Treatment with the cell remedy bemdaneprocel (BRT-DA01) led to reductions in motor symptom severity for folks with Parkinson’s illness in a small scientific trial, in line with knowledge introduced by BlueRock Therapeutics, its developer.
“We are excited to be sharing the results of this Phase I [trial] and look forward to advancing bemdaneprocel to the next stage of clinical testing,” Ahmed Enayetallah senior vice chairman and head of growth at BlueRock, stated in an organization press launch.
Based on the info, BlueRock plans a Phase 2 scientific trial to additional check bemdaneprocel in Parkinson’s and expects to begin enrollment within the first half of subsequent yr.
Parkinson’s is brought on by the progressive dying of cells within the mind liable for making the chemical messenger dopamine.
Standard therapy entails levodopa and its derivatives, which give the mind extra uncooked supplies to make dopamine. These therapies may be efficient for controlling their signs, however most individuals will expertise “off” durations over time, when signs aren’t adequately managed between doses.
With bemdaneprocel, dopamine-making nerve cells are transplanted into the mind by means of a surgical process. The remedy makes use of dopamine-making cells created with stem cells from human embryos.
What had been the outcomes of the Phase 1 trial of bemdaneprocel?
BlueRock sponsored a Phase 1 trial (NCT04802733) to check bemdaneprocel in folks with Parkinson’s. The research enrolled 12 sufferers who had been having off time regardless of levodopa. All had been handled with the cell remedy at both a low or excessive dose.
The research’s principal aim was to check the remedy’s security and the outcomes had been optimistic. At one yr after the remedy’s surgical implantation, no critical unintended effects associated to the therapy occurred. Two critical security occasions had been reported — one seizure associated to the surgical procedure and one case of COVID-19 — however neither was deemed associated to bemdaneprocel.
Imaging of sufferers’ brains confirmed proof the transplanted cells had been surviving and making dopamine as designed. Data additionally indicated bemdaneprocel eased motor signs and decreased off time.
Specifically, at one yr after therapy, common scores on the MDS-Unified Parkinson’s Disease Rating Scale Part 3 — a standardized evaluation of motor symptom severity — improved by 13 factors with high-dose bemdaneprocel and by 7.6 factors with the decrease dose.
Based on the affected person’s diaries, the quantity of every day off time decreased by practically two hours a day within the high-dose group and about 45 minutes a day within the low-dose group. Daily on time, when signs are adequately managed, confirmed corresponding will increase.
“The data from this Phase I open label study are extremely encouraging. While this is a small open-label study, meeting the study’s primary objective for safety and tolerability along with initial improvements seen in clinical outcomes represents a great step forward,” stated Claire Henchcliffe, MD, one of many research’s principal investigators on the University of California, Irvine. “The hope now is that these trends continue and translate into meaningful benefit for people with Parkinson’s disease in controlled clinical trials.”
BlueRock is a completely owned independently operated subsidiary of Bayer.
“The positive outcome of this Phase I clinical trial is a clear step forward and it brings us closer to delivering new treatment options to patients,” stated Christian Rommel, a member of the chief committee of Bayer’s prescription drugs division and head of analysis and growth.
