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Q: Adult Stem Cell Therapy to cure Macular Degeneration specifically Stargardt. ( Answered,   0 Comments )
Subject: Adult Stem Cell Therapy to cure Macular Degeneration specifically Stargardt.
Category: Health > Conditions and Diseases
Asked by: ds1990-ga
List Price: $40.00
Posted: 05 Jun 2006 09:41 PDT
Expires: 05 Jul 2006 09:41 PDT
Question ID: 735450
Can you explain to me in laymans terms what progress is being made
using adult stem cells in the treatment of dry macular degeneration
specifically stargardt?  I would also like to have information
regarding how to go about being a part of the clinical trials.  I am a
52 year old healthy male residing in Texas.
Subject: Re: Adult Stem Cell Therapy to cure Macular Degeneration specifically Stargardt.
Answered By: crabcakes-ga on 05 Jun 2006 12:36 PDT
Hello Ds1990,

    One of the factors affecting in the slow progress of finding a
cure for Stargardt?s is the nature of the gene responsible for this
retinal disease. Some people with the gene never develop the disease,
while others seem to develop it without the gene. Environmental and
unknown factors play a role as well.

   ?In the future, when a patient manifests a genetic defect, doctors
hope to be able to correct it and resolve the problems it causes. Gene
therapy involves inserting a functioning gene into human cells to
correct a genetic error or to introduce a new function to the cells.
The surgeon must transplant genes rather than cells, a strategy which
miniaturizes the process by a million fold. The surgical tools are not
hand-held instruments, but trained viruses. At the 1998 conference of
the Association for Research in Vision and Ophthalmology, a report
presented just such a technique for retinitis pigmentosa (RP), another
retinal degenerative disease. Researchers showed that a virus called
Lenti could carry a new gene into mouse photoreceptors and could
prevent the photoreceptors from degenerating for six months in a mouse
model of recessive RP. That's a long time for a mouse! In another
experiment, an adenovirus was used to deliver a gene designed to
prevent the expression of an abnormal dominant gene for RP. Thus, it
appears that both dominant and recessive forms of RP are amenable to
gene therapy -- at least in mice! Before we can begin human trials of
gene therapy for retinal degenerations, the long term effects of these
viruses, including rejection, must be better understood. Like
transplantation, this approach requires delicate subretinal surgery to
place the virus near the photoreceptors and the RPE. Regrettably, gene
therapy to cure AMD is probably still more than a decade away.?

?There is much interest in stem cell research, because it holds the
promise of replacing defective cells with new ones. However,
scientists are just beginning to learn how to make such cells develop
into specific tissue, and making them replace several different layers
in a very complex structure like the retina is a challenge that will
take many years. So in the foreseeable future this is not a realistic
option for retinal diseases.? (More from this site is posted below)

  It appears that stem cell therapy for Stargardt?s has not yet
reached the clinical trial stage in humans, in the US or Canada. I
have posted some research in India a it further down the page.  Gene
therapy however, has, and with some successful results. Currently it
appears there are no ongoing trials in the US for other Stargardt?s
therapies either, but please check the following sites often:'s&spell=1&access=p&output=xml_no_dtd&ie=UTF-8&client=NEI_frontend&site=NEI&oe=UTF-8&proxystylesheet=NEI_frontend'S

Sign up for this newsletter to be advised of new developments:

   India, at the Rajender Prasad Centre for Opthalmic Sciences at
AIIMS has been involved in  stem cell research for Stargardt?s.

From Blind World Online Magazine
??? There has been some success with transplantation of neural stem
cells, but this approach is limited by political and ethical
controversies related to the use of embryonic stem cells, as well as
by cell rejection. So we are using cells derived from patient?s own
body,?? said Dr Kumar.

The stem cells derived in this manner are injected into a loose tissue
near the cornea. ??This is done by a fine 25 gauge needle (less that
0.1 mm), which is barely visible to the naked eye. A dose of 0.1 ml is
injected under local anaesthesia,?? the doctor said.

A subsequent dose of antibiotics is given so that the area doesn?t
develop infection following the injection. Next morning, the patient
is discharged. ??We do a follow-up after one month followed by checks
after three, six and 12 months,?? added Dr Kumar.?

? Dr Rajender Prasad Centre for Opthalmic Sciences at AIIMS has been
studying the effect of stem cells in patients who suffer from
degenerative vision disorders. The study is being conducted on fifty
patients who are severely affected by age-related macular degeneration
or retinitis pigmentosa.

