# Ph1 Study of HSV G207 in Pediatric Malignant Cerebellar Tumors IND 16294 01/05/18.

> **NIH FDA R01** · UNIVERSITY OF TX MD ANDERSON CAN CTR · 2024 · $183,881

## Abstract

Project Summary/Abstract:
 Childhood brain cancer is the most common solid tumor in children affecting approximately 2,500 children
a year with an estimated 22,000 children living in the United States with a malignant brain tumor, which
establishes this as an orphan disease. Current therapies for malignant childhood brain tumors including surgery,
chemotherapy and radiation are very damaging to the developing brain of a child and can result in significant
long-term disabilities such as cognitive difficulties, neuroendocrine dysfunction, and neurosensory deficits in
survivors. Approximately 30-40% of children with malignant brain cancer do not survive, and high-grade tumors
that recur after current therapies are uniformly fatal. Therefore, novel therapies which target tumor cells while
sparing normal cells and stimulate an anti-tumor immune response are desperately needed.
 Oncolytic engineered herpes simplex virus (oHSV) therapy offers an inventive, targeted, less-toxic
approach for children with incurable brain tumors and may afford an improved margin of safety as an adjuvant
therapy for curable tumors allowing for lower doses and less toxicity from traditional therapies. HSV has been
successfully engineered to introduce mutations in the virus (e.g. γ134.5 neurovirulence gene) that prevent
infection in normal brain cells while maintaining the virus’ ability to kill cancer cells and stimulate an anti-tumor
immune response. UAB conducted 3 Phase I trials of oHSV G207, which has both copies of γ134.5 deleted and
an insertional deletion of the ribonucleotide reductase gene for added safety, given alone and with a single small
dose of radiation to enhance virus replication and an anti-tumor immune response, in adults with recurrent high-
grade glioma. These trials conclusively demonstrated safety of G207 inoculated intratumorally or in surrounding
brain tissue, and ≈half of patients had radiographic evidence of tumor response, including two long-term survivors
(>5.5 years). An active pediatric trial of G207 in supratentorial brain tumors has demonstrated safety of G207
alone with evidence of responses to G207 in 7 of 8 patients including a patient >21months post-G207 with an
ongoing response without any additional therapies. These trial data coupled with our preclinical data
demonstrating that aggressive pediatric brain tumors are highly sensitive to G207 and the lack of available
therapies for patients strongly support an oHSV trial for children with progressive malignant cerebellar tumors.
 We propose to conduct a Phase I clinical trial of G207 alone and combined with a single low dose of
radiation in children with recurrent cerebellar brain tumors. We hypothesize that G207 will be safe and tolerable
with evidence of efficacy in children with refractory cerebellar malignancies. Our primary goal is to determine
safety. Our secondary aims are to obtain preliminary information on the effectiveness of and immune response
to G207 and on tumor genotypic ...

## Key facts

- **NIH application ID:** 10469975
- **Project number:** 7R01FD006368-03
- **Recipient organization:** UNIVERSITY OF TX MD ANDERSON CAN CTR
- **Principal Investigator:** GREGORY K FRIEDMAN
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2024
- **Award amount:** $183,881
- **Award type:** 7
- **Project period:** 2019-09-15 → 2027-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10469975

## Citation

> US National Institutes of Health, RePORTER application 10469975, Ph1 Study of HSV G207 in Pediatric Malignant Cerebellar Tumors IND 16294 01/05/18. (7R01FD006368-03). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10469975. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*