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Foundation News » Other Retinal Diseases
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Researchers Developing Innovative Gene Therapy for Cone-Rod Dystrophy
In a mouse study, a Foundation-funded team at the University of Utah successfully halted retinal degeneration.
VisionWalk: $30 Million and Counting
Launched eight years ago, the Foundation’s 5K fundraising program reaches a hard-earned milestone while it continues to gain strength.
Drug Restores Vision in Retina without Functional Photoreceptors
Successful in a mouse model, the Foundation-funded treatment shows promise for eventually working in humans.
Vision-Saving Research Highlighted at Foundation’s Orange County Symposium

The event also focused on ways in which academics, venture capitalists and companies are collaborating to offer treatments for retinal diseases.

Aerobic Exercise Preserves Vision in Retinal Degeneration Lab Study
Researchers are hopeful that the low-cost, non-invasive intervention will prove effective in humans as well.
U.K. Company Receives $16 Million to Advance Choroideremia Gene Therapy
Called Nightstar, the company will eventually provide commercial support for treatments targeting a variety of diseases.
Vision Improvements Reported in Choroideremia Gene Therapy Clinical Trial

Launched in 2011, the U.K.-based study will soon be followed by two others focused on choroideremia—one in Canada, the other in the United States.

Canadian Team to Conduct Clinical Trial for Choroideremia Gene Therapy

Because the disease begins as a biomedical disorder, treating it relatively early in a person’s life could prevent vision loss altogether.

Spark Therapeutics Receives $50 Million to Advance Gene Therapies

A spinoff company of the Children’s Hospital of Philadelphia, it will move a landmark LCA treatment and other therapies toward commercialization.

The Foundation Invests $2.1 Million in Seven New Research Efforts

Among the projects, awarded by the Scientific Advisory Board, are those targeting specific as well as multiple diseases.

Gene Therapy for LCA Caused by RD3 Mutations Performs Well in Lab Study

Moving a step closer to treating humans, the therapy was administered with a delivery system designed by a Foundation-funded researcher.

Researchers Identify Better Virus for Retinal Gene Delivery
The new virus can penetrate most cells in the retina through a less-invasive injection.
Progress Reported in Gene Therapy Development for Best Disease

A study conducted by a Foundation-funded research team at the University of Pennsylvania serves as stepping stone to a clinical trial.

Retinal Symposium Focuses on Research and Financial Partnerships for Advancing Therapies

The Foundation-hosted event brought together 150 venture capitalists, drug company representatives, researchers and people affected by retinal diseases.

Bone Marrow Stem Cells in Clinical Trial for Retinal Diseases
Extracted from the hip bone, the cells may be able to heal tissue in people who have lost vision due to loss of blood flow. 

Stem Cell Pioneers Creating Retinal Patch to Restore Vision
The Foundation-funded project has the potential to help people affected by a variety of retinal diseases.

Six Emerging Retinal Therapies Receive $3.1 Million in Foundation Funding

Backed by a truly collaborative effort, each of the cutting-edge projects has the potential to eventually move into a clinical trial.

Increasing Blood Vessel Permeability to Get Treatments to the Retina

In a lab study, Foundation-funded researchers identified a potential way to help vision-saving treatments reach their intended destinations.

Stem Cells Restore Vision in Lab Study of Retinal Disease

The results will help researchers design potential treatments for people affected by a variety of vision-robbing conditions.

Scientists Report Significant Advancements in Clinically Focused Retinal Research

Gene therapies, stem cell treatments and vision-preserving plant extracts were among the subjects covered by participants in the Foundation’s annual TRAP meeting in Las Vegas.

Patients’ Skin Cells Help Researchers Move Closer to Treatments for Best Disease

A Foundation-funded study enabled a team led by Dr. David Gamm to understand critical elements of the disease process.

Researchers Measure Retinal Function Through Vision Map of the Brain

In the future, the technique could help determine changes in vision and the effects of treatments.

Foundation Invests $2.4 Million in Eight New Sight-Saving Research Projects

Aimed at providing treatments and identifying causes, the projects have the potential to help those with LCA, AMD, RP and Usher syndrome, among other diseases.

