ACT Files European Clinical Trial Application For Phase 1/2 Study Using Embryonic Stem Cells To Treat Macular Degeneration

Advanced Cell Technology, Inc. ("ACT"; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that it has filed a clinical trial application (CTA) with the European Medicines and Healthcare products Regulatory Agency (MHRA) seeking clearance to initiate its Phase 1/2 clinical trial using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs) to treat patients with Stargardt's Macular Dystrophy (SMD).

"With this filing, our initiatives in Europe are really starting to gain momentum," said Gary Rabin, interim chairman and CEO of ACT. "Through data from this proposed trial, and the two trials we are preparing to commence in the United States, we are eagerly anticipating beginning to assess the capabilities of our RPE cells to repair and regenerate the retina. As in the US, we also intend to file in Europe for clinical trials involving Dry Age-Related Macular Degeneration (Dry AMD) and other degenerative diseases of the retina, concurrently targeting the two largest pharmaceutical markets in the world."

The proposed clinical 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 to patients with advanced SMD, similar to the FDA-cleared U.S. trial which is set to commence in the first half of this year. During the CTA review process, which requires a minimum of 60 days, the reviewers decide if an applicant is permitted to proceed with its proposed clinical trial. Additional information may be requested from the applicant, which could extend the review period.

"We are very excited about this European filing, because our preclinical data from various animal models with hESC-derived RPE cells have been tremendously encouraging," said Robert Lanza, M.D., chief scientific officer at ACT. "In rats we have seen 100 percent improvement in visual performance over untreated animals without any adverse effects. Near-normal function was also achieved in a mouse model of Stargardt's disease."

In 2010, the US Food and Drug Administration (FDA) granted Orphan Drug designation for ACT's RPE cells for treating SMD, and earlier this year the company received a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) towards designation of this product as an orphan medicinal product for the treatment of Stargardt's disease. ACT anticipates adoption of the EMA's recommendation by the European Commission in coming weeks.

About Stargardt's Macular Dystrophy and Degenerative Diseases of the Retina

Stargardt's Macular Dystrophy (SMD) is one of the most common forms of macular degeneration in the world. SMD causes progressive vision loss, usually starting in children 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.

Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. As many as thirty million people in the United States and Europe suffer from macular degeneration, which represents a $25-30 billion worldwide market that has yet to be effectively addressed. Approximately 10% of people ages 66 to 74 will have symptoms of macular degeneration, the vast majority the "dry" form of AMD which is currently untreatable. The prevalence increases to 30% in patients 75 to 85 years of age.

Source: Advanced Cell Technology, Inc

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GIS Scientists Propose A New Paradigm For Embryonic Stem Cells, Potentially Speeding Up Development Of Disease Therapies

Scientists from the Genome Institute of Singapore (GIS) have put forward a novel explanation for the pluripotency[1] of embryonic stem (ES) cells. Their groundbreaking explanation opens new doors for understanding how stem cells create specific cell types, fundamental knowledge that will drive changes and improvements in the therapeutic and translational usage of stem cells. A better understanding of ES cells could help advance the development of treatments for diseases such as diabetes, Parkinson's disease, and Huntington's disease. The work, published in the journal Cell Stem Cell, was led by Dr Bing Lim, Senior Group Leader of the Stem Cell and Developmental Biology department at the GIS, and Kyle Loh, GIS student from Dr Lim's lab.

By re-examining current data with a fresh eye, Lim and Loh were able to suggest a novel paradigm that may resolve the 30-year-old mystery behind pluripotency. The prevailing model of stem cell pluripotency suggests that stem cell genes active in ES cells prevent these stem cells from turning into specific cell types. This model accounts for how ES cells can remain undifferentiated, but is unable to explain convincingly the ability of stem cells to create any bodily cell type. Lim and Loh suggest that, contrary to current thinking, individual stem cell genes do not completely suppress differentiation, but instead actively direct ES cells to produce particular bodily cell types. In their new paradigm, Lim and Loh propose that the activation of a combination of such stem cell genes within ES cells is what enables ES cells to create any bodily cell type.

[1] Pluripotency refers to the ability of ES cells to differentiate into all bodily cell types. ES cells can potentially create, on demand, any cell type that clinicians or scientists need for therapeutic, biotechnological, or research purposes. Hence, the cells are currently used as a source of specialized cell types used in cell replacement therapies. An understanding of how ES cells are able to produce all these cell types is of intense pragmatic and theoretical interest.

Source
Genome Institute of Singapore

New Insight Into Muscular Dystrophy Provided By Human Embryonic Stem Cells

Myotonic dystrophy type 1 (DM1) is the most common inherited muscular dystrophy in adults. New research published by Cell Press online on March 31st in the journal Cell Stem Cell, uses human embryonic stem cells to make a clinically-important contribution to the understanding of this disease, and highlights the incredible potential that embryonic stem cells hold for unraveling the complex molecular mechanisms involved in a variety of human conditions.

DM1 patients suffer from muscle wasting and multiple defects in their central nervous system. Although scientists have made progress finding the genetic mutation that causes DM1, the molecular mechanisms that underlie the disease, and thus could be targets for treatment, are not well understood. To look for new molecular factors involved in DM1, a research team led by Dr. Cecile Martinat, from France's Institute for Stem Cell Therapy (I-Stem), designed a study to search for differences between cells carrying the DM1 mutation and normal cells.

"We used pluripotent stem cell lines derived from human embryos characterized during preimplantation genetic diagnosis as carrying the gene for DM1," explains senior study author, Dr. Martinat. "These cells can self-renew indefinitely, making them available in large numbers, and they possess the ability to differentiate into any type of cell, allowing us to perform key functional studies."

The researchers looked at neural cells made from their embryonic stem cell lines, and found reduced expression of genes in the SLITRK family that was mirrored in brain biopsies from DM1 patients. SLITRK proteins are involved in the outgrowth of neurons and the formation of synapses, which are sites of communication between nerve and muscle cells. Martinat and colleagues looked at DM1 neurons cultured together with muscle cells, and found that the change in SLITRK expression caused defects in the cell-cell connections that formed.

"These neuropathological mechanisms may be clinically significant for the functional changes in neuromuscular connections associated with DM1," says Dr. Martinat. "In addition, our results highlight the tremendous value of human pluripotent stem cells as an appropriate model to decipher events involved in the pathogenesis of a disease state. This is especially relevant now, as the French Parliament voted in favor of a revised bioethics bill last February that will restrict embryo research." says Dr.Marc Peschanski, Head of I-Stem. In support of successful pathological models like the one described here, a broad consensus of researchers and clinicians is now urging the French Senate to overturn the ban in a vote scheduled for April 5th, and to explicitly authorize research on human embryonic stem cells.

Source:
Elisabeth Lyons
Cell Press