Children's Neuroblastoma Cancer Foundation and Solving Kids' Cancer team up to help fund international clinical trial of targeted therapy
Children's Neuroblastoma Cancer Foundation and
Solving Kids' Cancer team up to help fund international clinical
trial of targeted therapy
Charities, academic and pharmaceutical researchers collaborate to fast-track a promising therapy for neuroblastoma
A new drug that may overcome resistance to treatment in high-risk pediatric neuroblastoma patients is heading to clinical trial. The Children's Neuroblastoma Cancer Foundation has proudly partnered with Solving Kids' Cancer to help fund the trial by collaboratively funding $135,000.
The clinical study, led byAraz Marachelian, MD, a pediatric oncologist at The Children's Hospital of Los Angeles (CHLA), is focusing on a small subset of neuroblastoma patients with mutations in the anaplastic lymphoma kinase (ALK) gene. Abnormal ALK changes drive about 15 percent of high-risk forms of neuroblastoma. The study will be conducted in 15 hospitals through the New Approaches to Neuroblastoma Therapy (NANT) consortium.
According to Dr. Yael Mosse, one of the study's key researchers, the drug, known as PF3922, has very broad potency against a range of ALK mutations. If this trial is successful, this drug will be prioritized for use in frontline therapy in children with ALK-driven neuroblastoma. The drug trial, slated to open at the end of May, represents an unprecedented collaboration between academic researchers, industry, and charities to rapidly advance a promising new agent to the clinic.
"The importance of this trial can't be overstated," said CNCF founder and President Patricia Tallungan. "New treatment paths are being opened up with targeted therapies, and we intend to use these innovations to our advantage in the fight against neuroblastoma."
Childhood cancer is the leading causing of death by disease in children aged 0-17 in North American and Europe. Neuroblastoma is a pediatric cancer that strikes mostly infants and young children. The aggressive cancer manifests as tumors in the head, neck, abdomen and pelvis. High-risk neuroblastoma has a 40 percent survival rate and far lower rate after relapse.
"We're supporting a clinical trial site in the UK and are hoping the trial can be expanded to continental Europe as well," said Donna Ludwinski, director of research programs at Solving Kids' Cancer. "We're committed to helping neuroblastoma families wherever they are." The hope is that this trial will result in rapid FDA and EMA approval so that this drug can be moved into frontline therapy in the U.S. and Europe in the next three years.
Just 4 percent of research funding by the American Cancer Society goes to study pediatric cancers, and all pediatric cancers combined receive just 4 percent of federal funding for research. Every donation helps fill the funding gaps so that children with neuroblastoma have a fighting chance. To donate to the fight, visit www.cncfhope.org.
The Children's Neuroblastoma Cancer Foundation is a nonprofit national health organization committed to finding a cure for neuroblastoma through research, education, awareness and advocacy. The premier source for neuroblastoma information and resources, CNCF initiatives educate the public about a disease dramatically lacking in awareness and funding. It serves
as an advocate for families, as well as a liaison between healthcare providers and families. Visit cncfhope.org.
About Solving Kids' Cancer
Solving Kids' Cancer helps accelerate new, next-generation treatments, including immunotherapy, cancer vaccines, and new drugs by applying an understanding of the entire childhood cancer research landscape to wisely invest in innovative projects. Visit solvingkidscancer.org.
You can help fund critical trials for neuroblastoma and provide educational programs for parents and caregivers by making a donation today.