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Oxford-Harrington Rare Disease Centre to Advance Novel Treatment for Duchenne Muscular Dystrophy

Wednesday, August 03, 2022

The Oxford-Harrington Rare Disease Centre (OHC), a collaboration between the University of Oxford and Harrington Discovery Institute at University Hospitals in Cleveland, Ohio, has announced its endorsement of a groundbreaking treatment for Duchenne muscular dystrophy (DMD), a rare neuromuscular disorder characterized by progressive muscle degeneration and weakness. The research project is spearheaded by Angela Russell, DPhil, a Professor of Medicinal Chemistry at the University of Oxford and a 2021 Harrington UK Rare Disease Scholar.

Duchenne muscular dystrophy primarily affects boys, typically manifesting around the age of four. It is caused by a genetic defect that impairs the production of dystrophin, a protein that acts as a shock absorber during muscle contractions. Without dystrophin, muscles gradually deteriorate and weaken. Children with DMD experience difficulties in standing up and walking, eventually requiring the use of a wheelchair. Severe, life-threatening complications such as heart muscle disease and respiratory difficulties may arise. Unfortunately, individuals with DMD rarely live past their thirties.

Professor Russell and her team have been investigating a protein called utrophin, which exhibits similar protective properties to dystrophin in muscle function. The laboratory has identified new classes of molecules that enhance utrophin production and has been collaborating with Harrington's Therapeutics Development Center since 2021 to advance their research. Recently, the Oxford-Harrington Rare Disease Centre selected Professor Russell's project for further support in drug development and commercialization.

"Professor Russell's project exemplifies our commitment to developing new therapies that can prevent or reverse the debilitating effects of rare diseases in children," says Professor Matthew Wood, Director of the Oxford-Harrington Rare Disease Centre. "We are delighted to provide Professor Russell with a network of resources and expertise to facilitate the advancement of this exciting discovery towards benefiting patients."

"Less than five percent of rare diseases currently have approved treatments, and many of these conditions disproportionately affect children," notes Jonathan S. Stamler, MD, President of Harrington Discovery Institute and Robert S. and Sylvia K. Reitman Family Foundation Distinguished Professor of Cardiovascular Innovation and Professor of Medicine and Biochemistry at University Hospitals and Case Western Reserve University. "The establishment of the Oxford-Harrington Rare Disease Centre aims to accelerate the development of treatments to address this significant unmet need and enhance the health outcomes of individuals living with rare diseases."

The Oxford-Harrington Rare Disease Centre considers projects for support if they offer novel treatments for rare diseases, particularly those with a genetic basis or that impact children. Additional factors taken into account include the potential for clinical impact and the ability to address other diseases.

Through the OHC, successful Principal Investigators may be eligible for additional funding to expedite drug development. They will also benefit from:

  • Support in drug and business development.
  • Facilitated access to funders, contract research organizations, patient organizations, and a nucleic acid therapy facility.
  • Invitation to present at the bi-annual Oxford-Harrington Rare Disease Centre Symposium and the annual Harrington Discovery Institute Scientific Symposium.

"I am grateful for the support I have received from Harrington Discovery Institute, my university, and other funders to bring our work to its current stage," expresses Angela Russell, DPhil. "I look forward to collaborating with the Oxford-Harrington Rare Disease Centre to advance this drug candidate for utrophin replacement therapy toward clinical trials."

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