What Does It Mean To Win the Alpert Prize?
We sat down with Dr. Paul Negulescu, Senior Vice President and Site Head, San Diego Research, to find out what this award means to him.
June 21, 2018
As the head of our San Diego research site, Dr. Paul Negulescu has spent the better part of his career leading a team of passionate researchers and scientists who discovered the first and only precision medicines to treat the underlying cause of cystic fibrosis (CF). After delivering three transformative medicines, Paul’s team continues to work relentlessly to bring new and better medicines to people living with this life-shortening disease.
In recognition of these significant advances, Paul Negulescu was awarded the 2018 Warren Alpert Foundation Prize alongside Francis Collins, Lap-Chee Tsui, Michael Welsh, and Bonnie Ramsey. The Alpert Prize honors the group’s "transformative discoveries in the fields of genetics, physiology, pulmonology and pharmacology that led to the development of life-altering precision-targeted treatments for the devastating multi-organ disease cystic fibrosis (CF).”
What is the Alpert Prize and what does winning mean to you?
This Alpert Prize is an example and reminder of what we, as a scientific and medical community, are supposed to do. We are supposed to help people by understanding the cause of diseases and developing therapies that effectively treat or cure them. Seems simple, but it’s not, which is why we celebrate when it happens.
This award recognizes the remarkable path from the discovery of the CF gene to the development of therapies that treat the cause of CF. It has been a privilege for me to be part of this journey and I am honored to receive the Alpert Award with such accomplished scientists and physicians as Drs. Collins, Tsui, Welsh, and Ramsey. I am also humbled and grateful to represent the amazing team that discovered and brought these medicines to patients. This award is meaningful for all of us because it acknowledges that our work over the last 20 years has had a positive impact on people with CF and their families.
How did you first become interested in CF?
I studied epithelial cell biology and ion channels during graduate and post-doctoral work and hoped that someday I could apply this training to the discovery of new medicines. The chance to work on CF first arose when the CF Foundation approached Aurora Biosciences in 1998 to develop assays and screen for compounds to rescue the function of the CFTR protein. I was fascinated by the scientific challenge and deeply impacted by meeting people with CF and their families. It became clear to me that we had an opportunity to work on something special that could make a real difference in people’s lives. The team and I were very glad that we could continue this work after Vertex acquired Aurora in 2001.
"I was fascinated by the scientific challenge and deeply impacted by meeting people with CF and their families."
What has been the lasting impact of your work?
Twenty years ago most people in the scientific community thought that it would be impossible to rescue the function of the defective CFTR protein. So, one impact of our work is that we showed it was possible and established a scientific precedent for correction of other genetic diseases. At a personal level, it’s been deeply moving to see how the compounds we discovered are now impacting the lives of people living with CF and their families.
What have you decided to do with your prize money?
I plan to donate the prize money to an entity that is dedicated to treating another serious disease that does not have an effective therapy. I’d like to choose the recipient(s) with some of my Vertex colleagues and friends who have been on this journey with me for so long.
The CF story isn’t over for us. In fact, I believe the best is yet to come, with even better therapies to treat even more people with CF. Our job here isn’t done; we are looking and will continue to look for new compounds and pursue other methods of treatment until this disease is essentially cured.