Small positive step for BlueRock Therapeutics Stem Cell Therapy for Parkinson’s

Several groups including Viviane Tabar who is a founding scientist at BlueRock Therapeutics are moving forward on potential cell therapies for Parkinson’s Disease.

BlueRock just released some new data that’s worth a look. Check out the new BlueRock poster. In today’s post, I briefly discuss the new data.

I also go into the larger context of what this means.

BlueRock Therapeutics, Parkinson's disease, brain imaging
BlueRock Therapeutics trial brain imaging showing DOPA uptake levels (orange = increase and blue = decrease) in the striatum after introduction of Bemdaneprocel. It’s not clear from the poster what more specifically are the 4 brain images. Image from company poster.

BlueRock Therapeutics phase 1 trial results

Twelve trial participants received one of two doses of Bemdaneprocel or BRDT-DA01. The BlueRock cell therapy consists of pluripotent stem cell-derived dopaminergic neurons. The source cells are human embryonic stem cells.

How does this work? A surgeon implants Bemdaneprocel into the brain where the cells are hoped to take the place of lost dopaminergic neurons. The study reported a year of follow-up with a good safety profile overall.

There also were some indications of stabilization of Parkinson’s Disease in the participants. This evaluation of efficacy is based on defining off and on states, where symptoms are not controlled or are better controlled, respectively. The higher dose of cells seemed to show potential signs of improvement in symptoms without causing dyskinesias. The lower dose did not clearly help.

Looking ahead on stem cell therapy for Parkinson’s

This was a small, open-label study without controls so we have to be cautious. Still, the results are encouraging in that they reportedly showed both a good safety profile and signals of possible efficacy. Investigational cell therapies for Parkinson’s have the potential to cause new dyskinesias so overall signs of improvement of Parkinson’s symptoms here are hopeful for the higher dose.

As to the future, it’ll be important to see this trial’s results in a peer-reviewed publication. We’ll also need to closely follow the Phase 2 study.

Despite the limitations, this seems like a small but positive step forward for BlueRock Therapeutics and the field.

I’m excited to see trial results from other groups. A variety of approaches are being used including autologous cells.

4 thoughts on “Small positive step for BlueRock Therapeutics Stem Cell Therapy for Parkinson’s”

  1. Thank you for keeping us updated on this fascinating and rapidly progressing field.

    I was wonder Is anyone doing autologous ipsc derived cells? For parkinsons or for other things?

    Thank you again

    Thomas Ichim

        1. Thanks, Tom. It’s been a long road. We just got clearance for a Phase 1/2a trial of the first* autologous therapy for Parkinson’s disease…no immunosuppression necessary. *There was a single patient trial using this approach, but Aspen is planning a multicenter trial for multiple patients. 5 years after we started the company…including the 2 pandemic years, so 3, really!

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