Von Hippel–Lindau disease

SurvivorNet Expands Its Groundbreaking Resources Into Rare and Chronic Diseases

Retrieved on: 
Thursday, June 9, 2022

NEW YORK, June 9, 2022 /PRNewswire/ -- SurvivorNet, the nation's leading information platform for cancer, is branching out to helpmillions of people seeking better information to manage their rare and chronic diseases.

Key Points: 
  • NEW YORK, June 9, 2022 /PRNewswire/ -- SurvivorNet, the nation's leading information platform for cancer, is branching out to helpmillions of people seeking better information to manage their rare and chronic diseases.
  • "There are thousands of diseases which now have remarkable new therapies being brought to market for people with very few or no treatment choices.
  • The information options for these people are too often lacking, and that's the void weare filling," said SurvivorNet CEO Steve Alperin.
  • "Along the way, we discovered that people living with other chronic diseases also deserve better information.

XORTX Therapeutics Announces Filing of Provision Patent for Polycystic Kidney Disease

Retrieved on: 
Tuesday, December 21, 2021

The Company remains dedicated to redefining kidney disease in the future and advance our lead program XRx-008 - on behalf of individuals with progressing kidney disease due to autosomal dominant polycystic kidney disease.

Key Points: 
  • The Company remains dedicated to redefining kidney disease in the future and advance our lead program XRx-008 - on behalf of individuals with progressing kidney disease due to autosomal dominant polycystic kidney disease.
  • Polycystic Kidney Disease is characterized by an array of anatomic and physiologic abnormalities, for example hypertension, endothelial dysfunction, cardiovascular disease, liver disease, cardiovascular disease involving large, medium and small caliber blood vessels as well as other health problems are frequently encountered by patients with polycystic kidney disease.
  • Polycystic kidney disease is associated with genetic changes and described by two categories- autosomal dominant polycystic kidney disease (ADPKD) and autosomal recessive polycystic kidney disease (ARPKD).
  • ADPKD is leading cause of polycystic kidney disease is autosomal dominant polycystic kidney disease and contributes approximately 90% of all individuals.

VHL Community Celebrates Historic Treatment

Retrieved on: 
Wednesday, August 18, 2021

"The approval of belzutifan for treatment of VHL-related renal cancers, hemangioblastomas, and neuroendocrine tumors of the pancreas is a game-changer in the treatment of VHL patients," said Othon Iliopoulos, MD, Director, VHL Comprehensive Clinical Care Center at Massachusetts General Hospital and Harvard Medical School, Associate Professor of Medicine at Harvard Medical School.

Key Points: 
  • "The approval of belzutifan for treatment of VHL-related renal cancers, hemangioblastomas, and neuroendocrine tumors of the pancreas is a game-changer in the treatment of VHL patients," said Othon Iliopoulos, MD, Director, VHL Comprehensive Clinical Care Center at Massachusetts General Hospital and Harvard Medical School, Associate Professor of Medicine at Harvard Medical School.
  • The VHL Alliance (VHLA) is the preeminent resource and clearinghouse for those affected by von Hippel-Lindau disease, including patients, caregivers, researchers, and the medical community.
  • The VHL Alliance's vision is Curing Cancer through VHL.
  • For information about VHL and the VHL Alliance, please visit vhl.org .

Exelixis Announces Results From Two Renal Cell Carcinoma Cohorts of the COSMIC-021 Trial of Cabozantinib in Combination With Atezolizumab

Retrieved on: 
Monday, September 21, 2020

Patients received atezolizumab in combination with either a 40 mg or 60 mg daily dose of cabozantinib.

Key Points: 
  • Patients received atezolizumab in combination with either a 40 mg or 60 mg daily dose of cabozantinib.
  • Exploratory cohorts have the option to be expanded up to 80 patients (cabozantinib) and 30 patients (atezolizumab) total.
  • The von Hippel-Lindau tumor suppressor gene inhibits hepatocyte growth factor/scatter factor-induced invasion and branching morphogenesis in renal carcinoma cells.
  • Markedly increased amounts of messenger RNAs for vascular endothelial growth factor and placenta growth factor in renal cell carcinoma associated with angiogenesis.

FDA Grants Breakthrough Therapy Designation to Merck’s Novel HIF-2α Inhibitor MK-6482 for Treatment of Certain Patients With Von Hippel-Lindau Disease- Associated Renal Cell Carcinoma

Retrieved on: 
Wednesday, July 29, 2020

The FDA also granted orphan drug designation to MK-6482 for VHL disease.

Key Points: 
  • The FDA also granted orphan drug designation to MK-6482 for VHL disease.
  • These designations for MK-6482 support the potential of targeting HIF-2 in certain patients with von Hippel-Lindau disease, who currently have limited treatment options and face an increased risk for benign tumors as well as several types of cancer, including renal cell carcinoma.
  • Proteins known as hypoxia-inducible factors, including HIF-2, can accumulate in patients when VHL, a tumor-suppressor protein, is inactivated.
  • Patients with VHL disease are at risk for benign blood vessel tumors as well as several cancers, including RCC.

