Recommended reads: stem cells for ALS treatment trial, epigenome editing, astronaut mutations, ‘3-person embryo’ DNA

A new stem cells for ALS treatment trial represents a big step forward. It’s early days still but this trial shows feasibility and provides a helpful foundation for moving forward.

It also highlights challenges.

The work is from a team led by Clive Svendsen.

Stem cells for ALS treatment trial
Data from Stem cells for ALS treatment “e, Immunohistochemistry revealed GDNF production in all participants. f,g, H&E staining demonstrated an (f) exophytic mass composed of (g) spindle cells arranged in a haphazard and intersecting fascicular growth pattern. h, Immunohistochemistry showed that the spindle cells were positive for S100, consistent with a Schwann cell origin. i, Neurofilament staining showed numerous punctae and irregularly shaped structures consistent with disorganized and regenerative axons mixed with the Schwann cells. j,k, CD34 staining showed (j) normal-appearing blood vessels within the mass and (k) cells were only infrequently Ki67-positive. Scale bar, 1 mm (e, f); 100 µm (g–k).” Part of Fig. 3. Baloh, et al. Nat. Med 2022.

Stem cells for ALS treatment trial

Here’s the study. Transplantation of human neural progenitor cells secreting GDNF into the spinal cord of patients with ALS: a phase 1/2a trial, Nat. Med. The GDNF-secreting stem and precursor cell product was derived by transduction of human neurospheres with lentivirus.

Neurospheres are cultures of stem and precursor cells. The transplanted cells led to new cell growth and GDNF.

In this trial benign tumors called neuromas were frequently observed, which represents a challenge for this kind of stem cells for ALS treatment approach. Even such benign tumors could be a problem for patients. The data from this trial may help reduce or eliminate the incidence of neuromas in future work.

Recommended reads

$150 Million Gift Takes Stem Cell Research to New Heights, UCSD. Nice new gift from T. Denny Sanford to UCSD for more stem cell research, who has already given around $100 million previously.

Retrospective analysis of somatic mutations and clonal hematopoiesis in astronauts, Comm Bio.  Astronaut mutations were most commonly found in TP53 and DNMT3. Clonal expansion was found in blood cells as well.

Chroma Medicine and Tune Therapeutics: Two companies take up epigenome editing, Nat Biotech.

The stem cell and regenerative medicine field lost a great one this week with the passing of Susan Solomon. I’ve set up a live page of tributes to her so please send me yours or leave a comment and I can paste it in.

Human embryo research

Cancer in children born after frozen-thawed embryo transfer: A cohort study, PLOS Med.

Embryos with DNA from three people develop normally in first safety study, Nature News.This study in China focused on better understanding the safety of spindle transfer in human embryos. They found “mostly normal” development. I’m not sure if that’s encouraging or not. Here’s the PLOS Bio paper. Years ago I was a vocal skeptic of the wisdom of proceeding with what was sometimes called back then “3-person IVF“. The technology has faced many challenges in its various forms. It’s good to see more in vitro data, but I still wonder about the idea of actually using this for human reproduction. In the past, some government officials and scientists tended to ignore concerning data or gaps in knowledge.

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