PURPOSE: The study is a proof-of-principle trial evaluating toxicity, immune 
response, and clinical response in melanoma patients after combined therapy with 
temozolomide and the telomerase peptide vaccine GV1001. Our previous GV1001 
trials showed immune responses in approximately 60% of lung or pancreatic cancer 
patients.
EXPERIMENTAL DESIGN: Twenty-five subjects with advanced stage IV melanoma (M1B 
or M1C) received concomitant temozolomide and GV1001. Temozolomide was 
administered 200 mg/m² orally for 5 days every fourth week, and GV1001 as eight 
injections over 11 weeks. Immune response was evaluated by delayed type 
hypersensitivity, T-cell proliferation, and cytokine assays. The immunologic 
responders continued monthly vaccination.
RESULTS: The treatment was well tolerated. A GV1001-specific immune response was 
shown in 18 of 23 evaluated subjects (78%). Patients developing long-term T-cell 
memory survived more than those rapidly losing their responses. The immune 
response exhibited several characteristics of possible clinical significance 
including high IFNγ/IL-10 ratios, polyfunctional cytokine profiles, and 
recognition of naturally processed antigens. Survival compared favorably with 
matched controls from a benchmark meta-analysis (1 year: 44% vs. 24%, 2 years: 
16% vs. 6.6%). The clinical responses developed gradually over years, contrary 
to what is expected from chemotherapy. Five patients developed partial tumor 
regression and six more recorded stable disease. One patient has no remaining 
disease on fluorodeoxyglucose positron emission tomography scans after 5 years.
CONCLUSIONS: The immunologic response rate is considerable compared with 
previous GV1001 trials without concomitant chemotherapy, although low toxicity 
is retained. The results warrant further studies of GV1001/temozolomide 
treatment and support the general concept of combining cancer vaccination with 
chemotherapy.