Table 1.

Immunomodulators in DMD. NFAT, nuclear factor of activated T cells; PDE, phosphodiesterase.

Drug/compoundTargetPathological processPreclinical trialsClinical trials/use
Current treatments
Prednisone, deflazacortNF-κB, othersAnti-inflammatoryYesYes
VBP15NF-κB, membrane protectionAnti-inflammatory, sarcolemma stabilityYesYes*
CyclosporineNFATAnti-inflammatoryYesYes
AzathioprinePurine synthesisAnti-inflammatoryYesYes
PoloxamerMembrane protectionSarcolemma stabilityYesYes
Gene therapyDystrophin replacementSarcolemma stabilityYesYes
Exon skippingDystrophin replacementSarcolemma stabilityYesYes
TLR7/8/9 antagonistsTLR7/8/9Anti-inflammatoryYesNo
NEMO peptideNF-κBAnti-inflammatoryYesNo
InfliximabTNF-αAnti-inflammatoryYesNo
IL-2/anti–IL-2 complexTregsAnti-inflammatoryYesNo
PentoxifyllinePDE inhibitorAnti-fibroticYesYes
PirfenidoneTGF-β signalingAnti-fibroticYesNo
LosartanAngiotensin type 1 receptor inhibitorAnti-fibroticYesYes
LisinoprilAngiotensin-converting enzyme inhibitorAnti-fibroticYesYes
Anti–IL-6IL-6Anti-inflammatoryYesNo
Anti-myostatin antibodiesMyostatinAnti-fibrotic, hypertrophyYesYes
CromolynMast cellsMembrane stabilityYesNo
Future options
ChloroquineLysosomal pHAnti-inflammatoryNoNo
EculizumabComplement C5Anti-inflammatoryNoNo
RapamycinTregs +Akt/mTORAnti-inflammatory, regenerationYesNo
PlerixaforCXCR4Anti-inflammatoryNoNo
IL-10Alternatively activated macrophagesAnti-inflammatoryNoNo
Anti-osteopontin antibodiesOsteopontinAnti-inflammatory, anti-fibroticNoNo
CandesartanAngiotensin type 2 receptor inhibitorAnti-fibroticNoNo

*Phase 1, 2015. †Not effective. ‡Effective in heart.