Indoximod-based chemo-immunotherapy for pediatric brain tumors: A first-in-children phase I trial.

ABSTRACT:

Background

Recurrent brain tumors are the leading cause of cancer death in children. Indoleamine 2,3-dioxygenase (IDO) is a targetable metabolic checkpoint that, in preclinical models, inhibits anti-tumor immunity following chemotherapy.

Methods

We conducted a phase I trial (NCT02502708) of the oral IDO-pathway inhibitor indoximod in children with recurrent brain tumors or newly diagnosed diffuse intrinsic pontine glioma (DIPG). Separate dose-finding arms were performed for indoximod in combination with oral temozolomide (200 mg/m2/day x 5 days in 28-day cycles), or with palliative conformal radiation. Blood samples were collected at baseline and monthly for single-cell RNA-sequencing with paired single-cell T cell receptor sequencing.

Results

Eighty-one patients were

SUBMITTER: Johnson TS 

PROVIDER: S-EPMC10836763 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature