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

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

The collaboration  between a public excellent research center and  a solid  private pharmaceutical company allowed to achieve  an extraordinary result, entirely "made in Italy": the first medicinal product  containing  stem cells  approved 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 (Holostem  Advanced 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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"Holoclar is the very first medicinal product  based on  stem cells to be approved and  formally  registered  in the Western world," states Andrea  Chiesi, Director of R&D Portfolio Management of Chiesi Farmaceutici S.p.A. and CEO of Holostem Terapie Avanzate. "This record," continues Andrea Chiesi,  "shows that the  partnership  between the public and private sectors is not only possible, but  is probably the best strategy for the development of stem cell-based regenerative medicine, particularly when autologous cells are used.  Holostem  is now considered  as a  business model  to translate into clinics the results  obtained by  scientific research  in 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 process  has been long and  complex, but  the result achieved today  shows that  cells  can be cultured  according to  pharmaceutical standards appropriate to guarantee  safety and efficacy," adds Professor Michele  De  Luca, Scientific Director and co-founder of Holostem, as well as Director of the CMR of the University of Modena. "In addition, in  a period  of great confusion  about the real  therapeutic possibilities  of stem cells,  such as the one  we are living in, being able to demonstrate  that  stem cells can be definitely safe and successful in a controlled clinical setting is  more important than ever."
To explain how Holoclar^® works is Professor Graziella  Pellegrini, 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: "After developing  cell cultures based on epithelial stem cells  for the treatment  of  various  disorders of the stratified epithelia  - from the skin for full-thickness burns to the reconstruction  of the urethra  -  we discovered that  the stem cells  that allow  the regeneration  of the cornea  reside  in a  small area at the border  between the cornea  (the transparent part at the  center of the eye) and  the conjunctiva  (the contiguous white part), which is called  'the limbus'. When  thermal or chemical  burns of the ocular surface  damage  irreversibly  this  stem  cell reserve,  the  corneal surface  -  which  in a  healthy eye  completely renews itself approximately  every six/nine months  -  stops  regenerating  and the conjunctiva  gradually  begins to  cover the  cornea  with  a white coating,  that  prevents vision  and causes chronic  pain  and  inflammation. If in at least  one of the eyes of the patient  even  a  small  residue  of  undamaged limbus is left,  we areable to reconstruct  in  a  laboratory  the epithelium  that covers the  corneal surface, thanks to the  stem cells harvested  through a 1-2mm² biopsy. This graft of  epithelium  -  Holoclar^®, precisely -  that looks like  a kind of  contact  lens, is then  transplanted  into the patient  and  allows to obtain a long-term  transparent cornea  and  a full recovery  of visual acuity, without causing any  rejection reaction,  because it  consists of cells  of 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.

http://www.chiesi.com

http://www.holostem.com

http://www.cmr.unimore.it