Clinical Results

Clinical improvements observed in people with Parkinson’s disease receiving therapy

Oryon, in collaboration with expertise across Mass General Brigham led by the Neuroregeneration Institute at McLean Hospital, is evaluating its autologous dopaminergic neuron replacement therapy in an ongoing Phase 1/2a clinical study in people with Parkinson’s disease.

The first cohort of participants with Parkinson’s disease received neuronal implants in one side of the brain (unilateral implants). The data show improvements in motor function, together with neuroimaging evidence consistent with restoration of dopaminergic signaling in the transplanted brain region.

Key findings

  • Well-tolerated
  • No serious adverse events to date
  • Early and meaningful motor improvement in all participants
  • Continued improvement beyond six months in all participants
  • Imaging evidence of increased dopaminergic signaling on the implanted side of the brain, that aligned with functional improvements
  • Improvement in the Parkinson’s Disease Questionnaire-39 (PDQ-39), a self-report questionnaire designed to measure the health-related quality of life for individuals with Parkinson’s disease
  • No need for immunosuppression

Interim results from the first five participants showed substantial improvements in motor function. A sixth participant had not yet had their first post-implant assessment.

In each of the first five participants, OFF-state MDS-UPDRS Part III motor scores decreased by approximately 29%–62% from baseline at assessments 6–18 months after treatment. Improvements were observed in core Parkinson’s motor symptoms, including bradykinesia, rigidity, and gait.

Most study participants also showed improvements in additional functional measures, including mobility assessments such as Timed Up and Go. Several also experienced reductions in levodopa equivalent daily dose (LEDD), indicating a reduced need for dopamine-replacement medication.

Neuroimaging biomarker shows dopaminergic synapse restoration

Oryon Brain Scan Image
Dopamine transporter DaT-SPECT imaging demonstrated increased dopaminergic signal in transplanted brain regions. In one participant, dopaminergic signal in the transplanted putamen (the brain region most directly implicated in Parkinson’s motor symptoms) increased more than five-fold at six months, compared with baseline, while the untreated side of the brain showed a decrease in signal. Image credit: Courtesy of Dr. Penny Hallett

The clinical data presented are interim findings from an ongoing Phase 1/2a study and are preliminary in nature. Results are based on a limited number of participants and may change as additional data are collected. Oryon’s investigational therapy has not been approved by any regulatory authority, and its safety and efficacy have not been established.

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