Table 2.
The potential drugs for DMD anti-inflammatory therapy
| Target | Potential drug | Company/sponsor | Mode of action | Clinical status | Reference* |
|---|---|---|---|---|---|
| Calcium channel | |||||
| TRPV2 | Tranilast | Kissei Pharmaceuticals | TRPV2 blocker | Clinically used antiallergic drug | |
| RyR1 | Rycals (ARM210) | ARMGO Pharma | Stabilizes the closed state of RyRs and inhibits calcium release from SR | Phase I | |
| ROS | Idebenone | Santhera Pharmaceuticals | Antioxidant | Phase III completed, preparation for marketing authorization | NCT01027884 |
| EGCg | Charite University Hospital Berlin | Antioxidant | Phase II/III | NCT01183767 | |
| Flavocoxid | University of Messina | Antioxidant | Phase I completed | NCT0133529 | |
| NF-κB | CAT-1004 | Catabasis | NF-κB inhibitor | Phase I/II | NCT02439216 |
| VBP15 | ReveraGen Biopharma | NF-κB inhibitor, membrane stabilizer | Phase I | NCT02415439 | |
| Remicade | Merck/Schering-Plough | Antibody to human TNF-α | Preclinical | ||
| Etanercept | Wyeth | Blocks soluble receptor to TNF-α | Preclinical | ||
| Follistatin | AAV.CMV.FS344 | Nationwide Children’s Hospital | Delivery of follistatin using adeno-associated virus | Phase I/II | NCT02354781 |
| IGF-1 | INCRELEX | Children’s Hospital Medical Center | Recombinant IGF-1 | Phase II | NCT01207908 |
Note:
*
ClinicalTrials.gov identifier.
Abbreviations: DMD, Duchenne muscular dystrophy; EGCg, epigallocatechin gallate; IGF, insulin-like growth factor; NF-κB, nuclear factor-kappa B; NOX, nicotinamide adenine dinucleotide phosphate oxidase; ROS, reactive oxygen species; RyR, ryanodine receptor; TNF, tumor necrosis factor; TRPV, transient receptor potential vanilloid.