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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Neuralstem ALS Trial In Multiple Presentations At Upcoming American Academy Of Neurology Meeting

Neuralstem, Inc. (NYSE Amex: CUR) announced that the Phase I safety trial of its human spinal cord stem cells (HSSCs) in amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) is the subject of three presentations at the American Academy of Neurology (AAN) Annual Meeting, April 9-16th, in Honolulu, HI (click here for more information). Chief among these will be a presentation by Eva Feldman, M.D., Ph.D., who is an unpaid consultant to Neuralstem and the Principal Investigator in the ongoing ALS trial, entitled: "A Phase I, Open-Label, First-In-Human Feasibility and Safety Study of Human Spinal Cord-Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis," at 2:00 PM, April 11th, (click here for more information). Dr. Feldman, who is also Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System, will present interim safety data on the first nine patients in the study.

Dr. Feldman's presentation was also chosen to be highlighted in the "Scientific Program Highlights Plenary Session" mediated by the Chair of the AAN Science Committee at 5:15 on April 15th. Inclusion in this session is by invitation only; Dr. Feldman's presentation was considered by the committee to be in the top 5% of submitted abstracts, which numbered in the thousands.

"We are thrilled to receive this level of interest and review from our peers at this prestigious neurology conference," Dr. Feldman commented. "As a doctor who sees ALS patients in the clinic, I am particularly eager to share this promising research with the scientists and clinicians of the American Academy of Neurology who continue to battle this difficult disease for their patients."

In addition to Dr. Feldman's presentation, two others will focus on the Neuralstem ALS stem cell trial. Dr. Jonathan Glass, M.D., professor of neurology, Emory School of Medicine and Director of the Emory ALS Center, will join Dr. Nicholas Boulis, M. D., associate professor of neurosurgery at Emory School of Medicine, and Dr. Feldman for a presentation entitled: "Intraoperative Evoked Potentials for Monitoring Spinal Cord Stem Cell Injection in Patients with ALS," at 2:00 on April 14th. Dr. Glass is the site Principal Investigator for the trial and Dr. Boulis is performing the surgeries. The team will be joined by neurologist Seward Rutkove, MD, Chief of the Division of Neuromuscular Disease at Beth Israel Deaconess Medical Center, to present "Electrical Impedance Myography in a Safety Study of Fetal Stem Cell Implantation in Amyotrophic Lateral Sclerosis," at 5:00 on April 14th. Dr. Rutkove's innovative work in EIM, which uses electrical currents to measure muscle changes in ALS patients, was just awarded a $1 million Prize4Life award . Neuralstem's trial is the first ALS study to utilize this breakthrough technology.

About the Trial

This Phase I trial, which is evaluating the safety of the cells and the surgery procedure, is designed to enroll up to 18 ALS patients, at varying stages of the disease progression. 11 patients have been treated so far. The first six were non-ambulatory. Of these, the first three received five unilateral injections of HSSCs in the L2-L4 lumbar segments of the spinal cord. The next three received 10 bilateral injections. All of the subsequent patients were ambulatory. The first three of these received five unilateral injections in the L2-L4 lumbar segments. The next two have received 10 unilateral injections in the same region. Future patients will all be ambulatory with sequential levels of disability. If approved by the FDA and the trial's safety monitoring board (SMB), the trial will progress to cervical transplantation.

Source:
Deanne Eagle
Neuralstem, Inc.

 

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