A closer look at clinical trials from people who are desperate to join them

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

IN

April 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.

“The process,” says Jennie, “was like climbing Mount Everest without any equipment. Every time we submitted our application, they would want something more. When we finished the treatment on mice, for example, they said, ‘Now we want it on more mice.’ Once we had done that, they said, ‘Now we want it on large animals.’ When we completed that, they said, ‘Now we want a primate study.’ By the way, a primate study means breeding a monkey in utero to have Canavan disease, waiting for that monkey to grow, and then treating that monkey’s Canavan and showing the results. It was a ton of time wasted, and time was not on our side. Not to mention it was tons of money. A process we thought would take six months and one million dollars ended up taking four years and six million dollars.”

With Canavan, every second counts. Every single day there’s more brain damage. The Landsmans were racing against the clock. By the time the bureaucratic tape cleared, would their children even be healthy enough to receive the treatment?

When they finally met with the entire review board of the FDA, they had already been on this hamster wheel for three years. In the meantime, Benny and Josh were losing skills and functioning they had once had. “I was told not to bring Benny and Josh into the government building, but I brought them anyway,” Jennie admits. “The review board always refers to patients by their patient number, but I wanted them to see that there was a person behind that number.

“At the meeting, the review board was stuck on silly components like which syringes to use — things that really didn’t have anything to do with the main treatment. I stood up with Benny and said, ‘I was told he has three to ten years to live and he’s going to turn three in a month, so please don’t tell me that figuring out these details is going to stop you from saving his life.’ ”

Jennie’s words resonated. By the time the meeting was over, the boys had approval to begin treatment.

Not everyone has to jump through bureaucratic hoops and hurdles like the Landsmans did before being accepted into a clinical trial. It’s usually a relatively easy process for the thousands of people who take part in clinical trials every year to test new, not-yet-proven treatments. Some participants join because they’re desperate for a new treatment to cure their illness; others hope to advance medical research. According to the clinical trials government website, there are over 500,000 clinical trials being conducted in all 50 states and 200 countries — with the number increasing every year.

Clinical trials create new discoveries in medicine that allow people to live longer, healthier lives, but there can be risks when trying a brand-new protocol — ranging from disappointment to life-threatening complications. How do patients decide whether the possible benefits are worth the risk?