"The Obama administration has become so concerned about the slowing pace of new drugs coming out of the pharmaceutical industry that officials have decided to start a billion-dollar government drug development center to help create medicines.
. . . .
The National Institutes of Health has traditionally focused on basic research, such as describing the structure of proteins, leaving industry to create drugs using those compounds. But the drug industry’s research productivity has been declining for 15 years, 'and it certainly doesn’t show any signs of turning upward,' said Dr. Francis S. Collins, director of the institutes.
The job of the new center, to be called the National Center for Advancing Translational Sciences, is akin to that of a home seller who spruces up properties to attract buyers in a down market. In this case the center will do as much research as it needs to do so that it can attract drug company investment.
That means that in some cases, the center will use one of the institutes’ four new robotic screeners to find chemicals that affect enzymes and might lead to the development of a drug or a cure. In other cases, the center may need to not only discover the right chemicals but also perform animal tests to ensure that they are safe and even start human trials to see if they work."
Is a new billion-dollar government drug development center to help create medicines a good idea? Let's test the issue with an example.
It is known that in order to prevent certain diseases you need to find a means to prevent a specific protein from folding into its disease-associated conformation. With respect to these diseases, how does the federal government propose to address the problem? By using more efficient robotic screeners to throw ever larger libraries of molecules at the target protein? In essence, this is what Big Pharma has been doing for the past twenty years, and as can be seen in the charts accompanying the New York Times article, this effort is failing.
Is there an alternative? Absolutely.
A decade ago, a tiny Israeli biotech company predicted that Big Pharma would hit the wall in its drug discovery efforts, and undertook a long lonely effort to create discovery platforms which could accurately model biological processes at the molecular level and enable the computerized prediction and selection of therapeutic and diagnostic product candidates. Foreseeing that the era of trial and error in the world of drug discovery was yielding diminishing returns, Compugen sought to harness advanced mathematics and computer science to create the next generation of drugs addressing unmet medical needs. Compugen's "Blockers of Disease-Associated Conformation" Platform ("DAC Blockers" Platform), only one of the company's growing number of discovery platforms, was developed to identify synthetic peptides that could prevent proteins from folding into their disease-associated conformations.
What was required to create the DAC Blockers Platform? Among many other things, Compugen needed to map all proteins in the human body. Compugen also needed to identify the cleavage points for all of these proteins in order to map all peptides in the human body. Moreover, Compugen needed to know the exact locations where proteins fold into their disease-associated conformations so as to predict synthetic peptides that could block this folding.
Does Compugen's DAC Blockers Platform work, given that even a single mistake along the way would render all of Compugen's predictions meaningless? According to the company:
"Initial runs of the discovery platform resulted in the in silico prediction of therapeutic peptide candidates for approximately 40 drug targets of interest with potential usage for various indications, including solid cancers, inflammatory diseases, septic shock and viral diseases. Eleven of these drug targets were selected for initial experimental validation and potential peptide blockers were found for all eleven targets."
http://www.cgen.com/Content.aspx?Page=Disease_Associated_Conformation_peptide_blockers
Can the federal government replicate Compugen's work? No, because the federal government is not playing with a full deck of cards, i.e. it is lacking proteins, cleavage points, peptides, folding sites, and the integrative knowledge which gave rise to this body of information. Sure, there might be isolated instances where robots might toss a molecule at a target and notice some sort of change, but this is the approach used by Big Pharma for the past 20 years, and as evidenced by Big Pharma's rising expenses and declining rates of discovery, it is headed for extinction.
On the other hand, there could much value in having the U.S. federal government shepherd the unlimited number of drug candidates which today can be obtained via predictive discovery through the "Valley of Death", i.e. the period of transition when a candidate is thought to be promising, but at too early a stage to validate its commercial potential and attract necessary capital for continued development.
Predictive drug and diagnostic discovery is finally proving its worth, and I believe that if Dr. Collins were to meet with Compugen's scientists, he would learn that there are indeed signs that drug research productivity is turning upward. Should Dr. Collins meet with Compugen before initiating this new billion-dollar project? You bet!
[As noted in prior blog entries, I am a Compugen shareholder, this blog entry is not a recommendation to buy or sell Compugen shares, and in mid-September 2009 I began work as a part-time external consultant to Compugen. The opinions expressed herein are mine and are based on publicly available information. This blog entry has not been authorized or approved by Compugen.]
I think the company Proteostasis (proteostasis.com) has an original approach to developing new therapeutics as well.
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