Why use Contract Research Organizations?
How will our work with Contract Research Organizations to help us to find a therapy for LGMD2B/Miyoshi muscular dystrophy?
The Jain Foundation is excited to announce the initiation of several new projects with Contract Research Organizations (CROs) to help in our efforts to develop a treatment for LGMD2B/Miyoshi. Over the past seven years, we have learned a great deal about dysferlin and the consequences of its absence. Through new partnerships with Contract Research Organizations (CROs), we are now in a position where we can begin to translate some of this critical knowledge into assays and platforms specifically designed for the discovery of therapeutic interventions. Our vision is to continue to expand our knowledge base, through research funding in academic laboratories, and use those findings to refine and develop our screening platforms for developing therapeutics. In these CRO projects, we will attempt to develop high throughput methods for screening libraries of chemical compounds (potential drugs) for their ability to treat dysferlin deficiency in cultured cells, and develop methods to a assess the efficacy of positive “hit” compounds in mouse models of dysferlinopathy.
Libraries of chemical compounds contain thousands of potential drugs that act on diverse biological pathways. By screening these libraries, candidate therapies that could not have been found by rational design may be identified. Such screens are time consuming, not mechanistic and difficult to publish, making them unlikely to advance the careers of academic scientists. For this reason, the Jain Foundation has chosen two contract research organizations, BioFocus and Evotec, to develop the high throughput screening assays necessary for screening thousands of drug candidates. Once a screening assay has been developed, libraries of chemical compounds can be screened, and positive hits can be tested preclinically in mouse models of dysferlinopathy.
Testing candidate drugs requires a definitive read out that can be compared between control and drug treated dysferlin deficient mice. Unfortunately, none of the known dysferlin deficient mice have a strong clinical phenotype, so where to look for improvement in dysferlin deficient mice is not well defined. To this end, the Jain Foundation has selected two contract research organizations, Charles River Research Discovery Services and PsychoGenics with expertise in preclinical testing to compare control and dysferlin deficient mice using techniques unavailable to most academic laboratories. If a phenotype can be found, it will be used to evaluate positive hits from the chemical compound screening assays completed by BioFocus and Evotec.
The Jain Foundation team is looking forward to working with all of these contract research organizations to find a therapy for LGMD2BMiyoshi. We also look forward to sharing the results of these projects with the greater scientific community, so that we can make use of these findings and accelerate the pace of research on dysferlinopathies.