Stem cell developers Give Cures

CORALVILLE, Iowa—Aimed at overcoming obstacles to offeringpersonalized medicine, as well as accelerating the search for effectivetreatments, human stem cell biomanufacturer Cellular Engineering TechnologiesInc. (CET), and its nonprofit arm, the John Paul II Medical Research Institute(JP2MRI), have launched a partnership to develop a private stem cell biobank tocreate more than 5,000 patient and disease-specific stem cell lines.

While CET is a for-profit biotech company that manufacturesstem cells from normal volunteers and from patients with clinical disease tofacilitate the drug discovery process for the general scientific community,JP2MRI focuses on clinical and translational research, administrates clinicalproposals and recruits patients for research protocols, but works with privatehospitals and clinics around the country to procure tissue and collect clinicaldata on patients.

The objective of this venture is to create a database ofprivate-practice physicians who will refer patients and/or serve as collectionsites to procure tissue for producing patient and disease-specific stem celllines, and to create stem cell lines that can be used by the general scientificcommunity.

"The project is ongoing and indefinite," says Dr. Alan Moy,co-founder and CEO of CET and founder and scientific director of JP2MRI. "Wecurrently have several hospitals and clinics that are participating in ourprogram. The institute launched a program called Give Cures that is recruitingpatients and doctors nationwide. The ultimate objective is to partner with andmake available data and cell lines to academia, government and industry toadvance their respective drug discovery efforts."

The cell lines for the biobank are derived solely from adultstem cells, thus sparing the partnership the controversy associated with theuse of human embryonic stem cells (hESCs). Stem cell donations to the bank willcome directly from patients recruited from private practice doctors and privatehospitals.

The project also enables drug testing on patient-specificstem cells, in contrast to the currently used models involving animal testingand clinical trials that are vastly more expensive, time-consuming and lesseffective, says Moy, who also serves as an adjunct associate professor for theUniversity of Iowa's College of Engineering.

The biobank stem cell lines will serve as models to betterpredict the outcome of drug therapy in patients and dramatically advanceresearch to bring new treatments to market sooner and at lower cost.

There is a sense of urgency to create a stem cell biobank ofhuman somatic stem cells, induced pluripotent stem cell (iPSC) lines and othercritical human cell lines. The annual rate of drugs approved by the U.S. Foodand Drug Administration (FDA) has declined, while research and developmentcosts have significantly increased, Moy points out. The cost of bringing a drugto market is currently more than $1 billion and takes more than 10 years. A newheart drug has a 20-percent chance of succeeding in a clinical trial, and a newcancer drug has only an 8-percent chance of succeeding.

"The low success rates are due to the complexity and widevariability of drug efficacy and safety in patients," Moy says. "Lead compoundsthat are tested in animal models that ultimately graduate to clinical trialshave low success rates because animal models do not predict very well the humanresponses. In particular, animal models are poorly suited for predictingbiological agents in human, e.g., antibodies."

The advantage of human stem cells is that the cell line froma patient could differentiate into a specific tissue type that would containthe disease characteristic of a patient, such as a neurological disease, Moyexplains.

"The stem cell would provide more specific predictability orintuition on drug efficacy and safety, which would decrease the time and costbecause more specific analytical processes could be used that are more costefficient," he asserts. "We have tested this hypothesis on children with raregenetic diseases and found that the drug responses in their cells correlatedwith their clinical outcomes to the drug when administered clinically."

While the main objective of the partnership is "to create apatient and physician registry, recruit patients, harvest tissue and createstem cell lines, once the lines are developed, it is anticipated that we willpartner with other stakeholders working on their own drug discovery platforms,"Moy says.

Moy remains cautiously optimistic.

"We think that our infrastructure and partnership betweenCET and JP2MRI will dramatically reduce the barriers that government andacademic biobanks typically face," he says. This includes not only "cost andtime efficiency, but red tape and bureaucracy and limited patient referralsites," he notes. "Once we achieve our business and technical milestones, weanticipate that the process will scale-up."