I recently wrote about the upbeat early reports from two allogeneic trials of stem cells for Parkinson’s. Now there’s additional encouraging early news on this front but using patients’ own cells.
This is one of the most promising areas of regenerative medicine.
Stem cells for Parkinson’s: Aspen Neuroscience upbeat early data
California biotech Aspen Neuroscience posted an abstract last week on its autologous stem cells for Parkinson’s trial: Safety, tolerability, and efficacy of intracranial delivery of autologous iPSC-derived dopaminergic precursors in moderate to advanced Parkinson’s Disease, Parkinsonism & Related Disorders.
Like the two allogeneic trials, this one also uses human pluripotent stem cells. IPS cells in this case, which are employed to make the product ANPD001.
At this point the early data from Aspen included in the new abstract show solid safety and also possible hints of efficacy. As with the two other trials, there appear to be no therapy-induced dyskinesias, one of the main possible side effects.
Overall for this area, it will all come down to the data with more patients. I’m optimistic about us seeing one or more approved cell therapies for Parkinson’s within five years.
Kennedy & food safety at the FDA
I’ve written about major reasons for concern about HHS Secretary RFK Jr. in the biologics space. Through his impact on the FDA, he may promote marketing of unproven stem cells and other biologics like peptides. However, the FDA also regulates foods. In this area, Kennedy seems less extreme. For example, food additives need a closer look and more data should be required to back them up before use. Here’s a new item in this area. What Kennedy Gets Right About the Chemicals in Our Food, NYT Times.
Tucked away in this article by Julia Belluz is the implication that researchers Amy Shyer and Alan Rodrigues have seen that common food chemicals alter organoid development: “When added to a cell culture, for example, they found that aspartame altered the way cells collectively formed structures. What that means for human health is worth exploring. It suggests there may be multiple ways chemicals harm us that regulators aren’t currently looking at, Mr. Rodrigues told me.” Could organoids be a useful tool for exploring which chemicals added to our foods might be harmful versus safe?
I don’t see a publication or preprint on this line of research yet, but maybe it’s coming. I avoid aspartame and other artificial sweeteners. The apparent U.S. policy of just generally assuming food additives are safe is the opposite of the EU approach. We also shouldn’t just assume that natural additives like food dyes from natural sources are safe either.
Other recommended reads
- Exercise Boosts Stem Cell Therapy for Parkinson’s,Neuroscience News. This was a rat study, but it’s interesting. Exercise is one of the possible natural stem cell boosts I’ve written about before here on The Niche. I wonder for the pluripotent stem cell-based trials if exercise could impact engraftment or function of the cell therapies.
- US FDA to convene expert panel for Capricor Therapeutics’ DMD cell therapy, Reuters. This is for the product deramiocel. Anyone have a sense what’s going on here? As we’ve discussed before here on The Niche, how well is CBER functioning these days?
- Oncogene aberrations drive medulloblastoma progression, not initiation, Nature. From the study: “To dissect this interplay, we used single-cell technologies (single-nucleus RNA sequencing (snRNA-seq), single-nucleus assay for transposase-accessible chromatin with high-throughput sequencing (snATAC-seq) and spatial transcriptomics) on a cohort of group 3/4 medulloblastoma with known alterations in the oncogenes MYC, MYCN and PRDM6. We show that large-scale chromosomal aberrations are early tumour-initiating events, whereas the single-gene oncogenic events arise late and are typically subclonal, but MYC can become clonal upon disease progression to drive further tumour development and therapy resistance.” Some of my early work as a postdoc provided insights into MYCN’s roles in the normal development of the cerebellum and MYCN functions in medulloblastoma so I found this new study to be interesting. Admittedly, I would have thought MYCN or MYC changes might have occurred earlier, but it’s still possible expression of one or both is elevated early on too in addition to later changes like amplifications.
- FDA clears in vivo blood stem cell editing trial, EndPoints.
Harvard postdoc imprisoned on frog embryos
I Came to Study Aging. Now I’m Trapped in ICE Detention, NYT. This is a painful read. The Russian postdoc who wrote it, Kseniia Petrova, was doing important work and should be freed and allowed to continue. She made a mistake by not declaring some frog embryos in her luggage when she returned to the US from France. This mistake, which in the past probably would have just led to a small fine, does not justify how she’s been essentially imprisoned for the past three months. Making things much worse, the government has now decided to criminally charge Petrova when it seemed a judge might have been just about to order her freed.