(Alexandria, VA) The United Mitochondrial Disease Foundation (UMDF) will award three new research grants totaling $350,000 to three projects that will impact the diagnosis, treatment and clinical care of mitochondrial disease patients.  The projects and their prize winners will be awarded at the UMDF’s Mitochondrial Medicine 2019 Symposium.

Since 1996, the UMDF has funded only the most outstanding peer-reviewed research projects from around the world facilitating basic, translational and clinical research in mitochondrial disease.

“UMDF is excited to support these new projects as we continue to move fast towards a cure,” said Brian T. Harman, President and CEO.  “In the past 23 years, we have funded 123 projects, equaling nearly $11 million dollars. More importantly, UMDF’s commitment over the years to these projects has led to more than $140 million dollars of follow-on government funding in the field of mitochondrial disease,” Harman added.

In 2019, UMDF leadership, in close collaboration with its Scientific and Medical Advisory Board (SMAB), created two new award categories for application by the scientific community.  The first is the Experienced Investigator Prize to support established, more-senior principal investigators in taking their research in a new and novel direction.  Alternatively, the Early Stage Investigator Prize was created to aid and inspire the next generation of scientists who dedicate their work to mitochondrial disease research. Both of the 2019 prize winners submitted projects that study and advance science on potential treatments and therapies for mitochondrial diseases.  Both prizes will be awarded at the UMDF’s Evening of Energy Banquet on Friday, June 28, 2019.

An exciting third prize category was created this year, the accelerators. The accelerators was designed for postdoctoral fellows who were invited to submit their abstracts for peer review.   The UMDF’s SMAB then selected three finalists, all judged to be fundable based on scientific merit.  The finalists will have five minutes to present their project abstracts during the “Big Pitch” on Friday, June 28th, at Mitochondrial Medicine 2019.   Donors to the accelerators program will have an opportunity to watch the finalists either in person or via a live web stream and then vote on the project they feel most passionate about.  “This is really a win-win for the mitochondrial disease community,” said Dr. Philip Yeske, UMDF Science and Alliance Officer. “From UMDF’s perspective, we are confident that a scientifically meritorious project is funded; and for patient families that care deeply about supporting the mission of UMDF, they are able to lend a voice to the final selection process.”  The $50,000 accelerators Prize winner will be announced at UMDF’s Evening of Energy Banquet.

The UMDF Research Grant Program was established in 1996 at a time when no other organization existed to fund mitochondrial disease research.  Today, UMDF is the largest, non-governmental funder of basic and translational research designed to bring the best science from the bench to bedside.  All submitted research projects are peer reviewed by the top global scientific and medical experts in the mitochondrial research field.  In conjunction with the UMDF Scientific and Medical Advisory Board and select external experts, awards are made.  It is through the work of UMDF’s generous donors and supporters, who organize and donate to UMDF through our Energy for Life Walkathons, annual appeals, and other fundraisers that funding for these vital research project is made possible.


2019 Experienced Principal Investigator Prize – $200,000
Matthew Whiteman, Ph.D.
Professor of Experimental Therapeutics
University of Exeter
United Kingdom

Dr. Whiteman will be awarded the UMDF’s 2019 Experienced Principal Investigator Prize for his research project entitled “Can novel mitochondria-targeted hydrogen sulfide delivery molecules restore cellular bioenergetics in primary mitochondrial disease.” Dr. Whiteman extends his work over the past 5 years in other disease areas to primary mitochondrial disease using a well-established worm model of mitochondrial disease. This is a new approach in studying mitochondrial health and may lead to novel therapeutic opportunities to normalize, restore, and rescue loss of mitochondrial function and cellular energy.

2019 Early Stage Principal Investigator Prize – $100,000
Zarazuela Zolkipli Cunningham, MBChB, MRCP
Division of Human Genetics, Mitochondrial Medicine Frontier Program
Center for Mitochondrial and Epigenomic Medicine
Children’s Hospital of Philadelphia (CHOP)

Dr. Cunningham’s award winning project is entitled ‘Defining the Natural History of Mitochondrial Myopathy’.  Her project focuses on further exploring how the symptoms of individuals with skeletal muscle issues change over time and how best to measure those changes.  Dr. Cunningham’s research will capture the severity of patient symptoms, disease progression, and treatment response, with the ultimate goal of identifying clinically-meaningful interventions.

2019 accelerator Finalists – $50,000 Prize

Rachel Guerra
Institute for Research
Madison, WI

Project:  Structural and Functional Characterization of COQ9 in Facilitating Coenzyme Q Biosynthesis and Complex Q Formation.

Dr. Guerra’s  proposal aims to study how two such helper molecules called CoQ7 and CoQ9 work together to produce CoQ. Understanding this process could lead to insights on how to treat forms of mitochondrial disease related to CoQ deficiency.

Zachary Wilson
University of Utah
Salt Lake City, UT
accelerator Project: Manipulating Mitochondrial Metabolism Via the Mitochondrial Derived Compartment Pathway

Dr. Wilson’s project aims to study how molecules called mitochondrial transporters are produced and controlled, with a particular emphasis on how the transporter molecules are broken down and what impact that has on how well mitochondria work. Learnings from this research could help future researchers to develop treatments for mitochondrial disease that manipulate the nutrient pathway.

Arwen Gao
Ecole Polytechnique
Federale de Lausanne (EPFL)
Lausanne, Switzerland

accelerator Project: Identification of Novel Compounds to Treat Rare Mitochondrial Diseases

Dr. Gao’s goal for her research project is to identify novel compounds that increase the amount of mitochondria and/or activate the identified pathway in lab-based cell models.  The compounds that work best in the cell models will be subsequently tested in animal models of mitochondrial disease. Future work with top compound candidates have the potential to pave the way towards the development of novel drugs targeting rare mitochondrial diseases.

Mitochondrial diseases result from the failure of the mitochondria, which are located in the cells of our bodies. Mitochondria are responsible for creating more than 90% of the energy needed to sustain life and support growth. When mitochondria fail, less energy is produced causing cell injury or cell death. On a larger scale, organ systems begin to fail. The disease is often debilitating. In some cases, it may result in death. Adult onset is becoming more and more common. There is no cure for mitochondrial disease.

Founded in 1996, the United Mitochondrial Disease Foundation (UMDF) promotes research and education for the diagnosis, treatment and cure of mitochondrial diseases and to provide support for affected individuals and families. Since its inception, the UMDF has funded more than $11 million in research, making it the leading non-governmental contributor of grants focused solely on mitochondrial disease.  For more information about mitochondrial disease or the UMDF, visit umdf.org.