Author: BernardusMuller

  • Entrepreneurial scientist…

    Entrepreneurial scientist…

    HiResI regularly hear leading scientists say that patients can make a difference by being closely involved in projects that could ultimately lead to breakthroughs for their diseases. On the other hand, I also hear researchers say “leave the science to the scientists” and that we shouldn’t get involved because of our biased opinions. Contradictio in terminis or a rusty scientific paradigm?

    You can’t deny that a certain subjectivity exists when it comes to patients being involved in research projects related to their own diseases. With the amount of knowledge that’s now only a quick Google search away, I don’t blame researchers for their hands-off opinions when it comes to dealing with patients. However, this type of knowledge has become such an abundant, primary source of hope and inspiration for patients with rare diseases, that even when restricted by paywalls, these patients are often more up to date with the latest breakthroughs and information than their doctors.

    Certainly, entrepreneurial patients like myself have strong opinions. We don’t take no for an answer. We can be instrumental in the process to develop new drugs for our diseases. We can accelerate development times because of our devoted focus. We are the ones who can help translate basic research into real products. Why? Because we have a vested interest in how this all turns out. Our intimate involvement with our diseases gives us the passion and the power to combine our business skills with the scientists’ practical knowledge in a robust collaboration. Biased or not, it doesn’t really matter, the ultimate goal to find a cure is what binds us to our cause.

    In the biotech and pharma industry, translational research is more common in certain parts of the world, like the USA, where companies collaborate at an early stage with academic institutions. With rare diseases like ALS, it is essential to do so. However, there is still much to learn. Academics should share their discoveries with companies and patient groups at a much earlier stage, instead of caring about publication dates. Some breakthroughs take almost a year before becoming public knowledge. That’s another year lost translating them into real products. When the papers are finally published, accompanied by a positive press release, the scientists continue researching in another direction, leaving their discovery for the business world to pursue.

    Innovation, and ultimately curing patients, is halted by this scientific paradigm. This really should change. Scientists should actively engage with fledgling biotech companies and offer them their “off paper” insights in return for a small licensing fee when the efforts prove successful. At the end of the day, it’s all about shared value, shared success and curing patients. At least it should be… Researchers should transform themselves into entrepreneurial scientists, while the business men and women should become more like scientific entrepreneurs.

  • Our progress

    Our progress

    I haven’t been blogging for a while. It hasn’t been due to a lack of inspiration, but it’s been more a matter of finding the time, while trying to balance multiple priorities.

    The last two months have been exciting, but also hectic and exhausting. Where 2013 was a year of planning and making preparations, the time has now come to execute those plans. In January, I got together with fellow patients Robbert Jan en Garmt to discuss our strategy and to clarify the path forward. It turns out that we have 35 projects on our plate! So we have a lot of work to do, but we found time to make a nice picture of us three 🙂

    20140215-125209.jpg

    Along with former classmates, Garmt recently organized an MBA class with a group of MBA students to discuss our strategy, centered around the following 5 pillars: patients, funds, cause, cure and care. We received a lot of great feedback from a business perspective. On the other end of the spectrum, I collected some very useful scientific feedback while attending the ALS/MND congress last December. We’ve had multiple talks with large biotech investors, health insurance providers, and other influential stakeholders, including visits to research centers in Europe that are focusing on a variety of innovative technologies. We’ve discussed hypotheses with key thought leaders and investigated the overall ALS/MND market worldwide.

    We make every effort to prepare ourselves as thoroughly as possible and do not want our efforts to ever be compromised by a lack of resources. Because of this, we try to have a leader for each project and at least one or more patients involved, as well. We are assisted by numerous experienced, skilled and enthusiastic volunteers. You could say that it’s now full speed ahead!

    In the coming months, I will regularly update you on our progress, so stay tuned…

  • Future evolution

    Future evolution

    Following the news feeds of blogs and various websites in the healthcare and biotech industries provides you with insights about where we are heading in the future. In my blog post “So far, so good,” I wrote about the autoimmune illness that’s plaguing the world healthcare system. Today I’d like to take you with me on a journey into the future.

    It does’ t require a visionary perspective to come to the conclusion that the growing prevalence of cancer, obesity, diabetes, and numerous neurological disorders is a bit of a modern phenomenon in the timeline of human history.

    Whereas the very existence of mankind was once threatened with extinction by different viruses or bacteria in ancient times, then called plagues, nowadays we think we’re far too smart to be taken down by a few nasty microbes. However, when confronted by the widespread panic that occurs when certain viruses mutate, a looming antibiotics crisis, and the frightening fact that over 5% of the US population is infected with untreatable super bacteria, you suddenly have the impression that we’re all playing some real-life, global version of Plague Inc., a popular game where the primary goal is to wipe out the world’s population with viruses and bacteria.

    Many scientists blame the drastic changes in our environment for our modern  super diseases and to a certain extent, I agree. As a human race, we’ve survived an industrial revolution, an information revolution, and are now in the middle of a biotech revolution. We genetically modify our food to boost production, adding chemicals for sustained quality and improved shelf life without even knowing the long-term effects. No worries, I am not a strong advocate of everyone going organic. We simply can’t produce enough food in this manner to feed the world’s population. It is very noble to think we can, but lets’ be honest, we first have to reinvent the food chain and then we’ll all have to start eating algae.

