Positive Preliminary Results From Australian Parkinson's Trial.
The study, sponsored by ISCO subsidiary Cyto Therapeutics Pty Ltd, is being conducted at the Dept. of Neurology, Royal Melbourne Hospital, Melbourne, VIC Australia.
ISC-hpNSC is a cellular therapeutic comprising human parthenogenetic neural stem cells for the treatment of Parkinson's disease (PD).
Six of the 12 patients in the clinical trial of have now completed one year of follow-up observations and one patient in the low dose cohort has now been followed for two years. There have been no safety signals or serious adverse effects seen to date as related to the transplanted ISC-hpNSC cells.
"Patients followed for one year after cell transplantation have reported improvements in a Parkinson's specific quality of life measure when compared to baseline evaluations. In this context, the results are encouraging that the procedure of transplantation may be well tolerated," said Chief Scientific Officer Russell Kern.
The evaluation is based on more than 12 months of safety data from the first cohort (low dose), and six months of data from the second co-hort (mid dose). Each cohort includes four patients. Dosing of patients in Cohort 3, which is receiving the highest dose, is ongoing.
The Melbourne-based Data Safety Monitoring Board (DSMB) determined that the favorable safety profile of ISC-hpNSC justified proceeding from Cohort 1 to Cohort 2 and Cohort 3.
In terms of preliminary efficacy, where scores are compared against baseline before transplantation, we observed a dose-pendent response.
The percent OFF-Time, which is the time of day when levodopa medication is not performing optimally, and PD symptoms return, decreased an average of 24 percent for the first cohort and 49 percent for the second cohort at six months post-transplantation.
The same was true for percent ON-Time without dyskinesia, which is the time of day when levodopa medication is performing optimally without dyskinesia.
The percent ON-Time increased an average of 19 percent for the first cohort and 33 percent for the second cohort during the same period.
The quality of life of the patients as measured by the Parkinson's Disease Quality of Life Score-39 (PDQ-39) Summary Index, improved 15 percent for the first cohort and 47 percent for the second cohort at six months post-transplantation.
These results should take into account that this Phase 1 safety study (due to its size), lacks the statistical power to generate efficacy conclusions. In addition, there was no placebo control group.
Proper comparisons against a control group will be performed in the Phase 2 clinical trial that we are currently planning.
The Phase 1 clinical study is a dose escalation safety and preliminary efficacy study of ISC-hpNSC, intracranially transplanted into patients with Parkinson's disease.
The open-label, single center, uncontrolled clinical trial will evaluate three different dose regimens of 30 million to 70 million neural cells. Twelve participants with Parkinson's disease will be treated.
Following transplantation, the patients will be monitored for 12 months at specified intervals to evaluate the safety and biologic activity of ISC-hpNSC.
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|Title Annotation:||Clinical Trials|
|Publication:||Stem Cell Lab World|
|Date:||Nov 12, 2018|
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