Last fall marked the start of a Phase 1 clinical trial at Oklahoma Medical Research Foundation, a culmination of work from its researchers on a compound named OKN-007, which has shown marked success in animal trials.
The story behind the drug has the makings of good fiction: a family tragedy, an intrepid optimist and several researchers daring to think outside the box.
This trial also will be Larry Kennedy’s swan song, as he will soon retire from his career of helping bring life-changing drugs to the hands of the people who desperately need them. He may also help cure the disease that killed his own brother.
Kennedy was a young biochemist in Chicago when he received word that his younger brother, John Kennedy, only 45 years old, was diag nosed with glioblastoma and it would wreak havoc on John’s brain tissue.
He was in touch with many of the greatest minds of medical research, and he was told the same story from everyone: Little could be done.
This nasty cancer robs its victims of quality of life long before their deaths. The scientist watched helplessly as his brother deteriorated. Back then, surgery, radiation and chemotherapy were the only options.
Kennedy’s true calling
Kennedy’s inability to help his brother would follow him throughout his career. He said that many people doing scientific research also are motivated by a similar situation.
“It might not be as dramatic, but you find that there is something personal that drives them,” he said.
Kennedy left Chicago and came to OMRF so he could facilitate the availability of drugs in various testing stages. Since then, as vice president of technology transfer at OMRF, he has helped create hundreds of drugs to help untold numbers of people.
“I find a way for their development to benefit mankind,” he said.
However, no drug was more fortuitous than NXY-059, a drug originally developed for stroke victims.
Dr. Robert Floyd developed the compound, and it showed promise when administered to mice that had suffered strokes.
It was put to clinical trials in humans but eventually proved that was not as effective as experts hoped. However, no negative health risks were found. After a final phase of testing, the drug was shelved.
Enter Dr. Rheal Towner, a cancer researcher and OMRF MRI specialist who works with disease in rats and mice. Towner was working on the effects of several drugs on mice suffering from particular ailments, especially cancer. The soft-spoken scientist with a white board completely full of numbers and an office that smells of lavender is the OMRF whiz kid with an MRI.
His career started with his work on liver disease, and he also found success creating mice and rat models of human diseases.
Coming full circle
After Kennedy and Floyd’s urging, Towner gave the NXY-059 compound to rats with the rodent equivalent of glioblastoma, and an amazing thing happened: the tumors shrank.
Moreover, the shrinkage left surrounding tissue unharmed. And months later, the once-cancer-riddled rats showed no signs of recurrence.
Kennedy was beyond excited, he said. Could this once-“failed” drug become a lifesaver for patients with glioblastoma? Just months away from his Feb. 14 retirement as a career scientist, he might finally help cure the disease.
A new drug’s path from development to human use is long and expensive. After a drug shows some promise in small lab tests, it goes through three rigorous and costly phases. If a drug makes it through the first two, Phase 3 brings in thousands of patients, and costs can zoom past $50 million.
Kennedy discovered that, in this instance, there is yet another hurdle: Glioblastoma affects approximately 13,000 people in the United States annually. Pharmaceutical companies declined to fund research for a drug that might or might not help such a small number of patients. With the industry at an all-time low, Kennedy said, the cost and benefit must be carefully considered with every drug that could potentially reach the consumer.
Even with the obstacle, Kennedy was undeterred. While poring over data from the continued work of Towner and his colleagues, it occurred to him that they could do the early trials themselves.
The drug was proven safe during earlier trials, and the drug surplus was still in storage, so OMRF was already two steps ahead. The men approached the Food and Drug Administration with their prior evidence and asked to move forward with human clinical trials. The FDA said yes. Next, the group started fundraising to start Phase 1.
By fall, the newly renamed OKN- 007 was tested in human patients. If all goes well, there is a real possibility that Kennedy’s legacy will be one he set out to accomplish many years ago: to provide real hope and healing for other John Kennedys of the world before it’s too late.