Merck’s Novel HIF-2α Inhibitor Showed an Objective Response Rate of Nearly 30% in Patients with von Hippel-Lindau (VHL) Disease-Associated Clear Cell Renal Cell Carcinoma

Retrieved on: 
Wednesday, May 13, 2020

Von Hippel-Lindau disease is a rare hereditary condition affecting multiple organs, which puts patients at risk for several cancers, including renal cell carcinoma.

Key Points: 
  • Von Hippel-Lindau disease is a rare hereditary condition affecting multiple organs, which puts patients at risk for several cancers, including renal cell carcinoma.
  • Follow Merck on Twitter via @Merck and keep up to date with ASCO news and updates by using the hashtag #ASCO20.
  • This study is a Phase 2, open-label, single-arm trial evaluating MK-6482 for the treatment of VHL-associated ccRCC (ClinicalTrials.gov, NCT03401788 ).
  • Patients received MK-6482 120 mg orally once daily until disease progression, unacceptable toxicity, or investigators or patients decision to withdraw.

Genetic Cancer Patients Report COVID-19 Crisis Impacts Medical Care

Retrieved on: 
Wednesday, April 22, 2020

VHL, or von Hippel-Lindau disease, is a genetic disorder characterized by tumors in up to ten areas of the body.

Key Points: 
  • VHL, or von Hippel-Lindau disease, is a genetic disorder characterized by tumors in up to ten areas of the body.
  • The tumors can be benign or cancerous, and appear and grow unpredictably throughout a patient's life.
  • Renal and pancreatic neuroendocrine cancers, paralysis, loss of vision and hearing, are among the dangers faced by VHL patients.
  • The VHL Alliance (VHLA) is the preeminent resource and clearinghouse for those affected by von Hippel-Lindau disease, including patients, caregivers, researchers, and the medical community.

Peloton Therapeutics Announces Completion of Enrollment in Phase 2 Trial of PT2977 for Treatment of Von Hippel-Lindau Disease-Associated Renal Cell Carcinoma

Retrieved on: 
Tuesday, April 9, 2019

This open-label Phase 2 trial will evaluate the efficacy and safety of PT2977 in patients with VHL disease-associated RCC.

Key Points: 
  • This open-label Phase 2 trial will evaluate the efficacy and safety of PT2977 in patients with VHL disease-associated RCC.
  • The potential impact of PT2977 on VHL disease-associated non-RCC tumors, as well as the tolerability and pharmacokinetics of PT2977, will also be evaluated in this trial.
  • Further information on the clinical trial of PT2977 can be found on clinicaltrials.gov (Study identifier: NCT03401788).
  • The company is initially focused on developing PT2977 for the treatment of kidney cancer, specifically metastatic clear cell RCC and VHL disease-associated RCC.

Peloton Therapeutics Announces Clinical Data Update for PT2977 at European International Kidney Cancer Symposium

Retrieved on: 
Monday, April 1, 2019

Peloton Therapeutics, Inc., a clinical-stage biopharmaceutical company advancing first-in-class oral medicines for cancer and other serious conditions, announced today the presentation of data from the Phase 2 portion of the companys Phase 1/2 clinical trial of its lead drug candidate, PT2977, at the Fourteenth European International Kidney Cancer Symposium in Dubrovnik, Croatia.

Key Points: 
  • Peloton Therapeutics, Inc., a clinical-stage biopharmaceutical company advancing first-in-class oral medicines for cancer and other serious conditions, announced today the presentation of data from the Phase 2 portion of the companys Phase 1/2 clinical trial of its lead drug candidate, PT2977, at the Fourteenth European International Kidney Cancer Symposium in Dubrovnik, Croatia.
  • The Phase 1 portion included a once daily administration schedule dose escalation cohort of 37 patients with advanced solid tumors.
  • PT2977 has demonstrated anti-tumor activity with a favorable safety profile in an early-stage clinical study in patients with solid tumors.
  • Peloton is also currently evaluating PT2977 in an international Phase 2 trial in von Hippel-Lindau (VHL) disease-associated RCC.

VHLA Announces 2018 Research Grant Recipients

Retrieved on: 
Thursday, November 29, 2018

BOSTON, Nov. 29, 2018 /PRNewswire/ --Von Hippel-Lindau Alliance (VHLA), a national non-profit committed to research, education, and awareness of VHL, today announced it awarded three research grants totaling nearly $300,000 as part of the 2018 VHLA Research Grant Program.

Key Points: 
  • BOSTON, Nov. 29, 2018 /PRNewswire/ --Von Hippel-Lindau Alliance (VHLA), a national non-profit committed to research, education, and awareness of VHL, today announced it awarded three research grants totaling nearly $300,000 as part of the 2018 VHLA Research Grant Program.
  • Since its inception in 1998, the VHLA Research Grant program has awarded more than $2 million toward research.
  • In addition to the VHLA Research Grant Program, VHLA also co-developed MyVHL: The Patient Natural History Study, which collects key patient data to help researchers better understand VHL and other types of cancers.
  • VHLA is the leading funder of VHL research, funding $2 million in grant to support studies designed to find a cure.