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 Staking a claim
September 2011
SHARING OPTIONS:
According to
market research firm Frost & Sullivan, Big Pharma has been cautious in
approaching stem cell research, but companies have accelerated their research
presence and investment in stem cells since President Barack Obama lifted
restrictions on government funding of human embryonic stem cell (hESC) projects.
Since that time, there has been an increase in Big Pharma’s efforts in this
area, primarily in the way of drug screening and toxicity testing, says Frost
& Sullivan.
“Big Pharma
adopted a cautious role by engaging in strategic alliances to develop and
commercialize stem cell-based products,” says Frost & Sullivan analyst
Sandhya Kamath. “Autologous stem cells, screening drugs with stem cells,
induced pluripotent stem cells and stem cell lines are some areas where pharma
is concentrating their efforts and submitting several patents.”
Corporate
venture funds of pharma companies have been supporting stem cell efforts from
research institutes and companies, which could be with a strategic interest in
using startup companies they fund for research collaboration or future mergers
and acquisitions, adds Kamath.
Company: Novartis
AG
Focus: Neuromuscular disorders
 The Novartis
Institutes for BioMedical Research (NIBR), the global pharmaceutical research
organization for Novartis, says it is “committed to discovering innovative
medicines that treat disease and improve human health.” Much of the NIBR’s work
has focused on “pioneering a scientific approach that focuses on discovering
and developing medicines for diseases with significant unmet medical needs for
which the underlying mechanism of disease is known.”
Much of the
NIBR’s work with stem cells has focused on the use of hESCs, while
acknowledging that “our understanding of these cells is at an early stage; yet,
they hold immense potential for combating diseases such as Parkinson's,
Alzheimer's and diabetes.”
With these
concerns in mind, Novartis in 2002 established an ethics committee to assess
preclinical research projects involving human embryos and hESCs in order to
ensure compliance with the company's internal ethical guidelines. The committee
is comprised of external, independent specialists to oversee the company's
research efforts in the field of stem cell research and to advise Novartis’
executive committee on all bioethical issues of relevance for the company.
“Novartis
acknowledges that human embryonic stem cell research is an evolving field and
one characterized by ethical complexities,” the company says on its website.
“The role of our ethics committee is to help us establish criteria and
procedures which strike the right balance between freedom of research and the
legitimate demands of society.”
“Induced
pluripotent stem cells and motor neurons derived from those cells provide
cellular models to study disease etiology and help to identify and validate new
therapies,” says Tewis Bouwmeester, executive director of developmental and
molecular pathways at the NIBR.
Bouwmeester
explains that SMA is caused by a single gene defect in the SMN1 gene, which
produces the SMN protein, a lack of which leads to splicing errors. Some
patients experience progressive paralysis caused by the loss of motor neurons
in the spinal cord.
“Such new
research insights could potentially lead to better understanding of other
neurodegenerative diseases, such as Alzheimer’s disease,” Bouwmeester notes.
Location: Basel, Switzerland
Focus: Toxicology and drug development
 Although stem
cell research represents a small part of Roche’s global R&D efforts, the
company has several collaborations underway with outside partners involving
toxicological and safety tests and screening in various human stem cell lines.
Kyle Kolaja,
director and global head of predictive toxicology screens and investigative
toxicology screens for Roche in the United States, says Roche’s goal is to
“improve drug development while reducing animal testing and potential serious
adverse events in human trials.”
“We provide
external partners with compounds from our drug library for testing on hESCs,”
Kolaja says. “We think it is very meaningful to help identify compounds that
will have a better safety profile and a lower-stage attrition. If we can
identify and weed out bad-acting compounds and poor patient response, we can
screen them out early.”
For example,
Roche recently entered into a partnership with Cellular Dynamics International
Inc. to test whether potential new drugs damage heart tissue using stem cells.
“We are looking
for a better model of cardiotoxicity,” Kolaja says. "If you look at what’s
available even in a stem cell-derived model of the heart, they are either
rodent-based or don’t beat. A lot of them will undergo cell division. That
doesn’t happen in a normal heart. This slows it down to near or no
proliferation rate. We can already see that this cell is behaving very
differently. If you look at stem cell-derived cardiomyocytes, they beat at
about 40 beats per minute and don’t proliferate. That’s a very high level.”
In June, Roche
announced an agreement to provide the latest-generation microarray systems,
high-throughput screening instruments, genetic expression profilers and exome
sequencing technologies to cancer researchers at UCLA. Roche also serves on the
steering body of the U.K. consortium Stem Cells for Safer Medicines (SC4SM),
and has ongoing collaborations with Harvard University, Massachusetts General
Hospital and the Institute for Stem cell Therapy and Exploration of Monogenic
Diseases (I-STEM) in Paris.
