Unleashing the Promise - Pharmaceutical Executive


Unleashing the Promise
An up-close look at some companies that may soon bring stem cell-based therapies to market.

Pharmaceutical Executive

Financial status Aastrom has a sufficient amount of cash ($35 million, based on December 2004 pro forma results) to ensure coverage of operational and clinical costs through 2006. The company has also been vigilant about patenting its processes, devices, and clinical systems. "The potential market size for adult stem cell–based therapeutics is enormous. We have chosen to initially focus on the orthopedics and vascular markets," Anderson says.

Chris Calhoun, CEO, MacroPore Biosurgery
Celfcure Ajay Bakshi, MD, president of the biotech start-up Celfcure, is an experienced neurosurgeon, instructor, and faculty researcher at the Drexel University College of Medicine in Philadelphia. Much of his research focuses on using a patient's own stem cells to treat trauma, such as spinal cord and head injuries, and stroke.

Winning science In March 2004, Bakshi and his team took first place for best venture in the Babcock Elevator Competition at Wake Forest University School of Management. Then the following month, the team placed second in the highly competitive Wharton Business Plan competition. These accolades are based on sound science. Bakshi has developed a therapeutic system called Manipulated Autologous Stem Cell Therapy (MAST), which works by harvesting stem cells from patients' bodies, culturing and loading them with drugs in the laboratory, then re-transplanting them back to the patient for therapy.

According to Bakshi, "The MAST model is based on bridging the gap between scientific research labs, where cell culturing is traditionally handled, and hospitals, where clinical procedures take place." Bakshi's prize-winning process is not dependent on isolation of stem cells. Rather, using a minimally invasive procedure, Bakshi removes bone marrow from a patient's groin, expands the cells using the Aastrom's ARS and enhances them with nanoparticles loaded with drugs to achieve sustained drug release.

Stem Cell Primer
Into the clinic Bakshi is currently filing patents for the procedures and anticipates initiation of clinical trials in 2005. He stresses that the most important issue for him is not the strength of his intellectual property portfolio, but the biological soundness of the procedure. "Brain tissue is very sensitive, so the ability to use autologous stem cells is a big deal in central nervous system [CNS] transplantation," Bakshi says. "Using the lumbar puncture technique and injecting the cells into the cerebral spinal fluid allows the cells to migrate throughout the CNS and hone in on injured areas." At press time, Celfcure had more than $1 million in grants, including university and state support. "I see Celfcure diversifying into various areas of CNS and spinal injuries, including head injury and neonatal hypoxia," Bakshi says.

Business model Celfcure recently entered into a collaboration with Thomas Jefferson Hospital, the largest spinal cord injury center in the United States. "In the field of stem cell-based therapeutic development, one of the problems is that there isn't a model to make money from stem cell-based therapies," Bakshi says. "But I argue that if the science is sound, you can make money. Celfcure will make money by selling a service, rather than selling cells. It's a more robust business model."

BioE In May 2004, St. Paul, Minnesota–based BioE, a development stage biotech, announced a significant breakthrough in stem cell research. The company, which develops antibody-based diagnostic and therapeutic technologies, isolated and defined a unique population of stem cells with properties that distinguish them from embryonic hematopoietic (blood-forming) cells and bone marrow-derived adult stem cells, as well as other multipotential adult progenitor cells. In the course of their research, BioE scientists discovered that these cells, called multilineage progenitor cells (MLPCs), demonstrate self-renewal capabilities as well as the ability to expand exponentially and differentiate into a wide variety of tissue types, including bone, nerve, and muscle—a range much more extensive for adult stem cells than previously thought.


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