Dr. Eric Jonasch

von Hippel-Lindau (VHL) disease is a rare inherited mutation that affects approximately 10,000 individuals in the United States. However, these patients have hope on the horizon, according to Dr. Eric Jonasch.

A recent study — presented at the 2020 American Society of Clinical Oncology (ASCO) Virtual Scientific Meeting – showed that treatment with MK-6482 appeared to be well tolerated and induced clinical responses among patients with VHL–associated renal cell carcinoma (RCC).

The trial showed an objective response rate of 27.9%. Moreover, eight patients who had an unconfirmed response showed an objective response rate of 41.0%. Additionally, 86.9% of patients had a decrease in the size of their target lesions and median time to response was 5.5 months.

Jonasch, professor of genitourinary medical oncology at The University of Texas MD Anderson Cancer Center, discussed the trial results with 

, offering hope on the horizon for these individuals.

So, VHL disease at this time does not have an FDA approved therapy. It’s a hereditary disorder that affects the eyes, brain, spine, pancreas, adrenal glands, and kidneys. These individuals who have this — there are about 10,000, in the United States – basically face a lifetime of surveillance scans and surgical interventions or other types of interventions.

So, having something that can actually shrink these without undergoing surgery would be a huge boost.

The hope here is that this agent be approved and that in individuals who do have lesions that are starting to get to a size where they might need some type of intervention that they could get this drug instead of a surgical intervention and that they could be on this for some period of time. That would either delay or prevent the need for that surgical intervention.

The long-term dream scenario would be that, this is a disease in adolescents and young adults, so the first manifestations can occur in childhood or most commonly in adolescence or young adulthood, we could come up with a prevention strategy where individuals would receive this drug and then prevent the development of organ manifestations and that would require a follow-up study asking a different question. That could really help us to help this patient population more.

From a patient perspective, it’s been an incredible joy seeing how this drug really has helped the lives of individuals living with VHL. Being personally involved in seeing how this has provided them with benefit and as a person who has been working with individuals with VHL disease now for 20 years, it makes me incredibly happy to see that we finally have something that can help these people.

Videos

The HIF-2a Inhibitor induced clinical responses among patients with von Hippel-Lindau disease–associated kidney cancer, according to results presented at the 2020 ASCO Virtual Scientific Program.
 

Dr. Eric Jonasch

MK-6482 Shows Promise in von Hippel-Lindau Disease-Associated Renal Cell Carcinoma

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