Be aware that some trials may require children to visit the hospital more frequently than others.
Since she was diagnosed with acute lymphoblastic at age 5, Phoenix Bridegroom, now 9, has been in several . The first was a European study during her first battle with cancer, which required 2.5 years of treatment.
“We asked the typical question: Which one is going to save her?” says her mother Tammy Bridegroom. “They believed the new protocol [in the trial] would offer a better chance.”
Despite her involvement in the study, Phoenix relapsed soon after completing the treatment. She's since had a bone marrow transplant and is currently involved in another study.
Overall, leukemia survival rates have greatly improved the last 40 years, says Dr. E. Anders Kolb, director of the Nemours Center for Cancer and Blood Disorders in Wilmington, Delaware. These improvements are largely due to patients participating in clinical trials, he says.
“Cancer in children is a rare disease, but it’s still the second leading cause of death next to accidents, and the leading cause of death by disease in children in less than 15 years of age,” Kolb says.
For families with or another life-threatening medical condition, a clinical trial may provide hope for survival. U.S. News spoke with experts who weighed in on the pros, cons and what parents should know before enrolling their child in a clinical study. Con: There may be unforeseen side effects.“There’s a risk of potential side effects, all of which may or may not be known at the time of the trial,” says Dr. Robyn Dennis, a physician with Nationwide Children’s Hospital's hematology, oncology and bone marrow transplant team.
There is always a benefit versus risk associated with research, Kolb says. However, he adds that clinical trials go through a vigorous review process to make sure they're appropriate for a particular illness.
“[We would not] expose a child to a therapy we think is less superior to the standard care,” Kolb says.
Pro: Clinical studies offer potentially more effective treatments and perhaps a cure.
Some children with a life-threatening condition have a high risk for death or side effects from other treatments. In some cases, an experimental therapy may be their best option.
In those situations, "the chances of their child doing well with standard treatment are low enough that we think there’s an experimental therapy that may offer a better alternative,” Kolb says.
The Deno family participated in a clinical trial as part of their daughter’s . In 2010, Aubrey was 13 months old and diagnosed with an aggressive form of acute lymphoblastic leukemia.
After her second round of chemotherapy, she developed hepatic veno-occlusive disease, or VOD – when blood vessels that carry blood through the liver become swollen or blocked, causing the to fail. It’s often fatal.
“All the sudden, this animal we were fighting wasn’t just cancer,” Dustin Deno says.
The only hope was to try defibrotide, a medication not yet approved for children. It’s believed to work by preventing blood clots and swollen blood vessels.
In Aubrey’s case, it saved her life. She’s now 5 and has been in remission for four years.
Con: Some trials take years to complete.
It's important for parents to ask how long a study may take.
"I try to tell parents that while there may very well be benefit to their child right now, the one downside of the trial is they do take a long time to analyze. Some can take two, three or even four years to complete enrollment," Dennis says.
Pro: Trials often allow patients to receive treatments they would otherwise not have access to.
If your child has already gone through the traditional route of treatment, and his or her health hasn’t improved, specialized treatments available through may be an option.
“It’s a consideration for a parent who might be looking for a treatment more novel than they’ve already tried," Dennis says.
Josh Clark was only 1-day-old when he was sent to Riley Hospital for Children at Indiana University Health, where discovered a heart defect called Shone’s complex.
The surgeons explained to the Clarks that Josh was too small for a mechanical valve surgery, and that cadaver valves would wear out too quickly – requiring multiple surgeries. Instead, the Clarks opted for the then-investigational Contegra valve. Without the trial device, he may not have made it to his first birthday.
Now 11, Josh is doing well, his mother Laura Clark says. In the next year or so, Josh will need a new Contegra valve because he has begun to outgrow it. "Josh is nervous," she says, "but he knows they fixed his heart once, and he knows they'll do it again."