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.

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.

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.

Microsoft Mouse for those with Low Vision

This is an older video that I ran across a few weeks ago, but I thought it would be beneficial for those who didn't know this existed.

Its a Microsoft mouse with a zoom feature built it.  I use something similar with just keyboard hotkeys, but this might go on my christmas list.



This is Microsoft's site detailing all its products that have zoom features.

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!

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