The Centre of Excellence for the Prevention of Organ Failure (PROOF Centre) and MRM Proteomics Inc. will use their biomarker expertise in collaboration with the Jain Foundation, Inc. to identify blood-based biomarkers of disease in individuals with a rare form of muscular dystrophy, called Limb‐girdle muscular dystrophy 2B (LGMD2B) or Miyoshi Myopathy (MM). Since no effective treatment exists for LGMD2B/ MM, this study is an essential step towards developing therapies for patients living with these debilitating muscle diseases.
LGMD2B/MM are both caused by mutations in the gene that encodes the protein dysferlin, which is thought to play a role in skeletal muscle repair collectively called “dysferlinopathies.”
The Jain Foundation chose to collaborate with MRM Proteomics and the PROOF Centre because of their excellent track record of discovering molecular biomarkers in blood and developing them into tests that aid in the diagnosis and treatment of other diseases.
“The Jain Foundation was impressed by the success PROOF Centre has had in moving biomarker programs from concept to the clinic, particularly with their recent clinical progress in finding a biomarker for rejection of a transplanted heart. We believe that pairing MRM Proteomics’ cutting edge expertise in mass spectrometry with PROOF’s unique clinical biomarker expertise, offers the greatest chance of identifying a biomarker to move our clinical program forward.” said Plavi MIttal, President and CEO, Jain Foundation
The study will compare blood samples from individuals with LGMD2B/MM with samples from healthy, age and gender-matched controls. Differences in proteins and nucleic acids in the blood that reflect differences in muscle function will be tested and validated as possible biomarkers. These biomarkers will then be used to help track disease progression and the efficacy of potential treatments in future clinical trials.