FDA grants orphan drug designation for neuroblastoma vaccine

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 D-u 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 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."

Source: Solving Kids' Cancer