    According to Dr Atul Kumar, Professor of Opthalmology and head of
the team undertaking the research, significant improvement has been
noticed in vision of the patients after one month of injecting stem
cells. There is further improvement after a gap of three months.

    The researchers are using autologous (derived from patient's own
body) bone marrow derived stem cells and injecting them into a loose
tissue near the cornea. Follow-ups are then done after one, three, six
and 12 months.?

Dr. Atul Kumar

Contact Information

    ?A Connecticut ophthalmologist has been reporting success from a
deceptively simple treatment on patients with Stargardt's disease and
other non-neovascular forms of retinal disease. Dr. Gerard Michael
Nolan has performed the treatment, called ECHO therapy, for nearly
three years on more than 200 patients at the Nolan Eye Clinic in
Farmington, Connecticut, and he is now working to locate support for
FDA-approved clinical trials.

Dr. Nolan, a diplomat of the American Board of Ophthalmology, is a
graduate of Georgetown Medical School and completed his residency at
Cornell Medical Center in New York. In a discussion with people from
the MD Support email support group in early October 2003, he said, "In
the course of my ophthalmic practice, I discovered that a topical drop
of dilute echothiophate (ECHO) can restore lost visual acuity in some
cases of chronic retinal disease. ECHO appeared to increase the
capability of the few surviving neurons, endowing this reduced
population with an enhanced stimulus potential. These effects need to
be studied in multi-center controlled clinical trials."

?But this isn't the 20th century anymore. This is the 21st century and
people don't take answers lying down. They get up and find out all
they can about something that affects them and then they take action.
For Pam Roberts, that meant searching day and night on the Internet,
until finally she discovered Dr. Gerard Nolan.

Dr. Nolan has an office in Connecticut and has discovered that by
treating Stargardt's with Echotiophate Iodide, he can restore blood
flow to his patients' eyes and they are able to see again. The
medicated drops have been approved by the Food and Drug Administration
as a treatment for glaucoma, but they have not been approved for
Stargardt's, which means insurance won't cover the price of the visit
to Dr. Nolan or the drops themselves.?

Dr. Gerard M. Nolan
428 Burnside Avenue,
East Hartford, Connecticut (CT)

This clinical trial is intended to assess the effectiveness of the
Implantable Miniature Telescope (IMT) to safely improve vision in
patients 55 years of age or older who have advanced AMD or Stargardt?s
macular dystrophy. Patients who have dry or disciform (scarred macula
after wet AMD) macular degeneration in both eyes may be eligible for
this study. Patients with wet AMD are not eligible. Candidate patients
must be able to attend all follow-up visits for this 2-year study,
including scheduled vision training appointments after the
implantation procedure to help adapt to the IMT and magnified image.
The IMT is a micro-sized precision telescope, about the size of a pea,
that is designed to magnify images onto the retina. By magnifying
images, it is hoped the blind spot caused by macular degeneration can
be reduced in size, allowing for better central vision and associated
function in daily activities. The IMT is implanted in one eye by an
ophthalmologist in an outpatient procedure. The implanted eye provides
magnified central vision. The non-implanted eye provides peripheral or
"side" vision for mobility and navigation. Since the IMT is implanted
inside the eye, natural eye movements are used to scan the environment
and reading materials. The enrollment for this trial is complete but
you can sign up for updates if a new trial opens    ? This link
was broken when I tried it!  Their home page does work!

The above trial is over, but you should contact Mr. Allen Hill for future trials:

Allen Hill, President and CEO
VisionCare Ophthalmic Technologies, Inc.
(408) 872-9393

More information on the above study:

About the telescope:?The IMT is a micro-sized precision telescope,
about the size of a pea, that is implanted in one eye in an outpatient
surgical procedure conducted under local anesthesia. The IMT provides
magnification of 3.0X or 2.2X, depending on the IMT model used. A
magnified image is projected over a wide field of the retina to
improve the ability to recognize images that were previously either
difficult or impossible to see. The IMT is implanted in one eye of
patients who present with untreatable late-stage AMD (end-stage AMD).
The telescope provides central vision, while the non-implanted eye
provides peripheral vision.?

   ?The other model system that the Hurwitz lab is studying is retinal
degeneration. The human diseases that exhibit retinal degeneration are
retinitis pigmentosa and macular degeneration. Mutations in the
proteins that make up the phototransduction cascade as well as
proteins that support the survival of photoreceptors have been found
to result in retinal degeneration in animal models as well as cause
human disease. The Hurwitz lab is currently studying the therapeutic
delivery of two photoreceptor proteins to diseased animals.

