Monday, January 19, 2026 1/19/2026

Chemo-radio immunotherapy for pediatric brain tumors

Award Number: R01CA229646
ORGANIZATION: NATIONAL CANCER INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/01/2019
PERIOD OF PERFORMANCE END DATE: 08/31/2025

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Chemo-radio immunotherapy for pediatric brain tumors - Brain tumors are the most common solid tumor of childhood. Some can be cured, but for the 30-35% of pediatric patients who recur following front-line therapy – and for every child with diffuse intrinsic pontine glioma (DIPG) – there is essentially no chance of cure with standard treatment. Thus, more effective therapies are urgently needed. The scientific premise underlying the current proposal is that conventional radiation and chemotherapy release large amounts of antigen from dying tumor cells, but that this normally cannot trigger a useful immune response because immunogenic antigen-presentation is suppressed by tumor-induced mechanisms such as the indoleamine 2,3-dioxygenase (IDO) pathway, a natural mechanism that profoundly inhibits immune response to apoptotic cells. The proposal hypothesizes that adding immunologic therapy using indoximod, an oral inhibitor of the IDO pathway, in combination with radiation and chemotherapy, will allow prolonged survival in these otherwise refractory patients. The applicants have recently completed a first-in- pediatrics Phase 1 study of the proposed indoximod-based chemo-radio-immunotherapy approach in 53 children with recurrent brain tumors. This proof-of-concept study shows a median Overall Survival of 17.2 months, which is markedly superior to historical comparator studies, with low toxicity. Aim 1 will conduct a Phase 2, stratified-design trial of indoximod immunotherapy in combination with temozolomide (TMZ) chemotherapy, with or without the addition of novel Low-Dose Partial-field (LDPF) radiation, in 91 pediatric patients with recurrent ependymoma, medulloblastoma and GBM. Outcome measures will be 8-month Progression-Free Survival (PFS), and 18-month Overall Survival (OS) and Time to Regimen Failure (TTRF), as compared to PFS and OS from historical controls. Aim 2 will conduct a Phase 2 single-arm trial of 30 children with newly-diagnosed diffuse intrinsic pontine glioma (DIPG), to test the hypothesis that front-line indoximod added to standard conformal radiotherapy, followed by maintenance chemo-immunotherapy with indoximod + TMZ, will result in improved Overall Survival (OS), compared to well-documented historical controls. Aim 3 will use mechanistic predictions from novel preclinical models to identify innovative, hypothesis-driven immune biomarkers, combined with cell-intrinsic genomic and epigenomic features of the patients' original tumors, to ask whether these allow prognostic risk stratification of patient outcomes in Aim 1 and Aim 2. A successful outcome from the proposed clinical trials has the potential to fundamentally change the approach to treating children with recurrent or refractory brain tumors. It would also have major implications for incorporating immunologic therapy directly into the standard treatment for children with high-risk brain tumors, at the time of front-line treatment, when there is the real possibility of long-term cure.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = $0 )
2026	2023	AUGUSTA UNIVERSITY RESEARCH INSTITUTE, INC	AUGUSTA UNIVERSITY 1120 15TH STREET	AUGUSTA	GA	30912	RICHMOND	USA	Cancer Treatment Research	000	5	1/9/2026	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2023 ( Subtotal = $570,066 )
2023	2023	AUGUSTA UNIVERSITY RESEARCH INSTITUTE, INC.	1120 15TH ST STE CJ3301	AUGUSTA	GA	30912	RICHMOND	USA	Cancer Treatment Research	000	5	7/31/2023	NON-COMPETING CONTINUATION	$570,066
														Subtotal = $570,066

Issue Date FY: 2022 ( Subtotal = $570,066 )
2022	2022	AUGUSTA UNIVERSITY RESEARCH INSTITUTE, INC.	1120 15TH ST STE CJ3301	AUGUSTA	GA	30912	RICHMOND	USA	Cancer Treatment Research	000	4	8/26/2022	NON-COMPETING CONTINUATION	$570,066
														Subtotal = $570,066

Issue Date FY: 2021 ( Subtotal = $581,700 )
2021	2021	AUGUSTA UNIVERSITY RESEARCH INSTITUTE, INC.	1120 15TH ST STE CJ3301	AUGUSTA	GA	30912	RICHMOND	USA	Cancer Treatment Research	000	3	8/27/2021	NON-COMPETING CONTINUATION	$581,700
														Subtotal = $581,700

Issue Date FY: 2020 ( Subtotal = $581,700 )
2020	2020	AUGUSTA UNIVERSITY RESEARCH INSTITUTE, INC.	1120 15TH ST STE CJ3301	AUGUSTA	GA	30912	RICHMOND	USA	Cancer Treatment Research	000	2	8/19/2020	NON-COMPETING CONTINUATION	$581,700
														Subtotal = $581,700

Issue Date FY: 2019 ( Subtotal = $596,748 )
2019	2019	AUGUSTA UNIVERSITY RESEARCH INSTITUTE, INC.	1120 15TH ST STE CJ3301	AUGUSTA	GA	30912	RICHMOND	USA	Cancer Treatment Research	000	1	8/21/2019	NEW	$596,748
														Subtotal = $596,748

Grand Total All Awards = $2,900,280

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Chemo-radio immunotherapy for pediatric brain tumors

Award Number: R01CA229646

ORGANIZATION: NATIONAL CANCER INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 09/01/2019

PERIOD OF PERFORMANCE END DATE: 08/31/2025

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