Weekly reads: stem cells for MS, blood origins, iPSCs

How promising is the idea of stem cells for MS?

I have been working slowly as time permits on a piece more specifically about hematopoietic stem cell transplantation (HSCT) for MS.

Two new pieces caught my eye on this topic so I’ll start with those.

Stem cells for MS

NurOwn Found Safe, Shows Promise in Phase 2 Trial for Progressive MS, Multiple Sclerosis News Today. This is from BrainStorm Cell Therapeutics.

The primary paper on the trial is Evaluation of neurotrophic factor secreting mesenchymal stem cells in progressive multiple sclerosis, Multiple Sclerosis Journal. This is a small, open-label, uncontrolled study so it’s early days.

The good news is the safety profile looks good.

I’m less clear on the potential hints of efficacy here with the designer MSCs.

Look for a possible Phase III study in the future, which would really nail down whether NurOwn is a big deal new treatment.

Note that many unproven stem cell clinics sell MSCs from a variety of sources for MS. Unlike BrainStorm, the clinics aren’t generating good data and they are generally just out to make a quick buck. Watch out for those.

Real-world application of autologous hematopoietic stem cell transplantation in 507 patients with multiple sclerosis, J. Neurol. This is a study from Dr. Richard Burt, who used to run the autoimmune HSCT program at Northwestern. That program was shut down. I have had some major concerns about the Burt Northwestern HSCT program over the years.

The data here are encouraging for a particular type of MS. Stay tuned for a closer look at stem cells for MS generally and a discussion of this paper in a future post.

Other recommended reads

• Independent origins of fetal liver haematopoietic stem and progenitor cells, Nature.
• Dual genome-wide coding and lncRNA screens in neural induction of induced pluripotent stemcells, Cell Genomics.
• Vaccination-based immunotherapy to target profibrotic cells in liver and lung, Cell Stem Cell.
• CRISPR infusion eliminates swelling in those with rare genetic disease, Science. This is from Intellia Therapeutics for hereditary angioedema.
• ISCT statement on Judge Bernal’s flawed ruling on a chain of stem cell clinics here in California.

• Mouse embryo model derived exclusively from embryonic stem cells undergoes neurulation and heart development, Cell Stem Cell. The Zernicka-Goetz team publishes their paper that had been previously discussed as a preprint on embryo modeling with stem cells.