Phase I/II trial of iPS-cell-derived dopaminergic cells for Parkinson’s disease (Japanese trial)

[Farooqji]

nature.com/articles/s41586-...

[Ctime]

Looks like a robust study and promising news!

[Obiora]

great great great news after some big disappointments

[nednedned]

The study concluded that the transplantation of allogeneic iPS-cell-derived dopaminergic progenitors is safe and has potential clinical benefits for Parkinson's disease.Specifically, the researchers found that the transplanted cells survived, produced dopamine, and did not form tumors.

Primary Endpoint:

* The primary outcome of the trial focused on safety and adverse events.

* There were no serious adverse events reported.

* The majority of the 73 adverse events recorded were mild, with only one moderate case of dyskinesia.

Secondary Endpoint:

* The secondary outcomes assessed motor symptom changes and dopamine production over 24 months.

* Four out of six patients showed improvement in motor function during the off-time period, as measured by the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part III.

* Five patients showed improvement in the MDS-UPDRS part III ON scores.

* The average changes for all six patients were -9.5 points (20.4%) for the OFF scores and -4.3 points (35.7%) for the ON scores.

* Four patients showed improvement in Hoehn-Yahr stages.

* There was a 44.7% increase in Fluorine-18-L-dihydroxyphenylalanine (18F-DOPA) influx rate constant (K₁) values in the putamen, with higher increases observed in the high-dose group.

[Farooqji]

Data from the phase 1 exPDite clinical trial, published in Nature today, demonstrated that injections of dopamine-producing neurons derived from human embryonic stem cells was not only safe 18 months after treatment, but also that some participants patients "experienced visible reductions in tremors," the journal review noted. Results of second trial of human-induced pluripotent stem cells conducted in Japan reported similar results.

ucihealth.org/about-us/news...