TAMPA — Locked away in a brick-and-glass building spanning the equivalent of two city blocks, thousands of frozen human tissue samples await scientific use.
The barcode on each sample represents someone's worst nightmare: a diagnosis of cancer. But to the Tampa Bay region, the repository holds the promise of breaking scientific ground while creating high-paying biotech jobs.
That's why more than $35 million in public dollars were invested in M2Gen, a for-profit research subsidiary of Moffitt Cancer Center. But five years after its launch, its leaders say it's still too early to judge the results.
Now M2Gen faces challenges that could shape its future:
• A partnership with drug giant Merck that bankrolled nearly $100 million — the majority of the venture — expires at the end of the year. M2Gen is negotiating with Merck and seeking other sources of revenue, but officials acknowledge the sour economy is weighing down prospects.
• Explosive scientific change is outpacing technology considered cutting edge just a few years ago. Moffitt could see its most valuable asset — its detailed genetic analyses of nearly 17,000 of the cancer specimens it has stored — become outdated and less valuable.
• M2Gen has yet to turn a profit. Moffitt wants to use its enormous tissue and bioinformation database to quickly find patients for clinical trials who are most likely to benefit from new therapies. That could speed up the process of bringing drugs to market. But it's an unproven model.
"What we're trying to do is increase the probability of success — both for the patients as well as the drug company," said Dr. William Dalton, Moffitt president and CEO.
M2Gen has signed up nearly 80,000 patients whom Moffitt will follow for life and can recontact when promising new drugs surface.
And it has notched another important success: a 10-state coalition of hospitals, doctors and patients that has helped to propel Moffitt into the worldwide race to discover cancer's genetic vulnerabilities. That coalition, plus the tumor bank, are the linchpins of Moffitt's dreams for M2Gen.
But Moffitt has lots of competition in this race from other cancer centers, all entrenched in a reality that has dogged scientists and patients since President Richard Nixon declared war on cancer four decades ago.
As Dalton puts it: Progress may not be "as fast as we'd like it.''
Still, he says confidently of M2Gen's goals, "we are well on our way."
• • •
In the not-so-distant future, leading cancer centers expect to offer genetic testing to every patient, then use the results to tailor therapies.
For some tumors — such as certain breast cancers — some such treatment is available. But researchers say more extensive genetic profiling, coupled with precisely targeted cancer drugs, is needed to realize the vision of personalized medicine fully.
That's the goal of M2Gen, short for Moffitt Second Generation. Moffitt created the for-profit company, which is wholly owned by Moffitt and doesn't release its own financial information, to manage its multimillion-dollar partnership with Merck.
If it leads to any blockbuster drugs, Moffitt plans to channel its share of the profits back into research.
The research collaboration started out trying to discover genetic markers in cancers, then develop drugs around these unique attributes. While there haven't been headline-generating breakthroughs, researchers have seen quieter successes.
For example, M2Gen's chief scientific officer, Dr. Timothy Yeatman, points to Merck's identification of a molecular fingerprint that led collaborators to propose a drug combination that no one had ever considered before and that is being vetted in clinical trials. But he declined to say more, citing confidentiality agreements.
"We've yet to really see the full impact of this project, sort of like the best is yet to come," Yeatman said.
Next, M2Gen aims to leverage the database of information it has gathered to run more effective clinical trials, speeding a process bogged down by the difficulty of finding qualified patients.
"Smarter, better, faster," said Yeatman of the model, which he believes will allow companies to enroll patients in clinical trials in six to eight months, a process that now can take two years. "The advantage that we have over everyone else in the world is the ability to find a patient based on a pre-existing genetic map."
In a preliminary test of the concept, M2Gen recruited nearly 40 colorectal cancer patients to test a drug, completing the trial phase within a year. Although results haven't yet been published, leaders say they have drawn industry interest.
To prove it was more than a one-time success, M2Gen now is working on two more trials, with others in planning stages.
"It's a very new approach for (the companies) operationally," said M2Gen CEO Cathy Kerzner. "It's come as a result of the size of the database and how quickly it has progressed."
• • •
The initial Merck-Moffitt partnership, which Gov. Jeb Bush touted as "a milestone day for Florida," expires at the end of the year.
In February, Kerzner told the Times that Merck had signaled it would scale back its commitment. Merck has announced plans to cut thousands of jobs and close several research centers.
"It's been tough for (the pharmaceutical industry) the last five years," said Dalton, Moffitt's CEO. "That's part of why we've had to regroup."
Neither Moffitt nor Merck is commenting on their next steps together.
"Moffitt has positioned itself to be in an excellent position to tackle the next step of personalized medicine," said Dr. Boris Pasche, director of the division of hematology and oncology at the University of Alabama at Birmingham and an expert in cancer genetic research.
But that doesn't mean riches will follow.
"Profit is one thing, and not losing too much money is another thing," said Pasche. Other institutions, he said, have found that large tissue banks are incredibly expensive to maintain. "It certainly is not going to be a gold mine."
• • •
Another challenge to M2Gen: Technology is evolving so rapidly that advances can quickly become outdated.
For instance, in the past five years, the cost of running a genetic test that allows scientists to sequence the DNA of tumors has plunged more than 100-fold.
But it was still cost prohibitive when Moffitt set up its protocol. So rather than sequencing, it uses a testing method that studies the messages transmitted within cells to understand how cancer works.
Even as DNA sequencing becomes more prevalent, Moffitt officials believe that the information they have amassed will remain scientifically relevant for another 15 to 20 years. And during that time, they should gather more medical information on their patients, which could add value to the overall database.
How scientists collaborate on cancer genetics is also changing.
Moffitt's work remains largely private under the terms of its deal with Merck. But others in the research community increasingly are working together to map the cancer genome — and some think Moffitt should, too.
"Many in the community are awaiting the information and data from the major effort at the Moffitt Cancer Center to be made public, so it can be used efficiently to improve patient care," said Dr. Gordon Mills, co-director of the Institute for Personalized Cancer Therapy at the University of Texas MD Anderson Cancer Center.
Moffitt officials downplay the competition with prominent institutions like MD Anderson, arguing that the size and quality of their database sets it apart from others.
"We have been dedicating ourselves to building the infrastructure to build something that doesn't exist anywhere else in the world. To that end, we are successful," said Dalton, Moffitt's CEO. "We're as excited as we were five years ago, if not more excited."
Times researcher Caryn Baird contributed to this report. Letitia Stein can be reached at [email protected] or (727) 893-8330.