Four Patients Test Retinal Implant That Could Stop Age-Related Blindness

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Some parts of getting older are awesome. Retirement. Early bird specials. Free reign to whine about your various aches and pains.

Not so great? Your vision can start to go.

Luckily, a new retinal implant may soon help treat a common cause of age-related vision loss.

Non-neovascular age-related macular degeneration (NNAMD) (also known as “dry” AMD) causes a blurry area right in the middle of a person’s line of vision that can grow as the disease progresses. Sometimes, the previously blurry spot becomes simply blank.

In short, that can be debilitating, making it pretty impossible to live a normal life. After all, you need to be able to see what’s in front of you to do things like drive a car or cook a meal.

Currently, there’s no treatment for the advanced stages of NNAMD, but a research team led by Amir Kashani, an assistant professor of clinical ophthalmology at University of Southern California (USC), is hoping to change that.

NNAMD likely begins with the breakdown of cells in a membrane in the eye called the retinal pigment epithelium (RPE). Kashani and his team designed an implant to mimic the function of this membrane. The implant fits on the retina and is made of human embryonic stem cells placed on a base material.

The team had already tested a version of the implant on rodents, so the next step was to make the leap to humans. So the researchers placed their implant into the eyes of four people with advanced NNAMD. Then, they monitored those people for between four and 12 months.

According to the researchers’ study, published today in Science Translational Medicine, none of the four participants had any negative or severe side effects from the retinal implant, and experienced no vision loss over the course of the trial. Once participant even “demonstrated an observable improvement” in their vision.

When the team took post-operative images of the patients’ eyes, they saw that the stem cells had blended with the existing retinal tissue. That is, the retinas looked like they were regaining their RPEs. A good sign.

Of course, this was an exceptionally small sample size that delivered promising (but not overwhelmingly positive) results. So the researchers’ next step is to test their implant on a larger group.

If it works the way researchers hope, it may someday be a game-changer for thousands of visually-challenged seniors. After all, what good’s a senior discount on movie tickets if you can’t see the screen.

The post Four Patients Test Retinal Implant That Could Stop Age-Related Blindness appeared first on Futurism.


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The First Gene Therapy That Fixes Hereditary Blindness May Finally Get FDA Approval

Second Sight

Today, a panel will advise the US Food and Drug Administration whether Luxturna, a gene therapy treatment developed by Spark Therapeutics, should be approved for general usage. The treatment has already been used to improve the eyesight of more than two dozen people with retinal disorders.

Gene therapy typically uses an engineered virus to administer a patient with a faulty gene with a corrected version. Rather than simply responding to the symptoms of the condition in question, it attempts to make changes to the individual’s genetic make-up in order to solve the problem at its root.

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Luxturna fixes a mutation in a gene known as RPE65, which is responsible for telling the body how to produce a protein that’s essential for normal eyesight. It introduces billions of engineered virus particles bearing a corrected version of the gene to the retinal cell, via a quick injection to the eyes.

The company estimates that 6,000 people around the world could benefit from Luxturna, including between 1,000 and 2,000 people in the US who suffer from diminished eyesight. The majority of these people would eventually lose their current level of vision entirely without treatment, and there are currently no drugs designed for people with an RPE65 mutation that are approved by the FDA.

However, Luxturna is not without its drawbacks. It’s not an outright cure, and it doesn’t give recipients full 20/20 vision. There’s currently no data on how long its effects last, so there’s a chance that patients’ sight might begin to recede once again over time.

Cost is also a major factor in how accessible it is. Two of the treatment’s biggest competitors, Strimvelis and Kymriah, cost around $ 700,000 and $ 475,000 respectively. Consequently it seems likely that Luxturna have to drop in price to be a feasible competitor. They might be losing ground, as Spark has announced plans to set up a program to help patients cover out-of-pocket costs like travel to Spark-proffering facilities.

Gene Therapy

Gene therapy has the potential to make huge improvements to the quality of life of people suffering from various genetic diseases. This blossoming form of treatment could well be the wave of the future.

“This is what I believe medicine is going to be like for the next 20, 30, if not 50 years,” said Spark CEO Jeff Marrazzo, speaking to the MIT Technology Review. “I think this is the beginning of an age that is going to fundamentally change medicine.”

We’ve already seen projects that use gene therapy to tackle everything from brain diseases to broken bones. However, these treatments will all require FDA approval — so scientists working in this sphere will likely be watching today’s decision regarding Luxturna very closely.

The post The First Gene Therapy That Fixes Hereditary Blindness May Finally Get FDA Approval appeared first on Futurism.


Researchers May Have Discovered a Way to Reverse Blindness

Giving Sight to the Blind

Visual impairment is still rather rampant, according to the World Health Organization (WHO). Some 285 million people worldwide are considered visually impaired, and 39 million of them are blind. Thankfully, 80 percent of all visual impairment can now be treated or cured, except in cases of total loss of sight, particularly those due to severe retinal degeneration.

But what if it’s possible to restore visual function to blind patients? Laboratory tests in the University of Oxford demonstrate how this may be possible. In a study published in the journal of the Proceedings of the National Academy of Sciences (PNAS), the Oxford researchers led by Samantha de Silva showed how it’s possible to restore the sight of people suffering from blindness previously considered untreatable.

“Inherited retinal degenerations may result in blindness due to a progressive loss of photoreceptor cells,” the researchers wrote. “We assess subretinal delivery of human melanopsin using an adeno-associated viral vector to remaining retinal cells in a model of end-stage retinal degeneration.”

Light Sensitivity

Using gene therapy, the researchers introduced a viral vector in retinal cells found at the back of the eyes that weren’t originally sensitive to light. The viral vector introduces a light-sensitive protein called melanopsin, which enables these residual retinal cells to respond to light and send visual signals to the brain. In lab tests with mice suffering from retinitis pigmentosa, the most common cause of blindness in young people, the researchers were able to maintain sight in the mice for over a year. The mice demonstrated high levels of visual perception, recognizing objects in their environment.

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It’s worth noting that de Silva and her colleagues have also been successfully trialing an electronic retina. However, they observed that gene therapy may be simpler and easier to administer. Similar work has been done by others involving age-related blindness, while an FDA-approved gene therapy seeks to cure hereditary retinal blindness.

The results are quite promising, and de Silva noted how much hope this treatment gives to patients suffering from blindness. “There are many blind patients in our clinics and the ability to give them some sight back with a relatively simple genetic procedure is very exciting,” she said in a press release. “Our next step will be to start a clinical trial to assess this in patients.”

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