New Gene Linked to Blindness in Children

A Foundation-funded research collaboration has identified a new gene responsible for Leber congenital amaurosis, a devastating genetic form of blindness in newborns.

Chemical Restores Vision in Lab Study

In the early stages of a study involving an animal model, small amounts of what is known as AAQ enabled subjects to respond to light.

New Study Reveals That One Exposure to Nicotine Reduces Retinal Function

Researchers found that the equivalent of one or two cigarettes’ worth can adversely affect otherwise healthy eyes.

FDA Meeting Helps Pave the Way for Clinical Trial of Cross-Cutting Gene Therapy

The Foundation and its partner RetroSense Therapeutics continue to push an optogenetic approach to treating various diseases.

ARVO Delivered Good News from Several Retinal Treatment Clinical Trials

More than 12,000 eye experts attended the Association for Research in Vision and Ophthalmology’s annual conference in Fort Lauderdale.

Part of the Retina May Have the Potential for Self Repair

A recent study shows that, for some retinal diseases, the retina may have the potential to heal itself.

Healthy, Happy Living May Slow Retinal Degeneration

A recent study suggests that exercise, stress reduction and social activity have a positive effect on vision.

Charles Bonnet Syndrome Causes Blind People to "See Things"

A recent report in an ophthalmology journal highlights a surprising, and sometimes unsettling, experience for people who have lost their vision.

Generous Foundation Supporters Fuel the Fight Against Blindness
A handful of recent contributions showcase donors’ commitment to funding research focused on saving and restoring sight.

Vision Testing and Retinal Imaging in Clinical Trials: What Patients Can Expect

As more people are recruited for human studies, the demand for measuring the ways in which the eyes respond to treatments increases.

Researchers Move Closer to Developing Transplantable Retinal Tissue from Stem Cells

Dr. David Gamm and his team are developing vision-saving techniques that, just a few years ago, weren’t even on the Foundation’s radar screen.

Drug for RP and LCA Continues to Perform Well in Clinical Trial

Many of the patients in the study, being conducted by QLT, Inc., report improvement in visual acuity and the size of their visual fields.

Foundation Commits $2 Million to Development of a Cross-Cutting Drug Treatment

The start-up company MitoChem Therapeutics has identified three compounds that could benefit people affected by a variety of retinal diseases.

Second Eyes Treated Successfully in LCA Gene Therapy Clinical Trial at Children’s Hospital of Philadelphia

The Foundation-supported effort renders positive results after six months, marking an important advancement in retinal gene therapy.

Foundation Funding Developer of “Slimy” Gene Therapy for Restoring Sight

In pre-clinical studies, genes from algae are being used in a cutting-edge therapy known as ‘optogenetics.’

Researchers Find Further Evidence That Bile Acid Slows Vision Loss in Mice

A Foundation-funded team at the University of Iowa concludes that TUDCA shows promise for treatments of certain kinds of RP and Bardet-Biedl syndrome.

Pennsylvania-Florida Team Reports Promising Three-Year Results for LCA Clinical Trial

Fifteen participants receiving gene therapy continue to do well as investigators discover the best ways to deliver treatments.

Scientists Report Outstanding Progress in FFB-Funded Translational Research

An international team of retinal researchers funded by the Foundation's TRAP program report excellent progress in moving their emerging treatments and cures – including gene therapies, stem cell treatments and pharmacological compounds – toward human studies.

Progress in Development of New Gene Therapy for LCA and Cone-Rod Dystrophy

Success in a mouse model holds great promise for future clinical trials, or human studies, the Foundation is helping to make possible.

Advocating for Yourself

A retinal specialist provides patients with a list of topics, issues and questions which should be addressed during and soon after the delivery of a retinal disease diagnosis.

What Emerging Treatment Will Work Best for Me?

With the number of clinical trials targeting retinal degenerative diseases growing, Dr. Stephen Rose, the Foundation’s chief research officer, explains why it’s wise to keep track of most, if not all, of them.

