2021 is a banner year for the Mass General Cystic Fibrosis Marathon Team; the squad includes four runners with CF among its 14 runners. That would have been impossible even two years ago.
But in 2019, the FDA approved Trikafta®, a medicine that dramatically improves the lung capacity of CF patients. “Trikafta® is just short of a miracle,” says Allen Lapey, MD, Marathon team captain and emeritus director of the Cystic Fibrosis Center. He explains that the drug addresses two of the most common genetic mutations that cause the disease. “It’s a transformative therapy that helps adults who are quite sickly have new energy and a new life. It actually restores lung function.”
“Our [cystic fibrosis] program is unique because it serves both children and adults.”
Lengthening Lives
In the past, hospitals had no adult programs, because so few cystic fibrosis patients lived that long. With improved treatments — including aerosol antibiotics, bronchial dilators, specialized nutrition and exercise — the need for adult care has increased dramatically.
“We started our adult CF program 20 years ago, and now the majority of our patients are over 18 years old,” says Dr. Lapey, one of the longest practicing CF doctors in the nation, having started his career at Mass General in 1970. “Our program is unique because it serves both children and adults.”
The philanthropic support provided by the marathon team and other donors is critical to advancing research and transforming the future of CF care for patients around the world.
The Center takes a team approach to treating the disease, with specialists collaborating to combat CF’s most common complexities and symptoms, including pulmonologists, gastroenterologists, endocrinologists, obstetrics specialists, and psychiatrists.
“For example, we have been leaders from the very beginning in recognizing that mental health is a huge issue in the lives of CF patients,” explains Dr. Lapey. “We are leading the effort in this area globally, and marathon funds got that started.”
Incubating Innovative Researchers
Despite recent advances, there is still no cure for CF. Many patients are living with just 20 percent lung capacity while awaiting a lung transplant. That’s why research funding is critical to the Cystic Fibrosis Center’s mission. The Center’s research priorities include investigations into potential gene therapies, regenerative medicine to restore organ function, clinical trials to bring new treatments to patients, improvements in PET scan technology to treat airway inflammation and studies to develop hormonal treatments for the 30 percent of CF patients who develop diabetes.
Mass General receives significant funding from traditional grant-making institutions like the National Institutes of Health (NIH). Usually, this type of institutional grant funding is awarded to low-risk investigations led by scientists with long track records of success in their fields.
“We use the money raised by the marathon team as bridge funding to launch the work of young physicians who want to dedicate their careers to CF research.”
However, in research, it is well-known that transformative advances can come from the outside-the-box ideas. Investigators pursuing new or unproven areas of research can find it challenging to secure NIH funding for the kind of biomedical science that turns big ideas into bold breakthroughs. As a result, the philanthropic support provided by the marathon team and other donors is critical to advancing research and transforming the future of CF care for patients around the world.
“These researchers are early in their careers, and don’t yet have the credentials or research results to win the big NIH grants,” says Dr. Lapey. “We use the money raised by the marathon team as bridge funding to launch the work of young physicians who want to dedicate their careers to CF research.”
To learn more about the Cystic Fibrosis Center at Mass General, please contact us.
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