4:53 PM
Jan 31, 2019
 |  BC Innovations  |  Product R&D

BlueRock’s PD purity play

Why BlueRock’s iPS cells could fare better in Parkinson’s than fetal transplants

Editor's Note: This article was updated on Feb 01, 2019 at 1:18 PM PST

Regenerative medicine company BlueRock is gearing up to submit its first IND this year, banking on its approach to Parkinson’s disease to overcome the legacy of failed fetal transplants by offering a more stable and pure supply of dopaminergic neurons.

BlueRock splashed onto the scene at the end of 2016 when Bayer AG and Versant Ventures teamed up to launch the biotech with a record-setting $225 million series A financing (see “Starting Up Stem Cells”).

BlueRock is using its coffers to build a pipeline of induced pluripotent stem (iPS) cell-based therapies. The goal is to be among the first companies to advance iPS cell technology from research tool to clinical mainstay, grabbing territory in neurology, cardiovascular and autoimmune diseases in the process.

Its lead program is an allogeneic cell therapy designed to restore dopaminergic neurons in PD patients. PD has long been considered a promising target for regenerative medicine. By the time of diagnosis, patients have lost about 80% of their dopamine-producing neurons. SOC therapies help boost residual dopamine signaling but do nothing to prevent the cell loss.

Researchers have been trying since the 1980s to replenish dopamine neurons via transplantation of brain tissue from aborted fetuses, but the technique has not taken off due to ethical concerns, limited efficacy and side effects such as dyskinesias.

BlueRock CEO Emile Nuwaysir considers those trials proof of principal, since some patients showed long-lasting benefit.

In addition to getting around the ethical baggage of fetal tissue, Nuwaysir said adult-derived iPS cells should yield better efficacy and safety. The fetal brain, he argues, is difficult to consistently dissect, which leads to variable numbers of dopaminergic neurons in transplants and contamination from other cell types, including serotonergic neurons, which he said are responsible for producing the dyskinesia.

By contrast, iPS cells can be engineered to produce a pure population of dopamine neurons whose identity and dose can be verified prior to transplantation.

“We are doing something that’s never been done before. You couldn’t put back the authentic midbrain dopaminergic cell until today,” said Nuwaysir.

At least two companies and one academic group are already conducting Phase I trials of iPS or stem cell-based therapies that could replace dopamine neurons in patients, and at least two other companies are in preclinical development (see...

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