Cell therapy bemdaneprocel gets FDA RMAT status
The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to bemdaneprocel, a cell therapy BlueRock Therapeutics, a subsidiary of Bayer, is developing to treat Parkinson’s disease.
RMAT designation is granted to therapies that have shown potential to treat serious or life-threatening conditions. It provides a series of benefits, including early talks with the FDA about trial design, and potential for priority review of an eventual application seeking approval.
Parkinson’s is caused by the loss of dopaminergic neurons, the nerve cells responsible for producing dopamine, a chemical messenger important for motor control. The loss of dopamine causes the disease’s typical motor symptoms, such as tremors, muscle rigidity, and slowness of movement, as well an nonmotor symptoms.
While levodopa and its derivatives are the mainstay treatment for Parkinson’s by providing cells with the raw material to produce dopamine, these treatments’ efficacy weakens over time.
Therapy designed to generate new neurons
Bemdaneprocel, previously known as BRT-DA01, is designed to generate new dopaminergic neurons after being transplanted into the brain, specifically the putamen, a region involved in motor control. The cell therapy uses human embryonic stem cells to generate dopamine-producing cell precursors. The transplanted cells are expected to restore motor and non-motor function.
A Phase 1 clinical trial (NCT04802733) enrolled 12 people with Parkinson’s all of whom experienced off periods, when symptoms are not fully controlled by levodopa. The experimental cell therapy was transplanted at a low (0.9 million cells per putamen) or high dose (2.7 million cells per putamen).
Data obtained one year after the transplant showed that the cell therapy was safe and well-tolerated, without any serious side effects reported. Brain imaging scans showed that the transplanted cells produced dopamine, leading to an easing of motor symptoms and off time reduction.
These improvements were maintained 1.5 years after treatment, with cells surviving in the brain even after patients stopped a one-year immune-suppression regimen.
June 5, 2024
