Making Progress in Difficult Times Will Require a Collective Effort
"No individual is alone responsible for a single stepping stone along the path of progress." This remark by Nobel Prize winner Ernest Lawrence comes to mind as we take stock of all that happened during this past year in cancer research.
In 2007, NCI put on full display the teamwork, collaborative effort, and maturation of scientific knowledge that, even in a time of constrained resources, helped ensure steady progress - with one discovery building on another. Certainly the Institute was forced to make some tough choices in order to redirect a less-than-inflation allocation of federal dollars toward our highest priority programs. Even with those limited resources, NCI's scientific accomplishments this past year were, to say the least, impressive.
In 2007, our genome-wide association studies (GWAS) validated and placed a remarkable amount of data into the public domain. GWAS is a truly collective effort that pools data from researchers studying large patient cohorts in order to identify common genes that confer modest amounts of cancer risk. These germline sites in our DNA, which are predictive of increased risk, act collectively, not alone. GWAS studies will help identify new environmental factors and biological mechanisms of disease as we layer onto GWAS data the genetic changes that accumulate during one's life. These genetic data, carefully analyzed and constructed, will be valuable resources in identifying new prevention and treatment strategies. GWAS studies published this year - several sponsored by NCI's Cancer Genetic Markers of Susceptibility project - identified common genetic variants that show increased risk of developing breast, prostate, and colorectal cancers, along with one germline region that appears to be protective against the development of breast cancer.
In another important area of cancer research, cancer biologists this year worked to test the hypothesis that within each tumor there is a small subpopulation of cells with the genetic properties of tissue stem cells. Some refer to this population as "cancer initiator" cells, and evidence supports their potential importance both in the resistance of tumors to therapy and in the process of metastasis. Therapeutic approaches that specifically target this subpopulation of cells are now being combined with standard anticancer regimens. Early clinical trials include a compound called DMAPT that NCI's Rapid Access to Intervention Development program is helping researchers to investigate, through an approach that "awakens" these quiescent cells to make them more sensitive to existing therapies.
Representing a highly leveraged team project was the launch in June of the NCI Community Cancer Centers Program (NCCCP). Focusing on underserved communities and groups that are disproportionately affected by the disease, the NCCCP's pilot phase involves 16 community hospitals working with NCI to identify the best research-driven strategies for delivering state-of- the-art cancer care in the community, where the large majority of cancer patients receive care close to home. The research conducted through NCCCP will help NCI bring early-phase clinical trials to such settings, explore development of electronic medical records, and enhance our ability to practice highly personalized cancer care. The ultimate goal is to ensure that all people, no matter where they live, no matter what their education or economic status, will have equal access to our latest science.
There was no absence of significant clinical advances this year, including the introduction of the phase 0 clinical trial at the NIH Clinical Research Center. Made possible by an extensive NCI-FDA partnership that resulted in the creation of the FDA Exploratory IND Guidance, these early studies use pharmacodynamic measurements or imaging to find the biologically effective dose for first-in-human studies of newly developed therapeutics. This new approach has already demonstrated its ability to significantly shorten the drug discovery process.
Another important clinical trial highlighted the significant contributions of imaging to cancer diagnosis and care, demonstrating that adding MRI to standard mammograms detects virtually all cases of contralateral breast cancer. A separate patient study provided a potentially important clarification about the use of finasteride to prevent prostate cancer in high-risk men.
We continue to make progress against specific forms of cancer. For example, this year began a new era for the treatment of advanced liver cancer, which had previously been impervious to new treatments. In yet another difficult-to-treat cancer type, small-cell lung cancer, researchers demonstrated that prophylactic cranial irradiation not only reduced the risk of symptomatic brain metastases, it also improved survival.
In financially constrained times, such as the NIH has experienced over the past 4 years, we are placing a greater emphasis on collaboration and finding ways to leverage scarce resources through cooperative external investment. None of that would be possible without NCI's senior leadership, who time and again this year worked together to make difficult funding and priorities decisions. These decisions were made with the utmost integrity, based on a belief that, regardless of the challenges or obstacles before us, we must work even harder - certainly with more collaboration and innovation - to support the best science. In 2008, we will continue moving forward one stepping stone at a time as we navigate this path on behalf of our patients.
Dr. John E. Niederhuber