Programmed for success: SickKids research a gateway to discovery
When Dr. Aser Rothstein became the scientific director of the Research Institute, he decided it was time for a change. One of the major elements of his restructuring plan in 1972 was the introduction of the concept of the “research program” to SickKids.
At that time, he introduced two new programs to the Research Institute: the Respiration Program and the Neurobiology Program. Each program needed to have a particular research focus. This focus needed to have the potential to solve or prevent a clinical problem, make a unique contribution, cover an interdepartmental scope and be in an area of biology where breakthroughs seemed possible. Programs would be led by a director and have three to five investigators, making up the “core” of the program.
In 1973, the program concept continued with the introduction of three more new programs to the SickKids Research Institute: Cystic Fibrosis (CF), Genetic-Metabolic Disease and Diagnostic Biology.
One program of note is the Cystic Fibrosis Program with its first director, Dr. G. Forstner. Already world leaders in CF research, SickKids developed a successful program that may be seen as the gateway to a discovery that changed CF research forever. Just over a decade later, in August 1989, a team of SickKids scientists led by Dr. Lap-Chee Tsui discovered that a mutation in the CF trans-membrane regulator (CFTR) gene is responsible for the multi-organ disease that mainly affects the lungs and digestive system. This year marks the 20th anniversary of that discovery.
The discovery of the CF gene made it possible to detect whether someone is a carrier and has opened the door for infant screening, which has already begun in Ontario, Alberta and Saskatchewan. Sixty years ago people born with CF did not often live past their fourth birthday and nowadays, Canadians with CF are living into their late 30s.