First Patient Treated in Choroideremia Gene Therapy Clinical Trial in U.K.

The first-ever choroideremia gene therapy clinical trial is now underway at the John Radcliffe Hospital in Oxford, United Kingdom.

Foundation Provides Three-Year, $1.5 Million Grant for Choroideremia Gene Therapy

A clinical trial of gene therapy for choroideremia may be just three years away thanks to a $1.5 million grant awarded by the Foundation Fighting Blindness to Dr. Jean Bennett at the University of Pennsylvania.

Researchers Discover Retinal Disease in Which New Photoreceptors are Generated
A Foundation-funded researcher team has discovered a canine retinal disease with an amazing, unexpected effect. While the condition acts like a typical retinal disease and causes the loss of photoreceptors, it also leads to the proliferation of new ones.

Gene Therapy Revives Cones Long After They Stop Working

If you could do one thing for a person losing their vision to a retinal disease, saving their cones would be near the top of the list. That’s because cones are the retinal cells that allow people to see color and fine detail, enabling them to drive, read and see the faces of loved ones.

QLT Receives FDA Fast Track Designations for RP and LCA Treatment

September 14, 2011 - The FDA has granted the biopharmaceutical company QLT with Fast Track Designations for its synthetic retinoid treatment for people with Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP) caused by variations in the RPE65 and LRAT genes. Individuals with these genetic variations do not produce a retinoid critical for vision; QLT’s treatment serves as a replacement for that missing retinoid.

Foundation Fighting Blindness to Fund Nine New Research Grants

July 28, 2011 - The Foundation Fighting Blindness has announced funding for nine new 3-year grants in support of a wide range of innovative retinal research projects including the development of: enhanced genetic screening strategies, new gene therapies, vision-preserving drugs, and advanced retinal imaging techniques.

Emerging Treatment for Retinal Diseases Gets the Message Right

August 10, 2011 - In every cell in our body, DNA is sending messages, known as messenger RNA (mRNA), which tell our cells what proteins to make. Proteins are essential building blocks that provide our cells with structure and strength, regulate and protect our bodies’ chemistry, and facilitate the transport of oxygen and other essential substance. Proteins are also essential to the health of our photoreceptors—the light-sensing cells in the retinaand the biochemical process that makes vision possible. Hence, it is critical that the messages transmitted by our DNA are translated correctly, so that the right proteins are made.

Looking for Genes in All the Right Places

July 20, 2011 - An innovative, targeted search technique known as whole-exome sequencing is dramatically reducing the time, effort and expense it takes to discover genetic defects that cause retinal disease. While the increasing raw power of genetic-screening technologies is continually accelerating the pace of gene discoveries, the whole-exome search strategy is a major leap forward.

Gene Discovery Enhanced by Quickly Advancing Technologies

Thanks to accelerating advances in computing power and lab instruments, discovering the genetic defect that causes a person’s retinal degenerative disease is continually getting quicker and less expensive.

Sharing With Congress How High-Tech Drug Screening Speeds Research

 

One reason it takes so long to develop a drug that serves as either a treatment or a cure for a specific disease is that it often takes researchers years, even decades, to screen the innumerable compounds that go into making the drug. Or at least it used to. These days, thanks to recent advances in computer, robotic and data-processing technologies, such tests can take only weeks, as long as researchers have the right resources, which can be very costly and difficult to access.

Emerging Treatment “Gobbles Up” Culprit in Many Retinal Diseases

Foundation-funded researchers are developing an elegant approach to wiping out the toxic, vision-robbing molecules common to many retinal degenerative diseases. They hope to turn their emerging research into a cross-cutting treatment that has the potential to preserve vision in people with a broad range of retinal conditions.

Researchers Take another Critical Step toward Using Skin Cells to Treat Retinal Disease

A research team funded by the Foundation Fighting Blindness used an innovative repair technique to correct the disease-causing genetic defect in stem cells derived from a person’s skin — stem cells that hold the potential to treat their retinal degenerative condition.

