Tag: Amyotrophic Lateral Sclerosis

  • Beyond the Ice Bucket

    Beyond the Ice Bucket

    congress-blog

    Five years ago, I was unexpectedly struck by amyotrophic lateral sclerosis, or ALS. I have dedicated my life to moving mountains in what was, at first, an unfamiliar field. My entrepreneurial career included real estate, flowers, oil and shipping. But the past five years had challenged me with the steepest learning curve I’ve ever faced: my disease.

    During my crash course on ALS, often called Lou Gehrig’s disease, I noticed that regulatory review and approval seriously hindered the process of drug development and results in missed deadlines.

    Although I am an academic drop out, I’ve learned to appreciate science as the backbone of evidence-based theories. However, being a patient with a killer disease means never ruling out miracles – the desire for a cure drives everything I’ve come to appreciate the passion and compassion of people within the ALS community – the patients, researchers and companies that underpin that drive.

    I’ve also learned to apply the skills I developed in my previous careers to drive innovation in drug development. This has led to the development of what is currently one of the largest genetic datasets of ALS patients on the globe. The shift in focus from technology-driven to disease-driven drug development is necessary in an industry ruled by science but governed by non-disclosure agreements. The heart of next generation drug development lies here – with innovative research approaches that include the experiences of the patients that the industry is focused on helping.

    Today, real innovation increasingly comes as a result of patients’ involvement in drug development. Admittedly, most pharma and biotech companies have patient-centric advocacy embedded in their strategy and mission statements, but patients have traditionally formed just a small part of the drug development equation. The patient voice should echo in all parts of the development path, including governing bodies, ethical committees and expert panels. I’d go as far to say that if that was done 50 years ago, we probably would not have been able to increase fivefold the time it takes for a drug to come to market. Fortunately, this is starting to change.

    Patients are increasingly aware of their ability to play a significant role in the biotech R&D paradigm. Social phenomena like the Ice Bucket Challenge and increased patient involvement to accelerate drug approvals have served as good examples for both the industry and the patient community. We should not omit science and data from advocacy campaigns. R&D should remain a scientific process, not one driven solely by popular vote. However, placing patients at the center of the drug development process can help spur changes and direct attention to unmet medical needs.

    Industry and government also have responsibility to recognize this shift. Pending U.S. legislation like 21st Century Cures Initiative and industrywide conferences such as the BIO International Convention provide opportunities for patients to make themselves heard, and for stakeholders to recognize these new roles.

    I founded a biotech company that aims to address a deadly disease with innovative, transparent research, genetic data analysis and accelerated therapy development. Our for-profit status allows us to apply general business rules to attract capital and advance our mission. We still face the boundaries obstructing fast development tracks, but this generation of patients, “patients 3.0,” will eventually break down traditional boundaries and help streamline and expedite drug development. With more than 5,600 people diagnosed with ALS each year, the sooner, the better.

    Bernard Muller

    This blog was published as the Congress Blog on The Hill on June 10th 2015

  • Sterke Leeuw

    Sterke Leeuw

    sterke leeuw
    Richard Branson’s “screw it, let’s do it” is typerend voor ondernemerschap, gewoon doen en de onmogelijkheden oplossen wanneer ze zich voordoen. Ikzelf zeg altijd dat ondernemen draait om 3 dingen; mensen, geld en omstandigheden. En het is vooral het laatste dat bepaalt of je succes hebt. Je aanpassen aan de altijd weer veranderende omgeving, niet alleen zakelijk, maar ook in je privéleven.De manier waarop ik omga met het feit dat ik ALS heb, wordt gekenmerkt door aanpassingen en dus vooral mij te focussen op de dingen die ik nog wel kan. Dat lijkt eenvoudig gezegd, maar dat is het natuurlijk niet. De ziekte heeft voor iedereen een andere beleving, voor de één een kwelling, voor sommigen ook een openbaring, een zinbeleving, van waar draait het eigenlijk om in het leven. Die relativering kom ik bij heel veel mede patiënten tegen en verklaart ook die enorme saamhorigheid tijdens evenementen voor ALS.Mijn hele leven draait om mogelijkheden, ook wanneer onmogelijkheden aan de horizon ontstaan, probeer ik het negatieve om te buigen in het positieve, vaak daarbij denkend dat het leven maakbaar is. Maar ik ben ook een realist, wetend dat er zoiets als het lot bestaat. En als dan de koers van je leven een andere wending neemt, kan je maar beter je kompas pakken om in ieder geval bij te sturen.

