For the first time, a stem cell replacement therapy has been found to be safe in a stage 1 clinical trial of patients living with Parkinson’s disease, drug developer BlueRock Therapeutics announced Wednesday.

Stem cell therapy has recently gained interest as a treatment for Parkinson’s, in which patients lose nerve cells in the brain that produce dopamine, causing motor and non-motor symptoms that can disrupt daily life. The treatment surgically implants stem cells in the brain with the idea that they may become new, dopamine-producing nerve cells over time, potentially helping to restore patients’ motor and non-motor function.

The current study findings provided evidence that Bluerock’s stem cell therapy, called bemdaneprocel, was well-tolerated in all 12 study participants, with no major safety events, the company reported. Investigators also found evidence that the transplanted cells had survived and engrafted in the brain through one year. 

With positive phase 1 results in hand, the company intends to proceed with a phase 2 trial to test the therapy’s effectiveness in restoring lost function. The findings mark “a very important step in the development of a potential new therapy for patients with this disease,” said Ahmed Enayetallah, senior VP and head of development at BlueRock, a Bayer subsidiary. 

Medications can help patients with Parkinson’s relieve symptoms by replacing dopamine or reducing the loss of dopamine, but tend to lose efficacy over time and can cause side effects. There currently are no available treatments that can stop, slow or reverse disease progression.

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