Dr. Niederhuber's Remarks at the ASCO Opening Session
The following is an abridged version of the prepared remarks delivered by NCI Director Dr. John Niederhuber on May 31 during the opening session of the 2008 ASCO Annual Meeting in Chicago, IL. This text represents the basis of his remarks and may have been modified slightly during delivery.
Good morning and thank you for asking me to appear before you today. … We are extremely fortunate to have such outstanding leaders of [ASCO] - individuals who so clearly understand the times we live in and the challenges and opportunities that lie before us - as we lead a transformation of science and medicine in the post-human-genome era. Among our many challenges, there is none, I believe, greater than the necessity that we more rapidly translate our discoveries, our new interventions, to patients.
As oncologists, as cancer physicians, we become intimate with the fears of our patients. Sitting at the bedside, gazing into the frightened, tearful eyes of far too many men, women, and children just learning they have cancer, we know that statistics are never simply numbers. They are very real individuals, each in his or her own way grasping to cope with news that, as a young physician being treated for Hodgkin disease recently reminded me, "is a life-changing event."
Recently, cancer has deeply touched us. Last year, at this podium, we honored a lifetime of dedicated service and leadership by our great friend and revered colleague Marty Abeloff. This year, we are all diminished by his absence. His loss, although months past, is still fresh, still palpable, and still painful to his colleagues and friends.
We also remember - I, especially - Judah Folkman, a legendary surgeon, educator, and researcher, whose skill, vision, and compassion we dearly miss and shall never forget.
Cancer intrudes into our national consciousness, as well. Just a few weeks ago, the Honorable Edward Kennedy chaired a special hearing of the Senate Health, Education, Labor, and Pensions Committee to consider ways our country could invest in and reinvigorate its collective efforts against cancer. Today, Senator Kennedy is courageously negotiating the treacherous path of the very disease he has worked so hard, for so many years, to fight. It is impossible to escape the irony that one of the architects of the landmark National Cancer Act of 1971 now turns his battle inward.
I daresay none of us has escaped cancer's scars, whether we wear those marks personally or symbolically, on behalf of a loved one.
As a nation, I believe we must confront the fact that a rapidly aging population and its cancer burden will weigh heavily, not only on health care, but on the workforce and our economy. As cancer researchers and cancer physicians, we face an inescapable question: Can we sustain our momentum and continue to lessen the all too devastating impact of cancer?
I believe that we are all here in Chicago this week, thousands strong, to resoundingly answer: "Yes."
We are here to listen and learn and share and discuss - and even debate - the promise of science and the assurance that we will not rest until that promise is delivered to all of our cancer patients, whether they seek treatment in a world-renowned university cancer center or in a rural community hospital. And we must resolve to consign to history the inequalities of cancer treatment that arise from age, frailty, race, ethnicity, poverty, and language.
As director of the National Cancer Institute - your National Cancer Institute - I stand here this morning to recommit NCI to make every effort toward the healthy tomorrows we all envision. I extend this promise fully aware of the challenges [presented by 4 straight years of funding that have been consistently below the rate of medical inflation].
I also know that our cancer patients don't want to hear explanations about flat budgets. They want prevention options. They want effective, novel therapies. NCI has an obligation, to our patients and to our country, to set the agenda for and to facilitate cancer research in the decades ahead. I believe, as well, that NCI is uniquely poised to bring together industry, academia, and government in the search for solutions none could provide by itself.
We come together during a time of unparalleled discovery, of rapidly mounting information about the alterations of the code of life that become aberrant and lead to cancer. Consider, if you will, The Cancer Genome Atlas, often called TCGA for short, to denote the four DNA bases. Co-sponsored by NCI and our sister NIH institute, the National Human Genome Research Institute, this 3-year pilot project is an effort to test the feasibility of large-scale characterization and sequencing of patients' tumors. We have sequencing centers up and running, along with tissue characterization centers making sure samples archived for sequencing (up to 500 samples of both tumor and matched normal tissue) are of sufficient quality.
