Advanced Cell Technology initiates higher-dosage patient treatment in European clinical trial for Stargardt's Disease

Advanced Cell Technology, Inc., a pioneer in the field of stem cells for treating conditions such as macular degeneration. has announced treatment of the first patient in the third dosage cohort, and seventh patient overall, in its European Phase I clinical trial for Stargardt’s macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). 

The patient was injected with 150,000 hESC-derived RPE cells, as compared with the 100,000-cell dose used in patients of the second cohort. The surgery was performed on Friday, April 19, without any complications, and the patient is recovering uneventfully.

The company declared that they are past the halfway point in all three of their clinical trials on both continents. They have also announced that the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has officially granted their hESC-derived RPE cells orphan medicinal product designation for the treatment of SMD. This will likely provide a number of benefits to them, as in protection from competition, as well as for the patients through reduced fees.

The Phase 1/2 trial is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with SMD at 12 months, the study’s primary endpoint. It will involve a total of 12 patients, with cohorts of three patients each in an ascending dosage format.

Stargardt’s disease or Stargardt’s Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration. Clink here to read more, 

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Phase 2b/3 clinical trial of Emixustat Hydrochloride in subjects with geographic atrophy associated with dry Age-related Macular Degeneration

Acucela Inc., a clinical-stage biotechnology company focused on developing new treatments for sight threatening eye diseases, announced phase 2b/3 clinical trial investigating emixustat hydrochloride in subjects with geographic atrophy (GA) associated with dry age-related macular degeneration (AMD) has been initiated. 

AMD is the most common cause of irreversible vision loss in the developed world, the overwhelming majority of which is associated with dry AMD. There are currently no medications approved to treat GA associated with dry AMD. Emixustat hydrochloride is being studied to determine whether it slows the progression of GA in patients with GA associated with dry AMD. 

The SEATTLE study (for Safety and Efficacy Assessment Treatment Trials of Emixustat Hydrochloride) was initiated based on data from the recently completed phase 2a study, the results of which will be announced at the ARVO 2013 Annual Meeting, as well as feedback from the U.S. Food and Drug Administration (FDA).

Emixustat hydrochloride has a unique mechanism of action in visual cycle modulation, offers oral dosing and the ability to specifically target the visual cycle, representing a potentially novel therapeutic approach for the treatment of retinal diseases, such as GA associated with dry AMD.

The SEATTLE study of emixustat hydrochloride is designed as a phase 2b/3 multicenter, randomized, double-masked, dose-ranging study comparing the efficacy and safety of emixustat hydrochloride with placebo for the treatment of geographic atrophy (GA) associated with dry age-related macular degeneration (AMD). Approximately 440 patients with GA associated with dry AMD will be enrolled in the study across 56 sites, primarily in the United States.

There are more than 10 million people in the US and more than 120 million people worldwide who have age-related macular degeneration. AMD is associated with irreversible vision loss, the overwhelming majority of which is due to the dry form of AMD, representing approximately 90% of all cases. Dry AMD occurs when the light-sensitive cells in the back of the eye slowly deteriorate, gradually blurring the central field of vision. As the disease advances, and where patients typically present with GA, the blurred vision slowly progresses to blindness in affected areas of the eye.

To know more about AMD, please click here and here.

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A study for new treatments for wet age-related macular degeneration (AMD)

A University of Wisconsin School of Medicine and Public Health scientist, Dr Nader Sheibani, is spearheading a study of new treatments for wet age-related macular degeneration (AMD). Dr. Sheibani, a professor in the department of ophthalmology and visual sciences, was awarded $6.2 million over five years from the National Eye Institute. In the first year, the collaborators will receive $1.2 million.

The project is a collaboration among Dr. Sheibani’s lab and others in the department of ophthalmology and visual sciences; researchers in the UW department of pediatrics; scientists at Northwestern University Center for Developmental Therapeutics and Feinberg School of Medicine; and the University of Nebraska Center for Drug Delivery and Nanomedicine. Each specialist and team brings different skills and techniques to the question of treatment of exudative AMD.

Wet or exudative AMD is the leading cause of blindness among aging Americans. The global rate of AMD is expected to double in the next decade as the population ages.  A cause of vision loss in this type of AMD is associated with angiogenesis, the growth of new blood vessels which are leaky with severe consequences to vision.

