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

Note: Any medical information published on this website is not intended as a substitute for informed medical advice and you should not take any action before consulting with a health care professional. For more information, please read our terms and conditions.

Please note that we publish your name, but we do not publish your email address. It is only used to let you know when your message is published. We do not use it for any other purpose. Please see our privacy policy for more information.

If you write about specific medications or operations, please do not name health care professionals by name.

All opinions are moderated before being included (to stop spam)

Contact Our News Editors

For any corrections of factual information, or to contact the editors please use our feedback form.

Please send any medical news or health news press releases to:

Privacy Policy | Terms and Conditions

MediLexicon International Ltd
Bexhill-on-Sea, UK
MediLexicon International Ltd © 2004-2011 All rights reserved.

Clinical Trial Success For Crohn's Disease Cell Therapy

Speaking at the UK National Stem Cell Network annual science meeting, Professor Miguel Forte described research into a new cell therapy for chronic inflammatory conditions such as Crohn's disease. Patient's own blood cells are used to produce a type of cell - Type 1 T regulatory lymphocyte - that can reduce the extent of the disease.

Professor Forte said "T regulatory lymphocytes are amazing cells - they secrete proteins - cytokines - that dampen down the over active immune response that causes the terrible symptoms of chronic inflammatory diseases such as Crohn's. We know that treatments based on these cells can work but the challenge is to develop them in the clinic so as to maximise the benefits and minimise the risk. We must show that these cells are well tolerated and do a good job to treat the disease."

Professor Forte and his colleagues at TxCell in Valbonne, France, have used patient's own immune system cells derived from PBMCs - a type of blood cell - to treat patients with chronic inflammatory diseases like Crohn's disease. They used these cells, from patients with Crohn's, who had previously been treated with drugs and/or surgery but still had significant symptoms due to treatment resistance to make Type 1 regulatory T lymphocytes, which were then given back to the patients. The purpose of the study was to assess how well patients react in general to the treatment and also to check the efficacy of these cells for treating Crohn's disease. The preliminary results presented today show a good tolerability and, when given the correct dose, patients with severe Crohn's disease that do not respond to other treatments have an improvement in their condition.

Cell therapy approaches, like this one and also MSCs, aim at using living cells as innovative new treatments to address unmet medical needs.

Professor Forte continued "It's still early days but the preliminary results are really good. The treatment didn't make the patients ill in any way and there is an early indication that their Crohn's disease has improved. The next step will be to do what we call a "phase 2b" clinical trial to find out if the treatment definitely works, what types of chronic inflammatory disease it works for, more about any potential side effects and how to manage them, and to confirm our results on the best dose used."

Source:
Mike Davies
Biotechnology and Biological Sciences Research Council