The first cancers being studied in this pilot program are lung, ovarian, and glioblastoma multiforme. In part, because high-quality tumor tissue has been available, glioblastoma has led the way in this effort. To date, more than 234 tumors have undergone comprehensive characterization and 1,300 genes have been sequenced, and some fascinating associations have been revealed - specifically, two genes that are highly associated with glioblastoma: NF1 and Erbb2. Neither of these genes was previously known to have an association with this disease. In addition, previously unknown changes in the EGF receptor and the p53 tumor suppressor gene are now known to be part of glioblastoma's course. Based on this new, very preliminary analysis, at least four general subtypes of glioblastoma are emerging.
Tumor sequencing of this sort, along with the rapidly advancing field of whole-genome scanning, are, without a doubt, at the forefront of cancer science. Yet, for now, this is just information: a collection of T's, C's, G's, and A's. Our challenge is to convert this powerful information into knowledge of how gene expression is transcriptionally regulated; how that expression is translated into proteins affecting signal pathways; and how these changes eventually alter tissue function and disease phenotype. These determinations will not be easy.
Ultimately, the power of the genome will be to push forward the development of new strategies in prevention, new opportunities for early detection, essential biomarkers of disease, and, of course, novel drugs and new treatment approaches. We must be focused on enabling translation.
This need was made clear in a report in 2007 of NCI's Translational Research Working Group, which spent 2 years conducting a detailed analysis of how to realize the power of a bench-to-bedside approach. The report called on NCI to tailor both new and existing programs to facilitate early translational research progress. NCI is working hard to answer the report's call in five key ways.
The first area is clinical research. We are fortunate to have, on the NIH campus in Bethesda, the world's largest hospital principally devoted to clinical studies. It is the home to a dedicated cadre of NCI intramural science programs and scientists. But we have also come to believe that, in order to facilitate the best translational efforts, this outstanding facility should become a resource for the extramural community. For the first time, we have a trial open with an extramural co-principal investigator, and there are several more such trials pending.
Additionally, we are reaching out to both the new Walter Reed National Military Medical Center, which is due to begin construction across the street from NIH this summer, and Suburban Hospital, which is across the street on the other side of campus. These two facilities have the potential to give us an opportunity to work on prevention and to see more early stage patients.
Our second goal is drug development. NCI often works with orphan opportunities: small molecules and biologics that are not being developed by industry because they lack a significant market share or they are too risky to pursue. We must enable that work. We see drug development as a platform that encompasses a new chemical biology consortium and extends NCI's Developmental Therapeutics Program. We have a duty, I believe, to develop new agents and to carry them forward to first-in-human studies.
Drug development will make our third goal essential: Reaching out to work with both the Food and Drug Administration and the Centers for Medicare and Medicaid Services, connecting regulators and those responsible for reimbursement. Just one example is our Interagency Oncology Drug Task Force that, among other functions, sponsors NCI-FDA fellowships to train young scientists in research and in research-related review, policies, and regulations.
Public-private partnerships will also be increasingly necessary. As we move to a more personalized era of oncology, it is clear that we will require multiple agents to target multiple pathways in the same patient. Facilitating that future will challenge how we think of competition, intellectual property, and even the language of contracts. I believe NCI must step into those areas and become the facilitator between the public, private, and academic sectors. I believe it is a fair question to ask: If not NCI, then who will lead this absolutely vital effort?
Fifth and finally, if NCI is to appropriately facilitate translation, we must make every attempt to provide stable funding for some of the most important programs at the heart of translational research. Specifically, I am referring to clinical trials, the Specialized Programs of Research Excellence (SPOREs), the Community Clinical Oncology Program (CCOPs), and the Cooperative Groups.
Although it is entirely possible that NCI's below-inflation funding may continue for several more years, we will do - and must do - all we can to keep these crucial programs vigorous.
