Opening Remarks and Special Lecture on Belzutifan Development | Medical Symposium 2024 скачать в хорошем качестве

Opening Remarks and Special Lecture on Belzutifan Development | Medical Symposium 2024

A special video message from Dr. Kim Rathmell, the Director of the U.S. National Cancer Institute, opens the 16th International VHL Medical Symposium and highlights recent breakthroughs in VHL research. She discusses the persistence of the VHL research community, noting that challenges once thought insurmountable—such as targeting the "undruggable" HIF-2 protein—are now being addressed through innovation and collaboration. [YouTube:    / @ncigov;   X: @theNCI, @NCIDirector; LinkedIn:   / ;   Facebook:   / cancer.gov;   Instagram: @nationalcancerinstitute] Dr. James Brugarolas then shares his inspiring journey of confronting an undruggable target (HIF-2 alpha) and ultimately developing belzutifan. He explains that in healthy cells, the VHL gene regulates proteins known as hypoxia-inducible factors (HIFs), which help cells respond to low oxygen levels. In VHL disease, mutations disable this regulation, leading to uncontrolled HIF activity and tumor growth. Dr. Brugarolas and his team identified a small pocket in the HIF-2 alpha protein that could be targeted by a drug. They developed a molecule specifically designed to fit into this pocket, effectively blocking the HIF-2 alpha protein from promoting tumor growth. This innovative approach led to the creation of belzutifan, which offers hope for controlling VHL-related tumors without the need for surgery. He also addresses the challenge of drug resistance, where tumors can adapt and make the treatment less effective over time. His team is exploring solutions, such as using small interfering RNA (siRNA) therapies to silence the HIF-2 alpha gene in tumors. His presentation emphasizes the importance of identifying which tumors are most likely to respond to HIF-2 inhibitors and developing biomarkers to predict treatment success.