FDA launched the FDA Rare Neurodegenerative Disease Grant Program when the Accelerating Access to Critical Therapies for ALS was enacted. The agency awards grants and contracts to public and private entities to cover costs of research and development of medical products intended to prevent, diagnose, mitigate, treat or cure ALS and other rare neurodegenerative diseases in adults and children. This program supports research in ALS as well as other rare neurodegenerative diseases.
Neurodegenerative diseases occur when nerve cells in the brain or peripheral nervous system lose function over time and ultimately die. In general, they are incurable and debilitating conditions and are progressive. The term “rare disease or condition” is defined in 21 U.S.C. 360ee and generally they are considered by FDA as diseases with a prevalence of fewer than 200,000 people in the United States or in the case of an acute disease (e.g., less than one year duration), an annual incidence of fewer than 200,000 per year. Some examples of what FDA currently considers rare neurodegenerative diseases include ALS, Cockayne syndrome, ataxia telangiectasia, mucopolysaccharidosis (MPS) type III, familial dysautonomia, and Niemann-Pick Type C.
FDA announced two funding opportunities based on feedback from patients, researchers, nonprofit organizations, companies and other stakeholders on regulatory science research gaps that could advance medical product development.
- RFA-FD-23-008: Natural History and Biomarker Studies of Rare Neurodegenerative Diseases
- RFA-FD-23-030: Systematic Review of Clinical Outcome Assessments (COAs) for Communication Brain-Computer Interface Devices (cBCIs) in Amyotrophic Lateral Sclerosis (ALS)
Applications submitted to these funding opportunities were reviewed for scientific and technical merit by rare disease, natural history and regulatory experts, including representatives from academia, patient groups, the National Institutes of Health (NIH) and the FDA.
The following awards were funded in FY 2023:
- Blackrock Microsystems (Salt Lake City, Utah); Shana Rae Melby; Metrics for brain-controlled communication: A comprehensive review of clinical outcome assessments for communication brain computer interfaces in amyotrophic lateral sclerosis ($500,000 over two years)
- Johns Hopkins University (Baltimore, Maryland); Philip C Wong; Biomarker study in amyotrophic lateral sclerosis (ALS) to develop a diagnostic test for prodromal phase of ALS ($1.6 million over four years)
- Massachusetts General Hospital (Boston, Massachusetts); Alexander V Sherman; Prospective natural history study and biomarker study in familial amyotrophic lateral sclerosis and ultra-rare motor neuron diseases to create a disease-agnostic scalable platform for decentralized observational and validation of digital biomarkers( approximately $1.6 million over four years)
- Massachusetts General Hospital (Boston, Massachusetts); Thurman M Wheeler; Biomarker study in myotonic dystrophy to determine extracellular RNA biomarkers (approximately $1.6 million over four years)
- University Of Illinois at Chicago (Chicago, Illinois); Stephanie M Cologna; Biomarker study in Niemann-Pick type C to determine clinically relevant biomarkers($1.6 million over four years)
- University Of Minnesota (Minneapolis, Minnesota); Pramod Kumar Pisharady; Biomarker study in amyotrophic lateral sclerosis (ALS) to optimize and validate multimodal longitudinal imaging of brain and cervical cord as an ALS disease biomarker using microstructure statistics and morphometry ($1.6 million over four years)
In fiscal year 2022, FDA’s Office of Orphan Products Development (OOPD) funded three natural history studies under the Orphan Products Grants program which serve to meet the intent of the ACT for ALS including for ALS, Myotonic Dystrophy Type 1 and Ataxia-Telangiectasia. The study for ALS, partially funded by the National Institutes of Health (NIH) National Institute of Neurological Disorders and Stroke, has the potential to advance existing knowledge of the natural history of ALS, inform drug development and possibly support future regulatory decisions. The natural history studies include:
- University of Minnesota (Minneapolis, Minnesota); David Walk; Retrospective and prospective study in amyotrophic lateral sclerosis of clinic-based multicenter data collection; $1.6 million over four years
- Johns Hopkins University (Baltimore, Maryland); Howard Lederman; Prospective study in ataxia-telangiectasia; $1.6 million over four years
- Virginia Commonwealth University (Richmond, Virginia); Nicholas Johnson; Prospective study in myotonic dystrophy type-1 to establish biomarkers and clinical endpoints; $1.6 million over four years
FDA also awarded two contracts:
- RTI International, ALS Functional Ratings Scale-Revised Clinical Outcome Assessment Remote-Use Equivalency Study, ~$1.8 million over two years
The ALS Functional Rating Scale-Revised Clinical Outcome Assessment Remote-Use Equivalency Study will adapt a trial endpoint, called the ALSFRS, for remote use. Typically, the ALSFRS is an assessment done by a health care professional in the office. This study will determine whether this assessment could be done at home, decreasing the amount of travel for patients and their families to participate in clinical trials. Additionally, the contract will include a Patient Committee for advice, transparency and oversight and translate the remote tool to U.S. Spanish.
- RTI International, Landscape Analysis of Brain-Computer Interface Focused Patient Preference Studies in ALS Patients, ~$330,000 over one year
The goal of this contract is to perform a landscape analysis of patient preference information (PPI) studies focused on brain-computer interface (BCI) devices. FDA is specifically interested BCI devices that communicate with the brain providing patients with the ability to interact with their families and health care professionals. The contract will review the literature to determine what is already known about BCI devices and PPI studies in ALS. This perspective will help inform FDA’s assessment of the benefit-risk profile for these devices and may lay the foundation for future PPI studies for BCIs in ALS patients.
The FDA remains committed to supporting medical product development for all rare neurodegenerative diseases. The agency is continuing to develop additional grant and contract opportunities to meet the intent of Section 5 of the ACT for ALS. See funding opportunities for FDA’s current grant opportunities for rare disease research.