Weekly reads: cell therapy trials, melanoma, β-thalassemia

I’m kind of in grant writing hell at the moment, as many of you may have been at various times, but I’m still finding time to read a little on cell therapy and stem cell research. Here are some things that caught my eye as I work on multiple grants with impending deadlines.

Clinical trials including on cell therapy

Stem cells from umbilical cord ‘goo’ delay type 1 diabetes progression, New Scientist. I don’t have a subscription to this publication, but it seems the idea is MSC helping diabetes. The subtitle is “Infusions of stem cells taken from umbilical cord jelly helped people with newly diagnosed type 1 diabetes reduce their need for insulin injections in a small study.” I think this is the publication in question in the journal Diabetologia, “Umbilical cord-derived mesenchymal stromal cells preserve endogenous insulin production in type 1 diabetes: a Phase I/II randomised double-blind placebo-controlled trial.“ The idea here is to invoke an immunomodulatory effect.

The study was small as they noted: “Three participants were included in each dose cohort during the first part of the study. Fifteen participants were randomised in the second part of the study, with ten participants assigned to ProTrans treatment and five to placebo.”

I think cell therapy based on beta cells is more promising than MSCs, but we’ll see how things develop.

Athersys begins enrolment in final cohort of MultiStem trial, Clinical Trials Arena. Athersys could really use some good news. I predicted another bad year for them in my 2023 stem cell predictions, but I hope I’m wrong. This trial will “MultiStem’s safety and effectiveness for the prevention and early treatment of complications after severe traumatic injury.”

Parkinson’s disease drug ropinirole safely slowed the progression of ALS in clinical trial, Medical News Today. Here’s the source research paper in Cell Stem Cell: Phase 1/2a clinical trial in ALS with ropinirole, a drug candidate identified by iPSC drug discovery. This is another small study and had a high attrition rate too. ALS patients and families need more options. Ropinirole works by mimicking aspects of dopamine signaling.

More recommended reads

• Gene-edited and -engineered stem cell platform drives immunotherapy for brain metastatic melanomas, Sci Trans Med.
• Inhibition of FGF23 is a therapeutic strategy to target hematopoietic stem cell niche defects in β-thalassemia, Sci Trans Med.