Webinar hosted by the NBIA Disorders Association with organization President Patty Wood, Dr. Suzanne Jackowski of St. Jude Children's Research Hospital in Memphis, and Dr. Arnold Strauss of Cincinnati Children’s Hospital Medical Center.
October 2020
How the NBIA Disorders Association decides which research to fund
By Vanessa Van Oost
When you donate to the NBIA Disorders Association’s research fund, do you know what happens to your money?
For nearly two decades, the association has followed a strict protocol for awarding research grants so it can act as a good steward of every donated dollar. Each proposal for research funding undergoes a rigorous review that includes a series of steps to make sure the science is solid and the researcher is well-equipped to deliver results.
Explaining that process, which may not be well-known among donors, was the purpose of an Aug. 13 webinar the organization hosted to help families better understand grant decision-making. ”We know that easily accessible resources and accurate, reliable information are critical for your loved one,” NBIA Disorders Association President Patricia Wood said. “That is why we thought you would find this webinar on our research grant award process helpful.”
After all, families and their friends raise the bulk of the donations used to fund the organization’s grant program. Since 2002, The NBIA Disorder Association community has raised $2.2 million for research funding, including 41 grants and six contracts aimed at performing a specific task, such as raising a mouse colony. The organization currently has three grants and two contracts in progress, and sufficient funds earmarked to make awards for MPAN, BPAN and PLAN research.
Researchers and physicians serving on the association’s Scientific & Medical Advisory Board (SMAB) as well as outside reviewers when needed, scrutinize each proposal by asking such questions as: Does this research mesh with priorities for treating this disorder, how well-trained is the scientist doing the work and what new information will it add to the existing knowledge?
Two SMAB members participated in the webinar with Wood to provide guidance and answer questions: Dr. Suzanne Jackowski, PhD, a biomedical scientist of St. Jude Children’s Research Hospital and Dr. Arnold Strauss, a pediatric cardiologist at Cincinnati Children’s Hospital.
“The key for funding is that you have strong science. … What new information can be obtained that will help move the field forward and help understand the disorders, and ultimately, develop potential therapies for the disorders?” said Strauss. He has over 40 years of experience in genetic diagnoses of rare diseases and has reviewed grants for many different organizations, including the National Institutes of Health and other rare disease family organizations.
Jackowski has over 40 years of experience in science, research and editorial roles. Some grant proposals are simply “unfundable,” she said, because the feasibility of what they want to accomplish is not evident. This could be due to a lack of understanding of the disease, a lack of experience with the materials of the proposed experiments, or a measurement or biological system that is not appropriate for the study.
Grant proposal reviewers consider the proposal’s relevance, feasibility and scientific merit when scoring projects. Further, they determine whether the investigator is sufficiently trained and equipped to perform the research, with the necessary experience in the method of study and use of biological systems.
“It’s important to know that the proposed research fits into the current knowledge, that the person that proposes that grant has actually reviewed the field and knows what is already well known and proposes something that will extend that knowledge,” Strauss said.
The experiments must have a sufficient number of patients to test a treatment and to assign to a control group, as well as the ability to produce statistically significant results.
The grant review committee led by SMAB members strives to have at least five individuals with various scientific and medical backgrounds. Those varying backgrounds gives the committee the ability to comprehensively evaluate the grant proposals. The scores and recommendations are then sent to the organization’s Board of Trustees to make the final decision on which proposals to fund.
A grant agreement must then be signed, which holds the recipients accountable for their research. The
agreement stipulates that human studies must be approved by the Institutional Review Board (ethics committee), gene therapy studies must provide their NIH Recombinant DNA Advisory Committee Review, and clinical drug trials must meet Food and Drug Administration requirements.
In addition, the final research papers must be made available online following NIH Open Access policies. Any model organisms that were developed must also be shared following NIH guidelines. Grant funds can be forfeited if the investigators do not comply with updating financial and scientific reports or show negligence in the performance of their work.
Wood stated, “We are proud of the guidelines and safeguards that NBIA Disorders Association has put in place to ensure that the funds our families raise for NBIA research are making a difference, and have been responsible for many of the new discoveries in the field.”
Research Program Priorities
- Find cures for NBIA disorders
- Understand strategic work in basic, translational and clinical research.
- Accelerate the pace of discoveries that lead to new therapeutics by supporting pilot and high-risk projects.
- Generate essential resources for the scientific community to advance knowledge about NBIA disease processes
- Produce preliminary data to enable national and international funding to advance work.
