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An Alternative Culture Method to Maintain Genomic Hypomethylation of Mouse Embryonic Stem Cells Using MEK Inhibitor PD0325901 and Vitamin C
Published on: June 1, 2018
Leveraging Hypomethylating Agents for Better MDS Therapy
Terrence J Bradley1, Justin M Watts2, Ronan T Swords2
1Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, 90 SW 3rd Street #2210, Miami, FL, 33130, USA. Tjb127@med.miami.edu.
Purpose Of Review:
Myelodysplastic syndrome (MDS) is a clinically and molecularly heterogeneous disease, which primarily occurs in older adults. Although hypomethylating agents have survival benefit and are the current standard of care, many MDS patients will not garner a response from therapy. For those who do respond, most responses are not durable, and the only hope for a cure is allogeneic stem cell transplant. New therapies to improve outcomes are urgently needed.
Recent Findings:
Clinical trials combining standard hypomethylating agents with novel experimental agents are underway in an effort to improve clinical outcomes in MDS patients. Several of these small molecules have demonstrated the ability to augment the response rates of hypomethylating agents alone, including complete remission rates, in both the front line and refractory settings. Combination approaches utilizing hypomethylating agents and novel-targeted therapies have demonstrated the ability to improve response rates in MDS patients in both the front line and salvage settings, and thus may change the standard of care.
Insights
New therapies combining hypomethylating agents with novel treatments show promise for myelodysplastic syndrome (MDS) patients, improving response rates and potentially altering standard care for this heterogeneous blood cancer.
Area of Science:
- Hematology
- Oncology
- Molecular Biology
Background:
- Myelodysplastic syndrome (MDS) is a heterogeneous blood disorder primarily affecting older adults.
- Current standard treatments, hypomethylating agents, offer survival benefits but lack durable responses for many patients.
- Allogeneic stem cell transplant remains the only curative option for MDS, highlighting the need for novel therapies.
Purpose of the Study:
- To review recent findings on novel therapeutic strategies for myelodysplastic syndrome (MDS).
- To evaluate the potential of combination therapies in improving clinical outcomes for MDS patients.
- To identify emerging treatments that may change the standard of care for MDS.
Main Methods:
- Review of ongoing clinical trials combining hypomethylating agents with novel experimental agents.
- Analysis of data on small molecules designed to augment hypomethylating agent efficacy.
- Evaluation of combination approaches in both front-line and refractory MDS settings.
Main Results:
- Several novel small molecules have shown potential to increase response rates to hypomethylating agents.
- Combination therapies have demonstrated improved complete remission rates in MDS patients.
- These novel combinations show promise in both initial treatment and salvage settings.
Conclusions:
- Combination therapies involving hypomethylating agents and novel targeted agents are a promising avenue for improving MDS treatment outcomes.
- These novel approaches may lead to enhanced response rates and more durable remissions in MDS patients.
- Emerging combination strategies have the potential to significantly alter the current standard of care for myelodysplastic syndrome.
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