Success key No.3: Focus on the intractable
The Mayo Clinic is initiating a personalized medicine service line with a focus on cancer patients where physician care has been stymied.
At least two Mayo Clinic facilities, in Florida and in Arizona, are engaged in clinical trials for patients with intractable cancer, such as those of the brain, kidney, liver, pancreas, and skin, and other cases with patients who have cancer with suspected underlying genetic conditions, according to Matthew P. Goetz, MD, associate professor of oncology and pharmacology at the Mayo Clinic College of Medicine.
The protocols will be included in the Mayo Clinic's Individualized Medicine Clinic, scheduled to open this fall at the three locations. Patients have been enrolled at Mayo Clinic Florida and Mayo Clinic Arizona for a pilot on whole genome sequencing, although final numbers have not
While the clinical trials are under way, patient care will be evaluated, Goetz says. The plans include multidisciplinary approaches, including a team of oncologists, as well as genomics and cancer researchers. The Mayo Clinic's centers "as mandated, are to rapidly move forward in individualized medicine in different ways," says Goetz. "We are doing that by supporting research to the tune of millions of dollars." Goetz did not disclose the full amount of Mayo Clinic research linked to personalized medicine.
"If you look at the healthcare landscape right now, there's a sort of personalized approach that has been going on for decades. We didn't call it personalized," Goetz adds. "With the best data we have now, there is not a smoking gun for what causes cancer. We are left with how do we best understand genetic alterations, and the resistance to our best therapies. We are not going to get rid of the old therapies, but probably will continue them, and understand what are the mechanisms for resistance, and study them in a way it doesn't take 18 years to find an answer."
At the Mayo Clinic Florida and Mayo Clinic Arizona, hospital researchers are examining specific types of cancer and how personalized medicine may make a difference.
Patients enrolled at Mayo Clinic Florida are diagnostic odyssey cases–people whose symptoms are suspected of having an underlying genetic condition or heritable cause but have eluded traditional diagnosis. These are often rare or poorly understood conditions. Hospital officials believe the whole genome sequencing may offer more clues for diagnosis, as well as potential treatment and prevention, Goetz says.
At Mayo Clinic Arizona, researchers have been using whole genome sequencing to look for novel treatments for intractable and incurable cancers. Now, patients who have exhausted traditional treatments will have their tumor genomes sequences to identity pathways to make them suitable targets for therapeutic drugs. Then physicians can identify unusual or experimental chemotherapies, such as drugs that may be usually used for breast cancer, but also may be suited for lung cancer. The tumor genome analysis also can help researchers understand why some tumors respond better than others to chemotherapy, Goetz says.
A significant Mayo Clinic undertaking in personalized medicine includes its Breast Cancer Genome Guided Therapy Study, in which 200 patients have been identified and will participate in a clinical trial examining mutations that would allow some tumors to adapt and thrive during chemotherapy.
The project focuses on women with high-risk cancers. As part of the study, patients are paired with mouse "avatars" that are expected to help doctors identify the best individual treatment, according to Judy C. Boughey, MD, associate professor of surgery at the Mayo Clinic. The patients' tumor tissue will be kept alive by implanting cell lines in immune-compromised mice before and after chemotherapy. The use of the mice will let researchers study the impact of chemotherapy on each patient tumor without risk of harm to the patient.
The study has "real potential to bring individualized medicine to our patients," Boughey says. For patients who have a "higher risk for reoccurrence" of cancer, the project can open the door to "go forward and identify drug development and focus on these patients," she adds.
Patients are increasingly showing willingness to undergo tests to receive advanced experimental treatment in clinical trials, according to a study by the Mayo Clinic and the Translational Genomics Research Institute in Phoenix.
Success key No. 4: Pharmaceutical contracts
For personalized medicine, more hospitals are forming contractual relationships with pharmaceutical companies to develop drugs that may give them an upper hand in the race for improved cancer care.
"We have a long history of working with pharmaceutical companies, over five years," Moffitt's Dalton says. Moffitt has developed a strategic partnership with Merck & Co., which helped underwrite some of the cost of building the infrastructure and has partnered for scientific discovery, Dalton says. By collaborating on a framework with a pharmaceutical company, new drug targets are being developed, he says.
M2Gen, the wholly owned subsidiary of the Moffitt Cancer Center, is supporting the operation of the research initiative, according to the hospital's annual report. M2Gen is partnering with Merck and other pharmaceutical and biotechnology companies to launch clinical trials using the biorepository for data on an IT platform with Oracle.
The Moffitt-Merck research collaboration was formed in December 2006. Funding sources included Hillsborough (Fla.) County, the city of Tampa, and the state of Florida. According to the report, the M2Gen venture is "dedicated to using molecular technology to identify biological markers." Under the plan, M2Gen operations "assist in collecting and processing data, while also carrying out certain monitoring responsibilities."
Without disclosing specific details of the arrangement with the pharmaceutical firm, Dalton adds, "They don't know who the patients are, but they are able to mine the data we are creating and determine new drug targets, then develop drugs effective in patients not responding to new therapy."
Other healthcare enterprises also are working with pharmaceutical companies to develop personalized medicine programs. Earlier this year, the Cancer Treatment Centers of America announced it reached a research collaboration agreement with Merrimack Pharmaceuticals, a Cambridge, Mass., biopharmaceutical company, to better understand cancer on the molecular level and accelerate personalized treatments for the disease. As part of the collaboration, CTCA will provide archived tumor biopsies from its extensive tumor databank and collect tumor samples for analysis.
The collaboration could result in companion diagnostics to guide treatment. Merrimack CEO Robert Mulroy said in a statement that "the collaboration represents the future of individualized treatment, where a hospital and biopharmaceutical company work together on research, which we hope will ultimately result in much better treatment of cancer patients."
The Mayo Clinic also acknowledges the importance of working with pharmaceutical companies for potential personalized medicine drugs. Those discussions are for "early-stage development of drugs, or maybe drugs that haven't been developed yet and we provide a target," says Goetz, of the Mayo Clinic oncology department. "We are looking at collaborating with multiple pharmaceutical companies and an array of partners."
Indeed, pharmaceutical companies are actively pursuing personalized medicine, with as many as half the U.S. drug companies developing personalized medicine programs, according to Christopher-Paul Milne, DVM, MPH, JD, director of research at the Tufts Center for the Study of Drug Development in Boston.
"I haven't heard that companies are backing off their commitments to push forward with personalized medicine, especially as they are increasing their efforts to demonstrate the value of their products compared to competitor products or other therapeutic options," Milne says.
The government also has become deeply involved in promoting personalized medicine, with the Federal Drug Administration coordinating programs for oversight of its research centers to specialize in such programs.
More than 40 years after Nixon's declaration, the White House this year released its national bioeconomy blueprint that laid out strategies in U.S. biotechnology, noting that "advances in recent technologies have increased the momentum of personalized medicine, customized healthcare based on specific genetic or other information of an individual patient."
This article appears in the September 2012 issue of HealthLeaders magazine.