Cell therapy has eased Parkinson’s motor symptoms in the first few participants in a clinical trial, according to new company data.
The data also indicate the treatment appears safe and may restore lost nerve function, according to a company press release.
Penelope Hallett, sponsor of the trial and co-director of Mass General Brigham’s Neuroregeneration Research Institute, presented the data at the AD/PD 2026 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders, held from 17 to 21 March in Copenhagen.
Parkinson’s is caused by the death and degeneration of dopaminergic neurons, specialised nerve cells that produce dopamine, a signalling molecule that helps control movement. The loss of these cells disrupts dopamine signalling and leads to Parkinson’s symptoms.
The approach involves collecting blood cells from patients and growing them into induced pluripotent stem cells, or iPSCs, which are lab-generated cells that can develop into other cell types. The iPSCs then undergo a series of biological manipulations to grow into dopaminergic neurons, which can be implanted into the brain to replace lost cells and restore dopamine signalling.
In the ongoing clinical trial, NCT06422208, six participants have so far received the cell therapy into one side of the putamen, a brain region that usually houses many dopaminergic neurons and is heavily affected in Parkinson’s. Oryon said the therapy has so far been tolerated well, and no serious safety issues have been reported.
Five study participants have undergone post-transplant assessments. Data generally indicated improvements in motor function. Motor scores on the Unified Parkinson’s Disease Rating Scale, assessed when patients were off other Parkinson’s medications, improved by about 29 to 62 per cent from the start of the study to assessments carried out six to 18 months after treatment.
The company said improvements were observed in core Parkinson’s motor symptoms, including bradykinesia, or slowness, rigidity and gait. Some patients also showed improvements in other functional measures, and some were able to reduce their daily dose of levodopa, a standard Parkinson’s treatment that can help ease symptoms but can also cause uncontrolled movements as a side effect.
Imaging data from the patients suggested an increase in dopamine activity in the treated side of the putamen, whereas the untreated side tended to show a decrease in dopamine signalling consistent with the typical progression of Parkinson’s.
“These results provide encouraging evidence that replacing dopaminergic neurons may restore biological function in Parkinson’s disease,” Hallett said. “The alignment of clinical improvements, imaging evidence of dopaminergic activity, and reduced medication use suggests that the implanted neurons are integrating into the brain’s circuitry and helping restore function lost to the disease.”
The trial, which is enrolling by invitation at a site in Boston, will soon begin testing in a new group of patients who will receive the experimental cell therapy into both sides of the putamen.
“We are encouraged by these data from the first cohort in this clinical trial who received unilateral neuronal implants,” said Nikola Kojic, MD, PhD, co-founder and chief innovation officer at Oryon. “In collaboration with the team at Mass General Brigham, we expect to begin bilateral [both-side] implantations in a second cohort this quarter to test the hypothesis that outcomes will improve even further compared to those seen with unilateral [one-side] implants.”
Oryon recently announced it had raised US$21m in financing, which the company expects will help support the completion of this early trial and fund preparations for further clinical testing. Ole Isacson, MD, PhD, company co-founder, said Oryon’s recent milestones “reflect the tremendous progress we have made in moving this technology from the lab to the clinic, and will allow us to accelerate it through its next stages of development.”
