Immune Activating Gene Therapy for Glioblastoma Enters in Clinical Trial

Release Date: 19-Sep-2019

A new tumor-localized gene therapy has been tested for the first time in glioblastoma patients. The testing involves a two-part approach, which consists of receiving an injection of an immune-activator gene into the brain tumor site and after that a pill is swallowed which switches the gene. This approach resulted in the production of the activator-interleukin 12 (IL-12) and an infiltration of immune cells into tumor tissue. The results also hint that patient’s survival may be prolonged by the treatment.


According to the Fredrick Lang of the University of Texas MD Anderson Cancer Center in Houston, which is involved the project, all the recent evidence suggests that if you can really get the immune system to attack a tumor, then you have increased potential to cure that tumor, and this [work] is moving in that direction.


Glioblastoma is a type of cancer which affects the brain of the patient. It is an aggressive and deadly form of brain cancer with an average survival after diagnosis of just 6 to 9 months. The standard traditional treatment is not able to completely remove the chance of the reoccurrence of brain tumor. After completely removal of the tumor by surgery, Glioblastoma reoccurs within 7 month.


Some of the factors are responsible for making this cancer one of the most deadly cancer form. The major factor is presence of blood brain barrier. This barrier prevents the entry of most of the chemotherapeutic agents into the brain. So, it prevents the chemotherapeutics from reaching the tumor. The cells of Glioblastoma is rapidly increasing cells and replace the damaged cell rapidly. It also creates an immunosuppressive environment that limits the action of immune cells.


The trail involves the activity of Veledemix, which is able to cross the Blood Brain Barrier. About 50% of the total patient under the trail experienced a cytokine release syndrome as a result of the therapy and so ceased treatment. Based on the frequency and severity of this complication, together with the patients’ drug-taking compliance and survival data, the authors determined the optimum dose of veledimex to be 20 mg.

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