First NHS patient receives Chiesi’s advanced-therapy-medicinal product, Holoclar▼ for moderate-to-severe limbal stem cell deficiency (LSCD) caused by chemical or thermal eye burns

Newcastle upon Tyne Hospitals NHS Foundation Trust first to provide breakthrough treatment for rare eye condition that can result in blindness 

Manchester (UK), 31 October 2019 - Chiesi UK today announced that the first NHS funded patient with limbal stem cell deficiency (LSCD) caused by chemical eye burns has been treated with Chiesi's stem-cell based therapy, the first advanced therapy medicinal product containing stem cells to be approved in the European Union (EU).1 LSCD is a rare condition that can lead to blindness, and affects just 3.3 out of 100,000 people in the EU.1 

Chiesi's stem-cell based therapy is a type of advanced treatment called a ‘tissue engineered product’ which consists of cells taken from the patient’s healthy limbus (at the edge of the cornea) during a biopsy.2 The cells obtained during the biopsy are then transported to the manufacturing facility at Holostem Terapie Avanzate in Italy, where they are prepared and grown in a unique culture to create a new layer of healthy tissue.2 After at least 50 days, this layer of healthy tissue is delivered back to the treating hospital and implanted by a surgeon into the damaged eye helping it to heal and repair the damaged corneal surface.2 In case of a partial bilateral problem, the healthy cells are taken from a spared portion of a patient’s less damaged eye.2 

In 2017, the National Institute for Health and Care Excellence (NICE) recommended Holoclar as an option for adults with moderate-to-severe LSCD after eye burns, if it is only used to treat one eye and in those who have already had a conjunctival limbal autograft (or there is not enough tissue for a conjunctival limbal autograft or it is contraindicated).3  

Professor Francisco Figueiredo, Consultant Ophthalmologist at the Newcastle upon Tyne Hospitals NHS Foundation Trust and Professor of Ophthalmology at Newcastle University who treated the first NHS funded patient following NICE approval, said, “In Newcastle we are a leading centre with an international reputation in autologous limbal stem cell transplantation, and our significant scientific research and clinical experience in limbal stem cell deficiency has led to us having proudly performed the first Holoclar treatment on an NHS patient. This operation was the first of a series that may benefit a number of blind patients over the next few years, not only from Newcastle but from across the whole of the UK, helping to restore their sight and comfort”. 

Chiesi's UK Managing Director, Tom Delahoyde commented, “We are delighted that the first patient outside of a clinical trial has received this innovative and breakthrough medicine. This first NHS treatment marks a major milestone for those people with LSCD due to physical or chemical burns and we look forward to many more eligible patients benefiting from such a ground-breaking therapy. Chiesi would like to thank NHS England and the approved Trusts for their support on setting up this new service in the NHS over the last two years and reaching this milestone today.” 

LSCD is seriously debilitating condition affecting one or both eyes; left untreated it results in chronic pain, burning, photophobia, inflammation, corneal neovascularisation, stromal scarring and the reduction or complete loss of vision.4,5 Chemical and physical ocular burn injuries are thought to be the most common cause of LSCD.6  

About LSCD

LSCD is a rare and seriously debilitating condition affecting one or both eyes that left untreated results in chronic pain, burning, photophobia (eye discomfort in bright light), inflammation, corneal neovascularisation (new blood vessels growing across the front of the eye), stromal scarring and the reduction or complete loss of vision.4,5 In this condition, affected patients lack cells called limbal stem cells, which are found at the edge of the cornea (the transparent layer in front of the eye) and which normally continuously renew and repair the cornea.  

Chemical and physical ocular burn injuries are thought to be the most common cause of LSCD.6 The reported incidence of new cases of severe chemical corneal injury in the UK is 0.02 in 100,000 people per year, i.e. 12 new cases per year.7  

The management of LSCD depends on the extent of the damage and symptomatology and aims at achieving symptom relief, improvement in visual acuity and ocular stabilisation.8  

About the treatment Chiesi's stem-cell based therapy is licensed for the treatment of adult patients with moderate-to-severe limbal stem cell deficiency (defined by the presence of superficial corneal neovascularisation in at least two corneal quadrants, with central corneal involvement, and severely impaired visual acuity), unilateral or bilateral, due to physical or chemical ocular burns. A minimum of 1 - 2 mm2 of undamaged limbus is required for biopsy.9 

For full details of adverse reactions and contraindications, see the summary of product characteristics at www.medicines.org.uk/emc/product/8033/smpc.  

Adverse events should be reported▼. This medicinal product is subject to additional monitoring to allow the quick identification of new safety information. It is therefore important to report any suspected adverse events related to this medicinal product. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard. 

About Chiesi Group

Based in Parma, Italy, Chiesi Farmaceutici is an international research-focused Healthcare Group, with over 80 years of experience in the pharmaceutical industry, and with presence in 27 countries. Chiesi researches, develops and markets innovative drugs in the respiratory therapeutics, specialist medicine and rare disease areas. Its R&D organisation is headquartered in Parma (Italy), and integrated with 6 other key R&D groups in France, the USA, the UK and Sweden to advance Chiesi's pre-clinical, clinical and registration programmes. Chiesi employs over 5,700 people.  For more information, please visit www.chiesi.com. Chiesi Limited is the UK affiliate of Chiesi Farmaceutici S.p.A. It is headquartered in Manchester. For more information visit www.chiesi.uk.com.