MaRS Innovation is partnering with Dr. James Eubanks at the University Health Network (UHN) to develop a new treatment for Rett syndrome, a rare developmental disorder found almost exclusively in girls and women.
Infants with Rett syndrome develop and grow normally until the age of 8 to 12 months when symptoms begin appearing. Although the symptoms vary in type and severity, most of the girls and women with the disorder cannot speak or use their hands purposefully. Many also experience seizures that are difficult to control with medications. Presently, there is no cure for Rett syndrome, and the available treatments can only help alleviate symptoms.
Researchers have known for many years that most cases of Rett syndrome are caused by mutations in the MECP2 gene; however, the mechanisms that link these genetic changes to the syndrome’s diverse symptoms are not well understood.
“We believe that we have discovered an important piece of the puzzle,” says Dr. Eubanks, a Senior Scientist at the Krembil Research Institute (UHN) who has been studying the disorder for over 20 years.
MaRS Innovation will initially invest up to $400,000 to help translate Dr. Eubanks’ discovery into a new treatment for Rett syndrome. The funds will support the creation and evaluation of drug-like compounds that target TRPM2, dampening its activity, in the brain of Rett patients. Dr. Mark Reed, a medicinal chemist and head of Krembil’s Centre for Medicinal Chemistry and Drug Discovery, will oversee the work, which will be split between the Centre and the Charles River Discovery site in the United Kingdom. The new partnership will also give Drs. Eubanks and Reed access to MaRS Innovation commercialization services to support further development of any promising treatments.