Cyclica Inc. is pleased to announce a collaboration with Dr. James Dowling of The Hospital for Sick Children (SickKids) in Toronto, Canada. The goal of this collaboration is to support Dr. Dowling’s research on a rare neuromuscular disorder, myotubular myopathy (MTM).
MTM is a rare genetic disease that afflicts approximately 1 out of every 50,000 newborn males worldwide. MTM is caused by mutations in the MTM1 gene, which is responsible for the production of a protein called myotubularin, an enzyme critical for the proper development and maintenance of muscle cells. Accordingly, MTM affects the body’s muscles, resulting in severe muscle weakness with impairment of motor skills (requiring wheelchair dependence), and prevents infants from eating and breathing without mechanical assistance (e.g. ventilator dependent); MTM-linked muscle weakness also disrupts bone development, impairs vision and causes secondary complications including liver disease, ear and lung infections, and seizures. Unfortunately, most individuals with MTM do not survive past childhood, with few documented cases of patients living into adulthood with this disease. Therefore, there is a substantial need for therapeutics that improve the quality of life for patients diagnosed with MTM. Currently, most research efforts focus on the promise of gene therapy to repair mutations in the MTM1 gene; however, small molecule therapies have remained a relatively unexplored area of scientific pursuit.
Dr. Dowling, a leading authority on the diagnosis and management of congenital myopathies, will use Cyclica’s computational proteome docking platform to strategically investigate, identify, and prioritize lead small molecule candidates as potential therapies for MTM, a disease in which no effective therapy currently exists. Funding for this project is provided by the Myotubular Trust, a charitable organization that promotes the study of, and research into, the treatment and cure of the muscle weakness caused by MTM. “We look forward to working with Cyclica and are confident that their innovative platform will positively impact how we identify and further develop novel therapies for patients with this rare and debilitating disease”, Dr. Dowling explained.
Cyclica’s patented Ligand Express™ technology generates proteome docking profiles for any ligand it screens. Cyclica also offers a range of analytical utilities that assist in interpreting these profiles in a meaningful way. Researchers have used these analytical tools to differentiate between leading drug candidates based on their likelihood for interacting with disease-relevant proteins, as well as, evaluated the probability that identified interactions will have a therapeutic effect. Other insights that can help prioritize between drug candidates include: identifying off-target effects (i.e. side effects) and potential contraindications (i.e. other drugs that should be avoided while taking the new drug). Together, Dr. Dowling and Cyclica will use Ligand Express™ to rationally prioritize small molecule drug candidates for the treatment of MTM. “Dr. Dowling is a world-class researcher, and it is our privilege to work directly with him and his talented team. We are excited to be an integral part of translating novel discoveries from the bench – and computer – to bedside”, said Naheed Kurji, President & CEO of Cyclica.