Breakthrough Treatment for People with a Rare Eye Condition (Limbal Stem Cell Deficiency)

Limbal Stem Cell Deficiency (LSCD) is a rare, long-term debilitating eye disease, caused by autoimmune disorders, infection, congenital defects, and trauma to the eye including thermal and chemical burns. People diagnosed with this condition suffer from sustained pain, irritation and blurred vision, which can lead to blindness.

The disease is characterised by dysfunction or loss of limbal stem cells, which are located at the outer edges of the cornea (the clear dome shaped covering on the eye surface) and are responsible for restoration of any damage to the outer epithelial layer of the cornea. LSCD can be partial (limited number of functioning limbal stems remaining) or total (no evidence of functioning stem cells). The disease can also be unilateral (affecting one eye only) or bilateral (affecting both eyes).

This project was set up to take an advanced therapeutic medicinal product developed entirely by experts working for both Newcastle University and The Newcastle upon Tyne Hospitals NHS Foundation Trust (NUTH; operating as part of the Newcastle Academic Health Partners*) to market.  The treatment involves taking stem cells from the healthy eye of a patient with unilateral LSCD, expanding them in the lab and then transplanting them into the patient’s injured eye (this is described as an autologous limbal stem cell product).

*Newcastle Academic Health Partners is a collaboration that brings together world-class expertise to ensure patients benefit sooner from new treatments, diagnostics and prevention strategies.

Limbal Stem Cell Deficiency causes serious detrimental effects on productivity and quality of life. This innovative treatment aims to cure LSCD rather than just providing symptomatic relief.

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The AHSN NENC funding allowed us to examine every step of the process and ensure strict compliance with GMP manufacturing process which will lead to enhanced safety of stem cell transplantation for the patients
Professor Majlinda Lako, Newcastle University
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LSCD is a painful and blinding disease with marked patient morbidity.  Patients with severe LSCD require frequent visits to clinics including admission into hospital for intensive treatment. Most cases of LSCD affect young male patients with long life expectancies, thus causing serious detrimental effects on productivity and leisure activities. Corneal vascularisation and opacity have been estimated to cause blindness in 8 million people (ca.10% of total blindness) worldwide each year and this may cost as much as £150 billion per year. In cases of bilateral LSCD, most patients would be registered as severely sight impaired which requires social support for visual loss, making this a high cost disease with an enormous impact on a patient’s quality of life. The World Health Organisation estimates that one year with severe visual impairment is equivalent to the loss of 23 weeks of life in perfect health.

Therefore a curative treatment is sought which will eliminate the requirement for frequent clinical visits and restore sight (directly impacting on the person’s ability to work and increase their quality of life), as well as having significant benefits to the NHS.

Newcastle University and NUTH jointly developed a treatment for people with total unilateral LSCD. The procedure involves taking stem cells from the healthy eye via a small biopsy, reproducing them in a lab on human amniotic membrane before transplanting them back into the affected eye of the patient.

The aim of this project was to commercialise this Limbal Stem Cell product as a licensed medicine.

Funding was secured from the Academic Health Science Network for North East and North Cumbria (AHSN NENC) to support the commercialisation of the product. The Newcastle innovation team made significant progress with the regulators (European Medicines Agency – EMA) gaining scientific advice to help them understand how to achieve regulatory compliance for the product.

Whilst undertaking this project, the EMA granted a Marketing Authorisation to Chiesi Farmaceutici S.p.A for a similar product ‘Holoclar®’ to treat moderate to severe LSCD due to physical or chemical burns in adult eyes. Due to this development, a partnership has now been formed with Chiesi Farmaceutici S.p.A, to become a trial site for Holoclar®.

Newcastle will treat patients using its own product for people with indications that fall outside that of Holoclar®, including non- chemical/physical burns (under a ‘Specials’ manufacturing license).

To date, one LSCD patient has been treated under NUTH’s ‘Specials’ license and work is ongoing to treat eight additional patients. Funding for this treatment is being sought from NHS England.

  • Sight restored in patient treated with the Newcastle product.
  • One patent application filed relating to the manufacturing process, creating wealth generation opportunities.
  • Two jobs created (post-doctoral research scientist and GMP staff scientist).
  • Professional development opportunities for two healthcare professionals invited to participate on Industry Advisory Boards.
  • Publication in Experimental Eye Research to demonstrate steps required for ex vivo expansion of limbal stem cells under good manufacturing practice is under review.
  • Poster presented at the annual LSCD conference in Liverpool in 2016.
  • Strong collaborations developed between clinicians, scientists, Good Manufacturing Practice (GMP) manufacturers, regulators and industry representatives.
  • For patients – reduced morbidity, fewer hospital visits, improved quality of life, increased ability to engage in occupational life.
  • For the NHS the potential to create savings due to patient pathway changes.

The authors would also like to gratefully acknowledge financial support from the following organisations:

Awarding organisation: MRC UK (G0900879). Title: To evaluate the effectiveness of cultured human limbal epithelium for the treatment of limbal stem cell deficiency. Start date: 1.01.2010; End date: 31.12.2015.

Awarding organisation: BRC and Newcastle University (BH124190). Title of the project: Cultured human limbal stem cells translation program. Start date: 01.03.2014. End date: 28.02.2015.