December 1, 2020

The Niche

Knoepfler lab stem cell blog

World Stem Cell Summit 2015: IPS cells, #CRISPR, CAR-T

Mahendra RaoIt’s been a great couple days so far here at the World Stem Cell Summit in Atlanta. You can follow it on Twitter using the #WSCS15 hash tag.

I first attended it 5 years ago in Pasadena. One of the special aspects of WSCS is it brings together diverse stakeholders in a way that just doesn’t happen elsewhere. For instance, you can have the FDA, patients, physicians, scientists, and funding agency people all in one room together.

Some strong impressions so far include the following.

IPS cells. There is a great deal of excitement here about IPS cells of course, but Mahendra Rao providing a more sobering perspective. I’m more optimistic than he is. Here are his six “realities” of IPS cells, which I interpreted as his view of the top challenges.

  • Allogeneic therapy is not going to work unless HLA matched.
  • HLA matching requires a larger number of patients.
  • There is not enough cGMP capacity to make all the lines required.
  • The cost of making a line and the time required to make one make this an expensive therapy and probably not useful for a large number of diseases.
  • Cells may not be mature enough for use even if one could solve all of the above problems.
  • No real model for true autologous therapy where manufacturing is involved (no amortization and no comparability).

CRISPR and human germline modification. I was on a panel yesterday on human germline modification that I enjoyed a great deal. I was on the panel with Aubrey de Grey and Aaron Levine, who both gave fantastic talks. The audience asked spot on probing questions as well. There was some disagreement regarding the appropriate level of urgency for developing policies and/or regulations on human germline modification, but overall my sense was that us three panelists perceive that attempts at CRISPR-based human heritable modification are almost certain to occur in the near future.

There’s real promise for CRISPR-based therapies in the future for rare genetic diseases (not to mention all the great in vitro CRISPR research on humans cells in the lab that should continue), but to get from point A where we are now to point B where we might as a field hypothetically be ready to responsibly use CRISPR in the germline for humans is a long road. Thus, irresponsible clinical attempts in the next few years would be very dangerous and could be harmful to the field and society.

Still, patients in the audience passionately pointed out that in many cases there is a profound need for helpful therapies now and in the immediate future. That “patient drive” as it was termed at the Washington DC meeting last week on human germline modification is powerful and must be part of the discussion.

Innovation in brain cancer treatment including CAR-T. We also heard earlier about transformative approaching such as stem cells loaded with toxins and CAR-T therapies for cancer. Very exciting stuff!

Patients and stem cell clinics. In a session today that I chaired, we heard much more from patients. They articulated better than anyone else could about how difficult it is to get reliable information. How does one know if a certain therapy is legit, safe, and worth getting? Where are sources of information? I’d recommend some of the websites list in the link section of this blog (scroll down the right side of the page).

Dr. Steven A. Davis, co-founder of SymBioSys, Inc. gave a great talk using the example of the Texas stem cell clinic CellTex, now operating in Mexico, as a test case and as a way to introduce the key issues that need discussion more broadly. We then spent a good 45 minutes have questions and discussions. It’s clear that there are many gray areas and difficulties in this arena.

The leader of the Mexican Stem Cell Foundation (unfortunately I didn’t catch his name, but if I find it I will add it in here later) gave some helpful context for understanding where things stand in Mexico. His sense was that the Mexican equivalent of the FDA (COFEPRIS) is not currently equipped to carefully evaluate all the stem cell clinics there (often clustered on the border and catering mainly to American patients). However, he did indicate that his perception was that things are improving.

Overall. I have to thank Bernie Siegel, who is the organizer of this amazing meeting, and his team at the Genetics Policy Institute (GPI). They do a phenomenal job every year on the WSCS.

More talks are ongoing right now including a fascinating one on cord blood and its potential use for neurological disorders….stay tuned.

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