 The first uses a lentiviral vector to deliver the ABC4A protein, a
member of the ABCR transporter family and a protein responsible for
retinal transport. This protein is defective in juvenile macular
degeneration (Stargardt's Disease) and in some forms of autosomal
recessive retinitis pigmentosa. The second uses an adeno-associated
viral vector to deliver the PDE6 gene, the target enzyme of the
phototransduction cascade that is defective in some forms of autosomal
recessive retinitis pigmentosa. The ultimate goal of these studies is
to examine the toxicities of these agents and the eventual use of
different gene therapy techniques for the treatment of human ocular
Consider contacting Dr. Richard Hurwitz, a researcher in retinal
diseases, including Stargardt?s.

Trials are occasionally run by his research group

?Current research also shows that patients with Stargardt's disease
could slow its progression by wearing UV-protective sunglasses and
avoiding exposure to bright light. Researchers have observed that mice
which had a mutation of the ABCA4 gene, and which were reared in dark
environments had virtually no lipofuscin deposits.?

?Scientists have been able to develop mice that are missing the ABCA4
gene (so-called knockout mice), and are studying whether various drugs
or other interventions can slow or stop the accumulation of the
abnormal material in these mice. They have found that if you do not
expose the mice to light, the degeneration proceeds more slowly. It is
difficult to know how this applies to people, but some investigators
recommend that patients should try to wear sunglasses when they are
outdoors. They also found that giving the mice Accutane (a vitamin A
derivative used for severe acne that has serious side effects such as
birth defects and psychiatric side effects; do not use for yourself!)
slowed down the accumulation of the abnormal material. Research is
ongoing to find safe therapies that may help in this condition.

   ? Researchers are also studying ways to slow down cell death in
general. They are beginning a study, in patients with a severe type of
retinal degeneration, whether cell death can be slowed by growth
factors. They have made a small reservoir containing cells that have
been genetically engineered to make a growth factor. They will implant
this reservoir in the eye, and, like an internal factory, the growth
factor that is made will be able to diffuse out of the reservoir and
into the eye.?

? It is critical for any patient with blind spots in or near the
center of vision to learn how to move the eye so that the object of
interest is imaged onto a seeing part of the retina. Most Stargardt
patients do this quite well, and they tend to look slightly above what
they want to see. This has the effect of moving the blind spot up and
out of the way. The difficulty is that as one uses retina farther from
the center of the macula, larger letters are needed in order to see,
so that magnification using a variety of low vision devices can be
very helpful."

    "I have a special device called a scanning laser ophthalmoscope
(SLO), which can let us plot out the exact location of the blind spot
directly on a video image of the macula. This information helps us to
advise the patients on strategies for using the remaining seeing
retina in the most effective way. The SLO also allows us to do special
imaging of the macula to determine the stage and progression of the
condition. It also allows us to do autofluorescence imaging, which
helps to identify areas that have had a loss of cells underlying the

?Gene therapy shows promise for treating Stargardt disease
In a key step toward a clinical trial for gene therapy to treat
Stargardt disease in humans, FFB-funded investigators have developed a
safe and efficacious gene therapy for treating Stargardt disease in
mice. The research team used a lentivirus ? a manmade virus
specifically designed for gene delivery ? to get a normal human ABCR
(ABCA4) gene to the photoreceptors in mice lacking the gene, and
therefore served as a model for Stargardt disease in humans.?

Not stem cell therapy, but patients are being recruited for this
study: Effect of DHA Supplements on Macular Function in Patients with
Stargardt Macular Dystrophy and Stargardt-like Macular Dystrophy

This is the only current clinical trial for Stargardt:

   ?Clinical trials are not widely available. Generally speaking,
ophthalmology departments in large research hospitals tend to conduct
clinical trials. Some large, private ophthalmology practices also
participate in clinical trials. Because clinical trials have limited
availability, strict eligibility requirements, and most patients with
wet macular degeneration need to be treated quickly before further
vision loss occurs, it is not always possible to participate in a
clinical trial.?

?Recently, Foundation-supported scientists reported on the discovery
of stem cells and/or progenitor cells in the eyes of adult rodents.
These cells may confer even greater advantage in retinal cell
transplantation. Discovery of retinal progenitor cells in the eyes of
adult mammals has sparked intense scientific investigation to
determine what variety of cell types these cells are capable of
manufacturing, and whether progenitor cells can make the transition to
mature photoreceptor or RPE cells in the environment provided by the
adult eye.
Despite the presence of neural progenitor cells in the adult eyes of
mammals, these cells do not automatically repopulate the retina to
replace injured or diseased photoreceptor cells. In order to function
properly, transplanted stem cells and neural progenitor cells must
also be able to make connections with other nerve cells in the retina.
Extensive work is needed to identify the factors that lead to
successful integration of transplanted cells.?

