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Stem Cells in Blindness Treatment
Done By:
Rawan Abdulwali Alakwaa
In this presentation:
 Stem cells and their types.
 The eye and blindness.
 Limbal stem cells and treatment of LSC Deficiency.
 The macula.
 Age Related Macular Degeneration and its types.
 AMD stem cell based treatment promise.
Stem cells
 Adult stem cells
 Embryonic stem cell
(ESCs)
 Induced pluripotent stem
cells (iPSCs)

Limbal Stem Cells
Limbal Stem Cell Deficiency
(LSCD)
 Limbal Stem Cell
Deficiency (LSCD) can
lead to corneal opacity and
neovascularization, with
consequent visual
impairment or blindness
due to loss of corneal
transparency.
LSCD stem cells based treatment
(Holoclar)
Limbal Stem Cell Deficiency
(LSCD) treatment (Holoclar)
 The Holoclar® system uses
stem cells to repair
moderate to severe limbal
stem cell deficiency.
 As small area of the
patient’s eye is biopsied for
this procedure, it does not
involve surgery.
Limbal Stem Cell Deficiency
(LSCD) treatment (Holoclar)
 Holoclar® gives a
therapeutic chance to
severe limbal stem cell
deficiency that involves
both eyes.
The Macula
Age related macular degeneration
(AMD)
 AMD is a common eye
condition and a leading
cause of vision loss among
people age 50 and older.
Age related macular degeneration
(AMD)
 As AMD progresses, a blurred
area near the center of vision
is a common symptom 
grows larger or you may
develop black spots in your
central vision. Objects also
may not appear to be as
bright as they used to be.
AMD cause
Types of age related macular
degeneration
Treatment of AMD using stem cells
Treatment of AMD: RPE replacement
RPE replacement (clinical trials)
 The safety of embryonic stem cell-derived RPE cells
has been tested in phase I/II clinical trials for
patients with Stargardt’s macular dystrophy, and for
those affected by AMD by a stem cell biotech
company called Advanced Cell Technologies.
RPE replacement (clinical trials)
 The results of the trial, published in 2014,
demonstrated:
 Safety and showed engraftment of the transplanted
RPE cells.
 Some participants experienced adverse side effects from
the immunosuppression and the transplantation procedure
itself.
 Several patients also reported an improvement in
vision.
RPE replacement (clinical trials)
 A second Phase I/II trial exploring the use of RPEs
derived from human embryonic stem cells for
people with wet AMD is currently underway in the
United Kingdom. The first patient received their
transplant in September 2015. This work, led by
Prof Pete Coffey, is ongoing and is being carried out
at Moorfields Eye Hospital as part of the London
Project to Cure Blindness.
 Finally, Japanese
researcher, Dr Masayo
Takahashi is leading a
clinical trial in Japan
which transplants RPE
cells made from iPS cells
into patients with wet
AMD. and many await the
results.
AMD treatment: replacing retinal
cells
 Several studies have
now reported that both
embryonic stem cells
and iPS cells can be
turned into different
types of retinal cells in
the lab.
 Unlike RPE cell transplantation, direct repair of the
retina may allow patients who have already lost their
vision to have it restored to some degree.
 This gives hope for patients with disorders like late-
stage age-related macular degeneration, where the
light-sensitive photoreceptor cells in the retina have
already been lost.
AMD treatment: replacing retinal
cells
 This type of research may also provide new
treatments for people who suffer from retinal
diseases like retinitis pigmentosa and glaucoma.
 However, despite encouraging evidence, such
research is very much in its infancy. There are
currently no patient clinical trials planned using this
type of approach, as significant further research is
still required first.
AMD treatment: replacing retinal
cells
Stem cells are not a one-stop, generic cure, but
they do hold exciting potential for the
production of new biological components that
can be used to repair the eye.
THANK YOU
Done By:
Rawan Abdulwali Alakwa’a

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Stem Cells in Blindness Treatment

