Backed by a $20,000 HSC Foundation grant, clinical dietitian Rhiza Regalado Lam Chew Tun will investigate the drug’s potential to reduce the need for enzyme therapy in cystic fibrosis patients
Rhiza Regalado Lam Chew Tun, clinical dietitian at HSC, shares test results with a pediatric patient and his family in HSC’s Pediatric Cystic Fibrosis Clinic.
A recently approved modulator therapy drug is giving hope to people living with cystic fibrosis (CF), a progressive and often fatal genetic disorder. In Canada, Trikafta (elexacaftor/tezacaftor/ivacaftor) is a prescription drug approved for treating CF in patients aged two and older with at least one F508del mutation or a responsive mutation in the CFTR gene and is funded through public drug programs.
“This is a significant advancement, a highly effective, life-changing medication that works with the underlying cause of the problem,” says Rhiza Regalado Lam Chew Tun, clinical dietitian at HSC’s Pediatric and Adult Ambulatory Care Nutrition and Food Services Department.
CF is the most common fatal genetic disease in Canada, affecting approximately 4,500 people nationwide. It primarily impacts the lungs and digestive system, leading to severe respiratory problems and difficulties in absorbing essential nutrients. As a result, nearly 88% of CF patients experience pancreatic insufficiency (when the pancreas doesn’t produce enough of the enzymes required to properly digest food), making it challenging for them to maintain optimal nutritional status.
“The standard treatment to manage pancreatic insufficiency involves pancreatic enzyme replacement therapy,” Regalado Lam Chew Tun explains. “However, the long-term use of pancreatic enzymes can cause side effects, including fibrosing colonopathy, where there could be thickening, narrowing, and shortening of the colon wall.”
Prior to the use of CF modulator therapy, pancreatic insufficiency in CF was thought to be irreversible. Improvement in pancreatic function on modulator therapy has been reported but the mechanism is still unclear. Trikafta offers hope by possibly restoring pancreatic exocrine function in CF patients—which could potentially reduce or even eliminate the need for enzyme therapy.
Rhiza Regalado Lam Chew Tun, clinical dietitian at HSC, observing a lung function testing of a young patient in HSC’s Pediatric Cystic Fibrosis Clinic.
If Trikafta continues to prove effective in restoring pancreatic function, it could significantly improve the quality of life for CF patients, many of whom currently take multiple enzyme capsules with every meal or snack, totalling hundreds of pills each month.
“If Trikafta can reduce this need, it would be such a meaningful stride towards a future where all Canadians with cystic fibrosis can live longer, healthier, better lives.”
“With life-changing treatments available for many, this is something to celebrate—and a chance to learn about the new modulator therapy drugs.” Clinical guidelines on reducing or stopping enzyme use when pancreatic function is restored through these new drugs are not yet established. Continuous monitoring of pancreatic function will be crucial as Trikafta becomes more widely available.
In Manitoba, pancreatic function tests for CF patients on modulator therapies like Trikafta are not covered by Manitoba Health. Patients need to pay out of pocket if pancreatic function testing is requested and needed. A test that must be outsourced to another province.
To address this gap, Regalado Lam Chew Tun sought support from HSC Foundation donors. In early 2024, she received a $20,000 Allied Health Grant from the HSC Foundation to investigate the effectiveness of Trikafta in her pediatric patients. The funding will allow her to track changes in pancreatic function for children aged two-to-18 currently using the drug, helping determine whether enzyme replacement therapy can be reduced or discontinued.
“The potential impact of this research is monumental” says Regalado Lam Chew Tun. “It means fewer pills, decreased financial burden, less damage to the gastrointestinal tract, and overall, a better quality of life for patients and their families.”
Through this research, Regalado Lam Chew Tun aims to help in creating an evidence-based care plan for CF patients. She plans to share her findings at the North American Cystic Fibrosis Conference, and her research will also be published in peer-reviewed journals, contributing to the broader understanding of CF treatment in the era of modulator drug therapies.
“My patients and their families are deeply grateful for this opportunity to improve their quality of life. On their behalf, I want to extend our heartfelt thanks to HSC Foundation donors for making this critical research possible,” she adds.
Every year, the HSC Foundation provides grants to promising medical research projects led by HSC personnel. To learn more about the research initiatives being funded by the HSC Foundation and its donors, click here. Consider donating today to help move health care forward in our province. To donate, select ‘Research at Health Sciences Centre’ on our donation page or call us at 204-515-5612 or 1-800-679-8493 (toll-free).
By Jen Golletz
