The first stem cell therapy for use in the European Union was approved in December 2014 by the European Medicines Agency (EMA), a major advance for the technology. It will be used for a rare eye condition that can result in blindness, and could be available on the NHS within a few years.
Limbal stem cell deficiency (LSCD) is a condition where burns or toxic chemicals have damaged limbal stem cells. These specialised cells, at the border between the cornea and the sclera or white of the eye, are important for regenerating and healing damage to the outer layer of the cornea known as the corneal epithelium. LSCD affects roughly 3.3 out of 100,000 people in the EU. Symptoms include pain, sensitivity to light, inflammation, excessive blood vessel growth, clouding of the cornea, and eventually blindness.
Holoclar, developed by the Italian pharma company Chiesi Farmaceutici, is the first medicine recommended for LSCD. The new treatment takes a small sample of the patient’s healthy cornea, isolates the limbal stem cells from it and then grows them under carefully controlled conditions until there are enough to put back onto the eye. This can help prevent further corneal damage and loss of sight. When there is already extensive damage, the treatment may also help a corneal graft to heal more quickly.
‘This recommendation represents a major step forward,’ says Enrica Alteri, head of the EMA’s human medicines evaluation division. As the product was designated an orphan medicine, the agency was able to provide support, including several rounds of free scientific advice to Chiesi during Holoclar’s development.
The EMA decision to approve Holoclar will now be sent to the European Commission for market authorisation. It will then be up to member states, such as the National Institute for Health and Care Excellence (NICE) in the UK, to recommend the therapy for use in their country.
‘This is exciting,’ says Graham McGeown of the Centre for Experimental Medicine, Queen’s University Belfast, UK, ‘both because there are no other treatments for this sort of damage to the eye and because this is the first example in which a “medicine” based on stem cells has been approved for use in humans. Because the cells come from the patient themselves, there should be no worries that the body will reject them as can happen when using a transplant from another person. This treatment will only be allowed under carefully defined conditions, however, so that the outcomes can be carefully monitored to see if the treatment works and doesn’t have any unexpected side-effects.’
The therapeutic approach on which Holoclar is based is relatively well established and has been performed in multiple academic institutions around the world, comments Dusko Ilic of King’s College London, UK. ‘It was just a question of when it would be approved by regulatory agencies, including the UK. I hope that Holoclar is not just an exception, and that many more cellular therapy products will see market in the next year.’
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