The drug is safe, and one trial found it may extend survival and slow paralysis in functions like muscle control and speaking. A larger trial will be completed in 2024.The Food and Drug Administration on Thursday approved an experimental treatment for A.L.S., a severe neurological disorder that causes paralysis and death, despite questions about the therapy’s effectiveness.The treatment, conceived about a decade ago by two college students, was approved even though analyses by the F.D.A.’s reviewers concluded there was not yet sufficient evidence that the medication could help patients live longer or slow the rate at which they lose functions like muscle control, speaking or breathing without assistance. Nevertheless, the agency decided to greenlight the drug without waiting two years for results of a large clinical trial, citing data showing the treatment to be safe and the desperation of patients with a disease that often causes death within two-to-five years.The drug, which has the scientific name AMX0035, will be marketed as Relyvrio. In a summary memorandum about the drug the F.D.A. wrote that there was “residual uncertainty about the evidence of effectiveness,” but that “given the serious and life-threatening nature of A.L.S. and the substantial unmet need, this level of uncertainty is acceptable in this instance.” The memorandum also said that the benefits outweigh the risks because the treatment is “without any significant safety signals of concern.”The approval follows an impassioned campaign by patients and advocacy groups. In addition, doctors who treat A.L.S. patients had urged approval in a letter to the F.D.A. and testimony and in testimony before an F.D.A. advisory committee.“In your difficult job, there’s always going to be a chance of making a mistake; it comes down to which mistake you would rather make,” Dr. Richard Bedlack, director of the A.L.S. clinic at Duke University, testified this month. “To approve AMX0035 and find out in two years that it doesn’t work — I doubt many are going to be very angry because people with A.L.S. got to try something that was safe and appeared promising in 2022.”But, he added, “Can you imagine the mistake of saying no and then getting confirmatory evidence in two years that this really did work? And realizing all those patients were much more disabled or even dead when they didn’t need to be? I don’t know how you’ll be able to live with yourself if you make that mistake.”A.L.S., or amyotrophic lateral sclerosis, also called Lou Gehrig’s disease, is diagnosed in about 6,000 people worldwide each year. There are only two other approved A.L.S. medications in the United States: riluzole, approved in 1995, which can extend survival by several months, and edaravone, approved in 2017, which can slow progression by about 33 percent.The Fight Against A.L.S.The illness, also called Lou Gehrig’s disease, robs people of their ability to move, speak, eat and ultimately breathe.Relyvrio: The experimental treatment for A.L.S. conceived a decade ago by two college students received the Food and Drug Administration’s approval, despite questions about its effectiveness.A Runner’s Mission: After surpassing the average life expectancy for people with the disease, Andrea Peet decided to race a new kind of clock: 50 marathons in 50 states.Brain Implant: A man who is fully paralyzed by A.L.S. was able to communicate using only his thoughts.Rethinking Care: In 2017, Brian Wallach was diagnosed with A.L.S. Now, his startup aims to help other patients make the most of their time.Relyvrio was conceived by Justin Klee and Joshua Cohen when they were undergraduate students at Brown University. They proposed that combining taurursodiol, a supplement sometimes used to regulate liver enzymes, and sodium phenylbutyrate, a medication for a pediatric urea disorder, could protect neurons in the brain from damage in diseases like A.L.S. by preventing dysfunction of two structures in cells: mitochondria and the endoplasmic reticulum. They later founded a small Massachusetts company, Amylyx Pharmaceuticals.As patients learned about the compound, some began obtaining the ingredients on their own from Amazon and other sources. In June, Canada became the first country to approve the treatment, under a special condition requiring Amylyx to later provide better evidence that it worked. Advocacy groups predicted that some American patients would seek it from Canada, where it is marketed as Albrioza, if the F.D.A. didn’t approve it.The two-drug combination was conceived by Justin Klee, left, and Joshua Cohen when they were undergraduates at Brown University.Cody O’Loughlin for The New York TimesAmylyx did not immediately say what price it is considering for the treatment in the United States and has said the price is still being negotiated in Canada.  “Amylyx’s goal is that every person who is eligible for Relyvrio will have access as quickly and efficiently as possible as we know people with A.L.S. and their families have no time to wait,” the company said in a statement after the F.D.A. announcement.The medication, a bitter-tasting powder mixed with water and either drunk or ingested through a feeding tube, traveled an unusual and controversial path to approval. The F.D.A. typically requires two persuasive clinical trials, usually Phase 3 trials, which are larger and more extensive than Phase 2 studies. For serious diseases with few treatments, the agency can accept one trial plus additional confirmatory data.For Relyvrio, the data comes only from one Phase 2 trial in which 137 patients took either the drug or a placebo, plus an extension study that followed some patients after the trial ended when they were knowingly taking the drug.The Phase 2 trial involved patients considered to have fast-progressing disease. Two-thirds of participants received Relyvrio. Over 24 weeks, they experienced a 25 percent slower decline than participants receiving placebo — declining 2.32 points less on a 48-point A.L.S. scale that rates 12 physical abilities, including walking, speaking, swallowing, dressing, handwriting and breathing.The open-label extension study involved 90 of those patients, including 34 from the placebo group, who began taking the medication about seven months after those who received it from the beginning. Patients who received the treatment the longest had a median of about 6.5 months more time before being hospitalized, being put on a ventilator or dying, Amylyx reported. Researchers later published another analysis that suggested additional benefit.The F.D.A. initially recommended that Amylyx not apply for approval until the Phase 3 trial was completed in 2024.A.L.S. advocacy groups campaigned vehemently to persuade the F.D.A. to reconsider, especially after the agency’s controversial approval last year of the Alzheimer’s drug Aduhelm despite doubts about whether it worked. Soon after, F.D.A. officials began suggesting that Amylyx submit an application for approval using existing data.In March, a committee of independent advisers to the F.D.A. voted by a narrow margin that the treatment had not yet been shown to be effective, a conclusion also reached by the F.D.A.’s own reviewers. The agency then allowed Amylyx to submit more data and took the unusual step of scheduling a second independent advisory committee meeting on Sept. 7. In a report presented there, agency reviewers said they also considered the new data insufficient.But Dr. Billy Dunn, director of the F.D.A.’s office of neuroscience, told the advisory committee that “although some might reasonably argue that substantial evidence does not currently exist” to justify approval, the agency should exercise “the broadest flexibility” by also considering the seriousness of the disease and the dearth of available treatments.Dr. Dunn posed a question to the company: If the treatment received approval now and was shown to be ineffective in the Phase 3 trial, would Amylyx voluntarily withdraw it from the market, saving the agency a lengthy recall process? Mr. Klee said the company would.That commitment by Amylyx, plus emotional testimony from patients and doctors, persuaded more advisory committee members at the September meeting, where the vote favoring approval was seven to two.One of those who voted against approval, Dr. Kenneth Fischbeck, a distinguished investigator for the National Institutes of Health, said the company could provide the therapy to patients for free while waiting for better evidence, but “I don’t think it’s met the standard of evidence to allow them to sell the drug.” Mark Weston, a member of the advisory committee who has A.L.S., said he was disappointed that the new information the company presented wasn’t stronger. “I was hoping for something more,” he said. But Mr. Weston, who earlier in the meeting named A.L.S. patients who had died since the March hearing, said he voted for approval because “I can’t decouple my thoughts about that from my thoughts about the unmet need.”That need for treatments was the overwhelming message of patients who testified. “Instead of thinking you are protecting me I want you to recommend approval so that I have the chance to live,” said Brian Wallach, 41, who co-founded “I AM ALS,” an advocacy group, in testimony mostly read by a friend because A.L.S. has profoundly damaged his ability to speak.Gregory Canter, one of the clinical trial participants, said “the rate of my functional decline has slowed considerably” since beginning the drug over three years ago.“A.L.S. is the basement; this starts us up the stairs,” Mr. Canter said. “It won’t get us to the top alone, but each step up is important to point us in the right direction.”Calaneet Balas, president and chief executive of the A.L.S. Association, said in a statement that the F.D.A. decision was “a victory for the entire ALS community, which came together to advocate for early approval of AMX0035.” The association and other advocacy groups met with F.D.A. officials, submitted a petition with over 50,000 signatures and organized a campaign generating over 13,000 emails to the agency.The A.L.S. Association contributed $2.2 million to the development and study of AMX0035, using money raised through the 2014 Ice Bucket Challenge. Amylyx agreed to use sales of the drug to repay 150 percent of the association’s grant to fund more research.

