NEI at 50: Into the Future

[On-screen] The National Eye Institute: Celebrating 50 years of advances in vision research [On-screen] Into the future [Dr. Andrew Huberman] Right now we’re on
the cusp of delivering on a promise which we’ve heard about for a long time. [Dr. Terri Young] We are seeing therapies
now that we only dreamed about 20 years ago or 10 years ago. [Dr. Paul Sieving] The future looks very promising. [Dr. Paul Sieving] The
Eye Institute launched the Audacious Goals Initiative. We wanted to develop a moon-shot project that
would have a major impact on restoring vision. We began by calling together several hundred
vision scientists and we placed before them the task of developing an ambitious target. What they responded with was the idea of restoring vision to the eye that had lost cells. Primary examples are photoreceptor cells and
retinal ganglion cells. The NEI Audacious Goals Initiative is developing
regenerative medicine to restore these cells and restore vision, at least partially for people who have already lost some of their sight. [On-screen] Neuroregeneration [Dr. Andrew Huberman] I’ve spent my entire
adult life working on the visual system, in particular the retinal ganglion cells or the
cells that connect the eye to the brain. The work that we’re doing specifically for
the AGI effort is to try and understand how retinal ganglion cells can be regenerated
after injury. We’re asking whether or not neural activity
and gene therapy and growth factor therapy can be used in humans in order to save retinal
ganglion cells that are sick and that might otherwise die as well as to regenerate connections
between the eye and the brain. And the way we’re doing that in particular
is using tools like virtual reality to trigger the activity of the ganglion cells and very
specific ganglion cells, and in particular the ones that are sick, to see whether or
not that can recreate connections with the brain that are lost or slowly dying back. [On-screen] Gene therapy [Dr. Andrew Huberman] In the last ten years
in particular there’s been a absolute explosion of new and exciting techniques brought into
study the nervous system, in particular the visual system. [Dr. Gerald Chader] Let me give you one example:
gene therapy. Gene therapy, of course, is replacing the
mutated gene by a normal gene. [Dr. Andrew Huberman] A gene can be introduced
to the eye by packaging it in a virus. Typically we think of viruses as a bad thing,
cold virus, flu virus, because they make us feel bad, but there are some viruses which
can carry cargo with it. In the laboratory one can construct
one of these viruses with any kind of cargo that you want. A cargo for, say, growth to keep cells that
are sick in the eye from disappearing and shrinking. And so you can take that virus with this cargo,
this cell growth gene if you will, inject it into the eye and then some of the cells
within the eye will get infected by the virus, but again to make them healthier, not to make
them sicker. [Dr. Paul Sieving] Gene therapy is not just
theoretical—it is actually being used to treat eye disease in children. The breakthrough came in 2008 when gene therapy
was successful for treating childhood blindness, termed Leber Congenital Amaurosis, or LCA. This treatment reinvigorated the entire field
of gene therapy for all of medicine. What we learned through vision research can
now be extended to many diseases beyond the eye alone. [Dr. Andrew Huberman] It’s really exciting
to think about the eye and the Eye Institute funded programs as one of the first places
where gene therapy is actually being applied in humans. [On-screen] Breakthroughs happen where disciplines meet [Dr. Paul Sieving] NEI-funded researchers
work at the NIH and at institutions across the country. They advance discoveries, technologies and
treatments to address blinding diseases while they also create jobs and contribute to their
local economy. [Dr. Kapil Bharti] Before AGI there were different
labs doing their own things. There were some labs doing basic science research. There were some labs trying to translate into
clinic. But I think what AGI did is brought them all
together. Breakthroughs happen definitely at the crossroads
of different disciplines. I think you could not do any of these things
if you did not merge different disciplines. My specific research is that we’re trying
to treat the disease called age related macular degeneration. [Dr. Sheldon Miller]Age-related macular degeneration,
or AMD, significantly reduces the vision of millions of individuals. [Dr. Paul Sieving] AMD causes the retinal
pigment epithelium cells inside the back of the eye to die. In turn, this causes subsequent loss of central vision
for driving and reading. Kapil Bharti is working with adult-derived
stem cells [Dr. Sheldon Miller] prepared from the patient’s
own skin or blood. [Dr. Kapil Bharti] So what we have done in
the lab is— it started off for many of the labs around the world that take patient-specific
IPS cells, induced stem cells, and convert them into this layer of cells, the pigment
epithelium, in the lab, grow it as a tissue and take the entire tissue and put it back
into the patient’s eye before the photo receptors die off and with the hope that the
transplanted epithelium would support now the photo receptors and protect them from
dying. [Dr. Sheldon Miller] This is a very promising
approach and intended to slow or reverse the progress of this disease and the clinical
trial is expected to begin early next year 2018. [Dr. Kapil Bharti] So I would definitely say
it’s a hopeful time for those patients and we’re going to bring them a therapy that
they really, really need. [Dr. Sheldon Miller] There are many interesting
and important advances that are currently in progress. All of these efforts together hold great promise
for improving public health and providing safe and therapeutically beneficial interventions
against the diseases that annually affect millions of people. [On-screen] To learn more, visit


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