NEI launches iPS cell therapy trial for dry AMD

(Music) AMD, age-related macular degeneration, has a late stage called dry AMD. Patients go blind because the retina cells and.

(Music) AMD, age-related macular
degeneration, has a late stage called dry AMD. Patients go blind because the
retina cells and the photoreceptor cells, which
perceive light and convert it into electrical
signals, die off. So the way the eye sees
is it’s like a camera. The light passes through the
lens, through the front of the eye, hits the back of the eye,
the retina, and the cells in the retina called the photoreceptors
that collect the light, converts it into electrical
signals sent through the retina and the optic nerve to the
brain, so we can see. RPE, retinal pigment epithelium,
is a layer of cells that in a healthy eye nourishes and
supports the photoreceptors. But in AMD patients, RPE cells
degenerate and die off, and, as a result, photoreceptors lose
their nourishment and they die off and patients go blind. The types of people affected by
AMD are mostly older people. It starts manifesting over
the age of 60, 65, and then as age progresses,
it keeps getting worse. Above the age of 80, it’s
thought that one in four, one in five people
are affected by it. Symptoms of AMD start
off as spotty vision in the central part of your vision,
and sometimes blurry and wavy vision, but as
the disease progresses, people really lose a big
chunk of their central vision and go blind in the central
part of their vision. The procedure that we have
developed in this new clinical trial is actually aimed to
replace patients’ degenerated tissue. We have been able to make
healthy RPE cells from patients’ own stem cells, which, when
engrafted into the back of the eye, will replace that
degenerated layer. Once it integrates with
patients’ surrounding tissue, it will start supporting the
photoreceptors. It may halt and reverse
progression of disease and, as a result, it’s going to
restore patients’ vision. So this new treatment is
different from a previous treatment in
many different ways. One is that in the previous
treatments, people have just injected cells, and these
cells don’t work very well if they’re just injected. And second, people have
used embryonic stem cells. We are using patients’ own stem
cells, so they are more autologous to the patient’s own
body cells, so they won’t be immune-rejected by
a patient’s body. Third, our cells are on a
biodegradable scaffold, so they would have a much
higher chance of integrating in the back of the eye. Creating one RPE
patch for a given patient takes almost six months. We start with patient blood, we
program that into iPS cells, or induced pluripotent stem
cells, and these cells can make any cell type of the body. So we take these cells and
make RPE cells out of it and take those RPE cells. We grow an RPE patch from
these cells on a scaffold. We had to optimize the fiber
thickness, the hole diameter and everything so the cells
would form a monolayer on top of our scaffold. To transfer this RPE patch under
the patient retina, we pick up the patch in one orientation and
bring it to the back of the eye, guide the patch under
the retina as the retina is made ready for
transplantation. The scaffold that we’re
using is biodegradable. It allows the cells to secrete
their own matrix, the natural matrix that
they like to grow on, and the scaffold keeps degrading
and disappearing over time. So the cells would integrate on
top of their own matrix in the back of the eye with the
surrounding tissue. So one of the main hopes for us
is, for this phase 1 trial, is to demonstrate that
one can do clinical trials with patients’ own iPS cells. And then we can demonstrate that
a patient’s own RPE patch can be delivered safely to the back of
the eye, can stay and integrate safely to the back of the eye, and hopefully start
functioning over time. Patients are really the
champions of this trial because as you know, this is a phase 1
safety trial and patients have to commit to this
experimental procedure. So all their efforts are for
altruistic reasons, because this may one day
affect and one day help save the vision of
thousands of patients. (Music)

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