Novel Neuroblastoma Vaccine Trial

New York, NY — (November 1, 2011) — The Andrew McDonough B+ Foundation, a Delaware-based nonprofit,and NYC-based charity Solving Kids’ Cancer (SKC) announce their joint support of a clinical trial to test a new cancer vaccine against neuroblastoma – a common type of solid tumor that occurs in young children. The Andrew McDonough B+ Foundation granted $145,000 (70%) of the trial cost to Solving Kids’ Cancer for project management, and the entire grant will be applied to the trial via milestone payments as patients are accrued. The principal investigator for this study is Dr. Kenneth Lucas, professor of pediatrics at Penn State Hershey Children`s Hospital and a pediatric oncologist and hematologist. The funding provides key support to further Dr. Lucas’ ongoing work to find new treatments for neuroblastoma and other deadly childhood cancers. Only modest gains have been made in the survival rate of neuroblastoma over the past few decades, survival after relapse remains near zero, and it accounts for 15 percent of all pediatric cancer deaths.”

The survival rates for some children`s cancers like leukemia have improved greatly, but other cancers, such as neuroblastoma, currently have a survival rate of less than 40%,” said Dr. Lucas. “We are grateful for this support which gives our team the opportunity to create better treatments for the deadliest childhood cancers.

“Vaccines for cancer treatment differ from other vaccines in that they treat active cancers or help to prevent recurrence. SKC has provided $62,060 for initial funding for this project that exemplifies its high-prioritystudies that are novel, first-in-children, have high potential for impact, and are less toxic than currenttreatments. The project is a small proof-of-concept study of 15 patients, designed to answer scientificquestions more rapidly than conventional clinical trials. Expanding the study to numerous cancer centersto treat more children will follow promising results. Cancer immunology is currently understudied in pediatrics but holds great promise.

The project builds on five years of pre-clinical research which identified three new immunological targets that are specific to this pediatric cancer.This new study uses a vaccine created by the patient’s own white blood cells — dendritic cells — whichare isolated, modified to recognize the cancer antigens, expanded, and given back to the patient inrepeated doses over a period of four months. A demethylating agent called decitabine, given before each vaccine dose, markedly increases the expression of the three immunologic targets on the cancer cells (NY-ESO-1, MAGE-A1, MAGE-A3).

This combination therapy provides an ideal immune response environment for antigen-specific cytotoxic T lymphocytes to kill all the cancer cells. Since opening this fall, the study has accrued three patients and the first child has already shown a positive response after treatment. These are very early observations, said Scott Kennedy, SKC’s Executive Director, but we believe this therapy holds great promise.The next two patients have been screened and approved to be on the study in the next six weeks. The study will also open at the Dana-Farber Cancer Center/Boston Children’s Hospital, and rapid accrual of new patients is expected.

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