The fight to end cystic fibrosis relies on local chapter – Albany Times Union

ALBANY People in the rare disease community know that in order to make any progress finding cures, they have to band together.

So it was that in 1955 a group of concerned parents whose children werent expected to live past 5 years old came together to create the Cystic Fibrosis Foundation. Few people at the time had the genetic disease, which affects the way a persons body makes mucus and limits their ability to breathe over time. Today, 35,000 people in the U.S. are living with it.

Since the 1950s, treatment of the fatal disease has come a long way. Babies born with cystic fibrosis today are expected to live into their mid-40s and beyond in some cases. And a central reason why is the foundation those parents launched.

Working alongside the CF community, we have effectively transformed a genetic disease in a single generation making CF one of the most amazing stories in medicine today, said Kate Townsend, executive director of the foundations Northeastern New York chapter. Nearly every CF-specific drug available today was made possible with our financial support.

The foundations venture philanthropy model is admired around the world, she said.

By providing funding to companies during the early stages of drug discovery and development when the risk of failure is at its highest the organization incentivizes research that companies would be much less likely to undertake, especially for rare diseases that impact a sliver of the population.

But such research is essential for the people impacted by rare diseases.

The cystic fibrosis community saw an especially significant breakthrough in 1989 when scientists supported by the foundation discovered the defective gene responsible for the disease cystic fibrosis transmembrane conductance regulator and its protein product. The discovery paved the way for future treatments and in 1993 the U.S. Food and Drug Administration approved the first drug developed with financing from the foundation made specifically for cystic fibrosis.

More drugs and therapies have been developed since, but the community is particularly excited these days about advances in gene-based therapies, which seek to treat the underlying cause of the disease rather than its symptoms,Townsend said.

In early November, the foundation announced a first-of-its-kind collaboration with Pioneering Medicines, a division of the life sciences venture capital firm Flagship Pioneering, to spur the development of gene-based therapies that could potentially treat the disease. The foundation will invest up to $110 million in the project, with an initial commitment of $20 million, the organization reported on its website.

Such investments are made possible by the on-the-ground work of the foundations 70 chapters.

Our primary function in the local chapter is to connect with local families to support their needs, and to fund-raise through both individual giving and special events, Townsend said.

The Northeastern New York chapter based in Albany routinely hosts events around the region to raise money for the foundation, including walks, runs, climbs and galas. The chapter is offering a $75 Beer Trail Passport that gives buyers a special discount to 25 local restaurants, breweries, wineries and distilleries through Dec. 30. Information about the passport can be found at https://events.cff.org/NENYBeerTrail.

Townsend said the chapter was able to adjust when the coronavirus pandemic made in-person events impractical. The 65-mile challenge in which individual donors raise funds and complete 65 miles of walking, running, biking, hiking or kayaking over the course of six weeks could easily pivot to become a virtual event, she said.

So you know we just got a little more creative, innovative and we still were able to fund-raise, just not gathering large groups of people in person, Townsend said.

The hope with rare disease fundraising is that people beyond just those directly impacted by the disease will be willing to donate, she said.

Advances in gene-based therapies represent an opportunity to end CF as we know it, she said. We are committed to exploring and advancing all science that has real potential to deliver treatments for the underlying cause of CF ...but we know that no pace is fast enough when you or a loved one is waiting for a breakthrough.

Cystic Fibrosis Foundation Northeastern New York Chaptergive.cff.org/northeastern-newyork

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The fight to end cystic fibrosis relies on local chapter - Albany Times Union