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MyTrial: A Clinical Trial Incubator within the Movement Disorders Division and Khurana Lab at BWH
APPROACH IN THE CLINIC: Beyond conventional clinical trials, the Khurana lab works within the Division of Movement Disorders at Brigham and Women’s Hospital to develop innovative new approaches to clinical trials. We select certain patients for “deep phenotyping” – a program we call “MyTrial” – in which we follow the patients’ trajectory with clinical measurements, biometrics and biomarkers in the blood, spinal fluid and with MRI and PET brain imaging. MyTrial currently focuses on patients with three conditions: Parkinson’s disease, Multiple System Atrophy and Spinocerebellar Ataxia Type 3.
The idea is very much in the line with the well-known tenets of precision medicine: matching the right patient with the right drug at the right time. The overarching goal is to track a patient in the clinic and, once we know the natural history, provide that patient with a carefully selected drug.  While this technology is as yet nascent, stunning technological breakthroughs enable us to now conceive of doing this for degenerative brain disorders.
INFRASTRUCTURE: MyTrial utilizes the infrastructure of the Harvard Biomarkers Study (HBS 2.0), a biomarker study house at Brigham and Women’s Hospital that is one of the largest biobanks for Parkinson’s disease and related disorders in the world. The program also benefits from the infrastructure developed within the Brigham and Women’s Movement Disorders Genetics Clinic for obtaining whole-genome sequencing information. A skin biopsy enables us to develop a personalized stem-cell model from each patient.
PERSONALIZED STEM-CELL MODELS: From skin biopsies we, first, generate an induced pluripotent stem cell from the patient. This cell is an embryonic stem cell-like cell that enables us to generate neurons and other CNS cell types from each patient we see. Secondly, from skin (or spinal fluid or even a nasal brushing) we capture the toxic protein (alpha-synuclein, for example) that is aggregating in that specific patient using a technique called the “seeded amplification assay.” This allows us to create a truly personalized model from the patient – capturing the right cell type and the right form of the aggregating protein “in the dish” in our lab. This provides us with a personalized model in which we can test drugs to optimally match them to the right patient. 
“n-of-few” CLINICAL TRIAL APPROACH: Ultimately our approach culminates in the introduction of a drug at the right time.  For patients we actively track in the clinic, we will have the prior clinical and biomarker “history” of the patient, enabling us to better understand the response to that drug. Testing the drug in a patient’s own brain cells in the lab before a clinical trial is attempted should increase our chances of success in the clinic.
GENE THERAPY: Gene therapies: We are working with collaborators to better optimize gene knockdown and editing approaches (e.g. antisense oligonucleotides). We “de-risk” these therapies in stem-cell models matched to patients. The aim is to better understand a therapy before it is introduced into the patient.
For active clinical trials, please see our BWH Movement Division website.
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