Health Canada Allows Start of DA01 Trial Testing Cell Therapy

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by Diana Campelo Delgado |

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Health Canada

BlueRock Therapeutics has received permission from Health Canada to start a Phase 1 clinical trial evaluating DA01, an investigational cell therapy that aims to restore neurons lost to Parkinson’s disease.

This clearance comes three months after the U.S. Food and Drug Administration (FDA) accepted the company’s request to launch the trial in the United States.

“We’re excited to initiate this study with our expert collaborators at University Health Network in Toronto as we believe this trial could shift the treatment paradigm for PD [Parkinson’s] patients in Canada and around the world,” Emile Nuwaysir, PhD, president and CEO of BlueRock, said in a press release.

DA01 consists of dopamine-producing neurons derived from human embryonic stem cells (ESCs). ESCs, in turn, are derived from undifferentiated cells found in embryos, and are able to grow, or differentiate, into any type of cell needed for a therapy.

This cell therapy was developed to repopulate a patient’s brain with these neurons, and so potentially revert neurodegeneration. The goal is to help patients reclaim lost motor abilities.

“Our therapy is intended to replace the midbrain dopaminergic neurons lost in the degenerative condition to rebuild the neural circuit, and thereby restore motor control to Parkinson’s patients,” Nuwaysir said.

“If successful, this could demonstrate for the first time that the effects of degenerative disease are, in principle, reversible,” he said.

In preclinical studies, these neurons were found to produce and release increasingly large amounts of dopamine over time as they matured, and to ease motor problems in animal models of disease.

The open-label Phase 1 trial (NTC04802733) will test stem cell-derived dopaminergic neurons in 10 Parkinson’s patients, ages 60 to 76, from Canada and the U.S.

In Canada, the trial will be conducted at the University Health Network, in Toronto. In the U.S., recruiting is ongoing at Weill Cornell Medical College, in New York. More information and contacts can be found here.

“We are thrilled to be the first national trial site bringing leading-edge clinical research with life-changing potential to the nearly 8 million patients world-wide who are affected by Parkinson’s,” said Brad Wouters, PhD, executive vice-president of science and research at University Health Network.

The trial will evaluate the safety and tolerability of DA01 one year after patients receive a cell transplant. Secondary goals include the transplanted cells’ ability to survive, changes in patients’ motor abilities at one and two years post-transplant, and continued assessments of safety at two years.

“Exploring the potential for a cell therapy that could improve Parkinson’s rather than simply slowing its progress is a monumental step — few therapeutic approaches to date have been designed to truly restore function,” said Andres Lozano, MD, PhD, the trial’s lead investigator and senior scientist at University Health Network.