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A Roadmap to Terminal Disease Cures
I have spent the past 45 years in the medical R&D arena. The first 14 years at the Roche Institute of Molecular Biology and the National Institutes of Health (NIH) doing basic research and 31 years in the drug industry co-founding and leading five different companies. I have therefore traveled through every corridor of the system charged with cures in search of something to pin my ambition to rid us of the terminal disease nightmare and been universally frustrated. There is no logic to any of it and therefore the only solution I see is to write this book. I and my colleagues, many much smarter than me, have failed in the arena of cures. We were pushing against a wall of fog and until that changes the effort borders on futile.
The failure to cure terminal diseases is the greatest global threat to humanity. It dwarfs any other challenge we face as a society and yet it was not a major issue in the recent election. The delivery of healthcare was center stage as is appropriate but from the standpoint of saving human lives does not approach the importance of curing the terminal diseases. Cancer, degenerative diseases such as Alzheimer’s and Parkinson’s and cardiovascular disease prematurely end almost 2 million lives every year. What other threat to humanity approaches such consequences? If we took curing terminal diseases as seriously as providing healthcare we would very likely have been successful long before now. No one has dared to critique the entire system to determine why it has failed so dramatically but rather they just keep throwing more dollars at it. Broken is too kind a word to describe it since it is not a coherent entity; criminal negligence comes a lot closer to the reality.
We are playing parlor games with cures. The fault is not in us but rather in a system that was never designed for the task. The talent is there as well as the capital and infrastructure. The libraries are flooded with research papers so productivity is not an issue but yet the goal is apparently absent. The millions of research papers have served science and its practitioner’s well but has clearly failed when it comes to results. We are buried in science but cures seem to be an afterthought. The roadblocks we have created at almost every step of the process have made drug development a billion dollar decade long Sisyphean task. History clearly shows what we are doing has not worked and what follows will point out why and specify a course of action that will put the cure quest on a laser focused course to finally getting the job done.
We have witnessed quantum technological advances during the past several decades. Virtually every other aspect of our lives has been transformed over this period but when it comes to the most important of all challenges; terminal diseases cures, we have failed. Also, those past successes were made without the help of the super data management and communication capabilities we have had for the past several decades, so it is rather bizarre that the cures mission has failed so dramatically and did not justify the coordinated focused effort given to the conquest of space travel, cyberspace and computing. But rather was allowed to evolve into an academic exercise and be strangled by regulation. Having a super computer in your pocket, a nuclear power plant down the road and folks orbiting in a space station is fine but if terminal diseases were conquered that would be orders of magnitude more significant . This is not a simple task but neither were those other successes.
Curing terminal disease was left to the whim of researchers at universities, institutes and charities all proceeding to leisurely pick away at basic science in a totally unaccountable disorganized fashion in hopes of stumbling on something that might be relevant to these diseases. While the NIH is out there as the federal government’s gesture to health research it has yet to make major inroads into the terminal disease crisis after almost a century of supposedly trying. It consists of 27 institutes, which seems ridiculous given that just three disease classes take the great majority of all lives, and nowhere in its mission statement is curing terminal disease or any disease mentioned. We have engaged in science for science sake for the past 50 years and until that ends very little will change.
Another fundamental reason for the failure in the medical arena is that the entire development component is regulated to the point of strangulation by the FDA and encumbered by a patent and legal establishment that makes drug development extremely expensive and difficult. Terminal patients face certain death yet the regulators insist on usurping their right to life by practicing an arrogant academic purism that has held up drug approvals, made access to investigational drugs very difficult and transformed the price of drug approval into a several billion dollar venture. Until the regulators and legal system back off substantially regarding terminal disease therapeutics there will continue to be a very slow progress.
