When Families Participate in Research, Breakthroughs Happen
Published on in Breakthrough Report
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Published on in Breakthrough Report
To uncover breakthroughs in children’s health, Children’s Hospital of Philadelphia (CHOP) has two key pieces of the research puzzle:
The third critical piece of the equation, however, is having families willing to allow their children to participate in research.
“The No. 1 barrier to success of a research project is recruiting qualified participants,” says Chris Gantz, Manager, Recruitment Enhancement Core. “It can make or break a study.”
Gantz acts like a matchmaker. When researchers at CHOP are ready to recruit children and families for a research study, they contact him. He seeks families with children who meet the study’s criteria and connects them to the researchers.
For Grace Ebersberger, 18, coming to Children’s Hospital for a research study has two things going for it: “I like that it helps other people,” she says, “and they pay me.”
Ebersberger’s participation in research has had nothing to do with the numerous orthopaedic surgeries she’s had over the years. Instead, she has participated in a series of studies for the Center for Applied Genomics and the Division of Neurology that began by donating a blood sample when she was 8 years old, and have included several lengthy interviews, answering questions while in an MRI, and playing computer games to track her brain’s processing speed.
Families like Ebersberger’s are happy to help CHOP to fulfill its mission of discovering better ways to keep kids healthy, more effective treatments and, hopefully, cures.
While some research takes place exclusively in the lab with scientists huddled over microscopes, other studies — called clinical trials — need children to participate.
Some studies compare a new medication or intervention for a specific disease against the current standard of care to see which delivers the best outcomes. Other research focuses on uncovering the causes of disease, which can lead to targeted treatment or disease prevention.
Some researchers study how to prevent injuries or how physicians can deliver more effective care by using electronic health records. At a place as big as CHOP, there are hundreds of active studies going on all the time.
In addition to children who have a particular condition, many studies need well children to serve as “healthy control subjects,” so researchers can compare healthy children to patients with a specific disease. Similar to Ebersberger’s experience, children are asked to give blood or have an imaging test and their results are matched against patients of the same gender and age. Participants are usually reimbursed for travel expenses and parking; sometimes they receive a small stipend.
To make it easier for families to know which research studies might be a good fit, CHOP created the Clinical Research Finder, a searchable database of current studies. Families can also follow Children’s Hospital on social media to see posts that call for participants or learn about studies from their child’s doctors and nurses.
When Emily Herrera’s acute lymphoblastic leukemia (ALL) didn’t respond to traditional chemotherapy, she had genetic testing that revealed she had a high-risk type of ALL called Philadelphia-like. When asked by her oncologist, Sarah Tasian, MD, Herrera, now 19, agreed to join a clinical trial testing a new medication called ruxolitinib along with usual chemotherapy, a treatment researchers believe will work on her type of ALL.
Herrera is the first CHOP patient to take ruxolitinib as part of a larger, multi-institution study from the Children’s Oncology Group. “I wake up every morning happy to take my ‘genetic pill,’ and I’d encourage other patients to take part in clinical trials,” she says.
Her study is an example of the type of research that is now under the umbrella of the Roberts Collaborative for Genetics and Individualized Medicine. The collaborative will position CHOP at the forefront of pediatric genetics research by integrating the efforts of all clinical departments, divisions and centers involved in the care of pediatric patients and pursuit of genetic causes of disease.
The Roberts Center for Pediatric Research was named to recognize the Roberts family for its landmark $25 million gift that helped fund the collaborative.
Research at the Center for Autism Research (CAR) “has been a real benefit to us,” says Jennifer Nolte, mom to 9-year-old triplets Erik, Luke and Julia. Both Erik and Luke, who have autism spectrum disorder, have participated in CAR studies.
“They’ve had the opportunity to be in cutting-edge studies — all the latest advances like studying visual and motor acuity — and we end up with extra information we wouldn’t otherwise have,” she says. “It’s very reciprocal: We help the researchers and they help us.”
The Nolte family now comes to the Roberts Center when they’ve volunteered for a CAR study. They’re ushered to the Family Research Center on the fourth floor of the 21-story gleaming glass building that sits just off the east end of the South Street bridge. There, a concierge greets them.
The Family Research Center has waiting rooms, a lactation room, a family restroom and a pantry for families to use. Multiple evaluation rooms mean there is plenty of space for participants in studies for CAR, the Center for Injury Research and Prevention or other groups.
“I really encourage other parents to find out what is happening in research at CHOP to see if their children qualify for a study,” Nolte says. “It can be very affirming and exciting to be part of research.”
You can support CHOP by participating in research. Take a look at the clinical trials that are currently recruiting participants of all kinds, including healthy children. See our Clinical Trial Finder.