The FDA has a unique mechanism called RMAT or regenerative medicine advanced therapy designation. It’s intended to move investigational regenerative therapies forward more effectively. RMAT data I recently updated The Niche RMAT list and it has jumped to 85. The FDA has approved many new RMATs this year. The data on how many applications the FDA …
Let’s start this week’s digest with a discussion of clonal expansion and mutations during cell and gene therapy development. Here’s the article that brought this to mind. Sickle cell gene therapy process may cause cancer-linked mutations in blood stem cells, Fierce Biotech. Clonal expansion This is a story about a non-CRISPR gene therapy using a …
BrainStorm Cell Therapeutics is developing Nurown, an investigational therapy intended for treating ALS. I recently posted about BrainStorm and its prospects for providing real hope on stem cells for ALS. I see it as a long shot. The ALS community needs real hope, but one challenge is determining how good clinical data have to be to …
BrainStorm Cell Therapeutics Hail Mary with FDA on Nurown for ALS Read More »
It’s great finding stem cell videos on YouTube that are either excellent research talks or provide important information. I recently found one such video by Shiri Gur-Cohen, which I included below. Interesting data there and Shiri is such a compelling speaker. She also won The Niche image contest one year with a cool microscopy pic …
Weekly stem cell reads: Google Bard AI issues, fat ball reprogramming, BrainStorm on ALS Read More »
From the beginning of the pandemic, I have been mostly skeptical about the idea of cell therapy for COVID including stem cell therapies. However, at the same time, I’ve closely followed the data, especially up until 2022. Now having just had a nasty bout with COVID myself for the first time this summer, I recently …
Update on stem cells & cell therapy for COVID trials Read More »
Imagine regularly having someone rub your face all over with the equivalent of a small roller covered with spikes and doused with either your blood or someone else’s and you have what’s called vampire facials. There’s more news that this is a very bad idea. Vampire facials linked to more HIV cases “Vampire facials” promoted …
Weekly reads: HIV from vampire facials, CAR-T, FDA warns Regenative Labs Read More »
I’m kind of in grant writing hell at the moment, as many of you may have been at various times, but I’m still finding time to read a little on cell therapy and stem cell research. Here are some things that caught my eye as I work on multiple grants with impending deadlines. Clinical trials …
Weekly reads: cell therapy trials, melanoma, β-thalassemia Read More »
I’ve been writing The Niche now for more than 13 years and the idea of stem cells for ALS has been around that whole time. Unfortunately, it has been slow going on this front, but there are still reasons for hope. Probably now much more than ever before. I’ll start with news on one path …
Weekly reads: stem cells for ALS, cell wrinkles, editors quit Read More »
What is the cell therapy for ALS called NurOwn? How strong is its potential as a treatment for ALS? I’ve been following the biotech BrainStorm Cell Therapeutics for many years. Patients keep asking me about NurOwn ALS prospects. They’re looking for hope. Today’s post is a close look at NurOwn and at BrainStorm as a …
Does NurOwn from BrainStorm Cell Therapeutics still provide hope for ALS? Read More »
I’m sure many of you remember He Jiankui, the guy who made CRISPR babies. He ended up serving three years in jail in China. He seems to be trying for something of a comeback since his release. Were He Jiankui invitations a mistake? Some prestigious places have even invited him to give talks, which seemed …
Weekly reads: He Jiankui disappoints, ALS, Vor Biopharma Read More »