As bans and restrictions proliferate across the country, abortion pill providers are pushing the envelope of regulations and laws to meet the surging demand for medication abortion in post-Roe America.Some are using physician discretion to prescribe pills to patients further along in pregnancy than the 10-week limit set by the Food and Drug Administration. Some are making pills available to women who are not pregnant but feel they could need them someday. Some are employing a don’t-ask-don’t-tell approach, providing telemedicine consultations and prescriptions without verifying that patients are in states that permit abortion.These changes are easing access to the pills for patients in states that have curtailed abortion, and also in states where it remains legal, but where clinics have longer wait times as patients flood in from restrictive states.Some of the practices, like not confirming that telemedicine patients are located in states that allow abortion, may run afoul of anti-abortion state laws or fall into uncharted legal territory, but they may also be challenging to police, reproductive health experts said.“We’re going to see these different approaches by organizations as they assess what the laws say and develop their rationale for how to provide care,” said Elizabeth Nash, state policy analyst for the Guttmacher Institute, a research group supporting abortion rights. “We just don’t have a road map about how to provide medication abortion post-Roe, so it’s all being created right now.”Abortion opponents criticized the efforts, calling them risky for women. Tessa Longbons, senior research associate at the Charlotte Lozier Institute, an anti-abortion organization, said the method had greater rates of complications and failure after 10 weeks that “could just put women at risk.” Providing pills to women who are not pregnant, she said, creates a risk that a woman “could take it anytime or someone that it wasn’t initially prescribed to could end up taking it.”Medication abortion, which was legalized in the United States in 2000, typically involves two drugs: mifepristone, which blocks a hormone necessary for pregnancy development, followed 24 to 48 hours later by misoprostol, which causes contractions that expel pregnancy tissue.Patients with some medical issues, like bleeding disorders, are not prescribed abortion pills. But for the many patients who are medically eligible, data indicates medication abortion is safe and effective, with a small percentage of patients requiring a procedure to fully remove pregnancy tissue and an even smaller proportion experiencing serious complications.The method is less expensive, less invasive and, especially with telemedicine, more private than surgical abortions. By 2020, it accounted for over half of U.S. abortions and has become even more sought-after since Roe was overturned.In the fast-changing abortion landscape, new online medication-abortion services are starting, and existing services are expanding, often adopting some of the new practices. Legally, this is a new frontier, both supporters and opponents of abortion rights say. It’s unclear, for example, whether any anti-abortion state laws — which typically target providers, not patients — address providing pills to someone who is not pregnant.Read More on Abortion Issues in AmericaSounding the Alarm: A United Nations panel reviewing racial issues in the United States called on the Biden administration to take action to protect abortion access for minorities and low-income people, adding that these groups could be disproportionately hit by the end of Roe.In Michigan: A state board in Michigan refused to place an abortion rights referendum on the November ballot because of a dispute over word spacing on the petition, which included more than 750,000 signatures.Fetal Personhood: A push to grant fetuses the same legal rights as people is gaining momentum, as anti-abortion activists move beyond bans and aim to get the procedure classified as murder.“As these new practices are developing, it is likely that they will be in a legal gray area,” said John Seago, president of Texas Right to Life, who said current Texas laws only involve providing abortion to women known to be pregnant.Other practices, like those that help patients in states with bans obtain pills, might violate those laws. But questions remain. Most providers using these practices prescribe and distribute pills only within states where abortion is legal. If those legally prescribed pills are then used by residents of anti-abortion states, it’s unclear if those states can prosecute out-of-state providers.Dr. Seago, who has a doctorate in bioethics, said new legislation would probably be introduced to combat the new practices. “Legislators are committed to figuring out how to enforce these laws,” he said.Beyond the F.D.A. ThresholdIn the fast-changing abortion landscape, new online medication-abortion services are starting and existing services are expanding.Jeff Roberson/Associated PressBefore Roe was overturned, few American services offered pills beyond the F.D.A.’s 10-week threshold, but many are now.Abortion Telemedicine, which was started after a draft of the Supreme Court ruling overturning Roe leaked in May, serves patients throughout the first trimester, which is 13 weeks into pregnancy.That company, and others serving patients at 11 or 12 weeks’ gestation, can legally use medical discretion to do so because studies suggest that abortion pills are safe and effective at that stage. The World Health Organization supports medication abortion through 12 weeks’ gestation.Dr. Daniel Grossman, a professor of obstetrics, gynecology and reproductive sciences at the University of California, San Francisco, said that late in the first trimester, medication abortion is safe and effective, but that there’s “a somewhat higher risk of some complications, including heavy bleeding,” and an additional dose of misoprostol is often needed to fully expel the tissue.Some services, including Abortion Telemedicine, automatically send a second round of the four misoprostol tablets for patients undergoing late first-trimester abortions.Reproductive health experts said patients should be advised that, while earlier in pregnancy, expelled tissue resembles a heavy period, after 10 weeks, it can appear more fetus-like. Dr. Abigail R.A. Aiken, an associate professor at the University of Texas, Austin, who leads a medication-abortion research group, said preparing patients for what the tissue might look likecould also help them guard against legal risk in states banning abortion — for example, in a situation where a patient is surprised by what they see and “then is disclosing that to someone who’s like ‘Well, I’m going to report you.’”Joann, 23, a single mother, was already 10 weeks pregnant when she decided to abort, so she contacted Abortion Telemedicine. She said she initially planned to carry her pregnancy to term, but then her 3-year-old son was diagnosed with autism and her employer, the U.S. military, decided to transfer her to another state. Joann, who asked to be identified only by her first name to protect her privacy, was in Colorado at the time, where abortion is legal, but her community was conservative.The service’s nurse practitioner told her that since she’d be taking the pills after 10 weeks’ gestation, she should expect more pain and bleeding, and counseled that the expelled tissue might resemble a fetus “so that I would be prepared for it,” Joann said.At 11 weeks and two days pregnant, she took the mifepristone, followed by the first four-tablet dose of misoprostol the next day and the second round six hours later. The cramping hurt, but “it was bearable,” she said.Providing Pills Before PregnancyHana is not pregnant but sought abortion pills just in case she did have an unwanted pregnancy in a state with a strict abortion ban, a practice called advance provision.Adriana Zehbrauskas for The New York TimesSome services are offering “advance provision” of abortion pills to patients who aren’t pregnant. Christie Pitney, a midwife who works with Aid Access, a medication-abortion service based in Europe that works with U.S. providers, likened it to travelers who “get medication for traveler’s diarrhea or for altitude sickness before you actually need to use it.” She said patients completed medical consultations before receiving prescriptions and were asked to contact the service again for an evaluation before taking the pills. Mifepristone’s shelf life is three to five years and misoprostol’s is 18 to 24 months, experts say.Several reproductive health experts have recently endorsed advance provision, suggesting that safety risks are low if patients are medically screened before being prescribed pills and before taking them, and if they have access to medical care if needed. Still, providers acknowledge that in an environment where many states restrict abortion, some patients may not follow appropriate guidelines. An article supporting advance provision, co-written by Dr. Grossman at U.C.S.F., noted that patients could take the pills in inappropriate circumstances or give them to others, and recommended further study.Ms. Pitney said Aid Access, which began offering advance provision in the United States in September 2021, was receiving about 40 requests per day earlier this year, but that the week after the Supreme Court decision, over 10,000 requests poured in.Hana, 31, said she sought advance provision because she lives in Arizona, a conservative state where a ban or other restrictions could take effect, and which is one of 19 states prohibiting telemedicine abortion. Hana, a claims researcher for a health insurer, who asked to be identified by only her first name to protect her privacy, said she tried to order from Aid Access the day the Supreme Court overturned Roe, but couldn’t get onto the website because traffic was so heavy. Two days later, she succeeded.After patients complete medical consultation forms, Aid Access ships from within the United States to patients in states with legal telemedicine abortion, while patients in restrictive states, like Hana’s, receive pills from a pharmacy in India. In 2019, the F.D.A. tried unsuccessfully to get Aid Access to halt overseas shipping. The organization’s founder, Dr. Rebecca Gomperts, a Dutch physician, said that U.S. Customs had occasionally stopped packages, but that most arrived without incident.“I have it hidden in my closet,” said Hana, who lives alone, but doesn’t want guests to stumble upon the medication. “I’m a little nervous,” she said, but added, “It’s really nice to have it just in case.”An Honor System for Patients’ LocationAbortion medication in the storage bin of a shuttered abortion clinic in Texas.Evelyn Hockstein/ReutersSome services check IP addresses to ensure that during telemedicine-abortion consultations patients are located in states where the practice is legal, but a growing number have decided not to verify location.“We don’t have any barriers such as an ID verification or GPS validation,” Dr. Jayaram Brindala, the founder of Abortion Telemedicine, said. His company arranges video consultations in 17 states where telemedicine abortion is legal and its appointment-booking form asks patients where they live, but “it’s on their honor,” he said.Some patients indicate they reside in states with abortion restrictions and are traveling to states where abortion is legal to receive pills at an address there, where the company can legally ship them, Dr. Brindala said.Ms. Pitney, the midwife with Aid Access, who also runs a service based in the United States called Forward Midwifery, said a shipping address was the only geographical information those services required. Only if patients mention they are doing the medical consultation from a state that prohibits telemedicine abortion or shipping of pills will she say she cannot legally treat them and refer them to Plan C, an online clearinghouse for information about medication abortion.Plan C recently added information about “virtual mailboxes” with commercial mail-forwarding companies: addresses in states where pills can legally be shipped and forwarded to patients in restrictive states. Forwarding companies are most likely unaware of the contents of the nondescript packages.“You’re using a legal mailing service and you’re using a legal telehealth service and you’re getting F.D.A.-approved products from a clinician with a prescription and the providers are fully compliant with the rules,” said Elisa Wells, co-founder and co-director of Plan C, which tested mail-forwarding companies by shipping bottles filled with garbanzo beans to approximate the rattling of pill bottles.Some medication-abortion providers are trying to broaden access while following F.D.A. guidelines and verifying patients’ locations.Abortion on Demand, which serves patients only up to nine weeks’ gestation, checks that patients do their video consultations from one of 22 states or Washington, D.C., all locations where telemedicine abortion is legal, and it will not ship to mail-forwarding companies or P.O. Boxes.“We very explicitly say that we’re sort of banning anything that may not just put us in legal jeopardy, but really put patients and put patients’ friends who are helping them in legal jeopardy,” Leah Coplon, director of clinical operations, said.Still, she said, her organization recently expanded to Pennsylvania. That state allows telemedicine abortion, but since Abortion on Demand has no office in Pennsylvania, state law prevents it from shipping pills there. Instead, Pennsylvania patients pick up pills at FedEx sites in New Jersey or Maryland.“We’ve had a lot of interest,” she said. “If the Pennsylvania model works well, there may be the potential to expand it in other areas.”

