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Blazing the trail
September 2011
EDIT CONNECT
SHARING OPTIONS:
A number of
biotechnology firms dedicated to stem cells have been launched in the past few
years,
thanks to strong venture capital funding in geographic areas where
government and science have favored stem cell research, according to Frost
& Sullivan.
Notably,
California ranks highest, with a total of 10 firms dedicated to all kinds of
stem cell research receiving either venture or IPO-backed
funding. It's
interesting to note that Geron Corp., which works
with hESCs, has the maximum
market capital value at $605 million, despite the fact that adult stem cell
research receives more funding.
Geron, located
in Menlo Park, Calif., is considered by many to be the top biotechnology
company in the field of
stem cell research. Since its founding in 1990, Geron
has been granted more than 260 patents. The company has two anti-cancer
products in human
clinical trials. In 2009, Geron received FDA approval to
begin Phase I
testing in humans of GRNOPC1, an eSC-based drug designed to treat
specific forms of spinal cord injury through remyelination of damaged axons.
Geron
also has several other embryonic stem cell treatments that are still in
the preclinical phase, including GRNCM1, a treatment for heart disease, and
GRNIC1, a treatment for diabetes.
Here, we feature
a handful of other companies that stand out as
leaders in this market,
according to Frost & Sullivan.
Company: Aastrom
Biosciences Inc.
Founded: 1989 Location: Ann Arbor, Mich. Employees: 65 Focus: Developing enhanced autologous cellular therapies for the treatment of severe, chronic cardiovascular diseases ![]() Unlike some of
the other companies operating in the
same space, Aastrom manufacturers the
autologous cells it uses in-house. A small quantity of bone marrow is taken
from a patient's hip, and in a 12-
day process, Aastrom expands the cell
populations found in the extracted marrow. Then, in a single inpatient
procedure, the expanded mixed-cell therapy
produced from this process—called
ixmyelocel-T—is administered to the same patient to promote healing of the
affected tissues.
"We have
designed our own highly automated, fully closed cell processing system,"
explains Tim Mayleben, Aastrom's CEO,
president and director. "It is the best
device in the world for processing autologous cells. And our success has been
to think both strategically and
medically about the kinds of diseases our
particular technology is well-suited for."
Specifically,
those are critical limb ischemia and dilated cardiomyopathy—critical
cardiovascular diseases that currently have limited or no treatment options.
The
company is in a Phase III clinical trial to study ixmyelocel-T patients
with critical limb ischemia and two Phase II clinical trials to study the
therapy in patients with dilated cardiomyopathy.
"These chronic,
long-term, progressive diseases are
well-suited to our technology," says
Mayleben. "There are literally no treatment options for these patients, and of
the options that are available—
amputation, heart transplant, etc.—these are
limited in availability and expensive. Our vision at Aastrom is to get the
first autologous cell therapy
product in physicians' hands and used by
patients, so we can change the way that medicine is practiced. Our technology
also has applications in other
disease areas that we are just now starting to
explore."
Company: Advanced Cell Technology (ACT)
Founded: 1994 Location: Santa Monica, Calif., and Marlborough, Mass. Employees: 32 Focus: Retinal degeneration; blood and cardiovascular diseases ![]() First, the
company is focused on commercializing its hESC-based Retinal Pigment Epithelial
(RPE) therapy for degenerative
retinal disease, for which it recently initiated
two Phase I/II clinical trials. Retinal degeneration can cause a variety of
blinding diseases,
including retinitis pigmentosa, Stargardt's disease
(juvenile macular degeneration), age-related macular degeneration (AMD) and
other degenerative
diseases of the retina.
"One of the
great things about working with the eye is that you don't have
the immune
rejection issues you would find in other parts of the body," says Gary Rabin,
chairman and CEO of ACT.
ACT is
particularly focused on AMD, which afflicts more than 30 million people
worldwide and is the leading cause of
blindness in people aged 60 and older in
the United States. And as average life expectancy continues to rise, it is
predicted that the incidence of AMD
will only continue to rise in tandem. Dry
AMD, the most prevalent form of the disease, represents a $25 billion to $30
billion market in the United
States and Europe alone, and there are currently
no approved therapies available for this condition.
On
July 14, ACT
announced the dosing of the first patients in each of its two Phase I/II
clinical trials for Stargardt's macular dystrophy and dry AMD
using RPE cells
derived from hESCs. The patients were treated by Dr. Steven Schwartz, retina
division chief at UCLA's Jules Stein Eye Institute. Both patients successfully
underwent the outpatient transplantation surgeries and are recovering
uneventfully.
"It would be
great if the end result of these clinical trials is if we arrest the
decline of
age-related macular degeneration," says Rabin. "The math works out readily to
see how this could be the largest drug therapy of all time—
this could be bigger
than Lipitor."
ACT is also
developing its hESC-based Hemangioblast (HG)
platform for the treatment of
blood and cardiovascular diseases in a partnership with CHA Biotech of Korea.
Finally, the company is focused on advancing its Phase II-
approved Myoblast
autologous adult stem cell therapy for the treatment of chronic heart failure,
advanced cardiac disease, myocardial infarction and
ischemia.
ACT owns or
licenses more than 150 patents and patent applications related to stem
cell therapy and
regenerative medicine. The company also recently announced a collaboration with
Roslin Cells for the storage and distribution of eSCs using ACT's Blastomere
technology.
