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 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. The NRI pioneered the first stem cell-based dopamine transplant recovery in animal models and is now developing the last experiments to provide the final protocol to use in patients with Parkinson’s disease. We were recently awarded a highly competitive award by the NIH for this work (see Osborn et al., 2020).