Eflornithine (Ornidyl (for other indications), Vaniqa (topical formulation))

Other Medications · Approved since 1990

US Off-label ES Hospital use Oral 5 Clinical Trials
Ornithine decarboxylase (ODC)

Description

Eflornithine is an irreversible inhibitor of ornithine decarboxylase (ODC), an enzyme crucial for polyamine biosynthesis and cell proliferation. In colorectal cancer, it functions as a chemopreventive agent rather than active treatment, specifically used to prevent adenomatous polyp formation and reduce colorectal cancer risk. Based on clinical trial data, eflornithine is typically combined with sulindac for enhanced chemopreventive efficacy in high-risk populations, including patients with familial adenomatous polyposis (FAP) and those with previous colon polyps.

Mechanism of Action

Eflornithine irreversibly binds to and inhibits ornithine decarboxylase, the rate-limiting enzyme in polyamine synthesis. This inhibition depletes cellular polyamines (putrescine, spermidine, and spermine), which are essential for DNA synthesis, cell division, and tumor growth, ultimately leading to reduced cell proliferation and adenoma formation.

Molecular Targets

Side Effects

Diarrhea Hearing loss Thrombocytopenia Leukopenia Alopecia Nausea Vomiting Anemia

Not all side effects are listed. Side effects vary by individual. Always consult your oncologist.

Clinical Trials

NCT01483144 Phase 3
Archived
Trial of Eflornithine Plus Sulindac in Patients With Familial Adenomatous Polyposis (FAP)
United States, Belgium, Canada, Germany, Netherlands, Spain, United Kingdom
NCT01349881 Phase 3
Active, not recruiting
S0820, Adenoma and Second Primary Prevention Trial
United States
NCT00983580 Phase 2
Archived
Acetylsalicylic Acid and Eflornithine in Treating Patients at High Risk for Colorectal Cancer
United States
NCT00033371 Phase 2
Archived
Celecoxib With or Without Eflornithine in Preventing Colorectal Cancer in Patients With Familial Adenomatous Polyposis
United States
NCT00118365 Phase 3
Archived
Eflornithine and Sulindac in Preventing Colorectal Cancer in Patients With Colon Polyps
United States