Promising cancer drug trial marks a major first for Oklahoma. Here's what you need to know

The OU Health Stephenson Cancer Center on Wednesday announced a first for the state: a cancer drug, developed entirely in Oklahoma and without the help of a pharmaceutical company, is set to be tested in humans for the first time.

The drug, dubbed OK-1, is more than 25 years in the making and was created by Dr. Doris Benbrook, a professor in the department of obstetrics and gynecology at the OU College of Medicine.

Now, a clinical trial is set to begin at the Stephenson Cancer Center, which involves giving the drug to women with advanced-stage ovarian, endometrial and cervical cancer.

"This drug is not available anywhere else in the world right now," Benbrook said. "We believe it has tremendous potential for treating cancer without causing toxic side effects.”

Benbrook said her career’s goal has been to develop new treatments for cancer patients that can kill cancer cells without harming healthy cells or causing other side effects.

Preclinical research has shown OK-1 can do that, and it may even be able to prevent cancer, Benbrook said.

“As I've gone through this process, and faced multiple obstacles, I've benefited greatly from the collaboration of Oklahoma experts, and students and fellows who have worked on this project, and the infrastructure at the Stephenson Cancer Center,” she said. “We're taking this experience and using it to mentor the next generation of cancer researchers.”

Can cancer patients skip post-surgery chemo?  New research says some may be able to.

What is the drug? How does work?

OK-1 is derived from the natural compound vitamin A, which the body uses to make retinoic acid.

Some types of retinoic acid and synthetic versions of it called retinoids can be used to treat cancers like leukemia, but they’re highly toxic.

Benbrook worked with a colleague to modify the drug’s chemical structure and study how they affected cancer cells versus healthy cells.

“Because we changed the structure so much from retinoic acid, OK-1 doesn't work like a retinoic acid,” Benbrook said.

The drug works to kill cancer cells by taking away one of the defense mechanisms the cells use to survive.

Cancer cells try to thwart the body’s natural defenses by developing “chaperone proteins” that work like bodyguards to keep them from dying. OK-1 gets around that defense by binding to the chaperone proteins and disabling them, so the immune system or chemotherapy drugs can more easily kill the cancer cells.

'Just another challenge': Why this 76-year-old paused cancer treatment to run the OKC Memorial Marathon

Who will benefit from it?

The drug, which is given in capsule form, has the potential to be a game-changer for cancer patients, OU scientists and doctors said Wednesday.

Dr. Kathleen Moore, who will run the clinical trial, said OK-1 is a sort of “holy grail.”

“It’s the beginning of a really exciting research enterprise,” she said.

What differentiates OK-1 from other drugs is that in preclinical models, researchers have found OK-1 is effective at shrinking tumors without toxicity.

It also seems to work well in concert with other cancer drugs, making both work more effectively than either would on its own, Moore said.

“We’re expecting it to be very tolerable,” Moore said, in contrast to other cancer drugs that can help patients survive their cancer but still take a toll on the body.

Dena Newlun, a cancer patient at the Stephenson Cancer Center, knows firsthand how brutal the side effects of cancer treatment can be.

She’s in the middle of a separate clinical trial for a stage-four ovarian cancer drug, she said at Wednesday’s news conference, with her service dog Tripod Petey by her side.

“Look at me, I don’t look sick,” she said. “That’s to to say that I didn’t experience a lot of the side effects that they were talking about.”

Among her side effects was aphasia, a condition where a person has trouble speaking and communicating or understanding other people. It's commonly seen in stroke patients.

“The drugs, although they do have a benefit, there is a downside,” Newlun said. “So something like this? Oh my God. Wow.”

Toby Keith announces stomach cancer diagnosis, will postpone tour dates

What’s next?

In the Phase 1 trials set to get underway now, scientists will figure out the right dose of OK-1.

Then, in later trials, OK-1 will be tested in combination with other cancer drugs.

Benbrook also plans to test OK-1 in an ovarian cancer prevention trial through the Cancer Prevention Clinical Trials Network.

If it's shown to be effective at preventing cancer, it could be given to women with a genetic predisposition to cancer, like a BRCA gene mutation. Not everyone with those gene mutations will develop cancer, but they're at higher risk for breast or ovarian cancer.

This article originally appeared on Oklahoman: Trial for cancer drug made in Oklahoma, called OK-1, set to begin