Ophthalmologists have developed treatments known as anti-vascular endothelial growth factor (VEGF) to slow or stop new vessels from forming. The anti-VEGF treatments require frequent injections into the eye. They are an important tool for ophthalmologists to help save vision and their use has demonstrated that inhibiting VEGF slows damage from AMD. However, VEGF is essential for normal ocular integrity and function. Therefore, there is a great need for new treatments which preserve vision without interfering with normal functions.

Dr. Sheibani and his collaborators hope to develop treatments using small peptides that mimic those of the body’s own inhibitors of angiogenesis. These treatments will counter VEGF activity and inhibit vascular growth in eyes with exudative AMD while providing an environment resistant to new vessel growth. 

This study will develop peptide mimetics – shorter versions of the proteins the body makes – that will stop vessel growth. Researchers at Northwestern University are developing the peptides and those at the University of Nebraska are developing the drug delivery nanotechnology. Dr. Sheibani and his staff will test whether the treatments work in preclinical models of the disease for its translation to humans.

The hope is that this class of drugs, because they mimic the body’s own defenses, will be more effective and have fewer side effects than the treatments already available. Although human clinical trials are years away, this research is truly translational, with the potential to greatly impact quality of life by preventing a common cause of vision loss.

Source

Mum with ROP manages with a guide dog

Wendy Wright thought she was exceedingly clumsy in her early adulthood. The 49-year-old has managed her condition, known as Retinopathy of Prematurity (ROP), for decades, but now has a new-found freedom thanks to guide dog, Freya.

The mum of five has no peripheral vision, a result of her premature birth.

Ms Wright has had Freya (3) for six months and said it was “amazing” how much of a difference the dog had made to her life.

Freya and Ms Wright graduated as a guide dog team on Tuesday.

from www.inmycommunity.com.au

Ms Wright, manager of the |Needle and Syringe Exchange Program, commutes to work in the Perth CBD by public transport with Freya every morning.

“I can walk down the street, turn my head and look into a shop window without walking into the back of someone, if there’s a person there Freya will guide me around them,” she said.

“I had so much trouble using a white cane, I’d walk into people with their iPod headphones in and they’d get annoyed.

“She’s also taken it upon herself to be my companion, which I didn’t expect, I had some very bad news recently and she didn’t leave my side – that bond after six months just amazes me.”

In 2013 the Association for the Blind, which runs Guide Dogs WA, celebrates 100 years of service to the WA community.

Source

Being the person, not the 'blind' person...

"...The accompanist’s rapidly moving hands on the keyboard spark curiosity about the source of the prominent piano line. But curiosity may linger for those that notice he is not using sheet music. The pianist is Shane Dittmar, a freshman music education major, accompanying the Department of Performing Arts’ production “It Gets Better” last October. For more than four hours, Dittmar played an entirely memorized piano part in the back-to-back productions." from The Pendulum, Elon University's Student News Organization And for 18 years, Dittmar has been living with a disability. Dittmar is blind. Diagnosed with a genetic disease called Leber Congenital Amaurosis (LCA), Dittmar and his twin brother Derek have been blind since birth. Just like Dittmar had to memorize music measures for “It Gets Better,” he has had to memorize innumerable daily methods in order to adjust to life being blind.

Shane has also been trying out musical theatre.

from The North Raleigh News With a flash of casting instinct from the mind of a high school theater director, Shane Dittmar has claimed his home on stage - the musical theater stage, that is. That's not necessarily an unusual thing for a high school sophomore who is a perfect-pitch tenor with a charismatic personality - punctuated by an enviable wit and outstanding student achievement honors. Difference here: Shane is blind.

Some of Shane's videos...enjoy his voice, his music and his wit!

A new potential treatment for Retinitis Pigmentosa & Macular Degeneration

A team of researchers from the University of California, Berkeley, in collaboration with researchers at University of Munich and University of Washington in Seattle have found a chemical that can temporarily restore some amount of vision in blind mice.

This compound could eventually help those with Retinitis Pigmentosa, a common genetic disease that leads to blindness, as well as Age-related Macular Degeneration.

The chemical, referred to as AAQ, acts on the remaining cells in the retina, which are normally "blind" cells, sensitive to light. AAQ is a photoswitch that binds to protein ion channels on the surface of retinal cells; when switched on by light, it alters the flow of ions through the channels and activates these neurons similar to the way rods and cones are activated by light.

This chemical has been shown to eventually wear off, and hence may offer a safer alternative to other approaches that restore sight, such as gene or stem cell therapies, which may permanently change the retina. It is also less invasive than implanting light-sensitive chips in the eye.

