Synergistic Cysteamine Delivery Nanowafer as an Efficacious Treatment Modality for Corneal Cystinosis

• DOI: 10.1021/acs.molpharmaceut.6b00488
• Publication Date: 2016-08-29T21:11:59Z
• Authors: Daniela C. Marcano; Crystal S. Shin; Briana Lee; Lucas C. Isenhart; Xing Liu; Feng Li; James V. Jester; Stephen C. Pflugf...; Jennifer Simpson; Ghanashyam Acharya
• Keywords: Cystinosis; Cysteamine; Treatment modality

A synergy between the polymer biomaterial and drug plays an important role in enhancing therapeutic efficacy, improving stability, minimizing local immune responses development of delivery systems. Particularly, case ocular delivery, need for synergistic system becomes more pronounced because wet mucosal surface highly innervated cornea, which elicit a strong inflammatory response to instilled formulations. This article presents cysteamine nanowafer treat corneal cystinosis. Corneal cystinosis is rare metabolic disease that causes accumulation cystine crystals cornea resulting opacity loss vision. It treated with topical (Cys) eye drops be 6–12 times day throughout patient's life, side effects such as pain, redness, inflammation. As result, compliance treatment outcomes are severely compromised. To surmount these issues, we have developed clinically translatable Cys (Cys-NW) can simply applied on fingertip. During course release, Cys-NW slowly dissolves fades away. The vivo studies cystinosin knockout mice demonstrated twice efficacy containing 10 μg administered once day, compared 44 day. Furthermore, stabilizes up four months at room temperature frozen or refrigerated still remain active only 1 week. Cys-NW, its enhanced safety profile, extended stability temperature, rapidly translated clinic human trials.