2022 KDA Grant Proposal Announcement
As of November 2021, the KDA has awarded $1,880,339 in research grants to help find a cure or treatment for Kennedy's Disease. The 2021 recipients are listed below. To see all of the research grants funded, click here.
Because the KDA is relatively small and funding is limited, our focus in recent years has been to provide “seed-money” to post-doc and other young researchers who do not currently have the funding or credentials to receive funding from larger organizations such as the National Institute of Health or the MDA. This “seed-money” normally provides the researcher an opportunity to further his/her research while giving him/her time to apply for other grants
In recent years, the awarding process takes place in the fall. In the late summer, the KDA announces to all known Kennedy’s Disease Researchers that anyone interested should send in their grant requests as outlined in the proposal notification. The Scientific Review Board reviews all applications with a focus on research projects that are specific to or could be used in finding a treatment or cure for Kennedy’s Disease. The Scientific Review Board recommends to the Board of Directors which applicant(s) should receive research funding. The Board of Directors notifies all candidates and awards the grants normally in October.
"I received a pilot grant from the KDA early in my career at a particularly vulnerable time for young scientists, before receiving my first grant from the NIH. The foundation's support made a big impact, helping enable us to generate a mouse model that we continue to study to understand disease mechanisms and therapeutic targets."
Andrew Lieberman, MD PhD
University of Michigan Medical School
In 2021, research grants were awarded totaling $175,000. Award recipients are listed below as well as a link to previous years grant recipients.
Carlo Rinaldi, Oxford University ($75,000 for one year)
We plan to elucidate polyQ AR transcriptional altered activity and its role in SBMA pathogenesis, filling a fundamental gap in the understanding of this disease, and to unravel the therapeutic mechanism of action of AR45, a naturally-occurring AR isoform able to fine-tune AR transcriptional activity. Ultimately our goal is to bring a gene therapy approach based on therapeutic delivery of AR45 into first-in-man clinical testing for SBMA patients.
Shinichiro Yamada, Nagoya University: ($50,000 per year for two years)
Most patients with SBMA experience cold exposure, a worsening of muscle movement under cold temperature, which is caused by muscle membrane hyperexcitability due to abnormal sodium current alteration. Based on the clinical and basic studies, we carried out a placebo-controlled, randomized, double-blind, multicenter, crossover exploratory clinical study of the efficacy and safety of mexiletine hydrochloride, a sodium channel blocker, in SBMA patients. ALSFRS-R which reflect comprehensive motor function and quantitative muscle strength in SBMA patients were tend to be improved in the mexiletine group. Therefore, we will prepare a confirmatory clinical trial with change of ALSFRS-R as the primary endpoint. This study will be the first trial to confirm the efficacy of mexiletine hydrochloride administration in SBMA patients.
In this proposal, we are also planning a biomarker study in parallel to support the results of the clinical trials. We will measure urine titin and serum neurofilament light chain (NfL) as a biomarker of motor neuron degeneration in the patients with SBMA and female carriers in SBMA that may reflect early pathophysiology of SBMA patients.