GRN-1201 is a cancer vaccine aimed at boosting the body’s own immune response against tumor-associated antigens (TAAs) and activating tumor-specific T-cells. These infiltrate the tumor site and kill cancer cells. An antigen is a substance not normally found in the body which triggers an immune response.

The vaccine, developed by Japan’s BrightPath Biotherapeutics, is being studied to treat melanoma and non-small cell lung cancer (NSCLC).

How GRN-1201 works

GRN-1201 targets four novel TAAs. It is composed of four human leukocyte antigen (HLA)-A2-restricted peptides. These peptides are derived from four specific and separate TAAs expressed by melanoma cells.

HLAs are proteins found on the surface of all cells. They help the immune system recognize which cells belong to the body and which do not. HLA is the human version of the major histocompatibility complex (MHC), a family of genes that occurs in many species. In humans, they consist of more than 200 genes located close together on chromosome 6. They are divided into three classes: HLA-1, HLA-2, and HLA-3.

HLA-A2 is found in about 50 percent of Americans and Europeans, and 40 percent of Japanese.

Once the HLA-A2-restricted melanoma-specific peptides vaccine is given, the melanoma-specific antigens in the vaccine activate the immune system to exert a cytotoxic (or cell-killing) T-lymphocyte (CTL) response against HLA-A2-positive melanoma cells.

GRN-1201 in clinical trials

A Phase 1 study (NCT02696356) is currently evaluating the effect of GRN-1201 in patients with resected melanoma. The treatment will be combined with granulocyte-macrophage colony stimulating factor (GM-CSF), a protein that helps stimulate the immune system and increase immune response against tumor cells.

The trial will study safety as measured by the number of side effects and determination of a safe dose. It is taking place at several U.S. locations but is no longer recruiting participants.

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