The Foundation for the National Institutes of Health (FNIH) will award the 2026 Paul-Gallin Trailblazer Prize for Physician-Scientists to Michael Pacold, MD, PhD, of NYU Langone Health. Dr. Pacold and his team discovered a metabolic pathway that enabled them to effectively treat a severely debilitating and, in some cases, fatal ultra-rare neurodevelopmental disease. Furthermore, this breakthrough could open new treatment paths for other neurodegenerative conditions, such as Alzheimer’s and Parkinson’s disease.

“Dr. Pacold and his colleagues turned their critical basic science discovery into an effective treatment for a young patient facing a lethal genetic disease,” said Steven Paul, MD, Chair of the FNIH Board of Directors. “His pioneering and consequential work perfectly captures the spirit of the Paul-Gallin Trailblazer Prize for Physician-Scientists.”

Dr. Pacold and his lab discovered that the enzyme HPDL drives a previously unknown metabolic pathway that makes a critical part of coenzyme Q10 (CoQ10), an antioxidant needed by cells to generate energy. His team translated this finding into an experimental treatment for a boy with a very rare genetic condition caused by HPDL deficiency, which leads to worsening coordination and movement and, in some patients, early death. Once unable to walk more than short distances due to severe muscle spasms, the boy regained the ability to walk within a month of starting treatment — the first time any therapy has improved neurological symptoms in a primary CoQ10 deficiency. He has continued to show dramatic improvement over the past two years. Beyond rare disease, Dr. Pacold's discovery opens new avenues for treating other rare pediatric diseases caused by loss of CoQ10 synthesis, as well as neurodegenerative conditions like Alzheimer's and Parkinson's disease.

“I am very grateful to the FNIH, the founders of the Paul-Gallin Trailblazer Prize for Physician-Scientists, the jury, my family and mentors, the members of my laboratory, and our patients and families,” Dr. Pacold said. “Our discovery of fundamental metabolic processes and successful treatment of a patient demonstrates the tremendous power of basic research to improve the outcomes of patients with a previously untreatable disease.”

A radiation oncologist and biochemist, Dr. Pacold is Director of Basic Science Research in the Department of Radiation Oncology at NYU Langone Health, where he also works as a physician. Dr. Pacold is an associate professor in the Department of Radiation Oncology at NYU Grossman School of Medicine and has been elected to the American Society for Clinical Investigation.

The Paul-Gallin Trailblazer Prize includes a $100,000 honorarium and recognizes the outstanding contributions of early- to mid-career physician-scientists whose research translates basic discoveries into novel approaches for diagnosing, preventing, treating or curing disease and disability. The award honors two important physician–scientists, Dr. Paul and the late John Gallin, MD. Dr. Paul initiated the endowment for this prize with a generous $1 million gift. Dr. Gallin was the longest-serving director of the NIH Clinical Center, who, with his wife Elaine Gallin, PhD, created the Trailblazer Prize in 2018 in partnership with the FNIH.

A jury of distinguished biomedical research leaders selected Dr. Pacold as the 2026 Paul-Gallin Trailblazer Prize recipient. Michael J. Welsh, MD, Director, Pappajohn Biomedical Institute, University of Iowa, chaired the Trailblazer Prize jury, which also included Dr. Paul; Howard Y. Chang, MD, PhD; Helen H. Hobbs, MD; Timothy J. Ley, MD; Kim Rathmell, MD, PhD, MMHC; Christine E. Seidman, MD; and Huda Yahya Zoghbi, MD.

Dr. Pacold will receive the award at the FNIH Celebration of Science: 14th Annual Awards Ceremony on Oct. 28, 2026, in Washington, D.C. The 2026 Montrone-Seigel Prize in Biological Sciences; the Charles A. Sanders, MD, Partnership Award; and the Kovler Prize for Trust in Life Science Journalism also will be presented during the ceremony.

The FNIH gratefully acknowledges its Celebration of Science Premier Sponsors, Fred and Donna Seigel and Perry Steiner and Vanessa Kay, and Visionary Sponsors, Sherry and Alan Leventhal and Pfizer, Inc. For more information about the Trailblazer Prize, visit fnih.org/TrailblazerPrize.

About the Foundation for the National Institutes of Health

The Foundation for the National Institutes of Health (FNIH) builds public-private partnerships that connect leading biomedical scientists at the National Institutes of Health (NIH) with their counterparts in life sciences companies, academia, patient organizations, foundations, and regulatory agencies (including the Food and Drug Administration and European Medicines Agency). Through team science, the FNIH solves complex health challenges and accelerates breakthroughs for patients, regardless of who they are or what health threats they face. The FNIH contributes to the development of new therapies, diagnostics, and potential cures; advances global health; and helps train the next generations of scientists. Established by Congress to support the mission of the NIH, the FNIH is a not-for-profit 501(c)(3) charitable organization. For more information about the FNIH, please visit fnih.org.

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"Dr. Pacold and his colleagues turned their critical basic science discovery into an effective treatment for a young patient facing a lethal genetic disease. His work perfectly captures the spirit of the Paul-Gallin Trailblazer Prize."—Steven Paul, MD