Blindness Cured By Stem Cells

By Bera Te.
Published: June 23, 2010
Related

Yes that’s right, no miracle prayers, no miracle cures. Stem cells has cured people who were blinded or
suffered tremendous damage to their eyes by chemicals[such as the eye above]. In the Italian-led
research-treatment, 96 out of 107 patients were completely or partially cured of their blindness according
to the New England Journal of Medicine.

The stem cells (‘borrowed’ from the patients’ healthy eyes) were first multiplied in the lab which were then
transplantedinto the damaged eye(s) which then grew into new tissue fixing the damage. Furthermore, since
the cells were taken from the patients themselves, no drugs were required for their body system to forcefully
accept the transplants.

The results were not immediate, it took several months for those who underwent the transplants and even
longer for those who had even more damage done to their eye. Still, the patients’ visions were restored.

The use of stem cells by reversing blindness is relatively new while stem cells used to cure diseases like
leukemia have been in practice for quite a time now.

Though this is an excellent breakthrough, stem cells transplants in this manner cannot assist patients who
have taken damage to their optic nerve. Doctors say that completely blind people cannot be cured by this
process because the stem cells need to come from one healthy eye in order to have the cells grow into new
tissue.

This study, funded by also the Italian government, had doctors treat the one hundred and six patients
between since the late 1990?s. In describing the reaction of the cured patients, according to AP, the
University of Modena’s Center for Regenerative Medicine’s Head of Cell Therapy Graziella Pellegrini
described the patients as being “incredibly happy”.
Stem Cell Technique Restores Sight to Burn-Injured Eyes
Lab-grown graft provides 'biological contact lens' for cornea, experts say.

By Randy Dotinga
HealthDay Reporter

WEDNESDAY, June 23 (HealthDay News) -- A new Italian study describes a technique that doctors can
use to restore vision to some patients with severe burns to their eyes.

The therapy restores the burned cornea -- the outermost layer of the eye -- with a graft of healthy stem
cells grown in the laboratory.

The treatment "is like putting on a biological contact lens," said Dr. Stephen Pflugfelder, a practicing
corneal specialist and professor of ophthalmology at Baylor College of Medicine in Houston, who says
the technique works well.

A clear cornea -- essential to good vision -- "is the clear window on the eye," like a watchglass on a
watch, added Pflugfelder, who was not involved with the Italian study but is familiar with its findings. The
results are published online June 23 in the The New England Journal of Medicine.

In all eyes, the ultra-thin cornea is vulnerable because it doesn't have much protection. Burns can
severely damage the cornea and rob a person of sight. The burns can come from firecrackers, spills of
boiling liquid, battery acid, industry chemicals and even eggs that explode into people's faces when they
open the doors of microwaves.

Burns to the cornea from fires are less common, perhaps because burns severe enough to harm the
cornea are often fatal, Pflugfelder said.

The prognosis is generally good for patients with minor burns to the cornea. But deeper burns damage
the eye's limbal stem cells -- cells with the ability to transform into many types of cells -- and can cause
severe glaucoma, he said.

With the exception of stem cell treatments, "the patients have almost no alternatives, or very poor
alternatives," said study co-author Graziella Pellegrini, head of the cell therapy unit at the Center for
Regenerative Medicine at the University of Modena and Reggio Emilia in Modena, Italy.

Existing treatments for severe burns involve taking stem cells from the patient's healthy eye, if he or she
has one, or from the eyes of a relative or another person. A doctor transfers the stem cells to the burned
eye, where they may begin creating new cells that allow the cornea to work properly again.

The treatment described in the study takes a slightly different approach. Researchers coaxed the eye's
limbal stem cells to reproduce in a lab culture before implanting them in the damaged corneas of 112
patients.

"By the time [researchers] put them on the damaged cornea, the stem cells can cover the eye,"
Pflugfelder said.

The study reports that the treatment was successful in 76.6 percent of the patients and partially
successful in 13.1 percent. (Success was defined as an absence of all symptoms and the permanent
restoration of a renewing, transparent cornea.) In 10.3 percent of the patients, the treatment did not work,
in part due to surgical complications or the severity of injuries.

About half of those with deep damage to the eye recovered vision, while all of the rest did, Pellegrini
said.

