US health department recommends looser restrictions on cannabis

Published1 hour agoShareclose panelShare pageCopy linkAbout sharingImage source, Getty ImagesBy Max MatzaBBC News, SeattleThe US Department of Health and Human Services has called on the Drug Enforcement Agency (DEA) to loosen federal rules on cannabis. The drug is illegal at the federal level despite 40 of 50 US states having passed laws legalising its use in some form. Cannabis is currently listed in the same class of drugs as heroin and LSD. If the DEA changes its classification, it could mark the most significant shift in US drug policy in decades. Cannabis is currently classified as a schedule 1 drug under the Controlled Substances Act, meaning it has no medical use and a high chance of abuse. The change to schedule 3 would align it with drugs listed as having a low potential for dependency and abuse. Ketamine, codeine, and anabolic steroids fall under that classification. Last year, President Joe Biden asked his attorney general and health secretary to oversee a review on whether cannabis should be listed as a less serious drug.The proposal was presented to the DEA by the Department of Health and Human Services (HHS) on Tuesday.”As part of this process, HHS conducted a scientific and medical evaluation for consideration by DEA,” the agency said in a statement.”DEA has the final authority to schedule or reschedule a drug under the Controlled Substances Act. DEA will now initiate its review.”The HHS, in its statement, said “this administrative process was completed in less than 11 months, reflecting this department’s collaboration and leadership to ensure that a comprehensive scientific evaluation be completed and shared expeditiously”. What Biden’s cannabis review means for AmericansMaking it big selling legal weed is harder than it looksThe recommendation stops short of removing cannabis from the Controlled Substances Act’s list altogether. Some advocates have pushed the administration to de-schedule the drug, meaning repeal it from the Controlled Substances Act and regulate it in the same way as alcohol or tobacco.Rescheduling it could open it up to further research and allow banking in the cannabis industry to operate more freely. Currently, most marijuana businesses in the US are forced to operate in cash, due to tax laws banning banks from handling money generated from certain drug sales. Public opinion polling indicates that a majority of Americans support some form of legalisation of the drug. Cannabis is legal for adult recreational use in 23 states, including all west coast states and in Washington DC. It is permitted for medical use in 38 states. This video can not be playedTo play this video you need to enable JavaScript in your browser.More on this storyMastercard demands banks not accept debit for weedPublished26 JulyMaking it big selling legal weed is harder than it looksPublished18 December 2022

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Promising evidence for sickle cell gene therapy

New research published in the New England Journal of Medicine indicates that stem cell gene therapy may offer a promising, curative treatment for the painful, inherited blood disorder sickle cell disease (SCD).
The findings from a new clinical trial, published August 31, add to the body of evidence supporting gene therapy as a treatment for sickle cell disease, which primarily impacts people of color.
About 100,000 Americans have sickle cell disease, according to the U.S. Centers for Disease Control and Prevention. The condition, which can cause a lifetime of pain, health complications and expenses, affects one in 365 Black babies born in the U.S. and one in 16,300 Hispanic babies.
Until recently, the only treatment options have been intensive bone marrow transplants from siblings or matched donors. But other curative therapies are now on the horizon. The University of Chicago Medicine Comer Children’s Hospital was one of three sites to enroll patients in the clinical trial, which tested a stem cell gene therapy to treat sickle cell disease.
As part of the trial, researchers used CRISPR-Cas9 to edit specific genes in stem cells — the building blocks of blood cells — taken from each patient. The edits increased the cells’ production of fetal hemoglobin (HbF), a protein that can replace unhealthy, sickled hemoglobin in the blood and protect against the complications of sickle cell disease. The patients then received their own edited cells as therapeutic infusions.
The therapy was the second for this disease to use CRISPR-Cas9 technology and the first to target a new genetic area and use cryopreserved stem cells with the hope of increasing access to such a treatment. Other gene therapy studies for SCD have used lentiviruses — a type of virus often modified and used for gene editing which remain in the cell long-term. No foreign material remains in stem cells edited with CRISPR-Cas9.
Trial participants who received the CRISPR-edited stem cells reported a decrease in vaso-occlusive events, a painful phenomenon that occurs when sickled red blood cells accumulate and cause a blockage.

