Encouraging Stem Cell Trial Results

[jimcaster]

Top-line Parkinson's Disease Clinical Trial Results

ISCO successfully completed the treatment phase of its dose escalating phase 1 clinical trial (ClinicalTrials.gov Identifier: NCT02452723) evaluating the safety, tolerability, and preliminary efficacy of its lead candidate, ISC-hpNSC® for the treatment of Parkinson's disease (PD). Patients treated with the mid dose (cohort 2) have been observed for at least 36 months and patients in the low dose group (Cohort 1) have been followed for 48 months post transplantation. In terms of preliminary efficacy, where scores are compared against baseline before transplantation, we observed a potential dose-dependent response, with an apparent peak effectiveness at our middle dose. The % OFF-Time, which is the time during the day when levodopa medication is not performing optimally and PD symptoms return, decreased an average 47% from the baseline at 12 months post transplantation in cohort 2. This trend continued through 24 months where the %OFF time in the second cohort dropped by 55% from the initial reading. The same was true for % ON-Time without dyskinesia, which is the time during the day when levodopa medication is performing optimally without dyskinesia. The % ON-Time increased an average of 42% above the initial evaluation at 12 months post-transplantation in the second cohort. The %ON result improved in the second cohort to 65% above the baseline in month 24. The quality of life of the patients as measured by the Parkinson's Disease Quality of Life Score-39 (PDQ-39)Summary Index, improved 43% for the second cohort at twelve months post-transplantation. This improved to a 45% better score in cohort 2 at 48 months.

investors.internationalstem...

[Drummer67]

Thanks Jim, this is good news.I wish they could speed this process up though!!!

[MBAnderson]

Pretty good. Thanks Jim.

[PalmSprings]

Very interesting! Thanks

[Canddy]

Thank you for posting!

[jeffreyn]

Good news but old news. The info in this press release is virtually identical to that contained in their Jun 30 2021 press release (including the typo in the last sentence).

But one important sentence was not repeated: "An extension phase of the study will evaluate patients every 6 months for 5 additional years." My guess is that this is to make really sure that those stem cells don't do something unexpected. So, presumably, they will wait for the "extension phase" to be completed before starting a Phase 2 trial.

[jimcaster in reply to jeffreyn]

Thanks for catching that. I sure hope we aren't another 5 years before Phase 2. Ugh.

[WinnieThePoo]

Stem cells are the big hope but frustratingly slow. The 5 year open label extension does not mean a phase 2 will be delayed until it finishes. It is just a pretty obvious thing to do that you continue to monitor progress / adverse events over an extended period to provide long term results to support any phase 2 trial

But it's been 5 years since they treated the first group already. That standard unit of measure for PD research potential. 5 years is probably the very least time needed for phase 2 results. 10 years to clinic?

First results from this trial was the big news around the time I was diagnosed and I remember Jeanne Loring (due to start trials any day then) being scathing about any positive response being a placebo effect.

I think it's this team that have developed a gdnf hydrogel to use for future transplants.

There seems to be a lot of very promising potential therapies in development, but if they are todays bright idea they might be a clinical option in 15 years time

Unless you are a Caribbean coke snorter😉