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Clinical trials program at Baystate Health benefits both children and adults with diseases across the lifespan

December 18, 2015

“Research is an integral part of what we do at Baystate Health. We believe that a thriving clinical trials program for both adults and children is essential to being able to provide our patients with state-of-the art treatments. Our researchers are actively involved in cooperative clinical trial networks and industry-sponsored clinical trials for diseases across the lifespan, and these trials are available right here in western Massachusetts,” said Dr. Peter David Friedmann, MPH, Baystate Health’s newly-appointed Chief Research Officer.

Three-year-old Xavier Pereira of Ludlow is one of a number of children who participated in cancer clinical trials during the past year at Baystate Children’s Hospital – research which has turned children’s cancer from a virtually incurable disease 50 years ago to one with a combined 5-year survival rate of 80% today.

“Because pediatric cancer is so rare, no single hospital or health care institution has enough young patients to conduct their own research to compare the effectiveness of different treatments on childhood cancers,” said Dr. Matthew Richardson, a pediatric oncologist at Baystate Children’s Hospital. “For example, we had 14 patients enrolled in various pediatric oncology clinical trials in the past year.”

That’s where the Children’s Oncology Group (COG) plays an important role.

The Children’s Oncology Group is a National Cancer Institute supported clinical trials group and is the world’s largest organization devoted exclusively to childhood and adolescent cancer research. The COG unites more than 9,000 experts in childhood cancer at more than 200 leading children’s hospitals such as Baystate Children’s Hospital, universities, and cancer centers like the Baystate Regional Cancer Program, across North America, Australia, New Zealand, and Europe in the fight against childhood cancer.

“These large-scale clinical trials, in which children around the world are enrolled, are the only way doctors can determine if one treatment works better than another. These studies over the years have allowed us to cure many childhood cancers, not by trial and error, but through the scientific study in the way children respond to different treatment protocols for a particular cancer,” he added.

Xavier, who was diagnosed last year with acute lymphoblastic leukemia (ALL), was enrolled in a clinical study which looked at two standard medications – methotrexate and vincristine – in treating children with leukemia.

“The trial involves giving slightly different doses at different frequencies to see if the cure rate is the same as standard dosing and timing, but with fewer side effects,” said Dr. Richardson.

The Children’s Oncology Group has nearly 100 active clinical trials open at any given time. These trials include front-line treatment for many types of childhood cancers, studies aimed at determining the underlying biology of these diseases, and trials involving new and emerging treatments, supportive care, and survivorship. Their goal is to cure all children and adolescents with cancer, reduce the short and long-term complications of cancer treatments, and determine the causes and find ways to prevent childhood cancer.

Among the side effects children experience from chemotherapy are those similar to adults such as nausea and vomiting. Treatment can also result in a low blood count requiring blood transfusions, decreased immune function, and risk of damage to the liver and kidney. As children grow into adulthood, they may later experience infertility, cardiac dysfunction, and even a second, different cancer. Dr. Richardson explained that some chemotherapy works by damaging a cell’s DNA, and that if a healthy cell is damaged, then there is a rare chance that it could result in a cell with a cancerous mutation.

Dr. Richardson noted that most parents often don’t think to ask if there is a clinical trial available for their child.

“I think mostly because they are just so much in shock of their child’s cancer diagnosis that it is not their first question,” he said.

But, similar to Xavier’s parents, when others learn about the possibility of enrolling their child in a clinical trial, most are interested in participating for a variety of reasons, noted Dr. Richardson.

“Some parents enroll their children strictly for altruistic reasons. They realize it might not help their child, but others in the future. For some young patients, unlike in Xavier’s case, a clinical trial might make a new medication, which has not yet been formally approved for treatment, available to them. Still others appreciate that their child’s treatment is periodically reviewed by the Children’s Oncology Group during the trial,” said Richardson.

Xavier’s mom, Michelle, didn’t hesitate in saying “yes” to doctors at Baystate about enrolling him in the clinical trial.

“If it will help other children, then I’m okay with that,” she said.

Dr. Richardson said the results of the clinical trial that Xavier was enrolled in will not be known for several years.

“Treatment of ALL lasts two to three years, then researchers must watch for any increase or decrease in side effects for several years after treatment ends,” said the pediatric oncologist.

As for Xavier’s prognosis right now, there is good news in the new year  – he is celebrating just over one year of being in remission.

According to pediatric oncologist Dr. Joanna Luty – who serves as the principal investigator at Baystate Children’s Hospital for the Cooperative Children’s Oncology Group clinical studies – there are currently 15 pediatric cancer clinical trials open for enrollment at the hospital.

“These studies range from a trial investigating treatment for ependymoma, a rare brain tumor in children, to a study on the biology of transient myeloproliferative disorder, a type of leukemia found in newborns with trisomy 21, also known as Down syndrome. There are also trials open for the most common cancers of childhood, including ALL and neuroblastoma,” said Dr. Luty.

Baystate Children’s Hospital also has an additional 15 active clinical trials for children with other diseases than cancer. There are also some 60 active clinical trials for adults with a variety of medical conditions at Baystate Medical Center.

For more information about research and clinical trials at Baystate Health, click on Education and Research on the Baystate homepage at baystatehealth.org.