When I was talking with my mom the other day, she asked me about how things are going with stem cells for vision loss. Fortunately, her vision remains fine, but we both know people dealing with macular degeneration. Fortunately, there’s some more hopeful news in this space so let’s start there. Then we’ll get to the more complicated stuff.
Hopeful stem cells for vision loss trial results
Here’s the encouraging news: Safety and tolerability of RPESC-RPE transplantation in patients with dry age-related macular degeneration: Low-dose clinical outcomes, Cell Stem Cell. This is an exciting adult stem cell-based trial article from a great team. I’m still enthusiastic about pluripotent stem cell-based approaches to vision loss too, but it’s always better to have multiple approaches in the pipeline. From this paper:
“In conclusion, interim results from the clinical study of RPESC-RPE-4W replacement therapy for dry AMD met the primary endpoints: lack of significant inflammation, lack of tumor formation, and lack of IP-related SAEs. BCVA showed clinically meaningful gains in the worse-seeing subjects. The positive safety and tolerability found for cohort 1 have enabled progress to mid-dose cohort 2 RPESC-RPE-4W therapy for dry AMD.”
I’m excited to see more data from this work in the future.
Child’s death in brain gene therapy trial shakes up field
Child’s sudden death unnerves a promising area of gene therapy research. Trial was a first test of viruses engineered to get past the blood-brain barrier, STAT News. One of the more sobering elements of this must-read article is the table of recent deaths in gene therapy trials. The different potential mechanisms behind the deaths adds complexity here. Overall, gene therapy and clinical use of genome editing (overlapping areas) remain extremely promising, but I don’t know if the public realizes that some trial participant deaths in this large clinical research space are almost unavoidable. We have to take such outcomes very seriously but not overreact.
NFL team doc on false promises at cell therapy clinics
New York Giants team doctor: Clinics offering ‘miracle injections,’ ‘next generation cell therapies’ are selling false hope, STAT News. This is an interesting read from a doctor, Scott Rodeo, who’s both a researcher and the New York Giants team physician. It carries extra weight that he’s treated many NFL players, some who has also received unproven stem cells and other procedures at clinics. Dr. Rodeo has probably seen it all.
More recommended reads
- F.D.A. Turmoil Keeps Spotlight on Its Commissioner, NYT. This piece from Christina Jewett provides some new insights into the chaos at the FDA. It seems like Makary’s position is more precarious than I might have thought.
- H2AK119ub dynamics control hair follicle stem cell quiescence, Nat. Comm.
- ALS and the market for false hope, Science Based Medicine. Of course, some of the false hope comes from supposed stem cell treatments.