The Ohio State University Comprehensive Cancer Center found a new drug, nemtabrutinib, very effective in patients. Credit: Mackenzie Shanklin | Lantern File

A new drug, nemtabrutinib, might soon be available to patients with blood cancers that have stopped responding to typical treatments after Ohio State conducted a successful clinical trial. 

The Ohio State University Comprehensive Cancer Center found the drug effective in ¾ of participating cancer patients, which includes those with chronic lymphocytic leukemia and non-Hodgkin lymphoma, according to a Nov. 6 press release.

While there are multiple drugs available to treat these B-cell cancers, many patients with blood cancer experience disease progression after initial treatment, which can be especially difficult to further treat with today’s standard options, according to the center.

Dr. Jennifer Woyach, lead investigator of the study and co-leader of the Leukemia Research Program at the cancer center, said for patients whose cancer has stopped responding to standard treatments, nemtabrutinib’s effectiveness shows its potential viability as an alternative treatment option.

“The patient population this is being tested in really doesn’t have many good clinical options,” Woyach said. “Something where over 50 percent of the people respond, I think would be really good in this clinical scenario.”

Although this marks the first in-patient study of nemtabrutinib, Woyach said she and other researchers at the center conducted all of the preclinical work with the drug, which was published in 2018. 

Woyach said before nemtabrutinib, Ohio State was one of the first institutions to begin transitioning from chemotherapy to targeted therapy in the treatment of chronic lymphocytic leukemia, also known as CLL.

Woyach said she and her research team were some of the first to study Bruton’s tyrosine kinase — also known as BTK — inhibitors, which are drugs that have “revolutionized the treatment of CLL.”

“In the frontline setting with our standard best chemotherapy regimens, the average remission durations were about four years,” Woyach said. “With these BTK inhibitors in the frontline setting, the average remission durations went to about eight and a half years.”

She and her team then determined that despite the success of the new targeted therapy, patients’ CLL still relapsed on BTK inhibitors due to a mutation in the BTK enzyme, Woyach said. 

Nemtabrutinib and its success in the study show that BTK can be targeted in a different way that has the potential to lead to another set of prolonged remission for patients.

“CLL probably still isn’t curable even with drugs like this, but I think that there is a possibility that combinations may be curable for some patients,” Woyach said. “For sure not all patients, but some patients might be able to be cured with combinations of therapies that we have right now.”

Even if the cancer isn’t cured, Woyach said nemtabrutinib and other targeted therapies may act as “functional cures,” providing patients a period of remission that allows them to live their lives as if they are cancer-free after taking a medication.

“And for many people, especially in their 70s, 80s and 90s, that’s probably as good as curing people, if you can get a drug that people can tolerate, take for a long time and live their life as if they don’t have cancer,” Woyach said.

With the success of this phase-one study, Woyach said the drug will now move on to mostly phase-three studies, largely because nemtabrutinib is known to be “a good target.” While this study was conducted within only three different centers for the sake of more easily monitoring toxicity, Woyach said the next steps will be on a much larger scale.

“The phase-three study, you’re gonna want to get as broad of a population as you can so it’ll be all over the world,” Woyach said. “Those ones will compare nemtabrutinib either alone or in combination, depending on the trial, versus the standard-of-care regimen. And the goal of those trials will be to get the FDA to give its approval at the end.”