Last reviewed 2026-04-23 · How we verify

Alkylating-based chemotherapy

Alkylating agent that interferes with DNA replication

Alkylating agent that interferes with DNA replication Used for Various cancers.

At a glance

Mechanism of action

Alkylating agents work by adding an alkyl group to the DNA molecule, which interferes with DNA replication and transcription, ultimately leading to cell death.

Approved indications

• Various cancers

Common side effects

• Myelosuppression
• Nausea and vomiting
• Hair loss

Key clinical trials

• Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease (PHASE1, PHASE2)
• A Study to Learn About the Study Medicine PF-07985045 When Given Alone or With Other Anti-cancer Therapies in People With Advanced Solid Tumors That Have a Change in a Gene. (PHASE1)
• A Study to Learn About the Study Medicine PF-07934040 When Given Alone or With Other Anti-cancer Therapies in People With Advanced Solid Tumors That Have a Genetic Mutation. (PHASE1)
• Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy (PHASE1, PHASE2)
• Neoadjuvant Carbo/Paclitaxel Followed by Doxorubicin/Cyclophosphamide in Breast Cancer (PHASE2)
• Tarlatamab vs Standard of Care Chemotherapy in Patients With Pre-treated Advanced, Pulmonary or Gastroenteropancreatic Poorly Differentiated Neuroendocrine Carcinomas (NECs) (PHASE3)
• N10: A Study of Reduced Chemotherapy and Monoclonal Antibody (mAb)-Based Therapy in Children With Neuroblastoma (PHASE2)
• 131I-Omburtamab, in Recurrent Medulloblastoma and Ependymoma (PHASE2)

Primary sources

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