Weekly reads: death in CRISPR trial, sickle cell, nose picking & dementia, epilepsy

As both a scientist who works on stem cells, cancer, and CRISPR, and a research advocate I’ve been fortunate to meet many patient advocates over the years. Some have been participants in clinical trials themselves.

CRISPR gene editing has major therapeutic potential but there are risks too.

Benefits & risks of clinical trial participation

It is very sobering to find out that a clinical trial participant has died. I remember when ALS advocate Ted Harada passed away from a brain tumor. I had gotten to know Ted reasonably well over the years and his death was a shock. He had been a super patient advocate and participated in the Neuralstem trial. 

In that trial, neural stem cells were tested as an approach to ALS. While the trial didn’t pan out overall, Ted felt that the intervention had made a major positive difference for him. When he later passed away from a brain tumor, frankly my first thought was a worry that the injected stem cells could have contributed to that. My understanding later was that Neuralstem checked (genetic assay?) and the tumor was unrelated to the experimental ALS therapy. Still, injections of stem cells have risks including tumor formation. Fortunately, so far, tumors have been extremely rare in cell therapy clinical trials.

Overall, trial participants are putting themselves at some risk, often to possibly benefit other people in the future from knowledge gained. They are real heroes.

I’m going to start today’s recommended reads with a piece on the death of a CRISPR trial participant.

Recommended reads

Death in CRISPR gene therapy study sparks search for answers, AP. The participant who died was Terry Horgan, who had Duchenne muscular dystrophy. Unfortunately, nearly zero is known about what happened in this case.  For instance, it’s unclear if the gene therapy had any role in his death. I hope there can be more information provided as this is analyzed further. Since no information has been released so far, there is no way to gauge any potential impact on the field or other specific CRISPR trials.

Gene therapy education materials for the sickle cell disease community, NIH Human Genome Research Institute.

Can Picking Your Nose Really Cause Dementia? What to Know About the Trending Study, Prevention. The answer at this point is “no” and those promoting the idea are way out of line. In my view there’s some major hype here to try tie in the nose picking connection. Oof. Here’s the original mouse paper. Very little there to justify speculation about people.

In Defense of Basic Science, Caltech letters. It’s odd how almost nobody talks about basic science anymore.

Trial of Stem Cell-derived Therapy for Parkinson’s to Open in Sweden, Parkinson’s News Today. This is from a team led by Malin Parmar. See this resource too: Parkinson’s disease stem cell therapy research update.

OHSU second in country to perform regenerative brain cell therapy procedure, Mirage News. The idea here is to use inhibitory cells to lessen excess neuron activity in the brains of people with refractory epilepsy. It’s a cool hypothesis.

Safety and tolerability of bosutinib in patients with amyotrophic lateral sclerosis (iDReAM study): A multicentre, open-label, dose-escalation phase 1 trial, eClinicalMedicine. This study was based on work that made use of iPS cell-based drug screening.

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