Researchers Use Stem Cells to Build New Retina

A research team from Kobe, Japan has taken a major step forward in developing a functional retina from embryonic stem cells.

Gene Therapy Preserves Vision in Lab Study of Bardet-Biedl Syndrome

In a groundbreaking effort funded in part by the Foundation Fighting Blindness, scientists at Baylor College of Medicine used gene therapy to preserve vision in mice affected with a form of Bardet-Biedl syndrome (BBS), a devastating, multi-faceted disorder that causes significant vision loss from retinitis pigmentosa (RP). This research advancement represents the greatest success thus far in the development of a treatment for RP associated with BBS.

A Valuable Opportunity Exists in Federally Funded Research
An opinion editorial, penned by Foundation chief science staff members Drs. Steven Bramer and Stephen Rose, advocating for increased federal support of embryonic stem cell research was published on The Hill’s Congress Blog. Embryonic stem cells hold incredible promise for the development of treatments for all retinal degenerative diseases and the furthering of other areas in retinal research.

Artificial Retina Receives European Marketing Approval
The Argus II Retinal Prosthesis System, an implantable device that provides useful vision to people who are blind from advanced retinal degenerative diseases such as retinitis pigmentosa, will be available later this year in Europe.
Research Call: February 2010

This is a time of great optimism here at the Foundation Fighting Blindness.  Researchers funded by the Foundation are continuing to make tremendous strides in understanding the causes of retinal diseases and in the development of potential treatments and cures.  And what’s most exciting is that the progress we’re making will not just benefit one or two forms of blindness—but the whole range of retinal diseases.

Stem Cell Research - Frequently Asked Questions
What are stem cells?
Stem cells are cells that aren’t fully developed into a specific cell type. Stem cells have strong potential as medical treatments because researchers are able to develop them into a variety of cell types — for example, retinal cells, spinal cord tissue, and heart cells — that can be used to replace or repair cells and tissue lost to disease or injury. Stem cells can also be implanted to help prevent the death of cells that are affected by disease.

The Three Major Inheritance Patterns
Autosomal Recessive, Autosomal Dominant, and X-Linked

Most retinal degenerative diseases — including retinitis pigmentosa, Stargardt disease, and Usher syndrome — are inherited. They are caused primarily by genetic variations that are passed down from one or both parents, through one of the three inheritance patterns reviewed in this article. (The following discussion on inheritance patterns is not relevant for age-related macular degeneration (AMD), because risk of AMD is associated with a complex combination of genetic and lifestyle factors.) 
Clinical Trials are Expensive
Labor Intensive Clinical Trials Require Time, Effort and Financial Resources

We are no doubt in an exciting time as more clinical trials of promising treatments come to fruition. But as we have been reporting in recent years, clinical research brings a whole new financial challenge to the Foundation and our supporters. Human studies cost several million dollars each and are many times more expensive than laboratory studies.

Investigators Move Closer to Gene Therapy Trial for Choroideremia

Researchers are reporting that a clinical trial for gene therapy to treat choroideremia may be two years away thanks to successes in preclinical studies of gene therapy for choroideremia and human studies of gene therapy to treat Leber congenital amaurosis (LCA). 

Q&A with Dr. Steve Bramer: A Pivotal Role
Foundation Brings Knowledge and Expertise to Clinical Studies

Stem Cell Research: Recent Legal Decisions and Unauthorized Treatments

Update, September 9, 2010: A panel of the U.S. Court of Appeals ruled that the federal government, including the NIH, can resume funding of embryonic stem cell research while the court reviews the original order that banned funding.

On August 23, national media began focusing much attention on embryonic stem cells, because a judge from the U.S. District Court in Washington, D.C. issued a preliminary injunction banning federal funding for virtually all embryonic stem cell research.

Gene Therapy Reactivates Retina, Restores Vision

An international research team, funded in part by the Foundation Fighting Blindness, has used gene therapy to reactivate retinal cells that were previously unresponsive to light. The treatment was delivered to cones, the retinal cells that provide central and daytime vision. Investigators evaluated the gene therapy in two mouse models of retinal degenerative disease, as well as cultures of human retinal tissue. Using a variety of tests, the researchers demonstrated that the treated cells restored functional vision, and that the restoration of vision persisted over a long period of time.

