ICT-121 is an investigational immunotherapy vaccine being developed by ImmunoCellular Therapeutics to treat patients with recurrent glioblastoma, a difficult-to-treat brain cancer that often is resistant to radiation and chemotherapy.

ICT-121 is called a patient-specific, cell-based immunotherapy because it uses the patient’s own immune cells to fight off cancer cells.

How ICT-121 works

ICT-121 is made by collecting monocytes, a type of white blood cells, from a patient’s blood sample. These monocytes are then grown in the laboratory to mature into dendritic cells, master immune cells that activate and coordinate immune responses.

As the dendritic cells mature they are “educated” to recognize CD133, a protein that is overproduced by many types of cancers, including glioblastoma. The mature (educated) dendritic cells are then returned to the patient’s body by being injected into the skin. Once in the bloodstream, the educated dendritic cells recruit other immune cells and “teach” them to attack and destroy cells that express the CD-133 protein (CD-133-positive cells).

Because the activated, educated immune cells naturally multiply in the body, the vaccine has the potential to have a long-lasting effect.

ICT-121 in clinical trials

A multicenter, open-label Phase 1 trial (NCT02049489) to test ICT-121 in adults with recurrent glioblastoma is currently underway. During the trial, which began in December 2013, 20 patients receive weekly injections of ICT-121 for four weeks, then continue to receive injections once every two months until disease progression (or the tumor keeps growing), death, or discontinuation for other reasons.

The main goal of the study is to find out how safe ICT-121 is in these patients by keeping track of adverse events and toxicities. The trial also is assessing how well the patients’ immune systems are responding to the vaccine and how treatment affects survival rates.

In June 2017, investigators reported positive preliminary data from the trial at the annual meeting of the American Society of Clinical Oncology (ASCO). The treatment was considered safe and well-tolerated as tests showed evidence of immune response to the vaccine, and eight of the 20 patients who were treated with ICT-121 were still alive. This result is notable given that the mean overall survival rate in this patient population is normally five to nine months.

The trial is ongoing at different sites in the U.S., but no longer recruiting participants. It is estimated to be completed by November 2018.

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