Rare Neurodegenerative Disease Grants Awarded
FDA Office of Orphan Products Development (OOPD)
Information about awards in previous fiscal years (FYs) is available below. The FDA Rare Neurodegenerative Disease Grant Program aims to equally fund studies to support development of treatments for ALS and other rare neurodegenerative diseases. For current rare disease grant opportunities, including RND grants, see: Current funding opportunities.
FY 2024 Awards
In FY 2024, FDA received 15 grant applications and was able to award one new grant to support development of new retinal imaging (imaging the back of the eye) biomarker(s) in people with a gene defect that can cause Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy (CADASIL). CADASIL is a rare inherited disorder that occurs when the thickening of the walls of small- and medium-sized blood vessels blocks the flow of blood to the brain. People with CADASIL can suffer from multiple strokes, decline in memory and thinking skills, vision problems and seizures. This funded study is examining retinal imaging as potential biomarker(s) of CADASIL. Specifically, it will assess the association of this biomarker with the CADASIL disease manifestations and other non-retinal biomarkers. The findings from this study can inform clinical trials in CADASIL and similar neurodegenerative diseases.
In addition, in FY 2024, FDA provided an additional approximately $4.1 million to several ongoing projects to support studies for ALS and other rare neurodegenerative diseases including Niemann-Pick type C, myotonic dystrophy, and familial dysautonomia. Reference: FY 2024 Request for Applications
| Awardee | Principal Investigator | Description of Project | Amount (approximate) | Duration |
|---|---|---|---|---|
| University of Wisconsin-Madison (Madison, Wisconsin) | Jane Paulsen | Biomarker study in CADASIL to evaluate retinal imaging biomarkers | $5 million | 4 years |
In FY 2023, 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. 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 FDA. Reference: Requests for applications for Natural History and Biomarker Studies of Rare Neurodegenerative Diseases (RFA-FD-23-028) and Systematic Review of Clinical Outcome Assessments (COAs) for Communication Brain-Computer Interface Devices (cBCIs) in ALS (RFA-FD-23-030)
In addition, in FY 2023, FDA provided additional funds to a competing supplement for the ongoing Natural History study in ALS that will allow for longitudinal collection of biosamples, development of a biorepository, longitudinal evaluation of three candidate biomarkers, and whole genome sequencing. FDA also funded a clinical trial (co-funded with the Orphan Products Grants Program) for familial dysautonomia (FD) that has the potential to inform whether this medication can easily be administered at home to treat crises and prevent hospitalizations.
| Awardee | Principal Investigator | Description of Project | Amount (approximate) | Duration |
|---|---|---|---|---|
| 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 (ALS) | $500,000 | 2 years |
| Johns Hopkins University (Baltimore, Maryland) | Philip C Wong | Biomarker study in ALS to develop a diagnostic test for prodromal phase of ALS | $1,600,000 | 4 years |
| Massachusetts General Hospital (Boston, Massachusetts) | Alexander V Sherman | Prospective natural history study and biomarker study in familial ALS and ultra-rare motor neuron diseases to create a disease-agnostic scalable platform for decentralized observational and validation of digital biomarkers | $1,600,000 | 4 years |
| Massachusetts General Hospital (Boston, Massachusetts) | Thurman M Wheeler | Biomarker study in myotonic dystrophy to determine extracellular RNA biomarkers | $1,600,000 | 4 years |
| University Of Illinois at Chicago (Chicago, Illinois) | Stephanie M Cologna | Biomarker study in Niemann-Pick type C to determine clinically relevant biomarkers | $1,600,000 | 4 years |
| University Of Minnesota (Minneapolis, Minnesota) | Pramod Kumar Pisharady | Biomarker study in ALS to optimize and validate multimodal longitudinal imaging of brain and cervical cord as an ALS disease biomarker using microstructure statistics and morphometry | $1,600,000 | 4 years |
| University Of Minnesota (Minneapolis, Minnesota) | David Walk | Retrospective and prospective study in ALS of clinic-based multicenter data collection | $5,800,000 | 4 years |
| New York University School Of Medicine (New York, New York) | Maria Alejandra Gonzalez-Duarte Briseno | Phase 2 study of dexmedetomidine sublingual film for the ambulatory treatment of hyperadrenergic autonomic crisis in patients with familial dysautonomia |
$2,600,000 (co-funded with OGP) |
4 years |
In FY 2022, 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:
| Awardee | Principal Investigator | Description of Project | Amount (approximate) | Duration |
|---|---|---|---|---|
| Johns Hopkins University (Baltimore, Maryland) | Howard Lederman | Prospective study in ataxia-telangiectasia | $1,600,000 | 4 years |
| University of Minnesota (Minneapolis, Minnesota) | David Walk | Retrospective and prospective study in ALS of clinic-based multicenter data collection | $1,600,000 | 4 years |
| Virginia Commonwealth University (Richmond, Virginia) | Nicholas Johnson | Prospective study in myotonic dystrophy type-1 to establish biomarkers and clinical endpoints | $1,600,000 | 4 years |
FDA also awarded two contracts in FY 2022:
| Awardee | Description of Project | Amount (approximate) | Duration |
|---|---|---|---|
| RTI International | ALS Functional Ratings Scale-Revised Clinical Outcome Assessment Remote-Use Equivalency Study | $1,800,000 | 2 years |
| RTI International | Landscape Analysis of Brain-Computer Interface Focused Patient Preference Studies in ALS Patients | $330,000 | 1 year |
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. This contract is partially funded by the National Institutes of Health (NIH) National Institute of Neurological Disorders and Stroke.
The goal of the Landscape Analysis of Brain-Computer Interface Focused Patient Preference Studies in ALS Patients 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.