Demand for medication abortion is surging, setting the stage for new legal battles.In the hours after the Supreme Court released its decision overturning the legal right to abortion in the United States, nearly 100 requests for appointments flowed into Just the Pill, a nonprofit organization that arranges for patients to obtain abortion pills in several states.That was about four times the usual daily number of appointment requests for the organization, and many came from patients in Texas and other states that quickly halted abortions after the court ruling.Abortion pills, already used in more than half of recent abortions in the U.S., are becoming even more sought-after in the aftermath of Roe v. Wade being overturned, and they will likely be at the center of the legal battles that are expected to unfold as about half the states ban abortion and others take steps to increase access.The method, known as medication abortion, is authorized by the Food and Drug Administration for use in the first 10 weeks of pregnancy. It involves taking two different drugs, 24 to 48 hours apart, to stop the development of a pregnancy and then to cause contractions similar to a miscarriage to expel the fetus, a process that usually causes bleeding similar to a heavy period.Many patients choose medication abortion because it is less expensive, less invasive and affords more privacy than surgical abortions — the pills can be received by mail and taken at home, or anywhere, after an initial consultation with a doctor by video, phone, in person or even just by filling out an online form.The patient must participate in the consultation from a state that allows abortion, even if it simply involves being on the phone in a car just over the border. The IP address of the computer or phone they use allows the clinic to identify where they are.For states that ban all forms of abortion, medication abortion is likely to provide significant enforcement challenges. It is one thing to shut down a clinic; it is much harder to police activities like sending or receiving pills through the mail or traveling to a state where pills are legal to have a consultation and pick them up, legal experts say.“When people say we’re going back to the days before Roe, there’s no such thing as a time machine — we have a very different pharmaceutical landscape,” said Katie Watson, a constitutional scholar and medical ethicist at the Feinberg School of Medicine at Northwestern University.The abortion laws beginning to take effect in numerous conservative states ban all forms of abortion, including medication abortion. In addition, 19 states already had laws barring using telemedicine for abortion. Texas recently enacted a law prohibiting sending abortion pills through the mail. So groups and some state governments that support abortion rights are mobilizing to help patients obtain the pills in states where they are legal.Since October 2020, Just the Pill has provided more than 2,500 telemedicine consultations with doctors to supply abortion pills by mail to patients in Colorado, Minnesota, Montana and Wyoming. Within a few days, it plans to deploy in Colorado the first of what will become “a fleet of mobile clinics” to park along state borders, providing consultations for medication abortions and dispensing pills, said Dr. Julie Amaon, the organization’s medical director.Called “Abortion Delivered,” the clinic-on-wheels program, which will also provide surgical abortions for patients who prefer it or are too far along in pregnancy for a medication abortion, is designed to reach patients from nearby states like Texas, Oklahoma and South Dakota that quickly outlawed abortion after the court decision, as well as other states like Utah that are expected to ban or sharply restrict abortion.“By operating on state borders, we will reduce travel burdens for patients in states with bans or severe limits,” Dr. Amaon said. “And by moving beyond a traditional brick-and-mortar clinic, our mobile clinics can quickly adapt to the courts, state legislatures, and the markets, going wherever the need is.”Similar medication abortion providers are also planning for an influx. Hey Jane, an organization that has served nearly 10,000 patients in California, Colorado, Illinois, New Mexico, New York and Washington, plans to expand to more states. “We’ve ramped up our team to accommodate this significant increase in demand,” said its chief executive, Kiki Freedman.Anti-abortion groups are trying to counter the rise in interest in medication abortion by claiming it is unsafe, calling it “chemical abortion.” James Studnicki, vice president of data analytics at Charlotte Lozier Institute, an arm of Susan B. Anthony Pro-Life America, said on Friday that “the safety of the abortion pill is greatly exaggerated,” and called the rise in medication abortion “a serious public health threat.”Much remains unknown about how states that ban all or most abortions will try to enforce their laws in cases of medication abortion. But as the Biden administration scrambled to react to the court ruling, two cabinet members swiftly released statements vowing to protect the right to take medicines that had been approved by the federal government.“We stand unwavering in our commitment to ensure every American has access to health care and the ability to make decisions about health care — including the right to safe and legal abortion, such as medication abortion that has been approved by the F.D.A. for over 20 years,” Xavier Becerra, the secretary of Health and Human Services, said in his statement. In another statement, Merrick B. Garland, the attorney general, referred specifically to the first drug in the medication abortion regimen, mifepristone. In December, the F.D.A. made access to it significantly easier by permanently lifting the requirement that patients obtain mifepristone by visiting an authorized clinic or doctor in person.“We stand ready to work with other arms of the federal government that seek to use their lawful authorities to protect and preserve access to reproductive care,” Mr. Garland said. “In particular, the F.D.A. has approved the use of the medication mifepristone. States may not ban mifepristone based on disagreement with the F.D.A.’s expert judgment about its safety and efficacy.”But it is unclear what the Justice Department can do. Some legal scholars have argued that federal drug approval pre-empts state actions to restrict a drug’s use. Others say that has only applied to cases where a state claims that safety or efficacy is an issue.“Today, the Supreme Court said abortion, it can only be regulated in sort of a health and safety way when it’s permitted, but it can be completely banned,” Professor Watson said.As a result, she said, the ability of the federal government to assert that the F.D.A.’s approval takes precedence over state laws “is limited, given, traditionally, states get to regulate the practice of medicine.”Legal experts said there might be other ways for the Justice Department to become involved in fighting medication abortion restrictions, such as contesting laws that bar mailing pills, since the mail is under federal oversight.On Friday, the F.D.A. took a cautious stance, saying in a statement, “We have not had an opportunity to review the opinion, but we do note that F.D.A.’s independent and regulatory decisions are based on science and facts.”The agency added that “patients should have access to medications that are safe and effective for their F.D.A.-approved use.”Medication abortion became legal in the United States in 2000, when mifepristone was approved by the F.D.A. The agency imposed tight restrictions on the drug, many of which remain in place. But access to the method increased in 2016, when the F.D.A. expanded the time frame within which the drug could be taken — from seven weeks to 10 weeks into a pregnancy.Major medical groups cite years of data showing that medication abortion is safe. For example, a research program that the F.D.A. allowed to provide telemedicine consultations and send pills by mail reported that 95 percent of the 1,157 abortions that occurred through the program between May 2016 and September 2020 were completed without requiring any follow-up procedure. Patients made 70 visits to emergency rooms or urgent care centers, with 10 instances of serious complications, the study reported.As conservative states began passing more laws restricting access to surgical abortions, more patients opted for pills, especially because they could be taken in the privacy of one’s home.The Covid pandemic fueled that trend. The Guttmacher Institute, a research organization that supports abortion rights, reported that in 2020, medication abortion accounted for 54 percent of all abortions.As patients look for ways to obtain the pills, some are expected to turn to international websites like Aid Access, a European organization that the F.D.A. has tried — so far unsuccessfully — to stop from mailing pills to the United States, further complicating enforcement efforts.Mary Ziegler, a law professor at the University of California, Davis, who has written widely on abortion, said in an interview last month that there might be attempts by states that ban abortion to prosecute doctors and other health providers in other states who provide abortion services like consultations and pills to their residents, or to try to block organizations or funds that give financial help to patients to travel to other states.States where abortion remains legal are mobilizing to increase access stifle legal assaults from other states. Connecticut passed a bill that would prevent abortion providers from being extradited to other states, bar Connecticut authorities from cooperating with abortion investigations from a patient’s home state and allow Connecticut residents who are sued under another state’s abortion provision to countersue. Legislation in California would provide financial assistance to patients traveling from other states to obtain abortions and increase the number of abortion providers. Justice Brett Kavanaugh, in an opinion concurring with the Supreme Court decision, suggested that patients who traveled to other states to receive an abortion would be protected by the constitutional right to interstate travel.So far, most states that restrict abortion have long adhered to a principle of targeting providers and others who help patients, but not the patients themselves. Professor Ziegler said it was possible that could also change because, in circumstances where the abortion takes place outside state boundaries, “there may be absolutely no one else in that state to go after but the patient.”Katie Benner contributed reporting from Washington.

