Papers in Cerebellum introduce our new clinical trials approach for MSA and other synucleinopathies

Congratulations to Alain Ndayisaba, Ariana Pitaro, Ashlan Willett and Jisoo Kim, along with BWH collaborators Geoff Young, Tarun Singhal and others, on two papers published in Cerebellum.  These papers highlight our new approach to clinical trials for synucleinopathies like PD and MSA. In a process known as “deep phenotyping” we will evaluate our patients with clinical scales, biometrics and PET/MRI imaging in conjunction with genome sequencing and stem-cell degeneration. The idea, described in this video, allows us to understand our patient’s clinical trajectory before they receive a drug in order to determine whether that trajectory is improved after the patient is given a drug. Some patients may respond, others may not. Heterogeneous responses may be missed in conventional randomized clinical trials.