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Capitalizing on our differences

Personalized cancer therapy means delivering “the right dose of the right drug for the right indications to the right patient at the right time,” says Gordon Mills, M.D., Ph.D. , director of the Khalifa Institute. Photo by John Smallwood

By Dawn Dorsey

Each person is unique; each tumor is unique. This seemingly simple truth is the premise of personalized cancer therapy, a growing field based on analyzing these differences to find keys to unlock the mysteries of cancer.


At MD Anderson, the Sheikh Khalifa Bin Zayed Al Nahyan Institute for Personalized Cancer Therapy, named in recognition of a transformational gift from the Khalifa Bin Zayed Al Nahyan Foundation, is perfectly poised to lead the way in this growing field, which has the potential to revolutionize cancer care and prevention. By targeting specific genetic and molecular abnormalities in each tumor and each patient’s body, scientists are learning to match patient and tumor with the most effective treatments.

Listen and Learn
“In personalized cancer therapy, we listen to what the patient’s body and the tumor are trying to tell us,” says Gordon Mills, M.D., Ph.D., director of the Khalifa Institute for Personalized Cancer Therapy. “If we just pay attention, patients can teach us incredible things.”


“For instance, in many clinical trials a subset of patients shows remarkable responses. These patients are trying to tell us something if we will just listen. Their tumor or genetic makeup is saying, ‘This is the right drug for me.’ If we can identify more patients who ‘look’ just like these, then we can have another major leap forward.”


It’s not that researchers didn’t want to listen in the past, Mills says. They just didn’t have the tools.


“We now have incredible technologies that can analyze at diagnosis what is going on in each tumor and find out at an early stage how that tumor is responding to therapy,” he says. “Putting these together, we’re getting better and better at pairing patients with the right treatments.”


On a mission
With new leadership, including Mills and John Mendelsohn, M.D., who steps in as co-director as of Sept. 1, the Khalifa Institute is laying the groundwork to make quantum leaps in personalized cancer therapy. And they’re armed with a new mission statement that leaves no room for ambivalence.


The goal of the Khalifa Institute is for MD Anderson investigators to define the new standard of patient care ― making personalized cancer therapy available to all new patients by the end of five years, with full third-party reimbursement based on evidence from clinical trials. This research will revolutionize the way we manage cancer patients.


“Today this approach and most of the targeted treatments are considered experimental,” Mendelsohn says. “Our goal is to perform research proving efficacy, so this will become standard of practice, reimbursed by private and governmental payors. Our ultimate goal is to cure more patients with cancer.”


MD Anderson’s bold approach shows its deep commitment to personalized cancer therapy. Ambitious plans include completing by 2014 the Sheikh Zayed Bin Sultan Al Nahyan Building for Personalized Cancer Care, a 600,000-square-foot state-of-the art facility made possible by the above-mentioned generous gift from the Khalifa Foundation. A visionary pilot project dubbed T9 and led by Mills is one of the first orders of business.


Multiplying the possibilities
T9, short for Ten Thousand Tumors, Ten Thousand Tests, Ten Thousand Therapies, aims to bring the promise of personalized therapy to reality. During the next two years, researchers will analyze the cancer-causing genetic variations in the tumors of 10,000 patients with advanced cancers that have no standard therapy. Doctors will refer their patients to clinical trials of experimental drugs and other anti-cancer agents that target those specific genetic aberrations.


“T9 will bring together all the molecular knowledge we’ve gained during the past 20 years,” Mills says. “We’ll also gain new knowledge and pair it with advanced technology to deliver the right dose of the right drug for the right indications to the right patient at the right time.”

A ‘perfect storm’
A number of factors come together at MD Anderson to distinguish the institution as a driving force in personalized cancer therapy.


“Among centers providing care for cancer patients, MD Anderson is in the best position to lead in developing personalized cancer therapy,” Mendelsohn says. “We have the largest number of patients, the most experience in investigating experimental treatments and an outstanding faculty committed to this research. And we already have a track record of innovative clinical trials with experimental drugs targeting genetic aberrations in patients’ cancers.”


In fact, MD Anderson scientists have identified critical genetic aberrations across several tumor types, including melanoma and cancers of the breast, lung, bowel, pancreas, brain, ovary and endometrium, that account for the vast majority of deaths from cancer. And the Khalifa Institute has been involved in recent major studies with the potential to change the treatment of some types of melanoma and breast cancer.


“MD Anderson brings together all the elements to make progress in this exciting field,” Mills says. “We see 30,000 new patients a year, many of whom are interested in participating in research studies. We have an extraordinary faculty dedicated to this approach, and the institution encourages and rewards them for doing high-level, innovative research.”


MD Anderson has built a massive and effective infrastructure to begin to do this type of research, says Mills.


“There’s a perfect storm at MD Anderson that gives us the best opportunity to move personalized cancer therapy forward,” he says. “The Khalifa Institute’s work will help make personalized cancer therapy the routine way to help patients, not an exception.”



© 2014 The University of Texas MD Anderson Cancer Center