  • 1. Stem Cells in Blindness Treatment Done By: Rawan Abdulwali Alakwaa
  • 2. In this presentation:  Stem cells and their types.  The eye and blindness.  Limbal stem cells and treatment of LSC Deficiency.  The macula.  Age Related Macular Degeneration and its types.  AMD stem cell based treatment promise.
  • 3.
  • 4. Stem cells  Adult stem cells  Embryonic stem cell (ESCs)  Induced pluripotent stem cells (iPSCs)
  • 5.
  • 6.
  • 7.
  • 8.
  • 10. Limbal Stem Cell Deficiency (LSCD)  Limbal Stem Cell Deficiency (LSCD) can lead to corneal opacity and neovascularization, with consequent visual impairment or blindness due to loss of corneal transparency.
  • 11. LSCD stem cells based treatment (Holoclar)
  • 12. Limbal Stem Cell Deficiency (LSCD) treatment (Holoclar)  The Holoclar® system uses stem cells to repair moderate to severe limbal stem cell deficiency.  As small area of the patient’s eye is biopsied for this procedure, it does not involve surgery.
  • 13. Limbal Stem Cell Deficiency (LSCD) treatment (Holoclar)  Holoclar® gives a therapeutic chance to severe limbal stem cell deficiency that involves both eyes.
  • 14.
  • 16. Age related macular degeneration (AMD)  AMD is a common eye condition and a leading cause of vision loss among people age 50 and older.
  • 17. Age related macular degeneration (AMD)  As AMD progresses, a blurred area near the center of vision is a common symptom  grows larger or you may develop black spots in your central vision. Objects also may not appear to be as bright as they used to be.
  • 19. Types of age related macular degeneration
  • 20.
  • 21. Treatment of AMD using stem cells
  • 22. Treatment of AMD: RPE replacement
  • 23. RPE replacement (clinical trials)  The safety of embryonic stem cell-derived RPE cells has been tested in phase I/II clinical trials for patients with Stargardt’s macular dystrophy, and for those affected by AMD by a stem cell biotech company called Advanced Cell Technologies.
  • 24. RPE replacement (clinical trials)  The results of the trial, published in 2014, demonstrated:  Safety and showed engraftment of the transplanted RPE cells.  Some participants experienced adverse side effects from the immunosuppression and the transplantation procedure itself.  Several patients also reported an improvement in vision.
  • 25. RPE replacement (clinical trials)  A second Phase I/II trial exploring the use of RPEs derived from human embryonic stem cells for people with wet AMD is currently underway in the United Kingdom. The first patient received their transplant in September 2015. This work, led by Prof Pete Coffey, is ongoing and is being carried out at Moorfields Eye Hospital as part of the London Project to Cure Blindness.
  • 26.  Finally, Japanese researcher, Dr Masayo Takahashi is leading a clinical trial in Japan which transplants RPE cells made from iPS cells into patients with wet AMD. and many await the results.
  • 27. AMD treatment: replacing retinal cells  Several studies have now reported that both embryonic stem cells and iPS cells can be turned into different types of retinal cells in the lab.
  • 28.  Unlike RPE cell transplantation, direct repair of the retina may allow patients who have already lost their vision to have it restored to some degree.  This gives hope for patients with disorders like late- stage age-related macular degeneration, where the light-sensitive photoreceptor cells in the retina have already been lost. AMD treatment: replacing retinal cells
  • 29.  This type of research may also provide new treatments for people who suffer from retinal diseases like retinitis pigmentosa and glaucoma.  However, despite encouraging evidence, such research is very much in its infancy. There are currently no patient clinical trials planned using this type of approach, as significant further research is still required first. AMD treatment: replacing retinal cells
  • 30. Stem cells are not a one-stop, generic cure, but they do hold exciting potential for the production of new biological components that can be used to repair the eye. THANK YOU Done By: Rawan Abdulwali Alakwa’a