Treatment for Parkinson's using stem cells: Advantages and further details
In the ongoing quest for a cure for Parkinson's disease, stem cell therapy is making significant strides. Currently, clinical trials are in the safety demonstration phase (phase 1), with some trials set to move into the effectiveness proof phase (phase 2) in the near future.
Parkinson's disease is characterised by the destruction of dopaminergic neurons that produce dopamine. This neurological condition, with no current cure, is typically managed with medications such as Levodopa, Selegiline, and Dopamine agonists like Ropinirole or Pramipexole. However, over time, people tend to become less responsive to medication, and these treatments can cause side effects, including serious ones like psychosis.
Stem cell therapy, an experimental treatment, aims to replace the damaged or destroyed dopaminergic neurons in Parkinson's disease. The procedure involves a doctor injecting stem cells directly into the brain, usually in the substantia nigra. These stem cells can be obtained from embryos or reverted from skin and blood cells (pluripotent stem cells).
One of the most promising stem cell therapies for Parkinson's disease is A9-DPC (TED-A9), a stem cell-derived dopaminergic precursor therapy. In a Phase 1/2a trial involving 12 Parkinson’s patients, this therapy showed a consistent positive trend in motor symptom improvement and safety over one year. The trial is expected to complete in 2026.
Another promising development comes from Bemdaneprocel, an embryonic stem cell-derived cell replacement therapy developed by BlueRock. After encouraging early results, this therapy is advancing toward a Phase III trial, which may soon provide stronger evidence for regulatory approval.
Additionally, trials are underway testing the safety and tolerability of an autologous stem cell approach (ANPD001), where patient-derived cells are matured into dopamine-producing neurons. These studies are in the Phase 1/2 stage and currently accept patients by invitation only at several centres, including San Francisco and San Diego.
The general approach of these therapies centers on transplanting dopaminergic neuron precursors to compensate for neuron loss in the substantia nigra, addressing the core motor symptom cause in Parkinson’s disease.
Other related trials focus on brain stimulation or other adjunct therapies rather than stem cells directly, indicating a multifaceted research approach.
As these stem cell therapies progress from early-phase pilot studies showing safety and initial efficacy toward larger pivotal trials, they are becoming one of the most promising emerging treatment modalities for Parkinson’s disease. If successful, stem cell therapy could revolutionise the treatment of Parkinson's disease.
[1] ClinicalTrials.gov Identifier: NCT04024496 [2] BlueRock Therapeutics [3] Neuroscience News [4] Parkinson's News Today [5] The Michael J. Fox Foundation for Parkinson's Research
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