Bemdaneprocel still shows benefits 1.5 years after treatment in trial

Cell-based therapy continued to ease patients' motor symptoms, cut off time

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by Andrea Lobo |

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Use of bemdaneprocel (BRT-DA01), BlueRock Therapeutics’ investigational cell-based therapy, was found to be safe and well tolerated among people with Parkinson’s disease 1.5 years after treatment in a Phase 1 clinical trial.

Moreover, the data also showed both that cells survived in the brain even after patients stopped a one-year immune-suppression regimen, and that the levels of dopamine in the brain increased. Treatment also eased patients’ motor symptoms and reduced off periods — times between doses of a medication when symptoms of Parkinson’s aren’t adequately controlled.

The data were presented at the International Conference on Alzheimer’s and Parkinson’s diseases, being held March 5-9 in Lisbon.

“We are excited to see the continued positive trends in the data from bemdaneprocel’s Phase I trial at 18 months,” Ahmed Enayetallah, BlueRock’s senior vice president and head of development, said in a company press release.

“While it is still early days and there is more work to do, we look forward to further investigating bemdaneprocel in clinical studies,” Enayetallah said.

In fact, the company is now planning a Phase 2 study, which is expected to start enrolling patients later this year.

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Claire Henchcliffe, MD, one of the study’s principal investigators at the University of California, Irvine, said the new results build on positive data seen at the one-year mark.

“It’s exciting that bemdaneprocel met safety and tolerability criteria at 12 months, and now 18-month results suggest that these … cells survive and have potentially positive effects even after discontinuation of immunosuppressants,” Henchcliffe said.

Parkinson’s is caused by the loss of dopaminergic neurons, the nerve cells in the brain responsible for producing the chemical messenger dopamine, which is important for motor control. The loss of these neurons leads to the disease motor symptoms, such as tremors, muscle rigidity, and slowness of movement.

Standard treatments include levodopa and its derivatives, which provide the brain with raw material to produce dopamine. Although these therapies can effectively control symptoms, their efficacy decreases over time, leading to off periods.

Bemdaneprocel is designed to repopulate a patient’s brain with dopaminergic neurons, derived from human embryonic stem cells. These dopaminergic neuron precursors are surgically transplanted into the putamen, a brain region involved in movement control, where they have the potential to rebuild neuronal networks and ease disease symptoms.

We are on the leading edge in the research for new treatment options for Parkinson’s disease as bemdaneprocel, the most clinically advanced pluripotent stem derived cell therapy candidate to date for this disease, continues to show positive trends.

The Phase 1 clinical trial (NCT04802733) tested bemdaneprocel in 12 people with Parkinson’s, all of whom were experiencing off periods while on levodopa treatment. All received the experimental therapy, either at a high dose (2.7 million cells per putamen) or a low dose (0.9 million cells per putamen).

Treatment was shown to be safe and well-tolerated after one year, with no reported serious side effects related to the therapy. Moreover, imaging of patients’ brains revealed the transplanted cells were surviving and producing dopamine — and easing motor symptoms and reducing off time.

Now, data from 18 months or 1.5 years after treatment revealed average scores on the MDS-Unified Parkinson’s Disease Rating Scale Part 3 — a standardized assessment of motor symptom severity — improved by 23 points in individuals treated with the high-dose bemdaneprocel and by 8.6 points in those receiving the lower dose.

Further, the amount of daily off time decreased by 2.7 hours a day in the high-dose group and about 48 minutes a day in the low-dose group. Daily on time, when symptoms are properly controlled, showed corresponding increases.

All trial assessments will continue for up to two years, according to the company.

“We are on the leading edge in the research for new treatment options for Parkinson’s disease as bemdaneprocel, the most clinically advanced pluripotent stem derived cell therapy candidate to date for this disease, continues to show positive trends,” said Christian Rommel, a member of the executive committee of Bayer’s pharmaceuticals division, and the head of research and development.

BlueRock is a wholly-owned, independently-operated subsidiary of Bayer.