Azacitidine Vidaza, Onureg

Used in colorectal cancer outside its approved indication. It appears in 6 clinical trials.

Intravenous, Subcutaneous DNA methyltransferase 1 (DNMT1)

United States Off-label

European Union EMA approved Since 2005

Spain Funded by SNS Used off-label in colorectal cancer

What it is

Azacitidine is a hypomethylating agent and DNA methyltransferase inhibitor that belongs to the class of nucleoside analogs. In colorectal cancer, it is used off-label in combination regimens to reverse epigenetic silencing of tumor suppressor genes and enhance immune recognition. The drug has shown particular promise when combined with immune checkpoint inhibitors like pembrolizumab and durvalumab in microsatellite stable (MSS) colorectal cancer patients who typically respond poorly to immunotherapy alone.

How it works

Azacitidine incorporates into DNA and RNA during replication, where it irreversibly binds to and depletes DNA methyltransferase enzymes. This leads to global DNA hypomethylation and reactivation of epigenetically silenced tumor suppressor genes, while also enhancing tumor antigen presentation and immune cell infiltration into the tumor microenvironment.

Molecular targets

DNA methyltransferase 1 (DNMT1)

Common side effects

MyelosuppressionNauseaVomitingDiarrheaFatigueInjection site reactionsConstipationPetechiaePyrexia

Not all are listed, and everyone experiences them differently. If you notice any, tell your care team.

Trials with this drug

Phase 1a/1b Study of Aplitabart (IGM-8444) Alone or in Combination in Participants with Relapsed, Refractory, or Newly Diagnosed Cancers NCT04553692 Terminated Phase 1 United States, Australia, France and 2 more Azacitidine Combined With Pembrolizumab and Epacadostat in Subjects With Advanced Solid Tumors (ECHO-206) NCT02959437 Archived Phase 1 United States, Spain, United Kingdom Study of Azacitidine and Durvalumab in Advanced Solid Tumors NCT02811497 Archived Phase 2 Canada A Phase 2 Study of Pembrolizumab (MK-3475) in Combination With Azacitidine in Subjects With Chemo-refractory Metastatic Colorectal Cancer NCT02260440 Archived Phase 2 United States Azacitidine and CAPOX in Metastatic Colorectal Cancer NCT01193517 Archived Phase 1 United States Azacitidine and Entinostat in Treating Patients With Metastatic Colorectal Cancer NCT01105377 Archived Phase 2 United States

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