The BSi unites a cadre of talented Johns Hopkins researchers, neurologists and neuroscientists into a research core focused on neurodegenerative disorders including Alzheimer's disease, amyotrophic lateral sclerosis (ALS), Huntington's disease and Parkinson's disease. The mission of this diverse group is to understand the fundamental causes of these diseases and ultimately to develop biomarkers and effective interventions to mitigate and cure them.
Millions of Americans suffer with Alzheimer disease and related dementias in the United States at any given time, and there is no known cause or cure. The disease progressively robs sufferers of all cognition, and is devastating to affected individuals, their livelihoods, families and friends. The vast majority of dementia patients – approximately 90% – have no known inherited link to the disease.
There are currently no truly effective treatments that substantially alter the course of Alzheimer's disease (AD), despite decades of efforts to find improved medications for this devastating disorder. One reason for this outcome is that the non-human model systems that have been used to evaluate new drugs, prior to testing them in humans, do not adequately capture the essential features of the human disease.
ALS Ice Bucket Challenge video, 2014
The goal of the Answer AD program is to first generate a comprehensive patient derived induced pluripotent stem cell (iPS) core. These true human "biopsies" likely capture critical elements of the disease, so that they may lead to improved treatments for AD and related dementias.
"Answer AD" could grow to be the largest single coordinated and comprehensive effort to end dementia through a highly unique personalized brain health approach.
The mission of Answer AD is to:
Today, the BSi supports internal and external ALS granting programs through the Robert Packard Center for ALS Research. This program has already discovered new drugs for ALS and frontotemporal dementia as well as disease-causing biological pathways and biomarkers.
More recently, two programs were launched to build the most comprehensive clinical, genetic, molecular and biochemical assessment of Alzheimer's disease and ALS. These ongoing expansive programs, known as Answer AD and Answer ALS, are developing an enormous biological dataset and profile of people living with neurodegenerative disorders by generating brain cells from patient blood samples.
This "virtual brain biopsy" will provide a data trove to be mined for disease causes and subgroups and to allow Johns Hopkins scientists to develop targeted therapeutics for Alzheimer's, ALS and other neurodegenerative diseases.