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Curing cancer: our hope, our reality, our quest

This column appeared in The Boston Globe on December 4, 2013.

By Pier Paolo Pandolfi, MD, PhD

This is a moment of unprecedented opportunity in cancer research. It is reminiscent of where cardiovascular medicine was just 25 years ago, when a number of key scientific insights led to astonishing advances in treating heart disease. It’s my belief that in the not-too-distant future we will be describing similar outcomes for cancer patients, because we have embarked on a similar path, in which cutting-edge science and patient care will go hand-in-hand.

Personalized Medicine

Many of the breakthroughs in cancer are taking place under the banner of personalized medicine. This began with the Human Genome Project, which in 2003 sequenced the entire human genome, thus enabling the discovery of new genetic elements relevant to disease. Today we have built on that breakthrough, and are on the brink of developing genomic tests that can not only detect an individual’s propensity to disease, but can also predict its future course as well as the efficacy of various treatments. These discoveries have established that cancer is genetically diverse: each tumor type is actually representative of several genetically distinct tumor sub-types. It turns out that cancer, too, is personalized.

The Holy Grail

Targeted therapies are the holy grail of personalized medicine and have shown much promise, in some cases achieving dramatic results. But we should be mindful that cancer remains a wily enemy: it evolves rapidly, frequently leading to drug resistance. Rapidly advancing technical capabilities have enabled us to develop gene-based therapies that go far beyond our current broad spectrum, highly toxic chemotherapies. Thus, the “disease management” of today can be transformed into real cures.

Targeted cancer therapy based on the genetics of an individual’s cancer can work. I know this because I’ve seen it work. Early in my career, I had the privilege of directly participating in genetic research that led to the cure of acute promyelocytic leukemia, a once fatal form of cancer. Cancer can be cured. We have done it before, and we will do it again for other types of cancer.

Reasons for Optimism

My faith that cancer can be cured is further bolstered by recent scientific discoveries, which we have contributed to here at BIDMC, that disrupt long-held ideas about the genetic bases of cancer. The central dogma of biology held for many years that genetic information flows from DNA, to RNA, to proteins in the cell. Yet oddly, 98 percent of the human genome does not code for proteins, and so was viewed as nothing more than useless “junk DNA” filling the space between genes. Our discoveries, however, have highlighted the relevance of non-coding RNAs that don’t encode proteins — the so-called “non-coding RNAs” — in cell growth and tumor development. This in turn forces us to reconsider the role of “junk DNA,” and revolutionizes our approach to cancer by multiplying the amount of genetic material worthy of investigation, and expanding the possibilities for finding cures. In the coming years, cancer programs at academic medical centers will routinely sequence entire cancer patient genomes and go far beyond protein-coding DNA to analyze patients’ RNA and “junk” DNA as well. These developments promise to lead the way to true, precision cancer care, and allow us to target genetic defects we previously didn’t even know existed.

Rethinking Clinical Trials

This entirely new approach to cancer therapy will enhance the ever-expanding list of targeted therapies and require significantly more clinical trials. Unfortunately, the current process of testing new, targeted therapies is slow and cumbersome, and as cancer is not one but many different types, not enough cancer patients may be available to participate in the large trials necessary to evaluate safety and efficacy of each new therapy. Indeed, in a world of personalized medicine, clinical cancer research as it is currently conducted is not sustainable. Neither the time nor the resources exist to evaluate single drugs (let alone combinations) in every cancer. We have to rethink the cancer drug testing process, streamline it, and maximize its benefits to patients. Critically, we also have to increase the number of patients enrolled in clinical trials. Thus, as BIDMC has been realigning its network to better serve its patient community, so too must our Cancer Center evolve, expand and realign its operation and alliances to meet these challenges head on.

Co-Clinical Trials

Here at Beth Israel Deaconess we are adopting the use of genetically engineered mice to rapidly evaluate new therapies for cancer patients, in what we call “Co-Clinical Trials.” In this promising new paradigm, each cancer patient is assigned an avatar — a mouse with the same genetic profile as the human cancer patient. Both are enrolled in the same clinical trial and studied in parallel as their cancer, and treatment, progress, allowing us to ask and answer questions about why treatments are or aren’t working. This approach is now providing us with insights that can enable swift evaluation and approval of new, lifesaving drugs.

A Crucial Alliance

Although these approaches may seem fanciful to some, this new research platform can be developed at a far lower cost than conventional clinical trials, and so must be explored if we are to conquer cancer with our current resources. The experience of losing both of my parents to this devastating disease has been a powerful personal motivation in my quest to make targeted cancer therapies a reality, and my sense of urgency is shared by both my clinical and scientific colleagues at the Cancer Center at Beth Israel Deaconess Medical Center. I believe an alliance like the one we envision between medicine and science is crucial in a moment so transformative for cancer research and patient care. I invite patients, their families, our colleagues, and the greater Boston community to follow our progress as we move from offering a standard of CARE, to providing a standard of CURE.

Pier Paolo Pandolfi, MD, PhD, is Director of the Cancer Center at Beth Israel Deaconess Medical Center and
of the Cancer Research Institute