Holoclar, manufactured by Italian therapeutics company Chiesi, has been granted a conditional marketing authorisation by the European Commission.
While the European Medicines Agency recommended the treatment in December, this month’s announcement (20 February) enables the Italian company to market and sell the treatment in 28 EU member states, which may benefit thousands of people across Europe.The treatment is indicated for patients with limbal stem cell deficiency (LSCD) due to chemical or physical burns. Trauma to the cornea caused by burns can result in damage or loss of the reservoir of cells in the limbus which regenerate the tissue, which can ultimately result in blindness.
LSCD is estimated to affect 3.3 out of 100,000 people in the EU. Symptoms may include pain, hypersensitivity to light, inflammation, increased ingrowth of blood vessels in the cornea, a loss of corneal transparency, and eventually blindness.
The Holoclar treatment involves taking a small biopsy of healthy tissue from the patient’s cornea, a patch of just a few square millimetres from the limbus, and culturing the isolated limbal stem cells in the laboratory. A transparent layer of cells resembling a contact lens is grown, which is then grafted onto the patient’s damaged cornea.
The treatment is reported to work in around 80% of patients, and was trialled in 20 patients at Moorfields last year. As Holoclar uses the patient’s own cells, it could reduce the rate of rejection associated with corneal transplants using donor tissue and potentially reduce the need for immunosuppressant drugs.
Professor Michael De Luca, scientific director and co-founder of Holostem, a spin-off company of the University of Modena in Italy, which manufactures Holoclar, said: “The authorisation 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.”
He added: “Being able to demonstrate that stem cells can be definitely safe and successful in a controlled clinical setting is more important than ever.”
Image credit: Chiesi