The drug, crenezumab, failed to prevent early symptoms or slow cognitive decline, the latest setback in the long quest to find effective therapies for the disease.A closely watched clinical trial of a potential Alzheimer’s drug failed to prevent or slow cognitive decline, another disappointment in the long and challenging effort to find solutions for the disease.The decade-long trial was the first time people who were genetically destined to develop the disease — but who did not yet have any symptoms — were given a drug intended to stop or delay decline. The participants were members of an extended family of 6,000 people in Colombia, about 1,200 of whom have a genetic mutation that virtually guarantees they will develop Alzheimer’s in their mid-40s to mid-50s. For many members of the family, who live in Medellín and remote mountain villages, the disease has quickly stolen their ability to work, communicate and carry out basic functions. Many die in their 60s.In the trial, 169 people with the mutation received either a placebo or the drug, crenezumab, produced by Genentech, part of the Roche Group. Another 83 people without the mutation received the placebo as a way to protect the identities of people likely to develop the disease, which is highly stigmatized in their communities.The trial investigators had hoped that intervening with a drug years before memory and thinking problems were expected to emerge might hold the disease at bay and provide important insights for addressing the more common type of Alzheimer’s that is not driven by a single genetic mutation.“We’re disappointed that crenezumab did not show a significant clinical benefit,” Dr. Eric Reiman, the executive director of Banner Alzheimer’s Institute, a research and treatment center in Phoenix, and a leader of the research team, said at a news conference about the results. “Our hearts go out to the families in Colombia and to everyone else who would benefit from an effective Alzheimer’s prevention therapy as soon as possible. At the same time, we take heart in the knowledge that this study launched and continues to help shape a new era in Alzheimer’s prevention research.”The results are also another setback for drugs that target a key protein in Alzheimer’s: amyloid, which forms sticky plaques in the brains of patients with the disease. Years of studies with various drugs that attack amyloid in different stages of the disease have fallen flat. In 2019, Roche halted two other trials of crenezumab, a monoclonal antibody, in people in the early stages of the more typical Alzheimer’s disease, saying the studies were unlikely to show benefit.Last year, in a highly controversial decision, the Food and Drug Administration granted its first approval of an anti-amyloid drug, Aduhelm. The F.D.A. acknowledged that it was unclear if Aduhelm could help patients, but greenlighted it under a program that allows authorization of drugs with uncertain benefit if they are for serious diseases with few treatments and if the drugs affect a biological mechanism that is reasonably likely to help patients. The F.D.A. said that biological mechanism was Aduhelm’s ability to attack amyloid, but many Alzheimer’s experts criticized the decision because of the poor track record of anti-amyloid therapies. The trial results on Thursday only added to the disappointing evidence.Laura Cuartas caring for her son Dario, one of four of her adult children with Alzheimer’s disease.Todd Heisler/The New York Times“Wish there were something more positive to say,” said Dr. Sam Gandy, the director of Mount Sinai’s Center for Cognitive Health, who was not involved in the Colombia research.“The pathogenic mutation in the Colombian family is known to be involved in amyloid metabolism,” Dr. Gandy said, adding, “The thinking was that these were the patients most likely to respond to anti-amyloid antibodies.”Dr. Pierre Tariot, the director of the Banner Alzheimer’s Institute and a leader of the Colombian research, said some of the data did suggest that patients receiving crenezumab fared better than those receiving the placebo, but the differences were not statistically significant.He also said there were no safety problems with the drug, an important finding because many anti-amyloid therapies, including Aduhelm, have caused brain bleeding or swelling in some patients.Additional data from the trial will be presented at a conference in August. Dr. Tariot and Dr. Reiman noted that Thursday’s results did not include more detailed information from brain imaging or blood analysis of the drug’s effects on proteins and other aspects of the biology of Alzheimer’s. They also did not reflect increases in the dose of crenezumab, which researchers began giving to patients as they learned more about the drug, Dr. Tariot said. He said some patients received up to two years of the highest dose during the five to eight years they were in the clinical trial.Dr. Francisco Lopera, a Colombian neurologist and another leader of the research, began working with the family members decades ago and helped determine that their affliction was a genetic form of Alzheimer’s. He said the trial had convinced him that “prevention is the best way of looking for the solution for Alzheimer’s disease, even if today we don’t have a good result.”“We know that we did a big step in the contribution to the investigation of Alzheimer’s disease,” he added. “And now we are prepared to start other steps in looking at the solution for this disease.”One participant’s wife, Maria Areiza of Medellín, said her husband, Hernando, whose surname is being withheld to protect his privacy, was among the first patients to enroll in the trial. Hernando, 45, who worked fixing telephone cables, began developing symptoms of cognitive decline about eight years ago. He has since progressed to Alzheimer’s dementia but can still hold a conversation. Because his deterioration has been relatively slow, his family had been hopeful that he was benefiting from the trial.“I had put all my hopes in this study,” his wife said.Jennie Erin Smith contributed reporting from Medellín, Colombia.

The F.D.A. is also reviewing the treatment, Albrioza, but the agency’s scientists have raised questions about its effectiveness.An experimental therapy for A.L.S., the paralyzing and fatal neurological disorder, has been approved in Canada, adding a new treatment option for a disease for which there are few effective therapies.The approval, which comes with the condition that the drug company later provide better evidence that the treatment works, is likely to be of major interest to patients with A.L.S. (amyotrophic lateral sclerosis) in the United States, where the same therapy — AMX0035, to be marketed as Albrioza — is being evaluated by the Food and Drug Administration, which has raised questions about the treatment’s effectiveness.An F.D.A. review earlier this year found Albrioza to be safe, but said there was not enough evidence that it was effective either in helping patients live longer or slowing the rate at which they lose functions like muscle control, speaking or breathing without assistance. A committee of independent advisers to the F.D.A. voted by a narrow margin in March that the therapy was not ready for approval.The F.D.A. had been scheduled to issue a final decision this month, but recently extended the deadline to Sept. 29, saying it needed more time to review additional analyses of data submitted by the company.In the meantime, Calaneet Balas, president and chief executive of the A.L.S. Association, one of several patient advocacy organizations pressing for F.D.A. approval, said, “We expect that Americans living with A.L.S. will try to access Albrioza in Canada, just as we have heard reports of people trying to buy the ingredients on Amazon.” A.L.S., also called Lou Gehrig’s disease, is diagnosed in about 6,000 people worldwide each year and often causes death within two to five years. There are only two approved A.L.S. medications in the United States: riluzole, which can extend survival by several months, and edaravone, which can slow progression by about 33 percent.Albrioza is a combination of two existing drugs in the form of a bitter-tasting powder that is mixed with water and drunk or ingested through a feeding tube twice daily. It is produced by a small Massachusetts company, Amylyx Pharmaceuticals, whose founders, Justin Klee and Joshua Cohen, conceived of the therapy when they were undergraduate students at Brown University less than a decade ago.The Fight Against A.L.S.The illness, also called Lou Gehrig’s disease, robs people of their ability to move, speak, eat and ultimately breathe.Lacking Evidence: A new treatment held promise for slowing A.L.S., but an F.D.A. panel found that there’s not enough data to show that it works.A Runner’s Mission: After surpassing the average life expectancy for people with the disease, Andrea Peet decided to race a new kind of clock: 50 marathons in 50 states.Brain Implant: A man who is fully paralyzed by A.L.S. was able to communicate using only his thoughts.Rethinking Care: In 2017, Brian Wallach was diagnosed with A.L.S. Now, his startup aims to help other patients make the most of their time.Their idea was that combining taurursodiol, a supplement sometimes used to regulate liver enzymes, and sodium phenylbutyrate, a medication for a pediatric urea disorder, could safeguard neurons by preventing dysfunction of two structures in cells: mitochondria and the endoplasmic reticulum.The data comparing Albrioza to a placebo has so far come from one Phase 2 trial (which is smaller than the Phase 3 studies regulatory agencies generally require); additional information came from an open-label extension study that followed some patients after the trial ended, when they were knowingly taking the medication. The F.D.A. typically requires two persuasive clinical trials of a drug for approval, but in cases of severe disease with few available treatments, it can consider evidence from one clinical trial plus additional supporting data.A box of packets being used in a clinical trial of the two-drug combination AMX0035, which will be marketed as Albrioza.Aileen Perilla for The New York TimesHealth Canada greenlighted Albrioza under a program called Notice of Compliance with Conditions, which allows for approval of drugs that appear promising for serious diseases, but have incomplete evidence that they work. The central condition the agency set is that Amylyx “verify the clinical benefit of this drug” with data from a Phase 3 clinical trial that is underway and expected to conclude in 2024, according to agency documents sent to the company on Friday. The company must also conduct additional pharmacological studies and provide periodic safety reports. “Patients should be advised of the nature of the authorization,” the documents said.The F.D.A. has a similar program called accelerated approval that allows conditional approval of drugs with incomplete evidence of effectiveness, but that program also requires that a drug show that it targets part of the underlying biological mechanism of a disease. Experts have said that if Albrioza does not win standard F.D.A. approval, it would be unlikely to meet accelerated approval criteria because too little is known about the underlying biology of A.L.S., and how and whether Albrioza might address it.Last month, 38 doctors who treat A.L.S. patients sent a letter to the F.D.A. urging approval. The A.L.S. Association said its campaign for approval had in recent weeks generated more than 6,000 emails asking the agency to greenlight the drug.A member of the F.D.A.’s independent advisory committee, Dr. G. Caleb Alexander, who voted in March that there was insufficient evidence the therapy works, said he continues to think the F.D.A. should wait for the Phase 3 trial’s results and that “it would be a mistake to approve it based on the single trial alone.”Dr. Alexander, an internist and epidemiologist at the Johns Hopkins Bloomberg School of Public Health, said there was desperate need for effective therapies for A.L.S., but that for Albrioza, “it’s unfortunate, but the magnitude of unmet need is not matched by the quality of evidence to date.”He added that “approval in Canada could only further increase the pressure that the F.D.A. faces to rule favorably and to approve this product.”It is generally illegal for Americans to import drugs that haven’t been approved in the U.S. for personal use. But the F.D.A. website lists some exceptions that might apply to Albrioza, including if the drug has no serious safety issues and if it is to treat “a serious condition for which effective treatment is not available in the United States.”Dr. Angela Genge, director of the A.L.S. Global Centre for Excellence at the Montreal Neurological Institute, who has received fees from Amylyx for serving on an advisory board, said American patients would be legally able to receive Albrioza in Canada if it were prescribed by a Canadian physician and obtained from a Canadian pharmacy, though they would not be eligible for insurance coverage under Canada’s public or private system.In an interview, Mr. Cohen and Mr. Klee declined to disclose the price that Amylyx is considering for Albrioza, saying it was still being negotiated. They said that the therapy would be available in about six weeks for people who were paying privately, but would take longer, possibly months, for people to receive coverage under Canada’s public system. Amylyx has already been providing Albrioza at no cost under compassionate-use arrangements to 250 patients in the United States, they said.Until last summer, the F.D.A. had recommended that Amylyx not apply for approval until the drug had completed its Phase 3 trial, but in July, officials began suggesting that Amylyx submit an application for approval using existing data. The timing followed vociferous pressure from A.L.S. advocacy groups in the wake of the approval of the new Alzheimer’s drug, Aduhelm, which was controversial because many experts said there was insufficient data that Aduhelm worked.In the Phase 2 trial, two-thirds of the 137 participants received Albrioza, and over 24 weeks, they experienced a 25 percent slower decline than the participants receiving placebo — declining 2.32 points less on a 48-point A.L.S. scale that rates 12 physical abilities, including walking, speaking, swallowing, dressing, handwriting and breathing.The open-label extension study involved 90 of those patients, including 34 from the placebo group, who began taking the medication about seven months after those who had received it from the beginning. Those who received the treatment the longest had a median of 4.8 months more time before being hospitalized, being put on a ventilator or dying, Amylyx reported. Researchers involved in the study published more data last month that suggested additional benefit.Amylyx financed the bulk of its research on Albrioza, but the A.L.S. Association contributed $2.2 million, using money raised through the 2014 Ice Bucket Challenge. Amylyx has agreed to use sales of the drug to repay 150 percent of the association’s grant to fund more research.The clinical trials involved patients who developed symptoms within 18 months before the trial and were affected in at least three body regions, generally signs of fast-progressing disease. Health Canada’s approval did not place restrictions on which A.L.S. patients could take Albrioza, but the Amylyx founders and Dr. Genge said it is possible that such limitations might be established by the Canadian coverage system or by pharmaceutical formularies in some of Canada’s provinces.

One in five adult Covid survivors under the age of 65 in the United States has experienced at least one health condition that could be considered long Covid, according to a large new study by the Centers for Disease Control and Prevention. Among patients 65 and older, the number is even higher: one in four.In an indication of how seriously the federal health agency views the problem of long Covid, the authors of the study — members of the C.D.C.’s Covid-19 Emergency Response Team — recommended “routine assess­ment for post-Covid conditions among persons who survive Covid-19.”Long Covid is the term used to describe an array of symptoms that can last for months or longer after the initial coronavirus infection. The researchers identified post-Covid health problems in many different organ systems, including the heart, lungs and kidneys. Other issues involved blood circulation, the musculoskeletal system and the endocrine system; gastrointestinal conditions, neurological problems and psychiatric symptoms were also identified in the study.In both age groups, Covid patients had twice the risk of uninfected people of developing respiratory symptoms and lung problems, including pulmonary embolism, the study found. Post-Covid patients aged 65 and older were at greater risk than the younger group of developing kidney failure, neurological conditions and most mental health conditions.“It is sobering to see the results of this study again confirming the breadth of organ dysfunction and the scale of the problem,” said Dr. Ziyad Al-Aly, chief of research and development at the V.A. St. Louis Health Care System and a clinical epidemiologist at Washington University in St. Louis, who was not involved in the research.The study evaluated electronic medical records for nearly two million people — comparing those who had been infected with the coronavirus with those who were not. The most common post-Covid conditions, regardless of age, were respiratory problems and musculoskeletal pain.The risk of post-Covid patients aged 65 and older developing the 26 health conditions the study evaluated was between 20 percent and 120 percent greater than people who didn’t get Covid. Those aged 18 to 64 had a 10 percent to 110 percent greater risk than uninfected people of developing 22 of the health conditions. But in that age group, Covid survivors were no more likely than uninfected people to develop most mental health conditions, substance use disorders or strokes and similar cerebrovascular conditions.Dr. Al-Aly said the study results “can potentially translate into millions of people with new diabetes, heart disease, kidney disease, neurologic problems. These are lifelong conditions — certainly manageable, but not curable conditions.”The study analyzed records of 353,164 people who were diagnosed with Covid-19 in the first 18 months of the pandemic, beginning in March 2020. It compared them with the records of 1.64 million people who had a medical visit in the same month in which the Covid patients were diagnosed but did not become infected with the coronavirus during the study period, which ended on Oct. 31, 2021.People in both groups who had a history of one of the 26 health conditions in the previous year were excluded from the study — an attempt by the researchers to consider medical issues that patients developed only after they had Covid.The study, which involved patients seen at health facilities that use a record system managed by Cerner Corp., a large medical data company, said the Covid patients included people admitted to hospitals, seen in emergency departments or diagnosed in an outpatient setting. The researchers did not indicate how many patients were in each group, one of several limitations of the study’s findings.Between 30 days and 365 days after their coronavirus diagnosis, 38 percent of the patients experienced one or more new health problems, compared to 16 percent of the non-Covid patients, the study said. The younger age group, 18-to-64, was somewhat less likely to have those problems — 35 percent developed long Covid issues, compared with 15 percent of uninfected people. In the 65-and-older group, 45 percent had new health conditions, compared with 19 percent of uninfected people.Based on those percentages, the study authors calculated that nearly 21 percent of the younger group and nearly 27 percent of the older group developed health problems that could be attributed to long Covid.The study did not look at the vaccination status of the patients and did not report characteristics like race, ethnicity, sex or geographic location. It also did not identify which coronavirus variants were linked to each case.The C.D.C. authors concluded that post-Covid conditions might “affect a patient’s ability to contribute to the work force and might have economic consequences for survivors and their dependents.” They added that “care requirements might place a strain on health services” in “communities that experience heavy Covid-19 case surges.”Dr. Al-Aly said he agreed that people who had Covid should be medically evaluated for potential new health problems.“Now that we are in possession of knowledge that Covid-19 can lead to serious long-term consequences,” he added, “we need to develop additional tools to reduce the risk of long Covid.”

