HBO recently aired a documentary titled Life According to Sam. The film was an official selection at the 2013 Sundance film festival, and has also been making its rounds at other notable film festivals, including the AFI Docs Festival in Washington, D.C., the Nantucket and Martha’s Vineyard Film Festivals, the Woods Hole and Rhode Island International Film Festivals, among others. It has won numerous awards at these and other festivals.
Life According to Sam is a story about Sam Berns, age 13. But it is also a story about Sam’s physician parents, a clinical trial, other children like Sam, and the rare disease progeria. Progeria is a rare disease that causes accelerated premature aging. Children with progeria have an average life expectancy of around 13 years. There are believed to be between 200 to 250 children with progeria world wide. There is no cure for the disease.
Part of the film centers around Sam’s parents clinical trial for the disease. Children suffering from progeria and their parents come from all over the world come to participate in the trial. However, with so few children with progeria, they decide to give the active drug to all of the children. This means they do not use a placebo group in the testing. The doctors know that the “gold standard” of a trial is testing on patients, with half of the patients getting the active agent and half getting a placebo. However, they question how ethical it is to deny the active agent to those dying of the disease and with a population of people with the disease being so small. In the film, we see the study rejected by medical journals because the researchers broke from the standard by failing to use the placebo group.
Reviewing the U.S. Department of Health and Human Services page on Ethics in Clinical Research, there are seven main principles guiding the conduct of ethical research: (1) Social and clinical value, (2) Scientific validity, (3) Fair subject selection, (4) Favorable risk-benefit ratio, (5) Independent review, (6) Informed consent, and (7) Respect for potential and enrolled subjects. However, as noted by Life According to Sam, do the requirements for an ethical study change because the research is for a group of people affected with a disease like progeria. With only 200 to 250 cases worldwide, is it really ethical to deny those in the trial the active agent? Does the placebo effect become less important when the group is so small?
Further possible ethical issues arise because Sam’s mother, Dr. Leslie Gordon, is leading the clinical trial. Can a mother really provide the unbiased lead on a clinical trial? On the other hand, is it ethical to deny a doctor the opportunity to research a cure for a disease simply because her child suffers from that disease?
Sam turned 17 on October 23, outliving the average life expectancy for children with progeria by four years. His mother, Dr. Leslie Gordon, and father, Dr. Sam Berns, were “instrumental in identifying the gene defect that causes aging to accelerate.” With this in mind, it does appear that sometimes the strict ethical requirements like a placebo group and preventing a mother from leading the trail should be loosened based on particular circumstances.
Watch the trailer for Life According to Sam.
Learn more about the research involved here.