BOSTON, May 13, 2021 /PRNewswire/ — In remarks at the 2021 Warrior Call event last night, a signature event for the VHL Alliance during VHL (von Hippel-Lindau disease) Awareness month, William G. Kaelin Jr., MD, noted incredible progress in understanding how tumors grown and possible breakthroughs in cancer treatment. Dr. Kaelin, based at Harvard’s Dana-Farber Cancer Institute, is co-recipient of the 2019 Nobel Prize in Medicine for his team’s work on Hypoxia-Inducible factor (HIF), an important tumor growth mechanism.
Dr. Kaelin discussed the significance of the research that he and his team conducted into the influence of HIF on cells. Their work led to the discovery of HIF-2α. Normally, the VHL gene regulates the production of proteins involved in controlling tumor growth. In VHL patients the gene’s regulating mechanism fails, resulting in the over-expression of HIF proteins. This failure allows the growth of benign and malignant tumors. Understanding this mechanism in VHL patients is an important step in unlocking a cure for cancer.
Last night, while discussing his work with VHL patients, caregivers, and other interested groups at a virtual event hosted by the VHL Alliance, Dr. Kaelin talked about how VHL patients have impacted him. “In my first interaction with meeting VHL patients, over 20 years ago, I was struck by the patients who are so courageous, have such resilience, and have such marvelous philosophies about life… and I thought, wouldn’t it be great to get to a point where we can do something better for them. They are so inspirational. Show me tumors shrinking and patients living better lives, that is what it is all about to me.”
In March the FDA granted priority review of a new drug application for a promising drug that inhibits the HIF-2α protein, much like a normal VHL gene would. In a phase II trial for the treatment of VHL disease-associated clear cell renal carcinoma, nearly a third of enrolled patients showed tumor shrinkage of at least 30%. In addition, 86.9% showed some decrease in target lesions. The drug is expected to have broad use in treating kidney and other cancers.
VHL Alliance Executive Director Chandra Clark said, “Each May we celebrate VHL Awareness Month. For nearly 30 years the VHLA has served the patient and caregiver community and funded research. We have boundless gratitude for Dr. Kaelin and dozens of other researchers who dedicate their time, resources, and intellect to understanding this disease and searching for treatments and cures. The VHL community is particularly fortunate to have researchers who understand and empathize, with the human impact of this disease.”
Von Hippel-Lindau disease is a genetic condition caused by a mutation in the VHL gene, which regulates the major feeding pipeline of every tumor. VHL patients battle a lifetime of unpredictable tumor growth in the brain, spine, kidneys, and many other areas.
Curing VHL is one step closer to curing many other forms of cancer.
The VHL Alliance (VHLA) is a 501(c)(3) non-profit organization founded in 1993, which is dedicated to research, education, and support to improve awareness, diagnosis, treatment, and quality of life for those affected by VHL.
Our vision is Curing Cancer through VHL.
For information about VHL and the VHL Alliance, please visit vhl.org.
Contact: Heidi Leone, VHL Alliance
SOURCE VHL Alliance