From BW:
<<Turning Data into Drugs--Fast
This summer, scientists expect to finish sequencing a rough draft of the entire set of human genes. It is a monumental accomplishment, requiring the correct sorting and assembly of more than 3 billion bits of genetic information. But deciphering the genome is not an end--it's a beginning. 'Having just the sequence of the human genome is akin to [having] a dictionary that lists all the words in the English language but doesn't include their meanings,' says Dr. Robert Tepper, chief scientific officer for Millennium Pharmaceuticals Inc. (MLNM) 'It doesn't tell you much.'
Biotech companies are now hard at work developing the tools necessary to decode the meanings--the functions of the 100,000 or so genes contained in the human genome--and use that knowledge to develop new drugs. It is not a simple task. Drug development is a lengthy and unpredictable process that, on average, takes 15 years to complete. Less than 5% of a company's initial findings lead to drugs that ultimately reach the marketplace. And there is no guarantee that potential drug candidates won't turn out to have toxic side effects. Biological testing in animals and years of human trials are still a must--genomics doesn't do anything to shorten that process.
Two Cambridge (Mass.) companies, Millennium Pharmaceuticals Inc. and Vertex Pharmaceuticals Inc. (VRTX), are among the leaders in the frenzied race to pluck the best targets from among hundreds of thousands of molecules--in other words, to create meaningful sentences out some of those newly discovered words in the genetic dictionary.
Millennium expects to launch two drugs this year. The company already has six drugs in human trials, which are aimed at treating cancer, immune-system disorders, and obesity. In addition, Millennium has committed to delivering 225 targets over the next five years to German pharmaceutical giant Bayer.
Vertex has eight medicines in clinical trials. It won approval back in April, 1999, for an AIDS treatment--a protease inhibitor called Agenerase that it is co-developing with Glaxo SmithKline. The company also has another protease inhibitor in advanced human trials, which analysts say could be even better.
One of the astounding things about companies such as Vertex and Millennium is their ability to move from basic findings to the clinic in just a few years. In 1991, for example, Vertex began work on a drug to reduce the inflammation associated with rheumatoid arthritis after researchers had found a gene associated with inflammation. Vertex then accelerated the search for a drug by doing two things at once: It worked to determine the role of the gene in inflammation and simultaneously began developing compounds that might block the process.
The result, says Vertex CEO Joshua Boger, was 'biological, chemical, and structural data coming together all at the same time.' With volumes of data in hand, company researchers could make a more informed decision about which candidate drugs to keep and which to throw away three years after starting the project. The molecule they ultimately settled on, called VX-740, is now in advanced human trials with partner Hoechst Marion Roussel. If it's approved, it will be the first oral medicine for rheumatoid arthritis.
In the next few years, Millennium and Vertex researchers are hoping to shorten the drug development time even further. If they can, all those words in the genetic dictionary may form some fantastic new sentences.>>
MC for MLNM (~10 BB, and for VRTX (~1.8 BB).
Please J.B., borrow Levin's vision on few minutes. It shouldn't take longer!
Miljenko |