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Nitrous oxide: Laughing gas ban could harm users, experts warn

Published7 hours agoShareclose panelShare pageCopy linkAbout sharingImage source, Getty ImagesBy Yasminara Khan and Ali HamedaniBBC NewsnightThe proposed ban of nitrous oxide could stop users seeking medical help in hospitals, health experts have warned.In a letter to the government, seen by BBC Newsnight, 15 neurologists and related health experts say possession of the drug should not be criminalised. Despite rising numbers of hospital patients suffering the effects of so-called laughing gas, they warn the ban could worsen the stigma around users.The government has responded, saying it plans to go ahead with the ban. Nitrous oxide is a colourless gas often sold in metal canisters. It is one of the most commonly used drugs by 16 to 24-year-olds, Heavy use can lead to nerve-related symptoms – being unable to walk, falling over, or experiencing tingling or loss of sensation in the feet and hands. Some users have nerve-related bladder or bowel problems, erectile dysfunction or incontinence. Supply of nitrous oxide for recreational use is currently banned – but possession is not. And earlier this year, the government proposed an amendment to the law – which could come into force before the end of the year – meaning it would become illegal to possess the drug. Nitrous oxide: What is it and how dangerous is it?Ban will not stop laughing gas use, experts warnLaughing gas should not be banned, review saysWriting to the minister for policing, Chris Philp, the 15 medical professionals say making possession of the drug illegal was “unlikely to translate to health benefits in our patients”. That is despite leading medics telling Newsnight they have seen an increase of nitrous oxide patients.The author of the letter, Dr Alastair Noyce, Professor of Neurology and Neuroepidemiology, said the ban risked creating “fear of a criminal record” among young users. “People may delay coming to hospital at a time when their symptoms are treatable,” he said. “The net effect of that may be that they develop long-term harm damages.”He said there was “very little evidence that the criminalisation will lead to reductions in neurological harm and will impact people’s opportunities who are not in education and employment”. The letter urged the government to fund a national education campaign in schools and through the media “to ensure the public understand the risks posed by nitrous oxide misuse”.This video can not be playedTo play this video you need to enable JavaScript in your browser.Under the proposals, those found in possession of the drug could face up to two years in prison, an unlimited fine, or both. The government has launched consultations of the plan, both with experts and the public. Experts have previously said a ban on laughing gas will not stop people using it. The Drug Science scientific charity said a blanket ban was “completely disproportionate” and “would likely deliver more harm than good”.However, Mohammad Ashfaq, who runs Kick It, a grassroots organisation in Birmingham, said the proposed ban would help stop misuse of the drug. “At the moment, it is very difficult for the police to completely eradicate,” he said. “Communities are getting frustrated. The law will make a difference. It would be a lot clearer.” BBC Newsnight also spoke with Katherine Bramwell, from South Wirral, who said she didn’t know anything about nitrous oxide until her son got addicted. She says he found it hard to get off the substance due to the psychological dependence.”When you’re the one going to pick your son off from somewhere and finding him just basically lay there – can’t walk, can’t speak. You know, you’re waiting for a call to tell you that your son is dead.”In a statement to Newsnight, the government said it would proceed with the nitrous oxide ban. It said it had set out a “clear strategy” in its anti-social behaviour action plan “for the police to deal with the misuse of this substance”.