Considering that it is a simple chemical, it will be easy to change the dosage, use it in combination with other therapies, or discontinue the therapy if the need be. This would allow new and improved chemicals that may become available in time to be offered to the same patients.

The blind mice in the experiment had genetic mutations that made their rods and cones die within months of birth and inactivated other photopigments in the eye. After injecting very small amounts of AAQ into the eyes of the blind mice, the researchers confirmed that they had restored light sensitivity because the mice's pupils contracted in bright light. The mice also demonstrated light avoidance, a typical rodent behavior impossible without the animals being able to see some light. The researchers are hoping to conduct more sophisticated vision tests in rodents injected with the next generation of the compound.

It will be a while before this compound will find its way in humans. The researchers have to show that these compounds are safe and will work in patients the way they work in mice. BUt these preliminary results demonstrate that this class of compound restores light sensitivity to retinas blind from genetic disease.

The current technologies being evaluated for restoring sight to people whose rods and cones have died include injection of stem cells to regenerate the rods and cones; "optogenetics," a type of gene therapy where a photoreceptor gene is inserted into blind neurons to make them sensitive to light; and installation of electronic prosthetic devices, such as a small light-sensitive retinal chip with electrodes that stimulate blind neurons.

Eight years ago, Kramer, Trauner, a former UC Berkeley chemist now at the University of Munich, and their colleagues developed an optogenetic technique to chemically alter potassium ion channels in blind neurons so that a photoswitch could latch on. Potassium channels normally open to turn a cell off, but with the attached photoswitch, they were opened when hit by ultraviolet light and closed when hit by green light, thereby activating and deactivating the neurons.

Subsequently, Trauner synthesized AAQ (acrylamide-azobenzene-quaternary ammonium), a photoswitch that attaches to potassium channels without the need to genetically modify the channel.

Newer versions of AAQ now being tested have demonstrated better results, as per the researchers. They activate neurons for days rather than hours using blue-green light of moderate intensity, and these photoswitches naturally deactivate in darkness, so that a second color of light is not needed to switch them off.

Source

Musings over a cup of sambar

It's not just the sight, but smells and sounds have the ability to transport someone to a different place, a different time. It happens too often for someone who suffered progressive vision loss. Here's the experience of one of them.

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In spite of the clattering of plates and the high-decibel noise, the restaurant seemed more appealing. It doesn't require a genius to figure out why? The smell of coffee lured us from quite a distance, and as we wade through the crowd milling around the tables, we were assailed bythe smell of sambar.

The aroma transported me to a different place, to a different time.

Friday evening at our home town of Viruddachalam (better known these days as the former constituency of actor-politician Vijayakant). The bazaar road would be chaotic with fast moving 'town buses' and trucks carrying loads of sugarcanes and groundnuts. Roadside tea stalls did brisk business, and the police station that sat at the corner of the Double street looked more magical as the lone tube light inside flickered and flashed at regular intervals.

Having spent a full day playing cricket on the dry riverbank, myself and my brother would feel hardly tired as we looked forward to the prospect of eating the evening snack outside. We invariably went to the thatched roof stall of Adai Rayar, whose serves special 'onion adai' with tasty 'Milagai podi' (chilli powder) and gingili oil.

That was, of course, the popular choice. The thick dosa like dish would arrive the moment Rayar Mama saw uncle cycling with a nephew each on the front and back of the saddle. While everyone preferred to eat the crisp and wonderfully fried Adai with chilli powder, my choice was the other

one: vegetable sambar and coconut chatni. Rayar was too happy to serve the sambar several times, especially to me since I never used to waste even a drop. How can I? It was one of the best thing I had ever tasted.

It was harder to say what made the sambar tasty. It could be the dal or the native tamarind and vegetable. My guess was the proportion and the blend, which needed immense skill to reproduce without any change on a daily basis.

With the red mark on his forehead and customary dhoti-towel, not to mention the trademark smile, Rayar offered the best food in the town. The Adai, made with red gram-rice-red chilli paste, topped the billing.

The sambar we tasted at the restaurant didn't live up to the sweetness of its smell. But thoughts of Adai Rayar and the tasty sambar at his modest stall seemed to have done the trick. There was only the smile and no complaints of the tanginess due to excess tamarind juice. Oh yes, the mind often crawls through scenes of the past, morphing the unpleasantness of the present with fond memories.

- Learner