UA Professor of Cell Biology and Anatomy Eugene Gerner will run CPP with his colleague Frank L. Meyskens, Jr., Professor of Medicine and Director of the Chao Family Comprehensive Cancer Center at the University of California in Irvine.
Gerner is a member of UA’s BIO5 Institute and the Arizona Cancer Center, where he is the lead investigator on the National Cancer Institute-funded Specialized Program of Research Excellence (SPORE) in gastrointestinal cancers grant.
CPP currently is conducting FDA approval trials on Eflornithine, a drug that—in combination with an existing one—has reduced the recurrence of colon polyps in earlier clinical trials by 70 percent, and has reduced the recurrence of the type of polyps most strongly associated with cancer by 92 percent. Colon polyps of all types are possible precursors to colon cancer. Gerner, who has been studying Eflornithine for more than two decades, is hopeful that the drug could be available in as little as three years.
While there are many cancer treatment drugs available, drugs for preventing the disease are still rare. "What we're trying to do is pretty unique," Gerner says. "Our mission is to bring drug-based prevention into the clinical management of patients at high risk for cancer."
That uniqueness initially presented a challenge. When Gerner and Meyskens tried to interest existing drug companies in Eflornithine, the companies were unwilling to take on the liability—and uncertain profits—associated with a prevention-based treatment. Gerner and Meyskens formed CPP instead, to assure that Eflornithine has a way of getting from the lab into the clinic.
Eflornithine disrupts the body's synthesis of polyamines, compounds which are associated with abnormal cell growth—and thus cancer—in adults. The drug would be combined with Sulindac, an already available non-steroidal anti-inflammatory drug (NSAID) that also disrupts polyamine production—as well as fighting inflammation, which also is associated with cancer.
Earlier Eflornithine clinical trials focused strongly on safety, and on finding the lowest possible dose that would still be effective. Gerner says reducing side effects was critical for a drug meant to be taken not only by cancer patients, but also by currently healthy individuals at risk for cancer. Ultimately the researchers found a dose so low it had no more side effects than a placebo containing no medication but still effective.
"We're hopeful that the FDA approval trials will lead to physicians being able to prescribe and patients being able to obtain Eflornithine," Gerner says. CPP also is working on ways to target patients who are at the greatest risk of colon cancer—those who've had colon polyps in the past and those with strong family histories—since they stand to benefit most from the drug.
Eflornithine may one day help prevent other cancers as well. Preliminary studies indicate that it is effective in men with a high risk of prostate cancer, and studies are currently underway among women at risk for breast cancer.
The multi-million dollar NIH SPORE grant is funding a portion of the current trials, and additional grants are planned by Arizona Cancer Center investigators based on availability of Eflornithine, all of which bring further economic benefits to the state. The universities of Arizona and California hold the patent on the use of Eflornithine in cancer prevention and would receive a share of the profits from its prescription to patients.
Gerner says the new drug could lower cancer mortality rates significantly as well as the health costs associated with cancer treatment. "Our company is working to treat patients with relatively inexpensive drugs that have both low risks and very positive benefits," he says.
