A pair of scientists from the University of Arizona and the University of California, Irvine, has formed a company to bring to market a colon cancer chemopreventive that combines an existing drug used to treat African sleeping sickness and a common non-steroidal anti-inflammatory.
The company, Cancer Prevention Therapeutics, is putting the final touches on an agreement with the schools to license the intellectual property surrounding the preventive, one of the co-founders said this week.
In addition, CPT is using an undisclosed portion of a $12 million National Cancer Institute Specialized Program of Research Excellence grant to fund Phase I and II clinical trials for the preventive, but is seeking additional financing to help offset drug-manufacturing costs for the trials.
CPT was founded by Frank Meyskens, professor of medicine at UC-Irvine, and Eugene Gerner, a UA professor of cell biology and anatomy whose participation and ownership in the new venture was approved last week by the UA board of regents.
Meyskens and Gerner had collaborated as early as the mid-1970s on oncology-related research as faculty members at UA, which eventually evolved into a mutual interest in chemopreventive compounds, Gerner told BTW this week.
In particular, during time spent as a research scientist within the pharmaceutical industry, Gerner became familiar with a compound called α-difluoromethylornithine, or eflornithine, which was being explored as a cancer therapeutic.
Though eflornithine eventually fell out of favor as a cancer drug after several negative studies, it eventually resurfaced as an effective treatment for African trypanosomiasis, or sleeping sickness.
However, Gerner and Meyskens continued to test eflornithine as a potential chemopreventive – Gerner as a professor at UA and a member of the university's Bio5 Institute and Arizona Cancer Center; and Meysken as a professor at UC Irvine and director of its Chao Comprehensive Cancer Center.
They eventually discovered that when combined with another existing drug, the anti-inflammatory sulindac, it could reduce the recurrence of colon polyps, a possible precursor to colon cancer.
The pair worked with their respective institution's tech-transfer offices to file patents on the discovery. One of those patents, US No. 6,258,845, was awarded in 2001, while several other related patents are pending. UA and UC are co-owners of the patents.
At the annual meeting of the American Association for Cancer Research last April, Gerner and Meyskens presented data from an experimental Phase III clinical trial showing that low doses of the drugs administered in tandem were able to prevent the recurrence of all colon polyps by 70 percent, and the recurrence of advanced adenomas, the highest-risk polyp, by 92 percent.
"Our trial was the first to really show this benefit," Gerner told BTW this week. "The subtlety is that it is a cancer-prevention mode, not a cancer-treatment mode."
Gerner said that their data attracted a "significant" amount of interest from pharmaceutical industry representatives at the meeting. Believing this was the experimental breakthrough that was needed to attract a potential commercial partner, the researchers and their tech-transfer offices began shopping their IP around.
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This effort has been unsuccessful, however, as the scientists met with a common tech-transfer roadblock: The treatment was deemed too early-stage and too risky by potential commercialization partners.
"This is a difficult technology to commercialize," Nina Ossanna, business development director at the Bio5 Institute and UA, told BTW. "It has been hard to license out to existing companies because it's cancer prevention as opposed to a therapy."
Gerner explained that "the problem is cancer prevention is not part of any oncology company's business plan in 2009. They look at our results, they're impressed, but they look at a situation like Vioxx, which was developed for inflammation but tried in cancer prevention with [the non-steroidal anti-inflammatory] Celebrex. But [these drugs] caused unexpected cardiotoxicity and ended up costing those companies a lot of money."
Gerner added that he understands the skittishness, but said that he and Meyskens have conducted extensive toxicity tests on the eflornithine-sulindac treatment to make sure it has "a minimum amount" of toxicity.
Sulindac is similar to aspirin, and "even aspirin is not totally safe," Gerner said.
"Our goal is to develop drugs like this, that are not going to be totally safe for everyone but will generally not make you sick," Gerner said. "We're targeting populations with an increased risk of cancer – a genetic predisposition, family history, et cetera. We're trying to develop strategies that people with increased risk can use with the same kind of safety as a drug like aspirin."
Therefore, the researchers decided that the best way to do this would be to create their own company, CPT, which they incorporated late last year with personal funding and money from friends and family. The enterprise did not become official until last week, when the Arizona Board of Regents approved Gerner's participation.
"Anytime a faculty member owns more than 3 percent of a company, it has to be approved," Ossanna said. "He didn't get the blessing to do this until the board meeting."
It is unclear whether Meyskens needed to obtain similar permission from administrators at UC-Irvine or the UC system.
"The other issue is that our company is now trying to license the patent, to which UA and UC are assignees," Gerner said. "That's part of what led to that requirement from the board of regents to approve any conflict of interest. UA has specifically been trying to encourage university faculty to interact with the private sector or develop new companies that might help commercialize research conducted at the university."
As far as the licensing deal goes, it has been approved in principle by the board of regents, but is still undergoing "some final bits of review by our attorney and UA's attorneys," Gerner said.
As part of that deal, both UA and UC would likely retain some financial stake in the company, and would split any profits if CPT is able to commercialize the therapy, according to Ossanna and Gerner.
CPT's next goal is to move the treatment into clinical trials, "and that means we need to get an FDA-approved indication," Gerner said. "We're working with NCI and FDA to design clinical trials that could lead to approval, and we're hoping to complete those studies in the next two to five years."
The NCI has awarded Meyskens UC Irvine lab $750,000 in support of the trials, and CPT is using an undisclosed portion of its NCI SPORE grant to offset the costs. But the company is "definitely" seeking additional funding, Gerner said, mostly to manufacture the drugs for use in the trials.
"We are in the process of negotiating for a new CEO for the company, and one of their major tasks will be to establish contacts and find additional funding," Gerner said. "We're also close to signing two agreements with different major pharmaceutical companies … for the initial manufacture of these drugs, which will be a significant cost." He declined to disclose the companies, citing a desire to keep the ongoing negotiations confidential.