Clinical trials evaluating potential therapies for light chain (AL) amyloidosis

Eli Muchtar, Morie Gertz

Research output: Contribution to journalReview article

Abstract

Introduction: The field of systemic amyloidosis is experiencing major advances in diagnostic and prognostic methods coupled with a growing availability in treatment options. Areas covered: Treatment of AL amyloidosis traditionally targeted the clonal plasma cells, in order to block further production of amyloidogenic light chains. Currently, a research focus is placed on targeting the already formed amyloid deposits using monoclonal antibodies against epitopes on such deposits. Encouraging results were obtained from the three investigated antibodies: NEOD001, 11-1F4 and anti-SAP, but further validation is required before these antibodies can be commercialized. In this paper, we review the current active clinical research in AL amyloidosis, which includes the monoclonal antibodies targeting amyloid deposits, daratumumab, Venetoclax, doxycycline, green tea, pomalidomide, carfilzomib and ixazomib. Expert opinion: Monoclonal antibodies, targeting either the amyloid deposits or the plasma cell compartment will likely be integrated into routine treatment practice given their encouraging results and minimal toxicity in the fragile population of AL amyloidosis. Other therapeutic options hold promise to the field as well, but toxicity will likely challenge their routine use. Early recognition remains the best option for outcome enhancement.

Original languageEnglish (US)
Pages (from-to)655-663
Number of pages9
JournalExpert Opinion on Orphan Drugs
Volume5
Issue number8
DOIs
StatePublished - Aug 3 2017

Fingerprint

Amyloidosis
Amyloid Plaques
Clinical Trials
Light
Monoclonal Antibodies
Plasma Cells
Antibodies
Doxycycline
Expert Testimony
Tea
Therapeutics
Research
Epitopes
Population

Keywords

  • amyloid
  • clinical-trials
  • Monoclonal antibody
  • research

ASJC Scopus subject areas

  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
  • Health Policy
  • Pharmacology (medical)

Cite this

Clinical trials evaluating potential therapies for light chain (AL) amyloidosis. / Muchtar, Eli; Gertz, Morie.

In: Expert Opinion on Orphan Drugs, Vol. 5, No. 8, 03.08.2017, p. 655-663.

Research output: Contribution to journalReview article

@article{2f4c2b17ebe1408fa1b8cd5355d118d2,
title = "Clinical trials evaluating potential therapies for light chain (AL) amyloidosis",
abstract = "Introduction: The field of systemic amyloidosis is experiencing major advances in diagnostic and prognostic methods coupled with a growing availability in treatment options. Areas covered: Treatment of AL amyloidosis traditionally targeted the clonal plasma cells, in order to block further production of amyloidogenic light chains. Currently, a research focus is placed on targeting the already formed amyloid deposits using monoclonal antibodies against epitopes on such deposits. Encouraging results were obtained from the three investigated antibodies: NEOD001, 11-1F4 and anti-SAP, but further validation is required before these antibodies can be commercialized. In this paper, we review the current active clinical research in AL amyloidosis, which includes the monoclonal antibodies targeting amyloid deposits, daratumumab, Venetoclax, doxycycline, green tea, pomalidomide, carfilzomib and ixazomib. Expert opinion: Monoclonal antibodies, targeting either the amyloid deposits or the plasma cell compartment will likely be integrated into routine treatment practice given their encouraging results and minimal toxicity in the fragile population of AL amyloidosis. Other therapeutic options hold promise to the field as well, but toxicity will likely challenge their routine use. Early recognition remains the best option for outcome enhancement.",
keywords = "amyloid, clinical-trials, Monoclonal antibody, research",
author = "Eli Muchtar and Morie Gertz",
year = "2017",
month = "8",
day = "3",
doi = "10.1080/21678707.2017.1341834",
language = "English (US)",
volume = "5",
pages = "655--663",
journal = "Expert Opinion on Orphan Drugs",
issn = "2167-8707",
publisher = "Informa Healthcare",
number = "8",

}

TY - JOUR

T1 - Clinical trials evaluating potential therapies for light chain (AL) amyloidosis

AU - Muchtar, Eli

AU - Gertz, Morie

PY - 2017/8/3

Y1 - 2017/8/3

N2 - Introduction: The field of systemic amyloidosis is experiencing major advances in diagnostic and prognostic methods coupled with a growing availability in treatment options. Areas covered: Treatment of AL amyloidosis traditionally targeted the clonal plasma cells, in order to block further production of amyloidogenic light chains. Currently, a research focus is placed on targeting the already formed amyloid deposits using monoclonal antibodies against epitopes on such deposits. Encouraging results were obtained from the three investigated antibodies: NEOD001, 11-1F4 and anti-SAP, but further validation is required before these antibodies can be commercialized. In this paper, we review the current active clinical research in AL amyloidosis, which includes the monoclonal antibodies targeting amyloid deposits, daratumumab, Venetoclax, doxycycline, green tea, pomalidomide, carfilzomib and ixazomib. Expert opinion: Monoclonal antibodies, targeting either the amyloid deposits or the plasma cell compartment will likely be integrated into routine treatment practice given their encouraging results and minimal toxicity in the fragile population of AL amyloidosis. Other therapeutic options hold promise to the field as well, but toxicity will likely challenge their routine use. Early recognition remains the best option for outcome enhancement.

AB - Introduction: The field of systemic amyloidosis is experiencing major advances in diagnostic and prognostic methods coupled with a growing availability in treatment options. Areas covered: Treatment of AL amyloidosis traditionally targeted the clonal plasma cells, in order to block further production of amyloidogenic light chains. Currently, a research focus is placed on targeting the already formed amyloid deposits using monoclonal antibodies against epitopes on such deposits. Encouraging results were obtained from the three investigated antibodies: NEOD001, 11-1F4 and anti-SAP, but further validation is required before these antibodies can be commercialized. In this paper, we review the current active clinical research in AL amyloidosis, which includes the monoclonal antibodies targeting amyloid deposits, daratumumab, Venetoclax, doxycycline, green tea, pomalidomide, carfilzomib and ixazomib. Expert opinion: Monoclonal antibodies, targeting either the amyloid deposits or the plasma cell compartment will likely be integrated into routine treatment practice given their encouraging results and minimal toxicity in the fragile population of AL amyloidosis. Other therapeutic options hold promise to the field as well, but toxicity will likely challenge their routine use. Early recognition remains the best option for outcome enhancement.

KW - amyloid

KW - clinical-trials

KW - Monoclonal antibody

KW - research

UR - http://www.scopus.com/inward/record.url?scp=85026217008&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85026217008&partnerID=8YFLogxK

U2 - 10.1080/21678707.2017.1341834

DO - 10.1080/21678707.2017.1341834

M3 - Review article

AN - SCOPUS:85026217008

VL - 5

SP - 655

EP - 663

JO - Expert Opinion on Orphan Drugs

JF - Expert Opinion on Orphan Drugs

SN - 2167-8707

IS - 8

ER -