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Oct
10

Why we have to redouble our efforts to increase the efficiency of drug discovery – the need to collaborate

OK -I have not blogged for a few weeks or so as I have had a lot on my mind. A refresher – my past efforts have been pretty specific and tied into really well defined scientific issues around databases, collaboration etc, areas that only a few people are interested in (well a few hundred) and I have pulled no punches and even been critical of the efforts championed by the head of the NIH. I figure if I am going to engage in the blogosphere I need to champion a few causes (not political). Now I have no soap box here but I am not afraid to wear my heart on my sleeve. The following is why I engaged in science from a very young age, it is also what keeps me going through ups and downs. We sadly all probably have stories like this..but I am going to add it to the pile.

When I was a young kid one of my friends had leukemia..we were too young to understand the implications of this but we knew it was bad. I remember my parents helped to raise money to buy him orange squash (as we call juice you dilute with water in the UK). I know it sounds odd but the small comforts made a big difference. He did not survive. His memory has haunted me for decades, it helped drive me into drug research. Nowadays we are bombarded with the fact that childhood leukemia is now a survivable disease for many due to research around the globe. Unfortunately the arrival of treatments like gleevec and their ilk were too late for my friend who passed away too quickly in the 80’s. Remember that was the decade when drugs could be brought to market far quicker than now..and far cheaper.

Then in the 90’s my grandmother had cancer, she was told to treat it with stout..the beer, I kid you not. She was prescribed Guinness. She knew she did not have long to go and it was just a question of comfort.

In the early 2000’s it was the turn of my sister to be diagnosed with cervical cancer, she underwent chemo, was in remission but the toll of further chemo and side effects took their toll. She left behind two young children, a husband and a gaping hole in the family. Within a couple of days after her passing the first of the cervical cancer vaccines received approval. This was bittersweet, a vaccine that can prevent so many families from undergoing what my family went through, yet there was and is still no cure for cervical cancer or even hope (on the near horizon) that we can do the same for this disease as for childhood leukemia. I guess what we felt with the arrival of vaccine was why did it not arrive a few decades earlier when we were kids, my sister could have been vaccinated, may not have had cancer, end of story. Just like when the polio vaccine arrived on the scene it changed the way people looked at the disease, but it was also too late for many. The same could probably be said a decade earlier with antibiotics and their magic bullet status.

You are thinking when is this going to end and why just focus on cancer?  Well the answer is I also have an aunt and uncle who are cancer survivors and a cousin who was recently diagnosed with cancer too and is going through chemo now.  I see no end. As a kid I would never have imagined that so many of my family would face cancer. I am not blogging this because I think we are any different to any other family. Far from it. But within my lifetime I have seen some cancers become treatable if not curable or manageable. The challenge I think is to get all cancer treatments to this stage. one or two is not enough. We have spent billions of dollars, pounds and euros on research and we inch along – and this is not meant as a criticism of all the cancer researchers. Its just I think its time to take stock how we go about it from beginning to end.

What do I know ? I just cannot watch as we throw more money at cancer, ignore other diseases and make little progress. Its time to take a different tack. My work in recent years on neglected diseases made me realize that for all of our advanced technological status we are just so terribly backward about thinking about the big picture when it comes to thinking about diseases.  We are currently going through the Nobel prize season and this kind of hammers it home. Scientists giving other INDIVIDUAL scientists prizes. Do you see any of them giving prizes to massive collaborative efforts. No! We are still giving prizes to individuals. This reinforces in the public the figurehead scientist as a loner. We need our children to think of scientists as collaborators which many of them are. Nobel unfortunately does not award a prize to the best collaborative scientist. When are we going to think about changing the very nature of science to be more open and massively collaborative. Some of us put our stuff out there in papers, slides etc and hope others will build on it, some of us have to patent or keep work trade secrets as is necessary to keep a competitive advantage. But I ask you who are we kidding. I alone cannot have every great idea but if I put a piece of my work out there maybe a scientist in India or Russia could do something with it.

