This substance is a very important compound in the field of pharmaceutical research. Its most widely known name is Venetoclax. It is a groundbreaking anti-cancer drug jointly developed by AbbVie and Genentech.
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Summary of Core Information
· CAS Number: 885519-12-0
· Generic Name: Venetoclax
· Trade Name: Venclexta® (in the United States), Venclyxto® (in Europe)
· Chemical Name: 4-(4-{[2-(4-chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazine-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrazole-2-benzimidazol-5-yl oxy)benzoic acid
· Molecular Formula: C₄₅H₅₀ClN₇O₇S
· Molecular Weight: 868.44 g/mol
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What is it?
Venetoclax is an efficient and highly selective oral small molecule inhibitor. Its function is to specifically inhibit the BCL-2 protein.
· BCL-2 protein: Belongs to the Bcl-2 protein family and is an important "anti-apoptotic" protein. Its function is to prevent cells from undergoing programmed death.
· Mechanism of action: In many cancer cells (especially certain blood cancers), the BCL-2 protein is overexpressed, acting like a "guardian" to protect cancer cells from dying, allowing them to proliferate indefinitely. Venetoclax functions like a precise "key", able to bind to the active site of the BCL-2 protein and inhibit its function. This releases the signals that prompt cell apoptosis, ultimately leading to the self-destruction of cancer cells.
This "apoptosis-promoting" therapy targeting BCL-2 is a milestone in the field of cancer treatment.
2. Main Uses and Indications
Venetoclax is mainly used to treat certain types of hematological malignancies. It is typically employed for cases of recurrence/refractory conditions, or in combination with other drugs as the first-line treatment.
The approved main indications include:
· Chronic Lymphocytic Leukemia: For patients with CLL carrying the 17p deletion mutation, or for those with the same mutation but who have previously received at least one form of treatment.
· Small Lymphocytic Lymphoma: Also applicable to SLL patients in the aforementioned situations.
· Acute Myeloid Leukemia: Combined with demethylating drugs or low-dose cytarabine, used to treat adult AML patients who do not meet the criteria for intensive chemotherapy.
Furthermore, it has also been studied in numerous other clinical trials for blood cancers and solid tumors, demonstrating a broad range of potential applications.
3. Characteristics and Importance
1. First-in-class drug: Venetoclax is the world's first approved BCL-2 inhibitor, representing a significant breakthrough in the field of targeted therapy and apoptosis research.
2. Precise targeting: Its high selectivity enables it to precisely attack cancer cells, causing relatively less damage to normal cells.
3. Foundation of combination therapy: It has a synergistic effect with many other anti-cancer drugs and has become a core component of many combination treatment regimens.
4. Changed the treatment landscape: For some refractory leukemia and lymphoma cases, Venetoclax significantly improved the remission rate and survival period of patients, providing new hope for them.
4. Important Notes and Challenges
· Tumor lysis syndrome: This is the most serious and unique risk associated with Venetoclax. Due to the rapid onset of the drug, a large number of cancer cells are killed within a short period of time, and their contents are released into the bloodstream, potentially causing life-threatening conditions such as electrolyte imbalance and renal failure. Therefore, a "dose escalation" strategy is typically adopted in the initial stage of treatment, and close monitoring and preventive measures are required.
· Other side effects: Common side effects include bone marrow suppression, diarrhea, nausea, fatigue, and upper respiratory tract infections.
· Drug resistance: Like other targeted drugs, some patients may develop resistance after long-term use.
Summary
CAS 885519-12-0 (Venetoclax) is not merely a chemical substance; it is a groundbreaking anti-cancer drug. Through its unique "apoptosis-promoting" mechanism, it has brought revolutionary treatment options to many patients with blood cancers who previously had limited treatment options. Although there are risks such as TLS that require strict management, its outstanding efficacy makes it an indispensable and important weapon in modern oncology.
