Renewed Hope, Renewed Urgency for ALS Organizations

I understand that many funded clinical trials will fail. I understand that there will be research projects that lead to dead-ends. I am okay with mistakes or failures, but not with complacency or business as usual.
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People continue to dump ice water over their heads in the name of finding a cure for ALS. It is over a month since I saw the first videos start showing up on my Facebook feed. Since then it seems like most everyone in the world has done it, but still I see new posts each day. They continue to make me smile and they continue to give me hope that now we will have the resources to change the course of this disease. People now have a connection to ALS and ALS organizations have seen an unprecedented surge in funding. And it continues.

While buckets continue to be dumped and donations in quantity and in magnitude that we have rarely seen before continue to come in, we must also be focused on moving forward. I was encouraged last week when ALS TDI announced that the unanticipated $3 million in donations they received this month would allow them to fast-track two programs that had been in their pipeline. A drug trial that they thought would take two years for them to raise the necessary funds for, will now go forward. If it succeeds, that means patients get this treatment two years faster. That is almost an extra generation of ALS patients saved. If it fails, we learn WHY two years faster and can proceed accordingly.

Organizations like Project A.L.S. and the Packard Center have also stressed that additional funds will be put to use immediately. These funds will change what they can do and how fast they can do it.

The scale of the donations that the ALS Association has received far exceeds what any of the other organizations have generated. While ALS TDI can quickly put a 30% increase in their yearly budget to work, is unrealistic and imprudent for the ALS Association to hastily make a decision on what to do with $100 million. They also must make sure adequate funds are devoted to patient care and improving the quality of life of all pALS. However, that does not mean we can't do anything today. That does not mean we can proceed in a business as usual fashion. We should not wait until October to act.

For example, on August 11 the ALS Association announced 21 new research grants totaling $3.5 million. In the call for proposals, the maximum award was listed as $240,000. That means, on average, these grants covered roughly 60 percent of the maximum request. Some of the proposals may not have requested the maximum, but I think it is clear that many of these grants were not funded at the requested amounts due to the size of the budget. Increasing the scale of these awards would immediately change the pace of these research projects. In addition, limited funds likely restricted the number of grants awarded. There likely were additional worthy projects that went unfunded. With the new resources at their disposal, these projects could move forward quickly through additional grants. Though the ALS Association should take the time to think about the best uses of this unprecedented surge in donations, they also should make a point to fully fund promising research as soon as possible.

Similarly, we have been told the FDA is speeding up the trial process, but now institutional review boards are slowing us down. The Northeast ALS Consortium (NEALS) has been working to create a centralized system. Provide them with the resources they need to get it up and running. That will cut months or even years off each stage of clinical trials. Make sure we move this forward as fast as possible today. While the ALS Association makes a long-term plan, they should also be putting targeted resources into fully vetted, "shovel-ready" projects.

People have used the analogy of not wanting to end up like a lottery winner who four years later wondered where all the money went. For myself, my family, other families facing this disease today, and those families who will be cruelly introduced to the disease in the coming years, my biggest fear is we will be asking where all the time went. No matter what we do, we cannot get the time back.

If in four years' time we have spent the ice bucket money and have not found a cure, there still will be armies of people combating the disease. There still will be families pounding the pavement to raise additional funds. There still will be new patients being told they have been stricken with a uniformly fatal disease with no cure, whose friends and families will generously devote their time and resources to the fight.

I understand that many funded clinical trials will fail. I understand that there will be research projects that lead to dead-ends. I am okay with mistakes or failures, but not with complacency or business as usual. I will continue to pester my friends and family for donations as long as I feel like their money is bringing us closer to a cure. However, it will be hard to continue to make requests if the largest organization is sitting on a war chest. For years we have spoken about the urgent need for new funds. We have repeated the Dr. Appel's quote that, "ALS is not an incurable disease. It is an underfunded disease." Well now that some of the necessary funding has come in, let's respond with equal urgency in our actions.

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