Funding the Next Generation
2022 accelerators Prize Finalists
Leonard Burg, PhD
Identifying therapeutic leads to
treating Leigh Syndrome
in Zebra Fish
Sara Carli, PhD
Pre-clinical study on
novel treatment strategies
for RRM2B disease
Mitochondria have their own genetic material, the mitochondrial DNA (mtDNA). Having too little mtDNA is one of the causes that drives the onset of Mitochondrial Depletion Syndromes (MDSs). Patients with MDS experience a spectrum of symptoms which generally lead to death in a few months. mtDNA depletion could be due to a defect in the production of a primary building block or gene of the mitochondrial DNA. Currently, there are no animal models present to better understand MDS or to test the effectiveness of therapeutic treatments. This project proposes to create a mouse model that will be used to better understand the progression of MDS. With a mouse model available I will then test the effectiveness of gene therapy, an innovative therapeutic approach to restoring the defective gene that causes MDS.
Filomena Massa, PhD
Fondazione Telethon, Telethon Institute of Genetics and Medicine (TIGEM)
Generation of innovative gene-independent therapeutic strategies for mitochondrial optic neuropathies (MONs)
Our accelerators are engaged philanthropists. Through our annual livestream-pitch event, our members have the opportunity to cast their vote for the project they feel the most passionate about, and ultimately see the difference their contribution makes.
How It Works
UMDF earmarks the accelerators
($50,000 in 2022)
Grant applications submitted by promising post-doctoral fellows
Applications reviewed and finalists selected by UMDF Scientific and Medical Advisory Board
3-5 finalists prepare “fast pitches” to be broadcast live at UMDF Symposium
casts a vote for the project they feel most passionate about
Prize awarded to winner
Right now… a scientist believes his innovation may bring about an end to Leigh syndrome.
Right now… people affected by mitochondrial disease need energy…and YOUR energy can help….but we need to go fast.
It could be an innovation that will find the cause of mitochondrial disease. Or, it may be the research that develops an effective treatment. Now is the time to accelerate that science from bench to bedside. Our patients and families are counting on your energy to help UMDF move faster toward a cure.
When you give $500 or more (cumulatively in a year), you unlock your accelerators benefits! No matter how you give – through a special event, to a designated fund or as a tribute to a patient – when you reach the accelerators level you join a group of engaged philanthropists. You will get a first-hand look at the promising ideas being developed in mitochondrial disease research.
In addition to the opportunity to jumpstart discovery for the next generation of mitochondrial disease researchers, our accelerators receive:
- Name recognition at the annual Symposium
- accelerators lapel pin
- accelerators social media badge
2021 Prize Winner
Lia Mayorga, MD, PhD
2020 Prize Winner
Kinsley Christopher Belle
Our objective is to determine how internal factors, such as development and cell specification cues, as well as external stimulus, oxygen levels, energy substrates, and drug compounds influence mitochondria heteroplasmy. Our preliminary assessments suggest that cell-type development and cell division influence heteroplasmy in developing tissues, additionally our work on cell conditions, and small molecules has yielded promising preliminary results for possible therapeutics. This body of work serves as a template for discovering compounds that reduce mitochondrial heteroplasmy and thus disease burden in patients.
2019 Prize Winner
Ecole Polytechnique Federale de Lausanne (EPFL)
Scientists who are working fast toward a cure.
Vamsi K. Mootha, PhD
HARVARD MEDICAL SCHOOL • Boston, MA
“In 2004, I was a recipient of a $90,200 UMDF grant designed to support my efforts on using computational genomics to identify novel assembly factors for mitochondrial oxidative phosphorylation. With this support, I was able to recruit and hire a talented computational biologist, who proceeded to predict the mitochondrial proteome using a computational tool that led to the identification of several new disease genes, forming the basis for my lab’s first NIH grant.”
Anna-Kaisa Niemi MD, PhD
RADY CHILDREN’S HOSPITAL • San Diego, CA
“A UMDF Clinical Fellowship Award allowed me to focus on diagnosis and treatment of patients with confirmed or suspected mitochondrial disorders. The most impactful part of the fellowship for me was learning about the daily life of children and families affected by mitochondrial disorders. I now continue to use the knowledge and experience I gained that year in my work caring for critically ill infants with confirmed or suspected mitochondrial disorders.”
Michael J. Palladino, PhD
UNIVERSITY OF PITTSBURGH • Pittsburgh, PA
“In 2006, I received a $98,000 research grant from UMDF. This grant funded my research to further develop our Drosophila NARP/MILS model and allow our first venture into compound screening to identify specific drug therapies for mito patients. This award served as “bootstrap funding” helping me successfully apply to the NIH for support of numerous projects and helped secure more than $2.75M in NIH funding for mito research in my lab.”