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Biden Makes Lower Drug Prices a Centerpiece of His 2024 Campaign

President Biden’s Inflation Reduction Act allows Medicare to negotiate some drug prices, a change that the pharmaceutical industry and Republicans have opposed for decades.As he heads toward a re-election campaign next year, President Biden is betting that his success in pushing for policies intended to lower health care costs for millions of Americans will be rewarded by voters at the ballot box.In speech after speech, Mr. Biden talks about capping the cost of insulin at $35, putting new limits on medical expenses for seniors, making some vaccines free and pushing to lower the prices of some of the most expensive drugs in the world.At the White House, Mr. Biden and his advisers have already begun to elevate the issue as a centerpiece of his agenda. And at his campaign headquarters in Wilmington, Del., aides are preparing television ads, talking points and speeches arguing that Mr. Biden’s push for lower health care costs is a stark contrast with his Republican opponents.“The president will have a very strong case to make,” said Senator Amy Klobuchar of Minnesota, a member of the president’s national campaign advisory board. “Not only will people want to keep the benefits they have seen, they are going to want to get the benefits that are coming their way.”On Tuesday, the White House announced that the Biden administration will negotiate on behalf of Medicare recipients for lower prices on 10 popular — and expensive — drugs that are used to treat diabetes, heart disease and other chronic illnesses.The move was made possible by passage last year of Mr. Biden’s Inflation Reduction Act, which for the first time allows Medicare to negotiate drug prices for older adults, a change that has been opposed by the pharmaceutical industry for decades.Republicans also generally oppose giving the government the right to negotiate drug prices. But the candidates for the Republican presidential nomination have said little about the cost of medication, focusing instead on abortion, transgender medical issues and Covid lockdowns.In his speeches, Mr. Biden rails against the industry and his Republican adversaries in Congress, all of whom voted against the law that included the prescription drug provisions. Aides say it is an effective message.“Today is the start of a new deal for patients where Big Pharma doesn’t just get a blank check at your expense,” the president said at a White House event celebrating the change.Since signing the law a year ago, Mr. Biden has repeatedly called it one of his proudest legislative victories. But his approval numbers have hardly budged. And while polls show that the new policy is widely popular among Americans who know about it, they also suggest that far fewer people are even aware that the change was made.That is most likely because prices on just the first handful of drugs are not scheduled to actually drop until 2026 at the earliest, assuming Mr. Biden’s program survives legal challenges. Drug companies have filed numerous lawsuits against the administration that claim the law is unconstitutional. Court cases could drag on for years.In its lawsuit against the administration, the Pharmaceutical Research and Manufacturers of America, an industry trade group, called the plan for negotiated prices “a government mandate disguised as negotiation.”Even if Mr. Biden’s plan goes into effect, older adults who have made the choice to ration their drugs will have to continue doing so until more than a year after the 2024 presidential election.Danny Cottrell, 67, a pharmacist who owns his retail pharmacy group in Brewton, Ala., said he regularly advised his Medicare patients on the ins and outs of the government’s prescription program. He welcomed Mr. Biden’s changes, but said it would be up to people like him to explain the complicated process.“I got to remind them, this doesn’t start till 2026,” Mr. Cottrell said. “And then also remind them this thing will change several times between now and then.”Neera Tanden, Mr. Biden’s top domestic policy adviser, said the White House was confident that the plan would survive the legal challenges.“It is absurd to argue that negotiation is unconstitutional,” she said in an interview. “There’s nothing in the Constitution that says Medicare negotiating drug prices is unconstitutional.”But more broadly, Ms. Tanden said that she and the president’s other advisers in the West Wing were determined to make the push for lower health care costs a central part of Mr. Biden’s message to Americans.And next September, just weeks before Election Day, the administration will announce the results of the yearlong negotiations over the first 10 drugs.“We plan to work extensively, to really remind folks of this issue,” Ms. Tanden said.For the people leading Mr. Biden’s re-election campaign, the political benefits of focusing on lower health care costs are clear.Some polls show that 80 percent of Americans support giving the government the ability to negotiate lower prices for Medicare, much the way it already does for veterans and members of the military.Campaign aides said talking about lower costs of drugs or limits on out-of-pocket medical expenses is one way to help Mr. Biden win support among seniors, who traditionally have voted for Republicans in greater numbers. That is especially important in battleground states like Michigan, Arizona, Georgia and Ohio, where increasing support among older adults will be critical in close contests.The campaign’s early television ads have included numerous references to the president’s efforts to lower health care costs. A spokesman for the campaign said the issue of health care would be a central feature of a $25 million ad blitz focusing on what the president has done to lower costs overall and make economic progress.Kate Bedingfield, who served as Mr. Biden’s communications director for the first two years of his presidency, said the issue had political benefits even when it came to appealing to people who do not benefit directly from the specific cost reductions.“It draws a really clear contrast with the Republicans, who have stood in the way and continue to stand in the way of getting more done on this,” she said.Representative Michael C. Burgess, Republican of Texas and a doctor, said Mr. Biden’s drug price negotiations were akin to government-imposed price controls that would lead to drug shortages.“This administration’s approach goes beyond ‘negotiation,’” he said in a statement. “Instead, it holds pharmaceutical companies hostage, jeopardizing their future innovation and the well-being of American patients.”Mr. Biden’s campaign aides said a debate with Republicans about the cost of medical care was one they were eager to have.“MAGA Republicans running for president want to repeal the Inflation Reduction Act, which would deliver a massive win for Big Pharma and increase costs for the American people,” said Julie Chávez Rodríguez, the president’s campaign manager, referring to Republicans loyal to former President Donald J. Trump.She said the choice in the election was between Mr. Biden and “a slate of candidates focused on extreme policies that put their wealthy donors first.”Robert Jimison