Overarching all that we do, NCI has a duty to make every effort to reduce the disparities that affect cancer prevention and treatment. I am convinced that access to our latest science will be the greatest determinant of cancer mortality in the years to come.
To address this we are now beginning the second year of our NCI Community Cancer Centers pilot program, a research effort to study how we can best bring state-of-the-art, multi-specialty care, and earliest-phase clinical research - phase I and phase II studies - to patients in the communities where they live. It gives me great pride to hear the 16 community hospital [pilot] sites report on their enthusiastic efforts, and how much they have been able to leverage the modest funding provided by NCI. It is also extremely gratifying to know that these pilot sites, seven of which have links to the CCOP program, are participating in ASCO's Quality Oncology Practice Initiative.
All of NCI's efforts - and certainly all of your efforts as oncologists - are about individuals with cancer. We have made many, many advances, and today we are witnessing perhaps the most exciting era in science's history.
I would like to conclude this morning with the story of a patient. It is a story not about a common occurrence or even one that may be replicated. It is a story about the power and the importance of your National Cancer Institute. It is a story of NCI's work in drug development and of facilitating early translational research.
When our patient arrived at the NIH Clinical Center, she couldn't even make a fist. Her hands, wrists, elbows, and knees could scarcely bend. Her skin was cracking, and she suffered the pain of many open skin lesions. Her face was swollen and disfigured. A once-vibrant woman in her late twenties, she was now severely anemic, wheelchair bound, and wrapped in blankets to preserve the body heat her skin could no longer retain. Over 2 years, as she suffered the disabling and constantly progressive manifestations of cutaneous T-cell lymphoma, the nights spent in the hospital had come to greatly outnumber those spent at home.
She was in hospice care and lacked the strength to be with her two small children. She came to the Clinical Center virtually out of treatment options, and once there, an initial short list of experimental treatments all failed.
Having apparently run out of hope, our patient's story came to the attention of Dr. Martin Gutierrez, a staff clinician with NCI's Medical Oncology Branch. Dr. Gutierrez, who has spent his career working with T-cell lymphoma patients, suggested trying a new drug being developed through NCI's well-known RAID Program. The Rapid Access to Intervention Development Program, as you are well aware, exists to speed the translation of novel anticancer therapies from our laboratories into first-in-human studies.
The drug utilized by Dr. Gutierrez, fenretinide, was one that was developed but never marketed by a pharmaceutical company as a chemopreventive agent. It was brought to the RAID program by an extramural investigator from Los Angeles, who wanted to reformulate it as a potential chemotherapeutic drug, based on observations he had made in laboratory models.
In this case, the drug paid off, and paid off dramatically. Within the first few doses, Dr. Gutierrez began to see remarkable improvement. Within 7 months, the patient's symptoms were completely gone. Today, almost 2 years after her arrival at the Clinical Center, the patient's tests show no evidence of disease.
To me this story - not a report of a large clinical trial, but involving just one patient - illustrates many aspects of the role of NCI. It was not the most common cancer problem and not the most common potential therapeutic compound. Instead, it is a great example of the partnerships between the extramural community and NCI in the development of novel agents. It is, I believe, an example of the increasing need for the development of new therapies that are not attracting the prime interest of the private sector. It is about knowing the science and using that knowledge to drive drug discovery; about using genetics to drive high-throughput screening; about the power of a large workforce of chemists to address formulation; and about scientific opportunity, without the consideration of shareholder profit.
We are all looking to the day when stories like this are commonplace, when we make cancer a more chronic, manageable condition.
We have many miles and, yes, a few mountains ahead of us on [our] path. My commitment to you is that your National Cancer Institute will walk beside you, helping you negotiate every step.
May our days here in Chicago renew our spirits, renew our commitment, and recharge us for the walk ahead. Thank you for all you do and for your support, and thank you for being here this morning.