Originally created as a cancer vaccine, GV1001 is a 16-amino-acid peptide that contains a sequence from the human enzyme telomerase reverse transcriptase (TERT). This enzyme is highly expressed in most cancers, and the intention behind immunizing with GV1001 is to trigger the immune system’s response to identify and eliminate cancer cells. Although GV1001 received approval in Korea for pancreatic cancer immunotherapy, it didn’t demonstrate efficacy in two global pancreatic cancer trials (Middleton et al., 2014; Clinicaltrials.gov).

Studies suggest that the peptide can infiltrate cells, engage with HSP70 and HSP90, and exhibit direct anti-cancer and anti-viral properties regardless of its impact on the immune system (Kim et al., 2018; Kim et al., 2016).

Beyond its role in cancer treatment, GV1001 has displayed anti-inflammatory and antioxidant capabilities in non-cancerous cells. When tested in rat neuronal stem cells, GV1001 effectively countered Aβ oligomer-induced toxicity and boosted cell survival when exposed to oxidative stress (Park et al., 2014; Park et al., 2016). Researchers attribute these effects to GV1001’s ability to mimic TERT’s antioxidative, anti-apoptotic, and pro-survival functions.

Moreover, positive outcomes have been observed in animal models concerning hearing loss, macular degeneration, and stroke (Kim et al., 2018; Kim et al., 2018; Lee et al., 2020; Kwon et al., 2021).