Patients Undergo Embryonic Stem Cell Transplantation Treatment for Stargardt's Disease and Macular Degeneration

Advanced Cell Technology, Inc., a leading company in the field of regenerative medicine, has announced that two patients have been implanted with retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs) in each of its two Phase 1/2 clinical trials for Stargardt's macular dystrophy and dry age-related macular degeneration (AMD).

The patients were treated on July 12, 2011, at the David Geffen School of Medicine at UCLA's Jules Stein Eye Institute in Los Angeles, USA. Both patients successfully underwent the outpatient transplantation surgeries and are said to be recovering uneventfully.

One patient in each clinical trial, the Stargardt's trial and the dry AMD trial, underwent surgical transplantation of a small dose (50,000 cells) of fully-differentiated RPE cells derived from human embryonic stem cells. The patients appear to have tolerated the surgical procedures well.

Regarding the clinical trial:

Both the Stargardt's trial and the dry AMD trial will enroll 12 patients each, with cohorts of three patients each in an ascending dosage format. Both trials are prospective, open-label studies designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with Stargardt's and dry AMD at 12 months, the studies' primary endpoint.

This is the first step in the clinical trial, which will potentially define the dose required to help treat the condition. If the trial succeeds, it opens doors to a potentially significant and new therapeutic approach to treating Stargardt's disease and AMD.

The primary objective of these Phase 1/2 studies is to assess the safety and tolerability of these stem cell-derived transplants. The patients will be carefully monitored over the course of the trials, to observe and report for any untoward and adverse event that may happen.

To know more of this trial, please click here.

About hESC-RPE Cells

RPE cells are highly specialized tissue located between the choroid and the retina. RPE cells support, protect and provide nutrition for the light-sensitive photoreceptors. Human embryonic stem cells differentiate into any cell type, including RPE cells, and have a similar expression of RPE-specific genes compared to human RPE cells and demonstrate the full transition from the hESC state.

About Stargardt's macular dystrophy:

Stargardt's macular dystrophy is one of the most common forms of macular degeneration in the world. Stargardt's causes progressive vision loss, usually starting 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 or RPE cell layer.

About Age-related Macular Degeneration:

Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. Approximately 10% of people ages 66 to 74 will have symptoms of macular degeneration, the vast majority of them suffering from the "dry" form of AMD - which is currently untreatable. The prevalence increases to 30% in patients 75 to 85 years of age.

Dry AMD, the most common form of macular degeneration, Stargardt's and other forms of atrophy-related macular degeneration are untreatable at this time, and need effective therapies for treatment of these common forms of blindness.

Disease progression of both Stargardt's and dry AMD includes thinning of the layer of RPE cells in the patient's macula, the central portion of the retina and the anatomic location of central vision. When RPE cells die, the macular photoreceptors are lost, leading to loss of central vision. This approach focuses on treating these conditions by transplanting RPE cells in the patient's eyes before the RPE population is lost.

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