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Companies Racing to Use Stem Cells to Find and Test New Drugs

By Rob Waters
May 20 (Bloomberg) -- Two companies that produce different types of stem 
cells have signed contacts to sell their products to drugmakers, showing the 
new technology will be used to help discover medicines not just to repair or 
replace damaged cells.
California Stem Cells Inc., an Irvine, California, biotechnology company 
that turns embryonic stem cells into neurons, said today it's selling the 
brain cells to researchers trying to find drugs to treat Lou Gehrig's 
disease. CellDesign Inc., of New Haven, Connecticut, said it has contracts 
with four drugmakers seeking to use its product to find new medicines for 
conditions such as Parkinson's disease and schizophrenia.
The efforts of these two closely held companies indicate stem cells will aid 
in the search for old-fashioned drugs long before they're infused into 
patients. It also suggests that the first businesses to benefit from stem 
cell technology will be traditional pharmaceutical companies and their 
suppliers not developers of new kinds of therapies.
``It's similar to what happened in the last century with molecular biology'' 
and gene therapy, said John Hambor, CellDesign's founding chief executive 
officer, in a telephone interview yesterday. ``We're now going down a 
similar path with stem cell biology. While it may lead to cures by itself, 
it will drive the next generation of drug discovery.''
California Stem Cell will provide hundreds of batches of its neural cells 
over the next year to BioFocus DPI, a unit of the Belgian drug discovery 
company Galapagos NV, said Chris Airriess, the California company's chief 
operating officer, in a telephone interview yesterday.
Manipulating Cells
BioFocus researchers will manipulate the neurons so they match the damage 
found in patients with amyotrophic lateral sclerosis, or ALS, said Katherine 
Hilyard, BioFocus vice president for biological sciences. Then researchers 
will run the altered neurons through machines that can rapidly test huge 
libraries of so-called gene silencers -- bits of genetic material that can 
block the action of proteins -- to see if they can fix the damage.
``Stem cells let us create systems that mimic what's happening in the 
patient so we can find a better drug,'' Hilyard said in a telephone 
interview yesterday.
The project is funded by the ALS Association, a nonprofit research and 
advocacy group that works to develop treatments for ALS, also known as Lou 
Gehrig's Disease. The condition kills nerve cells in the brain and spinal 
cord, robbing patients of muscular control and eventually leading to 
paralysis.
``This is a quick way to look for potential targets,'' said Lucie Bruijn, 
the association's science director. ``To test all these things in an animal 
model is so much more expensive.''
Airriess said the agreement with BioFocus is one of many he and his 
colleagues are developing. The company also is negotiating with drug 
companies to supply neural and heart cells.
`Very Profitable'
``It's potentially a very profitable business,'' he said.
Three European pharmaceutical companies, Roche Holding AG, GlaxoSmithKline 
and AstraZeneca, announced last October they'd work together to develop ways 
to use stem cells for drug screening. The work will be coordinated by a new 
London-based organization, Stem Cells for Safer Medicines, funded by the 
companies and the British government.
The group's goal is to find more efficient ways to identify new drugs and 
test them for potential side effects earlier in the drug development 
process, said Philip Wright, the group's executive director.
``If you look at attrition or failure of drugs in clinical development, by 
far the largest cause is through unexpected toxicities that haven't been 
predicted'' in animal tests, Wright said.
Mouse Cells
Drug companies already use mouse embryonic stem cells to search for and test 
drugs and some are interested in using human embryonic cells, Wright said.
``But they're taking a very conservative approach'' because of the ethical 
controversies that surround the use of cells from human embryos, he said in 
a telephone interview yesterday.
Until recently, scientists recognized two basic types of stem cells. 
Embryonic stem cells, derived from embryos in the first few days after 
they're fertilized, have the potential to become any of the body's roughly 
210 cell types. Extracting the cells with current methods kills the embryo, 
prompting opposition from critics including President George W. Bush. Adult 
stem cells, found in developed tissue, have more limited potential to become 
other cells.
If scientists can refine a new technique that reprograms cells found in 
mature organs to give them the power of embryonic cells, drug companies will 
be interested in the method as a way to develop heart, liver and other cells 
that can be used to test drugs, Wright said.
Funding Research
Wright's group will soon award 1 million pounds ($1.97 million) to back 
research into the use of stem cells for drug testing to be followed with 
another 10 million pounds ($19.7 million) over five years.
Hambor, the chief executive of CellDesign and a former Pfizer Inc. 
scientist, said the potential for stem cells to find new medicines helped 
attract a venture capital firm, Toucan Capital Corp. of Bethesda, Maryland, 
to invest in his fledgling company. Toucan Capital, which confirmed its 
investment, declined to say how much.
``The immediate impact of stem cell technology will be as a tool for 
discovering new drugs,'' Hambor said. ``Where else could you get human brain 
at the scale needed to do drug discovery?''
To contact the reporter on this story: Rob Waters in San Francisco at 
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Last Updated: May 20, 2008 16:38 EDT

Rayilyn Brown
Board Member AZNPF
Arizona Chapter National Parkinson Foundation
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