What are the most significant stem cell and regenerative medicine good news stories of 2024?
Since it has been an eventful year, there are many possibilities.
Please see our poll at the bottom of this post and weigh in on what you think is the most important encouraging news. If you don’t see your choice let us know in the comments.
Top stem cell & regenerative medicine good news stories
Here are brief notes on the top candidate events in the poll.
Patients start to receive combo cell-gene therapy for sickle cell
Last year saw European and FDA approval of new combination cell-gene therapies for sickle cell disease. The FDA-approved products, Casgevy and Lyfgenia, have shown clear benefits in trials. This year patients have started receiving both new sickle cell therapies and so far they are reportedly doing very well.
As more people get these therapies, we’ll likely hear about some side effects, probably major ones in some cases. Note that Lygenia got the most serious type of FDA side effect warning called a black box warning because some blood cancers arose in the patients getting it during trials.
Promising research on stem cells for type 1 diabetes
Work by Vertex continues to look encouraging on cell therapy for Type 1 Diabetes. Vertex acquired the two other major players in this space, ViaCyte and Semma, and continues trial work using stem cell-derived pancreatic cells.
Stem cells for Parkinson’s trial starts
Aspen Neurosciences has now started its trial of iPS cell-based cell therapy for Parkinson’s Disease. The trial by Aspen, a biotech founded by Jeanne Loring, provides hope for a novel Parkinson’s therapy. Other trial work on cell therapies for Parkinson’s is also ongoing as well, further boosting hope.
Capricor encouraging Duchenne cardiac data
Capricor has some encouraging data on its cardiosphere product for Duchenne Muscular Dystrophy. They might even get FDA approval in 2025 given current agency trends although more data could be needed including from their newer HOPE-3 trial.
New investigational iPS-cell-based vision therapy looks exciting
Researchers in Japan used an iPS cell-based approach to partially restore vision in patients. The work was published in The Lancet. The treatment is for limbal stem-cell deficiency or LSCD. Hopefully longer-term studies of more patients will continue to look promising.
FDA steps up its game in 2024 on unproven cell & tissue therapies
The CBER branch of the FDA has issued 12 warning letters to sponsors so far in 2024. It’s hard to say what 2025 will bring but I see this increased FDA activity as a very positive development. Why is this good news? This is positive news for patients and the legitimate stem cell research field as it’s crucial to differentiate between legitimate stem cell research and risky clinics.
FDA wins big federal appeals case
The FDA prevailed at the 9th Circuit Court of Appeals on a key stem cell case. This ruling, jiving with an 11th Circuit Court ruling on a highly similar Florida case by the DOJ, solidifies the FDA’s authority to regulate some cell products like SVF as drugs. As with the increased FDA activity, this is a win for patients and the stem cell research field. Patients and the public are not always clear on what is good stem cell clinical science.
Poll on most important good news stories
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