The tests screen for cancers that often go undetected, but they are expensive and some experts worry they could lead to unnecessary treatments without saving patients’ lives.Jim Ford considers himself a lucky man: An experimental blood test found his pancreatic cancer when it was at an early stage. It is the among the deadliest of all common cancers and is too often found too late.After scans, a biopsy and surgery, then chemotherapy and radiation, Mr. Ford, 77, who lives in Sacramento, has no detectable cancer.“As my doctor said, I hit the lottery,” he said.Tests like the one that diagnosed him have won praise from President Biden, who made them a priority of his Cancer Moonshot program. A bill in Congress with 254 cosponsors would authorize Medicaid and Medicare to pay for the tests as soon as the Food and Drug Administration approved them.But companies are not waiting for a nod from regulators. One, GRAIL, is selling its annual test, with a list price of $949, in advance of approval, and another company, Exact Sciences, expects to follow suit, using a provision known as laboratory developed tests.The tests, which look for minuscule shards of cancer DNA or proteins, are a new frontier in screening. Companies developing them say they can find dozens of cancers. While standard screening tests are commonly used to detect cancer of the breast, colon, cervix and prostate, but 73 percent of people who die of cancer had cancers that are not detected by standard tests.Supporters say the tests can slash cancer death rates by finding tumors when they are still small and curable. But a definitive study to determine whether the tests prevent cancer deaths would have to involve more than a million healthy adults randomly assigned to have an annual blood test for cancer or not. Results would take a decade or longer.“We’re at a point now where the blood tests are in their early days,” said Dr. Tomasz Beer, a cancer researcher at Oregon Health & Science University, who is directing a GRAIL-sponsored study of the test that found Mr. Ford’s cancer. “Some people in an informed manner can choose to be early adapters.”The companies would like to get the tests approved with studies less rigorous than the F.D.A. typically requires, and they stand to make huge profits if that happens.“GRAIL proposes to test every Medicare beneficiary every year, making it the screening test that could bankrupt Medicare,” said Dr. H. Gilbert Welch, a senior investigator in the Center for Surgery and Public Health at Brigham and Women’s Hospital.With 44 million Medicare beneficiaries and an annual test costing about $1,000 a year plus expensive scans and biopsies for those whose tests are positive, the price tag could be substantial.He and other critics warn that the risks of unleashing the tests are substantial. Paradoxical as it may sound, finding cancers earlier could mean just as many deaths, with the same timing as without early diagnosis. That is because — at least with current treatments — cancers destined to kill are not necessarily cured if found early.And there are other risks. For example, some will have a positive test, but doctors will be unable to locate the cancer. Others will be treated aggressively with surgery or chemotherapy for cancers that, if left alone, would not have grown and spread and may even have gone away.Dr. Beer acknowledges that a cancer blood test “doesn’t come without risks or costs, and it is not going to detect every cancer.”But, he said, “I think there’s promise for a real impact.”Others experts are worried.Dr. Barnett Kramer, a member of the Lisa Schwartz Foundation for Truth in Medicine and former director of the Division of Cancer Prevention at the National Cancer Institute, fears that the tests will come into widespread use without ever showing they are beneficial. Once that happens, he said, “it is difficult to unring the bell.”“I hope we are not halfway through a nightmare,” Dr. Kramer said.The Damocles SyndromeWhen Susan Iorio Bell, 73, a nurse who lives in Forty Fort, Pa., saw an ad on Facebook recruiting women her age for a study of a cancer blood test, she immediately signed up. It fit with her advocacy for preventive medicine and her belief in clinical trials.The study was of a test, now owned by Exact Sciences, that involved women who are patients with Geisinger, a large health care network. The test looks for proteins and DNA shed by tumors.Ms. Bell’s result was troubling: Alpha-fetoprotein turned up in her blood, which can signal liver or ovarian cancer.She was worried — her father had had colon cancer and her mother had breast cancer.Ms. Bell had seen what happened when patients get a dire prognosis. “All of a sudden, your life can be changed overnight,” she said.But a PET scan and abdominal M.R.I. failed to find a tumor. Is the test result a false positive, or does she have a tumor too small to be seen? For now, it is impossible to know. All Ms. Bell can do is have regular cancer screenings and monitoring of her liver function.“I just go day by day,” she said. “I am a faith-based person and believe God has a plan for me. Good or bad, it’s his will.”Some cancer experts say Ms. Bell’s experience exemplifies a concern with the blood tests. The situation may involve only a small percentage of people because most who are tested will be told their test did not find cancer. Among those whose tests detect cancer, scans or biopsies can often locate it.But Dr. Susan Domchek, a breast cancer researcher at the University of Pennsylvania, warned that when large numbers of people get tested, false positives become “a real problem,” adding, “we need to know what to do with those results and what they mean.”Dr. Daniel Hayes, a breast cancer researcher at the University of Michigan, refers to the situation as a Damocles syndrome: “You’ve got this thing hanging over your head, but you don’t know what to do about it.”How Good Are the Tests?So far, the Geisinger study is the only published one asking whether the blood tests find early, undetected cancers.In addition to Ms. Bell, the study involved 10,000 women aged 65 to 75 who had the blood test and were encouraged to also have routine cancer screening.The blood test found 26 patients who had cancers: two lymphomas, one thyroid cancer, one breast cancer, nine lung cancers, one kidney cancer, two colorectal cancers, one cancer of the appendix, two cancers of the uterus, six ovarian cancers and one unknown case in which there were cancer cells in the woman’s body but it was not clear where the cancer started.Seventeen of these women, or 65 percent, had early stage disease.Conventional screening found an additional 24 cancers that the blood tests missed.Dr. Bert Vogelstein, a cancer researcher at Johns Hopkins Medicine who helped to develop the test, said the study was not designed to show risks and benefits. That will require much larger and more detailed studies.GRAIL’s study, led by Dr. Beer, involved 6,629 participants. Its interim data, presented at a professional meeting last year, showed the test found cancer signals in 92 participants. After these subjects had additional tests like CT and PET scans and biopsies, the researchers concluded that 29 had cancer. Among those cancers, 23 were new cancers and nine were early stage. The rest were recurrences in people who had already had cancer.New Developments in Cancer ResearchCard 1 of 7Progress in the field.

