FDA CBER Authorizes Direct Injection of DNG64-CAR-V Gene Therapy into CSF for 2nd Patient with Leptomeningeal Disease

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,

LOS ANGELES, CA, UNITED STATES, September 23, 2026 /EINPresswire.com/ — 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 CSF samples will be withdrawn from the Ommaya Reservoir before and after each injection to assess response to treatment.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. https://doi.org/10.3390/cancers17071125), with long term (10-18 years) survivors and no delayed adverse events reported (Al Shihabi et al., Mol Ther Oncol 201 doi: 10.1016/j.omto.2018.118; Liu et al., Clin Oncol 2021, http://clinicsinoncology.com/).

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

Erlinda Gordon
Aveni Foundation
+1 818-726-3278
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