Being a pioneer is never easy. You are charting unknown territory, tackling problems that have defeated others before you. You have to overcome so many obstacles that at times the challenge can seem insurmountable. But for those who succeed in reaching their goal, the rewards can be extraordinary.
Last month Italian researcher Graziella Pellegrini saw 25 years of work pay off when a treatment she developed to cure a form of blindness was given approval for sale by the European Commission.
This is quite an achievement as this means her treatment, called Holoclar, is among the first commercial stem therapies in the world (the first was Prochymal, which has been approved in Canada and New Zealand for the treatment of pediatric GVHD. This drug was developed by Osiris, which was led by our current President & CEO, Dr. Randy Mills.)
Holoclar uses stem cells to help stimulate the regrowth of a cornea. It can only be used for certain rare conditions, but that in no way diminishes its importance for patients or significance for the regenerative medicine field as a whole.
Nature recently sat down with Dr. Pellegrini to talk about her work, her struggle, and the many obstacles she had to overcome to get market approval for her work.
The interview makes for fascinating reading, and is a timely reminder why this kind of groundbreaking research never goes quite as quickly, or smoothly, as one would hope.
CIRM currently has a number of projects focused treating different causes of blindness on limbal cells (the kind Dr. Pellegrini worked on) and other forms of blindness; including a project to treat macular degeneration that has been approved for a clinical trial, and a therapy for retinitis pigmentosa that we hope will be approved for a clinical trial later this year.