Ever since graduate school, I’ve gravitated toward the nonprofit funding sector. There’s something about seeing all the people with their friends, fellow walkers, runners, dogs, and kids spending Saturday mornings walking, running or some other –athoning that makes me appreciate my paycheck a little bit more.
I’ve had the privilege of having my salary supported by more than one nonprofit through the years. I appreciate it, and it makes me work just a little bit harder, which is why it is disheartening to hear the kinds of misconceptions about medicine, science and research that seem to circle walkathons, clothing drives and support groups. I once heard that all research could be done on computers (the last time I’ll listen intently to one of the former “Bangles”), that bee stings cure multiple sclerosis and that scientists only want to study certain tumors.
At first I didn’t give comments like the last one much thought, respecting the process we all go through as we move from “un-informed” to “informed.” However, I think that widespread misstatements can be so damaging that they sometimes prevent us from getting proper care, feeling we are supported, and prevent us from taking action that will make a difference for ourselves and others. Although I respect Farrah Fawcett’s life experience and right to make statements she sees as important, I believe some of the comments she made on-tape for millions to see bear some attention.
In her documentary, Ms Fawcett stressed her confusion over medical issues that “get only passing mention” and why our healthcare system doesn’t embrace alternative treatments that have proven successful in other countries.
While I’d love to cite some secret-society funding and advancements in less-studied diseases, demonstrating that all is fair in the world of research, it is true that some diseases receive more attention than others. There are many reasons for this. One reason may be the availability of material to study. It stands to reason that rare diseases offer less opportunity for study than more common illnesses whether study occurs during illness or after someone has died.
Another limiting factor related to the availability of material for study is the ability of scientists to study that same material in vitro, or in a petri dish under a microscope. For example, some cancers that take up residence in the body don’t survive under laboratory conditions, making it difficult to study their progression and examine ways in which they could be treated. This is one of the reasons there are ten times more scientifically peer-reviewed publications in the genetics of glioma versus medulloblastoma even though medulloblastoma is the most common form of pediatric brain tumor.
A third limiting factor is the availability of appropriate animal models. Once scientists learned that animals were similar to humans, in physiology and anatomy, it became preferable to use animals rather than humans. Interestingly, diabetes, arthritis and hypertension all occur naturally within animals. By controlling the animal’s environment or “lifestyle” (i.e. food intake, exposure to toxins), scientists are able to introduce one variable at a time, observing the results of that one intervention. Genetic engineering added another layer of availability, allowing scientists to create models of disease not normally found in animals, like when researchers manipulate an animal’s genome to overproduce α-synuclein protein to mimic the early effects of Parkinson’s disease.
Animal models are important, not only for investigators but also patients. Indeed, what model is used for preclinical testing of new therapies will influence the success of these therapies in the clinic. Alas, some human diseases such as HIV-1 or AIDs do not have animal models available for study, because HIV-1 does not cause disease in any non-human species. Others, such as rodent models of Alzheimer’s, develop only some of the pathology we see in humans. No model develops real Alzheimer’s Disease pathology, which means that the predictive power of the model is poor and may lead to erroneous results.
Some models are available that have reasonable predictive power but are cost-prohibitive, difficult to sustain, or have other characteristics that make it difficult to obtain statistically-significant findings resulting in new advancements. For example, sheep have long been used to study fetal development for their gestational similarity to humans, but their relatively long (145 day) gestational periods along with the cost of care, housing, and feeding results in fewer publications and findings than studies that use, for example, mouse models.
Another reason for the difference we see in the amount of research done in one area versus another is funding. Many of us in research like to think of ourselves as independent thinkers and scientific entrepreneurs, but the fact is that we can’t study anything without funding. Funding levels tend to rise when conditions like autism reach the consciousness of federal funding agencies like the NIH, whose on-staff scientists are responsible for making sure we are covering any scientific gaps, or legislative stewards of research funding such as the House of Representatives' Committee on Science and Technology.
Anyone may draft a bill; however, only members of Congress can introduce legislation, and by doing so they become the sponsor(s). With few exceptions, bills are referred to standing committees in the House or Senate, reviewed, edited, debated and voted upon. After a bill has been approved by both the House and Senate in identical form, it is sent to the President who can sign it into law or veto it. HR2112, or The Lung Cancer Mortality Reduction Act of 2009, introduced April 27, 2009, would require NIH, the Centers for Disease Control and Prevention (CDC) and other federal agencies to establish research programs to reduce the mortality rate of lung cancer by 25% by 2015. The Lung Cancer Alliance, a nonprofit organization supported by donations, walkathons and other giving actions was a major supporter of this bill and instrumental in bringing its message to members of Congress.
Regarding Ms Fawcett’s second comment, about “why our healthcare system doesn’t embrace alternative treatments that have proven successful in other countries”- I think Ms Fawcett’s comments echo a recent shift in public perception over alternative therapies. Whereas 15 years ago, alternative therapy was considered “mumbo jumbo,” today, over one-third of Americans use alternative medicines; many of which are available on drugstore shelves, giving consumers the impression that such remedies are safe and effective.
However, many of these treatments fall under the category of “dietary supplements,” which means that the law doesn’t require them to go through rigorous testing to prove safety and efficacy. For dietary supplements such as tinctures and herbs, the government simply states that the ingredients must be listed on the label (the Dietary Supplement Health and Education Act of 1994). Supplements may contain varying amounts depending on the brand, may contain ingredients that are harmful to users, and may reduce the effectiveness of proven medicines.
Despite their increasing use, most alternative therapies, in the US and abroad, have not been rigorously evaluated for their safety and efficacy. One could, in fact, argue that there is no such thing as “alternative medicine.” There is only scientifically proven, evidence-based medicine supported by solid data. Whether a therapeutic practice is unconventional or not, widely known, or practiced under the auspices of religious tradition shouldn’t matter. What truly matters is whether clinician scientists and investigators have used the tools available to us to evaluate the risks, the benefits and whether an approach is more, less or similarly effective to current evidence-based practices. In 1998, the NIH formed the National Center for Complementary and Alternative Medicine (NCCAM) to “explore complementary and alternative healing practices in the context of rigorous science.” Ten plus years and 2.5 billion dollars later have found no cures from alternative medicine.
When considering alternative therapies, consider the case of Daniel Hauser, the 13-year-old boy from Sleepy Eye, Minnesota whose mother favored the unproven healing practices of the Nemenah Band of Native Americans. Daniel was diagnosed with Hodgkin’s lymphoma at a stage consistent with an 85% chance of survival with chemotherapy and radiation, yet almost certain death without treatment. After one round of chemotherapy, Daniel’s tumors shrank considerably. But, after seeing how tough chemotherapy can be, Daniel’s mother fled Minnesota with Daniel. Although Daniel is back at home and he and his parents have agreed to at least five rounds of chemotherapy, a CT scan shows that the tumor in his chest is larger than when it was originally diagnosed, is pressing against his chest wall, and is causing Daniel to have trouble breathing.