In an attempt to develop therapies for patients with a rare form of muscular dystrophy, the Centre of Excellence for the Prevention of Organ Failure (PROOF) and MRM Proteomics will collaborate with the Jain Foundation to identify blood-based biomarkers.
Limb-girdle muscular dystrophy 2B (LGMD2B) or Miyoshi myopathy (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.
In the study, blood samples from patients with LGMD2B/MM will be compared to samples from healthy, age and gender-matched individuals. 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, according to the companies.
“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,” said Plavi Mittal, Jain Foundation president and CEO .
In August, Evotec and the Jain Foundation extended a research collaboration in skeletal muscular dystrophy in which Evotec’s assay development and screening capabilities are utilized to screen compound libraries. This project aims to develop methods that may potentially evaluate drugs that could become treatments for LGMD2B.
The Jain Foundation is a privately funded nonprofit organization established by Ajit Jain of Berkshire Hathaway in the hopes of curing dysferlinopathy, which afflicts his son.
