Cyclophosphamide

Drug

Drug Profile

Cyclophosphamide is an alkylating chemotherapy and immunosuppressive medicine used in multiple cancers and severe autoimmune conditions. It is available orally and intravenously and is included on the WHO Essential Medicines list. It has long-standing clinical use in oncology and immune-mediated disease management.

Drug Class
Alkylating agent; nitrogen mustard
Approval Status
Approved for medical use in the United States in 1959; prescription-only
Mechanism of Action
Interferes with DNA duplication and RNA creation
Brand Names
  • Cytoxan
  • Endoxan
  • Neosar
Indications
  • Lymphoma
  • \Multiple myeloma\
  • Leukemia
  • \Ovarian cancer\
  • \Breast cancer\
  • \Small cell lung cancer\
  • Neuroblastoma
  • Sarcoma
  • \Nephrotic syndrome\
  • \ANCA-associated vasculitis\
  • \Post-organ transplant immune suppression\

Related News

Off-the-Shelf CAR-T Therapy CB-011 Shows High Response Rates in Multiple Myeloma; Broader CAR-T Advances Presented at 2026 Tandem Meetings

Updated data from the phase I CaMMouflage trial showed CB-011, the first allogeneic anti-BCMA CAR-T therapy with immune cloaking, achieved an approximately 92% overall response rate in relapsed/refractory multiple myeloma. The 2026 Tandem Meetings also highlighted advances in EB-103, KITE-753, and LV20.19 CAR-T constructs across lymphoma and CLL. Separately, NXC-201 reported a 95% complete response rate in AL amyloidosis.

WHO targets child-friendly cancer drug formulations amid treatment gap

WHO in January 2024 launched its first list of priority childhood cancer drug formulations, naming six medicines that still lack optimal child-friendly versions. The agency says about 400,000 children and adolescents develop cancer each year and is working to expand access to safe, effective paediatric formulations.

Novel GVHD Prevention Regimen Shows Strong Results Without Standard Immunosuppressants

Interim results from the ABC phase 1/2b trial show a calcineurin inhibitor-free GVHD prevention regimen combining posttransplant cyclophosphamide, bortezomib, and abatacept achieved low GVHD rates and favorable survival outcomes. The approach enables earlier introduction of posttransplant maintenance therapies while reducing toxicity associated with conventional immunosuppressants.

Related Clinical Trials

NCT ID Title Status Phase
NCT07614048

Intermediate-size IND for Treatment of Patients With Advanced Cancer Using T Cells Engineered to Express TCR Targeting Mutant KRAS

TEMPORARILY_NOT_AVAILABLE
NCT07613788

Rimegepant Plus Glofitamab and CD19 CAR-T Therapy in R/R LBCL

NOT_YET_RECRUITING PHASE2
NCT07613723

A Study to Test the Safety, Tolerability and Effect of ZI-MA4-1 for Patients With Locally Advanced or Metastatic Solid Malignancies

RECRUITING PHASE1
NCT07603557

Study of Zola-cel (BMS-986353), in Participants With Autoimmune Cytopenia (Breakfree-AiCE)

NOT_YET_RECRUITING PHASE2
NCT07598955

Target-Selected CAR-NK Cells (CD30, CD5, or Mesothelin) for Relapsed/Refractory B2 Thymoma or Thymic Carcinoma

RECRUITING PHASE1/PHASE2
NCT07594899

AI-Driven Treatment Strategy vs Pola-R-CHP in Untreated LBCL

NOT_YET_RECRUITING PHASE2
NCT07593482

Bendamustine Versus Fludarabine/Cyclophosphamide for Lymphodepletion in Chimeric Antigen Receptor T-cell Immunotherapy (CAR-T): a Randomized Trial.

NOT_YET_RECRUITING PHASE2
NCT07591649

Adapt NK for High Risk Myeloid Diseases as Bridge to Allo HSCT

RECRUITING PHASE1/PHASE2
NCT07589543

Dual-Target CAR-NK Cells in Recurrent or Refractory Epithelial Ovarian Cancer

RECRUITING PHASE1/PHASE2
NCT07583303

BSB-2002 After Cyclophosphamide and Fludarabine for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Patients With NPM1 Mutation

NOT_YET_RECRUITING PHASE1