Foundation-Funded Gene Therapy the Focus at International Biotech Conference
The success of vision-restoring gene therapy clinical trials continues to get outstanding recognition from a variety of media, research, and advocacy organizations.  Most recently, key leaders in the biotechnology community and the federal government have been enthusiastically telling the story.

Oral Compound Improves Vision in Three People with LCA
Three people with severe vision loss from Leber congenital amaurosis (LCA) have experienced improvements in vision after receiving an oral compound in an early stage clinical trial. QLT Inc., the developer of the compound, presented interim results of their clinical study on May 3 at the 2010 annual meeting of the Association for Research in Vision and Ophthalmology in Ft. Lauderdale.

Congressional Briefing Highlights Research Funding Need for Artificial Retina
Nearly 25 years ago, when Dr. Mark Humayun first envisioned the development of a bionic retina to enable the blind to see, scientific pundits equated the undertaking to a “moon shot,” implying that the project might be overly ambitious. However, thanks to sustained funding and innovative engineering by a team of engineers, scientists, and clinicians led by Dr. Humayun, 38 people with advanced retinitis pigmentosa, a vision-robbing retinal degenerative disease, are now successfully using implanted artificial retinas. Many of these individuals are participating in a clinical evaluation of the device led by Second Sight Medical Products, Inc.

Gene Therapy Shows Promise for Curing Day Blindness
A new advance in gene therapy is providing hope for people with a debilitating retinal disease known as achromatopsia or day blindness. Foundation-funded researchers from the University of Pennsylvania have used gene therapy to successfully treat dogs with the same condition.

Foundation Develops New Strategic Plan to Guide Future Research
In keeping with its mission, and to ensure it has a focused and clinically relevant research program, the Foundation Fighting Blindness uses a strategic planning process to develop recommendations regarding long-term research goals. Held every four to five years, the most recent strategic planning meeting occurred in late 2008. A summary of the final outcomes from this effort and their implications for the next four to five years is presented here.

Why It’s Important to Stay in Touch with Your Retinal Specialist

For many years, people with retinal degenerative diseases such as retinitis pigmentosa (RP), Usher syndrome, and Stargardt disease have been frequently told by their eye care professionals that “there’s nothing we can do.”

Researchers funded by the Foundation Fighting Blindness are moving promising treatments and cures into human studies, and in the not-too-distant future, there are likely to be therapies that will enable eye care professionals to better treat many retinal degenerative diseases.

Is a Genetic Test Right for You?

If you have an inherited retinal degenerative condition such as retinitis pigmentosa, Usher syndrome or Stargardt disease, should you get genetically tested? Is there a benefit in trying to determine the genetic variation that is causing your condition?

Latest Update: Gene Therapy Restores Significant Vision to Children with LCA
Gene therapy has taken another major step forward as a treatment for retinal diseases. Researchers conducting the landmark gene therapy clinical trial at The Children’s Hospital of Philadelphia (CHOP) have just reported that 12 people with Leber congenital amaurosis have now been successfully treated and are experiencing sustained, and in many cases significant, improvement in their vision.

Latest Update: Childhood Rediscovered: Gene Therapy Restores Vision for 9-year old with LCA
When it was his turn to take the stage at a press conference at The Children’s Hospital of Philadelphia (CHOP), Corey Haas got up from his seat in the auditorium and walked effortlessly up the four steps in front of him. Without hesitation, he crossed the stage, stepped around the table and took his seat next to Dr. Jean Bennett. His white cane was nowhere to be seen.

New LCA Gene Therapy Clinical Trial Recruiting Participants
Applied Genetic Technologies Corporation (AGTC) has launched a Phase I/II clinical trial of gene therapy to treat Leber congenital amaurosis, extending the work being done for similar treatments in three other Phase I clinical trials taking place at The Children’s Hospital of Philadelphia, Universities of Pennsylvania and Florida, and Moorfield’s Eye Hospital in the U.K. In this trial, AGTC will be evaluating higher doses of the treatment in younger participants with the hope of restoring even more vision than has been restored in the other three studies.

