Hemophilia future or investigational therapies: Difference between revisions
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==Overview== | |||
Future and investigational [[Therapy|therapies]] of hemophilia include co-transplantation of [[Endothelium|endothelial]] colony-forming [[Cell (biology)|cells]] (ECFCs) and [[placenta]]-derived [[Mesenchyme|mesenchymal]] [[stromal]] [[Cell (biology)|cells]] (PMSCs) at the [[Infant|neonatal]] age and prevention of the anti-[[factor VIII]] memory [[B cell|B-cell]] response. | |||
==Future and Investigational Therapies== | |||
Future and investigational [[Therapy|therapies]] of hemophilia include: | |||
'''Co-transplantation of endothelial colony-forming cells (ECFCs) and placenta-derived mesenchymal stromal cells (PMSCs)''' | |||
*This is done at the neonatal age.<ref name="pmid30670078">{{cite journal |vauthors=Gao K, Kumar P, Cortez-Toledo E, Hao D, Reynaga L, Rose M, Wang C, Farmer D, Nolta J, Zhou J, Zhou P, Wang A |title=Potential long-term treatment of hemophilia A by neonatal co-transplantation of cord blood-derived endothelial colony-forming cells and placental mesenchymal stromal cells |journal=Stem Cell Res Ther |volume=10 |issue=1 |pages=34 |date=January 2019 |pmid=30670078 |pmc=6341603 |doi=10.1186/s13287-019-1138-8 |url=}}</ref> | |||
*It is a potential strategy to achieve stable and long-term engraftment.<ref name="pmid30670078">{{cite journal |vauthors=Gao K, Kumar P, Cortez-Toledo E, Hao D, Reynaga L, Rose M, Wang C, Farmer D, Nolta J, Zhou J, Zhou P, Wang A |title=Potential long-term treatment of hemophilia A by neonatal co-transplantation of cord blood-derived endothelial colony-forming cells and placental mesenchymal stromal cells |journal=Stem Cell Res Ther |volume=10 |issue=1 |pages=34 |date=January 2019 |pmid=30670078 |pmc=6341603 |doi=10.1186/s13287-019-1138-8 |url=}}</ref> | |||
*This holds great promise for the [[Cell (biology)|cell]]-based treatment of hemophilia A. | |||
'''Prevention of the anti-factor VIII memory B-cell response''' | |||
*It is an investigational [[therapy]] to counter the formation of inhibitors. | |||
*This is achieved via selective [[Enzyme inhibitor|inhibition]] of the [[Bruton's tyrosine kinase]], (R)-5-amino-1-(1-cyanopiperidin-3-yl)-3-(4-[2,4-difluorophenoxyl] phenyl)-1H pyrazole-4-carboxamide (PF-06250112), to inhibit [[B cell|B-cell]] [[Receptor (biochemistry)|receptor]] signaling prior to challenge with [[exogenous]] [[factor VIII]].<ref name="pmid30545924">{{cite journal |vauthors=Delignat S, Russick J, Gangadharan B, Rayes J, Ing M, Voorberg J, Kaveri SV, Lacroix-Desmazes S |title=Prevention of the anti-factor VIII memory B-cell response by inhibition of the Bruton's tyrosine kinase in experimental hemophilia A |journal=Haematologica |volume= |issue= |pages= |date=December 2018 |pmid=30545924 |doi=10.3324/haematol.2018.200279 |url=}}</ref> | |||
==References== | ==References== | ||
Latest revision as of 15:32, 30 January 2019
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1];Associate Editor(s)-in-Chief: Sabawoon Mirwais, M.B.B.S, M.D.[2]
Overview
Future and investigational therapies of hemophilia include co-transplantation of endothelial colony-forming cells (ECFCs) and placenta-derived mesenchymal stromal cells (PMSCs) at the neonatal age and prevention of the anti-factor VIII memory B-cell response.
Future and Investigational Therapies
Future and investigational therapies of hemophilia include:
Co-transplantation of endothelial colony-forming cells (ECFCs) and placenta-derived mesenchymal stromal cells (PMSCs)
- This is done at the neonatal age.[1]
- It is a potential strategy to achieve stable and long-term engraftment.[1]
- This holds great promise for the cell-based treatment of hemophilia A.
Prevention of the anti-factor VIII memory B-cell response
- It is an investigational therapy to counter the formation of inhibitors.
- This is achieved via selective inhibition of the Bruton's tyrosine kinase, (R)-5-amino-1-(1-cyanopiperidin-3-yl)-3-(4-[2,4-difluorophenoxyl] phenyl)-1H pyrazole-4-carboxamide (PF-06250112), to inhibit B-cell receptor signaling prior to challenge with exogenous factor VIII.[2]
References
- ↑ 1.0 1.1 Gao K, Kumar P, Cortez-Toledo E, Hao D, Reynaga L, Rose M, Wang C, Farmer D, Nolta J, Zhou J, Zhou P, Wang A (January 2019). "Potential long-term treatment of hemophilia A by neonatal co-transplantation of cord blood-derived endothelial colony-forming cells and placental mesenchymal stromal cells". Stem Cell Res Ther. 10 (1): 34. doi:10.1186/s13287-019-1138-8. PMC 6341603. PMID 30670078.
- ↑ Delignat S, Russick J, Gangadharan B, Rayes J, Ing M, Voorberg J, Kaveri SV, Lacroix-Desmazes S (December 2018). "Prevention of the anti-factor VIII memory B-cell response by inhibition of the Bruton's tyrosine kinase in experimental hemophilia A". Haematologica. doi:10.3324/haematol.2018.200279. PMID 30545924.