Topical diacerein for epidermolysis bullosa: a randomized controlled pilot study

Wally, Verena; Kitzmueller, Sophie; Lagler, Florian B.; Moder, Angelika; Hitzl, Wolfgang; Wolkersdorfer, Martin; Hofbauer, Peter; Felder, Thomas K.; Dornauer, Michael; Diem, Anja; Eiler, Nora; Bauer, Johann

doi:10.1186/1750-1172-8-69

Cited by 50 publications

(43 citation statements)

References 5 publications

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“…Diacerein, an IL-1β inhibitor, reduced blistering in EBS in a small pilot study (Wally et al 2013). Small molecule-based approaches have recently emerged as a novel way to target keratinising diseases.…”

Section: Current Therapeutic Approachesmentioning

confidence: 99%

Keratins and skin disease

2015

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Mutations in keratin genes cause a diverse spectrum of skin, hair and mucosal disorders. Cutaneous disorders include epidermolysis bullosa simplex, palmoplantar keratoderma, epidermolytic ichthyosis and pachyonychia congenita. Both clinical and laboratory observations confirm a major role for keratins in maintaining epidermal cell-cell adhesion. When normal tissue homeostasis is disturbed, for example, during wound healing and cancer, keratins play an important non-mechanical role. Post-translational modifications including glycosylation and phosphorylation of keratins play an important role in protection of epithelial cells from injury. Keratins also play a role in modulation of the immune response. A current focus in the area of keratins and disease is the development of new treatments including small inhibitory RNA (siRNA) to mutant keratins and small molecules to modulate keratin expression.

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Section: Current Therapeutic Approachesmentioning

confidence: 99%

Keratins and skin disease

2015

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“…Based on our in vitro data, we launched a double‐blind, randomized, placebo‐controlled phase 1 pilot study of topical diacerein 1% cream in 5 patients with EBS‐gen sev . Topical formulation of oral diacerein (which is already approved for the treatment of osteoarthritis) should account for feasibility and future cost‐efficiency while minimizing the probability of systemic side effects.…”

Section: Clinical Researchmentioning

confidence: 99%

Epidermolysis bullosa: Advances in research and treatment

Prodinger

Reichelt

Bauer

et al. 2019

Experimental Dermatology

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Epidermolysis bullosa (EB) is the umbrella term for a group of rare inherited skin fragility disorders caused by mutations in at least 20 different genes. There is no cure for any of the subtypes of EB resulting from different mutations, and current therapy only focuses on the management of wounds and pain. Novel effective therapeutic approaches are therefore urgently required. Strategies include gene‐, protein‐ and cell‐based therapies. This review discusses molecular procedures currently under investigation at the EB House Austria, a designated Centre of Expertise implemented in the European Reference Network for Rare and Undiagnosed Skin Diseases. Current clinical research activities at the EB House Austria include newly developed candidate substances that have emerged out of our translational research initiatives as well as already commercially available medications that are applied in off‐licensed indications. Squamous cell carcinoma is the major cause of death in severe forms of EB. We are evaluating immunotherapy using an anti‐PD1 monoclonal antibody as a palliative treatment option for locally advanced or metastatic squamous cell carcinoma of the skin unresponsive to previous systemic therapy. In addition, we are evaluating topical calcipotriol and topical diacerein as potential agents to improve the healing of skin wounds in EBS patients. Finally, the review will highlight the recent advancements of gene therapy development for EB.

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“…As a consequence, research on these diseases has ultimately proved to be very useful for a better understanding of the mechanism of very common conditions, as they may represent a model of dysfunction of a distinct biological pathway. These findings paved the way for a new era of targeted therapeutic interference, in dermatological terms to mention icatibant for hereditary and sporadic angioedema, vismodegib for basal cell carcinoma and Gorlin syndrome, as well as diacerein for EB [14–16]. …”

Section: Perspectives Of Molecular Research In Rd – From the Rarest Tmentioning

confidence: 99%

Epidermolysis bullosa House Austria and Epidermolysis bullosa clinical network

Laimer

Pohla‐Gubo

Diem

et al. 2016

Wien Klin Wochenschr

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SummaryAccurately addressing the diverse and complex issues of rare diseases (RD) in terms of prevention, recognition, diagnosis, treatment, care and research along key RD specificities, such as great heterogeneity, a limited number of patients, scarcity of relevant knowledge and expertise as well as enormous costs for patient care is a challenging task for healthcare providers and authorities that makes a supranational approach particularly feasible. The European Union has acknowledged RD matters by several initiatives, including efforts to implement national centres of expertise and European reference networks as well as a cross-border referral mechanism to foster access to expert services and to boost dissemination of clinical expertise and research activities. Exemplified by the EB House Austria, a centre of expertise for epidermolysis bullosa cross-linked with international reference partner institutions, this strategy proves its potential to be translated into optimized patient care and to meet the major medical, scientific, social and health-economic impact of RD.

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Topical diacerein for epidermolysis bullosa: a randomized controlled pilot study

Cited by 50 publications

References 5 publications

Keratins and skin disease

Keratins and skin disease

Epidermolysis bullosa: Advances in research and treatment

Epidermolysis bullosa House Austria and Epidermolysis bullosa clinical network

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