Clinical Trials : Yielding New Therapies and Saving Lives

Some people may view clinical trials as just another form of research, but everyone at Ohio State’s Comprehensive Cancer Center – James Cancer Hospital and Solove Research Institute (OSUCCC – James) knows these studies are imperative for improving patient care.

“Clinical trials do involve research on selected groups of patients, but the ultimate aim is to improve cancer care by demonstrating the safety and effectiveness of new treatments, examining treatment strategies and looking at problems associated with existing therapies so refinements can be made,” says OSUCCC Director and James CEO Michael A. Caligiuri, MD, pointing out that patients in these studies often have no other therapeutic options and are receiving the very latest experimental treatments. “Simply stated, clinical trials are patient care.

“The importance of these studies cannot be overstated,” he continues, noting that hundreds of such studies are under way at the OSUCCC – James at any given time, including several that are available nowhere else. “Every cancer therapy in standard use today stems from a past clinical trial.”

One ongoing trial at Ohio State that represents the potential widespread benefit of these studies is called a “basket trial” because it involves patients with different cancer types that have common gene alterations.

This phase II trial, led by principal investigator Sameek Roychowdhury, MD, PhD, a specialist in translational genomics at the OSUCCC – James, is applying basic science knowledge and tools to help researchers develop better precision cancer drugs that molecularly target alterations observed in each patient’s genes.

“Precision cancer medicine is about looking at the genetic features and molecular changes acquired by each patient’s cancer that have allowed it to grow and spread, and then tailoring the therapy,” explains Roychowdhury, director of precision cancer medicine. Specifically, this clinical trial will determine how well a drug called ponatinib hydrochloride works in treating patients with cancer that has metastasized (spread) to other parts of the body, has not responded to previous therapy, and has one of several alterations, or mutations, in its DNA sequence.

“Ponatinib hydrochloride may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth,” says Roychowdhury, a member of the Translational Therapeutics Program at the OSUCCC – James. “It is not yet known whether a patient’s genetic alterations affect how well this drug works.”

Roychowdhury says there is “a seamless integration between this clinical trial and laboratory study to determine how cancers become resistant to therapy and why some patients respond to therapy and others do not, with a key connection of research tumor samples before and after treatment.”

The investigators are using a “team science” approach to 1) study gene and protein alterations via collaboration between the basic science labs of Roychowdhury and co-investigator John Hays, MD, PhD, also of the Translational Therapeutics Program, and 2) complete the clinical trial via collaboration between Roychowdhury and scientists at the University of Michigan, where Roychowdhury was a clinical lecturer in hematology/oncology before being recruited to Ohio State in 2012.

Donn Young: A Life Saved by a Clinical Trial

Of all the surprises that Donn Young, PhD, might have imagined receiving for his 60th birthday, a diagnosis of metastatic prostate cancer wasn’t one of them.

But that’s what he got. Just a few days before, during a pre-operation work-up for routine back surgery, he had requested a prostate-specific antigen (PSA) test, which can detect prostate cancer. “It was just on a whim,” Young recalls. “I had no symptoms, but it had been a few years since I’d had a PSA.”

Levels in healthy men are usually less than four; Young’s came back at 78, and a repeat test showed them at 90. As a biostatistician who had designed and managed clinical trials with the cancer program at Ohio State for more than three decades, Young says he had “worked with enough research data and crunched enough numbers” relating to prostate cancer and PSAs to know that something was terribly wrong.

On Dec. 5, 2006 – the day he turned 60 – Young had a prostate biopsy that confirmed cancer with bone metastasis (spread) to several vertebrae, his right shoulder and hip, and a couple of ribs. “Having your prostate biopsied instead of blowing out candles is no fun,” he says, adding that the results were even worse, since there is no known cure for his advanced illness.

Young turned to the OSUCCC – James, where Steven Clinton, MD, PhD, who directs the Prostate and Genitourinary Oncology Clinic, enrolled him in a clinical trial that involves a pair of drugs—one that stops the body’s production of testosterone and androgen, and another that blocks those hormones from binding to tumor cells. The trial was designed to determine whether an intermittently administered regimen of this therapy is as effective as continuous therapy in promoting overall survival.

“This was an equivalency rather than a curative trial,” Young explains, noting that the study opened in 1995 and accrued patients until October 2008. “So it’s taken all these years to find out whether the intermittent regimen is as good as what the standard of care was when the trial started.” The median survival time for men on this trial is 34 months. Now almost nine years later, Donn is still cancer-free.

“So far, so good,” he says, “but the reality is that at some point this cancer always escapes hormonal control. My hope is that, through this therapy, I can buy a number of years until they can find out why and devise a targeted therapy.”

He knows the only way to make that kind of progress against his and other forms of cancer is for more people to enroll in science-based therapeutic clinical trials that can turn discoveries into innovative treatments.

“I never have been much of a worrier or a negative person,” he says. “I just take each day as it comes and have fun doing what I want to do. It’s always a matter of what you make of life rather than how long you live – but I hope to live a long, long time.”

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