ICT-107 is a cancer vaccine being developed by ImmunoCellular Therapeutics for the treatment of newly diagnosed glioblastoma multiforme (GBM), the most common and lethal type of brain cancer.

It is designed to activate a patient’s own immune system to target six different tumor-associated antigens, or proteins, found on glioblastoma cancer cells.

ICT-107 was designed an orphan drug by the U.S. Food and Drug Administration (FDA) in 2010 and by the European Medicines Agency (EMA) in 2014.

How ICT-107 works

ICT-107 consists of a patient’s own dendritic cells (DCs), specialized immune system cells, which are pulsed with six synthetic peptides found on glioblastoma tumor cells.

Such DCs that produce tumor-associated antigens, when administered back to the patient’s body, can stimulate the immune system — and particularly a type of immune cell called cytotoxic (cell-killing) T-cells — to attack the cancer cells.

The six tumor-associated antigens are absent in melanoma 2 (AIM-2), melanoma-associated antigen 1 (MAGE-1), tyrosinase-related protein 2 (TRP-2), glycoprotein 100 (gp100), epidermal growth factor receptor 2 (HER-2), and interleukin-13 receptor subunit alpha-2 (IL-13Ra2).

ICT-107 in clinical trials

A Phase 1 trial evaluated the safety and immunogenicity (ability to trigger an immune response) of ICT-107 in 21 patients. The vaccine was administered three times at two-week intervals. Results showed that treatment correlated with better survival in patients whose tumors were known to express at least three of the antigens contained within ICT-107.

A randomized, double-blind, placebo-controlled Phase 2 study (NCT01280552) evaluated the safety and efficacy of ICT-107 in newly diagnosed patients with GBM following surgery and chemoradiation. A total of 124 patients were treated at 25 trial sites across the U.S. The study did not show an overall survival benefit, but did show two to three months progression-free survival that was statistically significant compared with patients treated with DCs that were not pulsed with antigens.

A Phase 3 clinical trial (NCT02546102) was started to study this vaccine in newly diagnosed GBM patients following surgery and chemoradiotherapy. Preliminary analysis showed that four of the targeted antigens were associated with prolonged survival. The randomized, double-blind, placebo-controlled trial was aiming to enroll around 400 patients, but was suspended in June 2017 because the company is unable to secure sufficient financial resources to complete it.


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