    The field of genetics is also evolving. We now know that our imprinted DNA is not necessarily what is biologically expressed in our bodies. Cells can have different DNA profiles and even tiny environmental changes can influence how these genes express themselves, resulting in unknown, complex, and rare diseases. With new technologies like proteomics, transcriptomics, and metabolomics, we are trying to find out what exact biological path a disease will follow. In the meantime, we still need to unravel the causes of many of these same diseases.

    The real question is if technology can keep up with the human race. With a world population of over 7 billion, an annual birth rate of over 130 million, and an average of around 100 de novo variants per person, that’s roughly 13 billion new human genetic mutations formed on an annual basis! That’s what Darwin called evolution…

    I am a passionate advocate for science and technology. Sadly, the scientific community is restricted and hampered by increasingly strict privacy legislation, ethical concerns, and a pervasive reluctance to change. We have the World Health Organization, but their task is primarily reactive. We don’t have a UN of science. In its place, there is a lack of coordination and collaboration. Only in despair and in cases of extreme crisis do we join forces. Then it’s usually already too late…

    As there are around 100 trillion cells in the human body, with each encoding 3 billion base pairs of DNA, it ‘s almost as if we’re like the ancient Greeks, discovering the stars and the universe, which after more than 2,000 years still remain a mystery. It might take decades, if not centuries to unravel the biological mysteries within our own bodies. And where Galileo ended up literally blinded by science, I surely hope we don’t.

    I am not trying to sketch a gloom and doom scenario. We know that nature has a tendency to balance itself. That’s one of the basic lessons that emerges from studying evolution. Luckily some governments, like the UK, are seeing the importance of life science technology and announcing large-scale genetic projects. Unfortunately, these initiatives are still a drop in the ocean of human biology and without lifting the restrictive rules surrounding it, we still have a very long way to go. A recent FDA ban on the genetic company 23andme, a pioneer in consumer genetics, offering their services at low costs to interested consumers, proves this point. I believe 23andme and the services they provide are a good step forward, but still not the giant leap that we desperately need. Certainly not now that the FDA ban takes us several steps backwards instead.

    Historically we have had major scientific breakthroughs that have reshaped our thoughts, cured diseases, and even taken us to the moon. Why can’t we experience another one tomorrow? I know that I’m not alone in my opinion, but we are unfortunately too outnumbered to convince the politicians and world leaders who set the scene for the majority of people. In my opinion, this is far more important than the issue of climate change, which after many years has been prioritized on everyone’s agenda.

    Keep the faith and stay hopeful. It’s never too late to change… our future evolution.

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  • Simply words…

    Simply words…

    Tag Cloud One Man
    Tag Cloud One Man

    When you are fighting a disease one is often motivated and inspired by quotes or words of admired leaders.

    All these One Man´s inspired thousands and thousands of people, changing existing paradigms and consequently changed the world.

    A tag cloud of almost 70.000 most used words from 20 speeches resulted in the beside standing image.

    Surprised? I wasn´t… The spoken word is the most distinguished gift that Nature gave to us and powerful words haven’t really evolved in time, as you can see. I used speeches from various people in history, from Socrates to Steve Jobs and from Jesus Christ to the one that gave name to my disease, Lou Gehrig.

    They use mostly simple words in their speeches in their fight against racism, apartheid, terrorism, crime or their hope for freedom, achievement, ending poverty and curing diseases.

    In my image the only difficult word, “Umkhonto”, came from Mandela’s speech “I am prepared to die” from 1961, which means spear and refers to the armed wing of the ANC, we all know how the story ends and how it changed history.

    So not intellectual words, complex verbs or scientific equations, but words that everyone understand are the way to change. It emphasises one’s belief that you can change the world and it might not even be so difficult, not by shouting it of roofs, but by inspiring others with simple words.

    One Man

    For the ones inspired and interested in the speeches I used, here’s my list:

    • The Fall of the Berlin Wall – Ronald Reagan
    • Going to the Moon – John F Kennedy
    • The Vast Wasteland – Newton Minow
    • The Most Successful Drop Out – Bill Gates
    • Don’t loose faith – Steve Jobs
    • The Mountaintop – Martin Luther King
    • Against Imperialism – Che Guevara
    • The Gettysburg address – Abraham Lincoln
    • Farewell to Baseball Address – Lou Gehrig
    • Depart! – Alexander the Great
    • Apology – Socrates
    • Wonders of science – Albert Einstein                                      
    • I am prepared to die – Nelson Mandela
    • Blessed are the poor in the spirit– Jesus Christ
    • The sun shall never set on so glorious a human achievement’ – Nelson Mandela
    • I have faith in the righteousness of our cause – Mahatma Ghandi
    • Give Me Liberty or Give Me Death – Patrick Henry
    • We are not only scientists, we are men too – J Robert Oppenheimer
    • Freedom of Choice is a universal principle – Mikhail Gorbachev
    • Time Has Come for Universal Health Care – Barack Obama
  • So far… but so good!

