NeuralStem Trying To Make Name In Functional Genomics
By BETH M. MANTZ
Of DOW JONES NEWSWIRES
NEW YORK -- Functional genomics seems like an area into which nearly every pharma and biotech company is diving head first these days.
With so many public and private players, companies need to demonstrate why their functional genomics technology - the study of what genes do and why, when they are defective and when they cause disease - would aid drug discovers and developers more than competitors' or carve out a niche of certain targets and disease pathways.
Privately-held NeuralStem BioPharmaceuticals Ltd. in College Park, Md., is trying to distinguish itself by focusing on the central nervous system. Chief Executive Richard Garr hopes his company's technology will be considered essential for discovering and testing potential compounds to treat central nervous system, or CNS, disorders.
"We want our cells to become the industry standard...the substrate for all CNS drug discovery throughout the world; we want to get our technology used in every testing format whether it is biochemical, gene chip, or ion channel screening," said Garr.
NeuralStem's technology will enable scientists to grow neural cells reliably in Petri dishes from human post-mortem fetal stem cells without any manipulation.
Stem cells are referred to as blank slates that have the potential to differentiate and form all of the tissues and organs of the human body. Once placed in their cell environment or manipulated, stem cells adopt the neighborhood's characteristics.
These new neural cells could be used to replace diseased and dead neurons with healthy cells for treating CNS disorders including Parkinson's, Alzheimer's and spinal regenerative diseases.
While it weighs this therapeutic option, NeuralStem is expending much energy on functional genomics. In fact, it has allotted nearly 90% of its funding for internal development of its technology for this use, said Garr.
NeuralStem is developing cell lines to use in screening synthetic and natural compounds for central nervous system activity; developing platforms for high throughput drug screening; detecting central nervous system toxins; and measuring normal changes in gene expression that occur as human neurons develop or when diseases progress.
NeuralStem licenses this technology to drug developers, drug discoverers, genomic information providers, and research institutions.
The company has already licensed the technology to Gene Logic Inc. (GLGC) for gene expression analysis and inclusion in the GeneExpress database. Gene Logic is the company's largest source of revenue, said Garr, who didn't disclose the revenue arising from this relationship.
Because NeuralStem's cells are destined to turn into neurons from the beginning, a drug discovery company wouldn't have to wait until human clinical trials to learn its investigational compound failed to have any activity on human neurons. NeuralStem expects to ink its first cell-line deal by the first quarter of next year with an unidentified pharmaceutical company, said Garr.
Additionally, this technology can read expression levels of all genes before and after a certain drug has been taken. "We are doing this so we can find out what is really happening when a person takes an antidepressant," said Garr.
The various uses of the technology convinces Garr the Gene Logic arrangement is one of a long line of agreements with companies that could benefit from NeuralStem's expertise, and envisions real revenue as a result. With licensing fees of $500,000 a year, he conservatively projects 2002 revenues in the high 20 million-dollar range.
"There is a huge appetite for our cells in the industry because we can cut down on the price of developing drugs in an ever expanding funnel of gene targets at the top and compounds at the bottom," he added.
NeuralStem hasn't decided whether it will become a publicly-traded company.
"But given our capital needs, going public is clearly the best way of access," said Garr.
Jim |
