CALABASAS HILLS, Calif., Sept. 15, 2004 (PRIMEZONE) -- The ALS Association (ALSA) today announced funding support of a new pre-clinical study at Ceregene, Inc. to help move viral delivery of insulin-like growth factor 1 (IGF-1) closer to the clinic for patients with amyotrophic lateral sclerosis (ALS).
This effort is part of several studies required for development and FDA approval of CERE-130. CERE-130 is a novel gene therapy product that involves the delivery of IGF-1 through an adeno-associated viral (AAV) vector. In partnership with The Robert Packard Center for ALS Research at Johns Hopkins and Project A.L.S., these efforts are vital to moving viral delivery of IGF-1 into clinical trials.
"I am very excited to be collaborating with Ceregene, The Robert Packard Center at Johns Hopkins and Project A.L.S in such an important effort," said Dr. Lucie Bruijn, science director and vice president of ALSA. The study is titled "Long-term toxicity and Expression of CERE-130 (AAV-IGF-1) Following Intramuscular Delivery in Rodents."
Bruijn explained that for many years, laboratory studies have suggested that growth factors (neurotrophic factors) such as IGF-1; GDNF (Glial Derived Neurotrophic Factor) and BDNF (Brain Derived Neurotrophic Factor) are important for neuron survival. However, clinical trials administering these growth factors have been disappointing thus far.
"The challenge of delivering these proteins to the site of damage is likely the underlying cause of the failures in the clinic," Bruijn said.
Ceregene's in vivo gene therapies utilize non-replicating viral vectors to deliver therapeutic genes to the nervous system, where they express proteins with the ability to prevent neurodegeneration and restore normal nerve function. The AAV vector is an important gene delivery tool which has demonstrated ability to efficiently and stably express genetic information in non-dividing target cells, such as neurons degenerating in ALS and to achieve potentially long-term therapeutic benefit.
A collaborative research team involving Fred Gage, Ph.D., and Brian Kaspar, Ph.D., at The Salk Institute for Biological Studies in San Diego, and Jeffrey Rothstein, M.D., Ph.D., of Johns Hopkins University in Baltimore, reported in the Journal Science in 2003 that IGF-1 can be efficiently transported from muscle to motor neurons using AAV as the viral transport mechanism. This discovery led to increased survival of transgenic mice expressing mutant G93A prior to and at symptom onset. Based on this encouraging data, a clinical research team at Johns Hopkins University has initiated plans for a human trial of AAV-IGF1 in ALS.
"Project A.L.S. has funded Dr. Gage's gene therapy work from the beginning," said Valerie Estess, co-founder of Project A.L.S. "It is a point of pride that we are now partnering with ALSA and The Packard Center on this project. Our triumvirate will work hard alongside Ceregene -- and on behalf of the entire community -- to deliver this potential therapy safely and effectively to trial."
"We knew from our mouse studies with Dr. Gage, and the previous clinical experience with IGF protein, that we needed to move into the clinic as soon as possible," commented Dr. Rothstein. "My colleagues and I are working closely with Ceregene and the appropriate regulatory agencies to design and implement a clinical trial as quickly as possible."
San Diego, Calif.-based biotechnology company Ceregene, Inc. (www.ceregene.com) is dedicated to the development and commercialization of therapies for neurodegenerative diseases using neurotrophic factors and gene delivery, with emphasis on Alzheimer's and Parkinson's diseases and ALS. In order to provide supporting data for an "Investigational New Drug" (IND) application with the FDA, Ceregene, Inc. is conducting a series of pharmacology and safety/toxicity studies using CERE-130. ALSA is sponsoring one of these studies to assess the safety of the therapeutic approach on long-term administration of CERE-130. This study will provide critical data on the long-term expression and toxicity of CERE-130.
"We are delighted to be working with such a dedicated and committed group of people and excited about the possibility of developing an effective treatment for ALS," said Dr. Raymond Bartus, vice president of Research and Development at Ceregene, Inc.
Completion of the mandatory pre-clinical safety/toxicity studies will guide the design of the clinical trial in ALS patients. Specifics of the clinical trial are under discussion with the appropriate, required regulatory agencies by the research team at Johns Hopkins, Massachusetts General Hospital and by Ceregene, Inc.
Additional information about the clinical trial will be available from ALSA (www.ALSA.org), Project A.L.S. (www.projectALS.org) and the Robert Packard Center (www.ALScenter.org).
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