Gene Therapy Field Advances With Important Clinical Findings Reported in the New England Journal of Medicine
SAN DIEGO, Calif., February 03, 2009 /PRNewswire-FirstCall/ -- Cardium Therapeutics today reported on recent clinical findings published in The New England Journal of Medicine showing the long-term outcome of a method of gene therapy to treat infant children with a fatal disorder of the immune system. The infant children studied had a rare form of severe combined immunodeficiency (SCID). SCID arises in infants with a genetic defect that leaves them unable to produce an enzyme called adenosine deaminase. This key enzyme is most active in lymphocytes, which are white blood cells vitally important for protection against infection by bacteria, viruses and fungi. Infants who inherit this fatal genetic abnormality develop impaired immune responses, leading to recurrent infections and a failure to thrive. Without adequate treatment, these children typically die within one year of birth.
The New England Journal of Medicine (NEJM) reported on the success of a DNA-based approach for the introduction of a normal adenosine deaminase gene into the patient's hematopoietic stem cells that have been removed from the bone marrow. After transfection of the cells with the gene, the cells are re-infused back into the patients after nonmyeloablative conditioning with busulfan. After a single treatment, all 10 children in the study survived and gene therapy was not associated with any adverse events during a median follow-up of 4 years. In 8 of the 10 patients, there was excellent and persistent immune reconstitution and protection from infection.
In an accompanying NEJM editorial entitled, Gene Therapy Fulfilling Its Promise, the authors noted, "The prospects of continuing advancement of gene therapy to wider applications remain strong. Ongoing and upcoming clinical trials will use safer designs of retroviral vectors, newer types of vectors for viral gene therapy, and emerging methods for direct in situ gene repair. These approaches to the treatment of hemoglobinopathies, hemophilia, muscular dystrophy, congenital retinopathies, neurodegenerative disorders, and other genetic diseases may further fulfill the promise that gene therapy made two decades ago."
"Historically, gene therapy has been long on promises and, all too often, short on clinical results. The success of this recent study marks an important milestone for the field, provides important insights into the use of advanced gene therapies, and sheds new light on the promise and new therapeutic realties of DNA-based approaches for the treatment of disease," stated Christopher J. Reinhard, Chairman and Chief Executive Officer of Cardium Therapeutics. "Cardium is developing a new class of growth factor biologics designed to take advantage of the body's own natural healing abilities for the regenerative treatment of ischemic conditions, such as chronic wounds, myocardial ischemia and heart attacks, and we look forward to completing our Phase 2b MATRIX gene therapy clinical trial that is evaluating the safety and efficacy of Excellarate for the potential treatment of non-healing diabetic foot ulcers."
Cardium Therapeutics, Inc. and its subsidiaries, InnerCool Therapies, Inc. and the Tissue Repair Company, are medical technology companies primarily focused on the development, manufacture and sale of innovative therapeutic products and devices for cardiovascular, ischemic and related indications.
Cardium's InnerCool Therapies subsidiary is a San Diego-based medical technology company in the emerging field of temperature modulation therapy to rapidly and controllably cool the body in order to reduce cell death and damage following acute ischemic events such as cardiac arrest or stroke, and to potentially lessen or prevent associated injuries such as adverse neurological outcomes. For more information about Cardium's InnerCool subsidiary and patient temperature modulation, including InnerCool's new RapidBlue(TM) System, which just received FDA clearance, and its CoolBlue(TM) System, please visit http://www.innercool.com.
Cardium also has two biologic candidates in clinical development. Cardium's Tissue Repair Company subsidiary (TRC) is focused on the development of growth factor therapeutics for the treatment of severe chronic diabetic wounds. TRC's lead product candidate, Excellarate(TM), is a DNA-activated collagen gel for topical treatment formulated with an adenovector delivery carrier encoding human platelet-derived growth factor-BB (PDGF-BB). Excellarate(TM) is initially being developed to be administered once or twice for the potential treatment of non-healing diabetic foot ulcers. Other potential applications for TRC's Gene Activated Matrix(TM) (GAM) technology include therapeutic angiogenesis (cardiovascular ischemia, peripheral arterial disease) and orthopedic products, including hard tissue (bone) and soft tissue (ligament, tendon, cartilage) repair. For more information about Cardium's Tissue Repair Company subsidiary, please visit http://www.t-r-co.com.
Cardium's Generx product candidate (alferminogene tadenovec, Ad5FGF-4) is a DNA-based growth factor therapeutic being developed for potential use by interventional cardiologists as a one-time treatment to promote and stimulate the growth of collateral circulation in the hearts of patients with ischemic conditions such as recurrent angina.
Except for statements of historical fact, the matters discussed in this press release are forward looking and reflect numerous assumptions and involve a variety of risks and uncertainties, many of which are beyond our control and may cause actual results to differ materially from stated expectations. For example, there can be no assurance that results or trends observed in one clinical study or procedure will be reproduced in subsequent studies or procedures, that human clinical trials can be conducted and completed in an efficient and successful manner, that FDA, CE Mark or other regulatory clearances or UL or other certifications, or partnering or other distribution agreements or other commercialization efforts will be successful or will effectively accelerate the business or market, that product modifications or launches will be successful or that the resulting products will be favorably received in the marketplace, that our products or product candidates will prove to be sufficiently safe and effective, that necessary regulatory approvals will be obtained, that third parties on whom we depend will perform as anticipated, or that our products or product candidates will not be unfavorably compared to competitive products that may be regarded as safer, more effective, easier to use or less expensive. Actual results may also differ substantially from those described in or contemplated by this press release due to risks and uncertainties that exist in our operations and business environment, including, without limitation, risks and uncertainties that are inherent in the development of complex biologics and therapeutic hypothermia devices and in the conduct of human clinical trials, including the timing, costs and outcomes of such trials, our ability to obtain necessary funding, regulatory approvals and expected qualifications, our ability to successfully accelerate the commercialization of our therapeutic hypothermia devices and launch new devices within the timeframes contemplated, our dependence upon proprietary technology, our history of operating losses and accumulated deficits, our reliance on collaborative relationships and critical personnel, and current and future competition, as well as other risks described from time to time in filings we make with the Securities and Exchange Commission. We undertake no obligation to release publicly the results of any revisions to these forward-looking statements to reflect events or circumstances arising after the date hereof.
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CONTACT: Bonnie Ortega, Director, Investor/Public Relations of CardiumTherapeutics, Inc., +1-858-436-1018, InvestorRelations@cardiumthx.com
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Posted: February 2009