Cardiac amyloidosis future or investigational therapies: Difference between revisions
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* Tamafidis<ref name="pmid22645360">{{cite journal |author=Bulawa CE, Connelly S, Devit M, ''et al.'' |title=Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade |journal=[[Proceedings of the National Academy of Sciences of the United States of America]] |volume=109 |issue=24 |pages=9629–34 |year=2012 |month=June |pmid=22645360 |pmc=3386102 |doi=10.1073/pnas.1121005109 |url=}}</ref><ref name="pmid22843282">{{cite journal |author=Coelho T, Maia LF, Martins da Silva A, ''et al.'' |title=Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial |journal=[[Neurology]] |volume=79 |issue=8 |pages=785–92 |year=2012 |month=August |pmid=22843282 |doi=10.1212/WNL.0b013e3182661eb1 |url=}}</ref> | * Tamafidis<ref name="pmid22645360">{{cite journal |author=Bulawa CE, Connelly S, Devit M, ''et al.'' |title=Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade |journal=[[Proceedings of the National Academy of Sciences of the United States of America]] |volume=109 |issue=24 |pages=9629–34 |year=2012 |month=June |pmid=22645360 |pmc=3386102 |doi=10.1073/pnas.1121005109 |url=}}</ref><ref name="pmid22843282">{{cite journal |author=Coelho T, Maia LF, Martins da Silva A, ''et al.'' |title=Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial |journal=[[Neurology]] |volume=79 |issue=8 |pages=785–92 |year=2012 |month=August |pmid=22843282 |doi=10.1212/WNL.0b013e3182661eb1 |url=}}</ref> | ||
* Eprodisate<ref name="pmid17554116">{{cite journal |author=Dember LM, Hawkins PN, Hazenberg BP, ''et al.'' |title=Eprodisate for the treatment of renal disease in AA amyloidosis |journal=[[The New England Journal of Medicine]] |volume=356 |issue=23 |pages=2349–60 |year=2007 |month=June |pmid=17554116 |doi=10.1056/NEJMoa065644 |url=}}</ref> | * Eprodisate<ref name="pmid17554116">{{cite journal |author=Dember LM, Hawkins PN, Hazenberg BP, ''et al.'' |title=Eprodisate for the treatment of renal disease in AA amyloidosis |journal=[[The New England Journal of Medicine]] |volume=356 |issue=23 |pages=2349–60 |year=2007 |month=June |pmid=17554116 |doi=10.1056/NEJMoa065644 |url=}}</ref> | ||
* Pomalidomide<ref name="pmid22493299">{{cite journal |author=Dispenzieri A, Buadi F, Laumann K, ''et al.'' |title=Activity of pomalidomide in patients with immunoglobulin light-chain amyloidosis |journal=[[Blood]] |volume=119 |issue=23 |pages=5397–404 |year=2012 |month=June |pmid=22493299 |doi=10.1182/blood-2012-02-413161 |url=}}</ref><ref name="pmid23572409">{{cite journal |author=Elkinson S, McCormack PL |title=Pomalidomide: first global approval |journal=[[Drugs]] |volume=73 |issue=6 |pages=595–604 |year=2013 |month=May |pmid=23572409 |doi=10.1007/s40265-013-0047-x |url=}}</ref> | |||
CPHPC, also called R-1-[6-[R-2-carboxy-pyrrolidin-1-yl]-6-oxo-hexanoyl]pyrrolidine-2-carboxylic acid, is a competitive inhibitor of SAP binding to amyloid fibrils. CPHPC is a [[proline]]-derived small palindromic molecule able to strip amyloid P (AP) from deposits by reducing levels of circulating [[serum amyloid protein]] (SAP). It also crosslinks and dimerizes SAP, which is cleared rapidly by the [[liver]]. The SAP-amyloid association has also been identified as a possible drug target for anti-amyloid therapy, with the recent development and first stage clinical trials of CPHPC for [[amyloidosis]]. | CPHPC, also called R-1-[6-[R-2-carboxy-pyrrolidin-1-yl]-6-oxo-hexanoyl]pyrrolidine-2-carboxylic acid, is a competitive inhibitor of SAP binding to amyloid fibrils. CPHPC is a [[proline]]-derived small palindromic molecule able to strip amyloid P (AP) from deposits by reducing levels of circulating [[serum amyloid protein]] (SAP). It also crosslinks and dimerizes SAP, which is cleared rapidly by the [[liver]]. The SAP-amyloid association has also been identified as a possible drug target for anti-amyloid therapy, with the recent development and first stage clinical trials of CPHPC for [[amyloidosis]]. |
Revision as of 19:24, 12 May 2013
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Raviteja Guddeti, M.B.B.S. [2]; Aarti Narayan, M.B.B.S [3]
Overview
New therapies targeting the serum amyloid protein (SAP), which is an excellent immunogen and a universal component of all amyloid deposits, using monoclonal antibodies are currently being investigated.
