The University of New Mexico Comprehensive Cancer Center successfully completed the first in-human testing of a promising new cancer drug called BXQ-350 that is meant to target solid malignant tumors.
Dr. Olivier Rixe has been involved in the development of BXQ-350 from the early stages of its conception and is the director of the clinical trial at UNM.
“You have a lot of steps with the (U.S. Food and Drug Administration),” he said. “It’s almost been 10 years from the initial concept to the first patients treated.”
The drug works by targeting a protein called phosphatidylserine, also known as PS, that is usually present in the inner cell membrane and plays a key role in cell signaling.
Rixe said in contrast to healthy cells, the PS protein is expressed on the outer membrane of cancerous cells.
“We don’t know why, but when cells become cancerous, there is a flip-flop of PS to the external part of the membrane,” he said.
BXQ-350 is constructed to specifically bind to PS proteins expressed on the outside of cancerous cells to then induce apoptosis — meaning cell death.
UNM Cancer Center is the lead institution for the BXQ-350 trials with three other universities participating: the Ohio State University Comprehensive Cancer Center, the University of Cincinnati Barrett Center and the University of Kentucky Markey Cancer Center.
Now that their application for the Investigational New Drug Process from the FDA is approved, Rixe and constituents can create a trial protocol to take the drug “from the bench to the bedside.”
The recent phase 1A clinical trial is the first in-human administration and is designed to study the safety of the drug, to find the proper active dosage through biopsies of tumors and to see “if (they) hit the target,” Rixe said.
“It’s really about the safety profile,” he said. “As the first-in-human and first-in-class, we have no comparison with other agents. It’s so brand new.”
The phase 1A clinical trial begins with giving patients a low dosage and requires a gradual increase over time so they can analyze the safety of the new drug.
The first phase of the trial will include 20 patients with all cancer types including glioblastoma, which is a particularly aggressive brain cancer with few treatment options, Rixe said.
“When (patients) have exhausted all the standard options, they come to us and say, ‘Do you have this type of new treatment?’” he said. “(It’s) very different from what they got before, because now they’re resistant to chemo, and (their) disease won’t respond to conventional therapies.”
Currently there is no method to mapping the position of PS proteins in patients, meaning this new trial is only available to patients who have exhausted standard therapy options.
The next step is to determine how to identify the best candidates, Rixe said.
“We are still in the discovery mode with a limited number of patients,” he said. “Really, the very first critical step is exploration and discovery.”
The next phase includes expansion in order to determine the efficacy of BXQ-350 and which patients respond to the treatment, Rixe said.
“It’s a long process, but we always try to expedite this, because we are testing this drug in brain tumors and in all types of cancer as well,” he said.
Once phase 1A is complete with positive results, the study will look to expand the number of patients and begin comparing the drug with other, more conventional therapies.
If the drug is determined safe and effective, it could take up to 10 more years to go to the FDA, Rixe said.
He said the interaction between the patients and their families with the physicians and research team is what makes this study unique.
“We have outstanding patients and families engaged in this project,” he said. “It’s about them, not about us — it’s about their commitment.”
He said the patients’ commitment to science and helping others is “absolutely incredible.”
“This, to me, is the real definition of altruism,” Rixe said.
Madison Spratto is a news reporter for the Daily Lobo. She can be contacted at email@example.com or on Twitter @Madi_Spratto.