In the history of CF research and therapies, collaboration is king
The progress researchers have made is staggering, but it required teamwork
The year is 1955, and a group of parents, including the late Doris Tulcin, decides to create an organization dedicated to improving care for children with cystic fibrosis (CF). That organization, the Cystic Fibrosis Foundation (CFF), continues to thrive today.
The year is 1989, and Lap-Chee Tsui, Jack Riordan, and colleagues at the Hospital for Sick Children in Toronto, along with Francis Collins and his colleagues at the University of Michigan, announce one of the most significant breakthroughs in medical history: They have discovered the gene that causes CF. This achievement, which received financial support from the CFF, occurred only after researchers began working together in earnest a few years prior. Researchers in a lab in London who were also trying to identify the genetic cause of CF faxed the team in Toronto to congratulate them, and then popped a bottle of Champagne to celebrate.
The year is 2000, and the CFF is in the early stages of its venture philanthropy model, in which a nonprofit collaborates with a for-profit company to achieve its goals. The foundation’s first significant investment under that model targeted “high throughput screening, a then-novel technology that uses robots to test the therapeutic properties of thousands of chemical compounds per day in cells in laboratory dishes,” the CFF notes on its website.
The year is 2025, and every year for the past half-century, people with CF, along with their families and friends, plus researchers, clinicians, and tens of thousands of others, have come together to raise millions of dollars for better medications and one day, a cure for CF.
Multiple ‘shots on goal’
When Tulcin and that group of visionary parents first started the CFF, cystic fibrosis was considered a fatal pediatric disease. Those parents understood the importance of community and working together. They understood that the whole is greater than the sum of its parts.
Despite all the challenges that come with CF, fortunately, it is caused by a single defective gene, making it a strong candidate for potential genetic therapies. Many other diseases have far more complicated causes. Like so many other stories, a breakthrough occurred after a critical mass of scientific discoveries was reached by dedicated researchers. In this case, after multiple scientists decided to work together, they were able to isolate the gene that causes CF. None of them could have done it alone.
Robert Beall, the former president and chief executive officer of the CFF, was fond of saying that it is important to have a lot of “shots on goal” in the CF therapy pipeline. This idea proved to be innovative, but it wasn’t as simple as encouraging pharmaceutical companies to develop new medications, because CF wasn’t considered a financially viable market. This led to the venture philanthropy model I mentioned above, whereby funds are invested in companies and clinical trials. Vertex Pharmaceuticals ultimately scored that goal and now markets several CFTR modulator therapies.
Investments continue today, and I believe there is a direct through line from the funds that families raise at the CFF’s Great Strides walks across the country to the almost unfathomable increase we’ve seen in CF life expectancy. It’s astounding to witness.
People often say that competition breeds innovation, but I don’t believe in that premise. While healthy competition can lead to progress, I believe that excessive competition can cause isolation, insecurity, anxiety, defensiveness, and secrecy. Collaboration, on the other hand, is synergistic. Working together has driven the incredible progress we’ve seen in the CF community. Working together also provides a sense of meaning, fulfillment, and joy.
The story of cystic fibrosis — a story filled with tragedy and triumph — is still being written. Perhaps the most humbling and inspiring part is that much of it is defined by collaboration and community. None of us could’ve done any of this alone, yet every single one of us has been a crucial cog in the machine. One day, that machine will produce a cure.
Note: Cystic Fibrosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Cystic Fibrosis News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to cystic fibrosis.
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