When people refer to the NIH as the “National Institutes of Hope,” Dr. Bill Gahl is one of the many people who come to mind. Dr. Gahl is a medical geneticist working to help patients with rare and undiagnosed diseases. His research group focuses on inborn errors of metabolism, which include defects in the body’s biochemical processes caused by rare genetic disorders, such as cystinosis, Hermansky-Pudlak syndrome, alkaptonuria, and ciliopathies. Transcending biomedical boundaries to take advantage of the IRP’s unique team-science environment, Dr. Gahl led the creation of the NIH’s Undiagnosed Diseases Program to provide answers and possible treatments for people with mysterious conditions that have long eluded diagnosis.
Pancreatic cancer kills more than 40,000 Americans each year, and just 6% of patients survive five years or more after diagnosis, because the disease metastasizes very early in its development and is resistant to most current treatments. Dr. Christine Alewine is a physician-scientist exploring new treatment strategies for pancreatic cancer. Her lab and clinic are testing and refining two recombinant immunotoxins that target a protein called mesothelin that is present on the surface of several types of cancer tumor cells, including pancreatic, ovarian, and some lung cancers. If clinical trials show that the drug is safe and effective, it could lead to much needed systemic therapies for these cancer patients.
Dr. Nehal Mehta currently directs the largest ongoing cohort study to date in psoriasis, which is an inflammatory skin disease associated with increased cardiovascular risk. His research is showing that local inflammation in different areas of the body such as the teeth, scalp, knees, skin, or gut has systemic consequences. And treating that local inflammation can help heal heart disease.
This page was last updated on Friday, January 14, 2022
