TWENTY-TWO YEARS AGO my son Bobby was born with cystic fibrosis, a rare genetic illness that attacks the lungs and other organs. When Bobby was born, the outlook for those with cystic fibrosis was dire – with a life expectancy lasting not much past early adulthood. In fact, kids like Bobby were lucky to make it to 30.
But five years ago everything changed. That year a new treatment was approved – developed right here in Massachusetts by Vertex Pharmaceuticals – that transformed Bobby’s life and the lives of thousands of other cystic fibrosis patients across the world. It not only gave them relief from the debilitating effects of the disease but also gave them something they thought they would never have – a future.
How we got to this point is a story that exemplifies why Massachusetts has become a leader of life sciences throughout the world. We did it by creating a vibrant ecosystem bringing together academia, industry, private investment, and government to find cures and improve lives. And it is why it is so appropriate that the North American Cystic Fibrosis Conference — a gathering of thousands of cystic fibrosis scientists, clinicians, and caregivers from across the globe – chose Boston to host its annual convention this past week. It’s the largest conference of its kind in the world.
In Bobby’s case, the breakthrough treatment came just in time. Growing up, Bobby would need to take up to 100 pills a day to deal with the impacts of cystic fibrosis on his organs to help him digest his food, and to prevent life-threatening infections. He would need to spend at least 45 minutes a day breathing medication through a nebulizer and using another device for 20 minutes to loosen mucus in his lungs.
Just prior to the approval of the new treatments in 2019, Bobby was also suffering from advanced liver disease due to cystic fibrosis and was facing the possibility of a liver transplant. The symptoms of the liver disease required him to no longer play hockey and kept him from many of the activities important to a young teenager.
And then came an answer to our prayers — the discovery of medicines to treat the underlying cause of cystic fibrosis. But it didn’t happen overnight. It took decades of advocacy by patients and families fighting for attention while hoping for a cure. It took scientists like Francis Collins who helped discover the gene that caused cystic fibrosis. It took the efforts of local philanthropists like the late Joe O’Donnell, who lost his own son Joey to cystic fibrosis at age 12, to raise hundreds of millions of dollars to fund research. It took former governor governor Deval Patrick – and former governor Charlie Baker and current Gov. Maura Healey – to make growing the sciences sector in Massachusetts a top priority. And it took private industry investing billions of dollars over decades to fund the long and arduous search for an answer to this horrible illness.
The results have been nothing but transformative. Life expectancy for those with cystic fibrosis has nearly doubled. Cystic fibrosis patients can now plan lives that for so long were denied them — to graduate from college, to start a career, and to get married and have a family of their own.
Look no further than our Bobby. Since he started the new treatment five years ago this fall, he is a new man. He has gained over 20 pounds, grown a foot, and his lungs are functioning as good as they were when he was a small child.
He graduated high school and went to college, graduating from UMass Boston just last May. This summer he started a new job and career. And we could not be more proud that the field he has chosen is life sciences, with a focus on helping companies and patients achieve their dreams.
But for every Bobby there are thousands of other patients suffering from illnesses and diseases for which there are no such curative treatments. But as we have shown with cystic fibrosis here in Massachusetts, when the patients, families, scientists, academics, political leaders, and life science companies come together as one, there is no cure that is beyond our reach. Just ask Bobby.
Robert Coughlin is a former Massachusetts state representative, undersecretary of economic development, and CEO of MassBIO. He is currently managing director of JLL’s life sciences industry practice.
The post We need more stories like Bobby’s appeared first on CommonWealth Beacon.