The Case for Granting Accelerated Approval to GM604

This past week I went to Washington DC to speak at a rally urging the FDA to grant Accelerated Approval for GM604. I saw old friends, made new friends, connected with many families in the ALS community, and met with members of Congress.

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While we do not yet know how effective GM604 will end up being, Accelerated Approval was specifically designed to allow patients access to treatments, that have been shown to be safe, for serious conditions with limited therapeutic options.

Please note that Accelerated Approval does not come at the expense of good science. Data will continue to be collected and full approval will not be granted until results of a confirmatory trial prove efficacy. There are simple statistical techniques that can be used to control for placebo effects even in single-armed trials.

Here is the text of my speech in which I lay out why I believe Accelerated Approval should be granted:

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March 25, 2015

Hello, my name is Stephen Finger.  It is an incredible honor to speak here today in front of so many families involved in this fight. I grew up across the river in Arlington, VA, but never anticipated coming to DC for this reason.

Two years ago I was diagnosed with ALS. Between my son James’s first birthday and his sister Mary Adair’s third, I was told, what I thought was a minor issue with my hands, was a disease that would rob me of my ability to play catch, to play tag, to walk, to speak, to eat, to breath.

My family’s lives where forever changed and if one thing was clear, it was that time was of the essence. And just as we recognized the need to live urgently; to hug today, to laugh today, to live today, to love today. We also recognized the need to act urgently, to act today. That is why we are here. Team Gleason says “Not quietly”. The ALS Association says “The time is now”. We are here telling the FDA that the status quo is not good enough and they need to do everything in their power speed the search for a cure. We are here urging our members in Congress to remind the FDA to work with the flexibility and urgency needed to fight this universally fatal disease.

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I have now been a part of this community long enough to see patients who were diagnosed with this disease after me lose their fight. Last year, I had the pleasure of meeting Trickett Wendler in the halls of Congress. A young mother with three kids, a wife, a friend, a relentless advocate, she was diagnosed in the summer of 2013 and passed away in less than 2 years. Always one to speak her mind, she has asked her friends to continue to fight. So we are all here on her behalf.  On Tony Conway’s behalf. On so many other great people’s behalf, who have been lost to this disease that we know can be solved.

We are not just here acting in their memory, we are here inspired by the way they fought this disease. Everything we do must be done with the recognition that every 90 minutes another mother, another father, another daughter, another son, another friend will be lost.

It is with that urgency that we are here today asking that the FDA grant GM604 Accelerated Approval. Let’s not force it to go more of the traditional trials that will take years to complete. Based on the results of the Phase I and II trials, let’s make it available immediately to patients who desperately need it, while we gather confirmatory data required of full approval. We are not asking today to rewrite any laws. We are not asking for a special exception. We are asking that the FDA uses the tools at their disposal to make this treatment available to as many people as possible in the shortest amount of time.

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Accelerated Approval is not a new program. It has been around for over 20 years, specifically designed for situations like this. Congress has provided them with the ability to act with the urgency this situation merits. The 2012 Food and Drug Administration Safety and Innovation Act, with near unanimous support in both the House and Senate, I quote: reiterated the “Agency’s longstanding commitment to regulatory flexibility regarding the evidence required to support product approval for the treatment of serious or life-threatening diseases with limited therapeutic options”.

That is us! Here is their chance to live up to their own directives. Today! We are simply asking that they use the tools at their disposal to act with the urgency Congress told them to act with. The urgency deserved by the families in this fight.   No ALS treatment has been granted Accelerated Approval before, so here is an opportunity to show a new way forward. The status quo is not good enough.

In GM604, we have a drug meets the FDA’s own criteria for Accelerated Approval. Its safety has been demonstrated according to traditional standards. Over 50 patients have received GM604 with no adverse events, consistent with the preclinical work.  Surrogate endpoints for efficacy have been reached. Even in a small trial, GM604 had a significant effect on Forced Vital Capacity, which we know is a predictor of survival. Because ALS is a complex disease and progressions vary, meeting traditional standards of efficacy will require additional large and lengthy trials. Well we don’t have time for that and Accelerated Approval was specially designed to address this. While we wait for confirmatory data, let’s give current patients a fighting chance.  That is the directive Congress has given.

Now sure after Phase I and Phase II trials there are still risks, but there will still be risks after Phase III and more importantly, we all know what the risk of inaction is. Accelerated Approval does not mean we enter the wild, wild west. Data will still be collected and approval could still be revoked if confirmatory trials do not go our way. But given the safety data, given the suggestion of efficacy, given the prognosis of the disease, given the urgency of the situation, isn’t that a risk we are willing to take?  Isn’t that a risk the FDA is supposed to take? Given the evidence from the competed trials, doesn’t that make more sense than telling current patients to sit tight for two years until another trial can be completed?  We won’t sit here quietly. Too many people have already been lost. Too many people have shown us the importance of acting today.

The FDA has recognized that different conditions require different acceptance of risk. They have granted Accelerated Approval status to cancer drugs, to HIV treatments. They have this tool at their disposal specifically for situations like this.  Just because ALS is not communicable does not mean that we can be complacent.

