The UC San Francisco Memory and Aging Center (MAC) has developed a new type of clinical trial for dementia aiming to accelerate treatments for neurodegenerative disease. By adapting one of the precision medicine approaches that have defined 21st century cancer therapy, the San Francisco-based team introduces “basket trials.”

What are ‘Basket Trials?

Basket trials group together patients with different forms of cancer based on shared genetics or other biological mechanisms. These trials have successfully shown that drugs developed for melanoma, for example, also can work in patients whose lung cancers are caused by the genetic defects, dramatically expanding the number of patients who stand to benefit.

Why Neurodegenerative Therapeutic areas are Comparable?

As it turns out much like cancer trials neurodegenerative trials include some common attributes which can support basket trails. For example, Nicholas Weiler writing for UCSF reports that despite considerable differences between Alzheimer’s with material memory impairments, and frontotemporal dementia, with its eroding of personality and decision-making, these and mother other types of neurodegenerative disease share a common element:  the buildup of one or more types of misfolded protein in different areas of the brain, which leads to the death of brain cells and gradual loss of brain function.

The Study

UCSF MAC published the results of a basket study in the November 11, 2019 issue of JAMA Neurology. It is the first basket trial in neurodegenerative disease, combining multiple forms of dementia believed to be triggered by a common underlying pathology: the toxic buildup of the protein known as tau. 

The study included 94 patients diagnosed with Alzheimer’s disease, progressive supranuclear palsy, and corticobasal syndrome, and assessed the efficacy of a drug that investigators sought could make up for cellular issues triggered by mutated tau.

The Interview

UCSF news interviewed Adam L. Boxer, MD, PhD, the Endowed Professor in Memory and Aging in the UCSF Department of Neurology and director of UCSF’s Neurosciences Clinical Research Unit, and the Alzheimer’s Disease and Frontotemporal Degeneration (FTD) Clinical Trials Program at the UCSF MAC, in regards to the new study and the possibilities of basket trials to accelerate dementia and neurodegenerative disease drug development. Follow the source below to read the entire interview.

Lead Research/Investigator

Adam L. Boxer, MD, PhD, the Endowed Professor in Memory and Aging in the UCSF Department of Neurology and director of UCSF’s Neurosciences Clinical Research Unit, and the Alzheimer’s Disease and Frontotemporal Degeneration (FTD) Clinical Trials Program at the UCSF MAC

Call to Action: Interested in learning more about basket trials in neuroscience? TrialSite News will monitor this topic. Also consider contacting Dr. Boxer at UCSF.

Source: UC San Francisco

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