Parents of sick kids know all too well the feeling of being powerless to help their children. When the illness is rare, it can be even more difficult as families face countless visits to a plethora of doctors, the so-called ‘diagnostic odyssey’ to determine what’s wrong. Those lucky enough to identify the rare disease are often faced with the reality that there are few, if any, treatment options.
That’s where the IGNITE Project (the short form of Orphan Diseases: Identifying Genes and Novel Therapeutics to Enhance Treatment, a project that received $200,000 from NSRIT) comes in. The passionate and dedicated team of doctors and researchers uses a unique combination of techniques to help develop drugs that are specifically targeted to these rare genetic disorders.
Dr Jason Berman, a key member of the team, is helping to unlock the puzzle using his expertise with zebrafish. The tiny, translucent fish provide a unique look into the genes behind the diseases, as well as a way to track the effects of new drugs. And it’s working – this team has discovered a drug that can treat a rare genetic blood disorder, bringing much-needed relief to affected families in the region. Ground-breaking work like this has now led to Dr Berman’s lab becoming a node of Canada’s esteemed Centre for Drug Research and Development.