Miguel Bronchud, Veteran Cancer Clinician and Researcher and Co-Founder of Regenerative Medicine Solutions, shared on LinkedIn:
“Good News from MD Anderson Houston Texas for a Rare Type of Cancer Prone Gene Mutation- With the FDA’s recent approval of belzutifan, a drug that promises to be a game changer?
The FDA approved the drug after a clinical trial involving 61 patients at MD Anderson and 10 other cancer centers produced encouraging results.
The clinical trial was designed to test the drug’s effectiveness in VHL patients who developed a type of kidney cancer called renal cell carcinoma.
This is one of the most common cancers in people with von Hippel-Lindau disease. At some point in their lives, about 70% of VHL patients will develop renal cell carcinoma.
After taking belzutifan daily for 18 months, almost half of participants saw their kidney tumors shrink at least 30%.
The drug also shrank tumors in the brain, spine and pancreas.
Belzutifán, marketed under the name Welireg, is an oral medication that acts as a hypoxia-inducible factor 2 alpha (HIF-2α) inhibitor.
What is it used for?
- Clear cell renal cell carcinoma (RCC): Treatment in adult patients with advanced disease who have already received prior immunotherapy or targeted therapies.
- Von Hippel-Lindau (VHL) disease: Treatment in adults with kidney tumors, hemangioblastomas of the central nervous system, or pancreatic neuroendocrine tumors that do not require immediate surgery.
How it works? It blocks the HIF-2α protein to prevent it from regulating genes that promote angiogenesis (growth of new blood vessels) and the proliferation of tumor cells.
The importance of oxygen has been understood for centuries, but how cells adapt to changes in levels of oxygen has long been unknown. William Kaelin, Peter Ratcliffe, and Gregg Semenza discovered how cells can sense and adapt to changing oxygen availability. During the 1990s they identified a molecular machinery that regulates the activity of genes in response to varying levels of oxygen.
People with a rare, inherited disorder called von Hippel-Lindau disease develop cancerous and noncancerous tumors in up to 10 different parts of their bodies.
‘Having von Hippel-Lindau disease can be overwhelming,’ says Eric Jonasch, who heads MD Anderson’s von Hippel-Lindau Clinic.
‘Tumors recur frequently throughout your lifetime, and they could only be removed surgically – until now.’
Genetic mutation leads to tumor growth
Von Hippel-Lindau disease is caused by a mutation in the VHL gene.
Normally, this gene keeps cell growth in check. But when the gene mutates, the gene malfunctions and cells multiply out of control.
Masses of cells develop into cancerous and noncancerous tumors in the brain, spine, eyes, ears, lungs, kidneys, pancreas, liver, adrenal glands, and reproductive organs.
As fast as surgeons removed them, new ones appear. A constant cycle of surveillance, surgery and recovery.
Other articles featuring Miguel Bronchud on OncoDaily.