Advanced Cell Technologies CEO Interviewed on Bloomberg Radio

I posted recently that ACT has gotten approval to start a phase I trial for treating Stargardts disease. 

The CEO of ACT was interviewed on the radio on March 9th. He speaks about stem cell treatment in general, as well has the health care industry.

Click the Title of the article to listen to the interview

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.

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.

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.

Happy Holidays - Brian McKeeve attempts to compete on both Olympic and Paralympic games this winter

For the holidays I thought I'd post this.  Its an brief article explaining Brian McKeever, a Canadian cross country skier with Stargardt's disease.  He is legally blind and attempting to compete in both the Olympic and Paralympic games this winter.  Here is the article in full.

Brian McKeever of Canmore, Alta., moved closer Tuesday to becoming the first ever winter athlete to compete in an Olympic and Paralympic Games.
The 30-year-old, legally blind cross-country skier dominated the 50km Haywood NorAm individual-start classic race in his hometown, part of a series of races Cross Country Canada is using to determine its final Olympic spots that will be announced in January. McKeever suffers from Stargardt's disease, which has left him only with 10 per cent of his vision, all peripheral. In 2007, he finished 21st in a 15km skate-ski race at the able-bodied world championships. "That is all I had [Tuesday] and I hope it is enough to convince the selection committee that I deserve a spot on the Olympic team," he said in a release. "I feel satisfaction right now."
Five summer-sport athletes have competed in both the Paralympics and Olympics.

Eat your Friuts and Vegetables.

A study has been published in the Journal of Food Science stating that nutrients in Green leafy vegetables and colored fruits and vegies can greatly aid vision.  Here is an excerpt from the article.

To reach the conclusion, authors from the University of Georgia compiled the results of multiple studies on the effects of the carotenoids lutein and zeaxanthin on visual performance. These carotenoids play an important role in human vision, including a positive impact on the retina.

After reviewing the various studies, the authors concluded that macular pigments, such as lutein and zeaxanthin do have an effect on visual performance. Lutein and zeaxanthin can reduce disability and discomfort from glare, enhance contrast, and reduce photostress recovery times. They can also reduce glare from light absorption and increase the visual range.

The article also states getting a healthy amound of lutein and zeaxanthin can reduce the risk of age related macular degeneration and cataracts.

I posted in a previous article one mans claims of the benefits of lutein.

Click the title for the full article.

Phase II trial for Wet Age related Macular Degeneration (In Europe)

A company called ThromboGenics is holding a phase II trail for treating West AMD with microplasmin.

Here is a description of what the drug does.

It was recently discovered that one-third of patients with AMD have focal vitreomacular adhesion, a condition in which the vitreous gel in the center of the eye has an abnormally strong adhesion to the retina at the back of the eye. The same adhesion occurs in patients with wet AMD. Microplasmin is designed to treat vitreomacular adhesion by separating the vitreous gel from the retina, potentially preventing the progression of wet AMD.

The MIVI5 (Microplasmin for IntraVitreous Injection) trial will enroll approximately 100 patients across up to 20 European medical centers. The goal is the non-surgical resolution of vitreomacular adhesion. Safety and efficacy will also be evaluated during a one-year follow-up period.

Click the Title for the full article

Marigolds may be cure for Dry age related macular degeneration?

This retired british Optician (Harry Marsland) claims he cured his dry age related macular degeneration by taking MACUSHIELD a supplementt that

 contains lutein, found in spinach, and zeaxanthin, the yellow pigment found in corn – both of which are used in other treatments. It also contains meso-zeaxanthin, derived from marigolds, which was a nutrient Mr Marsland had not tried before.

 
I am skeptical but curious.  I also dont know how beneficial it is to non age related MD.  I would suggest contacting your retina specialist before trying it.  It can be purchassed via the link below.

Click the title for the full article.

Embryonic Stem Cell treatment for Stargardt 's starting soon

A company call Advanced Cell Technology based in Massachusetts has applied for an IND (investigational new drug) application with the FDA.  This application would allow them to start a phase 1 trial for using Embryonic Stem Cells to treat Stargardt 's disease in 12 people. 

This would be only the 2nd time the FDA approves the use of Embryotnic Stem Cells for treatment.  The 1st was for the treatment of paraplegics but that study has been on hold since January.

ACT has had promising results with curing macular problems in rats and in other animal trials.  This would be a milestone for medicine in general opening up a new field called Regenerative Medicine.

Here is ACT's press release in its entirety.

WORCESTER, Mass., Sept 22 (BUSINESS WIRE)—Advanced Cell Technology, Inc. (OTCBB:ACTC.PK–News) provided an update on pre-clinical activities in preparation of its first IND filing with the Food and Drug Administration for its retinal pigment epithelium (RPE) cell program for the treatment of various eye diseases. In the next few weeks, the Company will be completing the preclinical work necessary for filing the IND. To date, no adverse events have occurred in testing. The results will be part of the submission which Advanced Cell expects to submit to the FDA prior to the end of the year.