Maybe, according to a growing number of studies, but there’s not yet a definitive answer.As the pandemic enters its third year, long Covid has emerged as an increasingly important concern. And many people are wondering whether getting a Covid shot can reduce their chances of developing long-term symptoms.What does the research show so far?The jury is still out, but a growing number of studies suggest that getting a Covid vaccine can reduce — though not eliminate — the risk of longer-term symptoms.The United Kingdom’s Health Security Agency conducted an analysis of eight studies that had been published on the topic before mid-January. It reported that six of the studies found that vaccinated people who became infected with the coronavirus were less likely than unvaccinated patients to develop symptoms of long Covid. The remaining two studies found that vaccination did not appear to conclusively reduce the chances of developing long Covid.How much protection could vaccines offer, according to the studies that found benefit?Some study results suggest substantial protection, while others find only a slight benefit.One large study of electronic records of patients in the U.S. Veterans Health Administration found that vaccinated Covid patients had only a 13 percent lower risk than unvaccinated patients of having symptoms six months later.Two studies in Britain found a bigger effect. One study of about 1.2 million people, based on patients’ reports via a phone app, found a 50 percent lower risk of lingering symptoms among vaccinated patients. Another, which has not been peer-reviewed and was based on surveying about 6,000 patients, found a 41 percent lower risk.A study of U.S. patients by Arcadia, a health care data firm, and the Covid Patient Recovery Alliance, a collaboration of leaders with health expertise in government and the private sector, found a larger benefit still. The study, which has not been peer-reviewed, analyzed records of about 240,000 patients infected with the coronavirus by May 2021 and found that those who had received even one dose of a Covid vaccine before their infection were one-seventh to one-tenth as likely to report two or more symptoms of long Covid 12 to 20 weeks later. That study also found that people who received their first vaccine dose after contracting the coronavirus were less likely to develop long Covid than those who remained unvaccinated, and the sooner they were vaccinated after infection, the lower the risk of long-term symptoms.A study in Israel, which has also not been peer-reviewed, found through surveys that people who received two doses of vaccine had between 54 percent and 82 percent lower risk than unvaccinated patients to report having seven of the 10 most common long-term symptoms. They were generally no more likely to report symptoms like headache, muscle pain and other issues than people in the general population who had never gotten Covid, the study said. (The authors said they could not confirm whether the patients were vaccinated before or after they had gotten Covid, but said that because of Israeli vaccination policy it was likely most people who received two doses of vaccine were infected with the coronavirus sometime after they had gotten their shots.)In the veterans’ study, also not yet published in a peer-reviewed publication, researchers compared about 48,000 patients who were unvaccinated when they got Covid with about 16,000 patients who were vaccinated. It found that vaccinated patients mostly benefited by being less likely to develop lung problems and blood-clotting difficulties, said one of the authors, Dr. Ziyad Al-Aly, chief of research and development at the V.A. St. Louis Health Care System and a clinical epidemiologist at Washington University in St. Louis. Other symptoms showed “very little risk reduction” from vaccines, he said.“The overall message is that vaccines reduce but do not eliminate the risk of long Covid,” said Dr. Al-Aly, adding that “reliance on vaccination as a sole mitigation strategy is wholly inadequate. It is like going to battle with a shield that only partially works.”A long-Covid patient was examined in a hospital in Tel Aviv in February.Amir Cohen/ReutersWhat about studies that don’t show any benefit?In an analysis of electronic medical records of patients in the United States, researchers in the United Kingdom compared about 10,000 people who had received Covid vaccines with a similar number of people who had not been vaccinated against the coronavirus but had received a flu vaccine — an effort to limit the number of people in the study who might be considered vaccine hesitant or who generally had less healthy behaviors.The study found that having a coronavirus vaccine before being infected did not reduce the risk of most symptoms of long Covid. There was some suggestion from the data that vaccinated people might be at lower risk of long-term symptoms like abnormal breathing and cognitive issues, the authors wrote, but those results were not statistically conclusive.The researchers said it was possible that because their data relied on electronic health records, the study might have captured only patients with the most severe symptoms, rather than a wider range of patients who did not seek medical attention for their symptoms.Why is the research conflicting?One reason is the differences in the studies themselves. Not all researchers have defined long Covid in the same way, measured the same symptoms or tracked patients for the same length of time. For example, some studies recorded symptoms that have lingered at least 28 days after infection, while others measured symptoms people were experiencing six months later. Studies relying on patient surveys may yield very different results than those based on electronic medical records. And some studies did not have very diverse populations. Patients in the veterans’ study, for example, were mostly older, white and male.Are the results different for different coronavirus variants?Much of the published data followed patients infected early in the pandemic. Some recently-published data included people infected by the highly contagious Delta variant, but it is too early for studies about vaccines and long Covid that include the Omicron variant. It’s also too early for studies evaluating the effect of boosters on long Covid.Is there anything scientists can conclude for sure?Yes. Vaccines are very effective at preventing people from getting seriously ill from infection by all the variants known so far. And many studies have found that Covid patients sick enough to be hospitalized were more likely to have lasting health issues. So, by keeping people out of the hospital, vaccines should reduce the chances of that type of long-term post-Covid case.Still, many people with long Covid had mild or even asymptomatic initial infections, and while some studies suggest vaccines have potential to ease their long-term symptoms, the evidence is not yet conclusive.Vaccines do offer some protection against getting infected to begin with — and avoiding infection, of course, is the surest way to prevent long Covid.Does the brand of vaccine make a difference in potential protection against long Covid?So far, studies have not found that different vaccines have different effects on long-term symptoms.What are the possible scientific reasons that vaccines might protect against long Covid?The cause of long Covid is still unclear, and different symptoms might have different underlying causes in different patients, scientists say. Some believe that the condition may be related to remnants of the virus or its genetic material lingering after the initial infection subsides. Another theory is that the continuing problems are related to inflammation or blood circulation problems spurred by an overactive immune response that is unable to shut down.Akiko Iwasaki, an immunologist at Yale, has said that vaccines may be able to provide lasting relief in people whose symptoms are caused by vestiges of the virus if the antibodies generated by the vaccines eliminate those remnants.But in people whose symptoms may be caused by a post-viral response resembling an autoimmune disease, she said, vaccines may help only temporarily, and problems like fatigue could re-emerge.One thing we know for certain is that vaccines are very effective at preventing people from getting seriously ill from infection by all the variants known so far. Kenny Holston for The New York TimesCan getting vaccinated help if you already have long Covid?When vaccines were first rolled out, some patients with long Covid were finding that symptoms like brain fog, joint pain, shortness of breath and fatigue improved after they had gotten vaccinated. Still, many people experienced no difference in their symptoms after vaccination, and a small percentage said they felt worse.A study by the Office for National Statistics in the United Kingdom found that in people ages 18 to 69 who reported their symptoms between February and September 2021, a first dose of a vaccine lowered the odds of reporting long Covid symptoms by 13 percent. A second dose further lowered the odds by 9 percent, the study found.The recent analysis by the U.K. Health Security Agency evaluated that study and seven others that examined whether vaccinating people with long Covid affected their symptoms. It found that in most of those studies, more people with long Covid reported improvement in their symptoms at some point after they were vaccinated. However, some people also reported worsening of symptoms, and in several studies the majority of people said their symptoms were unchanged.The agency noted that the definition of long Covid varied widely among the studies and that, because all the studies were observational, changes in symptoms could be due to factors other than vaccination.

Officials cited data showing the new Alzheimer’s drug has serious safety risks and may not help patients.For the past few months, Medicare officials have been inundated with impassioned pleas about how to handle coverage of the controversial new Alzheimer’s drug Aduhelm.Advocacy groups for patients have said the federal insurance program for people 65 and over must pay for a drug approved by the Food and Drug Administration. Many Alzheimer’s doctors and experts cautioned against broadly covering a treatment that scientific evidence shows has uncertain benefit and serious safety risks. Individual patients and families weighed in on both sides with emotional statements.On Thursday, Medicare officials announced their final decision. The program will cover Aduhelm only if people receive it as participants in a clinical trial, likely a small percentage of the estimated 1.5 million people in the United States who have mild Alzheimer’s-related cognitive decline, the condition Aduhelm was approved to treat.Chiquita Brooks-LaSure, the administrator of the Centers for Medicare and Medicaid Services said the decision was intended to protect patients while gathering data to indicate whether Aduhelm, an expensive monoclonal antibody given as a monthly infusion, could actually help them by slowing the pace of their cognitive decline.