Saving Vision with Skin Cells
Thanks to Foundation-funded researchers at the University of Wisconsin-Madison, skin cells might someday restore vision lost to retinal degenerative diseases.

Vision Improvements from Gene Therapy Persist After One Year
Researchers from the Universities of Pennsylvania and Florida have reported that vision improvement for three individuals treated with gene therapy in a Phase I clinical trial has persisted for one year. The trial participants, all in their early 20s and with severe vision loss from Leber congenital amaurosis (LCA), continue to experience significant increases in night and day vision. Most importantly, the safety of the treatment, which is the primary goal of this and all Phase I clinical trials, is now extended to one year.

Q&A on Modifier Genes with Dr. Stephen Daiger
Funded by the Foundation Fighting Blindness since 1985, Dr. Stephen Daiger, of the University of Texas—Houston, is a world-renowned expert in identifying and understanding the genes and related mutations that cause retinal degenerative diseases.

A Vision for Gene Therapy
FFB Partnership Accelerates Advancement of Gene Therapies into the Clinic

A decade ago, five scientific leaders with expertise in gene therapy formed a company to commercialize their promising innovations for the treatment of incurable genetic diseases. They named the company Applied Genetic Technologies Corporation, with their acronym, AGTC, cleverly representing the four organic compounds — adenine, guanine, thymine, and cytosine — that make up the genetic code found in virtually every cell on the planet.

FFB Partnering with AGTC to Accelerate Advancement of Gene Therapies into the Clinic
The Foundation Fighting Blindness (FFB) has announced that it is collaborating with Applied Genetic Technologies Corporation (AGTC), a leading gene therapy development company, to advance gene therapies for retinal degenerative diseases into clinical trials.

Neurotech Treatment Shows Biological Effect in RP Clinical Studies
Neurotech’s innovative treatment employing Encapsulated Cell Technology (ECT) demonstrated a biological effect after 12 months for people with retinitis pigmentosa (RP), Usher syndrome, and choroideremia participating in two Phase II/III clinical trials. Both trials are being funded in-part by the Foundation Fighting Blindness.

Promising Research Presented at 2009 ARVO Annual Meeting
Every year, thousands of eye researchers from around the world convene at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting to share the latest advances and breakthroughs in eye and vision research. ARVO’s Annual Meeting is regarded as the premier annual research conference for the ophthalmological community.

Dr. Dean Bok Receives Foundation Fighting Blindness Career Achievement Award
The Foundation Fighting Blindness honored Dr. Dean Bok, of the Jules Stein Eye Institute of UCLA, with the prestigious Llura Liggett Gund Award Career Achievement Award on May 7.  The highly coveted award was presented to Dr. Bok in recognition of his outstanding dedication and commitment to retinal science and his extraordinary career achievements that have significantly advanced the research and development of preventions, treatments and cures for retinal degenerative diseases that affect more than 10 million Americans. Dr. Bok received the award before an audience of his peers at a Foundation dinner following the Association for Research in Vision and Ophthalmology Annual Meeting in Ft. Lauderdale.

Leading Retinal Disease Gene Therapy Developer Receives $11.8 Million Investment

Applied Genetic Technologies Corporation (AGTC), a clinical-stage gene therapy development company, has raised $11.8 million in venture capital funding to advance treatments for the retinal degenerative diseases Leber congenital amaurosis (LCA) and achromatopsia. The investment will also be used to fund research for an early onset form of emphysema.

New Investment to Boost Gene Therapy Development

The National Neurovision Research Institute (NNRI), the Foundation Fighting Blindness’ clinical trial support organization, announced today that one of its key partners, biopharmaceutical company Oxford BioMedica, has established a collaboration with sanofi-aventis, a major international pharmaceutical company, to develop and commercialize gene therapy treatments for vision-robbing retinal degenerative diseases that affect tens of millions of people around the world.