For some patients with metastatic tumors not significantly affected by other forms of chemotherapy, the treatment halted their cancer’s growth.The patients had metastatic breast cancer that had been progressing despite rounds of harsh chemotherapy. But a treatment with a drug that targeted cancer cells with laserlike precision was stunningly successful, slowing tumor growth and extending life to an extent rarely seen with advanced cancers.The new study, presented at the annual meeting of the American Society of Clinical Oncology and published on Sunday in the New England Journal of Medicine, would change how medicine was practiced, cancer specialists said.“This is a new standard of care,” said Dr. Eric Winer, a breast cancer specialist, director of the Yale Cancer Center and head of the A.S.C.O. Dr. Winer was not involved with the study. He added that “it affects a huge number of patients.”The trial focused on a particular mutant protein, HER2, which is a common villain in breast and other cancers. Drugs that block HER2 have been stunningly effective in treating breast cancers that are almost entirely populated with the protein, turning HER2-positive breast cancers from those with some of the worst prognoses into ones where patients fare very well.But HER2-positive cases constitute only about 15 percent to 20 percent of breast cancer patients, said Dr. Halle Moore, director of breast medical oncology at the Cleveland Clinic. Patients with only a few HER2 cells — a condition known as HER2-low — were not helped by those drugs. Only a small proportion of their cancer cells had HER2, while other mutations primarily drove the cancer’s growth. And that posed a problem because the cancer cells evaded chemotherapy treatments.The clinical trial, sponsored by the pharmaceutical companies Daiichi Sankyo and AstraZeneca and led by Dr. Shanu Modi of Memorial Sloan Kettering Cancer Center, involved 557 patients with metastatic breast cancer who were HER2-low. Two-thirds took the experimental drug, trastuzumab deruxtecan, sold as Enhertu; the rest underwent standard chemotherapy.In patients who took trastuzumab deruxtecan, tumors stopped growing for about 10 months, as compared with 5 months for those who with standard chemotherapy. The patients with the experimental drug survived for 23.9 months, as compared with 16.8 months for those who received standard chemotherapy.“It is unheard-of for chemotherapy trials in metastatic breast cancer to improve survival in patients by six months,” said Dr. Moore, who enrolled some patients in the study. Usually, she says, success in a clinical trial is an extra few weeks of life or no survival benefit at all but an improved quality of life.The results were so impressive that the researchers received a standing ovation when they presented their data at the oncology conference in Chicago on Sunday.Trastuzumab deruxtecan was already approved for patients with HER2-positive breast cancer, but few expected it to work because other drugs for such cancers had failed in HER2-low patients.The drug consists of an antibody that seeks out the HER2 protein on the surface of cells. The antibody is attached to a chemotherapy drug. When trastuzumab deruxtecan finds a cell with HER2 on its surface, it enters the cell, and the chemotherapy drug separates from the antibody and kills the cell.But “what is unique and distinct” about trastuzumab deruxtecan, Dr. Modi adds, is that the chemotherapy drug seeps through the cell’s membrane. From there, it can move into nearby cancer cells and kill them as well.Like all chemotherapy, trastuzumab deruxtecan has side effects, including nausea, vomiting, blood disorders and, notably, lung injuries that led to the deaths of three patients in the trials.But, Dr. Winer said, “if I were a patient with metastatic breast cancer, and if I were to get a drug with chemotherapy’s side effects, I’d prefer this drug.”Doctors have said they are planning to try the treatment in their breast cancer patients who have metastatic HER2-low cancers.New Developments in Cancer ResearchCard 1 of 6Progress in the field.

The study was small, and experts say it needs to be replicated. But for 18 people with rectal cancer, the outcome led to “happy tears.”It was a small trial, just 18 rectal cancer patients, every one of whom took the same drug.But the results were astonishing. The cancer vanished in every single patient, undetectable by physical exam, endoscopy, PET scans or M.R.I. scans.Dr. Luis A. Diaz Jr. of Memorial Sloan Kettering Cancer Center, an author of a paper published Sunday in the New England Journal of Medicine describing the results, which were sponsored by the drug company GlaxoSmithKline, said he knew of no other study in which a treatment completely obliterated a cancer in every patient.“I believe this is the first time this has happened in the history of cancer,” Dr. Diaz said.Dr. Alan P. Venook, a colorectal cancer specialist at the University of California, San Francisco, who was not involved with the study, said he also thought this was a first.A complete remission in every single patient is “unheard-of,” he said.These rectal cancer patients had faced grueling treatments — chemotherapy, radiation and, most likely, life-altering surgery that could result in bowel, urinary and sexual dysfunction. Some would need colostomy bags.They entered the study thinking that, when it was over, they would have to undergo those procedures because no one really expected their tumors to disappear.But they got a surprise: No further treatment was necessary.“There were a lot of happy tears,” said Dr. Andrea Cercek, an oncologist at Memorial Sloan Kettering Cancer Center and a co-author of the paper, which was presented Sunday at the annual meeting of the American Society of Clinical Oncology.Another surprise, Dr. Venook added, was that none of the patients had clinically significant complications.On average, one in five patients have some sort of adverse reaction to drugs like the one the patients took, dostarlimab, known as checkpoint inhibitors. The medication was given every three weeks for six months and cost about $11,000 per dose. It unmasks cancer cells, allowing the immune system to identify and destroy them.While most adverse reactions are easily managed, as many as 3 percent to 5 percent of patients who take checkpoint inhibitors have more severe complications that, in some cases, result in muscle weakness and difficulty swallowing and chewing.The absence of significant side effects, Dr. Venook said, means “either they did not treat enough patients or, somehow, these cancers are just plain different.”In an editorial accompanying the paper, Dr. Hanna K. Sanoff of the University of North Carolina’s Lineberger Comprehensive Cancer Center, who was not involved in the study, called it “small but compelling.” She added, though, that it is not clear if the patients are cured.“Very little is known about the duration of time needed to find out whether a clinical complete response to dostarlimab equates to cure,” Dr. Sanoff said in the editorial.Dr. Kimmie Ng, a colorectal cancer expert at Harvard Medical School, said that while the results were “remarkable” and “unprecedented,” they would need to be replicated.The inspiration for the rectal cancer study came from a clinical trial Dr. Diaz led in 2017 that Merck, the drugmaker, funded. It involved 86 people with metastatic cancer that originated in various parts of their bodies. But the cancers all shared a gene mutation that prevented cells from repairing damage to DNA. These mutations occur in 4 percent of all cancer patients.Patients in that trial took a Merck checkpoint inhibitor, pembrolizumab, for up to two years. Tumors shrank or stabilized in about one-third to one-half of the patients, and they lived longer. Tumors vanished in 10 percent of the trial’s participants.That led Dr. Cercek and Dr. Diaz to ask: What would happen if the drug were used much earlier in the course of disease, before the cancer had a chance to spread?They settled on a study of patients with locally advanced rectal cancer — tumors that had spread in the rectum and sometimes to the lymph nodes but not to other organs. Dr. Cercek had noticed that chemotherapy was not helping a portion of patients who had the same mutations that affected the patients in the 2017 trial. Instead of shrinking during treatment, their rectal tumors grew.Perhaps, Dr. Cercek and Dr. Diaz reasoned, immunotherapy with a checkpoint inhibitor would allow such patients to avoid chemotherapy, radiation and surgery.New Developments in Cancer ResearchCard 1 of 7Progress in the field.

