Our unique breakthrough research
Prevent degeneration, restore function,
reduce symptoms
Lipid storage and lysosomal enzyme deficiencies create massive risk for Parkinson’s disease.
We are testing disease-provoking genetic and age-related changes in enzymes and molecules that control lipid levels inside neurons and glia. Specifically, the so-called GBA enzyme has been found to be altered in both genetic and all forms of age-induced Parkinson’s disease by NRI scientists. NRI scientists have found several key components in pathways of lysosomal and lipid function to be defective in Parkinson’s disease cells and tissues. New research at the NRI aims to elucidate which molecules can solve the lipid problem that causes Parkinson’s disease and some forms of dementia (see Isacson et al., 2019).
Inflammation in the brain prevents function leading to nerve cell degeneration.
The NRI was one of the first research teams in the world to demonstrate scientific findings that showed how the development of inflammation around neurons and glia in the brain results in cell death and loss of brain connection as in Parkinson’s disease, Alzheimer’s disease and other dementias. This research has progressed to a stage where a refined understanding of the inflammatory mechanisms that cause degeneration of the brain can be identified and potentially reversed by novel treatments. NRI has recently begun new projects to prevent nerve cell and glial degeneration caused by inflammatory disarray in the brain. These projects have potential to provide specific and novel therapies for many patients at risk for Parkinson’s disease and dementias (see Deleidi & Isacson 2012).
Cell-based therapies to provide new nerve cells and glia to Parkinson’s disease patients.
By the time symptoms emerge, a patient has lost over 60% of dopamine neurons and synapses due to the disease process. The NRI has developed cell-based restoration by transplantation in Parkinson’s disease, using the patient’s own cells which are reprogrammed into new dopamine neurons. This approach can restore all dopaminergic function lost by the disease. A potentially curative treatment for the well-established loss of dopamine neurons in the midbrain of patients with Parkinson’s disease is underway at the NRI and collaborative clinical institutions. Building on more than three decades of pioneering research in cell therapy for Parkinson's disease (see Osborn et al., 2020), the NRI has translated its discoveries from the laboratory to the clinic. This work has led to a first-of-its-kind Phase 1/2a clinical trial in which patients' own blood cells are reprogrammed into induced pluripotent stem cells (iPSCs) and differentiated into dopamine neurons for transplantation into the brain. The clinical study is evaluating the safety and feasibility of this novel cell replacement approach, as well as its potential to restore dopamine function and improve symptoms in patients with Parkinson's disease.