Friday, February 22, 2013

New Drugs for Bad Bugs?

For the last several decades infectious disease physicians have been warning about the ever-increasing prevalence of pathogens that are resistant to existing antibiotics. This includes some really scary organisms such as multidrug-resistant Enterococci and vancomycin resistant MRSA. Despite this rapidly evolving threat there has been relatively little research on new antibiotics within Big Pharma. Presumably the return on investment for this area is not attractive to the industry.


Thus it is cheering to learn that the European Union Innovative Medicines Initiative (IMI) is preparing to invest $550M in drug discovery for new antibiotics. This seems a very timely move. Contemporary high speed DNA sequencing technology now allows genomes of bacterial strains to be analyzed with ease thus potentially leading to the identification of many potential new targets. The IMI initiative will involve both public funding and the collaboration of academic institutions and drug companies. Thus it seems to share in the current trend for pre-competitive collaboration in drug discovery. While even half a billion dollars may not be enough to bring one or more new antibiotics all the may through clinical trials and into the marketplace, at least it seems likely that novel and important new lead compounds will emerge from this effort.


Despite somewhat of a surge in new drug approvals in 2012 (1) it remains clear that the purely profit driven model of Big Pharma is failing to address therapeutic areas that are vitally important to public health (2). Public-private partnerships with more interaction at the precompetitive level, like the present IMI effort, may help fill those gaps.


(1) K. Jiang. Near-record number of approvals signals drug development shift. Nature Medicine 19:114 (2013)


(2) R.L. Juliano. Pharmaceutical innovation and public policy: The case for a new strategy for drug discovery and development. Science & Public Policy (epub ahead of print, 2013) 

Thursday, February 14, 2013

Making academic research pay off- is that really the point?

An interesting commentary in Science by D. Malakoff describes the many approaches that universities are taking to assure that their research ‘pays off’ in the commercial arena. However, as the article points out, for every major success, such as NYU’s $650M from Remicade, there are many more failures with less than 1% of academic intellectual property earning $1M. Many technology transfer offices cost far more to run than any revenues they return to the university. To this observer this intense pursuit of commercial and entrepreneurial activity is another sign of the increasing corporatization of American universities and a turning away from their traditional role as the creators of new knowledge that broadly benefits society.

Many research-intensive universities, especially public ones, are struggling to demonstrate their economic relevance to their masters in state legislatures. However, trying to do this on the basis of patents issued or royalties generated is likely to be a losing proposition. Universities need to devise better appraisals of the positive impact of their research on economic wellbeing and not have it defined in narrow commercial terms. 

Friday, February 8, 2013

Who needs PhDs?

Now here is a website that should chill the marrow of all those grad students working hard on their PhDs. Innocentive is an online company that proposes to solve problems by crowd sourcing. This apparently includes complex scientific problems involved in drug discovery and development since the company includes Eli Lilly among its clients. Here is how it works. Clients (Seekers) can post ‘challenges’ via Innocentive. People (Solvers) can then respond to the challenge with their ideas. If a successful solution is provided the Solver gets a prize- whoopee!

Seemingly the client companies involved seem to think that instead of long term hiring of expensive scientific staff, they can cherry pick new technologies by offering booby prizes of a few hundred or a few thousand dollars. So who needs PhDs- maybe Joe the Plumber can come up with your next research breakthrough!

Outsourcing of research to China and India is bad enough, but this is worse. In this race to the bottom the desire to minimize costs is eviscerating the scientific and technological capabilities of the American pharmaceutical industry. By not investing in research Big Pharma is on the path of slow motion suicide. 

Friday, February 1, 2013

Long term federal funding and career development for young scientists.

An interesting article in C&EN News discusses attempts to secure multi-year funding for science as opposed to the current system of annual congressional appropriations. This has been a dream of many scientists for years, and such stability would no doubt enhance scientific productivity in the US. The President’s Council of Advisors on Science & Technology (PCAST) has now come out strongly in favor of the multi-year approach, but whether this will influence Congress to relax its hold on the purse strings is in doubt. One aspect of this that isn’t much discussed is the potential impact on the careers of young scientists. One of the most difficult aspects of career development in research institutions is the enormous uncertainty about grant funding. When NIH or NSF funding levels wobble a few percentage points from year to year it can be a career breaker for early stage scientists. While multi-year funding wont reduce the intense competition for grants, at least it would make the process more predictable and allow better career planning.  

Finally- an antisense drug makes it into the clinic!

Congratulations to the ISIS team for getting the first oligonucleotide drug into the clinic. It has been a long time coming, but maybe mipomersen will open the door to other molecules based on antisense, siRNA, or splice switching oligos. Kynamro (mipomersen) is an antisense that targets the RNA for apolipoprotein B100, a component of low density lipoprotein. A small cohort of people with homozygous familial hypercholesterolemia are resistant to statins and have highly elevated cholesterol levels that lead to serious cardiovascular problems at an early age. Kynamro should be able to help these individuals.