About 1 million people are living with multiple sclerosis in the U.S. alone with millions of other cases around the world so a huge effort has gone into trying to find new approaches to the disease including stem cells for MS. We’ll start our weekly recommended reads by talking about a new paper on MS and using cells called MSCs to try to tackle it.
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Stem cells for MS
Effects of Mesenchymal Stem Cell Transplantation on Cerebrospinal Fluid Biomarkers in Progressive Multiple Sclerosis, Stem Cells Translational Medicine.
Here’s the Clinicaltrials.gov listing. This is a very short study report tracking Neurofilament light chains (NF-L) as a marker of MS. CXCL13 was also tracked and fit with the pattern of change with NF-L.
This finding is a bit encouraging but we’ll see how the work progress with larger studies.
I’m not sure I get the rationale for why the marrow cells would help other than a possible anti-inflammatory action.
A different recent study linking MS to EBV was very interesting. I wonder if there’s some combo gene-cell therapy approach based on the EBV connection that could be helpful for patients or prevention.
The idea of stem cell therapy for MS has been around a long time with some encouraging results using true bone marrow stem cells. MSCs are more of a mixed bag and only a fraction are usually stem cells. The most promising approach here uses the combo of chemo and a hematopoietic stem cell transplant.
Other recommended reads
- CRISPR’s Nobel Prize winners defeated in key patent claim for genome editor, Science.
- How the Coronavirus Steals the Sense of Smell, NYT. Here’s the original Cell paper with my colleague Qizhi Gong as part of the team: Non-cell-autonomous disruption of nuclear architecture as a potential cause of COVID-19-induced anosmia.
- Myc Supports Self-Renewal of Basal Cells in the Esophageal Epithelium, Stem Cell Reports. This was a great collaborative project that I was part of about Myc’s interesting function and some pluripotency factors in the esophageal basal cells, which are stem cell-like cells.
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An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion, Nat Comm. This comes from Novartis.
- Genetic manipulation of stress pathways can protect stem-cell-derived islets from apoptosis in vitro, Stem Cell Reports.
- Meet the scientist at the center of the covid lab leak controversy, MIT Tech Review. In this piece, the author Jane Qiu spends time in a bat cave.
Thank you for bringing attention to Branaplam….it was given FDA fast-track status for Huntington. I had no idea that small molecules can trigger pseudo exon inclusion…
Here is link to fast track designation