FDA CBER Authorizes Direct Injection Of DNG64-CAR-V Gene Therapy Into CSF For 2Nd Patient With Leptomeningeal Disease

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The Cancer Center of Southern California is committed to conducting first in human clinical trials and developing drugs for cancer and neurodegenerative disorders

The Aveni Foundation mission is to expedite development of gene-targeted technologies for cancer and other unmet medical needs.

An innovative way of administering DNG64-CAR-V for treatment of brain tumors, leukemia, lymphoma and cancers that have spread to the brain and spinal cord,

This is an exciting medical milestone for neuro-oncology and patients fighting advanced central nervous system cancers and cancers that have spread to the brain.” - Sant P. Chawla, MDLOS ANGELES, CA, UNITED STATES, September 23, 2026 /EINPresswire / -- The Cancer Center of Southern California/Sarcoma Oncology Center and the Aveni Foundation are proud to announce FDA CBER's emergency use authorization for intraventricular injection of DNG64-CAR-V for the 2nd patient with brain cancer (Diffuse Midline Glioma ) and leptomeningeal disease (IND#33293). The process required submitting a Compassionate Use Request to the FDA Center for Biologic Evaluation and Research for“Right to Try” Intraventricular Injection of DNG64-CAR-V and the instillation of an Ommaya Reservoir for injection of DNG64-CAR-V directly into the cerebrospinal fluid (CSF). The treatment is given every week for about 4 months. CSF samples will be withdrawn from the Ommaya Reservoir before and after each injection to assess response to treatment.

According to Dr. Chawla:“This is an exciting medical milestone for neuro-oncology and patients fighting advanced central nervous system cancers and cancers that have spread to the brain, including leukemia, lymphoma, breast cancer, sarcoma and lung cancer. DNG64-CAR-V is the first tumor targeted gene therapy product to be authorized by FDA CBER for direct delivery into the CSF and a number of patients with leptomeningeal disease have already requested to be considered for this type of treatment. To serve this population more efficiently, a Phase I/II clinical trial for leptomeningeal disease is planned".

DNG64-CAR-V (DeltaRex-G) is a tumor targeted gene delivery system created by Drs. Erlinda M. Gordon and Frederick L. Hall at the USC Keck School of Medicine, Los Angeles CA. According to Dr. Gordon "DNG64-CAR-V can be administered intravenously, intratumor, intra-arterially and intrathecally to reach primary and metastatic sites of cancer. An off-the-shelf, injectable replication-incompetent chimeric (tumor-targeted) amphotropic retroviral vector encoding a Cyclin G1/CCNG1 inhibitor construct, DNG64-CAR-V not only kills cancer cells and its blood supply, but also kills growth-promoting, stroma-producing cancer-associated fibroblasts (CAFs) in the tumor microenvironment (TME). By reducing stroma which serves as an impermeable fortress for cancer cells, DNG64-CAR-V enables entry of chemo/immunotherapy drugs for enhanced drug concentration in the TME." Intravenously administered DNG64-CAR-V has been used successfully in phase I/II studies for sarcoma, pancreatic, and breast cancer, and in a phase II clinical trial for osteosarcoma that had failed standard chemotherapy (Chawla et al., 2025, Gene and Cell Therapy for Sarcomas: A Review. Cancers 2025, 17, 1125. ), with long term (10-18 years) survivors and no delayed adverse events reported (Al Shihabi et al., Mol Ther Oncol 201 doi: 1016/j.2018.118; Liu et al., Clin Oncol 2021, ).

DNG64-CAR-V is available in an expanded access program sponsored by the Aveni Foundation, a 501(c)(3 public charity (NCT04091295; ). For further information, please go to the following websites: , , or contact Dr. Gordon at... or.... To make a donation, please visit and click on the“donate” button for credit card donations. Wire transfer information may be provided upon request.

Erlinda Gordon
Aveni Foundation
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FDA CBER Authorizes Direct Injection of DNG64-CAR-V Gene Therapy into CSF for 2nd Patient with Leptomeningeal Disease News Provided By Aveni Foundation September 23, 2026, 10:00 GMT Share This Article Distribution channels: Business & Economy, Healthcare & Pharmaceuticals Industry, Human Rights, Insurance Industry, Science

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