Looking for some stem cell good news? You’ll like today’s post.
One mission of this blog The Niche is to promote rigorous science-based regenerative medicine, which can lead to investigating and writing about not-so-upbeat stuff. Risky clinics. People getting hurt. Patient lawsuits. Serious FDA, FTC, or state AG regulatory developments. Such actions can be good news in the sense that they are needed but they still aren’t necessarily upbeat.
However, there are more positive and encouraging developments too. Let’s go through some of the hopeful and exciting news.
Regenerative medicine & stem cell good news on clinical trials: real hope
- Parkinson’s Disease. I recently wrote about how Aspen Neurosciences had its first trial participant. There’s a long way to go, but this is encouraging news. Autologous iPS cell-based therapy for Parkinson’s makes so much sense.
- Spina Bifida. The MSC-based trial for spina bifida continues here at UC Davis. I’m not part of that study and it’s still ongoing. However, I feel hopeful about how it might turn out. The trial is led by Drs. Diana Farmer and Aijun Wang. The idea is to use placental mesenchymal cells as a protective cover for the developing spine.
- GvHD. Mesoblast got the nod to move forward with its BLA on cell therapy for GvHD. The FDA had been tough on this front for a long time so perhaps additional encouraging data pushed this into a positive direction on the oversight front.
- HSCT for some forms of MS. The general trend is very encouraging on autologous HSCT for some types of MS like RRMS.
- Epilepsy. Neurona Therapeutics has some encouraging trial data on its investigational cell therapy for epilepsy. While this is another situation of it being early days and the number of participants is small so far, some of them had very positive potential results. There’s a solid chance this is going to work for many people. Neurona also got some very positive coverage from Antonio Regalado over at MIT Tech Review.
- Sickle cell disease and B-thalassemia. Some of the best recent news was the approvals of gene and cell-based drugs for sickle cell disease and B-thalassemia. I believe Casgevy in particular provides so much hope. Realistically, because of the price tag, we’re going to need more affordable and widely available options, but this is a good starting point.
Other encouraging regenerative and stem cell news
The recent news on gene-edited pig organ transplants provides solid hope for a new future source of organ transplants. While there’s still a lot to do to make this a reality at a wider scale and more could go wrong, I see the latest steps as quite encouraging.
Concrete applications of AI in biomedicine are going to transform aspects of medicine and research including regenerative medicine. Yes, AI has been majorly hyped overall, but in some areas there is reason for excitement. Data suggest numerous AI applications are going to be a big deal in our field. Even just predictive technologies for health conditions look very promising.
RNAs as medicines. COVID vaccines were just one big application but RNAs as medicines looks like a larger, very exciting field. I should note that RNAs need to be proven to be effective and safe in strong trials before clinical use. I’ve been concerned about so-called supportive oligonucleotide therapies or SOT, which are already widely sold at clinics in the US with little robust trial data to back it up.
Did I miss any other good news stories in our field? Let us know in the comments.
Thanks, Paul. I still can’t quite believe that my dream of a neuron replacement therapy for Parkinson’s disease has come true!
Thanks for balancing out the skepticism with good news!
Are the stem cell therapies being done at the Riordan Institute legit?