Ok......here are some more responses from the NMSS (after reading this last batch of suggestions.) Sharon, they will be addressing yours shortly.
1. Something I have mentioned previously is that funding organizations need to hold the funded researchers accountable.
This is a good point and one that we work hard to achieve. Every single researcher receiving funds from NMSS has to provide us with an annual report of their research progress. We on the staff review the progress against the goals stated in the application. If there are questions about lack of progress, then we query the investigators. There are no free rides. Every researcher receiving support from NMSS knows that the funds can be taken away for lack of progress. In addition if a researcher is coming back to us for continued support, our reviewers will scrutinize the application to make sure that the scientist has actually been productive (in other words published research papers) with the money provided. If they haven’t shown a record of progress, it is highly unlikely that they would receive funds from us.
2. The NMSS should at least facilitate networking between researchers.
We try to do this in a few of ways. First we have two new funding programs (the MS Collaborative Research Centers and the Translational Research Partnerships on Nervous System Repair and Protection in MS) that are specifically targeted to create a network of researchers and to bring new researchers into the field of MS. Second, we provide money to support scientific meetings of researchers across disciplines. These are not meetings that we specifically organize. Rather they are organized by the researchers. We also do no attach any ‘strings’ to our support other than to report to us on the outcomes of the meeting. Third, we (NMSS) organize scientific workshops ourselves with the express purpose of bringing researchers together on timely topics related to MS. Recent examples were workshops on the genetics of MS, design of clinical trials for MS given the proliferation of new potential therapies, nerve repair and so on. When we organize these we try very hard to bring a cross-section of the research/clinical community together in order to create the networks suggested. Fourth, we also sponsor the annual ACTRIMS (Americas Committee on Treatments in MS) meeting. That meeting which is open brings together clinicians, scientists, and others to hear about the latest MS research and to give them an opportunity form networks. Every two years this meeting is held in conjuction with our European counterparts in order to facilitate networks across our respective continents.
I would suggest that Wesley try to attend the Federation of Clinical Immunology Societies (FOCIS) meeting (being held in Boston this year) or the annual meeting of the American Academy of Neurology (being held in Miami), or the annual meeting of the Society for Neuroscience (being held in Washington D.C). These meetings are attended by many MS researchers and I think would perhaps paint a more accurate picture of what is happening out there.
2. And then you mentioned neuro-protection. I would like to see a project, perhaps leading to clinical trials, of difluoromethylornithine (DFMO).
This is new to me and so I can’t really respond other than to say that it sounds interesting. I can pass it along to researchers who may be interested in finding leads for neuroprotective compounds. Thanks.
1. There should be much more international collaboration in MS research.
We are trying hard to that. To give you a few examples. First, we do support projects outside of the United States We are currently funding work in Italy, Israel, Australia, the UK to name a few. Second, our special project, the MS Lesion project, is in fact a large international collaboration of leading MS researchers in the United States and in Europe. Third, our efforts to try so solve the genetics of MS involves a collaboration of genetics researchers in the US and Europe. Finally, our new initiative on Neural Repair and Protection in MS specifically was targeted to attract large scale collaborative groups from around the world. Check out our web site in the summer to see which groups are picked for this new collaborative effort.
2. Researchers given substantial sums of money should be required to come up with a real advancement in the knowledge of MS.
I would be interested in hearing ideas for measuring real advancement. From our perspective we can point to the fact that there are many many more drugs being tested in clinical trials today than there were just 5 or 10 years ago. This is real progress and this depends on the many researchers working hard to test their ideas, develop better understandings of the disease course of MS and so on. We continue, of course, to desire more and more research that leads to new and better drugs to manage and ultimately cure MS. One has to be cautious though about jumping to conclusions too quickly about whether or not a particular line of research is or is not really advancing the knowledge of MS.
To give you but one example, the new drug Tysabri was developed from research done to try to understand the how immune cells leave the blood stream and get into particular organs (the nervous system in the case of MS). When the researchers (one of whom was in fact funded in part by NMSS) were looking at the question, they weren’t really thinking about creating a new drug for MS. They were trying to study how these immune cells move from one place to another and the rules governing that process in the context of MS. At that time scientists had great difficulties answering that very important question. These researchers came up with some molecules which helped them answer their question, and in the course of doing that someone came up with the idea that these sorts of molecules might be another possible way to help treat MS. Looking back one can say that that initial work was a true advancement, even though at the time they didn’t appreciate just how big an advancement that would be. I am optimistic that there are lots so similar examples out there.
3. More research in collaboration with researchers of other degenerative diseases eg Parkinsons, Alzeimers etc.
We are always interested in trying to foster collaboration between researchers within the MS community and from other diseases. We try to do that through our workshops and our MS Collaborative Research Centers program.
4. Much more research on neuro-protection.
We agree. That is why we created our program on Nervous System Repair AND Protection in MS. Up to now our understanding of how to go about that research was primitive, but now it seems to have reached a point where we can tackle it in a way that can lead to possible therapies.
5. Too much research has relied on EAE and there are some big questions as to how applicable this is to MS.
This is a good point and one that has been debated long and hard. Finding other models that mimic MS is not trivial but scientists are trying hard.
6. I would also like to see a research project which answers the question (once and for all) - is MS an auto-immune disease?
This is an interesting idea. I’m not certain that one could answer this question with one project or actually do so once and for all but one could try to get at the different parts of the issue.
1. My question is just simply what it will take to get some of that money put aside for some clinical studies of LDN. It looks like a researcher would have to put together a package stating what they would do? Can they be more precise about what would be needed. Perhaps if all of us knew exactly what it would take, we could start working on accomplishing that.
Raising money for a clinical trial for LDN would involve a few steps. First, the researcher would have to be willing to do a placebo-controlled trial (i.e some get LDN and their responses are compared against those not receiving LDN). This really the only way to validate the drug’s effectiveness in MS. Second, it the researcher would have to come up with a clinical trial design that asks the right questions in with the right group of patients. A poorly designed trial will only lead to confusing outcomes and that helps no one. Finally once they have the trial designed they would need to put together a proposal, determine how much it would cost and then approach the pharmaceutical company or a funding agency like NMSS or NIH for support. That’s the process in a nutshell. The key though is convincing the researcher to do the placebo-controlled trial. That is the gold standard for showing the effectiveness of a potential therapy.
1. ….if our MS societies will not become more proactive in directing certain trials which have limited profit potential, I’m afraid nobody will and it will be up to the MS patients themselves to try to get some cures working as is happening today. The limited profit potential might even be a good measure in deciding which trials should be sponsored by MS societies since it are these trials no company will invest in and clinical trials with already approved drugs can be done within a reasonable budget so are doable by the NMSS.
This is a good point as well. I guess, what I would need to know is what is considered a reasonable budget. To give you an example, a recent trial (which is being funded by NIH) to look at whether or not the combination of Avonex and Copaxone work better together than when taken individually is going to cost close to $30 million. That was a reasonable budget for the trial. That is close to what we spent last year for all of our research projects. I am not trying to whine, but to simply illustrate the challenge that we and other private funders face with regards to clinical trials. We keep an open mind, and do indeed fund smaller trials particularly in their initial stages.