So we are telling the FDA to act urgently, to act today, to act for the families in this fight, to grant Accelerated Approval to GM604.

Thank you.

If you have not done so, please sign the White House petition requesting that the President urge the FDA to grant Accelerated Approval to GM604.

https://petitions.whitehouse.gov//petition/request-fda-grant-accelerated-approval-potentially-life-saving-treatments-als-now-starting-gm604

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Genervon’s GM6

Over the last couple of weeks, you may have seen information regarding Genervon’s GM6 therapy. GM6 is a naturally occurring peptide that regulates multiple genes and has shown no health risks.  Results of the latest trial suggest that it is effective at hitting its targets and has a statistically significant impact on patients’ progressions. Because of this, Genervon is seeking conditional approval from the FDA so they can make the treatment broadly available to patients while they continue to collect data to ensure that the drug is safe and effective (http://friends4eric.blogspot.com/2015/01/condition-green.html). Over 75,000 members of the ALS community have signed a petition encouraging the FDA to grant the approval (https://www.change.org/p/food-and-drug-administration-accelerated-fda-approval-of-genervon-s-gm6-for-use-in-als).

If I went into a complicated surgery, I could tell the doctors to go to extraordinary measures to save my legs or my voice. It would be a tragedy if they couldn’t accomplish that goal. If I woke up without the ability to stand or to walk or to speak, the doctors would feel that they had failed. Yet supporting the status quo and suggesting Genervon should do a Phase 2b trial, and then go from there, is essentially resigning me to the same fate, if not much worse. There is nothing in the immediate research pipeline with the capability of truly regenerating nerve cells. Even the stem cell trials work by creating a healthier environment for the remaining motor neurons. So slowing or stopping the disease is the immediate goal. And the immediacy and the urgency is real. Without an effective treatment I know where I, and many of my friends, are heading. Life expectancy is 2-5 years after diagnosis.

GM6 may not eventually prove to be effective for everyone.  However, two safety trials have found no significant safety risks. The pathways that it affects are not inherently dangerous. The company laid out pre-specified biomarkers and clinical primary and secondary outcome measures. This 12-week study actually had one of the most extensive biomarker plans of any trial I am aware of. So the researchers could not just monitor symptoms, but could measure what was occurring around the motor neurons.  In the trial GM6 had a statistically significant impact on a number of these outcomes. The results were consistent with what they said the treatment should do and were consistent with its performance in animal models.

I have a PhD in an empirical field. Over the past 10 years or so I have worked with data for a living. I understand the limitations of a 12 person trial, but also understand how unlikely it would be to get these results at random. Something positive is going on and we have lots of evidence that the treatment is safe.  This is a perfect candidate for accelerated conditional approval. This type of approval would require full data collection and surveillance going forward and final approval would be based on the data. If serious adverse events occurred or the data didn’t hold, approval could be quickly pulled. Inaction in all likelihood would be much more dangerous than action. “Do no harm” takes on a different meaning in this community. The pharmaceutical company wants to move forward and 75,000 members of the community are ready to act.

Suggesting we wait two years for the results of another trial is suggesting I tell the doctors I don’t want them to go to extraordinary measures. It is suggesting I should resign myself to what this disease will do to me over the near-term. And when you have ALS two years isn’t the near-term, it is a lifetime. How many of us won’t survive the next two years?

Yes, the standard protocol would be for another small-scale trial, but two years ago at a hearing with the ALS patients, the FDA said they recognized the urgent need for an effective treatment and would be flexible in working with the ALS community.  The FDA was flexible in addressing the Ebola outbreak last year, and because of the heroic actions of doctors and governments, less than 10,000 people died from the disease. Without any changes, roughly 75,000 to 100,000 people will die of ALS worldwide this year. Let’s let someone try to be a hero. Please urge the FDA to grant GM6 accelerated approval.

https://www.change.org/p/food-and-drug-administration-accelerated-fda-approval-of-genervon-s-gm6-for-use-in-als

Logistics

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It is now almost 2 years since I was diagnosed. All in all, my progression continues to be relatively slow. I am almost embarrassed to talk about many of my symptoms given what some of my friends are going through. I can still drive, get dressed, and feed myself. I can still do most things I need to do for myself in my day to day life. However, each of these things is getting more and more difficult. They have moved from being harder than they should be, to hard. Showering and drying off and getting dressed is a tiring proposition, so I don’t do it that often. Just ask my wife. At this point we can see that I won’t be able to do some of these for too much longer. Driving the kids to school, taking them to the zoo on my own, this may be the year I have to give these things up. Not being able to drive is obviously scary because of what it means to my freedom and the time I get alone with the kids, but it’s more than that. It also takes away one of the ways I can still contribute to keeping the trains moving on time for our family.