Another patient who had the same treatment did not survive. But the demonstration of the technique could help with other cancers.Researchers have managed to tame pancreatic cancer in a woman whose cancer was far advanced and after other forms of treatment had failed.The experiment that helped her is complex and highly personalized and is not immediately applicable to most cancer patients. Another pancreatic cancer patient, who received the same treatment, did not respond and died of her disease.Nonetheless, a leading journal — The New England Journal of Medicine — published a report of the study on Wednesday.Dr. Eric Rubin, the journal’s editor in chief called the proof of concept experiment “an important step along the way” to devising similar treatments that might be applicable to lung, colon and other cancers.The experiment involved genetically reprogramming the patient’s T cells, a type of white blood cell of the immune system, so they can recognize and kill cancer cells. The technique was developed by Eric Tran and Dr. Rom Leidner of the Earle A. Chiles Research Institute, a division of Providence Cancer Institute in Portland, Ore.To turn a cancer patient’s T cells into a living drug, the researchers had to overcame serious challenges. Pancreatic cancer is one of the most difficult to treat. While new treatments have allowed patients with other cancers to live longer and to have a better quality of life, pancreatic cancer has stubbornly resisted these advances. Less than 10 percent of patients live past five years.For most patients, said Dr. William Jarnagin, a pancreatic cancer specialist at Memorial Sloan Kettering Cancer Center, who was not involved in the current experiment, the cancer has already spread by the time it is discovered. Even when the tumors are caught in the pancreas and surgically removed, about 85 percent of patients have recurrences.“Our treatments are not doing the job,” Dr. Jarnagin said.The technique described in the new paper, “is not off-the-shelf,” Dr. Tran said. He added that “it takes specialized facilities and expertise to manufacture the T cells.”But, Dr. Leidner said, “the beauty of it” is that the reprogrammed T cells will only attack cancer cells. Other cells will be left alone.The first problem in trying to entice T cells to kill cancer cells is that mutated proteins that drive the growth of cancer are hidden inside cells.There is, though, a hint to the immune system that the cancer cells are abnormal. They contain fragments of mutated cancer proteins on their surface, “kind of like molecular bread crumbs,” Dr. Leidner said. The challenge was to get T cells to see those crumbs.The solution employed was to collect the patient’s own T cells and genetically modify them in the lab to recognize and attach to those bits of mutated proteins. Then the T cells were infused back into the patient.In this case the target was KRAS, a mutated protein implicated in 25 percent of all cancers, including about 95 percent of pancreas cancers, 40 percent of colon cancers and a third of lung cancers.“Folks have been trying to target KRAS immunologically for more than 20 years,” said Dr. Robert Vonderheide, a pancreatic cancer specialist and director of the University of Pennsylvania’s Abramson Cancer Center.The mutated KRAS gene “is such a bull’s-eye,” Dr. Vonderheide said, that killing cancer cells by attacking cells with KRAS mutations has “major implications.”But the encouraging result comes with some real caveats. For starters, it is not clear why the other patient who died did not respond to the therapy.Dr. Elizabeth Jaffee, a pancreatic cancer specialist at Johns Hopkins Medicine also highlighted the location of the patient’s metastases, or where the cancer had spread to. Metastases arose only in the patient’s lungs. Most pancreatic cancer patients have metastases in their liver that are more difficult to treat.“I would like to see liver lesions go away,” Dr. Jaffee said.Kathy Wilkes, the patient who was successfully treated, is 71 and lives in Ormond-by-the-Sea, Fla. It is too soon to know if the cancer will come roaring back.New Developments in Cancer ResearchCard 1 of 6Progress in the field.