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Can taking statins after a bleeding stroke lower risk of another stroke?

People who have had a stroke called an intracerebral hemorrhage who take cholesterol-lowering drugs called statins may have a lower risk of having another stroke, especially ischemic stroke, compared to people who also had an intracerebral hemorrhage but were not taking statins, according to a new study published in the August 30, 2023, online issue of Neurology®, the medical journal of the American Academy of Neurology.
Intracerebral hemorrhage is caused by bleeding in the brain. Ischemic stroke is caused by a blockage of blood flow to the brain and is the most common type of stroke.
“Previous research has had mixed results on the risk of stroke in people who are taking statins and have already had a bleeding stroke, so we evaluated this further,” said study author David Gaist, MD, PhD, of the University of Southern Denmark in Odense and a member of the American Academy of Neurology. “We looked at whether use of statins after a bleeding stroke is associated with the risk of any additional stroke, including both those caused by bleeding and by blood clots. We found that those who used statins had a lower risk of stroke, notably ischemic stroke, while there was no change in the risk of bleeding stroke.”
For the study, researchers looked at health records in Denmark and identified 15,151 people who had a first bleeding stroke.
People were followed from 30 days after their first bleeding stroke until the first occurrence of another stroke, death, or the end of follow-up, which on average lasted 3.3 years. Researchers used prescription data to determine information on statin use.
Researchers then compared 1,959 people who had another stroke to 7,400 people who did not have another stroke who were similar in age, sex and other factors. Of those who had another stroke, 757 people, or 39%, took statins compared to 3,044 people, or 41%, of those who did not have a second stroke.
After adjusting for factors like high blood pressure, diabetes and alcohol use, statin use was associated with a 12% lower risk of another stroke.

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Lucy Letby: Inquiry given powers to compel witnesses to give evidence

