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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Potential Stem Cell Therapy For Age-Related Macular Degeneration

The notion of transplanting adult stem cells to treat or even cure age-related macular degeneration has taken a significant step toward becoming a reality. In a study published today in Stem Cells, Georgetown University Medical Center researchers have demonstrated, for the first time, the ability to create retinal cells derived from human-induced pluripotent stem cells that mimic the eye cells that die and cause loss of sight.

Age-related macular degeneration (AMD) is a leading cause of visual impairment and blindness in older Americans and worldwide. AMD gradually destroys sharp, central vision needed for seeing objects clearly and for common daily tasks such as reading and driving. AMD progresses with death of retinal pigment epithelium (RPE), a dark color layer of cells which nourishes the visual cells in the retina.

While some treatments can help slow its progression, there is no cure. The discovery of human induced pluripotent stem (hiPS) cells has opened a new avenue for the treatment of degenerative diseases, like AMD, by using a patient's own stem cells to generate tissues and cells for transplantation.

For transplantation to be viable in age-related macular degeneration, researchers have to first figure out how to program the naïve hiPS cells to function and possess the characteristics of the native retinal pigment epithelium, RPE, the cells that die off and lead to AMD.

The research conducted by the Georgetown scientists shows that this critical step in regenerative medicine for AMD has greatly progressed.

"This is the first time that hiPS-RPE cells have been produced with the characteristics and functioning of the RPE cells in the eye. That makes these cells promising candidates for retinal regeneration therapies in age-related macular degeneration," says the study's lead author Nady Golestaneh, Ph.D., assistant professor in GUMC's Department of Biochemistry and Molecular & Cellular Biology.

Using an established laboratory stem cell line, Golestaneh and her colleagues show that RPE generated from hiPS cells under defined conditions exhibit ion transport, membrane potential, polarized VEGF secretion and gene expression profile similar to those of a normal eye's RPE.

"This isn't ready for prime time though. We also identified some issues that need to be worked out before these cells are ready for transplantation but overall, this is a tremendous step forward in regenerative medicine," Golestaneh adds.

She explains that the hiPS-derived RPE cells show rapid telomere shortening, DNA chromosomal damage and increased p21 expression that cause cell growth arrest. This might be due to the random integration of viruses in the genome of skin fibroblasts during the reprogramming of iPS cells. Therefore, generation of viral-free iPS cells and their differentiation into RPE will be a necessary step towards implementation of these cells in clinical application, Golestaneh says.

"The next step in this research is to focus on a generation of 'safe' as well as viable hiPS-derived somatic cells," Golestaneh concludes.

Notes:

Other authors on the paper include first author Maria Kokkinaki, Ph.D., Department of Biochemistry and Molecular &Cellular Biology, and Niaz Sahibzada, Ph.D., Department of Pharmacology at GUMC.

This work was funded by the National Institutes of Health. The authors report no personal financial interests related to this study.

Source:
Karen Mallet
Georgetown University Medical Center