    Ik ben niet godsdienstig, maar geloof sterk in de evolutieleer van Darwin, die zegt: “Het zijn noch de sterkste, noch de meest intelligente die overleven, maar degene die zich het beste aanpassen” en zo is het in mijn ogen.

    Toch kent iedereen die angst en dat soms hulpeloze gevoel van die volgende dag, die siddering die je voelt als iets simpels je niet meer lukt, overslaand in woede en frustratie. Het omarmen van die angsten, dat gevoel accepteren is een overwinning op jezelf…!

    In de top 5 van grootste angsten in Nederland staan; de dood en spreken in het openbaar, nou beide heb ik inmiddels wel overwonnen en daarbij ben ik trots op mezelf. Die trots wil ik graag breken in 1500 stukjes en geven aan al mijn mede patiënten, want ik weet dat niet iedereen even sterk kan en wil zijn. Toch schuilt in deze gastblog ook weer een diepere betekenis omdat LeoForte staat voor “sterke leeuw” en laat dat nu precies mijn sterrenbeeld zijn en figuurlijk betekent het een zeer zware beproeving ondergaan, nou dat noem ik dan in ons geval een understatement…

    Maar laten we vooral vechten als leeuwen en de omstandigheden aangrijpen om het onmogelijke, mogelijk te maken…

    ——————————————————————————————–
    Deze gastblog is geschreven voor mijn mede patient Ries van der Velden en
    zijn stichting Leoforte. Kijk voor meer info op http://www.stichtingleoforte.nl

     

  • Building bridges…

    Building bridges…

    HiRes

    Someone recently called me a “patient” entrepreneur. Yes, I am an ALS patient, but no, I am definitely not patient! 🙂

    The things I undertake to battle my disease can be described as entrepreneurial. However, it’s just who and what I am. Do I have a choice? I certainly do, although I can’t help being me. Don’t put me in a wheelchair and ask me to just stare out the window. It would probably break me… Don’t sit me in front of a TV. I’d probably just switch that crap off! Don’t give me face time with politicians unless you want me to tell them what their doing wrong. I’ll lecture businesses on what to do and how to spend their money, and scientists on what they should try to discover. Now, surround me with my fellow patients and I immediately feel a compassion that fuels my drive to find a cure. Facing my family fills me with love—something that everyone needs in their life!

    On my journey, I encounter stigmas towards patients, rigid social dogmas and oftentimes, outdated scientific paradigms. I am surprised at how our society has embedded fear into the roots of the very stakeholders that should be striving to make a difference, instead choosing compliance and governance over innovation and revolution. What has happened to the revolutionary mind of mankind that brought us to where we are today? How can we call ourselves innovators, when in reality, we’re just maintaining the status quo?

    Cultural and national differences are amongst the challenges I face in my endeavours, but our disease is global. It has no borders and certainly needs a more cosmopolitan approach. One of the lessons I try to share with others is that despite the adversity being faced, cultural norms dictate that people are always approaching it from different angles. We must be compassionate, but not blind. In my opinion, this is the only way to fill the pre-judged gaps in our opinions.

    As human beings, we are the only species that can feel both love and compassion. We have evolved beyond the confines of natural selection where the weak are left to themselves and the strong survive. When we combine compassion with our will to survive, we can overcome many issues and hell yes, even make the weak stronger!

    Right is not always right, and wrong is not always wrong. Hence, why I keep building bridges…

  • 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…