The University of New Mexico Comprehensive Cancer Center played a key role in the first clinical trials of a new drug that targets solid cancer tumors. The Phase 1A clinical trials mark the first time ever the drug was used in people. It opened at several sites across the country. Olivier Rixe, MD, PhD, at UNM Comprehensive Cancer Center, oversaw the clinical trials’ national protocol development and directed the trial in New Mexico.
The drug, called BXQ-350, has been shown in pre-clinical studies to induce cancer cells to die but have little effect on normal cells. It is produced by Bexion Pharmaceuticals, a privately held biotech company, that focuses on cures for cancer. BXQ-350 is comprised of a human protein called SapC and a human lipid called DOPS.
Clinical researchers for the Phase 1A clinical trial tested the safety of BXQ-350. “It had a very good safety profile,” says Rixe. Rixe hopes the drug will continue to show promise for those whose brain cancer has recurred. He is especially hopeful for those diagnosed with glioblastoma multiforme, a particularly aggressive brain cancer.
New cancer drugs and treatments go through a series of steps before the United States Food and Drug Administration approves them for use in people. The entire process can take a decade or more and cost billions of dollars. The early phases test whether the new drug or treatment is safe, what its side effects are, and what the best dose for treatment is. Later phases test whether the new drug or treatment works better than the standard therapy. All clinical trials in the United States must meet stringent safety measures that the FDA enforces.
Terry Novak, RN, served as the Nurse Clinical Trials Coordinator for the BXQ-350 clinical trial at UNM Cancer Center. “It was an honor for me to be part of this trial,” she says. “The drug was very well tolerated with minimal side effects.”
The earliest clinical trials are called Phase 1A clinical trials. Clinical researchers make sure that the people who join a Phase 1A clinical trial follow the trial’s set of rules, called a protocol. The FDA approves the clinical trial’s protocol before it can begin. The protocol lists the details of how the drug or treatment will be given and what data will be collected. Importantly, it also describes the conditions under which researchers will shut the clinical trial down.
Phase 1 clinical trials test how safe a drug is for people and how it moves through people’s bodies. Extensive preclinical trials, which researchers conduct on animals, suggest a starting range for safe doses in people. Phase 1 clinical trials start at the lowest dose at which clinical researchers expect to see an effect. The protocols call for gradual dose increases while watching the people on the trial very carefully.
Clinical researchers may shut down a clinical trial at any time if the people in it have severe side effects. Phase 1 clinical trials do not use a placebo; everyone on the trial knows they are getting the new drug. “Patients on this trial are truly our heroes,” Novak says about the people on the BXQ-350 trial. Novak helped them meet the clinical trial’s protocol. She says, “Their courage and confidence in the UNM Cancer Center and in Dr. Rixe made this trial possible.”
Rixe originated the Phase 1 protocol and oversaw the protocol development for all of the sites at which the Phase 1A BXQ-350 clinical trials took place, in addition to directing the Phase 1A clinical trial at UNM Cancer Center. He was also involved in developing the drug through preclinical trials, to its Investigational New Drug application.
Rixe and his Phase 1 clinical trials team at UNM Cancer Center have completed 12 Phase 1 clinical trials in New Mexico. “We offer what other cancer centers, like Harvard and MD Anderson, offer their patients,” Rixe says. “The patients treated at UNM Cancer Center are among the first in the world to be given this drug.”
Olivier Rixe, MD, PhD, is a Professor in the Department of Internal Medicine, Division of Hematology/Oncology, at the University of New Mexico School of Medicine. He serves as the Associate Director for Clinical Research at the UNM Cancer Center. Trained as a medical oncologist, Dr. Rixe is internationally renowned for his work in Phase I clinical trials and as Principal Investigator for several neuro-oncology clinical trials. He was involved in the early development phases of many U.S. Food and Drug Administration approved agents, including oxaliplatin, camptothecins, and taxanes; and many targeted therapies including antiangiogenic compounds sunitinib, axitinib and VEGF-trap.
In July 2016, Bexion received a “Study May Proceed” letter from the FDA, allowing Bexion to initiate the first clinical trial using BXQ-350 to dose patients with cancer (solid tumors and glioblastoma). Information about the Phase 1 Study of BXQ-350 in Adult Patients With Advanced Solid Tumors trial can be found at https://www.clinicaltrials.gov/show/NCT02859857.
Bexion Pharmaceuticals is a privately-held biotech company focused on the development and commercialization of innovative cures for cancer. Bexion's first-in-class biologic, BXQ-350, has demonstrated selective tumor targeting with the potential for clinical efficacy in a broad range of cancers. In 2013 the NCI awarded Bexion a prestigious "Bridge Award" of $3MM to support testing of BXQ-350 in the clinic. In February 2015, the FDA granted Bexion Orphan Drug status for Saposin C, the active ingredient in its proprietary drug BXQ-350, for the potential treatment of glioblastoma multiforme (GBM), a type of brain cancer. In June 2015, Bexion won a Tibbett's Award by the Small Business Administration for exemplifying the very best in innovation. For more information, visit www.bexionpharma.com.
The University of New Mexico Comprehensive Cancer Center is the Official Cancer Center of New Mexico and the only National Cancer Institute-designated Cancer Center in a 500-mile radius. Its 125 board-certified oncology specialty physicians include cancer surgeons in every specialty (abdominal, thoracic, bone and soft tissue, neurosurgery, genitourinary, gynecology, and head and neck cancers), adult and pediatric hematologists/medical oncologists, gynecologic oncologists, and radiation oncologists. They, along with more than 500 other cancer healthcare professionals (nurses, pharmacists, nutritionists, navigators, psychologists and social workers), provided cancer care for nearly 60 percent of the adults and children in New Mexico affected by cancer. They treated 11,249 patients in 84,875 ambulatory clinic visits in addition to in-patient hospitalizations at UNM Hospital. These patients came from every county in the State. More than 12 percent of these patients participated in cancer clinical trials testing new cancer treatments and 35 percent of patients participated in other clinical research studies, including tests of novel cancer prevention strategies and cancer genome sequencing. The 130 cancer research scientists affiliated with the UNMCCC were awarded almost $60 million in federal and private grants and contracts for cancer research projects and published 301 high quality publications. Promoting economic development, they filed more than 30 new patents in FY16, and since 2010, have launched 11 new biotechnology start-up companies. Scientists associated with the UNMCCC Cancer Control & Disparities have conducted more than 60 statewide community-based cancer education, prevention, screening, and behavioral intervention studies involving more than 10,000 New Mexicans. Finally, the physicians, scientists and staff have provided education and training experiences to more than 230 high school, undergraduate, graduate, and postdoctoral fellowship students in cancer research and cancer health care delivery. Learn more at www.cancer.unm.edu.
Dorothy Hornbeck, JKPR, 505-340-5929, firstname.lastname@example.org
Michele Sequeira, UNM Cancer Center, 505-925-0486, email@example.com