There is precedent for collaborations on the scale we need to radically alter the pharmaceutical industry. During the war pharmaceutical companies pulled together for the war effort to manufacture Penicillin. Can you call it collaboration? -but regardless lives were changed and the pharmaceutical industry was seen as a life saver (when was the last time Joe and Josephine public thought that?). How about – during the 80’s when under great public and political pressure antivirals were quickly developed and approved for AIDS. Look at that disease now in the west it appears under control, patients can have an active life. We are instead  faced with the healthcare disparity with those in the west being treated and those in the third world continuing to die at an appaling rate. When there is enough will and focus from research and government we can get our act together. You just have to watch the vaccine frenzy everytime there is some flu alert. I think in the 2010’s we have an opportunity with tightened global budgets for everything including research to really use this time to do everything different, new approaches are needed and not those that have already been unsuccessful in industry.

In my lifetime I have seen computers go from taking up rooms to sitting in the pocket of my shirt. I have seen people design and simulate molecules, cells and organs on computers on a desktop PC, this is now possible on a phone. We need to do more to advance drug discovery at the same rate as computers. Drug research and computer technologies are converging and this needs to permeate the way we do industrial drug discovery. Having thousands of chemists working for you in China and India has not improved the drug discovery rate in the same way high throughput screening has had limited impact. We are at a turning point because there are ways to use computers to improve, enable and facilitate collaboration. We have created massive social networks so that people can pass links to blogs, papers around the web. I implore the NIH to foster collaboration (in more than words) through grantees. Look at the framework grants in the EU for inspiration and do it better? I think companies need to loosen up and put more of their molecules and data into the public domain. Here is a once in a lifetime opportunity to get some public goodwill. I saved the best till last.. The regulatory authorities, you know who you are..For all your attempts to add layer upon layer of hurdles to the industry as a way to ensure public safety ( and believe me I am all for pulling drugs that have life threatening side effects, or better still avoiding them in the first place) what has it achieved? The public is safe..because there are fewer drugs coming through. Its time to shift back the balance we need drugs for diseases and we need them now. Have I left anyone out..well yes you the reader, I ask you to be like the AIDS activists in the 80’s because with a stronger voice the message might get through faster to the governments and decision makers. If you are also a scientist, give a thought to how you could personally help increase the efficiency of drug discovery (maybe more than cutting coffee breaks), collaborate with someone new, who knows maybe it will lead to a new cure for cancer and perhaps it will be approved fast enough to save your life or that of someone in your family.   

 

 

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  1. Chris Waller says:

    Sean, thanks for sharing. As you point out, I do not think that your family is terribly unique from any of us. I suspect that most all of us have been impacted by the suffering of a friend, family, or loved one from a potentially curable (or at least manageable) disease such as cancer. You point out correctly the success of the collective when focused (and incentivized) properly on specific disease areas (HIV). I’m encouraged by the emergence of the new alliance model for research as demonstrated most obviously by Lilly and the open research model being nurtured. Pfizer, too, is experimenting with the concept of virtual research units to drive forward discoveries initially in the neglected and rare disease areas. I am hopeful that this new biotech revolution of sorts is more successful that the last. One downfall of the last evolution was the creation of data silos between potential collaborators and the inappropriate monetization of data as a corporate asset. I see interactive data sharing platforms such as those provided by Collaborative Drug Discovery and Scynexis’s HEOS platform as incredibly enabling once a collaboration opportunity has been identified. More recently, I’m supporting the efforts of start-ups specifically focused on identifying collaboration opportunities upstream of the data sharing platforms. Emerging solutions such as the one proposed by CollaborationFinder seem to fall directly on the critical path of the creation of Pharma 2.0.

    1. sean says:

      Many thanks Chris for expanding on the blog, I would really like to see more companies follow the lead of Lilly and Pfizer who are taking steps in trying to do research more collaboratively. The scale and funding of these efforts needs to increase, as with support of neglected and rare diseases, because unfortunately they are just a drop in the ocean.

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