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Limbal stem cell deficiency: Holoclar for the treatment of this rare condition caused by burns to the eye

The European Medicines Agency ( EMA ) has recommended Holoclar, the first advanced therapy medicinal product ( ATMP ) containing stem cells, for approval in the European Union ( EU ).
Holoclar is a treatment for moderate to severe limbal stem cell deficiency ( LSCD ) due to physical or chemical burns to the eye(s) in adults.
It is the first medicine recommended for LSCD, a rare eye condition that can result in blindness.

The active substance of Holoclar is ex-vivo expanded autologous human corneal epithelial cells containing stem cells.

Stem cells can act as a repair system for the body. Limbal stem cells are located in the eye at the border between the cornea and the sclera. These cells are important for regenerating and healing damage to the outer layer of the cornea ( corneal epithelium ).
Physical or chemical burns can cause loss of these stem cells, resulting in LSCD, a condition that is estimated to affect about 3.3 out of 100,000 people in the European Union.
Symptoms include pain, photophobia, inflammation, corneal neovascularisation ( excessive ingrowth of blood vessels into the cornea ), loss of corneal transparency, and eventually blindness.

Holoclar is a living tissue equivalent intended to be transplanted in the affected eye(s) after removal of the altered corneal epithelium. It is made from a biopsy taken from a small undamaged area ( minimum of 1-2 mm2 ) of the patient’s cornea and grown in the laboratory using cell culture.

Holoclar can offer an alternative to transplantation for replacing altered corneal epithelium in some cases, and it has been shown to increase the chances of a successful corneal transplant where the injury has caused extensive eye damage.
It reduces the risk of rejection compared with transplanting tissue from a donor and does not require surgery on the patient’s other eye as only a small biopsy is performed to collect the cells, thus reducing the risk of damage to the healthy eye. Therefore, Holoclar may also be suitable where both eyes are affected by moderate to severe LSCD.

The CAT ( Committee for Advanced Therapies ) and the CHMP ( Committee for Medicinal Products for Human Use ) have considered that Holoclar provides a first treatment option for this rare eye condition and have recommended a conditional marketing authorisation because, although the data supplied by the applicant show that the medicine's benefits outweigh its risks, the data are based on retrospective studies and are not yet comprehensive. Therefore, an additional study on the use of Holoclar should be conducted. ( Xagena )

Source: EMA, 2014