After 24 months, bemdaneprocel cell therapy by BlueRock Therapeutics is especially effective in higher doses
The prevalence of Parkinson’s disease has doubled in the past 25 years, with an estimated 10 million patients suffering worldwide. This number is expected to double again by 2050, with the biggest driving factor being an increased ageing population.
Several studies have taken place over the years, analysing the causes and potential treatments of Parkinson’s disease.
Earlier this year, John Hopkins University School of Medicine found that a pathological protein long associated with Parkinson’s disease triggers cells to increase protein synthesis, which kills the dopaminergic cells that Parkinson’s patients typically lose.
There have also been drug trials this year researching neuroinflammation, an immune response activated in the development of Parkinson’s and other neurodegenerative diseases.
Most recently, clinical stage cell therapy company BlueRock Therapeutics, a subsidiary of pharmaceutical company Bayer AG, has developed new cell therapy bemdaneprocel for Parkinson’s disease.
Phase one clinical trial exPDite examining the effects of bemdaneprocel has shown positive data after 24 months.
“We are very excited to share the 24-month data from the exPDite trial which shows that bemdaneprocel could be a potentially meaningful treatment option for individuals living with Parkinson’s disease,” said Amit Rakhit, the chief development and medical officer at BlueRock Therapeutics.
“The completion of this study marks an important milestone for bemdaneprocel and sets the stage for the next phase of clinical development.”
The goal of the therapy is to replace the dopamine-producing neurons that are lost in Parkinson’s disease patients.
With a surgical procedure, dopaminergic neuron precursors are implanted in a Parkinson’s patient’s brain. The aim is that the neuron precursors will reform the neural networks that have been impacted by Parkinson’s and restore the patient’s motor and non-motor function.
“There is considerable momentum in the concept of restoring dopamine inputs in the brain using transplanted cells, and the positive results from the exPDite trial leads the drive forward,” said Claire Henchcliffe, MD, the chair of the University of California at Irving Department of Neurology and one of the study’s principal investigators.
“The completed study demonstrates that the transplanted cells survive and there are early signs that bemdaneprocel may potentially help patients to better control their motor symptoms. These are exciting results that warrant further exploration in a next phase placebo-controlled study.”
After one year, the immunosuppression therapy was discontinued as per the study’s protocol, and since then, the transplanted cells have continued to survive and engraft in the brain. This 24-month safety profile is consistent with earlier findings that patients tolerate bemdaneprocel well.
The trial measured several significant Parkinson’s disease symptoms, including motor degeneration, impairment of daily living activities and dyskinesias (uncontrolled, involuntary movements).
Data showed consistent positive trends, with the seven patients receiving a high dose benefitting more than the five patients receiving a low dose. This was assessed using the MDS-Unified Parkinson’s Disease Rating Scale part II and III (MDS-UPDRS part II and III) and the Hauser PD Diary.
Those experiencing motor symptoms in the high dose cohort showed a mean reduction of 21.9 points compared to the baseline, while those in the low dose cohort still showed mild improvement with a mean reduction of 8.3 points.
The amount of time patients spent free from dyskinesias symptoms increased for high cohort patients, who showed a mean increase of 1.8 hours. Low cohort patients showed a mean decrease of 0.8 hours symptom-free, indicating that a high dose of bemdaneprocel was more effective.
Regarding the inability to complete daily living activities, the high dose cohort experienced a mean reduction in difficulties of 3.4 points compared to the baseline, while the low dose cohort showed a mean increase of 2.0 points, again highlighting the effectiveness of the higher dose of bemdaneprocel.
“The continued positive results and encouraging trends in exploratory clinical endpoints for bemdaneprocel after 24 months reinforce Bayer's commitment in developing innovative therapies that can significantly improve patient lives,” said Christian Rommel, the head of research and development at Bayer’s pharmaceuticals division.
Last month, the 24-month data was presented at the International Congress of Parkinson’s Disease and Movement Disorders in Philadelphia.