Future and Investigational Therapies
Possible future treatment options which are currently being investigated include:
CPHPC, also called R-1-[6-[R-2-carboxy-pyrrolidin-1-yl]-6-oxo-hexanoyl]pyrrolidine-2-carboxylic acid, is a competitive inhibitor of SAP binding to amyloid fibrils. CPHPC is a proline-derived small palindromic molecule able to strip amyloid P (AP) from deposits by reducing levels of circulating serum amyloid protein (SAP). It also crosslinks and dimerizes SAP, which is cleared rapidly by the liver. The SAP-amyloid association has also been identified as a possible drug target for anti-amyloid therapy, with the recent development and first stage clinical trials of CPHPC for amyloidosis.
References
- ↑ Bodin K, Ellmerich S, Kahan MC; et al. (2010). "Antibodies to human serum amyloid P component eliminate visceral amyloid deposits". Nature. 468 (7320): 93–7. doi:10.1038/nature09494. PMC 2975378. PMID 20962779. Unknown parameter
|month=
ignored (help) - ↑ Pepys MB, Herbert J, Hutchinson WL, Tennent GA, Lachmann HJ, Gallimore JR, Lovat LB, Bartfai T, Alanine A, Hertel C, Hoffmann T, Jakob-Roetne R, Norcross RD, Kemp JA, Yamamura K, Suzuki M, Taylor GW, Murray S, Thompson D, Purvis A, Kolstoe S, Wood SP, Hawkins PN (2002). "Targeted pharmacological depletion of serum amyloid P component for treatment of human amyloidosis". Nature. 417 (6886): 254–9. doi:10.1038/417254a. PMID 12015594.
- ↑ Sekijima Y, Dendle MA, Kelly JW (2006). "Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis". Amyloid : the International Journal of Experimental and Clinical Investigation : the Official Journal of the International Society of Amyloidosis. 13 (4): 236–49. doi:10.1080/13506120600960882. PMID 17107884. Unknown parameter
|month=
ignored (help) - ↑ Bulawa CE, Connelly S, Devit M; et al. (2012). "Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade". Proceedings of the National Academy of Sciences of the United States of America. 109 (24): 9629–34. doi:10.1073/pnas.1121005109. PMC 3386102. PMID 22645360. Unknown parameter
|month=
ignored (help) - ↑ Coelho T, Maia LF, Martins da Silva A; et al. (2012). "Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial". Neurology. 79 (8): 785–92. doi:10.1212/WNL.0b013e3182661eb1. PMID 22843282. Unknown parameter
|month=
ignored (help) - ↑ Dember LM, Hawkins PN, Hazenberg BP; et al. (2007). "Eprodisate for the treatment of renal disease in AA amyloidosis". The New England Journal of Medicine. 356 (23): 2349–60. doi:10.1056/NEJMoa065644. PMID 17554116. Unknown parameter
|month=
ignored (help) - ↑ Dispenzieri A, Buadi F, Laumann K; et al. (2012). "Activity of pomalidomide in patients with immunoglobulin light-chain amyloidosis". Blood. 119 (23): 5397–404. doi:10.1182/blood-2012-02-413161. PMID 22493299. Unknown parameter
|month=
ignored (help) - ↑ Elkinson S, McCormack PL (2013). "Pomalidomide: first global approval". Drugs. 73 (6): 595–604. doi:10.1007/s40265-013-0047-x. PMID 23572409. Unknown parameter
|month=
ignored (help)