The aftermath of Amylyx’s ALS drug failure, the murky meaning of "abortion," & the tests childhood cancer survivors don't get

And, of course, there's always more obesity drug news.

March 11, 2024 Check out STAT+

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By Theresa Gaffney Reporter & Podcast Producer

Morning, all. Several people sent me comments on the pharma ads during the Super Bowl last month (everyone disliked them). Here's what I'd love to know next: What did you think of Eli Lilly's anti-vanity ad campaign that ran during the Oscars last night? Send your thoughts on that and any movie hot takes to theresa.gaffney@statnews.com.

biotech Amylyx's ALS drug failed, but it was already FDA-approved. What now? Amylyx Pharmaceuticals via AP Last week ended with some truly stunning bad news: Amylyx Pharmaceuticals announced that its treatment for amyotrophic lateral sclerosis, called Relyvrio, failed to benefit patients in a large clinical trial. In the Phase 3 study of 664 people with ALS, Relyvrio failed to outperform a placebo on an ALS functional rating scale that measures patients' ability to walk, breathe, speak, and swallow.  STAT's Adam Feuerstein and Damian Garde wrote about some of the major lingering questions that patients, advocates, and researchers have in the wake of the announcement: Will Relyvrio, which was already approved by the FDA, be pulled from the market? Does the FDA need to rethink its "flexible" approach to approving drugs for rare diseases? And how will this setback affect Amylyx? "For all of us, our phones and our emails are on fire, and for good reason," said Sabrina Paganoni, a Massachusetts General Hospital neurologist who served as an investigator in Relyvrio's clinical studies. "This was perhaps the most anticipated event of 2024 for the ALS community, and it didn't yield the results we were hoping for. It's a huge setback." Read more from Adam and Damian on the far-reaching implications of the failure. reproductive health How do we actually define abortion? Abortion rights are positioned to be a major factor in this year's presidential election, the first since the Supreme Court's decision to overturn Roe v. Wade led to tighter abortion restrictions in several states. But across the country, there's widespread disagreement — among doctors, lawmakers, and the public — about what an abortion is. Is it an abortion when a doctor removes a dead fetus or ectopic pregnancy? What if a pregnancy is ended that wasn't known to the doctor treating the patient? The answer depends on whom you ask, reports STAT contributor Saima May Sidik. "You have a medical definition of abortion; you have 50-some-odd legal definitions of abortion; and then you have a bazillion public perceptions of abortion," said OB-GYN Cara Heuser, who practices in Utah, where abortion is banned after 18 weeks. And a study published last month shows that states' definitions just keep getting murkier. Read more on how the word's meaning has shifted over the years, and why it matters now more than ever. first opinion What the NIH needs to do to better include disabled scientists in academia Disabled people are the largest minority in the nation. Yet the representation of disabled principal investigators in academia has declined over recent years, dropping from 2% to 1.3% between 2008 and 2022. In a new First Opinion, two disabled researchers argue that the NIH needs to use its power over the scientific ecosystem to reverse this damaging trend for disabled scientists.  "We experience a unique set of physical and systemic challenges, including inaccessible research facilities and equipment; the inability to join in-person study sections, meetings, symposiums, and social events that build interpersonal relationships that often lead to professional opportunities; implicit biases; crumbling diversity and inclusion policies; and limited support services," write researchers Elizabeth Weaver II and Kiana Jackson. "Navigating these barriers creates an overpowering burden for disabled people entering the field. Without systems in place to increase equity, such as strong DEI, participation from disabled researchers will continue to decline." Read more on what the NIH can do.

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health care Change Healthcare payments should resume by the end of this week Patrick Sison / AP UnitedHealth Group, the parent company of Change Healthcare, said it'll have its electronic payment function up and running by late this week after a cyberattack took many of its systems offline. But its claims network and software will take longer to restore. Since the company announced the attack on Feb. 21, hospitals, pharmacies, and other health care providers have had varying degrees of difficulty getting paid for their services, with some racking up significant claims backlogs. While Change clients wait, UnitedHealth said it strongly recommends they use the workarounds it has established, particularly its new claims submission system. "The prospect of a month or more without a restored Change Healthcare claims system emphasizes the critical need for economic assistance to physicians, including advancing funds to financially stressed medical practices," said Jesse Ehrenfeld, president of the American Medical Association. Read more from STAT's Tara Bannow on when and how UnitedHealth expects to get its systems up and running. obesity revolution Wegovy gets FDA approval to add cardiovascular benefits to its label U.S. regulators approved a label expansion for Novo Nordisk's obesity drug Wegovy to tout its benefits for the heart, a move that could boost demand and insurance coverage for the already highly popular treatment, reports STAT's Elaine Chen. The new label indicates that Wegovy can reduce the risk of major heart complications — including heart attack, stroke, or cardiovascular-related deaths — for people with overweight or obesity and existing heart disease, Novo said in a statement on Friday. Novo said it has also filed for a label expansion in Europe and expects a decision this year. Read more from Elaine on the study results that led to the decision, and what we do (or don't) know about the mechanisms behind the benefit. research Study finds childhood cancer survivors aren't getting the tests they need in adulthood Most adult survivors of childhood cancer are not up to date with their recommended surveillance tests, despite increased risk for certain conditions, per a new study published in the Canadian Medical Association Journal. Among 3,241 survivors in Ontario, Canada, 99% were at elevated risk for cardiomyopathy, 10% for colorectal cancer, and 7% for breast cancer based on treatment they received as children. But only 53%, 13%, and 6% of those groups, respectively, had adhered to recommended surveillance. The researchers noted that the results mirror low proportions of adherence to guidelines in the U.S., despite the fact that health insurance is rarely a barrier there like it is here. They hypothesize that a lack of knowledge about the likelihood of late effects from childhood treatment among survivors and their doctors is a major barrier.

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What we're reading Social factors may 'disproportionately' affect transplant process, Washington Post Medicare announces loan program to aid providers affected by Change cyberattack, STAT "One little glimmer of hope": What IVF means to Mass. cancer patients in wake of Alabama ruling, Boston Globe In surprise move, FDA calls for advisory committee on Lilly's Alzheimer's drug, STAT My anxiety, New Yorker

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Thanks for reading! Until tomorrow — Theresa

theresa.gaffney@statnews.com

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