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Emerging targeted therapies for pediatric acute myeloid leukemia
Riccardo Masetti1, Katharina Kleinschmidt, Carlotta Biagi
1Paediatric Oncology and Haematology Unit "Lalla Seràgnoli", University of Bologna Sant'Orsola-Malpighi Hospital, 40138 Bologna, Italy. riccardo.masetti@gmail.com
Insights
Novel targeted therapies show promise for treating childhood acute myeloid leukemia (AML). Research reviews new drugs like monoclonal antibodies and epigenetic agents, improving cure rates and reducing toxicity in pediatric cancer treatment.
Area of Science:
- Pediatric Oncology
- Hematology
- Cancer Biology
Background:
- Childhood acute myeloid leukemia (AML) is a significant pediatric cancer, comprising 15-20% of childhood tumors.
- Recent decades have seen substantial improvements in AML outcomes, with cure rates reaching approximately 60% due to better risk stratification, effective chemotherapy, and supportive care.
Purpose of the Study:
- To review novel molecularly targeted drugs for treating childhood acute myeloid leukemia (AML).
- To analyze preclinical data and clinical trial results for recently patented targeted agents.
Main Methods:
- Literature review of preclinical studies and clinical trials.
- Focus on targeted therapies including monoclonal antibodies, signaling inhibitors, proteasome inhibitors, and epigenetic agents.
Main Results:
- Molecularly targeted therapies offer potential for increased efficacy and reduced toxicity compared to standard treatments for pediatric AML.
- Analysis includes data on monoclonal antibodies, signaling inhibitors, proteasome inhibitors, and epigenetic agents.
Conclusions:
- Advancements in understanding AML biology have led to the development of targeted therapies.
- These novel agents represent a promising direction for improving treatment outcomes in children with acute myeloid leukemia.
Abstract:
Acute myeloid leukemia is a heterogeneous group of diseases accounting for 15-20% of all tumors diagnosed in children under 15 years of age. The past few decades have yielded remarkable improvements in long-term outcomes for children with acute myeloid leukemia. A better risk-group stratification of patients based upon clinical and biologic features, a more effective use of anti-leukemic agents and enormous improvements in supportive care have increased the probability of cure by approximately 60%. The increase in our understanding of the biology of this disease has resulted in the development of molecularly targeted therapies that are potentially more effective and less toxic than the standard approaches. We here review novel molecularly targeted drugs for the treatment of childhood acute myeloid leukemia such as monoclonal antibodies, inhibitors of signalling molecules, proteasome inhibitors and epigenetic agents. For these recently patented agents, we also provide a detailed analysis of the published preclinical data and the clinical trials that have been completed.
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