What Can I Do?

The case for guinea pigs.

Jan 20, 2025

I’ve approached many patients over the years about participating in a clinical trial. Not every patient, however, is a candidate. I remember an old farmer who had a run-of-the-mill intermediate risk prostate cancer. His sister worked as a statistician in the 1980s and he told me about the important work she did in cancer research. “You can experiment on me all you want, doc,” he said earnestly.

When I assured him that he would not be on a trial and would be receiving standard treatment, he replied “I don’t even care. Just use me to learn something that can help someone else.”

I tried again to explain that we don’t just experiment on patients and that the current treatment was so successful there wasn’t really anything we needed to learn from his case. He just smiled and insisted that he wanted to help the next patient. I finally gave up and agreed that I personally would learn something from him.

Prior to the use of clinical trials, random experimentation was the most common (and therefore largely unsuccessful) manner by which medical discoveries were made. A person (who didn’t even have to be a trained physician) just thought of something and tested it on people with the BELIEF that it worked.

Notable examples of this method include:

Henry Freeman performing a lobotomy. Photo: BBC.

Prefrontal lobotomy to cure “agitation and depression” performed by Dr. Henry Freeman. President John F. Kenndey’s sister, Rosemary, underwent this procedure as a teenager and was reduced to the mental state of a toddler. She spent the rest of her life institutionalized in Wisconsin.
Forced sterilization of prisoners in the United States from 1907 to the 1960’s. Motivated by the popular belief that eugenics (the promotion of “superior” people and inhibition of growth of “inferior” people) would eliminate crime, over 60,000 people underwent vasectomies or tubal ligation without their consent. Judges today still accept sterilization promises in exchange for reduced sentences.

Dr. John H Kellogg. Photo source: Wikipedia.

John Kellogg was a physician who believed that masturbation was the source of many of society’s ills including back pain, bed-wetting, weak posture, hysteria in women and insomnia to name a few. At his sanatorium in Grand Rapids, Michigan he prescribed a diet of bland foods including his eponymous Corn Flakes to reduce the urge to masturbate.

Obviously, these treatments all fell out of favor as people realized that they didn’t work.

Well-designed clinical trials, on the other hand, must be based on solid science and pre-clinical data obtained in animal models.

(Guinea pigs are not often used in cancer research. Mice are far and away the most common model due to their ready availability, low cost and short lifespan.)

After mice models indicate a promising treatment, the next step is a Phase I trial or pilot study. Here the intervention or medication is tested in human volunteers to see if it is feasible. What are the side effects for example, or what is the maximum tolerated dose. In the case of a behavioral intervention like telehealth counseling, for example, will people be able to use a virtual platform or show up for exercise classes. Phase II trials follow and then Phase III trials where the intervention is tested against the current standard.

Other trials are registry trials where researchers are trying to get a sense of what happens in a large group of people. The Women’s Health Initiative and the Framingham study are two great examples of these types of studies. In each, the researchers followed large populations of people to learn more about heart disease in women.

The most robust clinical trial mechanism in cancer began in the 1970’s and tested different methods of treating childhood leukemia. As a result, the survival rate for children with leukemia has risen from 0% to 80% in the past 50 years.

Other clinical trial successes include treatment and prevention of HIV, development of the HPV vaccine leading to decreased cervical cancer cases and of course, the rapid implementation of the COVID-19 vaccine. Each of these successes would not have been possible without volunteers signing up to be “guinea pigs.”

Today, fewer than 1 in 20 patients with cancer participate in clinical trials. This is odd because 70% of people say they are inclined or very willing to participate in clinical trials.

In a recent blog, NIH director and cancer survivor Dr. Monica Bertagnolli highlighted NIH’s efforts to use AI to boost participation in clinical trials for all diseases including cancer. “TrialGPT” uses large language models to match patients to thousands of possible clinical trials which I think sounds awesome. I had to do it the old-fashioned way – remembering the last trial a colleague talked to me about and calling the research nurse.

It’s easy to feel helpless when you look around the world. What can I, just a single person, do to help?

May I suggest becoming a human guinea pig?

Listed below are a few trials related to cancer. I promise there is something out there for everyone. Yes, even you.

Men in the UK: watch for a letter from your GP to join the largest prostate cancer screening trial ever. The $20 billion TRANSFORM trial will start enrolling patients in early 2025 and provide vital information on how we can detect aggressive prostate cancer earlier.
US citizens without cancer age 30-70: Join the CONNECT for Cancer Prevention study. Sponsored by the NCI, participants answer questions about behavior and health patterns to help researchers understand the causes of cancer and how to prevent it.
In the FORTE Colorectal Cancer Prevention Trial, patients who have had a recent colonoscopy that showed 1-2 precancerous polyps are randomly assigned to two groups: one that has a colonoscopy in 5 years and the other to have a colonoscopy in 10 years. This trial seeks to determine which surveillance timing is better.
Patients who had surgery as part of their treatment for head and neck cancer can enroll in the BRIGHT study which is testing different interventions for coping with body image changes and mental health concerns. Treatment must be completed within one year of enrollment.
For the handful of Cancer Culture subscribers in the Netherlands, researchers are looking how to support cancer survivors returning to work.
The list goes on…