FDA OKs trial of pig livers as dialysis-like treatment for liver failure
WASHINGTON (AP) — U.S. researchers will soon test whether livers from a gene-edited pig could treat people with sudden liver failure — by temporarily filtering their blood so their own organ can rest and maybe heal.
The first-of-its-kind clinical trial has been cleared by the Food and Drug Administration, according to pig producer eGenesis, which announced the step Tuesday with its partner OrganOx.
An estimated 35,000 people in the U.S. are hospitalized each year when their liver suddenly fails. There are few treatment options and death rates as high as 50%. Many don't qualify for a liver transplant or can't get a match in time.
The new study, which is expected to get underway later this spring, is a twist on the quest for animal-to-human organ transplants. Researchers won't transplant the pig liver but instead will attach it externally to study participants.
The liver is the only organ that can regenerate, but the question is whether having the pig's liver filter the patient's blood for several days could give it that chance.
In experiments with four deceased bodies, that 'bridge' attempt showed the pig liver could support some functions of a human liver for two or three days, said Mike Curtis, CEO of Massachusetts-based eGenesis, which genetically modifies pigs so their organs are more humanlike.
The trial will enroll up to 20 patients in intensive-care units who don't qualify a liver transplant, he said. A device made by Britain's OrganOx, currently used to preserve donated human livers, will pump participants' blood through the pig liver.
It's the latest step in attempts to use gene-edited pig organs to save human lives. Pig kidneys from eGenesis and another pig producer, United Therapeutics, are being used in experimental transplants.
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The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Science and Educational Media Group and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
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Hamilton Spectator
43 minutes ago
- Hamilton Spectator
At least 8 dead in shooting near Israeli and US-supported aid sites in Gaza
KHAN YOUNIS, Gaza Strip (AP) — At least eight Palestinians were killed and dozens more wounded on Sunday in a shooting near Israeli- and U.S.-supported food distribution points in the Gaza Strip, according to health officials. Witnesses blamed the Israeli military, which did not immediately comment. Witnesses said Israeli forces opened fire around dawn toward crowds of desperate Palestinians heading to two aid sites in the southern city of Rafah. Experts and aid workers say Israel's monthslong blockade and military campaign have caused widespread hunger and raised the risk of famine in the population of over 2 million. The vast majority rely on international aid because the offensive has destroyed nearly all of Gaza's capacity to produce food. The war in Gaza rages more than 20 months after Hamas' Oct. 7, 2023, attack, which sparked a chain of events that helped lead to Israel's surprise attack on Iran on Friday. The shooting on Sunday happened close to the sites that are operated by the Gaza Humanitarian Foundation , a group that Israel and the United States hope will replace a system of aid distribution run by the United Nations, which has rejected the initiative, saying it violates humanitarian principles. A man described the distribution as 'a trap' There have been near-daily shootings near the sites since they opened last month. Witnesses say Israeli forces have repeatedly fired on crowds, and health officials say scores have been killed. The military has acknowledged firing warning shots at what it says were suspects approaching its forces. 'There were wounded, dead, and martyrs,' Ahmed al-Masri told The Associated Press on Sunday as he returned from one site empty-handed. 'It's a trap.' Umm Hosni al-Najjar said she joined the crowd heading to the aid point in Rafah's Tal al-Sultan neighborhood around 4:30 a.m. She said the shooting began as people were advancing to the site a few minutes after her arrival. 'There were many wounded and martyrs,' she said. 'No one was able to evacuate them.' The Nasser Hospital in the nearby city of Khan Younis said it received eight bodies after the shooting. Gaza's Health Ministry said Sunday that overall, the bodies of 65 people killed by Israeli strikes or gunfire had been brought to hospitals over the past 24 hours. The aid system rolled out last month has been marred by chaos and violence, while the U.N. system has struggled to deliver food because of Israeli restrictions and a breakdown of law and order, despite Israel loosening a total blockade it imposed from early March to mid-May. UN has criticized the new aid system Israel and the U.S. say Hamas has siphoned aid from the U.N.