Tag: biotech

  • 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…