Catalyst - Summer 2009 - The Pharmaceutilization of Stem Cells Celgene Cellular Therapeutics: On the Brink of a Breakthrough

In these days when technology makes the world spin at warp speed, it's almost hard to believe there was a time when stem cells were considered an "emerging" platform.

Yet, when his company, Anthrogenesis, was founded 11 years ago, Dr. Robert J. Hariri explained, the stem cell was still just promising science. Those in the medical community were beginning to understand the stem cell's "tremendous power."  

Dr. Hariri, now chief executive officer of Celgene Cellular Therapeutics (CCT) (since parent company Celgene acquired Anthrogenesis in December 2002), is a scientist, neurosurgeon, inventor, and businessman; he has established himself as a recognized leader in the development of new human cellular and tissue therapeutics. Prior to the acquisition, Anthrogenesis was a privately held New Jersey-based bio-therapeutics company and cord blood banking business. At the time, the company was developing technologies for the recovery of stem cells from human placental tissues following the completion of full-term, successful pregnancies.

Renamed Celgene Cellular Therapeutics, the company now operates as a wholly owned subsidiary engaged in the research, recovery culture-expansion, preservation, development and distribution of placental cells, including stem and progenitor cells, as therapeutic agents. And, when Celgene acquired the company – thanks to Dr. Hariri's vast experience – it was poised to translate the science of stem cells into a tool doctors can use in the healing process.

Dr. Hariri calls this the "pharmaceutilization of stem cells."

"In the mid 1990s, we knew that for stem cells to have a meaningful place in medicine they would have to be turned into a tool that could be used by a doctor the same way a medical device could be used," he explained. "The concept of creating a product that was a living cell that could be delivered to patients as a therapeutic was more in line with the way a drug is developed than anyone could have predicted."

This work starts with a suitable source of material and, he said, the placenta became an attractive target to pursue for stem cells because "it's readily available, a high quality human tissue that can be subjected to very rigorous standards for quality."

Plus, he pointed out, this is the place where science and business converge: turning placenta stem cells into an actual "product" is quite an attractive, economically viable proposition for the healthcare industry, Dr. Hariri added.

So, when he founded Anthrogenesis, a small biotech, he and his team set out to test the placenta hypothesis. "We were very successful early on in discovering, identifying the population of cells that could be recovered from a healthy placenta," he said. "These are high quality cells with potential clinical value. From that, we embarked on understanding how the cell functions and what specific activities were useful in a clinical way. Then, we worked on finding a way to produce those cells in a way that met the standards and requirements for the FDA."

Celgene Cellular Working to Get the Job Done 

Dr. Hariri's team of just under 200 employees at CCT's base in Warren, N.J., has been devoting considerable amount of time and money to creating a product with the highest standards for a human cellular product. The FDA accepted the company's Investigational New Drug Application for "PDA001." The designation permits clinical patient trials to determine safety and efficacy of the drug.

CCT's primary clinical cell-therapy candidate PDA001 is a novel culture expanded stem cell population that has broad therapeutic potential in areas such as immunology, inflammation, hematology and oncology, according to Wolfgang Hofgartner, M.D., DSc, and vice president of research at CCT. "PDA001 has potent immune-suppressive properties with several potential significant benefits in that the cells are derived from a safe and almost unlimited source (the placenta) and are scalable to a traditional pharmaceutical level," he said.

A Phase I multi-center clinical trial in the U.S. for patients with moderate-to-severe Crohn's disease, who are refractory to oral corticosteroids, such as prednisone, and immune suppressants, is currently ongoing. Dr. Hariri said Crohn's disease "offers an unmet medical need. It's a bad disease with a pretty good clear endpoint to follow. From our pre-clinical work, there is evidence that PDA001 would be effective."

The first half of the clinical trials, at many hospitals around the country including New York's Cedar Sinai, was recently completed. Dr. Hariri expects the entire study will be completed by the end of 2009. "At this time, we are looking into expanding the trial to additional clinical areas after the Phase 1 study." Other clinical areas might include auto-immune diseases such as Multiple Sclerosis and Rheumatoid Arthritis.

"The industry of human stem cell therapeutics is relatively new," CCT's CEO said. "Demonstrating safety and efficacy would lead to moving into a broader range of clinical areas. The hope is that stem cells would be used in a whole range of diseases including stroke, spinal cord injury, you name it."

When You Have the Right Leader… 

Dr. Hariri's work in the field of cell science spans many years. He has made his life's work the quest to bring cells into the mainstream therapeutic marketplace so we can better treat and in some cases eradicate diseases such as leukemia and other cancers, anemias, heart disease, diabetes, paralysis and neurogenerative diseases such as Parkinson's and Alzheimer's diseases.

In fact, during his tenure as chairman and CSO of Anthrogenesis, he developed proprietary technological solutions to enhance the processes involved in the collection, testing and storage of umbilical cord blood cells.

A former engineering undergraduate student turned medical student, Dr. Hariri has authored over 20 issued and pending patents in the areas of cell processing and surgical devices and techniques. He is well regarded as a scientist who continues to focus on breaking new ground in regenerative medicine by combining early cells with innovative biomaterial products to treat and cure disease.

The Science Behind CCT's Product 

The placenta, a by-product of birth, is usually discarded; however, the placenta is also a rich source of stem cells for research and therapies including hematopoietic (blood) and non-hematopoietic stem cells, said Dr. Hofgartner. "Unlike embryonic stem cells, placenta-derived stem/progenitor cells are regarded as ethically acceptable and non-controversial by most members of the public."

The expectation, he added, is that this product – PDA001 -- can fill unmet medical needs in certain diseases that traditional drugs or biologics can not fill.

At CCT, postpartum placentas from full-term healthy births are procured under informed consent at accredited hospitals, with collections performed by licensed healthcare professionals trained in the collection of placenta units. Of course, donor eligibility documentation is required and cells collected are subjected to a rigorous series of quality control tests, including serology, bacteriology and HLA typing. After collection, the placenta is processed using proprietary techniques to collect different cell populations.

"The beauty of this entire process," added Dr. Hariri, "is that this is a completely ethical solution. No one objects to placenta cells being used in healthcare solutions."

The Impact of Political Debate 

The political impact on whether stem cells are turned into medicine, in Dr. Hariri's mind, is "immaterial." Federal funding of stem cells research has very little to do with taking a technology, testing it and getting it approved, he said.

"The role that government can play in this arena is quite small. Politicians like to talk about stem cells because it's a lightning rod. It's a way to rekindle the prolife/prochoice debate. The fact is, nothing that has been done by politicians has had an impact on the development of stem cell science."

With the exception, of course, of possibly former President George W. Bush's ban on federal funding for embryonic stem cell research. Michael F. Christman, Ph.D., president and chief executive officer of the Coriell Institute for Medical Research in Camden, N.J., said Bush's ban took a "major, though perhaps not completely evident, toll on the field of stem cell research.

"Coming at a time of great promise, the ban resulted in the loss of a whole generation of young scientists who embraced embryonic stem cell research as a worthwhile path of discovery, but saw no future for it in the United States."

President Barack Obama lifted the federal ban on federal funding of stem cell research shortly after taking the presidency in January 2009. (For more on the president's actions in this area, see the related article in this issue of Catalyst.)

That being said, Dr. Hariri is convinced that industry has always been the biggest influencer and investor in stem cell research. "The process of reducing stem cells to a medicine is a process that requires industry scientists and clinical investigators to demonstrate that a product is safe and effective. I can't think of a drug product which has been approved and owes the approval to the federal government's support. It just doesn't happen that way in real life."


EisnerAmper's Catalyst: Summer 2009

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