Elesclomol-copper combination offers hope for a cure for Menkes Disease.
By Crystal Carter, Texas A&M Innovation – October 6, 2023
Dr. Vishal M. Gohil’s work to understand the effects of elesclomol combined with copper is leading to potential treatments for copper deficiency conditions such as Menkes Disease, a devastating condition that primarily affects young children. Dr. Gohil’s technology is at the center of a licensing agreement between The Texas A&M University System, managed through the Texas A&M Innovation office, and California-based Engrail Therapeutics.
Menkes Disease, also known as Menkes Syndrome or Kinky Hair Disease, is a rare X-linked recessive disorder characterized by a defect in copper absorption leading to a profound copper deficiency within the body. This condition leads to a range of severe symptoms, including neurological degeneration, connective tissue abnormalities, and hair that is often described as “kinky.” It is a fatal disease that affects approximately 1 in 35,000 live births and has no known cure.
A Texas A&M AgriLife Research team led by Vishal M. Gohil, Ph.D., Professor in the Department of Biochemistry and Biophysics in Texas A&M’s College of Agriculture and Life Sciences, has dedicated years to unraveling the intricacies of mitochondrial function and cellular copper regulation.
At a press conference held at his research laboratory, Gohil discussed his findings and the potential implications for Menkes Disease treatment. He explained, “We have long known that copper is vital for various biological processes, including the function of numerous enzymes and proteins. In Menkes Disease, a copper deficiency disrupts these essential processes, resulting in severe health issues. Elesclomol, when used in combination with copper, provides efficient means to deliver copper into cells and especially to mitochondria, thereby potentially mitigating the disease’s effects.”
The license agreement with Engrail, executed in 2021, set the stage for Engrail to conduct preclinical studies toward a cure for Menkes Disease. Even though it may take some time for the treatment to become available to patients, Gohil’s research has already garnered substantial attention from patients and their families who have long struggled with this devastating condition. An AgriLife Today video report received many inquiries worldwide from those seeking possible cures. “We received inquiries from families who are desperate because there is no cure,” he said.
Gohil completed his Ph.D. in Biological Sciences at Wayne State University in 2005 and a postdoctoral research fellowship at Harvard Medical School. For more information on Gohil’s Lab, visit http://www.gohillab.com/.
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