Maya Cohen’s entree into the world of obesity medicine came as a shock.In despair over her weight, she saw Dr. Caroline Apovian, an obesity specialist at Brigham and Women’s Hospital, who prescribed Saxenda, a recently approved weight-loss drug. Ms. Cohen, who is 55 and lives in Cape Elizabeth, Maine, hastened to get it filled.Then she saw the price her pharmacy was charging: $1,500 a month. Her insurer classified it as a “vanity drug” and would not cover it.“I’m being treated for obesity,” she complained to her insurer, but to no avail.While Ms. Cohen was stunned by her insurer’s denial, Dr. Apovian was not. She says it is an all too common response from insurers when she prescribes weight-loss drugs and the universal response from Medicare drug plans.Obesity specialists despair but hope that with the advent of highly effective drugs, the situation will change.Novo-Nordisk, the maker of the medicine Dr. Apovian prescribed, and patient advocacy groups have been aggressively lobbying insurers to pay for weight-loss drugs. They also have been lobbying Congress to pass a bill that has languished through three administrations that would require Medicare to pay for the drugs.But for now, the status quo has not budged.No one disputes the problem — more than 40 percent of Americans have obesity, and most have tried repeatedly to lose weight and keep it off, only to fail. Many suffer from medical conditions that are linked to obesity, including diabetes, joint and back pain and heart disease, and those conditions often improve with weight loss.“The evidence is now overwhelming that there are physical changes in weight regulating pathways that make it difficult for people to lose weight and maintain their weight loss,” said Dr. Louis Aronne, an obesity medicine specialist who directs of the comprehensive weight control center at Weill Cornell Medicine. “It’s not that they don’t have willpower. Something physical is holding them back.”Ms. Cohen in 2016.via Maya CohenDr. Aronne and other obesity medicine specialists emphasize that obesity is a chronic disease that should be treated as intensively as heart disease, diabetes, high blood pressure or any other chronic illness are. But, they say, that rarely happens.“Access to medicines for the treatment of obesity is dismal in this country,” said Dr. Fatima Cody Stanford, an obesity medicine specialist at Massachusetts General Hospital and Harvard Medical School.But even if a patient’s insurer will cover weight loss drugs, most doctors do not suggest the drugs and most patients do not ask for them, as they fail to realize there are good treatment options, said Dr. Scott Kahan, an obesity medicine specialist in Washington, D.C. And, he added, even if doctors and patients know there are F.D.A. approved drugs, many think they are “unsafe or not well studied and that everyone regains their weight.”The medical system bears much of the blame, Dr. Stanford said. Just 1 percent of doctors in the United States are trained in obesity medicine. “It’s the biggest chronic disease of our time, and no one is learning anything about it,” she said.Data on medication use by patients predate the newer, more effective and safe drugs made by Novo Nordisk and Eli Lilly. Still, obesity medicine doctors say, they doubt that the number has changed much from the earlier studies that found that less than 1 percent who are eligible obtained one of these drugs. That is the about the same percentage as those who get bariatric surgery which most insurers, including Medicare, pay for.“The perception is, ‘If you are heavy, pull yourself up from your bootstraps and try harder,’” Dr. Kahan said.And that, he adds, is a perception many patients, as well as doctors, share, making them reluctant to seek medical help or prescription medications.Then there is the problem Ms. Cohen ran into: Insurers that do not cover weight-loss drugs.But some obesity specialists have found a strange workaround to get an effective but expensive Novo Nordisk drug for patients with obesity whose insurers will not pay.Ozempic, or semaglutide, is often covered by insurance companies as a diabetes drug.Ryan David Brown for The New York TimesMs. Cohen took a mix of prescription weight-loss pills.Ryan David Brown for The New York TimesThe workaround exploits quirks in the way Novo Nordisk markets its drugs. The company sells a drug, semaglutide, for both diabetes and for obesity. As a diabetes drug, it is called Ozempic and has a list price of $892 for four weeks. It is easily available at pharmacies, and insurance companies cover it for people with diabetes.Novo Nordisk sells two weight loss drugs that are of the same class in two doses — liraglutide as Saxenda, and semaglutide at a higher and more effective dose as Wegovy. The list price — the suggested retail price — for both is about $1,350 a month. That means the same drug costs 51 percent more if it is used to treat obesity than if it is used for diabetes.But as an obesity drug, it is hard to get.Not only do most U.S. insurers decline to pay for Saxenda or Wegovy because they are weight-loss drugs, but Wegovy supplies are so limited that the company has asked doctors not to start new patients on it.Eli Lilly has a similar and seemingly more powerful weight-loss drug, tirzepatide, which it hopes to get approved for people with obesity. It was recently approved to treat diabetes under the name Mounjaro. As a diabetes drug, its retail price is $974 a month.Douglas Langa, an executive vice president at Novo Nordisk, said the Wegovy supply problem was caused by a manufacturing issue that should be resolved later this year.He also said that diabetes and obesity were “separate categories, separate marketplaces” to explain the difference in price between the companies’ two drugs that were based on the same medicine, semaglutide. He said Wegovy’s price “reflects efficacy and clinical value in this area of unmet need.”Dr. Stanford was appalled.“It’s unbelievable,” she said, adding that it was a gross inequity to charge people more for the same drug because of their obesity. She finds herself in an untenable situation: getting excited when her patients with obesity also have diabetes because their insurers pay for the drug.Dr. Apovian says she too finds herself rejoicing when patients have high blood sugar levels — and that was what ultimately resolved Ms. Cohen’s problem.Her insurance company would cover Ozempic, but it would not cover Saxenda. So she started taking Ozempic, with a $70 a month copay.Ms. Cohen — who measured at five feet fall and weighed 192 pounds when she saw Dr. Apovian — had a dramatic response to Saxenda. She has lost 54 pounds and now weighs 138 pounds. Her waist size, which was 46 inches, is now 33 inches. She has more energy and her joints do not hurt.“It has absolutely changed my life,” Ms. Cohen said.

