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New Assessment Tool Shows Potential Of Stem Cells In Restoring LSCD Patients' Sight: AlphaMed

1/17/2014 9:05:47 AM

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Durham, NC – A new assessment tool is helping scientists determine which treatments might benefit patients with a type of eye disorder called limbal stem cell deficiency (LSCD). The tool, developed by researchers at University College London and Moorfields Eye Hospital in London and funded by the UK’s National Institute for Health Research Biomedical Research Centre at these institutions, has already shown that the majority of these patients can benefit in the short term from a stem cell transplantation and up to 30 percent are still experiencing better sight three years later, according to the study published in the current issue of STEM CELLS Translational Medicine.

LSCD is an eye disorder in which the stem cells responsible for forming the surface skin of the cornea are destroyed by injury or disease. This results in pain, loss of vision and a cosmetically unpleasant appearance. Many new treatments, including limbal stem cell transplants, are emerging for this condition but their effectiveness remains to be proven.

“Assessing how well they perform has been severely hampered by the lack of biomarkers for LSCD and/or validated tools for determining its severity,” said Alex Shortt, M.D., Ph.D., of University College London’s Institute of Ophthalmology and lead investigator in the study. “In virtually all studies of limbal stem cell transplantation to date the clinical outcome has been assessed subjectively by the investigating clinician. This is clearly open to significant measurement and reporting bias.”

His team’s aims, then, were to design and test the reliability of a new tool for grading LSCD, to define a set of core outcome measures to use in evaluating treatments and to demonstrate the treatments’ impact on two common types of LSCD: a genetic disorder called aniridia and Stevens-Johnson syndrome (SJS), an inflammatory disorder.

They began developing an assessment tool by paring down a list of clinical signs taken from previously published studies to four key LSCD indicators: corneal epithelial haze, superficial corneal neovascularization, corneal epithelial irregularity and corneal epithelial defect. A standardized grading plate was then produced for each of these parameters, ranging from normal to severe. They named their assessment method the Clinical Outcome Assessment in Surgical Trials of Limbal stem cell deficiency [COASTL] tool and validated its performance in 26 patients with varying degrees of LSCD.

Once they had the COASTL tool in place, they used it to evaluate treatment outcomes in 14 patients with aniridia or SJS. All had undergone a limbal epithelial transplantation (allo-CLET), using cells taken from a deceased donor, cultivated in the lab before being transplanted into the recipient.

“The COASTL tool showed that following allo-CLET there was a decrease in LSCD severity and an increase in visual acuity up to 12 months post-treatment, but thereafter LSCD severity and visual acuity progressively deteriorated,” Dr. Shortt said. “However, despite a recurrence of clinical signs, the visual benefit persisted in 30 percent of aniridic and 25 percent of SJS patients at 36 months.

“A reliable method of obtaining objective outcome data for surgical trials of limbal stem cell deficiency will greatly contribute to the effective evaluation of current and new treatments,” said Anthony Atala, M.D., editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.

The full article, “Three-Year Outcomes of Cultured Limbal Epithelial Allografts in Aniridia and Stevens-Johnson Syndrome Evaluated Using the Clinical Outcome Assessment in Surgical Trials Assessment Tool,” can be accessed at

About STEM CELLS Translational Medicine: STEM CELLS TRANSLATIONAL MEDICINE (SCTM), published by AlphaMed Press, is a monthly peer-reviewed publication dedicated to significantly advancing the clinical utilization of stem cell molecular and cellular biology. By bridging stem cell research and clinical trials, SCTM will help move applications of these critical investigations closer to accepted best practices.

About AlphaMed Press: Established in 1983, AlphaMed Press with offices in Durham, NC, San Francisco, CA, and Belfast, Northern Ireland, publishes two other internationally renowned peer-reviewed journals: STEM CELLS® (, celebrating its 32nd year, is the world's first journal devoted to this fast paced field of research. The Oncologist® (, also a monthly peer-reviewed publication, entering its 19th year, is devoted to community and hospital-based oncologists and physicians entrusted with cancer patient care. All three journals are premier periodicals with globally recognized editorial boards dedicated to advancing knowledge and education in their focused disciplines.

About the NIHR: The National Institute for Health Research (NIHR) is funded by the Department of Health to improve the health and wealth of the nation through research. Since its establishment in April 2006, the NIHR has transformed research in the NHS. It has increased the volume of applied health research for the benefit of patients and the public, driven faster translation of basic science discoveries into tangible benefits for patients and the economy, and developed and supported the people who conduct and contribute to applied health research. The NIHR plays a key role in the Government’s strategy for economic growth, attracting investment by the life-sciences industries through its world-class infrastructure for health research. Together, the NIHR people, programmes, centres of excellence and systems represent the most integrated health research system in the world. For further information, visit the NIHR website ( NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology is a partnership between Moorfields Eye Hospital NHS Foundation Trust and the UCL Institute of Ophthalmology. Established in April 2007, its purpose is to conduct 'translational research' that is designed to take advances in basic medical research from the laboratory to the clinic, enabling patients to benefit more quickly from new scientific breakthroughs. Our centre is currently one of 12 biomedical research centres that were awarded in 2007 to NHS/university partnerships with an outstanding international reputation for medical research and expertise, and experience of translating that research into the clinical setting. For further information, please visit

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