The gift of breakthroughs in pediatric brain tumors

This academic year, Children’s National Hospital received a $96 million investment from an anonymous donor family to transform rare childhood brain tumor research and care. This donation is one of the largest in the hospital’s history and will allow Children’s National to recruit more top talent and advance the most promising research for safer, more effective treatments. The investment will elevate standards of care to help children with rare brain tumors thrive for a lifetime. Brain tumors are the most common solid tumors affecting children and are often the ones with the poorest outcome. They are especially challenging in kids because their brains are still developing. The disease and current treatments can put them at risk for lifelong complications.

The new funding will propel the hospital’s longstanding efforts to drive research and new clinical trials. It will allow us to lead the way to harness the power of immunotherapy to better treat these tumors and explore the ability of ultrasound to safely get agents to the tumor in ways not possible previously. It expands the Rare Brain Tumor Initiative, which is already leading a new collaborative with hospitals in North America, South America and Europe to better understand and find novel treatments for these rare diseases. The investment will also be used to explore multiple ways to perform safer, more effective neurosurgery and develop techniques to assess the molecular underpinnings of the tumor. Children’s National will recruit leading scientists to advance biology-informed therapies that can be targeted for children across a spectrum of brain tumors. Under the leadership of internationally renowned clinical and translational researchers such as Roger Packer, M.D., and Catherine Bollard, M.D., M.B.Ch.B., what is one of the institution’s largest donations will become a major driver in research advancements for pediatric brain tumors.

This transformational investment will have a far-reaching impact on our ability to save and improve the lives of children with brain tumors. Funds will fuel collaborative breakthroughs across a range of scientific and psychosocial approaches.

The partnership will supercharge highly individualized and promising treatments for children with brain tumors. We will radically transform the research landscape with a focus on:

• Low intensity focused ultrasound (LIFU) – Advancing laboratory research and a clinical program designed to treat childhood brain tumors with LIFU therapy
• Cellular immunotherapy – Testing “first in human” neoantigen-specific T cell therapies and novel gene-engineered immune cell therapy products and accelerating their integration into standard of care
• Rare Brain Tumor Program – Propelling new clinical trials through the hospital’s national and global leadership in pediatrics with the creation of a new collaborative with hospitals in North America, South America and Europe to better understand and find novel treatments for these rare diseases
• Neurosurgery innovation – Exploring multiple ways to perform safer, more effective neurosurgery and developing new methods to enhance drug/agent delivery
• Precision medicine – Recruiting leading scientists to advance biology-informed therapies that can be targeted for children across a spectrum of brain tumors
• Good Manufacturing Practices (GMP) facility – Expanding our GMP, one of the first standalone FACT accredited facilities in the country that focuses on the manufacture of novel cell therapies at a children’s hospital, to translate new discoveries into clinical trials more rapidly
• Additional priorities including expansion of clinical research infrastructure and growth of bioinformatics, brain tumor repository and molecular diagnostics initiatives

Delivering world-class care to every child who needs it requires partnerships, and this donor family’s generosity is remarkable. Pairing their philanthropy with our brightest minds in research and care will spark the development of new and better ways to care for children with rare brain tumors. This investment will fundamentally change the treatment and long-term outcomes for children with brain cancer.

The $96 million investment will transform rare childhood brain tumor research and care at Children’s National Hospital. The investment will be used to recruit top talent, advance promising research, elevate standards of care and expand the hospital’s GMP facility to translate new discoveries into clinical trials. This partnership will lift up one of the nation’s top pediatric brain tumor programs and bring new breakthroughs in therapies to children with brain tumors.