When his baby boy was diagnosed with the illness, he made it his mission to combat it. He later took his expertise back to his native Ghana.Soon after his first child, Kwame, was born on May 13, 1972, Dr. Kwaku Ohene-Frempong discovered that the boy had a fatal genetic disease.“I was holding Kwame, and he came upstairs with tears in his eyes,” his wife, Janet Ohene-Frempong, said in an interview, recalling the moment her husband broke the news. “He said, ‘Our son, Kwame, has sickle cell disease.’ He knew what that meant.” Sickle cell can result in searing pain, organ damage, strokes, susceptibility to infections and premature death.Dr. Ohene-Frempong, a medical student at Yale at the time, then called his mother at their family home in Ghana. “God is telling you something,” she told him. The message, she said, was to use his medical training to help combat the disease. And that is what he did “until he drew his last breath,” Ms. Ohene-Frempong said.“The most important thing that happened to us is Kwame’s birth,” she added. “It changed the trajectory of our lives and of hundreds and hundreds of people around the world. All the work he did — every bit of it — he did because of Kwame.”Dr. Ohene-Frempong, familiarly known by his initials, Kof (pronounced cough), died on May 7 in Philadelphia. He was 76. The cause was metastatic lung cancer, his wife said.Dr. Ohene-Frempong worked for decades at the Children’s Hospital of Philadelphia, part of the University of Pennsylvania. At CHOP, as it is known, he established its Comprehensive Sickle Cell Center.Dr. Alexis Thompson, a colleague and sickle cell expert there, said in an interview: “I relied on his wisdom at almost every turn in my career. Part of it was watching with this tremendous awe what his vision was and the things he thought to do to move this field forward.”Dr. Ohene-Frempong was a leader of a large, federally-funded study, the Cooperative Study of Sickle Cell Disease, that helped answer an important question: What is the natural course of the disease?Analyzing the study’s data, he found that the disease could result in blockages in blood vessels in the brain, leading to a high rate of strokes in children with sickle cell. That led other researchers to be able to predict which children were most at risk and to discover that regular transfusions could prevent most strokes in those children.In his native Ghana, Dr. Ohene-Frempong established a pilot program to provide screening for sickle cell disease among newborns in the southern city of Kumasi. It was the first such program in sub-Saharan Africa. In addition to identifying children with the illness, the program referred them to specialized clinics that provided treatments like antibiotics to prevent infections, routine immunizations and a drug, hydroxyurea, that can reduce the risk of complications from sickle cell.Kwaku Ohene-Fremong was born on March 13, 1946, in Kukurantumi, in eastern Ghana, to Kwasi Adde Ohene and Adwoa Idi Boafu. His father was a cocoa farmer and a prominent member of a royal family.Kwaku attended a boarding school, Prempeh College, then went to Yale University, where he majored in biology and was captain of the track and field team, setting indoor and outdoor records in the high hurdles. While a student, he met Janet Williams, who was attending Cornell University. They married on June 6, 1970, one week after they had both graduated.Dr. Ohene-Frempong said in an interview in 2019 that he first found out about sickle cell when he and some friends attended a lecture about the disease at Yale. As he sat listening, he said, he suddenly recognized the disease: It was in his family but had gone undiagnosed. One of his cousins had the symptoms and died at 14.“He was in pain,” he said of his cousin. “His eyes were very yellow, and he was very skinny.”Dr. Ohene-Frempong continued on to medical school at Yale, then went to New York Hospital Weill-Cornell Medical Center in Manhattan for his residency. He studied pediatric hematology at the Children’s Hospital of Philadelphia before moving to the Tulane University School of Medicine, where he was associate professor of pediatrics.In his six years at Tulane, he established the Tulane Sickle Cell Center of Southern Louisiana, a medical care facility, and helped the state health department develop a newborn-screening program for the disease.In 1986, Dr. Ohene-Frempong returned to Children’s Hospital and remained there for 30 years before leaving to work full time in Ghana, at the Kumasi Center for Sickle Cell Disease, a research and treatment center. He was still based there when he returned to Philadelphia for cancer treatment.“He was very, very aware of the limitations of working in Africa,” Ms. Ohene-Frempong said. “His goal was to raise the standards of care. He said, ‘It can be done in America, and that is our goal here.’”As part of that mission, Dr. Ohene-Frempong became president of the Sickle Cell Foundation of Ghana and the national coordinator for the American Society of Hematology’s Consortium on Newborn Screening in Africa.His honors and accolades were many, including, from Ghana, the Order of the Volta in 2010 and the Millennium Excellence Award in Medicine in 2015. In the United States, in 2020, he received the Assistant Secretary of Health Exceptional Service Medal, the highest civilian award given by the Public Health Service, part of the Department of Health and Human Services. The American Society for Hematology honored him in 2021 with its Stratton Award for Translational and Clinical Science.But despite the progress that Dr. Ohene-Frempong and others had made in caring for people with sickle cell disease, his son, Kwame, did not survive it: He died in 2013 at age 40, the father of two young children.In addition to his wife, Dr. Ohene-Frempong is survived by his daughter, Afia Ohene-Frempong; three brothers, Kwabena Ohene-Dokyi, Kwasi Ohene-Owusu and Reynolds Twumasi; a sister, Ama Ohene- Agyeiwaa Boateng; a grandson; and a granddaughter.

New treatments aim for a gene variant causing the illness in people of sub-Saharan African descent. Some experts worry that focus will neglect other factors.In a Zoom call this spring with 19 leaders of A.M.E. Zion church congregations in North Carolina, Dr. Opeyemi Olabisi, a kidney specialist at Duke University, asked a personal question: How many of you know someone — a friend, a relative, a family member — who has had kidney disease?The anguished replies tumbled out from the assembled pastors:A childhood friend died, leaving a daughter behind.A father and sister felled by the disease.Uncles and sons lost.Three cousins and a brother-in-law on dialysis.None of this surprised Dr. Olabisi, who disclosed that he, too, had lost family members to the disease. His best friend, who had taught him to ride a bike in his native Nigeria, died of kidney failure in his early 30s.Kidney specialists have long known that Black Americans are disproportionately affected by kidney disease. While Black people make up about 12 percent of the U.S. population, they comprise 35 percent of Americans with kidney failure. Black patients tend to contract kidney disease at younger ages, and damage to their organs often progresses faster.Social disparities and systemic racism contribute to this burden, but there is also a genetic factor. Many with sub-Saharan ancestry have a copy of a variant of the gene APOL1 inherited from each parent, which puts them at high risk. Researchers have known for a decade that APOL1 is one of the most powerful genes underlying a common human disease.But there is hope now that much of this suffering can be alleviated. As many as 10 companies are working on drugs to target the APOL1 variants. And Dr. Olabisi has a federal grant to test whether baricitinib, a drug that treats rheumatoid arthritis, can help kidney patients with the variants.Yet the promise of treatments comes with difficult questions.Should genetic testing be offered and, if so, to whom? Although the variants increase risk, they do not preordain kidney disease. If someone knows that they have the variants, will they live in fear of kidney failure?There are as yet no proven ways to reduce the risk of kidney disease in those with two copies of the variants. Rigorous control of blood pressure — a major risk factor for progression of kidney disease — can be difficult to achieve in those who have the variants.