Advanced Cell Technologies granted orphan drug designation from FDA

I have written earlier about Advanced Cell Technologies filing an IND (Investigational New Drug) application with the FDA.  The application would allow them to start a phase 1 trial for using Embryonic Stem Cells to treat Stargardt's disease in 12 people.

This application was approved on March 2nd and the trial is now able to move forward.  This could be a very promising treatment for those with Stargardt's Disease.

“We are pleased that the FDA has, for the first time, granted orphan drug status for the use of an embryonic stem cell derived therapy in treating an unmet medical need,” said Edmund Mickunas, Vice President Regulatory. “We believe that our terminally differentiated RPE cells represent a promising treatment for patients with SMD and expect to be in a position to accelerate clinical development and hopefully make RPE cellular therapy available to the majority of patients sooner.”

Here is a description of the treatment.

Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world, and as many as ten million people in the United States have photoreceptor degenerative disease. While most of these patients have Age-Related Macular Degeneration (AMD), a smaller number have Stargardt’s, an Orphan disease and to date an untreatable form of juvenile macular degeneration leading to blindness in a much younger group of patients than are affected by AMD. ACT’s treatment for eye disease uses stem cells to re-create a type of cell in the retina that supports the photoreceptors needed for vision. These cells, called retinal pigment epithelium (RPE), are often the first to die off in SMD and AMD, which in turn leads to loss of vision.

While there is currently no treatment for SMD, several years ago ACT and its collaborators discovered that human embryonic stem cells could be a source of RPE cells. Subsequent studies found that the cells could restore vision in animal models of macular degeneration. In a Royal College of Surgeons (RCS) rat model, implantation of RPE cells resulted in 100% improvement in visual performance over untreated controls, without any adverse effects. The cells survived for more than 220 days and sustained extensive photoreceptor rescue. Functional rescue was also achieved in the ‘Stargardt’s’ mouse with near-normal functional measurements recorded at more than 70 days.

Click the title for the full Press Release

Brian Mckeever to compete Sunday

I've dedicated a few posts to Canadian Olympic cross country skier Brian Mckeever.  He is 30 years old, has Stargardts disease, is legally blind, and is competing in both the Olympic and Paralympic games this winter.  He will be the first athlete in history to do so.

He is competing in the Men's 50km Mass Start Classic, It starts Sunday February 28th at 9:30 AM PSD.  If you are on the east cost set your DVR's.

Again we'll be cheering for him even though he's Canadian.

Update: (3/2/2010):  Unfortunately according to Wikipedia Brian did not compete on Sunday. 

however Canada's coach decided to replace him with a skier who did well at an earlier event at the 2010 games and thus he will not become the first athlete in the world to compete in the Winter Paralympics and Winter Olympics in the same year.

Simple steps to protect your eye sight

This article speaks of a few basic steps to preserve your sight.

1. Get regular eye exams

2. Stay informed about eye diseases

3. Exercise Regularly

4. Wear sunglasses and protective eye wear

5. Avoid eye fatigue

6. Provide your eyes with critical nutrients

Click the title for the full article.

There is also an article that states eating a 'Mediterranean diet' protects eye health. The article states

A study from the Centre for Eye Research Australia (CERA) found that people who consume at least 100 millilitres of olive oil a week are almost 50 per cent less likely to develop macular degeneration than those who eat less than 1 millilitre per week.

The study also found that people who eat other foods which are rich in omega-3 fatty-acids, such as fish and nuts, are 15 per cent less likely to develop macular degeneration.

February is AMD/Low Vision Awaremess month

Sorry Its been a pretty slow news month.

Age-related Macular Degeneration, which impacts the vision of more than 2 million Americans 50 and older, is one of the leading causes of blindness. Yet many people aren’t even aware of the perils of this eye disease, which is why Prevent Blindness America has declared February AMD/Low Vision Month. 

 So tell a friend.

Reading glasses specifically for those with Low Vision

I posted earlier about a Dr. Sonsino creating a set of portable reading glasses for those with low vision.  He is back in the news and it sounds like the glasses are coming along nicely.

If you didnt read the previous post about these glasses here is a quick description from Dr. Sonsino.

He says, "The glasses combine three aspects. The first is magnificaion, the second is a little prism to allow the eyes to stay straight, instead of curving in, and the third is LED lighting."

 The article describes briefely how the glasses help and who would have a need for them.  They also have a website www.lowvisionreaders.com which says they are currently working on a prototype and best estimates are these will be released in summer of 2010.

Click the title for the full article

Google has a search engine specifically for those with Low Vision

Did you know google had a search page for those with low vision?  It is only in its preliminary stages but it could be helpful.  It responds to hot keys, magnifies the results you have selected, and beeps when you change the focus on the page.

Give it a try

Google Accessible Web Search for the Visually Impaired

Should your eye doctor sell supplements?

This article raises a good point.  Doctors should not be in the position of selling you products.  And supplements benefits are inconsistent at best.  Here is an excerpt from the article.

There's something fishy about this, besides the fish oil, in my opinion. If a doctor wants to suggest that this product may help, show me the research and tell me that I can buy it in places besides your office. I later discovered that a similar formula with Lutein, Zeaxanthin and fish oil is available at Sam's Club for half the cost. I suspect it's available at any retailer that sells a lot of supplements and vitamins.

What do you thing?  Click the title for the full article.

Brian Mckeever will compete and make history!