A participant in the Italian study described as Patient 93, for example, had such poor vision after an acid
burn to his eye that he could only see with it well enough to count his fingers; a graft the researchers
developed from limbal stem cells restored normal vision.

A graft of limbal stem cells also restored the corneal surfaces of Patient 24 -- who had had severe burns
to both eyes -- and improved his vision, the study noted.

Interestingly, the procedure was successful in some patients who had been burned years before entering
the study. Patients 22, 26 and 46, for example, were nearly blind in one of their eyes, with completely
opaque corneas, after suffering alkali burns; they had undergone unsuccessful surgery, respectively, 13,
30, and 3 years before admission to the study. The researchers were able to restore a stable corneal
covering to all three, with patients 22 and 46 regaining complete visual acuity, the study reported.

At about $20,000-$30,000, the treatment is relatively inexpensive, Pflugfelder said. It's not clear how
long the treatment will last before it has to be repeated, although some patients in this study did well for
as long as 10 years.

Currently, this specific stem cell treatment is not approved for use in the United States, Pflugfelder said,
although it is performed in other countries.

More information

For more about the cornea, visit the U.S. National Library of Medicine.

(SOURCES: Stephen Pflugfelder, M.D., professor of ophthalmology and director, Ocular Surface Center,
Baylor College of Medicine, Houston; Graziella Pellegrini, Ph.D., head, Cell Therapy Unit, Center for
Regenerative Medicine, University of Modena and Reggio Emilia, Modena, Italy; July 23, 2010, "Limbal
Stem-Cell Therapy and Long-Term Corneal Regeneration," The New England Journal of Medicine.)
Limbal Stem-Cell Therapy and Long-Term Corneal Regeneration
Paolo Rama, M.D., Stanislav Matuska, M.D., Giorgio Paganoni, M.D., Alessandra Spinelli, M.D., Michele
De Luca, M.D., and Graziella Pellegrini, Ph.D.

ABSTRACT

Background Corneal renewal and repair are mediated by stem cells of the limbus, the narrow zone between
the cornea and the bulbar conjunctiva. Ocular burns may destroy the limbus, causing limbal stem-cell
deficiency. We investigated the long-term clinical results of cell therapy in patients with burn-related corneal
destruction associated with limbal stem-cell deficiency, a highly disabling ocular disease.

Methods We used autologous limbal stem cells cultivated on fibrin to treat 112 patients with corneal damage,
most of whom had burn-dependent limbal stem-cell deficiency. Clinical results were assessed by means of
Kaplan–Meier, Kruskal–Wallis, and univariate and multivariate logistic-regression analyses. We also
assessedthe clinical outcome according to the percentage of holoclone-forming stem cells, detected as cells
that stain intensely (p63-bright cells) in the cultures.

Results Permanent restoration of a transparent, renewing corneal epithelium was attained in 76.6% of eyes.
The failures occurred within the first year. Restored eyes remained stable over time, with up to 10 years of
follow-up (mean, 2.91±1.99; median, 1.93). In post hoc analyses, success — that is, the generation of normal
epithelium on donor stroma — was associated with the percentage of p63-bright holoclone-forming stem
cells in culture. Cultures in which p63-bright cells constituted more than 3% of the total number of clonogenic
cells were associated with successful transplantation in 78% of patients. In contrast, cultures in which such
cells made up 3% or less of the total number of cells were associated with successful transplantation in only
11% of patients. Graft failure was also associated with the type of initial ocular damage and postoperative
complications.

Conclusions Cultures of limbal stem cells represent a source of cells for transplantation in the treatment of
destruction of the human cornea due to burns.

Source Information

From San Raffaele Scientific Institute, Ophthalmology Unit, Milan (P.R., S.M., G.P., A.S.); and the Center
for Regenerative Medicine Stefano Ferrari, University of Modena and Reggio Emilia, Modena, Italy (M.D.L.,
G.P.).

This article (10.1056/NEJMoa0905955) was published on June 23, 2010, at NEJM.org.

Address reprint requests to Professor Pellegrini at the Center for Regenerative Medicine Stefano Ferrari,
University of Modena and Reggio Emilia, Via Glauco Gottardi 100, Modena, Italy, or at
graziella.pellegrini@unimore.it.