By: Crystal Carter
Professor Receives Chancellor’s Innovation Award for Breakthrough in Copper Metabolism Therapy
During the 2025 Innovation Awards Luncheon, Dr. Vishal M. Gohil, a professor in the Department of Biochemistry & Biophysics at Texas A&M University, was honored with the prestigious Chancellor’s Innovation Award, recognizing his groundbreaking research on mitochondrial diseases and copper metabolism.
Dr. Gohil’s laboratory made a major breakthrough when they discovered that elesclomol, a compound initially developed as a cancer drug, can transport copper into mitochondria—rectifying biochemical defects associated with genetic copper deficiency. This finding has enormous implications for the treatment of Menkes disease, a rare, fatal pediatric disorder characterized by insufficient copper delivery to cells, resulting in developmental delays, uncontrollable seizures, and death by the age of three.
In her remarks at the luncheon, Janie Hurley, Senior Director of Licensing, praised Dr. Gohil’s work, calling it “a true example of innovation that begins at the lab bench and extends to life-changing therapies for patients in need.”
From Cancer Drug to Lifesaving Therapy
Dr. Gohil’s research focuses on disorders affecting mitochondrial energy production, with an emphasis on copper as a critical micronutrient. Copper is essential for mitochondrial function, but delivering it effectively into cells—especially into mitochondria—has been a long-standing challenge in medicine.
In a pioneering drug screen, Dr. Gohil’s lab discovered that elesclomol is capable of binding copper in the bloodstream and delivering it directly into mitochondria. In preclinical studies, elesclomol was shown to restore copper-dependent mitochondrial activity and correct metabolic defects in copper-deficient zebrafish and mice models. This work paved the way for what may be the first effective therapy for Menkes disease.
This innovation led The Texas A&M University System to enter into an exclusive license agreement with Engrail Therapeutics, allowing the company to advance development of the therapy. Backed by this collaboration, preparations are now underway for clinical trials in the United States.
Compassionate Use and Real-World Impact
In a testament to the life-changing potential of this discovery, the Spanish Agency of Medicines and Health Products approved elesclomol for compassionate use in children with Menkes disease. To date, five children have received the drug, with remarkable results. One child, previously bedridden and given a poor prognosis, began treatment at 20 months old and is now four and a half years old—able to walk and speak in two languages.
The FDA has granted Orphan Drug Designation and Rare Pediatric Disease Designation to elesclomol, recognizing its potential to address an unmet medical need in a small patient population. These designations position the therapy for regulatory support as it enters clinical trials.
Recognizing a Legacy of Innovation
Dr. Gohil joined Texas A&M in 2012 and has steadily built a national and international reputation for excellence in biomedical research. He was promoted to Full Professor in 2023 and previously received the Vice Chancellor’s Award in Excellence for Research (2021) and was named a Chancellor EDGES Fellow (2024).
He earned his Ph.D. in Biological Sciences from Wayne State University and completed postdoctoral research at Harvard Medical School. His work has been recognized with numerous honors, including the Ivano Bertini Award for contributions to Copper Biology and Medicine, the Chairman’s Prize from the United Mitochondrial Disease Foundation, and the Martin Research Prize for Excellence in Basic Research from Massachusetts General Hospital.
Dr. Gohil’s research is supported by the National Institutes of Health, The Welch Foundation, and Engrail Therapeutics.
