After the good news of Friday that a stem cells for type 1 diabetes therapy appears to be effective in a small trial, we should also note that a second teenager has died after receiving Sarepta’s Elevidys gene therapy for Duchenne muscular dystrophy. The cause of death again was liver failure. Sarepta has limited shipments after this event and there will be more analysis of the situation.
Another death after receiving Sarepta’s Elevidys
Liver toxicities are not unusual with AAV-based gene therapies more generally. Zolgensma is another example. It’s not fully understood why, but seems to involve immune responses to the virus.
When something is both risky and can be beneficial. Another challenge is keeping things in perspective when a therapy has both proven benefits but also clear, serious risks including possibly death. Especially when a disease has no other meaningful treatments, some patients may be up for taking the risks. What do you think?
FDA approval of Sarepta‘s Elevidys was controversial. This death just raises the pressure here. At a more political level, it’s notable that current FDA CBER Director Vinay Prasad had criticized Elevydis.
Now that he’s both the biologics leader and has been appointed to other FDA leadership roles, could Prasad take steps that limit established and investigational gene therapies? I’m also curious how he will approach emerging cell therapies. At the recent FDA Cell & Gene Therapy roundtable hosted by Prasad, the emphasis was more on speed than extensive trialing.
Update: Prasad fired Verdun
As to FDA speed or the lack thereof, placing the FDA’s Nicole Verdun on leave (appears to be effectively a firing), which was reportedly Prasad’s decision, could slow CBER way down or lead to mistakes inside the agency when less experienced people assess candidate therapies. Or if Prasad relies too much on AI, decisions could be too fast or slow or just erroneous.
STAT had more on Friday on Verdun’s firing by Prasad, which could have impact on agency evaluation of Capricor’s deramiocel, also for Duchenne. Deramiocel is a cell therapy approach specifically for cardiac muscle injury associated with this form of muscular dystrophy.
For context, Verdun was opposed to the Elevidys approval, which got the nod from Peter Marks anyway.
Here’s my updated full list of FDA-approved cell and gene therapies.
More reads
- Even full fetal personhood shouldn’t end abortion rights, STAT News. I’ve written many times about personhood, the idea that even one-cell embryos are people just like you and me. Personhood makes no scientific or even common sense to me.
- ESC models of autism with copy-number variations reveal cell-type-specific translational vulnerability. Cell Genomics.
- Lilly to acquire Verve Therapeutics to advance one-time treatments for people with high cardiovascular risk, Lilly. I’ve regularly covered Verve here.
Human-animal chimeras
- Mice with human cells developed using ‘game-changing’ technique Human cells injected into amniotic fluid find their way into fetal mouse organs, Nature News.
- Tiny human hearts grown in pig embryos for the first time The hearts started to beat in the pig–human hybrids, which survived for 21 days, Nature News.
These two newsy items from Nature report on talks given at the annual ISSCR meeting. So there are no papers out on these yet.