    So far… but so good!

    One of the conclusions that came out of a report on the financial crisis was the complexity and lack of transparency that led to the ultimate meltdown of a system that was put in place over 40 years ago. Forced by public opinion, the excessive bonus culture of the financial industry came under intense scrutiny and the banking world was forced to reinvent itself, ultimately returning to a more sensible definition of home base.

    Many high-profile economists agree that our medical and health care system is about to collapse, hence prompting the Harvard Business Review to open @Hbrhealth and inspiring countless blogs with just as many opinions. Can we really fix the health care system from a business angle, like a failing bank, or might there be a different approach?

    Healthcare systems differ per country, but basically the concept is the same. Workers pay taxes and/or insurance premiums, while insurance companies and national health institutions provide the insured with care, medication, and more. From an economic point of view, the two sides of this equation should balance out. So far, so good…

    Let’s move on to pharmaceutical companies, many of whom made billions with blockbuster drugs which they invented in the sixties and seventies when development costs were mere peanuts compared to now. Some scandals at that time forced them to cooperate with government agencies to ensure that safety comes first in clinical trials. This has evolved into one of the most complex governance models there is on the globe. These companies now spend a lot of their earnings in research and development on trying to develop drugs for lifetime use. So far, so good…

    So where’s the catch? Well, as we say in the business world, “cash is king” and this is what is completely different from the financial crisis. Big pharmaceutical companies are sitting on huge piles of cash, scanning the market for small, innovative biotech companies to buy instead of using their capital to develop drugs themselves. The small biotech companies are doing their utmost to garner attention from the major players in the pharmaceutical industry with smartly designed trials which show efficacy to their bigger brothers.

    Is the system in an equilibrium? Hell no! Health care costs are skyrocketing and have doubled in most developed countries in the last 10 years. The pharmaceutical industry is struggling with longer development times and huge governance costs, not to mention the pressure to find cures…

    I sincerely think we face a chronic autoimmune disease in the health care system, where the system is attacked by its own antibodies, placed in the system many years ago, and is now suffering from a chronic illness. Eerily similar to what it was meant to prevent, isn’t it?

    But what if we apply some of the lessons learned from the global economic crisis in order to cure the current illness plaguing the health care system?

    Remember what it’s all about…

    The people at the top of the health care food chain are cognitively disassociated from the people it was designed to serve…the patients! It’s just like how the executives in the financial sector forgot about their customers.

    My idea would be for pharmaceutical/biotech companies to team up with insurance companies, health care institutions, and patients to create dedicated task forces per disease with a shared cost/revenue model. Pharmaceutical companies with great views and expertise will be in more task forces and thus make more money. Innovative biotech pioneers can look for neglected task forces for rare diseases. This is what I call a shared, innovative health care system, where everyone benefits and gets what they want. Drug companies make money for their shareholders, governments reduce health care spending, and last but not least, patients get their cures.

    Simplicity rather than complexity…

    Bigger doesn’t always means better. With the global population doubling in just over half a century, in parallel we created the most complex system on earth to provide for our health care. An average blockbuster drug costs over 1 billion dollars and between 12 to 15 years to make it available to the public. A clinical trial culture aimed to design and meet pre-defined outcomes, with many patients failing to meet the inclusion criteria, is thus left to its own devices. In a recent blog, Dan Ollendorf, Chief Review Officer at the Institute for Clinical & Economic Review, pleads to implement the so called “adaptive licensing” model, whereby a drug is admitted earlier to the market, with open access to all patients. This should be paired with a monitoring system that prevents us from creating a “learning” system, but rather a “curing” system. If we could implement platforms that make this model more easily and readily available for patients, with less knowledge being hoarded by the system, I sincerely hope that we would be able to remove the one and only obstacle to true change… the existing mindset.

    Transparent as glass…

    The data that comes out of drug trials and the research undertaken by task forces should be made public at all times. In a system that basically has been paid for by the people, it should not be allowed to put information behind paywalls. Trial data should not be protected by intellectual property rights and I am not the only one with that opinion. 4 senior scientists from the European Medicines Agency write in the leading New England Journal of Medicine that putting clinical trial data in the public domain will make it more cost-effective to develop new medicines.

    The system itself has a responsibility to serve the people it is meant to protect, inform, and heal. Companies are rewarded for the results they achieve in the task forces, and publishers should develop a different business model aimed at elucidating rather than educating.

    Reinvent the governance model…

    We all have to rely on science, otherwise we would be forced to jump out of an airplane without a parachute and learn to fly (or wait for God to save us). Undoubtedly science should be at the core of all the above. Companies that claim over-the-counter-drug effects from fuzzy trial data can be part of the sketched model and scientifically prove their concept. It is, at last, a shared model, but also with shared responsibilities. No tricky business, just a clear, open, and honest approach between task force members, supervised by independent bodies with a clear mandate and the authority to protect the system from becoming ill again.

    Without ignoring the current costs of health care, I think we should start at the root of the problem. This will automatically lead to a significant reduction in the rapidly emerging problems of a global health care system on the verge of collapse.

    So far…but so good!

    One Man

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