Aspen Neuroscience said its experimental Parkinson’s disease treatment, made from a patient’s own cells, showed encouraging results in a small study, reports Endpoints News.
The clinical trial builds on decades of stem cell research and marks an early test of a personalised approach to treating the neurological disease.
In patients with Parkinson’s, neurons die and can no longer make dopamine, which is critical for movement. Aspen converts a patient’s skin cells into stem cells, then turns them into implantable dopamine-producing cells with the goal of replacing neurons.
Competitor BlueRock Therapeutics has reported early clinical data ahead of Aspen. Unlike Aspen, BlueRock relies on donor cells, an off-the-shelf approach that may be faster to manufacture, but that typically requires immunosuppressive drugs to prevent the body from rejecting the therapy.
In an eight-person phase 1/2a study conducted by Aspen, patients showed improvements in standard Parkinson’s measures over one year, such as fewer movement problems and longer periods when symptoms were well-controlled.
On average, patients gained roughly two additional hours per day of good “on time” smooth movement, and motor scores improved by about 13 to 15 points on a commonly used scale.
Brain scans also suggested the transplanted cells survived in the brain. No serious safety concerns were reported, including no major surgical complications or severe side effects.
The results of the trial were shared last at the International Conference on Alzheimer’s and Parkinson’s Diseases. The study was open-label, meaning there was no placebo comparison.
“What’s really beyond expectations is that we’re seeing very significant improvements in quality of life,” Aspen CEO Damien McDevitt told Endpoints News.
McDevitt said he was optimistic that patients would keep improving beyond one year, but it’s too soon to say. Based on the results, Aspen plans to move into a phase 3 study.
Stem cell pioneer Jeanne Loring’s lab at Scripps Research spun out Aspen. Loring, who has persevered through many setbacks, helped advance induced pluripotent stem cells, which are created by reprogramming adult cells into an embryonic-like state.
Aspen’s results could add to the momentum behind using patient-derived cells, also known as autologous cells, in therapies.
Ted Dawson, director of the Johns Hopkins Institute for Cell Engineering, said Aspen’s results so far appear “pretty similar” to BlueRock – though with the advantage of not needing immunosuppressive drugs.
“I’m sure this will cause a lot of excitement. It looks really encouraging,” said Dawson, who was not part of the study. But he added that further clinical data will determine which approach is best and how these experimental therapies stack up against current treatments.
For Aspen, one challenge is the added complexity of personalising therapies for each patient. McDevitt said the company invested five years ago in AI, genomics, robotics and other technologies to scale manufacturing.
“We knew that it is going to be so important to be able to industrialise manufacturing of autologous cell therapy,” McDevitt said.
Endpoints News – Aspen’s personalised Parkinson’s therapy shows early promise (Open access)
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First human trial on stem cell treatment for Parkinson’s
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