“It’s our obligation at C.M.S. to really make sure it’s reasonable and necessary,” Ms. Brooks-LaSure said in an interview Thursday. “The vast majority” of the approximately 10,000 comments the agency received on its website, she said, were in favor of “really limiting coverage of Aduhelm to a really controlled space where we could continue to evaluate its appropriateness for the Medicare population.”A major issue for Medicare had been how to deal with other similar drugs for Alzheimer’s, several of which are likely to be considered for F.D.A. approval soon. In a proposal in January, C.M.S. had said it would cover them in the same way as Aduhelm because it typically made coverage decisions for an entire class of drugs.People in favor of wider access to Aduhelm held a rally outside the Department of Health and Human Services last month.Kenny Holston for The New York TimesBut after both experts and advocacy groups raised concerns, Medicare officials said Thursday that they would not automatically apply the same restrictions to each new drug. If, unlike with Aduhelm, the F.D.A. finds that there is clear evidence that a drug can help patients, Medicare would cover it for all eligible patients and would only impose a requirement that the patients’ experience be tracked.Dr. Lee Fleisher, the chief medical officer at C.M.S., said the two-track way of dealing with the fast-developing field of Alzheimer’s therapies, a program called Coverage with Evidence Development, “is meant to be nimble and really respond to any new drugs in this class that are in the pipeline, and do demonstrate clinical benefit.”The decision is extremely unusual for Medicare, which almost always automatically pays for drugs that the F.D.A. has approved, at least for the medical conditions designated on labels. Understand the New Alzheimer’s Drug AduhelmF.D.A. Approval: In a contentious decision, the agency approved the first new Alzheimer’s treatment in nearly two decades. A Fierce Debate: Alzheimer’s patients are desperate for new options, but some scientists say there isn’t enough evidence Aduhelm works.Potential Safety Risks: Concerns over Aduhelm intensified after a 75-year-old woman in a clinical trial developed brain swelling and died.Medicare Limits: Officials said the federal program should cover the drug only for patients who are participating in approved clinical trials.The Campaign for Coverage: A powerful Alzheimer’s advocacy group is pushing relentlessly for broad Medicare access to the drug.But Aduhelm’s path has been very unusual, too. The F.D.A. itself acknowledged that it was unclear if the drug was beneficial when it approved Aduhelm last June. It greenlighted the drug under a program called “accelerated approval,” which allows authorization of drugs that have uncertain benefit if they are for serious diseases with few treatments and if the drug affects a biological mechanism in a way considered reasonably likely to help patients.The clinical trial evidence reviewed by the F.D.A. showed that patients in one trial appeared to experience slight slowing of cognitive decline, while patients in a nearly identical trial didn’t appear to benefit at all. About 40 percent of patients on the dosage later approved experienced brain swelling or brain bleeding, often mild, but sometimes serious. Both a council of senior F.D.A. officials and the agency’s independent advisory committee had said there wasn’t enough evidence for approval.The drug, manufactured by Biogen, takes the form of a monthly intravenous infusion.Pool photo by Jessica RinaldiQuestions about the approval, and whether the F.D.A. worked too closely with Biogen, Aduhelm’s manufacturer, have prompted investigations by congressional committees, the Health and Human Services department’s inspector general, the Federal Trade Commission and the Securities and Exchange Commission. Major medical centers, including the Cleveland Clinic, have declined to offer Aduhelm. As a result of concerns raised by Alzheimer’s experts and some groups, Medicare officials announced several other changes to their earlier proposal. Instead of requiring randomized controlled trials approved by C.M.S., Medicare will cover participants in any trial approved by the F.D.A. or the National Institutes of Health. It will allow those trials to be conducted in a broader array of locations, not just hospital settings, and to include people with other neurological conditions like Down syndrome, many of whom develop Alzheimer’s but had been excluded from the earlier proposed plan.The trials will still need to comply with a Medicare requirement to recruit a racially and ethnically diverse group of participants, contrasting with the previous trials of Aduhelm, in which most participants were white.In the trials, “the manufacturers will have to come to us with how are they going to include all patients that represent the Medicare population, and how are they going to ensure that all of these patients are getting appropriate medical treatment and monitoring of their treatment while they’re in each of these studies,” Tamara Syrek Jensen, the director of coverage and analysis for C.M.S.’s Center for Clinical Standards and Quality, said in an interview.The F.D.A. has also required Biogen to conduct another clinical trial to determine if the drug provided any evidence of benefit, but it said that in the years it will take for that trial to be completed, Aduhelm would be available to patients. Under Thursday’s decision, Medicare would cover the costs for participants in Biogen’s trial. Medicare’s coverage evaluation team makes decisions without considering the cost of a drug, but the Aduhelm decision could ease some concerns about how covering the drug might affect the pocketbooks of the country’s millions of Medicare beneficiaries.Counterprotesters at the Aduhelm rally in Washington in March.Kenny Holston for The New York TimesLast year, Medicare’s actuarial division, acting without knowing what the coverage decision would be, imposed one of the biggest-ever increases in Medicare Part B premiums for 2022, partly driven by the possibility of coverage for Aduhelm, which at the time was priced by its manufacturer at $56,000 a year.Since then, Biogen, facing weak sales of the drug after many hospitals and doctors would not prescribe it, lowered the price to $28,800 a year, still much higher than many analysts have said is warranted.Xavier Becerra, secretary of health and human services, had said that he would consider lowering premiums after the final coverage decision for Aduhelm was made, adding that “We’re going to make sure that seniors don’t pay more than they have to.”In the interview Thursday, Ms. Brooks-LaSure, the C.M.S. administrator, said “The secretary told us to look at it, and we are going to engage in the process of reviewing the Part B premium.”Advocacy groups, several of which receive some funding from Biogen and other pharmaceutical companies, had campaigned vigorously for broad Medicare coverage. These groups said patients should be able to decide with their doctors whether to try an F.D.A.-approved drug and claimed it was discriminatory to only reimburse participation in clinical trials that may not be easily accessible to many patients.“We just can’t let it stand as it is,” Harry Johns, the chief executive of the Alzheimer’s Association, told the organization’s staff, according to a recording of the meeting obtained by The New York Times.In an interview before the Medicare announcement, Mr. Johns indicated that the association wouldn’t be satisfied if Medicare’s restrictions applied only to Aduhelm, saying, “We absolutely believe there is sufficient evidence to provide coverage for the first approved treatment.”

The Alzheimer’s Association has pushed relentlessly to get broad access to Aduhelm, despite safety risks and uncertain evidence that it helps patients.The day after Medicare officials announced a preliminary decision to sharply limit coverage of the controversial new Alzheimer’s medication Aduhelm, citing its unclear benefit and serious safety risks, the nation’s most prominent Alzheimer’s advocacy organization convened its policy team.The agenda: fighting Medicare’s proposal.“This is our top priority,” Robert Egge, the association’s chief public policy officer, said at the Jan. 12 session, according to recordings obtained by The New York Times.The next day, the chief executive, Harry Johns, took the mission to the entire staff, saying, “You can count on us being relentless.”Medicare’s proposal to pay for Aduhelm only for participants in randomized clinical trials was welcomed by many Alzheimer’s experts and other doctors. They say it would protect patients and families whose desperation might lead them to try anything, while also rigorously evaluating whether Aduhelm actually works.But the Alzheimer’s Association and similar groups, along with Aduhelm’s manufacturer, Biogen, strongly object. They say that Aduhelm, a monoclonal antibody that is the first drug approved for Alzheimer’s in 18 years, should be covered for anyone with mild Alzheimer’s-related cognitive decline and that usage decisions should rest with patients and their doctors.The association’s campaign makes little mention of Aduhelm’s safety risks and uncertain benefit, arguing that restricting coverage of an F.D.A.-approved drug is “shocking discrimination against everyone with Alzheimer’s disease.”With Medicare’s final decision due by April 11, the group has organized tweets directed at President Biden, emails directed at Congress, and ads on social media and websites including Univision, Essence and Martha Stewart.It has orchestrated over 400 meetings in which people with Alzheimer’s, their family members or supporters have implored their members of Congress to press the Centers for Medicare and Medicaid Services (C.M.S.) to broadly cover the drug. In sessions prepping for those meetings, association officials suggested that people say versions of phrases like “I have a fatal disease and I want more time with my family” and to discuss “how access to F.D.A.-approved treatments will impact me and my family.”Leaders also encouraged people to submit comments to C.M.S. in ways that weren’t scripted. “It is important that individuals not use our talking points,” Beth McMullen, senior director of grassroots advocacy, instructed at one meeting. Employees filing comments “cannot submit as a staff member of the Alzheimer’s Association,” she said.The push reflects growing activism by advocacy groups involved in various diseases, which sometimes campaign for therapies despite medical experts’ concerns about insufficient data. Some are partially funded by pharmaceutical companies. In fiscal year 2021, the Alzheimer’s Association received $1.6 million from manufacturers developing monoclonal antibodies, including $487,500 from Biogen.Biogen, Aduhelm’s manufacturer, gave the Alzheimer’s Association $487,500 in fiscal year 2021.C.J. Gunther/EPA, via ShutterstockOther Alzheimer’s advocacy groups are also campaigning, including UsAgainstAlzheimer’s. It has run television ads featuring patients and has written in strident language to C.M.S. that “the government would be consigning millions of Americans to inevitable decline and death, with no possibility of appeal.” The Alliance for Aging Research led a rally of about 100 people outside the Department of Health and Human Services, which houses C.M.S. (Afterward, about half the alliance’s scientific advisory board quit in opposition to the group’s position.)Understand the New Alzheimer’s Drug AduhelmF.D.A. Approval: The agency approved the first new Alzheimer’s treatment in 18 years despite fierce debate over whether it works.Potential Safety Risks: Concerns over Aduhelm intensified after a 75-year-old woman in a clinical trial developed brain swelling and died.Medicare Limits: Officials said the federal program should cover the drug only for patients who are participating in approved clinical trials.Understand Alzheimer’s: Get answers to common questions about the disease, which affects about 30 million people globally.Medicare’s coverage proposal came after Aduhelm, an expensive monthly infusion that also has the scientific name aducanumab, was approved in June by the Food and Drug Administration despite contradictory scientific results. Participants in one clinical trial appeared to experience slight slowing of cognitive decline, while patients in a nearly identical trial didn’t appear to benefit at all. About 40 percent of patients on the dosage later approved experienced brain swelling or brain bleeding, often mild, but sometimes serious. Both a council of senior F.D.A. officials and the agency’s independent advisory committee had said there wasn’t enough evidence for approval. Questions about the approval, and