Macular Degeneration and Stem Cells

1. Macular Degeneration and Embryonic Stem Cell Therapy

According to Advanced Cell Technology's website:

"The Phase I/II trial will be a prospective, open-label study that is designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation into patients with Dry AMD."

The site that is participating in this clinical trial is Jules Stein Eye Institute, UCLA School of Medicine.

Macular Degeneration Research and Embryonic Stem Cells

There are three layers of tissue that support the retina - the choroid, Bruch's membrane and the retinal pigment epithelium.

Advanced Cell Technology will use retinal pigment epithelial (RPE) cells derived from human embryonic stem cells to replace the the lost RPE cells

Many people have ethical concerns regarding the use of embryonic stem cells. Here is the company's response to this issue.

According to Gary Rabin, Advanced Cell Technology's chief executive, "Advanced Cell Technology uses a proprietary technique to extract a single cell from a young embryo, allowing the rest to remain intact and develop normally.

A similar method is routinely used to biopsy embryos for pre-implantation genetic diagnosis, in which embryos created through in vitro fertilization are scanned for genetic disorders before being transferred to a uterus."

As with any Phase I clinical trial, the focus will initially by on safety.

Once the safety trials are completed then subsequent clinical trials will focus on the effectiveness of the stem cell.

The hope of future studies will be to show that the RPE cells that are injected into the retinal space will be capable of slowing or possibly halting the progression of dry macular degeneration.

There is even some hope of potentially even restoring some lost visual acuity.

Eligibility Criteria

However there are many other criteria listed for this study for a complete listing go to

Stem Cells and Macular Degeneration Clinical Trial

Description of Procedure

"Each eligible patient who signs a consent form and fulfills all (Inclusion/Exclusion) criteria will receive a single uniocular (one eye) subretinal infusion of MA09-hRPE cells in one of four dose levels.

Three patients - 50,000 cells transplanted

Three patients - 100,000 cells transplanted

Three patients - 150,000 cells transplanted

Three patients - 200,000 cells transplanted

Six weeks after the first patient in each dose cohort receives the cell transplant, the Data Safety Monitoring Board (DSMB) will review the clinical data and recommend if the next two patients in the dose cohort may be treated.

Each cohort of 3 patients will be reviewed by the DSMB when the 3rd patient completes 4 weeks of follow-up."

Stem Cell Update

According to published results in Lancet on January 24, 2012:

"After surgery, structural evidence confirmed cells had attached and continued to persist during our study."

"... it is encouraging that during the observation period neither patient lost vision. Best corrected visual acuity improved from hand motions to 20/800 (and improved from 0 to 5 letters on the Early Treatment Diabetic Retinopathy Study [ETDRS] visual acuity chart) in the study eye of the patient with Stargardt's macular dystrophy, and vision also seemed to improve in the patient with dry age-related macular degeneration (from 21 ETDRS letters to 28)."

"The hESC-derived RPE cells showed no signs of hyperproliferation, tumorigenicity, ectopic tissue formation, or apparent rejection after 4 months.

The future therapeutic goal will be to treat patients earlier in the disease processes, potentially increasing the likelihood of photoreceptor and central visual rescue."

Dry Age Related Macular Degeneration

"In the case of the Dry AMD trial, four of the initial patients that principal investigator Dr. Steven Schwartz of UCLA had identified as good candidates from which to select patients two and three wound up being screened out of eligibility in the final battery of tests. The patients all passed initial health screenings, complete reviews of their medical records and extensive optical analyses. Sadly, on the eve of the surgeries, the longer lead time screenings for cancer and viral infections yielded previously unidentified positive results."

AND

"The Food and Drug Administration’s (FDA) protocol for our surgeries, as you are also likely aware, involves a course of immune suppression before and for a period of time after the transplant, and a patient’s ability to tolerate this without other adverse effects is a critical factor in inclusion in the study. The extremely poor visual acuity requirements in the FDA protocol result in a decidedly elderly patient population (with patients typically being 77 years old or so at the youngest). When that factor is combined with the requirement of an intact Bruch’s Membrane, and the general health-related restrictions mentioned above, it makes for a more challenging patient screening process in the dry AMD trial relative to that for the SMD trial."

AND the GOOD NEWS

"That being said, I am pleased to inform you that we are currently in the advanced stages of screening several more clinical trial candidates and are very confident that we will soon be able to treat two additional Dry AMD patients, rounding out the first cohort in the AMD trial."

2. Autologous Bone Marrow Transplant for Macular Degeneration

The stated purpose is:

"The purpose of this study is to evaluate the behavior of intravitreal injection of of autologous bone marrow stem cells in patients with age related macular degeneration."

Eligibility Criteria

* Male or female macular degeneration patients 55 to 80 years old * In either eye, diagnosis of geographic atrophy or choroidal neovascularization due to age related macular degeneration

* ETDRS best corrected visual acuity of 60 letters or worse in the study eye.

Description of Procedure

1) severe visual loss, defined as a drop in 15 letters on ETDRS visual acuity scale;

2) decrease in ERG response;

3) decrease in 5 square degrees on visual field;

Secondary secondary safety outcomes to be evaluated are:

1) increase in intra-ocular inflammation defined herein as anterior chamber cells and flare higher than 3+ for more than 1 month after injection according to a classification described elsewhere ;

2) decrease in CMT more than 50um;

3)genesis of abnormal tissues (teratomas) or tumors;

4) qualitative changes in retinal or choroidal perfusion, like macular nonperfusion

To read more about this macular degeneration research go to: Intravitreal Bone Marrow-Derived Stem Cells in Patients With Advanced Age-related Macular Degeneration (AMDCELL)

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