LIFE ACCORDING TO SAM explores the remarkable world of Sam Berns and his family. Now a high-school junior about to turn 17, Sam embraces his circumstances with admirable courage, showing wisdom beyond his years. “I didn’t put... More
LIFE ACCORDING TO SAM explores the remarkable world of Sam Berns and his family. Now a high-school junior about to turn 17, Sam embraces his circumstances with admirable courage, showing wisdom beyond his years. “I didn’t put myself in front of you to have you feel bad for me,” he says at the beginning of the film. “I put myself in front of you to let you know you don’t need to feel bad for me. I want you to know me. This is my life, and progeria is part of it. It’s not a major part of it, but it is part of it.” An excellent student, he excels at music, enjoys friendships and is an avid sports fan. “You are handed something, and what you do with it is what matters, and that’s what Sam is doing,” says his mother. “I’m impressed continually by him.” When Sam was diagnosed with progeria, Leslie was a resident intern. With the support of her husband, Scott, a pediatric ER doctor, and the rest of her family, she devoted herself and her career to studying the disease. In fewer than four years, they established The Progeria Research Foundation (PRF), headed by Leslie’s sister, Audrey Gordon, raising $1.25 million towards identifying the gene that causes it. Everyone’s body contains the protein progerin, which ages some aspects of the body, such as the cardiovascular system, but not all. Though they are mentally and emotionally the same age as their peers, kids with progeria possess an abnormal amount of progerin, so gaining a better understanding of how the disease works could lead to breakthroughs in treating heart disease and aging in the general population. After identifying the gene, PRF-funded researchers found the first potential treatment using experimental drugs called farnesyl transferase inhibitors (FTIs). Leslie then assembled a team of doctors, scientists and statisticians, and in 2007 launched the first progeria drug trial with the FTI lonafarnib, involving 28 children, including Sam, from 16 countries. The two-year trial brought participants to Boston three times a year for a battery of tests to gauge whether this experimental drug had any measurable effect. Normally, drug trials are conducted on hundreds or thousands of patients, with half receiving a placebo, or no medicine, and half receiving the drug. Due to the small sample size of her study, Leslie’s team made the crucial ethical decision to give all the children the drug.