Emerging Treatment Stabilizes Vision in People with Dry AMD

Owings Mills, MD - March 26, 2009 — An innovative technology, employing a tiny capsule implanted in the eye, is stabilizing vision in people suffering from dry age-related macular degeneration (AMD). Encapsulated Cell Technology (ECT), developed by Rhode Island-based Neurotech, preserved vision in a majority of the 51 people who participated in a Phase II clinical trial.

Foundation Appoints Four New National Trustees to Advance Mission

The Foundation Fighting Blindness has appointed four new national trustees to further advance the organization’s fundraising, organizational development, and volunteer recruitment efforts.

2009 Day of Science: Cures Coming into Sight

Highlights from the 2009 Day of Science Meeting Hosted by the Foundation Fighting Blindness

Dr. Al Maguire captivated the Day of Science audience when he played a video of an eight-year-old boy, who after being treated with a landmark gene therapy, was able to quickly navigate an obstacle course he was previously unable to follow. The audience erupted in loud applause when Dr. Maguire added, “That boy has put away his blind cane and is now enrolled in a mainstream school.”

Retinal Nourishment is Key in Preventing Vision Loss
Nourish the retina. That's the concept behind two newly identified strategies for keeping the retina healthy and preventing vision loss. They were the findings of Foundation-funded researcher Constance Cepko, Ph.D., of Harvard Medical School, who studies the degeneration of photoreceptors in animal models of retinal disease.

Gene Therapy Shows Promise for Curing Day Blindness

A new advance in gene therapy is providing hope for people with a debilitating retinal disease known as achromatopsia or day blindness. Researchers from the University of Pennsylvania have used gene therapy to successfully treat dogs with the same condition.

Light of Day

Gene Therapy Shows Promise for Curing Day Blindness

A new advance in gene therapy is providing hope for people with a debilitating retinal disease known as achromatopsia or day blindness. Researchers from the University of Pennsylvania have used gene therapy to successfully treat dogs with the same condition.

Neurotech Capsule Receives Fast Track Status from FDA

Neurotech’s innovative encapsulated cell technology (ECT), which shows promise for treating a wide range of retinal diseases, has received Fast Track status from the FDA. Fast Track status is given to therapies that address an unmet medical need. Also, the FDA will review an application for marketing approval of a Fast Track treatment within six months. Neurotech plans to release results from their three Phase II/III clinical trials in spring 2009. At that point the company may seek FDA marketing approval for the treatment to make it generally available and/or conduct additional clinical studies.

Emerging Treatment Provides Crucial Biochemical for Restoring Vision
People with certain forms of Leber congenital amaurosis and retinitis pigmentosa have new hope for vision restoration thanks to a treatment being developed by QLT, Inc., a biopharmaceutical company in Vancouver, Canada.
Researchers Coax the Retina into Rebuilding Itself

In a pioneering study at the University of Washington, FFB-funded researchers discovered a way to prod retinal cells to regenerate after injury. If perfected, the approach could be used to restore retinal tissue, and vision, for people with a variety of retinal degenerative diseases including retinitis pigmentosa and macular degeneration.

Hill Briefing Highlights Research Effort that is Helping the Blind to See

Two of the world’s top retinal researchers came to Capitol Hill on June 24 to educate congressional staff about their landmark clinical trial in which they used gene therapy to restore some vision in three young adults who were virtually blind from a severe form of retinitis pigmentosa known as Leber congenital amaurosis (LCA). Seventy people attended the briefing, which was hosted by the Alliance for Eye and Vision Research and the Foundation Fighting Blindness.

New Model of Artificial Retina Moves into Human Studies

For people with the most advanced retinal degenerative disease - those who are blind or have only light perception - artificial retinas are providing promise for restored vision.

Second Sight® Medical Products is now conducting a Phase II human study of its second-generation artificial retina - the Argus II - in clinical centers in the U.S., Mexico, and Europe (England, France, and Switzerland).

