Phase I Trial Based on UC Basic Research Successfully Completed

A Phase I, first-in-human clinical trial, testing the safety and antitumor activity of a treatment that was developed based on laboratory findings from a University of Cincinnati (UC) College of Medicine researcher, was just successfully completed.

This brings researchers another step closer to a potential treatment for advanced solid tumors and cancers like glioblastoma multiforme, the most common type of brain cancer. 

Bexion Pharmaceuticals and Clinical Trial and Consulting Services (CTI) are the trial sponsors of BXQ-350, which was created using technology developed by Xiaoyang Qi, PhD, a UC College of Medicine and Cincinnati Cancer Center researcher. This compound was studied in a national Phase I clinical trial, including at the UC Cancer Institute. It was led locally by John Morris, MD, director of the UC Cancer Institute’s Phase I/Experimental Therapeutics Program. The UC Cancer Institute has the only Phase I program in the region. 

"The trial was designed to determine the maximum tolerated dose of BXQ-350 that could be administered without any serious dose-limiting effects being seen in the patients,” Morris says. 

BXQ-350 was well-tolerated at all five dose levels with no dose limiting toxicities observed and with no serious adverse effects attributed to the therapy. The highest dose tested will be used in an upcoming Phase Ib part of the trial to study the effectiveness to BXQ-350 against cancers.

In 2013, Bexion received a $2.9 million Small Business Innovation Research Bridge Award from the National Cancer Institute, with Qi as co-principal investigator, to help it bring BXQ-350 into the clinical trial phase.

The treatment is based on research from Qi’s lab that began in the 1990s at Cincinnati Children’s Hospital Medical Center. 

Qi, now a professor in the Division of Hematology Oncology at UC, developed SapC-DOPS, a combination of a lysosomal protein, saposin C (SapC), and a phospholipid, dioleoylphosphatidylserine (DOPS), that assembled into tiny cavities, or nanovesicles, that can kill many forms of cancer by selectively targeting cancer cells and inducing natural "cell death” programs in the tumor cells.

Lysosomes are membrane-enclosed cellular organelles that contain enzymes capable of breaking down all types of biological components; phospholipids are major components of all cell membranes and form lipid bilayer of cell membranes.

Qi found that the combination of these two natural cellular components, or SapC-DOPS, caused cell death in many human cancer cell types, including brain, lung, skin, prostate, blood, breast and pancreatic cancers, while sparing normal cells and tissues in animal cancer models. 

With numerous basic studies under his belt, Qi formed a partnership with Bexion to create the BXQ-350 compound.

"This is very exciting as it demonstrates true translational research—from a scientific lab to actually being evaluated in patients,” says Morris, who is a professor at the UC College of Medicine. "With Bexion leading the way, the next steps are to determine the effectiveness of the drug in a larger number of patients. This part of the study is set to begin in the next few weeks.”