Researchers led by professors Mark Saltzman, PhD, and Ranjit Bindra, MD, PhD, have developed a treatment for medulloblastoma, a deadly brain cancer affecting children.

The treatment involves specially designed drug-carrying nanoparticles administered directly into the cerebrospinal fluid (CSF).

Mice with medulloblastoma receiving this nanoparticle-based treatment lived significantly longer than those in the control group.

Medulloblastoma tends to spread rapidly along the leptomeninges in the central nervous system, making it challenging to target with traditional treatments.

The nanoparticles adhere to tumors and slowly release talazoparib, an FDA-approved DNA repair inhibitor that belongs to the PARP inhibitor class.

The nanoparticle treatment is injected intrathecally, between the leptomeninges protecting the CSF, allowing long-term retention of nanoparticles in the fluid space.

The method offers a potential solution for treating brain cancers by overcoming the blood-brain barrier, a natural defense system.

The researchers combined the nanoparticle treatment with an oral dose of the chemotherapy drug temozolomide, aiming for synergistic tumor cell killing with minimal systemic toxicity.

Mice receiving the nanoparticle-based treatment lived longer and showed less cancer spreading compared to those receiving traditional drug therapy or no treatment.

The next steps involve validating the approach in larger animal models and eventually conducting human testing, with plans to explore the treatment's effectiveness in other cancers that tend to spread to the brain.