Company: Aldagen
Founded: 2002 Location: Research Triangle Park in Durham, N.C.
Employees:
16
![]() In 2002,
Aldagen's founders took technology developed at Johns Hopkins University and
Duke University and started a commercial enterprise. The technology identifies,
selects and isolates specific adult stem cells that
express high levels of the
enzyme ALDH, which plays an important role in controlling the developmental
state of stem and progenitor cells. It converts
vitamin A into retinoic acids,
which regulate genes and influence the differentiation of blood, neural, endothelial
and other types of stem and
progenitor cells.
Because Aldagen
uses this technology to collect autologous cells
from a patient's own bone
marrow, the risk of potential rejection of the injected cells in a patient is
minimalized, says Edward L. Field, the
company's chief operating officer.
"When you
compare us to other companies, we have a
unique cell population that no one
else has," Field says. "What we do is very different, but also very simple. We
don't manipulate, culture or
expand cells, so we are able to fairly rapidly
produce and deliver them to the patient."
Compared to
some
of the other companies we are featuring here, Aldagen is fairly young, having
operated for less than 10 years. Field attributes the company's
youth to "the
fact that a lot of us are trying to do what has never been done before."
"If you
think
about commercializing new technology in our industry, it usually takes 20 to 25
years from the seminal event to get commercial products to
market, and stem
cells are no different," he says.
Companies like
Aldagen will be able to realize
the promise of this groundbreaking research "if
we can achieve the next milestone in stem cell research, which is putting Phase
III studies and data
sets on the table," says Field.
Aldagen is
headed in that direction, as it is beginning Phase II
studies involving
therapeutic candidates for critical limb ischemia and ischemic heart failure.
Also, in May, the company—along with its collaborator,
the Los Angeles Brain
and Spine Institute (LABSI)—
announced the enrollment of the first subject in a
clinical trial that studies ALD-401, a unique stem cell population derived from
a patient's own
bone marrow, for the treatment of ischemic stroke.
"The stroke
market alone presents billion-dollar
market opportunities," says Field,
"because there are no therapeutic options or small-molecule drugs to treat it.
In addition, our therapy is a
regenerative, restorative kind of therapy that
has the potential to take costs out of the healthcare system by keeping
patients ambulatory. We have
talked to payors, and they are very encouraging as
we move this program forward."
Company: Neuralstem Inc.
Founded: 1996
Location: Rockville, Md., and San Diego; plans to
open subsidiary in
China soon Employees: 16
Focus: Using its patented neural stem cell technology to treat central nervous system diseases ![]() Nearly 20 years later, Garr is the president and CEO of Neuralstem Inc., a publicly traded biotherapeutics company that is making headlines for its stem cell clinical trials to treat amyotrophic lateral sclerosis, (ALS, commonly known as Lou Gehrig's disease) and depression—and eventually, other diseases of the central nervous system (CNS). Bonded to the
father of one of his son's classmates, Dr. Karl Johe—a staff scientist at the
Laboratory of Molecular Biology of the
National Institute of Neurological
Disease and Stroke—
Garr saw an opportunity to alleviate Matt's suffering. In
1996, he and Johe joined forces to commercialize Johe's Human Neural Stem Cell
technology.
The technology
allows for the isolation of CNS stem cells from tissue; the expansion, in vitro, of each
cell up to a
billion-billion times, or 60 doublings; and the controlled differentiation of
the cells into mature, physiologically relevant human
neurons and glia that can
be used to treat CNS diseases and conditions. This provides a unique window for
traditional drug discovery and genomic
applications, Garr says.
"We screen against ourselves, and we screen against physiologically relevant human cells, so we can really see what's actually happening, which is predictive of what will happen in humans," he explains. Neuralstem has 14 patents to its name—issued all over the world—with more than 20 others pending. The company's work with stem cells is evenly divided between using them to develop actual therapeutics and to screen against for drugs. The company
currently has two Phase I
clinical trials underway. The first is evaluating the safety of Neuralstem's spinal cord stem cells in the treatment of ALS, and notably, is the first ALS stem cell trial to be approved by the FDA. In June, Neuralstem announced that 12 patients being treated at Emory University in Atlanta safely received injections in the lumbar region of the spine, prompting the trial's Safety Monitoring Board (SMB) to unanimously approve advancing the trial to transplantations in the cervical region. Neuralstem hopes these injections can help alleviate the breathing and swallowing difficulties that often lead to death in ALS patients.
Neuralstem's
second Phase I trial is testing
NSI-189, the lead compound in the company's
neuroregenerative small-molecule drug platform, for the treatment of major
depression. Phase Ia is testing
the drug in healthy volunteers for safety and
tolerability.
Neuralstem hopes to conclude both trials within the next five years, and is preparing to do a stroke trial in China. In fact, the company has several projects in the works that will give it a significant presence in Asia as well as the United States. "We have a fairly global ambition in terms of rolling out products and studies, and we're growing our infrastructure in that direction," says Garr. RELATED STORIES:
Staking a claim
Growing stem cell market entices Big Pharma to harness its power for drug screening, toxicity testing A roundup of North America's top five
academic research
institutions in the stem cell arena
ONLINE BONUS:
Tools of the trade Life science tool providers provide constant support to ever-evolving stem cell research community Code: E091128 Back |
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