Accelerating Research for Near-Term Impact
NOVEMBER 3, 2022
Cystic Fibrosis Canada invests in research to help improve the health outcomes and quality of life of people living with CF. While we generally fund basic science and clinical research projects in academic laboratories, universities and hospital research institutes, in 2016, an opportunity arose for us to invest in a Laurent Pharmaceuticals, a Montreal-based company currently focused on treatments for cystic fibrosis and COVID-19. Laurent Pharma’s lead molecule, LAU-7b, has the potential to benefit people with cystic fibrosis (CF), regardless of their mutation(s), by resolving inflammation of the lungs that CF patients experience, a leading cause of lung tissue destruction in CF, while preserving overall lung function.
“An anti-inflammatory drug will be really important for people living with CF,” said Dr. John Wallenburg, Chief Scientific Officer, Cystic Fibrosis Canada. “Inflammation is a well-known, unresolved problem in cystic fibrosis. In 2016, several anti-inflammatory drugs were under development, but we knew new approaches were needed. All of them, except LAU-7b, have since failed in clinical trials.”
The path from laboratory discovery to an approved therapy for patients is long, difficult, and expensive. Many great ideas in the lab never make it to clinical practice for a variety of reasons: they may not end up being safe or as effective in the real world; they may be too difficult, expensive or burdensome for the patient; their ‘cost-benefit ratio’ may be lower than other therapies; or the academic researchers or start-up company leading the transition from lab discovery to therapy may not have the funding, resources or knowledge to navigate this challenging, expensive and lengthy pathway to market. This makes investing in the early stages of development high risk, but also high reward.
LAU-7b was originally an invention by a group of McGill University researchers led by Dr. Danuta Radzioch that came from academic research that Cystic Fibrosis Canada funded. Wallenburg remembered the invention from his days at McGill and coincidentally was reaching out to the company when the company reached out to Cystic Fibrosis Canada. LAU-7b had just completed phase 1b of clinical trials, and Laurent Pharma was looking for investors to allow phase 2 trials to commence. It was a critical time for the company.
Ultimately, Cystic Fibrosis Canada saw the potential in this molecule as a therapy and saw the importance of running the phase 2 trial. Our investment, as well as the Cystic Fibrosis Foundation’s investment, and those that followed, allowed for the continuation of the clinical trials and bridged the gap from phase 1b to phase 2.
“The timeline for development of LAU-7b is not that unusual for an academic invention,” said Dr. Wallenburg, “but the process can absolutely be quicker and that is what we should be trying to achieve as an organization by making meaningful investments at critical timepoints in the development process.”
Many people with cystic fibrosis continue to suffer from inflammation, regardless of their mutation or access to modulators. Our research funding programs, made possible through the generosity of our donors, can not only help enhance this type of work, but also make the path to laboratory discovery easier for researchers. A treatment such as LAU-7b may help anyone with CF, so we eagerly await the results of the phase 2 study, which we hope will be made available at the North American Cystic Fibrosis Conference at the beginning of November.