September 5, 2019
NBIA Disorders Association Announces Research Grants for Neurodegeneration with Brain Iron Accumulation (NBIA)
About NBIA Disorders Association
The NBIA Disorders Association, formerly known as Hallervorden-Spatz Syndrome Association, (HSSA) was originally founded in 1996 by President, Patricia Wood. The goals of the association are to raise funds to support research pertinent to NBIA; to provide emotional support to those afflicted with NBIA and their families; and to raise public awareness of NBIA. If you would like further information, please email at pwood@NBIAdisorders.org, or telephone at (619) 588-2315.
The NBIA Disorders Association is currently accepting applications for one-year grants for clinical and translational research studies related to the early detection, diagnosis, or treatment of patients with two forms of NBIA: PLA2G6-Associated Neurodegeneration (PLAN), caused by a mutation in the PLA2G6 gene; and Fatty Acid Hydroxylase-associated Neurodegeneration (FAHN), caused by a mutation in the FA2H gene.
Neurodegeneration with Brain Iron Accumulation (NBIA) is a group of rare, genetic, neurological disorders characterized by the accumulation of iron deposits in the brain and progressive degeneration of the nervous system. It typically first appears in childhood. Presenting signs and symptoms may include difficulty walking, loss of balance, and problems related to speech. Those affected suffer a progressive loss of muscle control, sudden involuntary muscle spasms, and uncontrolled tightening of the muscles. Symptoms may also include seizures and deterioration of intellectual ability. At the present time, symptoms for the disorders may be treated but there are no cures.
The purpose of the NBIA Disorders Association Research Grant Program is to encourage meritorious research studies designed to improve the diagnosis or treatment of NBIA. The research can be conducted in the United States, countries of the European Union, Canada, Australia, New Zealand, Brazil, Argentina, Chile, South Africa, Japan, or Israel, and in other countries where adequate supervision of grant administration is possible.
Evaluation of proposals will follow NIH guidelines and include careful consideration of experimental or protocol design, objectivity or relevance of parameters measured, and statistical analysis plan.
Our research priorities reflect our goal to find cures for NBIA disorders and the understanding that strategic work in basic, translational and clinical research will be required to reach this goal. We strive to accelerate the pace of discoveries that lead to new therapeutics by supporting pilot and high-risk projects. The projects we support are expected to generate essential resources for the scientific community, advance knowledge about NBIA disease processes, and produce preliminary data to enable national and international funding to carry the work forward.
Our research priorities for the PLAN and FAHN 2019 Fall Grant Cycle are:
- Develop model organisms that complement existing models, especially for use in high-throughput screening of potential therapeutics
- Discover and validate biomarkers
- Delineate the molecular cascade that leads to early cellular changes
- Develop rational therapeutics, including for gene therapy
- Establish outcome measures to be used in clinical trials
- Develop other essential resources to substantially prepare the patient, family and investigator communities for clinical trials
Information is provided below about existing resources. Please explore these resources further to avoid duplication of effort and to propose how your project might complement or exploit these resources.
- Patient cell repository
- Mouse and fly mutants (published models may be shared)
- Disease natural history study
- Patient cell repository
- Mouse mutants https://www.komp.org/geneinfo.php?geneid=57584 and a published model
- Disease natural history study
- Fly model was unsatisfactory – Scientific Report
A grant not to exceed $45,000 for one year will be awarded by the NBIA Disorders Association for each disorder. Natural history studies must have a component that includes participation in the International NBIA Patient Registry & Biobank.
Research resources, including model organisms developed under this funding initiative must be shared per National Institutes of Health guidelines. These guidelines can be found at http://grants.nih.gov/grants/policy/model_organism . In addition, investigators are encouraged to share novel cell and animal models or technologies that are not yet defined in the NIH guidelines.
- Applications due by November 4, 2019
- Award announcement by February 14, 2020
- Grant funding to begin March 1, 2020
Please see the Grant Application to the right or below (mobile) for further details.
August 23, 2019
The 2019 Million Dollar Bike Ride Pilot Grant Program is now open! The MDBR Pilot Grant Program provides a one-year grant to support research related to a rare disease represented in the 2019 Million Dollar Bike Ride.
This Request for Applications (RFA) is open to the international community. All individuals holding a faculty‐level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation are eligible to respond to this RFA. Letters of Interest (LOIs) are due no later than Monday, September 23, 2019 by 8pm EST. Full proposals are by invitation only and due no later than Thursday, October 24, 2019 by 8 pm EST.
If questions, please contact Samantha Charleston, Assistant Director of Patient Outreach and Program Management, ODC at firstname.lastname@example.org, or (215) 573-6822.
Team NBIA Disorders represented the NBIA Disorders Association at the sixth annual Million Dollar Bike Ride for rare disorders in June. They, along with many BPAN families holding community fundraisers, raised $31,245, for BPAN research and $30,000 was matched by the University of Pennsylvania’s Orphan Disease Center Pilot Grant Program.
One grant of $61,245 will be awarded for Beta-propeller Protein-Associated Neurodegeneration (BPAN).
BPAN RFA Information
BPAN- A Neurodegeneration with Brain Iron Accumulation Disorder: One pilot grant for $61,245 is available for clinical and translational research studies related to the detection, diagnosis, or treatment of this rare, X-linked disorder caused by mutations in WDR45. BPAN typically is recognized in early childhood with delayed development and seizures. In adulthood, people with BPAN develop rapidly progressive parkinsonism. At the present time, symptoms may be treated but there are no cures.
Grants are expected to generate essential resources for the scientific community, advance knowledge about BPAN disease processes, and produce preliminary data to enable national and international funding to carry the work forward. Examples of priority topic areas include developing disease models that complement existing models, identifying biomarkers, delineating the molecular cascade that leads to early cellular changes, developing rational therapeutics, establishing outcome measures to be used in clinical trials, and developing other essential resources to substantially prepare the BPAN community for clinical trials. Natural history studies must have a component that includes participation in the International NBIA Patient Registry & Biobank. This grant is made possible by Team NBIA and BPAN families with the NBIA Disorders Association.
Please see 2019 MDBR RFA for more information.