In response to a recent question; the following are my choices of the "big 5" questions that are currently being asked in MS research.
1. CURE: Can we cure MS by rebooting the immune system early in the course of the disease?
This question is currently being asked by treating MS'ers early in the course of their disease with drugs such as alemtuzumab and cladribine or with autologous bone marrow transplantation. We know these therapies often render MS'ers disease-activity free (no relapses, no MRI activity and no disability progression), however, will these treatments prevent these MS'ers developing secondary progressive MS? To answer this question we will need to wait about 15-20 years for an answer. If these treatments don't prevent the secondary progressive MS then it is likely that MS is a primary neurodegenerative disease.
2. NEUROPROTECTION: Can we protect nerves and axons during acute focal inflammatory events, for example optic neuritis, or in progressive MS so that they can be saved to preserve function?
Several treatment strategies are currently being tested in clinical trials to answer these questions. If these trials are positive this will almost certainly lead to large combination therapy studies, with one therapy targeting inflammation and another therapy aimed at protecting nerves to maintain function.
3. PREVENTION: Can we prevent MS with physiological vitamin D supplementation?
The hurdles and logistics of doing the correct studies to test this hypothesis are enormous. However, I am confident that we will eventually get public health officials to see the light (sunlight) and to start a population-based food fortification study. The costs of not doing something are simply too high not to do something. Can we let a whole new generation of MS'ers be born?
4. CAUSATION: How do all the risk factors for developing MS interact with each other?
Over the last 20 to 30 years several genetic and environmental risk factors for developing MS have been identified. If we can work out how these factors interact with each other, and the timing of these interactions, we may have a better idea of how to prevent MS in people at high-risk of developing MS (siblings and children of MS'ers). Several research groups, including ours, are working on this problem. This research question gets to the heart of the cause of MS.
5. SYMPTOM MANAGEMENT: Symptomatic therapies for MS'ers are in general sub-optimal. In particular our ability to treat fatigue, cognitive impairment, spasticity and pain. Developing and optimising symptomatic therapies will have a major impact on the quality of life of MS'ers.
We are and others are working on symptomatic therapies. The solution to these problems, however, lie with big pharma. Pharma have the resources to best tackle these problems.
You will be disappointed, particularly if you have progressive MS, that I have not added restoration of neurological function to this list. The reasons for this are complex; firstly, I don't want to raise unrealistic expectations (neurorestoration in the MS arena is a long way off) and secondly, the evidence that we can do this is poor at present. The improvement we see in people with highly-active MS going onto an effective DMT is probably due to recovery from natural mechanisms as a result of the particular DMT suppressing further damage, rather than the DMT triggering repair mechanisms directly. However, I remain open to the idea that some DMTs may have a dual action; on the one hand suppressing inflammation and on the other hand stimulating recovery. In my experience recovery of function tends to occur in MS'ers early in their disease course rather than later in the disease, presumably because compensatory mechanisms fail or burn out with time and possibly with age. The latter seems to a common theme across the spectrum of neurological diseases.
The holy grail of MS is neurorestoration!
I would be interested to know what you think of my "big 5"? Do you agree? If you disagree please tell me why?