Lap-Chee Tsui
2018 Warren Alpert Foundation Prize Symposium
Posted on by Dr. Francis Collins
I was deeply honored to be among the five recipients of the 2018 Warren Alpert Foundation Prize. All five recipients were recognized for their discoveries in contributing to the development of life-altering treatments for cystic fibrosis (CF). The other recipients were: Paul Negulescu, Vertex Pharmaceuticals, Boston; Bonnie Ramsey, University of Washington School of Medicine and Seattle Children’s Research Institute; Lap-Chee Tsui, The Academy of Sciences of Hong Kong; Michael Welsh, University of Iowa, Iowa City. We were recognized at an afternoon symposium titled Cystic Fibrosis: From Gene Discovery to Basic Biology to Precision Medicines. The symposium was held at Harvard Medical School, Boston, on October 4, 2018. The video posted here shows my presentation that afternoon. But if you would like to see more, there is a full video of this fantastic symposium.
Cystic Fibrosis: Keeping the Momentum Going
Posted on by Dr. Francis Collins
Caption: Lower left, me, Lap-Chee Tsui, and John Riordan celebrating our discovery of the cystic fibrosis gene. Right, Robert J. Beall, me, and Doris Tulcin at a November Cystic Fibrosis Foundation event honoring Dr. Beall.
It’s been more than a quarter-century since my colleagues and I were able to identify the gene responsible for cystic fibrosis (CF), a life-shortening inherited disease that mainly affects the lungs and pancreas [1]. And, at a recent event in New York, I had an opportunity to celebrate how far we’ve come since then in treating CF, as well as to honor a major force behind that progress, Dr. Bob Beall, who has just retired as president and chief executive officer of the Cystic Fibrosis Foundation.
Thanks to the tireless efforts of Bob and many others in the public and private sectors to support basic, translational, and clinical research, we today have two therapies from Vertex Pharmaceuticals that are targeted specifically at CF’s underlying molecular cause: ivacaftor (Kalydeco™), approved by the Food and Drug Administration (FDA) in 2012 for people with an uncommon mutation in the CF gene; and the combination ivacaftor-lumacaftor (Orkambi™), approved by the FDA in July for the roughly 50 percent of CF patients with two copies of the most common mutation. Yet more remains to be done before we can truly declare victory. Not only are new therapies needed for people with other CF mutations, but also for those with the common mutation who don’t respond well to Orkambi™. So, the work needs to go on, and I’m encouraged by new findings that suggest a different strategy for helping folks with the most common CF mutation.