Congressional Briefing Underscores the Importance of Vision Research Funding
Top leaders from the vision research community came to Capitol Hill on October 31 to brief 70 congressional staff and guests about the critical need for research funding to eradicate vision-robbing retinal diseases that affect more than nine million Americans. Representatives from the Foundation Fighting Blindness, National Eye Institute and Alliance for Eye and Vision Research held the briefing to highlight the public importance of funding for eye research.
Now They See - Breaking News from the Foundation Fighting Blindness

Three young adults with virtually no vision can now read several lines on an eye chart and see better in dimly lit settings thanks to an innovative gene therapy aiming to reverse blindness in a severe form of retinitis pigmentosa known as Leber congenital amaurosis or LCA. One person was even able to better navigate an obstacle course several weeks after receiving the therapy.

Paris Research Center Focused on Moving Promising Treatment into Clinical Trials

In 2005, French researchers José Sahel, M.D., and Thierry Léveillard, Ph.D., won the Foundation’s Annual Trustee Award for the most promising advance of the year: Identifying a protein that shows great potential for preserving vision in people affected by a variety of retinal degenerative diseases. Sahel and Léveillard, both with the Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts in Paris, are now part of a new FFB-funded research center chartered with moving this promising protein into clinical trials.

Nanoparticles Show Promise for Delivering Vision-Restoring Genes to the Retina
Nanoparticles are a rapidly emerging technology that is appearing in emerging treatments for retinal diseases, cancer, HIV, and a variety of other conditions. These microscopic particles — which are like tiny rocks as small as one fifty-thousandth of an inch — are uniquely able to penetrate cells and tissue for the delivery of therapeutic genes and molecules.
Genetic Breakthrough Gives New Hope to Family’s Fight Against Blindness

Shortly after Alan Brint was born in 1997, his parents David and Betsy realized that his vision was not right. They were devastated to learn from retinal experts that young Alan was nearly blind because of a genetic disease called Leber congenital amaurosis (LCA). “It’s every parents’ worst nightmare,” says David, “to find out that something is tremendously wrong with your child. Blindness never even occured to us until the doctor said, “Your child is blind and there is nothing we can do at this time.”

Human Neural Stem Cells Show Potential for Vision Rescue

An FFB-funded research team from the University of Wisconsin and the University of Utah used human neural stem cells — cells derived from the brain — to rescue vision in a rodent model of retinal degenerative disease.

Potential Cure for Blind Children Moves into a Human Study

Children born blind from a devastating retinal condition called Leber congenital amaurosis (LCA) may soon be able to see thanks to an innovative gene replacement therapy that has just moved into a clinical trial.

Umbilical Cord Tissue Shows Promise as Retinal Disease Treatment

The umbilical cord is not only the lifeline for a developing baby, but it might also save sight for people affected by a variety of retinal degenerative diseases.

Dr. Matthew LaVail Receives Llura Liggett Gund Award for Excellence in Retinal Degeneration Research

Dr. Matthew LaVail was awarded the Llura Liggett Gund Award—the most prestigious honor awarded by The Foundation Fighting Blindness (FFB)—for lifetime achievement in retinal disease research.

Retinal Chip Study Expanded

A 10-person evaluation of the Artificial Silicon Retina (ASR) microchip, developed by Optobionics (Naperville, Illinois), is being expanded to 20 patients at three sites: Johns Hopkins School of Medicine (Wilmer Eye Institute), Emory University School of Medicine/Atlanta Veterans Administration Medical Center, and Rush University Medical Center.

FFB Research Center: University of Iowa’s Genetic Testing Initiative will Bolster Progress in Treating RDDs

Ed Stone, Val Sheffield, Tom Casavant, and their colleagues at the University of Iowa’s Center for Macular Degeneration and Allied Retinal Diseases see no reason why there shouldn’t be cost-effective, efficient, and accurate genetic testing available for every person who has an inherited retinal degenerative disease (RDD). They’re certain that more and better treatments will be attainable, if genetic testing — and the information gleaned from it — is more accessible to researchers, ophthalmologists, and patients. With support from The Foundation Fighting Blindness and other organizations, they are well poised to make this plan for ubiquitous testing a reality.

 
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