Europe Approves Holoclar, the First Stem Cell-Based Medicinal Product

PARMA and MODENA, Italy, February 23, 2015 /PRNewswire/ --

The collaborationbetween a public excellent researchcenteranda solidprivate pharmaceuticalcompany allowed toachievean extraordinary result, entirely "made in Italy":the first medicinal productcontainingstem cellsapproved in the Western world

The European Commission has granted a conditional marketing authorization, under Regulation (EC) No 726/2004, to Holoclar, an advanced therapy based on autologous stem cells and capable to restore the eyesight of patients with severe cornea damage. Holoclar is manufactured by Holostem Terapie Avanzate (HolostemAdvanced Therapies) - a spin-off of the University of Modena and Reggio Emilia - at the Centre for Regenerative Medicine "Stefano Ferrari" (CMR) of the same University.

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"Holoclaris theveryfirstmedicinalproductbased onstem cellsto beapproved andformallyregisteredin the Western world," states AndreaChiesi, Director of R&D Portfolio Management of Chiesi Farmaceutici S.p.A. and CEO of Holostem Terapie Avanzate. "This record," continues AndreaChiesi,"shows that thepartnershipbetween the public and privatesectorsis not only possible,butisprobably the best strategy for the development of stem cell-based regenerative medicine, particularly when autologous cells are used.Holostemisnowconsideredasabusiness modeltotranslate into clinicstheresultsobtained byscientific researchin this field." Underlying Holoclar are more than 20 years of excellence in research, conducted by a team of internationally renowned scientists in the field of epithelial stem cell biology aimed at clinical translation. European Directive 1394/2007 substantially equalizes advanced cell therapies to medicines and imposes, among other things, that cell cultures has to be manufactured only in GMP-certified facilities (GMP: Good Manufacturing Practice). Thanks to the investments of Chiesi Farmaceutici, the Centre for Regenerative Medicine in Modena - where Holostem operates - was certified as GMP compliant and continue to follow the path towards the registration of this newly developed advanced therapy.

"The authorization processhas been long andcomplex, butthe resultachievedtodayshows thatcellscan beculturedaccording topharmaceutical standardsappropriateto guaranteesafety and efficacy," adds Professor MicheleDeLuca, Scientific Director and co-founder of Holostem, as well as Director of the CMR of the University of Modena. "In addition,ina periodof great confusionabout the realtherapeutic possibilitiesof stem cells,such as the onewe are living in, being ableto demonstratethatstem cells can be definitely safe and successful in a controlled clinical settingismore important than ever." To explain how Holoclar works is Professor GraziellaPellegrini, Coordinator of cell therapy at CMR, as well as director of R&D and co-founder of Holostem, who authored, together with Professor De Luca, the research and designed the product development: "Afterdevelopingcell culturesbased onepithelial stem cellsfor the treatmentofvariousdisorders ofthestratifiedepithelia-from the skinfor full-thicknessburnsto the reconstructionof the urethra-wediscoveredthatthe stem cellsthat allowthe regenerationof the cornearesidein asmall areaatthe borderbetween the cornea(the transparent partat thecenter of the eye)andthe conjunctiva(the contiguous white part),which is called'the limbus'.Whenthermal or chemicalburnsof theocular surfacedamageirreversiblythisstemcellreserve,thecorneal surface-whichin ahealthy eyecompletely renews itself approximatelyeverysix/ninemonths-stopsregeneratingand the conjunctivagraduallybegins tocover thecorneawithawhite coating,thatprevents visionand causes chronicpainandinflammation.Ifinat leastone of the eyes of the patientevenasmallresidueofundamaged limbus is left,we areable to reconstructin a laboratorythe epitheliumthat covers thecorneal surface,thanks to thestem cells harvestedthrough a 1-2mmbiopsy.Thisgraftofepithelium-Holoclar, precisely-that looks likea kind ofcontactlens,is thentransplantedinto the patientandallows to obtain along-termtransparent corneaanda full recoveryof visual acuity,without causing anyrejection reaction,because itconsists of cellsof the patient him/herself."

This therapy, experimentally applied for the first time in humans in the nineties, and designated as orphan drug in 2008, thanks to the registration obtained today, in the near future will be available to all European patients who have suffered workplace injuries (caused, for example, by burnt lime, solvents or acids), domestic accidents (for example eye burns caused in adults and children by detergents or abrasive agents) or - as unfortunately reported by the press in the past few months - in the cases of assault with chemical agents.

Meanwhile, the research in Modena does not stop. The next goal of the team of Emilian researchers and entrepreneurs is to develop new advanced therapy products, such as the gene therapy for the treatment of epidermolysis bullosa, or "Butterfly disease", to date used successfully in the first two patients ever. And to develop new experimental and clinical protocols using different stem cells of stratified epithelia, such as conjunctiva, urethra, oral mucosa and respiratory epithelia.

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Europe Approves Holoclar, the First Stem Cell-Based Medicinal Product