Published11 hours agoShareclose panelShare pageCopy linkAbout sharingImage source, Cheshire PoliceBy Nick TriggleHealth correspondentThe inquiry into how nurse Lucy Letby was able to murder seven babies will now have greater powers to compel witnesses to give evidence.In a significant move, ministers upgraded the independent inquiry after criticism from families of the victims that it did not go far enough.The inquiry, ordered after Letby was found guilty this month, was not initially given full statutory powers.Health Secretary Steve Barclay said he had listened to the families.He said he had decided a statutory inquiry led by a judge was the best way forward and “respects the wishes” of the families.Mr Barclay said the key advantage was the power of compulsion.”My priority is to ensure the families get the answers they deserve and people are held to account where they need to be,” he added.He said an announcement about who would chair the inquiry would be made in the coming days – ministers have already said it will be a judge.Richard Scorer, a lawyer who is representing two of the families, welcomed the government’s announcement.”It is essential that the chair has the powers to compel witnesses to give evidence under oath, and to force disclosure of documents. Without these powers, the inquiry would have been ineffectual and our clients would have been deprived of the answers they need and deserve,” he said.Shadow health secretary Wes Streeting said it was right the families’ wishes have now been taken into account, adding that “no stone can be left unturned in getting to the truth”.Matthew Taylor, chief executive of the NHS Confederation, said it was “vital that lessons are learnt by the NHS, its regulators, clinicians and leaders”. “There are, of course, a series of questions that are being raised by the events in Chester and the inquiry will be best placed to establish the facts of these events and to draw conclusions and recommendations for the trust and the wider NHS,” he added. Baby serial killer Lucy LetbyWhat I learned about Lucy Letby after 10 months in courtThe text messages Letby sent as she killed babiesWho is baby serial killer Lucy Letby? Doctors’ warnings ignored as Letby killed more babiesLetby, 33, was given a whole life sentence for murdering seven babies and attempting to kill six more while working at the Countess of Chester Hospital neonatal unit between 2015 and 2016, meaning she will spend the rest of her life in prison.She was found not guilty on two attempted murders and the jury could not reach verdicts on six others during the 10-month trial.The conviction made her the most prolific child serial killer in modern British history. The BBC has since been told hospital bosses failed to investigate allegations against Letby and tried to silence doctors.The hospital also delayed calling the police despite months of warnings that the nurse may have been killing babies, according to doctors who worked at the hospital.The unit’s lead consultant Dr Stephen Brearey first raised concerns about Letby in October 2015.No action was taken and she went on to attack five more babies, killing two.Hospital management had demanded doctors write an apology to Letby and told them to stop making allegations against her.The senior managers involved went on to work in other high profile roles in the NHS, prompting calls for tighter regulation of NHS managers.Unlike doctors and nurses there is no national regulation of managers.The move to make the inquiry statutory is being seen as crucial to finding out exactly what happened and what lessons should be learnt.Although some have pointed out statutory inquiries can take longer to hold – something ministers had originally said was the justification for making the inquiry non-statutory.Meanwhile, the Ministry of Justice has proposed new laws to try to force criminals to attend sentencing hearings.Letby is one of a number of high-profile offenders who have refused to appear.Powers already exist to compel people to attend but Ministry of Justice sources say they are not often used.A Ministry of Justice source said clear legislation to allow judges to increase sentences by two years was likely to encourage them to do so.Sign up for our morning newsletter and get BBC News in your inbox.More on this storyDo public inquiries answer families’ questions?Published12 hours agoWarnings ignored as Letby killed more babiesPublished18 August

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Pedal power pays off: Mountain biking benefits outweigh risks, research finds

