PMID- 33287176 OWN - NLM STAT- PubMed-not-MEDLINE LR - 20240330 IS - 1999-4923 (Print) IS - 1999-4923 (Electronic) IS - 1999-4923 (Linking) VI - 12 IP - 12 DP - 2020 Dec 3 TI - Recent Research in Ocular Cystinosis: Drug Delivery Systems, Cysteamine Detection Methods and Future Perspectives. LID - 10.3390/pharmaceutics12121177 [doi] LID - 1177 AB - Cystinosis is a rare genetic disorder characterized by the accumulation of cystine crystals in different tissues and organs. Although renal damage prevails during initial stages, the deposition of cystine crystals in the cornea causes severe ocular manifestations. At present, cysteamine is the only topical effective treatment for ocular cystinosis. The lack of investment by the pharmaceutical industry, together with the limited stability of cysteamine, make it available only as two marketed presentations (Cystaran((R)) and Cystadrops((R))) and as compounding formulations prepared in pharmacy departments. Even so, new drug delivery systems (DDSs) need to be developed, allowing more comfortable dosage schedules that favor patient adherence. In the last decades, different research groups have focused on the development of hydrogels, nanowafers and contact lenses, allowing a sustained cysteamine release. In parallel, different determination methods and strategies to increase the stability of the formulations have also been developed. This comprehensive review aims to compile all the challenges and advances related to new cysteamine DDSs, analytical determination methods, and possible future therapeutic alternatives for treating cystinosis. FAU - Castro-Balado, Ana AU - Castro-Balado A AUID- ORCID: 0000-0001-5168-114X AD - Pharmacy Department, University Clinical Hospital of Santiago de Compostela (SERGAS), 15706 Santiago de Compostela, Spain. AD - Pharmacology Group, Health Research Institute of Santiago de Compostela (FIDIS), 15706 Santiago de Compostela, Spain. AD - Department of Pharmacology, Pharmacy and Pharmaceutical Technology, Faculty of Pharmacy, University of Santiago de Compostela (USC), 15782 Santiago de Compostela, Spain. FAU - Mondelo-Garcia, Cristina AU - Mondelo-Garcia C AUID- ORCID: 0000-0001-8555-1415 AD - Pharmacy Department, University Clinical Hospital of Santiago de Compostela (SERGAS), 15706 Santiago de Compostela, Spain. AD - Pharmacology Group, Health Research Institute of Santiago de Compostela (FIDIS), 15706 Santiago de Compostela, Spain. FAU - Varela-Rey, Iria AU - Varela-Rey I AD - Pharmacy Department, University Clinical Hospital of Santiago de Compostela (SERGAS), 15706 Santiago de Compostela, Spain. AD - Pharmacology Group, Health Research Institute of Santiago de Compostela (FIDIS), 15706 Santiago de Compostela, Spain. AD - Department of Pharmacology, Pharmacy and Pharmaceutical Technology, Faculty of Pharmacy, University of Santiago de Compostela (USC), 15782 Santiago de Compostela, Spain. FAU - Moreda-Vizcaino, Beatriz AU - Moreda-Vizcaino B AD - Department of Pharmacology, Pharmacy and Pharmaceutical Technology, Faculty of Pharmacy, University of Santiago de Compostela (USC), 15782 Santiago de Compostela, Spain. FAU - Sierra-Sanchez, Jesus F AU - Sierra-Sanchez JF AD - Pharmacy Department, Hospital de Jerez de la Frontera, Jerez de la Frontera, 11407 Cadiz, Spain. FAU - Rodriguez-Ares, Maria Teresa AU - Rodriguez-Ares MT AD - Ophthalmology Department, University Clinical Hospital of Santiago de Compostela (SERGAS), 15706 Santiago de Compostela, Spain. FAU - Hermelo-Vidal, Gonzalo AU - Hermelo-Vidal G AD - Pharmacology Group, Health Research Institute of Santiago de Compostela (FIDIS), 15706 Santiago de Compostela, Spain. FAU - Zarra-Ferro, Irene AU - Zarra-Ferro I AD - Pharmacy Department, University Clinical Hospital of Santiago de Compostela (SERGAS), 15706 Santiago de Compostela, Spain. AD - Pharmacology Group, Health Research Institute of Santiago de Compostela (FIDIS), 15706 Santiago de Compostela, Spain. FAU - Gonzalez-Barcia, Miguel AU - Gonzalez-Barcia M AD - Pharmacy Department, University Clinical Hospital of Santiago de Compostela (SERGAS), 15706 Santiago de Compostela, Spain. AD - Pharmacology Group, Health Research Institute of Santiago de Compostela (FIDIS), 15706 Santiago de Compostela, Spain. FAU - Yebra-Pimentel, Eva AU - Yebra-Pimentel E AD - Department of Applied Physics, Optometry, Faculty of Optics and Optometry, University of Santiago de Compostela (USC), 15782 Santiago de Compostela, Spain. FAU - Giraldez-Fernandez, Maria Jesus AU - Giraldez-Fernandez MJ AD - Department of Applied Physics, Optometry, Faculty of Optics and Optometry, University of Santiago de Compostela (USC), 15782 Santiago de Compostela, Spain. FAU - Otero-Espinar, Francisco J AU - Otero-Espinar FJ AUID- ORCID: 0000-0001-9030-2253 AD - Department of Pharmacology, Pharmacy and Pharmaceutical Technology, Faculty of Pharmacy, University of Santiago de Compostela (USC), 15782 Santiago de Compostela, Spain. FAU - Fernandez-Ferreiro, Anxo AU - Fernandez-Ferreiro A AUID- ORCID: 0000-0002-7348-7337 AD - Pharmacy Department, University Clinical Hospital of Santiago de Compostela (SERGAS), 15706 Santiago de Compostela, Spain. AD - Pharmacology Group, Health Research Institute of Santiago de Compostela (FIDIS), 15706 Santiago de Compostela, Spain. LA - eng GR - XVI Convocatoria de Ayudas a la Investigacion en Salud/Fundacion Mutua Madrilena/ GR - FEFH 18-19/Sociedad Espanola de Farmacia Hospitalaria/ GR - JR18/0004/Instituto de Salud Carlos III/ GR - CM18/00090/Instituto de Salud Carlos III/ GR - Asociacion La Lucha de Iker/Asociacion La Lucha de Iker/ GR - Mejor Proyecto I+i 64 Congreso SEFH Sevilla/Sociedad Espanola de Farmacia Hospitalaria/ PT - Journal Article PT - Review DEP - 20201203 PL - Switzerland TA - Pharmaceutics JT - Pharmaceutics JID - 101534003 PMC - PMC7761701 OTO - NOTNLM OT - analytical chemistry methods OT - cysteamine OT - cystinosis OT - drug delivery systems OT - ophthalmic administration COIS- The authors declare no conflict of interest. EDAT- 2020/12/09 06:00 MHDA- 2020/12/09 06:01 PMCR- 2020/12/03 CRDT- 2020/12/08 01:07 PHST- 2020/11/10 00:00 [received] PHST- 2020/11/27 00:00 [revised] PHST- 2020/12/02 00:00 [accepted] PHST- 2020/12/08 01:07 [entrez] PHST- 2020/12/09 06:00 [pubmed] PHST- 2020/12/09 06:01 [medline] PHST- 2020/12/03 00:00 [pmc-release] AID - pharmaceutics12121177 [pii] AID - pharmaceutics-12-01177 [pii] AID - 10.3390/pharmaceutics12121177 [doi] PST - epublish SO - Pharmaceutics. 2020 Dec 3;12(12):1177. doi: 10.3390/pharmaceutics12121177.