News & Information | START In The News

September 4th, 2014

FDA approves drug that was first tested by SA-based START

SAN ANTONIO (September 4, 2014) -- Patients with advanced melanoma will soon have access to a new treatment option, thanks to FDA approval today of MK-3475, or, pembrolizumab.  The drug, a Merck compound, underwent its initial testing at START in San Antonio.  The principal investigator on the trial was Dr. Amita Patnaik, and the first 17 patients treated with the drug and first responses were at START.  Merck plans to market pembrolizumab under the brand name Keytruda.

Dr. Patnaik, Associate Director of Clinical Research at START in San Antonio, calls this drug "transformative" in the treatment of advanced melanoma which causes about 75% of the skin cancer deaths in the U.S., and has proven to be very resistant to traditional forms of treatment.  

"Approval of this drug is wonderful news for cancer patients and the doctors who treat them," said Dr. Patnaik.  "A breakthrough of this proportion happens rarely and will give both Oncologists and patients a significant, new treatment option and will likely become the standard of care for patients with late stage melanoma."

Pembrolizumab is a PD-1 inhibitor (programmed death receptor-1) which is thought to work by stimulating the patient's own immune system to mount a T cell-mediated immune response against the cancer.  Pembrolizumab is one of a number of what are called 'targeted therapies' which researchers at START are optimistic can 'zero in' on specific malignant cells, without the dangerous and debilitating side effects of radiation and chemotherapy.

"This FDA approval is also important news for San Antonio." Patnaik said, "In the world of cancer research, San Antonio is ground zero and what many may not realize is that a large proportion of FDA-approved anticancer agents were first used in patients here -- not in cancer centers in Houston, or New York or anywhere else.   In other words," Patnaik said, "one of the most recognizable names in cancer therapies were tested by the researchers at START in San Antonio."

START's researchers -- Dr. Amita Patnaik, Dr. Anthony Tolcher, Dr. Kyriakos Papadopoulos and Dr. Drew Rasco -- have had direct, hands-on involvement with the clinical development of more than 18 anticancer drugs that, in the past 16 years, have obtained FDA approval. These drugs include Erlotinib, Gefitinib, Sorafenib, Panitumumab, Temsirolimus, Lapatinib, Bortezomib, Vorinostat, Pemetrexed, Abiraterone, Ixabepilone, Cabazitaxel,  Eribulin, Trametinib, Kadycla, Stivarga, Zaltrap, and Carfilzomib.  

Because of the work being done by START, San Antonio's reach has been global.

START serves patients and leads research efforts from its headquarters in the South Texas Medical Center and operates four Phase I clinical research centers on three continents, using an integrated team of researchers working around the clock to develop the most effective treatment therapies.  This innovative global approach to research has one singular goal -- to accelerate the development of new drugs for the treatment and eventual cure of cancer.

With Phase I centers strategically situated in Madrid, Spain, Shanghai, China and the US, START's team of physicians and researchers are using all 24 hours of the clock. When one center closes for the day, another one is just beginning its day.

In April of 2013 pembrolizumab was granted breakthrough therapy (BT) designation by the U.S. Food and Drug Administration which is reserved for drugs considered to offer a significant improvement in the safety or efficacy of the treatment of a serious condition.  Breakthrough therapy designation generally shortens the drug's FDA review period from 10 months to 6 months.  Data on MK-3475 (pembrolizumab ) were first presented at the 2012 ASCO Annual Meeting by Dr. Patnaik and her colleagues and subsequently published in the New England Journal of Medicine in July of 2013. These early results looked at 135 patients with advanced melanoma given MK-3475 every 2 to 3 weeks. Confirmed response (reported in the NEJM) to the treatment was found in 38% of patients.

START is based in San Antonio, with clinic sites in Madrid and Shanghai, and operates the world's largest Phase I program for new anti-cancer agents.  

You can find Merck's release on the news here.

About Advanced Melanoma.  Melanoma is the most serious form of skin cancer and is the 19th most common cause of cancer death in Europe. In 2012, there were an estimated 22,200 deaths from malignant melanoma in Europe. There were an estimated 232,000 new cases of melanoma diagnosed worldwide in 2012.

About Pembrolizumab. Pembrolizumab (MK-3475) is an investigational, selective, humanized, monoclonal anti-PD-1 antibody designed to reactivate anti-tumor immunity. Pembrolizumab exerts dual ligand blockade of the PD-1 pathway by inhibiting the interaction of PD-1 on T cells with its ligands PD-L1 and PD-L2.

ABOUT START:  With centers located in Madrid, Spain, Shanghai, China and San Antonio, Texas, START operates the largest, global, 24-hour Phase I research organization.  START's mission is to accelerate the development of new anticancer drugs for the purpose of improving the quality of life and survival for patients with cancer.  START Madrid opened in 2008. For information on START visit