News Release

FDA orphan drug designation announced for childhood cancer vaccine

Business Announcement

Solving Kids' Cancer

The U.S. Food and Drug Administration (FDA) granted orphan drug designation for a neuroblastoma vaccine from MabVax Therapeutics, providing development incentive with market exclusivity of the novel treatment for children with this deadly childhood cancer.

The vaccine harnesses the power of a child's own immune system to recognize and destroy cancer cells that express the two antigens most commonly found on the surface of neuroblastoma cells—GD2 and GD3, potentially promoting long-term remissions. A phase I trial with the vaccine was recently completed at Memorial Sloan-Kettering Cancer Center with 15 patients and demonstrated very promising results; the results were published in the March 2014 issue of Clinical Cancer Research.

"The coveted FDA designation for this agent to treat neuroblastoma represents a win for the childhood cancer community. This will continue to encourage drug development for pediatric cancer, an arena which is often ignored by companies due to the small patient populations," said Scott Kennedy, Executive Director of Solving Kids' Cancer.

This past May, INBRACED--an international neuroblastoma research collaboration founded by the Neuroblastoma Children's Cancer Alliance (NCCA UK), Joining Against Cancer in Kids (J-A-C-K), and Solving Kids' Cancer--announced its first grant award of $400,000 to fund a phase I clinical trial using the MabVax vaccine. The clinical trial will enroll children with relapsed or refractory neuroblastoma at Memorial Sloan-Kettering Cancer Center in New York City, Bristol Children's Hospital in the UK, and the Hospital Sant Joan de Déu Barcelona, in Spain.

"It is vitally important to speed the exploration and evolution of potential therapies for the children and their families blighted by neuroblastoma," said Richard Brown, Chair at J-A-C-K. "Ensuring that children have access to the most novel and effective treatments will help change the future of this deadly disease."

Neuroblastoma is a cancer of the nerve cells and most often occurs in children 5 years and younger. The disease is primarily treated with chemotherapy, surgery, radiation, and stem cell transplant, but the cancer returns in half of all children within five years.

"For children with relapsed neuroblastoma, chemotherapy is highly toxic and not curative," said Woody Thompson, President of the Ronan Thompson Foundation. "We are honored to be a funding partner with INBRACED to support this novel and promising vaccine. This is the type of work that would make our son Ronan proud."



The International Neuroblastoma Research and Collaboration for Effective Delivery is a new collaboration between charities, researchers and clinicians with the shared aim of accelerating the development of new, more effective therapies for the childhood cancer neuroblastoma. To learn more, visit


Joining Against Cancer in Kids (J-A-C-K) provides advice and support for children and families wishing to undertake innovative or experimental clinical trials, treatments for neuroblastoma. For more information, visit

Neuroblastoma Children's Cancer Alliance UK

Established in 2006, the NCCA UK is a children's cancer charity working to help more children survive neuroblastoma through research, education and helping families with access to treatments. See more at

Ronan Thompson Foundation

The Ronan Thompson Foundation is a non-profit 501(c)(3) organization dedicated to finding a cure for neuroblastoma, a form of childhood cancer. We are currently fundraising to create a world-class neuroblastoma research and care center dedicated to funding both traditional and non-traditional treatments to reduce the number of children affected by this disease and increase survival rates.

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 about our work, please visit

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