whether the F.D.A. worked too closely with Biogen, have prompted investigations by congressional committees, the Health and Human Services department’s inspector general, the Federal Trade Commission and the Securities and Exchange Commission. Major medical centers, including the Cleveland Clinic, have declined to offer Aduhelm.After C.M.S. announced its proposal, Dr. Lee Fleisher, the agency’s chief medical officer, said, “Our foremost goal is to protect beneficiaries from potential harm from an intervention without known benefits in the Medicare population.”The proposal has attracted extraordinary interest, with C.M.S. receiving 9,957 comments, some organized by advocacy groups on both sides of the debate.The Alzheimer’s Association, with revenue of about $400 million, is highly influential in the Alzheimer’s field: It runs an international conference, provides research grants and publishes a journal.In recordings of the staff meeting and two prep sessions, association officials didn’t mention Aduhelm’s safety risks.In an interview, Mr. Johns, the chief executive, said, “We take the safety concerns seriously,” adding, “The risks are real, but they’re not worse than treatments that are covered by Medicare today.” He proposed that patients enroll in a registry where negative effects could be reported. But many experts say that wouldn’t sufficiently evaluate the drug. At the staff meeting, Maria Carrillo, the association’s chief science officer, addressed issues of whether Aduhelm works by saying, “One of the questions that you may be asked, or maybe you’re asking yourself is: Is the scientific evidence sufficient to conclude” that Aduhelm and similar monoclonal antibodies “improve health outcomes for Medicare beneficiaries?” The answer, she said, was “yes.”While some patients enthusiastically back the association’s efforts, its stance troubles some longtime supporters of the organization, including Dr. Daniel Gibbs, a retired neurologist in Portland, Ore., with early-stage Alzheimer’s. Dr. Daniel Gibbs, a retired neurologist with early-stage Alzheimer’s, agrees with Medicare’s proposed restrictions on Aduhelm.Amanda Lucier for The New York TimesHe participated in an aducanumab trial, experiencing brain swelling and bleeding that required treatment in an intensive care unit. “I want aducanumab to be successful, and I really think that it might be useful,” he said. However, he added, he considers Medicare’s proposed restrictions appropriate.He called the Alzheimer’s Association campaign “shocking and irresponsible” and said he’ll stop donating to the organization, except for his local chapter. “The vast majority of experts in Alzheimer’s are not on the side of the Alzheimer’s Association,” he said.Several members of the association’s scientific and medical advisory group said they had concluded the drug shouldn’t yet be approved. Still, the association pushed for approval.The F.D.A. itself acknowledged the unclear benefit when it greenlighted Aduhelm under “accelerated approval,” which allows authorization of unproven drugs for serious diseases with few treatments, if the drug affects a biological mechanism in a way considered “reasonably likely to predict clinical benefit.”The agency’s justification was that Aduhelm targets a protein, amyloid, that forms plaques in the brains of Alzheimer’s patients. But many Alzheimer’s experts say that years of data have not shown that reducing amyloid can slow cognitive decline.In the association staff meeting, Dr. Carrillo described experts who criticized Aduhelm’s approval as “naysayers” who “are mainly anti-amyloid researchers, in conflict, in my view.”Several scientists critical of Aduhelm’s approval support the idea that targeting amyloid might help patients; they simply found Aduhelm’s results unconvincing.Mr. Egge, the chief public policy officer, did say at one meeting: “It’s not certain that this first treatment will work, certainly, for everybody, but it could.” He said that it’s common to have “first treatments making marginal differences” but that later drugs often work better. Mr. Johns said the association and its lobbying affiliate, the Alzheimer’s Impact Movement, have spent hundreds of thousands of dollars on the campaign.At the staff meeting, Mr. Johns addressed another issue: “Of course, you’ll hear people claim that we’re doing this because we receive some funding from the pharmaceutical industry or these companies.”He said corporate contributions “are very small, less than 1 percent of our total revenue,” adding, “We would never be affected by any kind of fund-raising.”One attendee asked: “Since the amount of funding we receive from biotech companies is so low, why not refuse to take it and take away the appearance of possible conflicts of interest?”Mr. Johns replied, “We’ve contemplated this many times,” but concluded it would have unintended consequences, like preventing the organization from accepting corporate matching contributions for participants in association fund-raising walks who happened to work at pharmaceutical companies.In the interview, Mr. Johns echoed these explanations. Asked if association officials had communicated with Biogen since Medicare’s proposal, he said, “I think probably some of our folks have had a conversation or more along the way” and that Biogen’s staff “occasionally pass along a piece of information, but I can tell you that we just don’t coordinate with them.”In the recordings, association officials told advocates that their annual meetings with local members of Congress would involve different “asks” than usual: They should request that lawmakers give a floor speech, write to C.M.S., or post on social media to urge Medicare to broadly cover Aduhelm and any other F.D.A.-approved Alzheimer’s treatment.One official, Jennifer Pollack, suggested that advocates should give congressional staff a “leave-behind” that included a sample letter, and social media posts drafted by the association and “sample talking points for a floor speech.”Kate Johnson, another official, told advocates: “If you do not know the answer to a question, that is totally OK.” In that case, “please always revert to our safest phrase,” which, she said, was: “‘I’m not sure, but I can pass that question on to a staffer at the Alzheimer’s Association who can provide some more information.’”She also urged advocates to take photos or screenshots of the congressional meetings to post on social media, tagging the lawmaker to show “we’re not going to give up on the issues that we’re passionate about.”Christopher Masak, director of advocacy, role-played being an Alzheimer’s patient’s son meeting with a lawmaker.“My mom is living with a fatal disease, and the bottom line is, I want more time,” his character said, adding, “Thinking of people like my mom who can’t access this medication, it just, it breaks my heart.”Medicare officials said that limiting coverage to clinical trial participants is especially important because Aduhelm is intended for people with early mild disease, who could potentially be sacrificing years of remaining function and quality of life if a drug’s harms outweigh any benefit.Mr. Johns said he understood the view that “risk could be better tolerated later in the disease” but said that the milder early period is the time many people most want to try to extend. C.M.S. officials also said Aduhelm data isn’t sufficiently representative because most trial participants were white, while Blacks and Latinos have greater Alzheimer’s risk. So, Medicare’s proposal requires that new clinical trials reflect racial and ethnic diversity.Association officials said Medicare’s proposal could have the opposite effect because people in underserved communities might have less access to clinical trial sites.A few issues mentioned by advocacy groups have also been raised by those who don’t oppose limiting coverage for Aduhelm. One is that, as currently worded, Medicare’s decision would apply to all monoclonal antibodies for Alzheimer’s, several of which are in late-stage trials and may soon be considered for F.D.A. approval.“It is essentially denying access to all current and future F.D.A.-approved treatments targeting amyloid in those living with Alzheimer’s disease,” Ms. Pollack said in one recording.The American Academy of Neurology wrote to C.M.S. calling the proposal to cover only patients in clinical trials “prudently designed to investigate the safety and effectiveness,” but asked that it be limited only to Aduhelm. In a statement, a C.M.S. spokeswoman, Beth Lynk, said coverage criteria could be re-evaluated for subsequent monoclonal antibodies. “C.M.S. intends to be nimble and respond to new evidence as it is made available — especially related to the clinical benefit of future therapeutics,” she said.Mr. Johns, who said that he recently attended three meetings with C.M.S. officials, including one with the agency’s administrator, indicated in the interview that the association wouldn’t be satisfied if Medicare’s restrictions applied only to Aduhelm, saying, “We absolutely believe there is sufficient evidence to provide coverage for the first approved treatment.”Advocacy groups’ efforts have gotten some lawmakers’ attention. Recently, a bipartisan group of 40 House members wrote asking C.M.S. to provide broad coverage.Earlier, 78 House Republicans sent a letter criticizing C.M.S.’s proposal, quoting the Alzheimer’s Association and UsAgainstAlzheimer’s. Both advocacy groups were described on Senator Susan Collins’s website as supporting a letter she and another Republican, Senator Shelley Moore Capito, wrote.Some lawmakers, including Democrats, have tweeted a version of the association’s language.But other lawmakers praised Medicare’s proposal, including Representatives Carolyn B. Maloney and Frank Pallone, Democrats who chair committees investigating the F.D.A.’s approval of Aduhelm.Mr. Johns said he didn’t know of any lawmakers who had switched from supporting to opposing Medicare’s proposal. But he said the association’s congressional meetings “have had positive outcomes, even for people who might still have a different opinion. There is at least a different level of understanding.”He declined to provide the number of tweets, letters or other measures of the campaign’s reach, saying: “There are those who are working against access. We are not inclined to share details of our tactical approaches with them. Our activities will be measured by outcomes that grant those with Alzheimer’s disease access to current and future F.D.A.-approved treatments without unnecessary barriers.”

Covid-19 may cause greater loss of gray matter and tissue damage in the brain than naturally occurs in people who have not been infected with the virus, a large new study finds.The study, published Monday in the journal Nature, is believed to be the first involving people who underwent brain scans both before they contracted Covid and months after. Neurological experts who were not involved in the research said it was valuable and unique, but they cautioned that the implications of the changes were unclear and did not necessarily suggest that people might have lasting damage or that the changes might profoundly affect thinking, memory or other functions.The study, involving people aged 51 to 81, found shrinkage and tissue damage primarily in brain areas related to sense of smell; some of those areas are also involved in other brain functions, the researchers said.“To me, this is pretty convincing evidence that something changes in brains of this overall group of people with Covid,” said Dr. Serena Spudich, chief of neurological infections and global neurology at the Yale School of Medicine, who was not involved in the study.But, she cautioned: “To make a conclusion that this has some long-term clinical implications for the patients I think is a stretch. We don’t want to scare the public and have them think, ‘Oh, this is proof that everyone’s going to have brain damage and not be able to function.’”