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Updated: Aug 8, 2026

Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 19, 2013
Phase I and pharmacologic study of the tyrosine kinase inhibitor SU101 in patients with advanced solid tumors
S G Eckhardt1, J Rizzo, K R Sweeney
1Institute for Drug Development, Cancer Therapy and Research Center, San Antonio, TX 78229, USA. geckhardt@saci.org
Purpose:
To evaluate the clinical feasibility and pharmacologic behavior of the platelet-derived growth factor (PDGF) tyrosine kinase inhibitor SU101, administered on a prolonged, intermittent dosing schedule to patients with advanced solid malignancies.
Patients And Methods:
Twenty-six patients were treated with SU101 doses ranging from 15 to 443 mg/m(2) as a 24-hour continuous intravenous (IV) infusion weekly for 4 weeks, repeated every 6 weeks. Pharmacokinetic studies were performed to characterize the disposition of SU101 and its major active metabolite, SU0020. Immunohistochemical staining of PDGF-alpha and -beta receptors was performed on malignant tumor specimens obtained at diagnosis.
Results:
Twenty-six patients were treated with 52 courses (187 infusions) of SU101. The most common toxicities were mild to moderate nausea, vomiting, and fever. Two patients experienced one episode each of grade 3 neutropenia at the 333 and 443 mg/m(2) dose levels. Dose escalation of SU101 above 443 mg/m(2)/wk was precluded by the total volume of infusate required, 2.5 to 3.0 L. Individual plasma SU101 and SU0020 concentrations were described by a one-compartment model that incorporates both first-order formation and elimination of SU0020. SU101 was rapidly converted to SU0020, which exhibited a long elimination half-life averaging 19 +/- 12 days. At the 443 mg/m(2)/wk dose level, trough plasma SU0020 concentrations during weeks 2 and 4 ranged from 54 to 522 micromol/L. Immunohistochemical studies revealed PDGF-alpha and -beta receptor staining in the majority (15 of 19) of malignant neoplasms.
Conclusion:
SU101 was well tolerated as a 24-hour continuous IV infusion at doses of up to 443 mg/m(2)/wk for 4 consecutive weeks every 6 weeks. Although further dose escalation was precluded by infusate volume constraints, this SU101 dose schedule resulted in the achievement and maintenance of substantial plasma concentrations of the major metabolite, SU0020, for the entire treatment period.
Insights
The platelet-derived growth factor (PDGF) inhibitor SU101 was well tolerated in patients with advanced solid tumors. This dosing schedule achieved sustained plasma concentrations of its active metabolite, SU0020.
Area of Science:
- Oncology
- Pharmacology
- Molecular Biology
Background:
- Platelet-derived growth factor (PDGF) signaling is implicated in various cancers.
- Targeting PDGF tyrosine kinase is a potential therapeutic strategy for solid malignancies.
Purpose of the Study:
- To assess the clinical feasibility of SU101, a PDGF tyrosine kinase inhibitor.
- To characterize the pharmacologic behavior of SU101 on a prolonged, intermittent dosing schedule.
- To evaluate SU101 in patients with advanced solid tumors.
Main Methods:
- Twenty-six patients with advanced solid malignancies received SU101 via 24-hour continuous IV infusion weekly for 4 weeks, repeated every 6 weeks.
- Doses ranged from 15 to 443 mg/m(2)/wk.
- Pharmacokinetic studies analyzed SU101 and its metabolite SU0020.
- Immunohistochemistry assessed PDGF-alpha and -beta receptor expression in tumors.
Main Results:
- SU101 was administered over 52 courses (187 infusions) with manageable toxicities (nausea, vomiting, fever).
- Grade 3 neutropenia occurred in two patients at higher doses.
- SU101 rapidly converted to SU0020, with a long elimination half-life (avg. 19 days).
- Trough SU0020 concentrations were maintained, and PDGF receptors were expressed in most tumors.
Conclusions:
- SU101 demonstrated good tolerability at doses up to 443 mg/m(2)/wk using a 4-week continuous IV infusion schedule.
- Infusate volume limited further dose escalation.
- The dosing schedule successfully achieved and maintained significant plasma concentrations of the active metabolite SU0020 throughout treatment.
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