The APBD Research Foundation is pleased to share that Felix Nitschke, PhD (University of Texas Southwestern Medical Center) and Wyatt Yue, PhD (Newcastle University) are the recipients of rare disease research grants from the University of Pennsylvania’s Million Dollar Bike Ride (MDBR) program. Dr. Nitschke and Dr. Yue, both past recipients of MDBR grants, are eager to expand on the foundation of their previous work and drive it forward.

From left to right: Dr. Felix Nitschke, Dr. Mayank Verma, Dr. Nirbhay Yadav, and Dr. Wyatt Yue
For the past nine years, our Foundation has partnered with the University of Pennsylvania’s Orphan Disease Center to participate in — and raise funds for — the Million Dollar Bike Ride program. This program provides a one-year pilot grant to support research related to a rare disease represented in the Million Dollar Bike Ride.
Through this partnership, our researchers at Columbia University, Duke University Medical Center, UT Southwestern Medical Center, University of Florida, Newcastle University, and Hadassah Medical Organization, have collectively received approximately $873,000 in pilot grants managed on our behalf.
Dr. Felix Nitschke | UTSW Medical Center
Research Project: From Mouse to Human – Establishing Novel Biomarkers for APBD
Grant Amount: $56,857
Dr. Felix Nitschke is an Assistant Professor at UT Southwestern Medical Center in the Departments of Pediatrics and Biochemistry. He is a leading expert in carbohydrate (sugar molecule) research and serves as the co-chair of the APBDRF’s Scientific and Medical Advisory Board. His work focuses on advancing our understanding of glycogen metabolism (how our body creates energy) and pioneering new therapies for rare diseases. In 2020, Dr. Nitschke received an MDBR grant to design and test a gene therapy using an APBD mouse model. Building on the progress made in his laboratory, he is now directing his efforts toward developing biomarkers – measurable characteristics of the body that indicate presence, stabilization, or reduction of disease – an essential step in paving the way for future clinical trials.
For his winning proposal, Dr. Nitschke teamed up with co-investigators Mayank Verma, MD, PhD (UT Southwestern) and Nirbhay Yadav, PhD (Johns Hopkins University). Together, they seek to develop reliable MRI imaging, urine, and blood biomarkers to monitor disease progression and evaluate treatment efficacy in future clinical trials.
Dr. Nitschke explained, “With several treatments for APBD in preclinical development, the identification of valid surrogate biomarkers (measurable biological attributes used in clinical trials as a substitute for clinical endpoints, which can take a long time or be challenging to assess) is critical to getting clinical trial approval.
As a team with diverse expertise, we are proposing a multi-dimensional approach that includes the APBD mouse model and patients. At the same time, we are probing fluid biomarkers but also those that can be detected non-invasively by novel MRI techniques. The MDBR funding enables us to work directly toward improving clinical trial readiness in APBD.”
Dr. Wyatt Yue | Newcastle University
Research Grant Project: Development of Glycogen Synthase Inhibitors for APBD
Grant Amount: $56,857
Dr. Wyatt Yue, a Professor and the Chair of Structural Biology at Newcastle University Biosciences Institute, leads pioneering research into the molecular mechanisms of rare metabolic diseases. To date, his team has determined the 3-D structures of over 70 different human metabolic enzymes. Dr. Yue and his team are focused on translating foundational scientific insights into the development of small molecule therapeutics.
Dr. Yue’s award-winning proposal builds on an innovative screening approach made possible by the MDBR grant he received in 2021. By integrating this cutting-edge method with his team’s exceptional chemical and biochemical expertise, he is seeking an oral therapy for APBD patients.
The approach will investigate certain molecules that may be able to effectively downregulate glycogen synthase, thereby preventing the abnormal accumulation of glycogen in the body. The team will also analyze and optimize these molecules to ensure they possess critical properties, such as the ability to reach damaged cells in the brain.
Dr. Yue explained, “Our vision is to develop a transformative oral therapy for APBD patients. As the key step towards this goal, we are developing small molecules that target the glycogen synthase enzyme as a drug starting point. This generous funding enables us to pursue multiple paths towards optimizing these small molecules to have drug-like properties, and move them into the clinic in the near future.”
Susan Coddon, a member of the APBD Research Foundation’s Board of Directors, shared, “Making treatments a reality was important to me when my husband LD was alive. At the time, I joined the APBDRF Board of Directors and my husband and I invested in research. Today, we still have no treatments, but our researchers are developing preclinical therapies that are on the cusp of clinical trials. This tells me that investing in research has never been more important. For the past nine years, our partnership with the UPenn Orphan Disease Center has helped us to raise critical funds and offer seed grants, paving the way and building momentum for innovative APBD research. I’m grateful to every supporter, the ODC, and our incredible research grant awardees — Dr. Nitschke, Dr. Verma, Dr. Yadav, and Dr. Yue — for their commitment to APBD and rare disease research.”