RAD001

RAD001

RAD001, also known as everolimus, is an immunosuppressant drug used primarily in the treatment of various types of cancer and in the prevention of organ transplant rejection. It is a derivative of sirolimus (rapamycin) and functions as an mTOR (mammalian target of rapamycin) inhibitor.

Mechanism of Action

Everolimus works by inhibiting the mTOR pathway, which is crucial for cell growth, proliferation, and survival. The mTOR pathway is often dysregulated in cancer, leading to uncontrolled cell division and tumor growth. By inhibiting mTOR, everolimus can slow down or stop the growth of cancer cells.

Medical Uses

Everolimus is approved for use in several medical conditions, including:

• Renal Cell Carcinoma: It is used in the treatment of advanced kidney cancer.
• Breast Cancer: Specifically, hormone receptor-positive, HER2-negative breast cancer in postmenopausal women.
• Neuroendocrine Tumors: Used for certain types of neuroendocrine tumors of pancreatic origin.
• Tuberous Sclerosis Complex: For the treatment of subependymal giant cell astrocytoma (SEGA) and renal angiomyolipoma associated with tuberous sclerosis complex.
• Organ Transplantation: Used to prevent rejection in liver and kidney transplants.

Side Effects

Common side effects of everolimus include:

• Stomatitis (mouth ulcers)
• Infections
• Fatigue
• Diarrhea
• Rash
• Edema

Serious side effects can include:

• Non-infectious pneumonitis
• Hyperglycemia
• Hyperlipidemia
• Renal failure

Pharmacokinetics

Everolimus is administered orally and has a bioavailability of approximately 30%. It is metabolized in the liver by the cytochrome P450 3A4 enzyme and is excreted primarily in the feces.

Research and Development

Everolimus continues to be the subject of research for its potential use in other types of cancer and diseases. Ongoing clinical trials are exploring its efficacy in combination with other therapies.

Also see

• mTOR inhibitors
• Sirolimus
• Renal cell carcinoma
• Breast cancer
• Neuroendocrine tumor

RAD001
Trade names	Afinitor, Zortress
Synonyms	RAD001
Chemical nomenclature
IUPAC name	40-O-(2-hydroxyethyl)-rapamycin
Routes	Oral
Pregnancy category	C
Bioavailability	30%
Metabolism	Liver (CYP3A4)
Half-life	30 hours
Excretion	Feces (80%), urine (5%)
Legal status	Rx-only
Identifiers
CAS Number	159351-69-6
PubChem	6442177
DrugBank	DB01590
ChemSpider	4946845
UNII	9HW64Q8G6G
KEGG	D03210
ChEMBL	1201187