A really insightful and sobering article on the process of translating a basic research finding into a therapeutic product DOI, well worth a read and sharing.
By highlighting only the success stories, and using words like "pipeline" are we giving the false impression that there is a direct relationship between scientific discovery and novel drug, and the translation of one into the other is an inevitable and reliable process?
Why Most Clinical Research Is Not Useful
An interesting publication in PLOS Medicine titled “Why Most Clinical Research Is Not Useful” DOI.
John P. A. Ioannidis suggests that a series of features that make clinical research useful can be identified, including those relating to problem base, context placement, information gain, pragmatism, patient centeredness, value for money, feasibility, and transparency and concludes …
Overall, not only are (clinical) most research findings false, but, furthermore, most of the true findings are not useful. Medical interventions should and can result in huge human benefit. It makes no sense to perform clinical research without ensuring clinical utility. Reform and improvement are overdue.
Given the costs involved I suspect this final point may catch the eye.
Reform is needed. Altering our approach could easily produce more clinical research that is useful, at the same or even at a massively reduced cost
An article on BuzzFeed suggests that Pharma companies are investigating the use of ResearchKit in clinical trial studies.
ResearchKit is an open source framework introduced by Apple that allows researchers and developers to create powerful apps for medical research. Easily create visual consent flows, real-time dynamic active tasks, and surveys using a variety of customizable modules that you can build upon and share with the community. And since ResearchKit works seamlessly with HealthKit, researchers can access even more relevant data for their studies — like daily step counts, calorie use, and heart rate
GlaxoSmithKline apparently is currently working on integrating (ResearchKit) into clinical trials and planning to start in coming months, whilst Purdue Pharma are in the early stages of exploring whether Apple’s new tool for research data collection can be used as part of its own drug R&D efforts.
So far, ResearchKit apps are being led by academic medical centers like the University of California, San Francisco, and nonprofits like Sage Bionetworks and the Michael J. Fox Foundation for Parkinson’s Research. LifeMap Solutions, a company that develops mobile health apps, helped create the asthma app in partnership with the Icahn School of Medicine at Mount Sinai. The first ResearchKit apps signed up more than 75,000 participants in just the first few months
A little while ago I mentioned The Community for Open Antimicrobial Drug Discovery effort to provide free compound screening against a variety of infective agents. I now have a few more details of what you might be able to access for a 1mg sample.
Test against key ESKAPE pathogens, E. coli,
K. pneumoniae, A. baumannii, P. aeruginosa,
S. aureus (MRSA), as well as the fungi C. neoformans and C. albicans, at a single concentration.
Hit Confirmation:- Confirm activity with minimum inhibitory concentration and counterscreen for cytotoxicity and membrane interaction.
Hit Validation:- Test the positive hit against a broader panel of microbes and evaluate the basic drug qualities of actives.
CO-ADD will screen your compounds for free and make no claim to IP
Annual Site Review
At the end of each year I take the opportunity to look at the website analytics to see what parts of the website are the most popular. Overall there was a 15% increase in the number of page views up to 75,000. Average time on a page was 2 mins suggesting the content is engaging with the viewers.
Nine of the top ten most popular pages were from the Drug Discovery Resources Pages which I am delighted to see, since it suggests that the work entailed in putting the resources together is worthwhile.
The most viewed pages were
Seeding Drug Discovery
The closing date for the next round of the Wellcome Trust Seeding Drug Discovery initiative is November 8 2013.
Funding to facilitate early-stage small-molecule drug discovery. The awards help applicants with a potential drug target or new chemistry embark on a programme of compound discovery and/or take later stage projects towards clinical trials. The aim of Seeding Drug Discovery is to develop drug-like, small molecules that will be the springboard for further research and development by the biotechnology and pharmaceutical industry in areas of unmet medical need.A two-point entry system has been introduced to enable projects at an earlier stage in development to be competitive for funding as well as to progress later-stage projects further towards clinical trials.
Separation of PK and PD
I’ve just added a section on compounds with extended off rates from the protein target.
For indications for which require an extended pharmacological profile a compound with a long binding half-life can have a duration of action which extends beyond the presence of drug levels in plasma needed for biological activity. In particular it may be possible to extend duration of action at the intended target whilst limiting activity at off-target proteins. Whilst this could be achieved by irreversible covalent binding there is now a growing body of evidence that many small molecules can display slow off rate kinetics
European Lead Factory
I’m delighted to see the announcement about the European Lead Factory, hopefully this will be a huge asset for Drug Discovery. http://www.nature.com/news/europe-bets-on-drug-discovery-1.12372
Two sites shuttered by the pharmaceutical giant Merck, one in Scotland and one in the Netherlands, will soon be humming again with the work of drug discovery. But the hum will not be business as usual. It will be the sound of a public–private consortium placing a high-stakes wager: a nearly €200-million (US$271-million) bet that it can boost a languishing pharmaceutical sector by fusing academic innovation with industrial-scale screening, using robots to test chemicals for biological activity….Any academic group or company can also propose assays to test molecules in the library for biological activity. Lead-factory scientists will run these assays free of charge and confirm any promising results, working mainly in laboratory space closed by Merck in 2011 at Oss in the Netherlands. Follow-up work will be done at the University of Dundee in Scotland. Results will be provided confidentially to the groups that proposed the assays so that they can pursue further work and publications.
FiercePharma are obviously on a recruitment drive, I have to confess I’m already a subscriber. Makes an interesting read over the early morning cup of coffee.
A virus that kills cancer
Ad5[CgA-E1A-miR122]PTD is a virus created by Professor Magnus Essand that has been shown in animal models to infect and kill cancer cells. The work has been published and is thus not patentable, however they hope to develop the virus with the support of charitable funding and crowdsourcing.
If you would like to find out more or donate visit http://www.fundingjar.com/projects/130/project-info/