How do genetic variants disrupt brain cells and cause abnormal movements and memory loss? Can addressing these molecular glitches before disease advances lead to better outcomes?
The new Precision Neurology Program (PNP) at Brigham and Women’s Hospital (BWH) and Harvard Medical School addresses these key questions.
Researchers in the program, based in the BWH Department of Neurology, are drawing upon the full spectrum of basic research, clinical work, and discovery of targeted treatment for movement and memory disorders.
“Instead of treating patients based on clinical symptoms, we are on a mission of identifying and targeting the disease driver in each patient,” says Clemens R. Scherzer, MD, director of the Precision Neurology Program, and head of the Neurogenomics Laboratory at BWH. “We believe that breaking down the silos of biology, clinic, and computation will lead to a quantum leap for neurology.”
The PNP will focus initially on Parkinson’s and expand to related movement and memory diseases including Lewy body dementia and multiple system atrophy. Its goals are to transform clinical trials and drug discovery and to create a “Google” for Parkinson’s disease to match targeted drugs with a patient’s genome.
A backbone of the work is the Harvard Biomarker Study and the Partners Biobank repositories. These biosources include longitudinal and molecular data on more than 2,700 people with neurodegenerative diseases with DNA, RNA, plasma, cryopreserved cells for on-demand production of personal stem cells, and in subsets, cerebrospinal fluid and brain banking.
The program’s components at the outset also include:
- human brain cell and stem cell platforms to rapidly translate human genetics into drug targets and clinical trials in a dish (including the work of investigators Tracy Young-Pearse, PhD, and Vik Khurana, MD, PhD, associate director of the PNP)
- a computational neurology team that applies machine learning to torrents of digital, genomic and other omics data generated from patients to predict and prevent their disease progression
- population-wide drug screening investigation in partnership with the University of Bergen (Norway) to repurpose approved drugs for Parkinson’s and other neurologic diseases
- a biomarkers incubator designed to speed up the biomarker test cycle from years to weeks.
Launched through philanthropic funding, the PNP is located in the Hale Building for Transformative Medicine at BWH, and involves physicians and scientists working in the Ann Romney Center for Neurologic Diseases, the Department of Neurology, and the precision medicine initiative. “We partner with like-minded investigators in the Harvard system and in biotech,” says Dr. Scherzer.
“Our goal is to transform reactive care into predictive, proactive, and personalized neurology,” Dr. Scherzer says. “We have a head start in Parkinson’s disease, and are working on pinpointing biomarkers and tailored drugs that would be useful for patients with the same specific disease driver, but manifesting with different movement and memory disorders.”