November 30, 2020

The Niche

Knoepfler lab stem cell blog

CIRM Grantee Meeting on Wednesday March 6: fantastic talks by Drs. Storb & High

I’m here in San Francisco at the annual CIRM Grantee Meeting. As President Alan Trounson said, this is the best stem cell meeting in the world.

We had two speakers tonight to kick off the meeting. Let me tell you my thoughts on each one.

First, we had Rainer Storb from The Fred Hutchinson Cancer Research Center (aka “The Hutch”, where I did my postdoc), who gave his talk “Allogeneic Hematopoietic Cell Therapy for Blood Disorders: the Road to Success”.  It was a fascinating history of the development of bone marrow and hematopoietic stem cell transplantation. It took decades to turn these technology into widely available treatments that have now saved tens if not hundreds of thousands of lives. One thread running through Dr. Storb’s talk that really stuck with me is how a stem cell treatment has a life of its own inside the recipient. It can cause all kinds of reactions such as Graft Versus Host Disease that can be fatal. Scientists need time to learn about these side effects and find creative ways to deal with them.

The second speaker was Katherine High from The University of Pennsylvania whose talk was entitled “Clinical gene therapy comes of age: Lessons from an Eventful Youth”. The audience was also wow’d by this talk. Dr. High gave a history of gene therapy including warts and all. She provided key lessons along the way including the notion that clinical researchers must be humble and learn lessons as they go, not go into things believing they know everything.

Again, as with Dr. Storb’s talk, Dr. High emphasized how treatments can have unexpected consequences and patient safety is a top concern. In the case of gene therapy, a number of patients died in the early days directly from the therapy itself including perhaps most notably Jesse Gelsinger. Other potential side effects include potential germline transmission of transgenic alleles used in the gene therapy via their uptake into gametes of patients. Very sobering.

Dr. High sprinkled her talk with slides about lessons learned. One that stuck out to me that should be taped to the wall of anything doing or contemplating stem cell transplants into patients:

You have to solve problems you are dealt. You cannot plead lack of expertise or interest.

She also pointed out how often studies in animals do NOT correlate with the behavior of treatments including side effects in humans. She said that in a general sense that 30% of the time animal studies on a treatment basically do not work to inform us of what will happen in humans. That’s a frighteningly high number.

Tomorrow looks to be great as well!