A recent research study on high-risk acute lymphoblastic leukemia (ALL), funded in part by the St. Baldrick’s Foundation, has improved the cure rate and cut the relapse rate by using an old drug (methotrexate) in a new way!
“It’s just amazing that the funds we help raise go directly to childhood cancer studies like this,” said Melinda Baderman, mother to Spencer who was diagnosed with ALL when he was 6 and has shaved his head for the Foundation every year since. “It doesn’t go to a bunch of different places. It goes to helping cure our children of cancer. This is huge, it’s amazing.”
Methotrexate has been a large part of Spencer’s treatment plan. He is now out of treatment and is most excited about getting to play soccer again – something this 5th grader hasn’t been able to do since kindergarten.
ALL is one of the most common forms of childhood cancer, accounting for 25% of all cancers in children. Each year, there are about 2,900 new cases of children and adolescents diagnosed with ALL in the United States.
In the 1940s, all children diagnosed with ALL died within a few months of their diagnosis. The first effective chemotherapy medicine for childhood ALL was Aminopterin, a relative of Methotrexate, which was introduced in the late 1940’s. The subsequent use of chemotherapy treatments in sequential clinical research trials has led to a steady improvement in the outcome for children with ALL.
In 1999, a Children’s Oncology Group (COG) task force, led by Dr. Eric Larsen, medical director at Maine Children’s Cancer Program, was formed to find new treatments for high-risk ALL that would improve cure rates while also reducing side effects and chances of relapse.
“When we first started, we wished we had an extensive menu of new drugs to choose from,” said Dr. Larsen, “but it had been many years since any new drugs had come on the horizon for ALL treatment, so we were tasked to look at the drugs we had and use them in a better way.”
And they did exactly that. Methotrexate was a drug that had been around longer than any other for ALL and had become an important part of a child’s treatment. Dr. Larsen and his team decided to test the effects of their proposed alternative, high-dose methotrexate, compared to the then current standard of care, lower dose Capizzi methotrexate.
The study opened in January 2003 at 180 institutions in the U.S. In January 2011, initial results showed a clear benefit to high-dose methotrexate: improved cure rates, reduced relapse rates and surprisingly fewer side effects!
This exciting finding established high-dose methotrexate as the new standard of care for high-risk ALL patients and soon became the back-bone for all other ALL studies.
“One of the greatest lessons of this study is that we should consider using established drugs in new ways,” said Dr. Larsen. “This approach is one that can be applied to research for every type of childhood cancer.”
Read more about Spencer’s childhood cancer story.
This is one of St. Baldrick’s Childhood Cancer Research Outcomes. Learn about the rest.