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Phase I trial of GBS-01 for advanced pancreatic cancer refractory to gemcitabine
Masafumi Ikeda1, Akihiro Sato2, Nobuo Mochizuki3
1Department of Hepatobiliary and Pancreatic Oncology, National Cancer Center Hospital East, Kashiwa, Japan.
Abstract:
GBS-01, an extract from the fruit of Arctium lappa L. is an orally administered drug rich in arctigenin, which has been reported to exert antitumor activity by attenuating the tolerance of cancer cells to glucose deprivation. We investigated the maximum tolerated dose of GBS-01 based on the frequency of the dose-limiting toxicities (DLTs) and pharmacokinetics in patients with advanced pancreatic cancer refractory to gemcitabine. GBS-01 was given orally at escalating doses from 3.0 g (containing 1.0 g burdock fruit extract) to 12.0 g q.d. A DLT was defined as a grade 4 hematological toxicity and grade 3 or 4 non-hematological toxicity appearing during the first 28 days of treatment. Fifteen patients (GBS-01 dose level 1 [3.0 g], three patients; dose level 2 [7.5 g], three patients; and dose level 3 [12.0 g], nine patients) were enrolled. None of the patients at any of the three dose levels showed any sign of DLTs. The main adverse events were increased serum γ-glutamyl transpeptidase, hyperglycemia, and increased serum total bilirubin; however, all the toxicities were mild. Of the 15 patients, 1 showed confirmed partial response and 4 patients had stable disease. The median progression-free and overall survival of the patients were 1.1 and 5.7 months, respectively. The pharmacokinetic study revealed a high bioavailability of arctigenin and rapid conjugation of the drug with glucuronic acid. The recommended dose of GBS-01 was 12.0 g q.d, and favorable clinical responses were obtained. This trial was registered at UMIN-CTR (http://www.umin.ac.jp/ctr/index-j.htm), identification number UMIN000005787.
Insights
GBS-01, a burdock fruit extract, shows good tolerability and bioavailability in advanced pancreatic cancer patients. The recommended dose is 12.0 g daily, with mild side effects and some positive clinical responses observed.
Area of Science:
- Oncology
- Pharmacology
- Natural Products
Background:
- Arctium lappa L. fruit extract (GBS-01) contains arctigenin, known for antitumor properties.
- Arctigenin may enhance antitumor activity by reducing cancer cell glucose deprivation tolerance.
- Pancreatic cancer often shows resistance to standard chemotherapy like gemcitabine.
Purpose of the Study:
- Determine the maximum tolerated dose (MTD) of GBS-01 in patients with advanced pancreatic cancer.
- Evaluate the pharmacokinetics (PK) of GBS-01 in this patient population.
- Assess preliminary clinical activity and safety of GBS-01.
Main Methods:
- An escalating oral dose study of GBS-01 (3.0 g to 12.0 g daily) was conducted in 15 patients.
- Dose-limiting toxicities (DLTs) were defined as specific grade 4 hematological or grade 3/4 non-hematological toxicities within 28 days.
- Pharmacokinetic analysis of arctigenin and its metabolites was performed.
Main Results:
- No dose-limiting toxicities (DLTs) were observed at any dose level (up to 12.0 g daily).
- Main adverse events included mild elevations in serum enzymes (γ-glutamyl transpeptidase, total bilirubin) and hyperglycemia.
- One partial response and stable disease in four patients were noted; median progression-free survival was 1.1 months, and overall survival was 5.7 months.
- High bioavailability of arctigenin and rapid glucuronidation were observed in pharmacokinetic studies.
Conclusions:
- The recommended dose of GBS-01 is 12.0 g daily due to favorable tolerability and pharmacokinetics.
- GBS-01 demonstrated potential clinical activity in gemcitabine-refractory advanced pancreatic cancer.
- Further investigation of GBS-01 in pancreatic cancer is warranted.
