Novel Immunotherapy Targeting Brain Tumors Fast Tracked by US FDA

Diakonos Oncology Corporation today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the Company's dendritic cell vaccine, DOC1021.

The pivotal FDA designation can help propel Diakonos closer to delivering DOC1021 to glioblastoma multiforme (GBM) patients, of which only 7% survive more than five years.

GBM is the most common malignant brain tumor, with an annual incidence of 3.19 per 100,000 persons in the U.S.

About 61% of patients have the unmethylated subtype, demonstrating a median survival of 15 months compared to 21.7 months for methylated GBM patients when treated with the standard of care.

Developed at the Texas Medical Center in Houston, Texas, DOC1021 represents a novel immunotherapy approach to fighting cancer that harnesses the body's natural anti-viral immune response.

By mimicking a viral infection with the patient's cancer markers, DOC1021 leverages the body's innate ability to detect and eliminate infected cells.

"The FDA's decision acknowledges the potential of this new treatment approach for a very challenging disease," said Mike Wicks, Chief Executive Officer of Diakonos, in a press release on October 17, 2023.

At the core of DOC1021 is Diakonos' proprietary "double-loading" technique, which stimulates a previously undiscovered viral recognition and response pathway.

Using a patient's dendritic cells, a type of white blood cell that detects threats, the unique cancer markers are loaded both internally and externally into the immune cells, which would simultaneously occur in a viral infection.

Once the patient's individualized treatment is prepared, it is administered through three precise injections targeting the deep cervical lymph node chains.

This approach results in immune responses that directly target the central nervous system.

This revolutionary approach has shown remarkable outcomes, as the earliest patients exceeded survival expectations. DOC1021 also maintains an impressive safety profile.

As the treatment enters the final stages of the clinical trial, no serious adverse events have been linked to it. In addition, without genetic modification or artificial stimulation, DOC1021 further stands apart from other cancer immunotherapies.

The DOC1021 clinical trial is underway at the MD Anderson Cancer Center at Cooper University Health Care in New Jersey and the University of Texas Health Science Center in Texas. This clinical trial is expected to be completed in 2023.