Clinical Trials

MTX110 is a soluble form of panobinostat, which has been shown to be effective against brain cells in pre-clinical studies. For this study, MTX110 is injected directly into the tumor using small, flexible catheters inserted into the tumor.  The drug is slowly injected throughout the tumor over several hours.  This technique is called convection-enhanced delivery or "CED."  When the drug is given by CED, it allows the drug to bypass the blood brain barrier and remain in the tumor.  After each CED treatment with MTX110, the insertion catheters are removed and can be replaced every 4 to 8 weeks as needed for repeat treatments. Funding is provided by Cannonball Kids’ Cancer, The Cure Starts Now, and Midatech Pharma Inc.

Immunotherapy is emerging as new treatment for a variety of malignancies, including pediatric DIPG and HGG. Immunotherapy uses one’s own anti-tumor immune capabilities and has gained recent attention as a complementary approach to traditional therapies like radiation. One protein called programmed cell death-1 (PD-1) can be made by tumor cells as a mechanism to shut off the body's immune response to tumor. PD-1 works by binding to the body’s T-cells and giving them a message to turn off. Preclinical studies looking at blocking PD-1 in brain tumors has shown that PD-1 blockade increases survival and leads to long-term responses in mice. Additionally, PD-1 blockade combined with radiation appears to heighten the local immune response and increase the number of active T-cells surrounding the tumor. Hypofractionated radiation (higher doses given over fewer days) may heighten the effects by further increasing local inflammation and leading to greater numbers and activation of tumor-associated immune cells. In DIPG, hypofractionated radiation may also improve quality of life by shortening radiation periods and decreasing time spent in the hospital. These findings have led to interest that PD-1 blockade combined with radiation, either standard or hypofractionated, could be a new way to treat brain tumors.

REGN2810 is a novel, human antibody directed at the PD-1 receptor that prevents PD-1 activation by blocking interaction with its signal proteins, PD-L1 and –L2. PNOC013 combines REGN2810 with radiation as a new treatment strategy for children and young adults with newly diagnosed DIPG, newly diagnosed, and recurrent HGG, as well as children with recurrent solid tumors. The study is a multi-arm clinical trial that will randomize patients with DIPG or HGG to different radiation schedules (standard or hypofractionated) to be given in combination with REGN2810. REGN2810 will then continue as monotherapy once radiation is complete. PNOC013 will evaluate the safety of this treatment approach, as well as look at the impact on survival and disease responses in patients treated on this study.  Funding is provided by Regeneron Pharmaceuticals.

This is an open label trial to assess the safety and tolerability of the adult-tolerated dose of hyperpolarized pyruvate for metabolic imaging in children with brain tumors who do not require sedation for their MR imaging. Patients will receive a single MR imaging examination that includes the acquisition of hyperpolarized 13C metabolic data in combination with anatomic, diffusion, perfusion and lactate edited 1H spectroscopic imaging data. The data will be processed using custom designed software to estimate changes in levels of lactate/pyruvate and to relate them to abnormalities observed in the data from other MR modalities. Funding is provided by Kure-It. *Please note, this study has closed to accrual and is no longer accepting patients. 

Irinotecan is an important anti-cancer drug, but cannot distribute well into the brain or brainstem because of the blood brain barrier.  For this study, nanoliposomal irinotecan is injected directly into the tumor using small, flexible catheters inserted into the tumor.  The drug is slowly injected throughout the tumor over several hours.  This technique is called convection-enhanced delivery or "CED."  Nanoliposomal irinotecan means the irinotecan has been "packaged" into a nanoparticle that remains active in the tumor for weeks.  When the drug is given by CED, it allows the drug to bypass the blood brain barrier and remain in the tumor.  After each CED treatment with nanoliposomal irinotecan, the insertion catheters are removed and can be replaced every 4 to 8 weeks as needed for repeat treatments. Funding is provided by The V Foundation.

This is a study of an immunotherapy vaccine for pediatric glioma, including diffuse intrinsic pontine glioma (DIPG). Recent genetic studies have revealed that malignant gliomas in children often show recurrent missense mutations in H3F3A, which encodes the replication-independent histone 3 variant H3.3. This study will evaluate safety and immunological activity of a vaccine using a specific synthetic peptide for the H3.3K27M epitope in HLA-A2+ children with newly diagnosed DIPG or other gliomas who are positive for the H3.3K27M mutation. Funding is provided by The V Foundation and the Mithil Prasad Foundation.

Recent advances in gene expression and sequencing technologies allow oncologists now for the first time to use these technologies in real time for clinical decision making and to determine an individualized treatment plan based on each child’s tumor characteristics. This trial will test the feasibility using a precision based medicine approach in collaboration with the Translation Genomic Research Institute (TGEN) for children with newly diagnosed diffuse intrinsic pontine glioma (DIPG). Based on the molecular characteristics of each child’s tumor a specialized tumor board will recommend a treatment plan based on the results of the molecular profiling. All current FDA approved drugs will be considered for the treatment plan. This will be the first trial in the US to test such an approach for children with DIPG. Funding provided by the Pediatric Brain Tumor Foundation and the V Foundation. *Please note, this study has closed to accrual and is no longer accepting patients.