New Curtin research into injuries sustained by trail users has found mountain biking is not the dangerous, injury-plagued sport reserved for thrill-seekers that it is often perceived to be and that the health benefits outweigh the risks.
Researchers analysed data from dozens of studies across the world, including Australia, encompassing 220,935 injured mountain bikers and 17,757 injured hikers. The study aimed to pinpoint the injury types and affected body areas in order to gain insights into the medical treatment of such cases.
Lead author PhD candidate Paul Braybrook, from Curtin’s School of Nursing, said mountain bikers were primarily injured on their upper limbs, mostly resulting in bruises, scratches and mild cuts while hikers were prone to injuring their legs and ankles, suffering mostly blisters and ankle sprains.
“Mountain biking and hiking are some of the fastest growing recreation activities in the world, so understanding the spectrum of injuries becomes paramount for effective medical care,” Mr Braybrook said.
“Despite a common perception of mountain biking as an ‘extreme’ sport, we found most reported injuries were of low severity. Although there were high proportions of ankle sprains in hikers and arm fractures in mountain bikers, with one study of the latter reporting more than half suffered head injuries, highlighting the importance of a good quality helmet.
“As the popularity of both pursuits has increased, so too has the standard of trails, bikes, footwear and protective gear, reducing the risk of serious injury.
“In the case of mountain biking there has also been a cultural shift from the more extreme or ‘radical’ style of riding synonymous with the sport when it first evolved decades ago in places like Colorado and California.”
Mr Braybrook said the risk of injury from mountain biking or hiking was outweighed by the considerable benefits.
“Mountain biking and hiking bring economic gains through tourism and the obvious health benefits of physical activity including improvements in cardiovascular health and reducing the risk of high blood pressure, obesity, high blood cholesterol, and type 2 diabetes,” Mr Braybrook said.
“With Spring weather upon us, people should take the opportunity to regularly head out to their nearest trail for a ride or hike — these are fun activities, great for fitness and with only the occasional scratch or bruise likely to result.”

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Genetic factor fends off Alzheimer's and Parkinson's

About one in every five people carries a version of a gene that, although largely unsung, appears to confer protection against both Alzheimer’s disease and Parkinson’s disease, Stanford Medicine investigators and their colleagues have learned. These lucky people may someday benefit all the more from a vaccine that could slow or stall the progression of these two most common neurodegenerative conditions.
An analysis of medical and genetic data from hundreds of thousands of people of diverse ancestries from several continents has revealed that carrying this gene version, or allele, reduced people’s chances of contracting either Parkinson’s or Alzheimer’s by more than 10% on average.
The evidence suggest that a protein called tau, which is notorious for aggregating in the brains of Alzheimer’s patients, may also be involved, in some mysterious way, in the development of Parkinson’s disease.
The findings and implications are described in a paper published online Aug. 29 in the Proceedings of the National Academy of Sciences. Emmanuel Mignot, MD, PhD, the Craig Reynolds Professor in Sleep Medicine and a professor of psychiatry and behavioral sciences, shares senior authorship with Michael Greicius, MD, the Iqbal Farrukh and Asad Jamal Professor and a professor of neurology and neurological sciences, and Jean-Charles Lambert, PhD, director of research for Inserm at the University of Lille in France. Lead authors are Yann Le Guen, PhD, assistant director of computational biology in Stanford Medicine’s quantitative sciences unit; Guo Luo, PhD, an instructor of sleep medicine; former postdoctoral scholar Aditya Ambati, PhD; and Vincent Damotte, PhD, a bioinformatician associated with Lambert’s group.
The protective allele identified in the study is called DR4.
“In an earlier study we’d found that carrying the DR4 allele seemed to protect against Parkinson’s disease,” Mignot said. “Now, we’ve found a similar impact of DR4 on Alzheimer’s disease.”
The Stanford Medicine team combined dozens of medical and genetic databases collected from numerous countries — in Europe, East Asia, the Middle East, and South and North America. All told, the databases included more than 100,000 people with Alzheimer’s disease and more than 40,000 with Parkinson’s disease. The scientists contrasted the incidence and age of onset of Alzheimer’s and Parkinson’s among people with DR4 versus those without it and found a roughly 10% risk reduction in those carrying DR4.

