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Stem cell developers Give Cures
February 2013
by Lori Lesko  |  Email the author


CORALVILLE, Iowa—Aimed at overcoming obstacles to offering personalized medicine, as well as accelerating the search for effective treatments, human stem cell biomanufacturer Cellular Engineering Technologies Inc. (CET), and its nonprofit arm, the John Paul II Medical Research Institute (JP2MRI), have launched a partnership to develop a private stem cell biobank to create more than 5,000 patient and disease-specific stem cell lines.  
While CET is a for-profit biotech company that manufactures stem cells from normal volunteers and from patients with clinical disease to facilitate the drug discovery process for the general scientific community, JP2MRI focuses on clinical and translational research, administrates clinical proposals and recruits patients for research protocols, but works with private hospitals and clinics around the country to procure tissue and collect clinical data on patients.  
The objective of this venture is to create a database of private-practice physicians who will refer patients and/or serve as collection sites to procure tissue for producing patient and disease-specific stem cell lines, and to create stem cell lines that can be used by the general scientific community.  
"The project is ongoing and indefinite," says Dr. Alan Moy, co-founder and CEO of CET and founder and scientific director of JP2MRI. "We currently have several hospitals and clinics that are participating in our program. The institute launched a program called Give Cures that is recruiting patients and doctors nationwide. The ultimate objective is to partner with and make available data and cell lines to academia, government and industry to advance their respective drug discovery efforts."  
The cell lines for the biobank are derived solely from adult stem cells, thus sparing the partnership the controversy associated with the use of human embryonic stem cells (hESCs). Stem cell donations to the bank will come directly from patients recruited from private practice doctors and private hospitals.  
The project also enables drug testing on patient-specific stem cells, in contrast to the currently used models involving animal testing and clinical trials that are vastly more expensive, time-consuming and less effective, says Moy, who also serves as an adjunct associate professor for the University of Iowa's College of Engineering.  
The biobank stem cell lines will serve as models to better predict the outcome of drug therapy in patients and dramatically advance research to bring new treatments to market sooner and at lower cost.   There is a sense of urgency to create a stem cell biobank of human somatic stem cells, induced pluripotent stem cell (iPSC) lines and other critical human cell lines. The annual rate of drugs approved by the U.S. Food and Drug Administration (FDA) has declined, while research and development costs have significantly increased, Moy points out. The cost of bringing a drug to market is currently more than $1 billion and takes more than 10 years. A new heart drug has a 20-percent chance of succeeding in a clinical trial, and a new cancer drug has only an 8-percent chance of succeeding.
"The low success rates are due to the complexity and wide variability of drug efficacy and safety in patients," Moy says. "Lead compounds that are tested in animal models that ultimately graduate to clinical trials have low success rates because animal models do not predict very well the human responses. In particular, animal models are poorly suited for predicting biological agents in human, e.g., antibodies."  
The advantage of human stem cells is that the cell line from a patient could differentiate into a specific tissue type that would contain the disease characteristic of a patient, such as a neurological disease, Moy explains.  
"The stem cell would provide more specific predictability or intuition on drug efficacy and safety, which would decrease the time and cost because more specific analytical processes could be used that are more cost efficient," he asserts. "We have tested this hypothesis on children with rare genetic diseases and found that the drug responses in their cells correlated with their clinical outcomes to the drug when administered clinically."  
While the main objective of the partnership is "to create a patient and physician registry, recruit patients, harvest tissue and create stem cell lines, once the lines are developed, it is anticipated that we will partner with other stakeholders working on their own drug discovery platforms," Moy says.  
Moy remains cautiously optimistic.  
"We think that our infrastructure and partnership between CET and JP2MRI will dramatically reduce the barriers that government and academic biobanks typically face," he says. This includes not only "cost and time efficiency, but red tape and bureaucracy and limited patient referral sites," he notes. "Once we achieve our business and technical milestones, we anticipate that the process will scale-up."  
Code: E021323



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