July 9, 2020

The Niche

Knoepfler lab stem cell blog

stem cell clinical trials

4 min read

Clinical trials using organoids are still rare and a Clinicaltrials.gov search for such organoid work largely brings up in vitro studies. A new clinical study in Japan where organoid material will actually be transplanted into patients is causing a lot of excitement. Masayo Takahashi (高橋 政代) is a pioneer in stem cell-based clinical trial work and more specifically induced pluripotent stem cell (IPSC)-based trials. Her new clinical study is another groundbreaking step in that it is organoid based, in this case for Retinitis pigmentosa. …Read More

6 min read

Stem cells for autism? After watching this area for many years, I’m not sure there’s any “there there”. There are logical reasons to think that certain stem cells might help many specific diseases, but in my view autism isn’t likely to be one of them. I hope I’m wrong, but today at least in 2018, it’s not looking promising. To be clear, I’m not talking about indirectly using stem cells for non-clinical trial research to provide conceptual and translational insights into autism spectrum disorder, …Read More

3 min read

We heard this week about the encouraging news of a new CRISPR-stem cell collaboration between stem cell biotech ViaCyte and CRISPR Therapeutics. The goal of the team is to make genetically engineered pancreatic derivatives from stem cells that would have a unique immune stealthiness. If such an approach was successful, patients would not need to take immunosuppressive drugs. I followed up with five questions for ViaCyte. Their CEO Paul Laikind sent along some answers that provide new, deeper insights. You can see a cool …Read More

2 min read

One of the most famous living biological scientists, Sir Ian Wilmut, just announced that he has Parkinson’s Disease. I wish him the best in dealing with this illness. Wilmut is very well-known for having cloned the first mammal, Dolly the Sheep. This work followed on the earlier breakthrough by Sir John Gurdon of cloning the first vertebrate with his work in frogs. it just occurred to me: I’m not sure if the first cloned frog had a name! Over the years there has been …Read More