MAY 26, 2017 10:49 AM PDT

FDA Approved Cancer Drug Based on Mutation, Not Location

WRITTEN BY: Xuan Pham

For the first time, the US FDA approved a cancer drug for patients based on presence of genetic biomarkers. Whereas cancer drugs have been traditionally been approved based on the location of the tumor, Keytruda (pembrolizumab) is approved for cancer patients with genetic defects classified as “microsatellite instability” or “mismatch repair” deficiencies.

"KEYTRUDA binds to the PD-­1 receptor and blocks its interaction with PD-­L1 and PD-­L2, which helps restore the immune response." - Keytruda.com

"This is an important first for the cancer community," said Richard Pazdur, acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research and director of the FDA's Oncology Center of Excellence. "Until now, the FDA has approved cancer treatments based on where in the body the cancer started - for example, lung or breast cancers. We have now approved a drug based on a tumor's biomarker without regard to the tumor's original location."

Keytruda is a humanized antibody that works as a second-line immunotherapy agent to destroy the cancer. This drug targets the programmed cell death 1 (PD-1) receptor, a protein that’s responsible for preventing the immune system from attacking its own cells. Cancers can take advantage of this system by making its own proteins that bind to PD-1, which shuts down the body’s ability to kill the cancer. As an immunotherapy, pembrolizumab boosts the body’s ability to kill the cancer again. The drug is already approved for the treatment of several types of cancer, including non-small cell lung cancer, melanoma, and some head and neck cancers, and even mesothelioma.

But the new approval could significantly expand uses of this drug. In particular, Keytruda may now be used to treat any solid tumor as long as the patients have the indicated biomarkers: microsatellite instability-high (MSI-H) or mismatch-repair deficient (dMMR). These types of deficiencies are estimated to be present in about 4 percent of cancers, including colorectal, endometrial, and gastrointestinal cancers.

Notably, the drug’s new indication would expand access to cancer patients with solid tumors that have progressed despite treatment and for which there are no other therapy. For colorectal cancer, this means patients who have exhausted their chemotherapy defenses with drugs such as fluoropyrimidine, oxaliplatin, and irinotecan, Keytruda can be a new option.

“The FDA’s approval of this new indication for Keytruda further supports Merck’s commitment to helping people with difficult-to-treat cancers,” said Roger M. Perlmutter, president of Merck Research Laboratories, the drug’s manufacturer.

 

About the Author
  • I am a human geneticist, passionate about telling stories to make science more engaging and approachable. Find more of my writing at the Hopkins BioMedical Odyssey blog and at TheGeneTwist.com.
You May Also Like
JUN 05, 2018
Cancer
JUN 05, 2018
Precision vs. Personalized Medicine Part II: Personalized Approach
So often the phrases "Precision Medicine" and "Personalized Medicine" are used interchangeably. Are they really synonyms? Part II looks at Personalized Medicine...
JUN 19, 2018
Cancer
JUN 19, 2018
Cancer-Related Cognitive Impairment aka "ChemoBrain" Needs YOU!
There is a need for neuroscientists to investigate what is known as chemobrain; survivors and providers are looking for ideas for approaches to mitigating this late effect chemo related issue...
JUL 17, 2018
Cancer
JUL 17, 2018
New Small Cell Lung Cancer Subtype Identified
A new subtype of small cell lung cancer is reported to account for potentially 20% of small cell lung cancers diagnosed; it has unique molecular biomarkers and regulatory mechanisms....
AUG 26, 2018
Cancer
AUG 26, 2018
Can antireflux surgery prevent Esophageal cancer?
Heartburn or gastroesophageal reflux disease (GERD) is a widespread health problem affecting 10 to 20% of adults in Western populations. If GERD left untre...
SEP 26, 2018
Cancer
SEP 26, 2018
Rare Cancer the Focus of Mesothelioma Awareness Day
More than 2,500 Americans are diagnosed with mesothelioma cancer each year. The cancer is rare but very serious - most patients diagnosed with the disease live just a short 12 to 21 months....
OCT 06, 2018
Drug Discovery
OCT 06, 2018
New Class of Drugs for Breast Cancer Therapy
Scientists at Stevens Institute of Technology have designed a new class of molecules that may hold the potential to add to the arsenal of drugs actively be...
Loading Comments...