Gemcitabine in Newly-Diagnosed Diffuse Intrinsic Pontine Glioma
Status:
Recruiting
Trial end date:
2022-12-01
Target enrollment:
Participant gender:
Summary
Diffuse Intrinsic Pontine Glioma (DIPG) is an aggressive childhood brain tumor that, despite
many past clinical trials, has never been shown to respond to chemotherapy. Radiation therapy
(RT) is effective in extending life but is not curative; median overall survival is 11
months. It is still unclear why the hundreds of clinical trials involving chemotherapy of
DIPG have failed to demonstrate any activity against the tumor. Given that many agents tried
in clinical trials cross the blood brain barrier (BBB), it is possible that there are factors
specific to DIPG and its location that prevent adequate drug penetration. Gemcitabine has
been selected for this study because there is strong evidence of DIPG cell line inhibition in
vitro and good BBB penetration. Furthermore, pediatric dosing and toxicity has been
established in prior studies of children with relapsed solid tumors and leukemia.
The primary aim of this study is to determine the presence of gemcitabine in childhood DIPG
tissue after systemic treatment with the drug. The secondary aim is to quantify the
intratumoral gemcitabine concentration after systemic treatment.
Participants in this study will be given a one time IV dose of gemcitabine prior to having
standard of care surgery. During surgery biopsies will be obtained for clinical and research
purposes along with a blood sample. Because patients will be undergoing this biopsy as part
of their standard of care therapy here at Children's Hospital Colorado, this is an optimal
time to obtain a tumor biopsy for this study. The biopsy will serve to see if the study drug
is penetrating the tumor. Patients will then enter a follow-up period for 30 days post
surgery.