The problems in specific areas of the medical R&D establish have only recently begun to be appreciated but no one has put the entire establishment under a microscope in one place and proposed solutions; this is the reason for the book that follows. Once the reasons for failure are exposed and reforms proposed that will speed this seemingly endless waiting game for cures and the hope is that the public will rise up and demand action. Unless this is made an issue by the electorate nothing of any consequence will happen. The stakeholders will get in the way, especially those that actually benefit from the status quo which is just about all of them. I am proposing substantial changes in virtually every aspect of the system and there will be kickback. Congress will have to override that but if the public makes it clear that how they vote will be determined by what the congress mandates then this will happen.
The AIDS situation is a good example of what can happen if the public rises up and makes it clear that they want results. The AIDS crisis was championed by a small group of sophisticated individuals that took to the streets, marched into the halls of government and demanded action. Washington got the message loud and clear. The NIH went into high gear and identified the virus rapidly as did the Pasteur institute. The FDA stepped out of the way and accelerated the approval of the drug cocktail that transformed AIDS from a terminal disease to a treatable one thereby saving thousands of lives. Unfortunately that mentality did not stick because the FDA was not organically changed as regards its mission, management and attitude about a patient’s right to life as I am proposing. The NIH was also not reformed as is urgently required.
Providing the ammunition to start a movement to get serious about the cure crisis is a major goal of this book. We cannot be satisfied with millions dying every year while researchers proceed to amble through their individual research ambitions; drug companies have no incentive to develop cures; the NIH and academia proceeds in an unfocused and unaccountable manner; the FDA strangles the drug industry; a patient’s right to life is destroyed and the patient-physician relationship disappears . If we fail to make a serious effort to reform the cure effort and continue to pour money into a failed system the road to cures will be a very long one. These issues have percolated in the political arena but it has always been about more money to build more laboratories and never about how we craft a global effort to achieve success. It is not about money but rather about strategy and repurposing what already exists.
This book seeks to raise awareness about the folly that is the medical R&D enterprise. My primary target audience is the public and providing it with the ammunition necessary to fight this battle. I use the term ammunition because this is a war against the status quo and vested interests. I also hope the stakeholders will look at the issues raised herein but the realist in me expects that they will want to protect their turf. The book is critical of virtually every corner of the enterprise; academia, the drug and biotech industry, thought leaders, venture capitalists, charities, the FDA, the patent codes, the legal system, the healthcare delivery system. In order to accomplish the task the discussion will be in two parts. The first dissects the drug development establishment and points out reasons for the paralysis of progress in plain non-technical terms. The second part suggests solutions that if appropriately thought out and put in place will transform the current quest for cures into an organized, accountable effort with the focus, urgency and strategy required for success.
For decades we have all watched loved ones die needlessly and live in fear of terminal disease. Cancer and Alzheimer’s are two of the worst experiences a family can face. One kills the body, the other the mind, and yet we seem pacified by sporadic reports of science breakthroughs that never lead to anything tangible aside from Nobel prizes. It does not have to be this way if we finally get serious about ending nightmare by applying our magnificent abilities in the same manner that that produced the wonders that have transformed every other aspect of our lives.
The reforms proposed are extensive and include; 1- A total re-thinking of the NIH and the FDA whereby the two agencies work together as partners with industry when it comes to terminal disease. 2- The requirement for all academic research to be conducted under strict quality control. 3- reducing the cost and time of the development path by two thirds for terminal disease drugs. 4- Reforming the patent process for terminal disease drugs making it easier to get marketing exclusivity for existing science and drugs. 5- Encouraging maverick science and shifting to more applied rather than basic research. 6- Restoring the patient-physician relationship by making the patient a direct consumer of healthcare. 7- Restoring the patients right to life.
A Roadmap for Curing Cancer, Alzheimer’s, and Cardiovascular Disease, by Paul J. Marangos, serves as a starting point to generate serious global discussions about fixing the entire terminal disease R&D process and finally cure them thereby saving 2 million lives each year and dramatically reducing healthcare costs. The book is therefore a springboard to achieving that long overdue goal and the first comprehensive treatment of this subject.
The book is expected to publish on May 12, 2017. If you would like to pre-order a copy, please visit the Elsevier Store. Apply discount code STC317 at checkout and receive up to 30% off the list price, and free global shipping upon publication.
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