“Now we know that the reason you can’t get your blood pressure down is because you have APOL1 kidney disease that is ferociously raising your blood pressure,” said Dr. Jeffrey Kopp, a kidney researcher at the National Institutes of Health. “It’s not your fault.”Despite their elation at the progress being made, some experts like Dr. Olabisi say that a laser focus on variants may let policymakers ignore the social and economic disparities underlying the disease.But, he added, “we don’t want to pretend that the biology doesn’t exist.” That, he said, “would not be doing the community any good.”Dr. Opeyemi Olabisi in the Duke Molecular Physiology Institute in Durham, N.C. He has lost friends and family members to kidney disease.Cornell Watson for The New York TimesA Farmer Provides a ClueWhile it has long been known that kidney failure occurs in African Americans five times as much in as it does in white Americans, “We had never been able to understand all the reasons,” said Dr. Neil Powe, a professor of medicine and an epidemiologist at the University of California, San Francisco.Researchers began looking for a genetic cause. Finally, a little more than a decade ago, a Havard team led by Giulio Genovese, Dr. David Friedman and Dr. Martin Pollak found it: variants of APOL1 that ramped up the gene’s activity.Understand Sickle Cell DiseaseThe rare blood disorder, which can cause debilitating pain, strokes and organ failure, affects 100,000 Americans and millions of people globally, mostly in Africa.The Global Epicenter: In Nigeria, where 150,000 babies are born each year with sickle cell disease, the effects of the condition are pervasive and devastating. On the Edge of Fear: A cure for the disease, which in the United States mostly affects Black people, seems near. For some, it may come too late.Preventing Complications: A legacy of neglect toward Americans with sickle cell means that patients may not receive the treatments needed to stave off the disease’s risks. A Haunting Memory: The Times reporter Gina Kolata shares her experience reporting on the inequities in access to medical advances in the treatment of the disease.It was a complete surprise. APOL1 is part of the immune system and can destroy trypanosomes — protozoa that can cause illnesses. But no one expected it to have anything to do with the kidneys.It turns out that the variants rose to a high frequency among people in sub-Saharan Africa because they offer powerful protection against deadly African sleeping sickness, a disease caused by trypanosomes. It is reminiscent of another gene variant that protects against malaria but causes sickle cell disease in those who inherit two copies. That variant became prominent in parts of Africa and other areas of the world where malaria is common, but sickle cell variants are much less common than APOL1 risk variants.About 39 percent of Black Americans have one copy of the gene’s risk variants; another 13 percent, or nearly 5.5 million, have two copies. Those with two copies are at increased risk for fast progressing kidney disease that often starts in young adulthood. Approximately 15 percent to 20 percent of those with two copies develop kidney disease in their lifetime.In contrast, 7.7 percent of Americans with African ancestry have one copy of the sickle cell variant, and 0.3 percent have two copies.“What nature gave with one hand, it took away with the other,” Dr. Olabisi said.One way to treat kidney disease might be using medicines that block the gene and its variants from acting in the body. But researchers had to find out if APOL1 was necessary for kidney function. If it was, drugs that blocked it might do more harm than good.Researchers found an answer: A farmer in India had no APOL1 gene. His kidneys were totally healthy.Often, in drug development, Dr. Friedman says, the drug dose has to be fine tuned — too much is dangerous and too little is useless. The discovery of the farmer, he said, “tells you you can probably drive the level of the APOL1 protein very low.”But ethical issues have tempered some experts’ enthusiasm about the genetic discoveries.Harriet A. Washington, a lecturer in ethics at Columbia University and author of the book “Medical Apartheid,” worries that knowledge of the role of APOL1 variants can drive the medical establishment toward “a blame-the-victim approach signaling an inherent flaw in African Americans.”The implication, she said: “This is something happening in nature, so what can we do about it?” Such an attitude, she added, “invites futility and absolves health care from treating sufferers.”Joseph L. Graves, Jr., a professor of biological sciences at North Carolina Agricultural and Technical State University, raised another issue. “We don’t want to fall into the myth of the genetically sick African,” he said.“All populations have genetic variants, but the action of those variants is determined by the environment in which those people live,” Dr. Graves explained. “People want to find simple explanations for complex phenomena. Find a genetic variant and make the story simple, but that’s not how it works. Environmental effects are really important.”He added that for Black Americans, profound environmental effects rise from structural racism — inequitable effects of law and policies — which can lead to a lack of access to health care, including preventive care to ward off chronic illness.Erika Blacksher, an ethicist at the nonprofit Center for Practical Bioethics in Kansas City, Mo., added that while finding “a treatment that might counteract the effect of the genetic variant is good news,” she worried that social inequities could not be disentangled from the high rate of kidney disease among those with sub-Saharan African ancestry, not all of whom have the APOL1 variant. Emphasizing the variants, she said, “deflects from our social responsibility to actually change the conditions that contribute to the onset of chronic kidney disease.”Making a PlanMalcolm, left, and Martin Lewis, 26, have lupus, an autoimmune disease that attacks the body’s tissues and organs.Amir Hamja for The New York TimesMartin and Malcolm Lewis, 26-year-old identical twins, have lupus, an autoimmune disease that can ravage the body’s organs. So when Martin developed kidney disease at age 10, his doctors said lupus was to blame.In July 2020, Martin, an actor who lives in Brooklyn, was visiting Malcolm, a data analyst who was hospitalized at Duke with a lupus flare-up. There, the brothers met Dr. Olabisi, who told them about APOL1.They discussed his research project, which involves testing Black Americans and enrolling those with the variant and with kidney disease in a study of the arthritis drug. He invited them to participate and asked if they wanted to know if they had APOL1 variants.“I was all for it,” Malcolm said. So was Martin.When they were tested, the brothers learned they had the variants and that the variants, not lupus, most likely were damaging their kidneys. They hardly knew how to react.“I am still trying to grapple with it,” Malcolm said.But Dr. Olabisi was not surprised. Researchers think the variants cause kidney disease only when there is a secondary factor. A leading candidate is the body’s own antiviral response, interferon, which is produced in abundance in people with lupus.High levels of interferon also occur in people with untreated H.I.V. As happens in people with Covid-19, they can suffer an unusual and catastrophic collapse of their kidneys if they have the variants. Other viral infections, including some that may go unnoticed, can elicit surges of interferon that could set off the APOL1 variants. Interferon is also used as a drug to treat some diseases including cancer and was tested as a treatment for Covid patients.For now, there is little Malcolm and Martin can do except take medications to control their lupus.Martin said he understands all that, but he’s glad he learned he has the variants. Now, he knows what he might be facing.