Researcher-led Phase II trials at the University of Texas MD Anderson Cancer Center show that treatment with Belztifan, a small molecule inhibitor of hypoxia-inducing factor (HIF) -2a, is a potent clinical practice in patients with renal cell carcinoma. Shows that activity has been achieved. (RCC) and von Hippel-Lindau (VHL) disease-related non-renal cell carcinoma neoplasms. Research today The New England Journal of Medicine.
Objective response rate Patience After a median follow-up of 21.8 months, the RCC was 49%. In addition, 92% of patients had a reduced size of the target lesion. In 24 months Progression-free survival It was 96%.
Originally known as MK-6482, Belzutifan Approved by the Food and Drug Administration August 13, 2021, based on previously reported results from this trial. This is the first FDA-approved treatment for VHL disease.
“Patients with von Hippellindau disease are at risk of developing several types of cancer and require repeated surgical procedures to manage the tumor,” said Principal Investigator Eric Jonash, MD. Professor Oncology) said. “These results will significantly change the way we manage patients with VHL disease and provide influential benefits to the majority of patients with VHL.”
Treatment of cancer associated with VHL disease represents an important unmet need
VHL disease is caused by a rare hereditary mutation VHL It is a gene and is associated with tumors formed by multiple organs. Some of these tumors are benign, but they can grow and damage organs. VHL can also cause cancerous tumors in the kidneys and pancreas. RCC affects about 40% of people with VHL disease and is one of the most common causes of disease-related death in these patients.
NS VHL Mutations cause cells to lose the ability to respond appropriately to oxygen levels, leading to the accumulation of HIF proteins.This process falsely informs cells that they are deficient in oxygen, causing blood vessel formation and operation. tumor growth. Inactivation of VHL tumor suppressor proteins is also observed in more than 90% of sporadic RCC tumors. Belzutifan directly targets HIF-2a and interferes with the growth, spread and abnormal blood vessel development of cancer cells.
Treatment of renal tumors associated with VHL disease consists of active monitoring until surgery is required for tumors larger than 3 cm to prevent metastatic disease. Repeated surgical procedures can cause serious complications, as many patients develop renal failure. Surgery does not cure VHL disease in RCC patients, but it is intended to prevent death from metastatic kidney cancer.
“Half of the patients in this study responded objectively, and almost all patients saw a reduction in lesion size,” Jonash said. “Patients with VHL can get a better quality of life because this treatment can delay or avoid the need for surgery.”
Studies suggest promising new treatment options for VHL-related kidney cancer
The single-arm clinical trial enrolled 61 patients in 11 centers in the United States, Denmark, France and the United Kingdom. In this study, germline mutation diagnosis of VHL disease, no previous systemic cancer treatment, measurable non-metastatic RCC tumors, and adult patients with a performance status of 0 or 1 in the Eastern Cooperative Oncology Group (ECOG). Was registered.
Patients were orally administered Belztifan once daily until the disease progressed, unacceptable toxicity, or a decision to discontinue. Tumor size was assessed at screening and every 12 weeks thereafter. No patient suffered from progressive disease during treatment, and 54 patients (89%) continued treatment.
Most treatment-related adverse events (AEs) were grade 1 or 2 in severity. The most common adverse events were anemia (90%) and fatigue (66%). There were no deaths due to treatment-related adverse events.
Responses were also observed in 77% of patients with VHL-related pancreatic lesions and 30% of patients with VHL-related central nervous system hemangioblastoma. Of the 12 patients with baseline retinal hemangioblastoma, 100% were rated as showing improvement.
“These data suggest that HIF-2a inhibition provides effective treatment options with manageable side effects for patients with VHL-related renal cell carcinoma and other VHL-related tumors,” Jonasch said. Said. “We are excited to offer this impactful treatment to patients who have been waiting for new options for a long time.”
The study is limited due to the lack of controls and the small sample size. Designing a randomized controlled trial is an ethical challenge, as Jonasch explained, as there are no approved non-surgical treatment options for VHL disease.Future studies may include testing whether this treatment can prevent the development of new lesions in VHL patients. disease..
New England Journal of Medicine (2021). www.nejm.org/doi/full/10.1056/NEJMoa2103425
University of Texas MD Anderson Cancer Center
Quote: Bell’s Tiffin elicited a strong response in patients with kidney cancer (November 24, 2021) associated with von Hippel-Lindau disease. von.html
This document is subject to copyright. No part may be reproduced without written permission, except for fair transactions for personal investigation or research purposes. Content is provided for informational purposes only.
Belzutifan induced strong responses in patients with von Hippel-Lindau disease-associated kidney cancer Source link Belzutifan induced strong responses in patients with von Hippel-Lindau disease-associated kidney cancer
The post Belzutifan induced strong responses in patients with von Hippel-Lindau disease-associated kidney cancer appeared first on California News Times.