Phase I and early phase II studies on human urinary colony stimulating factor

Area of Science:

• Hematology
• Clinical Pharmacology
• Biotechnology

Background:

• Leukocytopenia is a common side effect of chemotherapy and irradiation.
• Colony-stimulating factors (CSFs) are used to mitigate chemotherapy-induced neutropenia.
• Human urinary colony stimulating factor (CSFHU) is a novel therapeutic agent.

Purpose of the Study:

• To evaluate the safety and efficacy of CSFHU in humans.
• To determine the optimal dosage for treating leukocytopenia.

Main Methods:

• Phase I study: 6 healthy volunteers received CSFHU intravenously (2.5 x 10^5 to 10^6 units).
• Phase II study: 6 leukocytopenic patients received 7-day intravenous CSFHU injections (7 x 10^6 to 1.4 x 10^7 units).
• Safety assessments included monitoring blood pressure and adverse events. Efficacy was assessed by leukocyte and granulocyte counts.

Main Results:

• Phase I: CSFHU was well-tolerated with no severe adverse effects. Minor side effects included transient blood pressure decrease and mild infusion-related symptoms.
• Phase II: Doses of 1.3-1.4 x 10^7 units of CSFHU resulted in complete or partial recovery of leukocyte and granulocyte counts.
• Lower doses (7 x 10^6 units) did not show significant recovery of leukocyte numbers.

Conclusions:

• CSFHU demonstrates a favorable safety profile in healthy volunteers.
• Higher doses of CSFHU appear effective in treating leukocytopenia induced by anticancer therapies.
• Further large-scale Phase II studies are warranted to confirm efficacy in leukocytopenic patients.