?RPE Cell Transplants
RPE cell rescue transplants might prevent, halt or slow the progress
of the disease by transplanting healthy RPE cells, which support the
function of photoreceptor cells in the retina. Because RPE cells are
frequently implicated in macular degeneration, researchers think
transplanting these cells may offer a particularly effective treatment
for macular degeneration.
In Foundation-funded laboratory studies, RPE cell transplants have
delayed vision loss in an animal model. These important studies
provide ?proof of principle? that RPE cell transplants can delay
vision loss. Unfortunately, subsequent studies in humans found that
transplanted RPE cells evoke damaging immune responses.

Currently, Foundation researchers are working to overcome immune
response complications so that the effectiveness of this treatment can
be evaluated in clinical trials. For example, The Foundation is
collaborating with Oculex, a biotechnology company that has developed
a drug delivery device that slow-releases a steroid to prevent immune
complications after retinal cell transplantation. Such a device could
help elevate transplantation therapies to clinical trials.

Foundation researchers are collaborating with Neurotech
Pharmaceuticals to develop and test the use of ?immortalized? RPE
cells. These RPE cells have been genetically engineered to eliminate
characteristics that may irritate or stimulate the patient?s immune
system. In preliminary studies, transplanted immortalized RPE cells
prevented vision loss without invoking immune responses. Such cells
may offer a safe, abundant, disease-free source of donor cells for use
in transplantation.?

Stargardt patients sought:

You need to register (free) to read articles on Stargardt?s. I
registered, but have not yet received my log in information. I suggest
you register, if you have not already done so, to learn of recent

Another type of therapy:
?Micro Current Stimulation (MCS) therapy is a noninvasive procedure
which involves stimulating the retina and nerve fibers with very low
intensity electrical current using a FDA and CE Mark approved
electrical stimulation device. The current is delivered in the micro
Amp range at different electrical frequencies through electrodes
applied over closed eyelids. The treatment causes no discomfort or
pain and is administered for 12 minutes, twice each day. While a very
effective form of treatment, MCS therapy is not a cure for retinal
diseases and must be continued for the life of the patient. Overall,
no side effects or adverse reactions related to this procedure have
been observed.?

This is an excerpt from a longer article, which is a fee based article:
?Stephen P. Daiger 

Human Genetics Center, School of Public Health, and Department of
Ophthalmology and Visual Science, The University of Texas Health
Science Center, 1200
Herman Pressler Street, Houston, TX 77030, USA 
One of the great success stories in retinal disease (RD) research in
the past decade has been identification of many of the genes and
mutations causing inherited retinal degeneration. To date, more than
133 RD genes have been identified, encompassing many disorders such as
retinitis pigmentosa, Leber congenital amaurosis, Usher syndrome and
macular dystrophy. The most striking outcome of these findings is the
exceptional heterogeneity involved: dozens of
disease-causing mutations have been detected in most RD genes;
mutations in many different genes can cause the same disease; and
different mutations in the same gene may cause different diseases.
Superimposed on this genetic heterogeneity is substantial clinical
variability, even among family members with the same mutation. The RD
genes involve many different pathways, and expression ranges from very
limited (e.g. expressed in rod photoreceptors only) to ubiquitous.
These findings raise several general questions - in addition to the
extraordinary number of specific, biological problems revealed. What
fraction of the patient population can now be accounted for by the
known RD genes? How many more RD genes will be found, and how should
we find them? Are we dealing with just a handful of disease mechanisms
or are there many different routes to retinal degeneration? How will
this extreme heterogeneity affect our ability to diagnose and treat
patients? These questions are considered in this summary.?

You may purchase the entire article for $25 here:

Additional interesting reading:

Other investigational therapies

  When evaluating any ?new? therapy for Stargardt?s, please discuss
the process with your ophthalmologist first. Some trials require your
ophthalmologist?s recommendation for trial participation.

I hope this has helped you! If anything is unclear, please request an
Answer Clarification, and allow me to respond, before you rate. I?ll
ew happy to assist you further on this question, before you rate.

Sincerely, Crabcakes

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Retinal Diseases + Stargardt's + research + 2006
Stargardt?s + stem cell therapy
macular degeneration + latest research + 2006
Research + Stargardt?s + 2006
clinical trials + Echothiophate
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