Issues in ALS: Why fair funding will increase innovation
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Amyotrophic lateral sclerosis (ALS) is a devastating fatal condition, in which the nerve cells controlling muscles (motor neurons) are lost, followed by the muscles themselves. This results in paralysis and eventually death, usually within 4 years. ALS is the most common motor neuron disease, with a 1:400 risk to be affected at any time during adult life. The first diagnosis of what later would become known as ALS was given about 150 years ago, but it took until the late 20th century to start discovering the disease process and mechanics. Still, ALS is poorly understood, and only two treatments with small effects are currently available. This is likely the result of several issues on different levels, defined here as biological, technical, and structural. These problems especially restrict research progress for complex disorders such as ALS. Solving these might thus bring us closer to effective treatments. First, the biological problems facing ALS research are the complex processes that cause it and the differences between patients. A number of complicated biological processes have been associated with ALS. To make matters worse, they are not all causative, making it difficult to distinguish the ones that can be targeted for therapy. Additionally, not all these processes are shared between patients, with the likelihood and form of ALS dependent on genes, environment, and aging. This makes it hard to find shared factors and processes that cause the disease. Second, technical problems to research include the difficulty checking disease progress and poor design of studies. Disease risk, outlook, treatability, and the effects of treatment are measured with certain markers. However, as we do not understand the disease, making effective and reliable markers is very hard. It is also not possible to diagnose ALS before any symptoms, which causes an average delay between onset and treatment of about a year. Without reliable markers, putting patients into groups is difficult, making it hard to design studies and interpret their results. Additionally, as ALS is poorly understood, animal models are oversimplified to the point they do not reflect the disease process very well. Testing treatments in these simple models has been ineffective for finding good therapies for ALS patients. Third, structural problems to science are formed by high competition for funding and the way we reward research. Here, competition can lead to a lack of collaboration and exploration of unconventional approaches, both of which would contribute to studying complex diseases. On the other hand, the quantity of research is often rewarded instead of its actual quality, which pushes short and simple studies. As a result, researchers cannot perform extensive complex research or develop better methods and models. Especially for complex diseases like ALS, this is a major issue, as an extensive and thorough approach is necessary. Such an approach would be designed to take the biological issues into account, would give more time to develop suitable methods and disease models, and would allow the publication of all results, not just exciting ones. Effectively, there could be a large increase in new discoveries, possibly leading to the development of better treatments. To solve the problems posed to ALS research, a serious change is required in how research is performed and valued. Competition for funds can be limited by more general funding, where all qualified research groups get some basic funds. Additionally, research quality should be valued over quantity, considering more than just the number and score of publications. These changes would create a better environment for studying complex subjects like ALS. In turn, this would allow a deeper understanding of its cause and different forms, the improvement of markers, methods, and models, and finally the development of effective therapies.