GlycoNet Grants $3.9M to Canadian Researchers to Study Sanfilippo, Other Diseases

The Canadian Glycomics Network (GlycoNet) has granted $3.9 million in funding for research projects aimed at mitigating the healthcare burden in Canada and contributing to a better understanding of diseases, including Sanfilippo syndrome.

The recipients — multidisciplinary research institutions and their industry partners — will use the funding to address unmet needs in cancer, cystic fibrosis, Parkinson’s disease, and autoimmune disorders, as well as Sanfilippo.

“Investing in health research projects that improve the quality of life of Canadians is part of GlycoNet’s mission,” Todd Lowary, PhD, GlycoNet’s scientific director, said in a press release.

“These investments also help foster collaborations between academic institutions and industry stakeholders, making the transition of new scientific knowledge, research techniques and collected data into real-world applications faster,” Lowary said. “Through funding these projects, we want to make sure those who are learning how to treat diseases, who are finding ways to prevent infections, who are inventing new technologies and creating jobs in the industries have the support and facilities they need.”

The hope is that these projects will ultimately accelerate the development of diagnostics, treatments, and clinical management of disorders at different levels of Canada’s healthcare system, thereby lowering the country’s healthcare burden.

One GlycoNet-funded team will test innovative therapeutic approaches, including gene or stem cell correction and small molecules, to cure or reduce symptom severity in Sanfilippo, a lysosomal storage disorder that affects about 1 in 70,000 live births.

Also known as mucopolysaccharidosis type III (MPS III), Sanfilippo syndrome is a rare genetic lysosomal storage disorder characterized by the absence or reduced activity of enzymes involved in the breakdown of a complex sugar molecule called heparan sulfate, leading to its toxic buildup.

With no approved therapy that targets the disease’s cause, treatments have focused on easing its symptoms, specifically the deterioration of neurological function.

While there have been increasing efforts to develop enzyme replacement therapies that would regularly deliver a healthy version of the deficient enzyme to the body, the challenge is getting such treatments to reach the brain.

Because of the blood-brain barrier — a protective membrane that restricts passage of certain molecules from the blood into the brain — large molecules such as enzymes cannot reach the brain. Central nervous system-targeted gene therapy, which uses harmless viruses, can potentially overcome this challenge. But such therapies are still in the developmental stages.

The team’s lead investigator for the funded project is Alexey V. Pshezhetsky, PhD, of the CHU Sainte-Justine research center, a professor at the University of Montreal. Co-investigators are Gregory Lodygensky, MD, and Christian Beauséjour, PhD, both of CHU Sainte Justine, and Christopher Cairo, PhD, of the University of Alberta.

Other projects supported by GlycoNet include the development of next-generation treatments for chronic inflammation in the lower gastrointestinal tract, and an economic evaluation of a new sugar-based prostate cancer diagnostic tool. Funding also will go to projects seeking to develop treatments for patients unresponsive to current cancer immunotherapies, and to create a carbohydrate-based diagnostic tool to identify children with cystic fibrosis who are likely to fail conventional antibiotic treatment.

GlycoNet works to develop new carbohydrate-based therapies, vaccines, and diagnostics in collaboration with academia and industry.