“I’m the kind of person who likes to plan,” he said. “It does make a difference.”From a Gene to DrugsWhile Dr. Olabisi is waiting to start his study, a drug company, Vertex, has forged ahead with its own research. But there was no agreement on how APOL1 variants caused kidney disease, so it was not clear what a drug was supposed to block.“If you don’t understand the mechanism, that means you can’t measure effects in a lab,” said Dr. David Altshuler, chief scientific officer at Vertex. “And if you can’t measure effects in the lab, that means you can’t correct them.”It was known how the APOL1 protein protected against sleeping sickness — it punched holes in the disease-causing trypanosomes, making them swell with fluid and burst.Vertex researchers hypothesized that the variants spurred APOL1 proteins to punch holes not just in trypanosomes but also in kidney cells.What followed was years of work in lab studies and in animals given genes for human APOL1 variants and then screening about a million compounds that might block APOL1.Finally, the researchers settled on a drug that worked in animal models.Vertex tested the experimental drug in a 13-week study in patients with advanced kidney disease. The drug reduced the amount of protein in their urine by 47.6 percent, a sign of improved kidney function.In late March, the company announced it would take the next step — a clinical trial that would enroll approximately 66 patients in the first phase, to find the best dose, and 400 in the next phase, to see if the drug could improve kidney functions in patients with the risk variants and kidney damage and protect them from developing kidney failure or dying.Other companies began later and have revealed less about their plans and progress. AstraZeneca, for example, would only say that it was in the early stages of testing a drug that could bind to APOL1 mRNA, the messenger that carries instructions from the gene to cells’ protein making machinery.MAZE, a small biotech company, is pursuing a strategy similar to the Vertex one, said Dr. Sekar Kathiresan, a co-founder and board member.“I’m optimistic this can move quickly,” Dr. Kathiresan said.Using Their PulpitsAt the meeting with the pastors in North Carolina, Dr. Olabisi said he hoped to test 5,000 Black members of the community for kidney disease with a simple urine test and to use a saliva test to detect APOL1 variants. Testing of the arthritis drug would follow.“I’m in,” said the Rev. Dr. Daran Mitchell, the pastor of Trinity A.M.E. Zion Church in Greensboro, N.C.He and the other pastors were enthusiastic. It would be a community effort, led by people in the community and promoted on social media. Subjects could be tested in churches or in community centers or in their homes. And it was a way to advance the day when a treatment would be available.Dr. Olabisi smiled.“This gives me energy and a lot of hope,” he said.

Kidneys from Black donors are automatically downgraded in transplant assessments, but studying a gene variant could help change that.Transplant specialists, when evaluating kidneys that come from donors, try to work out how likely it is that the kidney will fail after being transplanted into a recipient. Their risk calculations consider factors including the donor’s age, height, weight and history of diabetes. And, to the dismay of some researchers, it also includes the donor’s race.Kidneys from Black donors, living or dead, are automatically downgraded as higher risk.Some experts are now asking if there is a better way of evaluating kidneys from Black donors, one that can rely more on genetic screening rather than race to assess the risk of failure.The proposed genetic screening would check whether donors carry two copies of variants in a gene, APOL1, that are strongly associated with kidney disease. Because most Black donors do not have those genetic variants, the experts argue, their kidneys should not be automatically downgraded.But before instituting that change, researchers say they have to determine if, in fact, kidneys from donors that have the risk variants of APOL1 are more likely to fail.The first hint came from a study by Dr. Barry Freedman, of Wake Forest University in North Carolina involving 1,153 deceased donor kidney transplants performed at 113 different transplant programs. It found that kidneys from deceased donors with two risk variants were twice as likely to fail rapidly compared with kidneys from donors who have one gene variant or none.But that finding will need to be replicated in a bigger research effort. It is getting underway with APOLLO, a large study sponsored by the National Institutes of Health, to assess living and deceased donors. Study researchers are testing kidney donors for APOL1 and following the fate of thousands of transplant patients who have received kidneys from Black American donors at more than 97 transplant programs.In the study, living donors can decide if they want to learn the result of their genetic test and if they want the recipient of their kidney to know the result as well. Medical privacy regulations forbid doctors from telling kidney transplant candidates if a living donor has the variants without the donor’s consent.Dr. Freedman said that whatever results come from the research, more transplant centers are broaching the idea of genetic testing people who want to donate kidneys.Until recently, he added, “many transplant centers said they don’t want to talk about it.”

The data have not yet been peer reviewed or published. But experts said the drug may give people with obesity an alternative to bariatric surgery.An experimental drug has enabled people with obesity or who are overweight to lose about 22.5 percent of their body weight, about 52 pounds on average, in a large trial, the drug’s maker announced on Thursday.The company, Eli Lilly, has not yet submitted the data for publication in a peer-reviewed medical journal or presented them in a public setting. But the claims nonetheless amazed medical experts.“Wow (and a double Wow!)” Dr. Sekar Kathiresan, chief executive of Verve Therapeutics, a company focusing on heart disease drugs, wrote in a tweet. Drugs like Eli Lilly’s, he added, are “truly going to revolutionize the treatment of obesity!!!”Dr. Kathiresan has no ties to Eli Lilly or to the drug.Dr. Lee Kaplan, an obesity expert at the Massachusetts General Hospital, said that the drug’s effect “appears to be significantly better than any other anti-obesity medication that is currently available in the U.S.” The results, he added, are “very impressive.”Dr. Kaplan who consults for a dozen pharmaceutical companies, including Eli Lilly, said he was not involved in the new trial or in the development of this drug.On average, participants in the study weighed 231 pounds at the outset and had a body mass index, or B.M.I. — a commonly used measure of obesity — of 38. (Obesity is defined as a B.M.I. of 30 and higher.)At the end of the study, those taking the higher doses of the Eli Lilly drug, called tirzepatide, weighed about 180 pounds and had a B.M.I. just below 30, on average. The results far exceed those usually seen in trials of weight-loss medications and are usually seen only in surgical patients.Some trial participants lost enough weight to fall into the normal range, said Dr. Louis J. Aronne, director of the comprehensive weight control center at Weill Cornell Medicine, who worked with Eli Lilly as the study’s principal investigator.Most of the people in the trial did not qualify for bariatric surgery, which is reserved for people with a B.M.I. over 40, or those with a B.M.I. from 35 to 40 with sleep apnea or Type 2 diabetes. The risk of developing