-run system, while U.N. officials say there is no evidence of systematic diversion. The U.N. says the new system does not meet Gaza's needs, allows Israel to control who gets aid and risks further mass displacement as people move closer to the sites. Two are in the southernmost city of Rafah — now mostly uninhabited — and all three are in Israeli military zones that are off limits to independent media. The Gaza Humanitarian Foundation says there has been no violence in or around the distribution points. It has warned people to stay on designated routes and recently paused delivery to discuss safety measures with the military. Separately, Israel's military body in charge of aid coordination in Gaza, COGAT, said 292 trucks of aid from the U.N. and international community entered Gaza over the past week. About 600 trucks entered per day during the latest ceasefire. Hamas started the war with its Oct. 7 attack on southern Israel as Palestinian militants killed around 1,200 people, mostly civilians, and took another 251 hostage. The militants still hold 53 hostages , fewer than half of them alive, after most of the rest were released in ceasefire agreements or other deals. Israel's military campaign has killed over 55,300 Palestinians , according to Gaza's Health Ministry. It says women and children make up most of the dead but doesn't distinguish between civilians and combatants. Israel says it has killed more than 20,000 militants, without providing evidence. The war has destroyed vast areas of Gaza and displaced around 90% of its population, often multiple times . ___ Magdy reported from Cairo. ___ Follow AP's war coverage at Error! Sorry, there was an error processing your request. There was a problem with the recaptcha. Please try again. You may unsubscribe at any time. By signing up, you agree to our terms of use and privacy policy . This site is protected by reCAPTCHA and the Google privacy policy and terms of service apply. Want more of the latest from us? Sign up for more at our newsletter page .
Yahoo
2 hours ago
- Yahoo
Sarepta Therapeutics Presents Data at the American Society of Gene & Cell Therapy Conference, Including Statistically Significant Functional Outcomes for 8- and 9-Year-Old Patients in New Data Analysis of EMBARK Part 2
Significant functional benefits for 8- and 9-year-olds with Duchenne in Part 2 of the EMBARK study, contributing to the evidence of stabilization or slowing of disease progression in later childhood when muscle weakness typically progresses Statistically significant differences were observed on all key endpoints including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (P=0.0097) compared to a well-matched external control cohort Five abstracts, including two oral presentations at American Society of Gene & Cell Therapy Conference, span Sarepta's portfolio of approved and pipeline therapies across Duchenne and limb-girdle muscular dystrophy CAMBRIDGE, Mass., May 16, 2025--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today presented new data from Part 2 of the EMBARK study that continue to support the clinical benefits of ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy. These data are among other ELEVIDYS data from Sarepta's portfolio presented during the 28th annual meeting of the American Society of Gene & Cell Therapy (ASGCT) Conference. In the recent analysis of Part 2 of the EMBARK study, participants with Duchenne muscular dystrophy who had received a placebo in Part 1 and were aged 8 to 9 years (n=14) at crossover were included. At one year post ELEVIDYS treatment, there were between-group differences (least square means) on all key endpoints that were statistically significant, including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (P=0.0097) compared to a well-matched external control cohort. "The latest data from the EMBARK study highlighting motor function improvements in 8- and 9-year-old boys is encouraging and adds to the growing body of evidence supporting ELEVIDYS," said Aravindhan Veerapandiyan, M.D., Associate Professor of Pediatrics at the University of Arkansas for Medical Sciences and Arkansas Children's Hospital. "What stands out is that these patients were treated at an age when motor decline is typically expected in those with Duchenne. Yet, those who received ELEVIDYS demonstrated statistically significant and clinically meaningful functional improvements compared to external controls." The results presented at ASGCT are from the ongoing analysis of results from Part 2 of EMBARK, which compared two-year outcomes from 63 participants against data from an external control group of untreated individuals with Duchenne. Results at two years post-treatment showed that individuals treated with ELEVIDYS had better outcomes in multiple motor function measures, compared to a well-matched external control group. Additionally, no new safety signals were observed in the EMBARK study over the two-year duration and, in a subset of patients (n=16), micro-dystrophin expression and sarcolemmal localization was sustained from Week 12 to Week 64. "This has been a significant year for our neuromuscular portfolio, with multiple, ongoing analyses and longer-term data on efficacy and safety presented for ELEVIDYS," said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research and development, Sarepta Therapeutics. "Building on the topline EMBARK Part 2 data from earlier this year, we're committed to sharing ongoing analyses as fast as possible. The one-year results of patients treated with ELEVIDYS at 8 to 9 years old provide evidence that those treated with gene therapy outperform those who don't receive it at a critical point when more dramatic functional decline is expected." A full listing of Sarepta's presentations at ASGCT are below. Abstracts