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How to help prevent childhood stunting

A relatively small intervention could have a huge impact on a damaging condition that stalks children in the developing world. A new Stanford-led study shows that adding zinc to farmland soil can help prevent childhood stunting, a condition due to chronic undernutrition that is associated with poor brain development and long-lasting harmful consequences, such as reduced school performance and increased disease risks. The paper, published Aug. 21 in Scientific Reports is the first large-scale study to examine the association between children’s nutritional status or health outcomes and soil mineral availability in India, where more than a third of children under five suffer from stunting.
“Our results add to a growing body of literature suggesting that interventions like micronutrient-enriched fertilizers may have a positive effect on health,” said study lead author Claire Morton, an undergraduate in mathematics and computational science at Stanford University. “This doesn’t prove that those interventions would be cost-effective for India, but it’s an exciting indication that they are worth testing.”
The researchers analyzed health data from nearly 300,000 children and one million women across India with over 27 million soil tests drawn from a nationwide soil health program. They found that that the presence of zinc in soil helps prevent stunted childhood growth, and iron in soil helps keep hemoglobin — a protein in red blood cells that carries oxygen — at healthy levels. The results suggest that fortifying soil with minerals could be a beneficial health intervention.
The link between soil zinc and childhood stunting is particularly robust — a one standard deviation increase in satisfactory soil zinc tests is associated with approximately 11 fewer children stunted per 1,000, according to the research. As a result, the researchers suggest that the potential benefits of using zinc-enriched fertilizers as health interventions deserves more consideration in India specifically and perhaps more generally.
“We’re not saying that geography is destiny, but soils really do seem to play a role in shaping child health,” said study senior author David Lobell, the Gloria and Richard Kushel Director of Stanford’s Center on Food Security and the Environment and professor of Earth system science in the Stanford Doerr School of Sustainability. “Even if this is only a small role, understanding it could help to identify better approaches to solving child stunting in India, which is one of the single biggest and longstanding challenges in global food security.”

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Antibody shows promise for preventing organ rejection after transplantation

A human-made antibody successfully prevented organ rejection when tested in primates that had undergone a kidney transplant, Duke Health researchers report.
The finding clears the way for the new monoclonal antibody to move forward in human clinical trials. Results of the study appear online Aug. 30 in the journal Science Translational Medicine.
“Current medications to prevent organ rejection are good overall, but they have a lot of side effects,” said lead author Imran J. Anwar, M.D., a surgical research fellow in Duke’s Department of Surgery. “These therapies suppress the immune system, putting patients at risk of infections and organ damage, and many cause non-immune complications such as diabetes and high blood pressure.
“The push over the last 30 to 40 years has been to develop new, less toxic drugs,” Anwar said. “We are hopeful this antibody moves us closer to that goal.”
Anwar and colleagues, including co-senior author Allan Kirk, M.D., Ph.D., chair of the Department of Surgery, focused on a monoclonal antibody identified as AT-1501. It was engineered to minimize the risk of blood clots, which had become problematic for an earlier version of this therapy.
In studies using primates that had undergone kidney transplantation, AT-1501 prevented rejection without the need for additional immunosuppressive drugs or promoting blood clots, confirming its immunosuppressive potential.
In animals that had undergone islet transplantation, AT-1501 alone did not lead to uniform rejection control, but it was effective in combination with existing immunosuppressive agents. The combination therapies in islet transplantation led to uniform islet graft survival without weight loss or infections that can typically arise. The islet transplants were performed by Norma Kenyon, Ph.D., co-senior author and professor at the University of Miami.
“These data support AT-1501 as a safe and effective agent to promote both islet and kidney transplant survival and function and allow us to advance into clinical trials right away,” Kirk said. “This less toxic approach has been pursued for over 20 years, and I think we are finally at a turning point. This could be a great advance for people in need of organ transplants.”
In addition to Kirk and Anwar, study authors include Dora M. Berman, Isabel DeLaura, Qimeng Gao, Melissa A. Willman, Allison Miller, Alan Gill, Cindy Gill, Steve Perrin, Camillo Ricordi, Philip Ruiz, Mingqing Song, Joseph M Ladowski, and Norma S. Kenyon.
The study received funding support from the National Institutes of Health (U19-AI051731), the Diabetes Research Institute Foundation, and Anelixis Therapeutics, now Eledon Pharmaceuticals, which is developing AT-1501 for kidney and islet cell transplant.

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