A chemotherapy compound designed at Stony Brook University has moved out of the laboratory and into a human clinical trial. The drug, now called RP-001, is being tested in adults with advanced and metastatic colorectal cancer, the university announced on November 5.

Where it came from

The compound traces back to the lab of Iwao Ojima, a SUNY Distinguished Professor in Stony Brook's Department of Chemistry who leads the university's Institute of Chemical Biology and Drug Discovery. Ojima and his team spent years developing what they call next-generation taxanes, a class of anti-cancer agents that stop cancer cells from dividing and push them toward programmed cell death.

Taxanes are already a mainstay of cancer care in the form of paclitaxel and docetaxel. The university notes that tumors often become resistant to those drugs and that their side effects can limit treatment. Ojima's second-generation Stony Brook taxane, known as DHA-SBT-1214, was designed to get around that resistance. It is linked to DHA, the omega-3 fatty acid found in fish oil.

A Stony Brook spinout company, TargaGenix, then worked with Mansoor Amiji of Northeastern University on a nanoemulsion, a form of the drug suspended in particles small enough to collect in tumor tissue. In animal studies, the university reports, that formulation was effective against tumors that resist multiple drugs and against tumors started from cancer stem cells.

From the lab to a trial

The SUNY Research Foundation gave TargaGenix the rights to develop the compound in 2016. TargaGenix later passed those rights to Recurv Pharma, which received a $24 million investment to develop it. Earlier this year, the Food and Drug Administration cleared Recurv to begin an open-label, multicenter phase 1/2a trial measuring the drug's safety, how well patients tolerate it, and its effect on tumors.

The trial is being run at sites in Texas and the United Kingdom, not on Long Island, and Recurv plans more trials in the UK. According to the company's chief executive, James E. Egan, the first phase is a safety and dose-escalation study. A phase 2 study in pancreatic and colorectal cancer is to follow, and Recurv also plans to test RP-001 alongside immunotherapy as a treatment for pancreatic cancer.

Why it matters here

Colorectal cancer remains one of the most common cancers in the United States. The National Cancer Institute reports that close to two-thirds of patients survive at least five years after diagnosis. RP-001 is aimed at patients whose disease is advanced or has spread.

Raymond Bergan, who directs the Stony Brook Cancer Center, described Ojima's work as a new form of cancer therapy aimed at the patients who most need one. Ojima's group is already working on third- and fourth-generation Stony Brook taxanes and other classes of anti-cancer compounds.

What patients should know

RP-001 is investigational and is available only through the trial. Long Island patients with questions about clinical trials for colorectal or pancreatic cancer can ask their oncologist or contact the Stony Brook Cancer Center, which runs its own trials program.

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