A Leap into the Lab  

How much of a medical gamble would you take to live a longer, healthier life? How far would you go to save your child’s life? A closer look at clinical trials from people who are desperate to join them

INApril of 2019, Jennie and Gary Landsman walked into a federal building to meet with the entire review board of the FDA — all 26 members. “I felt like a warrior walking into that room with my family and our small group of lawyers and scientists,” Jennie remembers. “I was literally fighting for my children’s lives.”

For three years, Jennie and Gary had been battling the FDA, trying to get them to approve an experimental treatment to save their sons, Benny and Josh, who have an extremely rare degenerative disease called Canavan that affects white matter in the brain. People diagnosed with Canavan disease suffer from diminishing motor and cognitive skills and have a life expectancy of ten years.

“Back in 2016, when we first submitted our application to the FDA for Compassionate Use, we thought we’d get a quick approval and begin treating Benny and Josh within six months,” Jennie shares. “Instead, thirty days after we submitted our application, we found out that the FDA was putting a stop on all gene therapy trials unless they were highly regulated. There had been contamination issues in another trial and the FDA wanted to investigate why and how this had happened. In the meantime, they put any other trial on hold unless it was highly regulated.”

The FDA was adamant: If the Landsmans wanted to treat their sons, they needed to run a full-blown pharmaceutical trial. Undeterred, Gary and Jenny got to work, even quitting their jobs so they could focus exclusively on the task. “There were many sleepless nights when we were working twenty-four seven to make this happen,” Jennie relates. “We did an immense amount of fundraising, working with the media, reaching out to large companies to get them to do matching campaigns. My father asked every senator and councilman to write letters to the FDA. We hired a lawyer who works on clinical trials to help us with the process, we had a team of scientists, and we had the entire Jewish community on our side.