can be found at Data from presentations are embargoed until 6:00 AM CT on the presentation day for oral abstracts and until 6:00 AM CT on May 13, 2025 for poster abstracts. Oral Presentations (*Previously presented at MDA 2025 and supplemented with additional data) Title Date, Time Long-term Functional Outcomes and Safety Following Delandistrogene Moxeparvovec Treatment in DMD: EMBARK 2-Year Results* May 164:30 – 4:45 p.m. CSTRoom 393-396 Cardiovascular Investigation of SRP-9005 ( in Non-Human Primates: A Gene Therapy for Limb-Girdle Muscular Dystrophy 2C/R5 May 145 – 5:15 p.m. CSTNew Orleans Theater B Poster Presentations (*Denotes encore presentation) Poster # Title #1350 3-Year Functional Outcomes of Patients with Duchenne Muscular Dystrophy: Pooled Delandistrogene Moxeparvovec Clinical Trial Data vs. External Controls* #1353 Assessment of Cardiac Outcomes in Delandistrogene Moxeparvovec Clinical Trials for Duchenne Muscular Dystrophy* #1422 In Situ Biodistribution and Localization of Bidridistrogene Xeboparvovec (SRP-9003) in LGMD2E/R4 Mice After 1 Year of Follow-up About EMBARK, Study SRP-9001-301 Study SRP-9001-301, also known as EMBARK, is a multinational, phase 3, randomized, two-part crossover, placebo-controlled study of ELEVIDYS in individuals with Duchenne muscular dystrophy between the ages of 4 to 7 years. The primary endpoint is change from baseline in NSAA Total Score at Week 52 following treatment. Eligible participants received a single dose of ELEVIDYS during either Part 1 or Part 2 of the study. In Part 1, participants (n=125) were randomized according to age (≥4 to <8 years) or NSAA Total Score at screening (>16 to <29) and received either 1.33 x1014 vg/kg of ELEVIDYS or placebo with a follow-up period for 52 weeks. In Part 2, participants cross over - meaning, those who were previously treated with placebo in Part 1 receive ELEVIDYS and participants who were previously treated with ELEVIDYS receive placebo, with a follow-up period for 52 weeks. All patients remained blinded through Part 1 and Part 2. Secondary outcome measures in EMBARK include the quantity of micro-dystrophin produced by ELEVIDYS at week 12 (in a subset of participants) as measured by western blot, timed function tests, stride velocity and validated patient reported outcome measures for mobility and upper limb function. One-year results from the Part 1 placebo-controlled period of the EMBARK study were published in Nature Medicine in October 2024 and quantitative muscle MR (magnetic resonance) outcomes from part 1 of EMBARK were published in JAMA Neurology in May 2025. About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). IMPORTANT SAFETY INFORMATION CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate. ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available. Discontinue infusion for anaphylaxis. Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended. Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient's clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. Advise patients to contact a physician immediately if they experience cardiac symptoms. Preexisting Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies. Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia. Report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). For further information, please see the full Prescribing Information. About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at We encourage investors and potential investors to consult our website regularly for important information about us. Forward-Looking Statements This statement contains "forward-looking statements." Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believe," "anticipate," "plan," "expect," "will," "may," "intend," "prepare," "look," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, research and development programs, clinical trials and the potential benefits and risks of ELEVIDYS. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials are positive, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; success in clinical trials does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; we may not be able to comply with all FDA requests in a timely manner or at all; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business; and those risks identified under the heading "Risk Factors" in our most recent Annual Report on Form 10-K for the year ended December 31, 2024 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law. View source version on Contacts Investor Contact: Ian Estepan617-274-4052iestepan@ Media Contacts: Tracy Sorrentino617-301-8566tsorrentino@ Kara Hoeger617-710-3898KHoeger@
San Francisco Chronicle
2 hours ago
- San Francisco Chronicle
Black dads go public with support for their kids with autism -- and each other
