JAN 21, 2020 7:44 PM PST

Drug Targets Gastrointestinal Cancer

WRITTEN BY: Nouran Amin

The FDA has recently approved Ayvakit (avapritinib) for the treatment of unresectable and metastatic gastrointestinal stromal tumor (GIST) that occurs most commonly in the stomach or small intestine. The treatment is specifically for GIST that harbors a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation. For Ayvakit, it works as a kinase inhibitor that blocks a type of enzyme called a kinase and keeps the cancer cells from thriving.

“GIST harboring a PDGFRA exon 18 mutation do not respond to standard therapies for GIST. However, today’s approval provides patients with the first drug specifically approved for GIST harboring this mutation,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “Clinical trials showed a high response rate with almost 85% of patients experiencing tumor shrinkage with this targeted drug.”

GISTs originate from from specialized nerve cells found in the lining of gastrointestinal tract. A mutation in any of these nerve cells likely lead to the development of GIST and more than half of GISTs start in the stomach and the small intestine.

Ayvakit was approved based on results of a clinical trial of 300 mg or 400 mg dose given to patients with GIST harboring a PDGFRA exon 18 mutation. The study examined the efficacy of Ayvakit’s ability to shrink tumors. Some of the noted side effects include swelling, fatigue, and loss of focus, abdominal pain, constipation, and rash. Some dangerous complications include the possibility of intracranial hemorrhage and mood disorders.

Clinicians should counsel pregnant women of the harms of Ayvakit to a developing fetus or newborn baby, and females of reproductive age.

Source: U.S Food and Drug Administration

About the Author
  • Nouran is a scientist, educator, and life-long learner with a passion for making science more communicable. When not busy in the lab isolating blood macrophages, she enjoys writing on various STEM topics.
You May Also Like
MAR 20, 2021
Drug Discovery & Development
Weekly Insulin for Type 2 Diabetes Proved Effective
MAR 20, 2021
Weekly Insulin for Type 2 Diabetes Proved Effective
A study found that a new once-weekly basal insulin injection holds similar efficacy, safety and lower rate of low blood ...
APR 14, 2021
Cancer
Cell Viability- Where to Begin?
APR 14, 2021
Cell Viability- Where to Begin?
Written By: Author As I pondered the title of this article, my thoughts immediately went back to a request by my Ph.D. a ...
MAY 03, 2021
Drug Discovery & Development
Low Dose Radiation May Improve Symptoms of Alzheimer's
MAY 03, 2021
Low Dose Radiation May Improve Symptoms of Alzheimer's
While high doses of radiation are known to be harmful, low doses may be able to help the body protect and repair. And no ...
MAY 04, 2021
Health & Medicine
Micro-dosing Inhaler Breakthrough for Medical Cannabis
MAY 04, 2021
Micro-dosing Inhaler Breakthrough for Medical Cannabis
In a leap that will eliminate the risks of vaping or smoking, Canada has just approved an Israeli-made inhaler that is a ...
MAY 13, 2021
Immunology
Salmonella Vaccine Uses "Molecular Telephones" to Talk to the Immune System
MAY 13, 2021
Salmonella Vaccine Uses "Molecular Telephones" to Talk to the Immune System
Salmonella infections are a massive problem—Over 95 million people are affected by this foodborne illness every ye ...
JUN 03, 2021
Drug Discovery & Development
Smartphone App More Effective than Traditional Treatment for Osteoarthritis
JUN 03, 2021
Smartphone App More Effective than Traditional Treatment for Osteoarthritis
  Researchers at the University of Nottingham in the UK and Joint Academy have found that a clinical evidence-based ...
Loading Comments...