Bone-marrow cells enriched with functional mitochondria
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Solution Overview
Problem
Current treatment options for mitochondrial diseases are limited, with vitamins and other agents showing limited effectiveness, and there is a need for potent short- and long-term remedies to address the dysfunction of mitochondria in these diseases.
Innovation Solution
The use of bone-marrow cells enriched with functional mitochondria, obtained from healthy donors, which are treated ex-vivo and returned to the patient, allowing for the transfer of healthy mitochondria to damaged cells, eliminating the need for HLA-matching and immunosuppression therapy, and enabling the replacement of defective mitochondrial genes or proteins.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If conventional treatments (vitamins and agents) are used for mitochondrial diseases, then treatment options are available, but effectiveness is limited
Solution Approach 1:
The patent uses bone-marrow cells as intermediary carriers to deliver functional mitochondria to patients. The bone-marrow cells are enriched with healthy mitochondria ex-vivo, then infused into the patient where they serve as mediators to transfer functional mitochondria to damaged cells, overcoming the limitation of conventional direct treatments
Solution Approach 2:
The treatment process is segmented into distinct phases: (1) isolation of bone-marrow cells from patient, (2) ex-vivo enrichment with functional mitochondria, (3) infusion back to patient, and (4) in-vivo transfer of mitochondria. This segmentation allows each step to be optimized independently, improving overall treatment effectiveness
2Reliability
If allogeneic cell therapy is used to treat mitochondrial diseases, then functional mitochondria can be transferred, but HLA-matching and immunosuppression are required
Solution Approach 1:
The patent uses the patient's own autologous bone-marrow cells as the delivery vehicle for functional mitochondria. This self-service approach eliminates the need for HLA-matching and immunosuppression therapy that would be required if allogeneic cells from donors were used, while still achieving effective mitochondrial transfer
Solution Approach 2:
Instead of using donor cells to deliver mitochondria (allogeneic approach), the patent inverts the approach by using the patient's own cells (autologous approach) that have been enriched with donor mitochondria ex-vivo. This inversion eliminates immunological complications while maintaining therapeutic effectiveness
Data Source
AI summary
The present invention provides human bone-marrow cells enriched with functional mitochondria, methods for their production, and therapeutic methods utilizing such cells.