We’re featuring the work of CIRM-Funded Research on World Parkinson’s Day that is focuses on promising ideas to treat patients with this debilitating neurodegenerative disease.
CIRM Grant: Quest Award – Discovery Stage Research
Birgitt Schuele, Parkinson’s Institute
Research: Birgitt is part of a team at the Parkinson’s Institute in Sunnyvale, California. They are using CRISPR gene editing technology to reduce the levels of a toxic protein called alpha synuclein. Alpha synuclein builds up in the dopaminergic brain cells affected by Parkinson’s disease.
“My goal is to help stop Parkinson’s disease progression. If we can develop a drug that clears the accumulated proteins in the brain, we may be able to prevent further cell death. For someone with early‑onset PD who has only mild tremor and minor walking issues, stopping the disease and using a low dose of dopamine therapy to manage symptoms would come close to a cure.”
CIRM Grant: Quest Award – Discovery Stage Research
Jeanne Loring, Scripps Research Institute
Research: Jeanne Loring works with a team at the Scripps Research Institute in La Jolla, California. There they are deriving dopaminergic neurons from the iPSCs of Parkinson’s patients. Their goal is to develop a personalized, stem cell-based therapy for PD.
We are developing a patient‑specific neuron replacement therapy for Parkinson’s disease. By the time PD is diagnosed, patients have already lost more than half of the dopamine neurons in a key brain region, and this loss continues. No current drug can halt this decline or restore the neurons’ function, so replacing the lost dopamine neurons offers the best chance for long‑term symptom relief.
We generate induced pluripotent stem cells from each patient and differentiate them into the exact type of dopamine neuron that has died. Using a patient’s own cells eliminates the need for risky immunosuppressive drugs. We aim to begin treating patients within one to two years, pending FDA approval.”
CIRM Grant: Quest Award – Discovery Stage Research
Justin Cooper-White, Scaled BioLabs Inc.
Research: Justin Cooper-White and his team at Scaled Biolabs in San Francisco are developing a tool that will make clinical-grade dopaminergic neurons from the iPSCs of PD patients in a rapid and cost-effective manner.
Treating Parkinson’s disease with iPSC‑derived dopaminergic neuron transplants has strong scientific and clinical support. However, even the best differentiation protocols are long, complex, and produce dopaminergic neurons with highly variable quality and yield. Scaled Biolabs has built a platform using high‑throughput microfluidics, automation, and deep data to streamline and optimize the entire process from iPSCs to dopaminergic neurons, enabling efficient and rapid transition to GMP‑grade production.
In the first six months of our CIRM‑funded work, we have already accelerated and simplified production of a key intermediate progenitor population, increasing purity from the reported 40–60 percent to more than 90 percent. Our ultimate goal is to deliver dopaminergic neurons to the clinic in a robust, economical way and speed treatments for people with Parkinson’s disease.
CIRM Grant: Basic Biology V
Xinnan Wang, Stanford University
Research: Xinnan Wang and her Stanford University team study how mitochondrial dysfunction affects the brain cells involved in Parkinson’s disease.
“Mitochondria act as the cell’s power plants, supplying nearly all its energy. When aging neurons face stress, damaged mitochondria release reactive oxygen species that poison the cell and trigger neurodegeneration. We hypothesized that Parkinson’s‑mutant neurons have impaired mitochondrial quality control, which drives this degeneration. To test this, we used induced pluripotent stem cell–derived neurons taken directly from Parkinson’s patients.”
