Drug intelligence / Profile preview

isotretinoin + temozolomide + thalidomide

Development stage
Unknown
Lead developer
Roche
Modality
Small Molecules
Administration
Oral
01

Overview

This is a combination therapy consisting of three small molecule drugs—**isotretinoin**, **temozolomide**, and **thalidomide**. Each component has distinct mechanisms of action: - **Isotretinoin** is a retinoid, primarily used for severe acne but also investigated for its ability to induce differentiation and apoptosis in certain cancer cells. - **Temozolomide** is an oral alkylating agent used mainly in the treatment of glioblastoma multiforme and other brain tumors; it works by methylating DNA, leading to tumor cell death. - **Thalidomide** is an immunomodulatory and anti-angiogenic agent approved for multiple myeloma and erythema nodosum leprosum; it modulates cytokine production (notably TNF-alpha), inhibits angiogenesis, and induces selective degradation of transcription factors involved in cancer cell survival. The combination has been explored in clinical research settings, particularly for refractory or high-risk cancers such as neuroblastoma or gliomas. The rationale behind this regimen is to combine cytotoxic effects (temozolomide), immune modulation/anti-angiogenesis (thalidomide), and differentiation/apoptosis induction (isotretinoin) to enhance antitumor efficacy.

02

Targets

RARA (Retinoic acid receptor alpha)CRBN (Cereblon)DNA

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