“We are very pleased with the long-term safety and efficacy data,” said Dr. Robert Lanza, ACT’s Chief Scientific Officer. “We have carried out pre-clinical studies using these cells in multiple animal models, and to-date have not seen any teratoma formation or untoward pathological reactions. We are optimistic that human embryonic stem cells (hESCs) will serve as a potentially safe and inexhaustible source of RPE for the treatment of a range of macular degenerative diseases.”

“We are pleased with the progress we have made in preparation for the FDA submission,” said William M. Caldwell IV, Advanced Cell’s Chairman and CEO. “In the retina, compromised RPE function can lead to deteriorated vision and photoreceptor loss in both age-related macular degeneration and other forms of degenerative eye disease. We look forward to further studying the role that our RPE cells can play in providing a potential solution to this problem.”

This is very exciting and we'll have to keep an eye on this company and this trail.

Click the title for full article

Update: The application has been approved!

Video of Corey Haas 9 year old boy from gene therapy trial

In my post yesterday I spoke of 12 patients who went through gene therapy to restore their sight.  Here is a video of the youngest participant Corey Haas and his family


Watch CBS News Videos Online

Gene therapy transforms eyesight of 12 with retinitis pigmentosa

This is very exciting stuff. 

The Children's Hospital of Philadelphia and the University of Pennsylvania School of Medicine performed a gene therapy study with 12 participants of varying ages.  All participants had a specific form of retinitis pigmentosa called Leber's congenital amaurosis.  Each patient had the therapy done to one eye.  After a few weeks every patient showed measurable improvements in their vision.  Here is the article in its entirety.

A single injection in a patient's eye brings 'astounding' results. The findings may offer hope for those with macular degeneration and retinitis pigmentosa.

Pennsylvania researchers using gene therapy have made significant improvements in vision in 12 patients with a rare inherited visual defect, a finding that suggests it may be possible to produce similar improvements in a much larger number of patients with retinitis pigmentosa and macular degeneration.

The team last year reported success with three adult patients, an achievement that was hailed as a major accomplishment for gene therapy. They have now treated an additional nine patients, including five children, and find that the best results are achieved in the youngest patients, whose defective retinal cells have not had time to die off.

The youngest patient, 9-year-old Corey Haas, was considered legally blind before the treatment began. He was confined largely to his house and driveway when playing, had immense difficulties in navigating an obstacle course and required special enlarging equipment for books and help in the classroom.

Today, after a single injection of a gene-therapy product in one eye, he rides his bike around the neighborhood, needs no assistance in the classroom, navigates the obstacle course quickly and has even played his first game of softball.

The results are "astounding," said Stephen Rose, chief scientific officer of Foundation Fighting Blindness, which supported the work but was not involved directly. "The big take-home message from this is that every individual in the group had improvement . . . and there were no safety issues at all."

The study "holds great promise for the future" and "is appealing because of its simplicity," wrote researchers from the Nijmegen Medical Center in the Netherlands in an editorial accompanying the report, which was published online Saturday by the journal Lancet.

The 12 patients had Leber's congenital amaurosis, which affects about 3,000 people in the United States and perhaps 130,000 worldwide. Victims are born with severely impaired vision that deteriorates until they are totally blind, usually in childhood or adolescence. There is no treatment.

Leber's is a good candidate for gene therapy because most of the visual apparatus is intact, particularly at birth and in childhood. Mistakes in 13 different genes are known to cause it, but all 12 of the patients suffered a defect in a gene called RPE65. This gene produces a vitamin A derivative that is crucial for detecting light.

About five children are born each year in the United States with that defect, which was chosen because researchers at the Children's Hospital of Philadelphia and the University of Pennsylvania School of Medicine had cloned the gene, making copies available for use.

The study, led by Dr. Katherine A. High, Dr. Albert M. Maguire and Dr. Jean Bennett of those two institutions, enrolled five people in the United States, five from Italy and two from Belgium. Five were children, and the oldest was 44.

The good copy of the RPE65 gene was inserted into a defanged version of a human adenovirus. The engineered virus then invaded retinal cells and inserted the gene into the cells' DNA.

Maguire used a long, thin needle to insert the preparation into the retina of the worst eye in each of the patients. Within two weeks, the treated eyes began to become more sensitive to light, and within a few more weeks, vision began to improve. The younger the patients were, the better they responded. That was expected, Bennett said, because similar results had been observed in dogs and rodents.

By both objective and subjective measures, vision improved for all the patients. They were able to navigate obstacle courses, read eye charts and perform most of the tasks of daily living. The improvement has now persisted for as long as two years.

The children who were treated "are now able to walk and play just like any normally sighted child," Maguire said.

Bennett noted that the oldest patient in the trial, a mother, had not been able to walk down the street to meet her children at school. "Now she can. She also achieved her primary goal, which was to see her daughter hit a home run."

