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Published on: October 5, 2012
Venetoclax: Bcl-2 inhibition for the treatment of chronic lymphocytic leukemia
G Del Poeta1, M Postorino2, L Pupo2
1Hematology, Dipartimento di Biomedicina e Prevenzione, Universit à degli Studi di Roma Tor Vergata, Rome, Italy. g.delpoeta@tin.it.
Abstract:
Venetoclax (ABT-199) is a small-molecule selective oral inhibitor of the antiapoptotic protein Bcl-2 that promotes programmed cell death of chronic lymphocytic leukemia (CLL) cells regulating the release of proapoptotic factors, such as Smac/Diablo, apoptosis-inducing factor (AIF) and cytochrome c. In April 2016, the U.S. Food and Drug Administration (FDA) granted accelerated approval to venetoclax for patients diagnosed with CLL with 17p deletion, as detected by an FDA-approved test, who have received at least one prior therapy. This review will focus on the mechanism of action, preclinical studies and clinical development of venetoclax both as a monotherapy and in combination with other drugs for CLL in the current milieu of therapy dominated by novel tyrosine kinase inhibitors such as ibrutinib and idelalisib.
Insights
Venetoclax is an oral BCL-2 inhibitor promoting cancer cell death. It received FDA accelerated approval for chronic lymphocytic leukemia (CLL) patients with 17p deletion who have had prior therapy.
Area of Science:
- Oncology
- Pharmacology
Background:
- Chronic lymphocytic leukemia (CLL) is a B-cell malignancy.
- The antiapoptotic protein BCL-2 is upregulated in CLL cells.
- Novel therapies are needed for relapsed/refractory CLL.
Purpose of the Study:
- To review the mechanism of action, preclinical data, and clinical development of venetoclax.
- To discuss venetoclax's role in CLL treatment, including monotherapy and combination strategies.
- To contextualize venetoclax within the current CLL treatment landscape dominated by tyrosine kinase inhibitors.
Main Methods:
- Review of preclinical studies on venetoclax's mechanism of action.
- Analysis of clinical trial data for venetoclax in CLL.
- Evaluation of venetoclax in combination with other CLL therapies.
Main Results:
- Venetoclax selectively inhibits BCL-2, inducing apoptosis in CLL cells.
- Accelerated FDA approval granted for venetoclax in relapsed/refractory CLL with 17p deletion.
- Ongoing research explores venetoclax combinations for improved efficacy.
Conclusions:
- Venetoclax represents a significant advancement in CLL therapy.
- Its targeted mechanism offers a new approach to managing CLL.
- Further studies are evaluating its optimal use in various CLL patient populations.
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