All of these
efforts will lead Roche down a path to realizing the promise of personalized
medicine—which has become a buzzword in the pharma realm in recent years, says
Kolaja.
“To me,
personalized medicine means getting the right dose of the right drug to the
right patient at the right time,” he says. “In doing so, this will link all of
the arms of the Roche group together, from our diagnostic arm, to our applied
science arm and to our pharma division.”
Company: GlaxoSmithKline
PLC (GSK)
Focus: Therapeutic agents; regenerative medicine
 For U.K.-based
GSK, stem cells are viewed as complementary to many of the pharma’s drug
discovery platforms. This means the industry could be able to test compounds in
human cells instead of non-human cells, thereby improving the prediction of
preclinical screens for efficacy and safety, a spokesman tells ddn.
“They have the
notable advantage that potentially they can be used to produce multiple mature
human cell types that previously were either impossible or difficult to obtain,
such as neurons or cardiomyocytes,” GSK says. “We are also excited about the manipulation
of the naturally occurring stem cells within the body to increase their
abilities to repair tissues, although this is a longer-term project.”
Prioritization
of this research depends on the goals of the R&D organization, GSK says.
“Stem cells provide
novel opportunities that didn't previously exist—they could ultimately be
beneficial to drug discovery in many ways, including as tools and targets for
drug discovery and as new therapeutic agents,” GSK’s spokesman says. “In the
generation of tools, stem cells can be induced to generate specific human cell
types for research and compound screening. It may also become possible to
generate human cells of relevance to diseases, thereby improving the relevance
of cell models to human diseases. As therapeutic targets, adult stem cells
residing in multiple organs of the body could be modulated by therapeutic
reagents to induce regeneration. For instance, in Alzheimer’s disease, if a
drug could be found that is able to induce the production of new neurons from
neural stem cells, there is the potential of returning lost function. Finally,
stem cells have the potential to be used as therapeutic agents. Stem cells and
their progenies grown and manipulated outside the body might be introduced into
the body for therapeutic purposes.”
One of the
pharma’s most advanced clinical development programs, one being developed by
GSK’s Rare Diseases unit, is a stem cell gene therapy for severe combined
immune deficiency (ADA-SCID), a rare and life-threatening immune deficiency
disorder. Results from these clinical studies were published in 2009 in the New England Journal of Medicine.
GSK has allied
with the Harvard Stem Cell Institute to develop new medicines, and with the
Fondazione Telethon and Fondazione San Raffaele to research and develop novel
treatments to address rare genetic disorders, using gene therapy carried out on
stem cells taken from the patient’s bone marrow. GSK is also a founding member
of the Stem Cells for Safer Medicine (SC4SM) initiative in the United Kingdom,
which brings together pharmaceutical companies and public-sector organizations.
Company: Pfizer Inc.
Focus: Drug screening
 When people
think of human embryonic stem cells, they don't often imagine the power of
these cells in drug discovery, but Pfizer says it has been using animal or
adult stem cells in its laboratories for more than a decade to help screen new
compounds and identify safer and more effective medicines.
“Pfizer has
begun to explore accessing drug development technology from leading academic,
biotechnology or pharmaceutical partners around the world, who also have
experience with currently available, human embryonic stem cell lines that meet
the highest ethical standards set by leading scientific authorities,” the
company says on its website.
In April, Pfizer
established a new biotech unit in Cambridge, the United Kingdom, that will
combine research in pain, sensory disorders and regenerative medicine. Taking
Pfizer’s expertise in the field of sodium ion channels, Neusentis scientists
will apply this knowledge to deliver new medicines across all forms of pain
influenced by this mechanism, as well as a regenerative medicine portfolio.
Neusentis is developing a cell-based therapy for age-related macular
degeneration constituted by retinal-pigmented epithelium made in vitro from a continuously growing
human pluripotent stem cell line. Pfizer and Neusentis initiated their first
stem cell clinical study for patients with ulcerative colitis this year.
Some of Pfizer’s
external partners include the University College of London and the University
of Wisconsin’s Alumni Research Foundation.
RELATED STORIES: Backed by strong venture capital funding, biotechs organize around promise of stem cell research A roundup of North America's top five
academic research institutions in the stem cell arena
ONLINE BONUS: Life science tool providers provide constant support to ever-evolving stem cell research community Code: E091127 Back  |
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