There are clear limitations to the study. The patients' vision was not corrected to normal because of the damage that had already been done to the retina, and only one eye was treated.

"The big elephant in the room is: Can you treat the other eye?" Rose said.

The foundation will put more funding into the research "to make sure that if you go back and treat the other eye, it won't ablate the positive results in the first eye due to an immune reaction or something else."

Researchers also have not optimized the dosage of the adenovirus used to carry the gene into the eye. Those issues will be studied in Phase 2, a larger clinical trial that they hope to begin soon.

Meanwhile, the team has begun treating some patients at the University of Iowa.

Researchers also hope they will be able to translate the results to other congenital conditions using different genes.

Leber's is one form of retinitis pigmentosa, which affects an estimated 100,000 Americans.

The findings might be applicable to macular degeneration, which affects an estimated 1.25 million Americans and is the major cause of visual impairment in the elderly.

I also received an email from The Foundation Fighting Blindness stating that Dr Rose would be interviewed tonight (10/25/09) on CBS Evening News at 6pm.  Now my schedule doesn't list the evening news being on tonight, so i'm not sure if this is a misprint but it also says the interview will also be run tomorrow morning on the CBS Early Show.

Near blind can be Oceanographers.

This  article is interesting for a few reasons.  It is about Amy Bower who is an oceanographer despite having macular degeneration and retinitis pigmentosa.  One affecting her central vision, and the other her peripheral vision.  The article is an inspiration for those with low vision who think they may not be able to perform a particular job.   It is also a testiment to the technology avialable to help those of us with low vision.
In Amy's words

"For anyone who finds themselves in the situation I was in 25 years ago, they need to learn to become a very strong self-advocate," Bower said. "And hopefully, you want to pursue something that you're passionate about, because you're going to need the energy that comes from such a passion to push through the challenges."

Click the Title for the full article and a short video.

The blind can climb mountains

This article follows Justin Grant who has Stargardt's disease and a team of blind, low vision, and sighted climbers who go on expedition to climb mountains.  They have climbed Mt. Kilimanjaro, Mt, Rainier, and up Machu Picchu.  The article explains how Justin got into climbing, as well as introduces the rest of the team, all of which are members of Team Sight Unseen. It shows that adversity drives people to acheive.  So having a disability is in many ways a benefit.  I don't agree with Justin when he said "If there was a cure for me, I’m not even sure I would want it."  I feel that my disability has given me a sense of drive that I may not have had otherwise, but getting a cure wouldn't remove that drive.

Click the Title for the full article plus some photos and a video

Do you have hallucinations in your blind spot?

Oliver Sacks studies the visual regions of the brain and hallucinations.  In this talk he describes people with sugnifficant vision loss seeing everything from geometric shapes to people and cartoons.  It is a very interesting talk.



I do see patterns in my blind spot.  Its not anything I can describe.  Its just chaos or fractals.  I'm not user if this is normal or if I have what he describes.

Low Vision Glasses An Option for Sight-Impaired

 This story is a few days old, but it seems very interesting to me, it is a special pair of reading glasses for those with low vision.  It says it improves reading ability up to 90% at close range.  Here's the bulk of the article

Optometrist Jeffrey Sonsino of Vandervilt Eye Institute created Illuminated Low Vision Glasses. They combine a high-powered L-E-D light in the frame, magnifying lenses, and prism correction that prevents eye fatigue.

"Those three things together provide magnification and enhanced contrast when the patient is reading at very close distance," Sonsino says.  "This doesn't cure anything, but what this does, is it allows people with the condition to function better."

In a study, the glasses improved patients' reading abilities by almost 90-percent compared to regular glasses.

Click the title for the full story and video

This really intrigued me.  I did some digging about Jeffrey Sonsino, he is an optomatrist and Director of the Center for Sight Enhancement at Vanderbilt Eye Institute, located in Nashfville TN.

The glasses may just be a set of powerful reading glasses with LED lights in the frame.  I found this PDF that is a brief description of the glasses.  Here is an excerp

Illuminated Low Vision Glasses are portable, far less bulky, easier to use than other reading aids, relatively inexpensive to manufacture and do not need to be prescribed by a low vision specialist. Illuminated Low Vision Glasses use high powered LED lighting built into the spectacle frame, magnifying high powered lenses and prism correction to fi x the reading distance.

I tried looking for the patent but couldn't find it.

Microchip in the Eye Seeks to Restore Vision

This technology isn't my favorite, but it is something that is making progress.

Patients who receive the implant will wear a pair of glasses that has a tiny camera attached to it. The camera will send images to a microchip implanted in the eyeball that channels the input to the brain.

It won’t entirely restore normal vision, say the researchers, but it will offer just enough sight to help a blind person navigate a room.

This is a good solution for those who are completely blind.  I'd prefer not needing an external camera to see.  The upside would be that you could possibly alter the camera to see infared and other light spectrums.

click the title for full story