Healthcare Industry News: gene therapy
News Release - December 12, 2006
Northern Therapeutics and MaxCyte Initiate Landmark Engineered Cell Therapy Trial for Fatal Lung DiseaseFirst Patient Treated in PHACeT Study at St. Michael's Hospital in Toronto
TORONTO, and GAITHERSBURG, Md., Dec. 12 (HSMN NewsFeed) -- Northern Therapeutics, Inc., a Canadian biotechnology company, and MaxCyte, Inc., a U.S. therapeutics company, announced today the commencement of the first-ever human trial of a novel, engineered cell-based therapy for the treatment of idiopathic (primary) pulmonary arterial hypertension (PAH), a rare but fatal cardiovascular disease that can strike in the prime of a person's life. The first patient in the trial received therapy at St. Michael's Hospital on November 7, 2006, and the procedure was well tolerated.
The trial, called PHACeT (Pulmonary Hypertension: Assessment of Cell Therapy), is the first to use genetically engineered progenitor cells, which are similar to adult stem cells* but act in different ways, for the treatment of a cardiac or vascular condition. For the two companies, the trial represents the fruition of a successful partnership to develop a cellular treatment for PAH.
"There is a tremendous unmet need for effective therapies to treat this devastating disease, and using engineered cell-based therapy to regrow and repair blood vessels in the lungs is a new approach to treatment," said Dr. Duncan Stewart, Chief Scientific Officer of Northern Therapeutics. "We anticipate that this trial's results will lay the foundation for further research and bring us closer to a therapy that reverses PAH, instead of merely addressing its symptoms as current treatments do."
PHACeT is an open-label, dose-ranging Phase IIa trial that will enroll up to 18 patients with PAH at two sites in Canada in a careful dose-escalation protocol. In the novel clinical therapy used in the trial, engineered progenitor cells are injected into the lung to repair and regenerate damaged blood vessels, and potentially reverse the progress of the disease. Expected to be completed within 18 months, the trial is being conducted at St. Michael's Hospital in Toronto and the Jewish General Hospital in Montreal. The trial's primary endpoint is safety and tolerability, but data will also be collected to establish the potential efficacy of this innovative treatment.
In the PHACeT trial, stem cell-like "endothelial progenitor cells" (EPCs) are harvested from the patient's own blood. These cells are then engineered using a DNA vector containing the plasmid for endothelial NO-synthase (eNOS), an enzyme needed for the production of nitric oxide. People with PAH exhibit low production of nitric oxide, which is a vasodilator that also plays a key role in the growth and repair of blood cells.
During the procedure, the eNOS plasmid is loaded into the patient's EPCs using a non-viral delivery process called electroporation. A defined electrical field is generated around the cells that allows the eNOS plasmid to be introduced. For this study, Northern Therapeutics has partnered with MaxCyte, which provides expertise and a technology solution that allows for safe and effective cell engineering that maximizes cell loading while minimizing cell stress and damage to the cells. A day after processing, the eNOS-engineered cells are delivered via catheter to the heart and pulmonary arteries of the patient from whom they were harvested.
"We are excited about working with Northern Therapeutics to help move its PHACeT study into clinical trials. Our technology has been shown to be extremely safe and efficient, and it is capable of producing cell-based therapeutics that are highly reproducible in quantities appropriate for clinical trial and commercial use," said Douglas Doerfler, President and CEO of MaxCyte.
About Pulmonary Arterial Hypertension (PAH)
PAH is a life-threatening disease that affects the blood vessels in the lungs. PAH is characterized by the deterioration of blood vessel walls, the accumulation of platelets and the disruption of smooth muscle cell function. These conditions cause blockages that impact the ability of the blood vessels to function properly, thereby impairing blood flow between the heart and the lungs. The result is an elevation in pulmonary blood pressure, which causes an increasing strain on the right side of the heart as it tries to pump blood to the lungs. There is currently no cure for PAH, and patients die, on average, within five years of their diagnosis. The disease affects young adults, primarily women.
About Northern Therapeutics
Northern Therapeutics is a privately held Canadian biotechnology company whose mission is to develop a unique cell and gene therapy platform technology for the therapy of chronic and life-threatening cardiopulmonary diseases.
MaxCyte is a clinical-stage cell therapeutics company with a rapidly growing pipeline of product development partnerships in cell-based therapies. The Company's proprietary ex vivo cell loading technology overcomes critical obstacles such as safety, scalability and reproducibility, which are fundamental to successful cell-based therapies. MaxCyte has demonstrated the value of its versatile technology in partnered therapeutic programs in oncology, pulmonary, metabolic and infectious diseases as well as in development collaborations with leading researchers: one in Phase I/II clinical trials for treatment of chronic lymphocytic leukemia (CLL) and several preclinical programs in oncology and regenerative medicine. More than 16 commercial and academic partners are currently using the MaxCyte technology. The MaxCyte system has a Food and Drug Administration (FDA) Master File in place at the Center for Biologics Evaluation and Research (CBER).
* Please note: This research does NOT involve fetal or embryonic tissue.
Source: Northern Therapeutics
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