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 earned her BS and MS in Biology at IUPUI and currently shares her love of science by teaching. She enjoys writing on various topics as well including science & medicine, global health, and conservation biology. She hopes through her writing she can make science more engaging and communicable to the general public.
You May Also Like
DEC 06, 2019
Health & Medicine
DEC 06, 2019
Federal Restrictions Make It Hard For Cannabis Researchers
With marijuana use becoming more popular in aiding health, scientists race to find clarity around its health benefits and risks. Since the legalization of...
DEC 15, 2019
Microbiology
DEC 15, 2019
Potential Therapeutics for Nipah Virus Are Identified
The fatality rate of Nipah virus has an estimated range of 40 to 75 percent...
DEC 21, 2019
Drug Discovery & Development
DEC 21, 2019
Improving Vaccines for Meningitis
Scientists at the University of Nottingham are seeking new ways to improve vaccine use in the protection against the bacterium, Neisseria meningitides that...
JAN 23, 2020
Cell & Molecular Biology
JAN 23, 2020
Scientists Engineer Venom-Producing Organoids
Snake venom is also a source of therapeutics, and a potential source of new medicines....
FEB 11, 2020
Cannabis Sciences
FEB 11, 2020
Traffic Light System Best Way to Label Cannabis Edibles
Cannabis products have become increasingly popular. Yet, as they are still relatively new to the market, research has found that most consumers don’t...
FEB 13, 2020
Cell & Molecular Biology
FEB 13, 2020
Study of Early-Onset Parkinson's Reveals Potential Therapeutic
Around 500,000 Americans are diagnosed with Parkinson's disease every year, and the rate of the disease is rising....
Loading Comments...