January 15, 2021

The Niche

Trusted stem cell blog & resources

stem cell clinical trials

2 min read

I’ve been closely following the arena of stem cells for COVID-19 since the pandemic broke including the efforts of Mesoblast. Mesoblast unlikely to meet primary endpoints in Phase III COVID trial Now, the firm got bad news as its DSMB reported that they are unlikely to meet the primary endpoint for their Phase III trial of their stem cell product for COVID-19 based on preliminary analyses. Mesoblast had set the endpoint as a “43% reduction in mortality at 30 days for treatment with remestemcel-L.” …Read More

7 min read

IPS cell research is a growing area of promise for regenerative medicine. These stem cells, also known as iPSCs or more formally as Induced Pluripotent Stem Cells, are engineered cells that are programmed to function the same way as embryonic stem cells. They can be differentiated into possibly any type of cell that is needed for therapeutic purposes. Since iPSC production was first reported by Shinya Yamanaka using mouse and human cells in 2006 and 2007, respectively, cells from a whole host of species have …Read More

3 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

4 min read

Multiple sclerosis (MS) can be an extremely debilitating disease that ultimately is fatal in some patients and reduces life expectancy and quality overall substantially. An MS patient named Caroline Wyatt, who works at the BBC, had the courage to open up in full detail on the BBC about having MS, her experience getting an experimental treatment of stem cells in Mexico, and how she is doing now. It’s a powerful new piece that brings home the complexities of having MS and of getting this hopeful, …Read More

4 min read

Is “snake oil” too harsh a term for some of what is sold at some of the worst stem cell clinics? Is the phrase “stem cell clinics” itself accurate and useful in a broad sense? When we read or write the phrase “stem cell clinics”, most people generally know what we’re talking about, but it is not a very precise use of language, especially these days with so many different clinics out there, and there is a lot of room for error. There are …Read More

4 min read

I’ve been blogging about stem cells now for about 8 years and the recent level of hype about and fluffy media coverage of Celularity, the biotech spun out of Celgene, ranks right up here with the most extreme past cases I’ve seen. What is Celularity and who is its leadership? It’s a new biotech focused on placental stem cells with some familiar, well-known leaders including Bob Hariri and Peter Diamandis, and famous tech names on their board including John Scully and Bill Maris, along …Read More

3 min read

A team of researchers at RIKEN and Kobe City Medical Center reported at a press conference today a serious adverse event in a clinical trial participant receiving an induced pluripotent stem cell (IPS cell)-based therapy for vision loss. According to Japan Times and Yahoo! Japan (in Japanese), the patient in question in the IPS cell trial for macular degeneration developed a problem with his eye following the cell transplant. From Yahoo! Japan: “The patient had an operation on Monday to remove pre-retinal membrane, deemed to …Read More