ASCO GU Presentation: Positive Data on Cabometyx for Renal Cell Carcinoma


The American Society of Clinical Oncology will soon hold their Genitourinary Cancers Symposium (ASCO GU) from February 11 through 13, 2021. The symposium, held virtually during COVID-19, will feature a poster from biotechnology company Exelixis Inc. (“Exelixis”) covering data on Cabometyx (cabozantinib) for patients with brain metastases stemming from renal cell carcinoma (RCC). According to the retrospective study, Cabometyx showed a significant intracranial response.

Renal Cell Carcinoma

While the exact cause of renal cell carcinoma (RCC) is not known, many researchers believe smoking represents a significant risk factor, as does a history of kidney problems. PRCVHL, and TFE3 have also been linked to this easily metastasizing kidney cancer. Typically, renal cell carcinoma begins as a single tumor in one kidney. However, it may later spread to the lungs, liver, or even brain. Because of the blood-brain barrier, brain lesions caused by RCC are usually difficult to treat.

Although RCC is a rare cancer, it is also one of the most commonly diagnosed kidney cancers in adults. It is more prevalent in males than females. Often, the cancer develops between ages 50-70. Without early detection, the 5-year survival rate for RCC is low. Unfortunately, many patients are asymptomatic until the cancer has metastasized. Symptoms that do appear include:

  • Bloody or rust-colored urine
  • Fever
  • Unintended weight loss
  • Abdominal and back pain
  • Appetite loss
  • Fatigue
  • High blood pressure
  • Anemia (low red blood cell count)
  • Abdominal lump
  • Enlarged testicles or varicose testis veins

Learn more about renal cell carcinoma here.


Currently, Cabometyx is approved for use in the United States for patients with advanced RCC who are taking no other medications or in conjunction with nivolumab. It is also approved for patients with hepatocellular carcinoma previously treated with sorafenib. Exelis has granted exclusive rights to Takeda Pharmaceutical Co. Ltd. (“Takeda”) for development in Japan, and to Ipsen for development in other areas of the world.

During the retrospective analysis, researchers analyzed medical records from patients treated with Cabometyx throughout the U.S., Spain, France, and Belgium. Patients were split into two groups: those who already had brain metastases and those who did not. A majority of the patients were considered poor risk and had already been treated. Researchers found a 61% intracranial response rate for those who had started the study with progressing brain lesions, and a 57% response rate for those without. The median survival rate was 14.7 months. Overall, researchers determined that Cabometyx could be beneficial for patients with brain lesions associated with RCC that are considered difficult to treat.

Safety Information

Those who are breastfeeding should not use Cabometyx and should not breastfeed until at least 4 months following the last dose. Patients should attempt to not take this medicine with CYP3A4 inhibitors or inducers. Side effects include:

  • Poor wound healing
  • Nausea and vomiting
  • Diarrhea or constipation
  • Fatigue
  • Appetite loss
  • Adrenal insufficiency
  • Abdominal pain
  • Hoarseness
  • Unintended weight loss
  • High blood pressure
  • Hepatotoxicity
  • Fistulas and gastrointestinal perforations
  • Hemorrhage
  • Stomatitis (inflammation of the mouth and lips)
  • Rash
  • Hypothyroidism
  • Proteinuria (excess protein in the urine)
  • Jaw osteonecrosis

Read the source article here.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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