ATLANTA (AP) — When Tyrone Green's youngest son was diagnosed with autism, his wife was immediately ready to get the 3-year-old the support he needed. But Green was stuck: He had questions about his son's future and an overwhelming feeling of loneliness — like no one, not his wife, not his friends, understood his experience. ' ... (M)y wife couldn't understand what I was going through as a Black father, all these hopes and dreams I had for my kid," said Green, who lives in Michigan. 'She didn't feel the same way.' In 2021, he joined a Black fathers' support group and met a few other dads eager to discuss their unique challenges. They started their own podcast in 2023 called AutisHIM, a place where Black dads talk about the wins and setbacks of having autistic children. Green is among a growing number of Black fathers of autistic children looking to be more visible in the national autism conversation through podcasts, nonprofits and summits that specifically address their experience. These men say that their hope is not only to be considered more than sidekicks to mothers of the children, but also to help other Black dads accept autism diagnoses and not prolong getting kids the help that they need. Autism is a neurodevelopmental disorder that affects how people communicate, process information and interact with the world around them. Federal data shows that since 2020, Black children have had a higher prevalence of autism spectrum disorder than white children — a change experts credit mostly to better awareness of autism in underserved communities. Health Secretary Robert F. Kennedy Jr. recently announced plans to have the federal government do a broad study for the causes of autism, even though it's been looked at by researchers for decades. He has said autism is a 'tragedy' that 'destroys families' and that some people with autism will never hold a job, pay taxes or go on dates. But many people with autism live successful, socially rich and independent lives, which makes a narrative like Kennedy's dangerous, said Michael Hannon, a counseling professor at Montclair State University who studies the social and emotional aspects of autism on Black fatherhood. It 'can literally diminish hope for any father or father figure or family,' Hannon said of Kennedy's framing of autism. But affinity groups for Black men who have kids with autism are a successful way to get the dads to engage with their emotions, Hannon said. 'The challenge is convincing people to (talk openly and honestly), because the practice of doing that is rare, not just among Black men, but people in general,' he said, adding that people might think it will reflect on their ability to parent. Evan Polk said a big part of navigating his 13-year-old daughter's diagnosis was learning to sit with emotions that weren't simply 'happy and mad.' In the beginning, he was very protective. 'I became a helicopter dad,' said Polk, who started AuSome Kicks, an art therapy nonprofit for autistic children near Philadelphia earlier this year. 'I didn't want nobody or nothing to harm her whatsoever. When I found out she was autistic, she'd be outside with knee pads and elbow pads looking crazy.' He said he later taught his family to be more patient with his daughter, as opposed to traditional parenting styles of being firm and hoping that she would fall in line. Dr. Berry Pierre said he initially was on the sidelines of his autistic daughter's support team as his wife, Maria Davis-Pierre, did the bulk of advocating. The Florida couple founded Autism in Black and for the first five years, he said the organization didn't specifically tailor messaging to Black dads. 'Whether it be in schools, the (individual education plan) meetings, the mothers were just there.' Pierre said. 'But as we started to kind of try to go deeper and figure out 'Alright, what's going on? Where are the guys?' we started to realize that a lot of them will be there.' Many Black dads, Pierre found out, were equally involved as the moms, and Pierre wanted to get more of them talking publicly about autism. 'The dads are there, but we know the general public doesn't realize that yet,' Pierre said. 'So we try to serve as this engine to shine a light on what's really happening. The dads are there, they're attentive. And even with this diagnosis, they're going even harder.' Some dads, like Nicholas Love in North Carolina, said they first hesitated to openly share their journey of raising their kids with autism in fear that people may not understand. 'I was very guarded for a while in talking about my children both being on the spectrum,' said Love, who is CEO of the marketing agency The Kulur Group. 'Even in how you take pictures that you upload on social media, being cognizant and thinking about, 'Well is this a picture that looks, dare I say, the perception of what normal looks like?'" Now, he's an open book about them, is understanding when employees need a little extra time for urgent family needs and has advocated that men receive more paid leave so they will have time to be more involved with their kids. 'I got to a point where it's like, 'OK, this is my reality … I need to do my part in normalizing this," Love said. Green said that while his podcast and platforms like Autism in Black make it easier for Black fathers to share their stories of their kids' wins and losses, he'd like to see 'more support groups out there, more podcasts, more conversations.' 'I see a lot of Black women doing their thing and I highly appreciate that, but I think there definitely needs to be more conversations surrounding (Black fatherhood and autism) because, for myself, I'm a Black man," Green said. "I have a Black family, but this is never really the topic of discussion.' ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Science and Educational Media Group and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
