Tests of a promising anti-cancer drug developed in Buffalo begin Monday on patients in a trial considered to be a milestone for the region's small but growing biotechnology industry.
The drug, which is referred to as KX2-391, belongs to an emerging new family of targeted cancer treatments called protein kinase inhibitors. The Buffalo-developed drug blocks a specific chemical messenger in the body called Src kinase that tells cancer cells to keep growing.
"What's remarkable about this compound is that, in preclinical testing, it has worked against every cancer and in every animal model," said David Hangauer, a University at Buffalo chemistry professor whose work was integral to the drug's creation.
These drugs work differently from chemotherapy, which poisons cancer cells as well as normal cells. It is hoped that targeted therapies will prove more effective and less harmful to normal cells.
A handful of kinase inhibitors have been approved for cancer treatment, including Novartis' Gleevec and AstraZeneca's Iressa. Many others are being tested in cancer clinical trials.
The Buffalo-developed drug stood out in nonhuman tests, showing effectiveness in a range of cancers, with fewer toxic side effects. It works in a way uniquely different from other kinase inhibitors under study.
"As we go further in the drug's development, and do broader testing, we get better and better data. If the drug works half or a third as well as it's worked in preclinical trials, it will have blockbuster, billion-dollar potential," said Allen Barnett, chief executive officer of Kinex Pharmaceuticals, the Buffalo company developing the drug.
Roswell Park Cancer Institute and M.D. Anderson Cancer Center in Houston are performing the phase one trials, which are expected to enroll 50 patients with advanced cancer who have not responded to other treatments. Clinical trials of experimental drugs are conducted in phases, with the first phase aimed at evaluating a drug's safety and dosage limits, and identifying side effects.
Related drug compounds under development at Kinex show potential for treating such autoimmune diseases as lupus, ulcerative colitis and rheumatoid arthritis, said Hangauer, who is also a co-founder and senior vice president at Kinex.
Protein kinases include many chemical messages in the body's cells that signal whether to grow. In cancer, abnormalities in the signaling cause cells to grow out of control, creating cancerous tumors.
KX2-391 targets an important cell signaler called Src kinase, a protein linked to the fast growth and spread of cancer cells. A potential advantage of Kinex's kinase inhibitor is that it disrupts Src at a site different from where other currently studied compounds are targeted, reducing the chance that patients will develop resistance to the new drugs, Hangauer said.
The final dosage for human trials was prepared in UB's New York State Center of Excellence in Bioinformatics and Life Sciences, where Kinex is headquartered.
Kinex started in 2003 and counts among a small but growing number of life science ventures that have opened in the Buffalo area in recent years, following millions of dollars of investment to create a biotechnology industry here.
The company has attracted significant interest from venture capital firms, private investors and several major pharmaceutical companies to fund the phase one trial and other phases, Barnett said.
Marnie LaVigne, director of business development at UB's Center of Excellence, described the drug's progression to human trials as "a critical milestone" in the growth of Buffalo's life-sciences economy.
"We're banking on our ability to create private-sector jobs in drug discovery and development, rather than licensing university-developed technologies to out-of-state firms, which had been the more common path for moving locally grown inventions from the lab to the marketplace," she said.