diabetes is many times higher for people with obesity than for people without it.An Eli Lilly spokeswoman said the company did not have a public timeline for seeking approval of the drug with the Food and Drug Administration.Because obesity is a chronic medical condition, patients would need to take tirzepatide for a lifetime, as they do for blood pressure or cholesterol drugs, for example.Dr. Robert F. Kushner, an obesity expert at Northwestern University’s Feinberg School of Medicine and a paid consultant to Novo Nordisk, said the new drug along with a similar but less effective one by Novo Nordisk, can close a so-called treatment gap.Diet and exercise, combined with earlier obesity drugs, usually yield perhaps a 10 percent weight loss in patients. That is enough to improve health, but not nearly enough to make a big difference in the lives of peoples who are obese.The only other treatment is bariatric surgery, which can result in substantial weight loss. But many people are ineligible or simply do not want the surgery.With the Eli Lilly drug and Novo Nordisk’s semaglutide, which was recently approved, “we really are on the cusp of a new way of treatment,” Dr. Kushner said.Semaglutide, marketed as Wegovy, Novo Nordisk’s own treatment for diabetes and weight loss.Cristian Mihaila/Novo Nordisk, via Associated PressBut prices may be a barrier. Insurers often will not pay for weight loss drugs. The Novo Nordisk drug, whose brand name is Wegovy, has a list price of $1,349.02 per month.Experts worry that tirzepatide, if approved, might carry a price in the same range. Many people who could most benefit from weight loss may be unable to afford such expensive drugs.The Eli Lilly study lasted 72 weeks and involved 2,539 participants. Many qualified as obese, while others were overweight but also had such risk factors as high blood pressure, high cholesterol levels, cardiovascular disease or obstructive sleep apnea.They were divided into four groups. All received diet counseling to reduce their calorie intake by about 500 a day.One group was randomly assigned to take a placebo, while the other three received doses of tirzepatide ranging from 5 milligrams to 15 milligrams. Patients injected themselves with the drug once a week.Those taking the highest dose lost the most weight, the investigators found. Participants taking a placebo lost 2.4 percent of their weight, an average of 5 pounds, typical for a diet study.Dr. Nadia Ahmad, senior medical director of Eli Lilly’s obesity program, said that seeing the results was an emotional moment for her.“I don’t think I ever imagined we could reach this degree of weight loss with a medicine,” she said. “We only got this far with surgery.”For decades, people who are overweight or have obesity were told that solving the problem was up to them. Diet and exercise were the prescriptions, and they simply did not work for many people. Most tried diet after diet, only to regain any weight they lost.Last year, the situation began to change when Novo Nordisk received approval from the Food and Drug Administration to market semaglutide. The drug can elicit a 15 percent to 17 percent weight loss in people with obesity.The medications are among a new class of drugs called incretins, which are naturally occurring hormones that slow stomach emptying, regulate insulin and decrease appetite. The side effects include nausea, vomiting and diarrhea. But most patients tolerate or are not bothered by these effects.Incretins raise the bar for the sort of weight loss possible with drugs. But they also pose difficult questions about whether bariatric surgery is becoming a relic of the past. Already there are new versions of incretins in development that might be even more powerful than the Eli Lilly drug.Even without them, Dr. Aronne said, the reductions observed with the Eli Lilly drug are “squarely in the range of surgical weight loss.”Some patients who have had bariatric surgery describe mixed results. Sarah Bramblette, a board member of the Obesity Action Coalition, had bariatric surgery only to regain the weight.Now 44, she weighed 500 pounds when she had the operation 20 years ago, which enabled her to get down to 250 pounds. Over the years, though, her weight crept back up to 490 pounds. She needed heart surgery but was too heavy for the operating table. Diets — and she has tried them repeatedly — did not help.Novo Nordisk’s semaglutide enabled her to get down to 430 pounds. Now, Ms. Bramblette said, she would like to try the Eli Lilly drug if it becomes available.“Trust me, I would not choose to be this size,” Ms. Bramblette said. “I need to lose weight.”

A promo for an upcoming Fox show describes a testosterone “calamity” among American men, along with an unlikely treatment.Are you a man worried about your testosterone levels? Hoping to give them a boost? Tucker Carlson, the Fox News host, has a solution.A promotional video for a new installment in a video series by Mr. Carlson describes a “total collapse of testosterone levels in American men,” positing an explanation for what he and many conservatives see as a creeping loss of masculinity in today’s society.Chock-full of oiled, shirtless men performing vaguely masculine tasks, like turning over giant tires and throwing a javelin, the video has already been widely remarked upon on social media for its bizarre erotic imagery.But one shot in particular stands out: a naked man atop a rock pile, limbs outflung, exposing his genitals to the red light issuing from what appears to be a waist-high air purifier. Something very like the theme from “2001: A Space Odyssey” plays in the background.This is the treatment proposed by Mr. Carlson’s “documentary”: Revive your underperforming testicles with red light, in particular a device made by a little known company called Joovv.A leading endocrinologist says — no surprise — the whole thing is ridiculous, and not just because of the man receiving light therapy atop a pile of stone slabs in the dead of night.First, there is precious little evidence that testosterone “levels are declining by roughly 10 percent per decade, completely changing the way people are at the most fundamental level,” as Mr. Carlson has said.Studies examining changes in testosterone over time are challenging for several reasons, including difficulties in recruiting large populations of normal subjects, daily circadian changes in testosterone, and differences in testing methods over time, noted Dr. John Amory, an expert on male reproductive health at the University of Washington.Mr. Carlson and Fox News did not respond to requests for comment.Tied to the anxiety over testosterone is another hotly disputed assertion: that sperm counts have been declining among men in the Western world for decades. Huge numbers of studies have been done, and there is no scientific consensus on the scope of the problem or whether it exists at all.So what’s all this about bathing one’s testicles in red light?Scott Nelson, co-founder of Joovv, said in an email, “The published data around light therapy and testosterone production is pretty light, but the limited evidence is fairly compelling.” He provided a link to a study on the Joovv website, apparently unpublished, reporting that red light worked best to boost testosterone in four men who were also on a ketogenic diet.Dr. Amory was not impressed. “Obviously, doing two interventions — red light and diet — at once in a non-randomized, non-blinded, underpowered study with unclear methods isn’t of much use to understanding cause and effect,” he said. “Biological plausibility here is also very weak.”The bottom line, for readers anxious about the decline of American virility: “In the absence of any evidence of benefit from clinical trials, I wouldn’t currently recommend this as a treatment for testosterone or symptoms of low testosterone,” Dr. Amory said.“It’s notable that there is a long history of pseudoscientific treatments for low testosterone that have not proven to be useful over time,” he added.