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

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

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!