for landmark discoveries of the molecular, biochemical, and functional defects underlying cystic fibrosis and the identification and development of medicines that reverse those defects and can treat most people affected by this disorder. Together, these discoveries and medicines are alleviating human suffering and saving lives.
The Shaw Prize in Life Science and Medicine 2022 is awarded in equal shares to Paul A Negulescu, Senior Vice President and Site Head, San Diego Research, Vertex Pharmaceuticals Incorporated, USA and Michael J Welsh, Professor of Internal Medicine – Pulmonary, Critical Care and Occupational Medicine, Professor of Neurosurgery, Neurology, Molecular Physiology and Biophysics and Director of Pappajohn Biomedical Institute, University of Iowa, USA for landmark discoveries of the molecular, biochemical, and functional defects underlying cystic fibrosis and the identification and development of medicines that reverse those defects and can treat most people affected by this disorder. Together, these discoveries and medicines are alleviating human suffering and saving lives.
Cystic fibrosis (CF) is one of the most common, severe single-gene disorders, affecting more than 80,000 people globally. The single gene in which the disease-causing mutations fall is called CFTR (Cystic Fibrosis Transmembrane Conductance Regulator). The CFTR protein ensures the proper flow of chloride, a component of salt, that is present in secreted body fluids such as sweat, saliva and mucus. These fluids keep cells lubricated and are thus vital for the proper function of organs. In CF patients, these secretions become thick and sticky and, rather than acting as lubricants, clog passageways, especially in the lungs. The disease is fatal. There are many different mutations in CFTR that cause the disease, but a mutation called F508del is particularly important, and is present in about 90% of patients.
The Shaw Prize in Life Science and Medicine 2022 is awarded in equal shares to Paul A Negulescu and Michael J Welsh for landmark discoveries of the molecular, biochemical, and functional defects underlying cystic fibrosis and the identification and development of medicines that reverse those defects and can treat most people affected by this disorder. Together, these discoveries and medicines are alleviating human suffering and saving lives.
Cystic fibrosis (CF) is one of the most common, severe single-gene disorders, affecting more than 80,000 people globally. The single gene in which the disease-causing mutations fall is called CFTR (cystic fibrosis transmembrane conductance regulator). The CFTR protein ensures the proper flow of chloride, a component of salt, that is present in secreted body fluids such as sweat, saliva and mucus. These fluids keep cells lubricated and are thus vital for the proper function of organs. In CF patients, these secretions become thick and sticky and, rather than acting as lubricants, clog passageways, especially in the lungs. The disease is fatal. There are many different mutations in CFTR that cause the disease, but a mutation called F508del is particularly important, and is present in about 90% of patients.
Paul A Negulescu is currently Senior Vice President and Site Head, San Diego Research, Vertex Pharmaceuticals Incorporated, USA. He received his BS and PhD in Physiology from the University of California, Berkeley, USA in 1986 and 1990 respectively. He carried out postdoctoral works at the University of California, Berkeley and the University of California, Irvine. He joined Aurora Biosciences, San Diego, USA in 1996 as one of the first employees and grew with the company to become a Senior Vice President of Discovery Biology (1999–2001). He has been appointed Senior Vice President of Research (2001–) after Vertex acquired Aurora in 2001.
Michael J Welsh is currently Professor of Internal Medicine – Pulmonary, Critical Care and Occupational Medicine, Professor of Neurosurgery, Neurology, Molecular Physiology and Biophysics and Director of Pappajohn Biomedical Institute, University of Iowa, USA. He received his MD from the University of Iowa, USA in 1974. He was a Research Fellow at the University of California, San Francisco, USA and the University of Texas, USA. He then worked at the University of Iowa, where he was successively Assistant Professor (1981–1984), Associate Professor (1984–1987), Professor of Internal Medicine (1987–), Director of Cystic Fibrosis Research Center (1988–) and Professor of Molecular Physiology (1989–). He is also an Investigator of the Howard Hughes Medical Institute (1989–), a member of the US National Academy of Sciences, the US National Academy of Medicine and the American Academy of Arts and Sciences.