I posted earlier that Brian Mckeever was attempting to compete in both the Olympic and Paralympic games this winter.  Well it is now official he has quallified to be a part of the Canadian Cross Country Olympic team.  Brian is 30 years old and suffers from Stargardts disease.  Here is an excerpt from the article.

The 30-year-old Canmore, Alta., resident will be selected to Canada's Olympic cross-country ski team on Friday, making him the first athlete to compete at both the Olympic and Paralympic Winter Games.

Mr. McKeever, who suffers from Stargardt's disease and is legally blind, stamped his double pass to Whistler by winning an able-bodied 50-kilometre Haywood NorAm race last month in Canmore. The race was one of four Olympic trials established by Cross Country Canada.

We'll be rooting for him, (even though he's Canadian).

Click the title for the full article.

Low vision aides show to have a drastic difference in reading speeds

I know it sounds obvious but reading the numbers is interesting to me.  Here is the short article.

Low vision aids can significantly improve reading speed and reading ability in patients with age-related macular degeneration, but better overall visual acuity still correlates with better reading ability.

According to a retrospective study of 530 patients with AMD who were provided either optical visual aids or closed-circuit TV systems as a low-vision aid, the added magnification of the system helped improved words read per minute.

For the entire group of patients, mean reading speed improved from 20 ± 33 words per minute (wpm) to 72 ± 35 wpm. However, among patients with a visual acuity less than 0.1, reading speed improved from 0.4 ± 3.8 wpm to 40 ± 13 wpm compared with 20 ± 28 wpm to 84 ± 30 wpm among patients with a visual acuity score of 0.1 or better.

Still, in the study, low vision aids had a marked impact on reading ability: Only 16% of patients were able to read before receiving a low vision aid compared with 94% after.

Click the title for full story

Macular Degeneration Cause discovered on a molecular level

Researchers at University College London have discovered the chemical proccess that causes Macualr Degeneration.  They state it is caused by the interaction of two protiens blood protein Factor H, and C-reactive protein.  These proteins work together to clear out the debris of dead cells in the retina, but if the levels are not optimal or if someone has a genetically different form of Factor H then dead cells are not cleaned up properly and for a deposite called drusen.  These deposits take the place of new cells and also restrict the bloodflow to neighboring cells causing them to die.  At least that is how I understood it.  here is the article in full.

Researchers at University College London say they have gleaned a key insight into the molecular beginnings of age-related macular degeneration, the No. 1 cause of vision loss in the elderly, by determining how two key proteins interact to naturally prevent the onset of the condition.

In a paper to be published in a forthcoming issue of the Journal of Biological Chemistry, the team reports for the first time how a common blood protein linked to the eye condition reins in another protein that, when produced in vastly increased amounts in the presence of inflammation or infection, can damage the eye.

"By starting to understand these interactions in greater detail, we can begin to devise methods that will ultimately prevent the development of blindness in the elderly," said Zuby Okemefuna, the lead author of the paper to be published Jan. 8.

Age-related macular degeneration, or AMD, is painless but affects the macula, the part of the retina that allows one to see fine detail. One form of the debilitating condition, known as "wet" AMD, occurs when abnormal and fragile blood vessels grow under the macula, leaking blood and fluid and displacing and damaging the macula itself. The second form, "dry" AMD, occurs when light-sensitive cells in the macula slowly break down.

It is believed that both forms start on a common molecular route and then deviate into dry or wet AMD, explained the research leader, Steve Perkins.

"The earliest hallmark of AMD is the appearance of protein, lipid and zinc deposits under the retinal pigment epithelial cells," he said, adding that the yellowish deposits, usually discovered by an ophthalmologist, are commonly known as "drusen."

The researchers studied two proteins involved in drusen formation -- blood protein Factor H and a second blood protein known as C-reactive protein -- and showed that Factor H binds to C-reactive protein when C-reactive protein is present in large amounts, as in the case of infection, to reduce the potentially damaging effects of an overactive immune system.

"In the eye, during the normal processes of aging, cells will die naturally for all sorts of reasons," Okemefuna said. "The blood supply to the eye will bring C-reactive protein with it, and a low level of C-reactive protein activity will enable the normal processes of clearance of dead cells at the retina through mild inflammation. In conditions of high inflammation, the levels of C-reactive protein in the retina will increase dramatically."

Uncontrolled C-reactive protein activity causes damage to the retina, which is followed by more inflammation and then even more damage to the retina, and so forth.

"It's the debris of broken up retinal cells, some of which is caused by this cycle, that is deposited as drusen," Okemefuna said.

The team also found that a genetically different form of Factor H does not bind to the C-reactive protein quite as well as the normal one, making people who carry the modified protein more vulnerable to an immune system attack in the eye and, thus, drusen buildup.

"In normal individuals, further damage to the retina by prolonged exposure to high levels of C-reactive protein is prevented by Factor H. C-reactive protein also prevents Factor H from clumping together and initiating the processes that lead to drusen formation," Perkins said. "Both these 'good' activities of Factor H are much reduced in the genetically different form of Factor H."

While there is no known cure for AMD, existing therapies aim to treat the symptoms and delay progression.

"It is interesting how the interaction of these two blood proteins protects the eye during crisis," Perkins said. "The two proteins also can be involved in a rare and often fatal cause of kidney failure in children. We now are better positioned to begin to work out preventative strategies for these diseases."

Ruodan Nan, Ami Miller and Jayesh Gor also were co-authors on the study, which was funded over the past three years by University College London, the Biotechnology and Biological Sciences Research Council, the Mercer Fund of the Fight for Sight Charity and the Henry Smith Charity.

Click the title to read the full article.