Every day we hear about a new drug, screening tool or technology that promises to improve our knowledge of health and disease. Generally, the news is good, or at least hopeful, reassuring the public that important research is progressing. Often, the new breakthroughs have been demonstrated in the lab or in animal models and has not yet been studied in humans, a process that could take years. Sometimes the lab findings do not translate well to the human and the researcher may have to start all over with a new strategy. This is particularly frustrating for individuals who have a disease or condition that is debilitating and could be deadly. The new movie, Extraordinary Measures, is the story about a father who self-funds a researcher to accelerate the process for a promising drug for Pompe disease because his children have the condition. It is a great example of how driven and focused we can become when our own loved ones are ill.
The research community is challenged when what we know now (based on the science technology and data available at the time), changes when new technology allows us to further explore a particular issue. Or when there is more data in a statistically significant pool that truly reflects our population diversity and allows us to determine differences that may exist among different races and ethnicities. This could lead to different approaches for certain populations. We’d like to think of research as finite but it is a constantly revolving cycle. One important example is the recent mammography guideline debate. Several years ago, mammography screening became mainstream: most women knew the guidelines, advocates worked to pass laws to assure that all women had access, and early studies showed that finding breast cancer early, in many cases, improved survival rates. Last November the U.S Preventive Task Force new mammography guidelines that were posted by Candace questioned the benefit of the current guidelines as applied to the general population. It should be noted that this is the first update by the Task Force since 2002. This led to conflicting reports of support verses outrage. There are some valid arguments on both sides and the new report was based on additional data. The outrage of strong proponents of mammography often overlooked the details of the report that acknowledged that high-risk women may want to discuss their screening intervals with their health care provider. On the other hand, the team behind the report were criticized for not fully explaining that there were high-risk populations that required further study. These exceptions would not change their overall recommendations, but they could have made it clearer that there could be exceptions that do not apply to the population at large and that they were sensitive to the emotional backlash this report would produce.
Advocacy is a powerful activity that has led to increases in research dollars and more sensitive care. But sometimes advocates are so focused that they do not see the big picture. An example of advocacy compromising good science is the debate in the 1990′s about High-Dose Chemotherapy with Bone Marrow Transplant or Stem Cell Support (HDCBMTSCS) as a treatment for breast cancer. HDCBMTSCS was seen as a promising aggressive treatment for the growing number of women who were being diagnosed each year with this disease. As appropriate, randomized controlled clinical studies (the gold standard that compared a new treatment with the latest standard of care) opened and tried to recruit women. However, many interested women did not want to be in the control group. After all, more is better! To respond to this demand from advocates, many hospitals opened bone marrow transplant units and insurance companies were pressured to cover this “experimental” treatment despite the lack of clinical trial evidence. And what happened? Women by-passed the trials and went to the institutions that offered the treatment outside of clinical trials. This delayed the evidence-based findings that eventually concluded that HDCBMTSCS was not better than standard treatment for breast cancer (it does offer a good option for certain other cancers) and that many women suffered far more severe side effects (that sometimes resulted in death) with little improvement in outcome.
So what do we do? It is important for men and women to understand enough science to enable us to interpret studies without the bias of headlines. Programs like Project Lead sponsored by the National Breast Cancer Coalition trains consumers to understand the basic scientific principles behind research studies. We need greater participation in well-designed clinical trials so we can get answers sooner so better therapies become available sooner. Finally, look for portals to research studies that not only study disease, they also look for better ways to prevent disease. One such portal is the Illinois Women’s Health Registry. Hopefully, more of these gateways will become available.