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.
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.
Chris Calhoun, CEO, MacroPore Biosurgery
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
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.
Stem Cell Primer
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."
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.