CpG ODN-miR-142 Compounds for Myeloid Leukemia Treatment
Find Innovative SolutionsGenerate Solutions
Solution Overview
Problem
Current treatments for acute myeloid leukemia (AML), particularly secondary AML and blast crisis chronic myelogenous leukemia, are ineffective and often exclude these patients from clinical trials, highlighting a significant unmet need for novel and more effective therapies.
Innovation Solution
The development of compounds comprising a CpG oligodeoxynucleotide (ODN) covalently bonded to a hybridized nucleic acid sequence, where the hybridized sequence includes a miR-142 passenger strand sequence hybridized to a miR-142 guide strand sequence, which are administered to patients to treat myeloid leukemia.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If current therapies are used to treat secondary AML and blast crisis CML, then treatment is provided, but treatment effectiveness is poor and patient outcome remains poor
Solution Approach 1:
The patent changes the molecular parameter by targeting miR-142, a microRNA that is downregulated in secondary AML and blast crisis CML. By restoring miR-142 expression through CpG ODN therapy, the treatment effectively modifies the molecular profile of the leukemia cells, leading to improved treatment response and patient outcomes compared to conventional therapies.
Solution Approach 2:
The patent uses CpG ODNs as intermediary molecules that mediate the restoration of miR-142 expression. These CpG ODNs act as intermediaries between the external treatment and the internal cellular mechanism, binding to TLR9 and triggering the upregulation of miR-142, thereby improving treatment effectiveness in secondary AML and blast crisis CML patients.
2Adaptability or versatility
If conventional therapies are applied to secondary AML and blast crisis CML patients, then treatment is administered, but these patients are often excluded from promising clinical trials
Solution Approach 1:
The patent creates a universal treatment approach using CpG ODNs that can be applied across different subtypes of myeloid leukemia, including secondary AML and blast crisis CML. This multi-functional therapy restores miR-142 expression and improves outcomes in patients who were previously excluded from clinical trials, thereby enhancing both accessibility and effectiveness.
3Reliability
If miR-142 levels are reduced in leukemia patients, then disease progression occurs, but restoring miR-142 requires novel therapeutic compounds
Solution Approach 1:
The patent employs CpG ODNs, which are relatively simple and cost-effective molecular compounds compared to complex protein-based therapies. These disposable-like molecules can be easily administered and rapidly degraded, providing a cost-effective solution for restoring miR-142 expression and controlling disease progression without requiring complex therapeutic compounds.
Applied Scientific Principles
This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.
Function Achieved in This Case
These compounds demonstrate potential in effectively treating myeloid leukemia by targeting specific molecular pathways, thereby improving treatment outcomes for patients with poor prognostic factors such as secondary AML and blast crisis chronic myelogenous leukemia.
Implementation Method 1
the hybridized nucleic acid sequence comprises a miR-142 passenger strand sequence hybridized to a miR-142 guide strand sequence
Data Source
AI summary
The disclosure provides, inter alia, compounds comprising Toll-like receptor 9-binding nucleic acid sequences and nucleic acid sequences comprising a microRNA-142 passenger strand sequence hybridized to a microRNA-142 guide strand sequence; pharmaceutical compositions comprising the compounds; and the use of the compounds and pharmaceutical compositions to treat myeloid leukemia.