Starting tomorrow I’m going to be at the ISSCR 2016 meeting live blogging it in San Francisco.
The annual ISSCR meeting is a great place to hear about all the latest research from basic findings to reports on pre-clinical and clinical studies. There are always valuable and unique insights from chatting with people too.
I plan to report here on the latest science and policy developments including hopefully promising data on the goal of getting new stem cell therapies of various kinds moving from the lab toward helping patients.
Stay tuned.
Here at the clinical translation workshop at ISSCR, we just heard from Masayo Takahashi. The clinical trials for AMD have restarted, using retinal pigment epithelium (RPE) derived from iPSCs with 6 HLA matching the patients- allogeneic, but a close match. The trials are now far more complex, with Kyoto making the iPSCs, Riken making RPEs and transplants taking place at multiple sites.
The reason for the change from autologous iPSCs to allogeneic iPSCs is the availability of iPSC lines that were made at CiRA that have multiple HLA matches with a large segment of the Japanese population. But preclinical autologous iPSC studies are still being done.
Thanks, Jeanne. This is really helpful to hear about this. It’s encouraging that things are firing up again on this trial.
Any mention of the mutation issue such as the specific genomic locations (e.g. certain genes or intergenic?), etc?
Are your efforts going to remain squarely focused on autologous moving ahead?
Paul
Hi Paul- Dr. Takahashi said that the “mutation” was a copy number variation (CNV) in a gene called STS. CNVs are common in the normal population- I have quite a few myself. There wasn’t enough information provided to know whether this CNV was present in the patient’s fibroblasts.
STS is steroid sulfatase (microsomal), isozyme S. There is no link with this gene and cancers…but the concern was that it might affect differentiation or survival of the cells.
Dr. Takahashi sequences all of her iPSCs- we do too. She and I agree that quality control is paramount in development of a therapy- and assessment beyond the minimum required by the FDA is critical to avoid problems.
The switch to allogeneic was motivated by several factors- the major ones were the availability of HLA-matched iPSCs that are good matches for 17% of the Japanese population and the fact that RIKEN was undergoing a social upheaval at the time (STAP).
Japan needs about 140 homologous HLA types to cover almost all the people of Japan. The estimate for the US is 1,000 to 5,000. I’ve only found one homozygous HLA person in our iPSC bank- from Kenya.
So, yes, for the US and the rest of the world, autologous is still necessary to avoid problems with rejection.
Thank you! And please, if you are fortunate enough to find/meet some patients who have benefited from non- approved stem cell treatment, or qualified doctors who are sticking their necks out to save patients…and have…please, won’t you sit down with them in an non-reporter frame of mind…and listen like a “friend”?
“There are more things in heaven and earth, Horatio, than are dreamt of in your philosophy”.
Lynn