Toronto Scientist Wins Award for Work Into Alternative Way of Treating CF

Medicine by Design, which supports work in regenerative medicines, gave a 2020 New Ideas Awards to support a researcher exploring an alternative pathway to the CFTR protein as a possible treatment for cystic fibrosis (CF).

The awardee, Amy Wong, PhD, works in developmental and stem cell biology at the Hospital for Sick Children (SickKids) in Toronto. She is one of the three investigators chosen for the prize, worth CA$100,000 (about $76,000) a year for up to two years.

Wong will use a combination of computational modeling, gene editing, and stem cell biology to assess if an alternative protein channel found in cells lining airway surfaces might compensate the lack of functional CFTR protein in CF patients.

“If successful, our study will be the first to provide proof-of-concept that this alternative approach to treating cystic fibrosis is effective,” Wong said in a press release. She is also an assistant professor in the department of laboratory medicine and pathobiology in the University of Toronto’s Temerty Faculty of Medicine.

The project builds up on research carried out at SickKids, where the CFTR gene, which causes CF when mutated, was first identified some 30 years ago.

According to Wong, while her project’s idea is not revolutionary in itself, but the combination of tools at her disposal might turn it into “a potential game changer.”

“We have access to an incredible resource of primary cells and stem cells from more than 100 individuals with cystic fibrosis harboring various mutations,” Wong said.

“Our lab has developed human lung models from stem cells that can be used to model lung disease such as cystic fibrosis. And with new advanced tools in single-cell genomics and gene-editing, coupled with key collaborations for computational modelling, we are poised to find new therapeutic targets for cystic fibrosis,” she added.

The two other investigators chosen for New Ideas Awards by Medicine by Design, part of the University of Toronto, are Leo Chou, PhD, an assistant professor at the university’s Institute of Biomedical Engineering, and Hyun Kate Lee, PhD, an assistant professor of biochemistry at the Temerty Faculty of Medicine.

Chou will lead a project focused on developing cell transplants for the retina — the region at the back of the eye that enables sight. Lee will focus on identifying target genes that might improve the overall health of neuromuscular junctions, the regions where nerve fibers and muscle cells communicate. Damage to and loss of these structures is associated with diseases that include amyotrophic lateral sclerosis.

Medicine by Design also gave four one-year Seed Fund Awards, each worth CA$100,000, to support projects ranging from studying heart repair in children undergoing heart surgery, to treating premature babies with necrotizing enterocolitis, a potentially life-threatening inflammation of the intestines typically affecting premature babies.

In total, Medicine by Design invested $1 million to support these seven research projects at the University of Toronto and its affiliated hospitals.

“Our 2020 New Ideas project portfolio integrates mathematical modelling, physics and computational biology with stem cell biology and biomedical engineering, and strengthens engagement with clinicians who are key to translating our research into patient impact,” said Michael Sefton, executive director of Medicine by Design.

“We are particularly delighted this year to support so many outstanding early-career researchers, who will ensure Toronto remains a global leader in regenerative medicine for years to come,” Sefton added.

Winning projects were selected from a shortlist of 36 proposals submitted by university and hospital clinicians and researchers, and evaluated through an external peer review process.

The Medicine by Design regenerative medicine initiative was partly made possible by a grant from the Canada First Research Excellence Fund.