One year after his last treatment, a six-year-old boy with recurrent neuroblastoma is in complete remission for his high-risk metastatic cancer. Doctors reported this case study in the January 2013 issue of Pediatrics, the journal of the American Academy of Pediatrics, which was funded in part by a joint grant from the Andrew McDonough B+ Foundation, Pierce Phillips Charity and Solving Kids' Cancer.
Current treatments for high-risk neuroblastoma patients include chemotherapy, radiation therapy, surgery, stem cell transplant, and immunotherapy. Less than half of the children survive in spite of the intensive and toxic standard therapy. Long term survival after a relapse is less than 5%.
Previous clinical trials in adult solid tumors have successfully used cancer-specific targets (NY-ESO-1, MAGE-A1, and MAGE-A3) to kill cancer cells. Now, scientists at the University of Louisville have used these same targets for neuroblastoma by creating a vaccine that causes the body's own immune system to attack the tumor cells. Dendritic cells are immune cells collected from the patient and grown in cultures after they are exposed to specific antigens. The dendritic cells "teach" the patient's T-cells to seek out and kill the cancer cells after they are returned to the patient through a series of injections.
Cancer treatment vaccines differ from other vaccines in that they treat active cancers or help to prevent recurrence. The principal investigator for this study, Kenneth Lucas, M.D., is the division chief of Pediatric Hematology-Oncology and Stem Cell Transplantation at the University of Louisville Department of Pediatrics. The funding provided critical support to further Dr. Lucas' ongoing work to find new treatments for neuroblastoma and other deadly childhood cancers.
In the case study, one year after the patient's last vaccination, the tumors cells that were located in the boy's bone marrow disappeared and he now shows no evidence of disease.
The study includes children with sarcomas as well as neuroblastoma, and will be completed in 2013.
For patients with relapsed neuroblastoma, there are few promising treatment options in clinical trials. More effective and less toxic treatments are desperately needed.
"This research builds on five years of pre-clinical research, which identified three new immunological targets that are specific to this pediatric cancer," said Scott Kennedy, the Executive Director of Solving Kids' Cancer. "The case study highlights the potential therapeutic progress that can be made against neuroblastoma, and brings hope to patients and their families in finding a lasting cure."
Andrew McDonough B+ Foundation
The Andrew McDonough B+ Foundation honors the memory of Andrew McDonough, founded by Andrew's mom, dad and sister. The overall goal of The B+ Foundation is to "Do Good." Providing financial assistance to families of children with cancer, The B+ Foundation has awarded over $500,000 to hundreds of families from around the country in their time of greatest need. The B+ Foundation also sponsors research to find cures for childhood cancers and advancements in chemotherapy. Information about the Foundation and its activities can be found at BePositive.org.
Pierce Phillips Charity
The Pierce Phillips Charity was founded in loving memory of our son, Pierce Alexander in June 2011. Our mission is devoted to the eradication of pediatric cancer through funding research and to improving the lives of the children and their families who are battling this disease. For more information, please visit Piercephillipscharity.org.
Solving Kids' Cancer
Created by two fathers who lost children to pediatric cancer, Solving Kids' Cancer is committed to significantly improving survivorship of the deadliest childhood cancers. 100% of all donations are used to find, fund, and manage clinical trials and scientific programs to rapidly develop more effective and less toxic treatments. Solving Kids' Cancer is a 501(c)(3) public charity. To learn more, please visit SolvingKidsCancer.org.