For example, we have our house on the market right now. It’s not the easiest thing to keep the house in “showing” condition with crazies running around with toys galore. So while we typically keep the house in pretty good shape, it’s a scramble when someone calls and wants to see it. The most efficient way to clean up is for me to take the kids and get out of Dodge. Then Cara’s left to actually get things done. This has been our strategy for lots of things around the house. I disappear with the kids, and she works. Obviously, this isn’t an equitable division of labor, but it has become the unfair default. When I can no longer drive, we will have to change the dynamics once again.

So in many ways, this year is going to be about logistics. What do we have to do to keep our heads above water? Steve Gleason is famous for saying that given where medicine is right now for ALS, “Technology is the cure”. I can see that day coming, but for us right now, logistics is the cure. Figuring out the hired help we need, how to get it consistently, and how to make sure everything doesn’t just fall on Cara. How do we make our daily lives manageable enough so that we can focus on other things?

If the kids are fed, laundry is done, and everyone gets a bath at least once a week, then we are winning. Then days are again about doing puzzles and reading stories and playing in the yard and going to ballet and getting the kids down and having a moment together of peace and quiet on the couch. Then physical limitations are just annoyances. These aren’t insurmountable tasks, but ones that need to get done.

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Once we accomplish them, then I know we will spend another year making incredible memories.  Like the afternoon in the fall when we had our family pictures taken and the kids somehow acted perfectly. Afterwards we noticed a brewpub at the edge of a pond in the same complex. So we sat on the dock in the sun, the kids feeding the blue gills and turtles and Cara and I enjoying a beer, laughing, waiting for one of the kids to fall in, and absolutely amazed when neither did. Memories like these:

Once we figure out the logistics, my third year with this disease will be remembered as the one in which we find a cure!

TDI Weekend – November 2014

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Cara and I spent this past weekend up in Boston visiting ALS Therapy Development Institute. ALS TDI is a nonprofit biotech solely focused on finding a treatment for ALS. It was started by a patient and his family and continues to be closely aligned with patients, having a patient as the chairman, and family members of patients making up the majority of the board seats. The funds we raised this summer from our event in New York went to TDI and Cara and I continue to strongly believe in their mission and team.

On Thursday, TDI hosted a Leadership Summit. They had presentations from a number of scientists and a panel focused more on the business side of drug development. Then Saturday night was their annual gala. And on Monday, I got to tour the lab and gave my samples for their Precision Medicine Program.

This is a program that got accelerated by an influx of funds this summer. They originally planned to collect data from 25 patients, but now are enrolling hundreds. On Monday alone, they had six patients in. They took blood and a skin samples from me to be used to map my genome and to grow stem cells. Then they will test 50,000 drugs against each of our stem cells to try to see if they can find subgroups of responders. One of the most difficult things about this disease is its heterogeneity. Patients differ in their age at onset, the location of their onset, their progression, etc… So it seems crazy to think that some drug will affect everyone the same, but that’s how most traditional trials are set up. This program will be able to move somewhat in reverse, first identify who responds and then look for commonalities in patient’s backgrounds or genetics. I also get to wear accelerometers that will track my movements so they can look back over time and see what influences progression. (Right now the devices are kind of distracting as I keep waiting for them to start flashing to let me know my table is ready.) TDI is using technology that has only recently become feasible on a large scale and are quickly deploying funds to try to find answers as fast as possible.

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I think the two most amazing things from the weekend were meeting the patients that were there and the team from TDI. I got the chance to speak with a number of scientists and operations people throughout the weekend. You cannot help but come away incredibly impressed by their passion for what they were doing. Not just that they thought the science was interesting, but they were truly devoted and passionate to finding a treatment. They were emotional when speaking with patients, recognizing they weren’t trying to solve some puzzle, but they were trying to save our lives. Many of the employees that TDI came to the organization because they had some connection to ALS, but the others are hooked as well. They recognize how far this science has come, but more importantly realize it has to be pushed further to truly make an impact. One moment that stood out to me was towards the end of the summit Dr. Perrin, the CEO, said, “I am not going anywhere. I’m staying here until this is done.” That was sense you got from each individual. They haven’t just bought in to the mission of the organization, but they see it as their own personal mission to end this disease.

I was one of six people honored with their annual leadership awards at the summit. You had Anthony Carbajal, who always wears his heart on his sleeve, perfectly cry through his speech (https://www.youtube.com/watch?v=zvivmqeIxfQ). Hope and Steve Dezember perfectly danced through theirs (https://www.youtube.com/watch?v=mJdNapQgJ5A). Pat Quinn was partying at a buddy’s wedding, but sent a video and you could see his determination to make a difference (https://www.youtube.com/watch?v=vxyDyRgc6fg). Then there was Deb Quinn, a patient who movingly spoke about her family being ravaged by familial ALS. More than 20 people in her family have died of ALS and now she has a son with the SOD1 gene causing her to proclaim, “I will never be silent about ALS.” Her speech is here, https://www.youtube.com/watch?v=nGJDoUP4KK4. It’s amazing.

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At the cocktail hour on Friday and at the gala we were able to meet other patients and family members. People who I talk to online. Families committed to pushing TDI forward. Advocates fighting to improve patients’ lives. People who of lost loved ones to this disease, still doing everything they can to change its course. (Sarah Coglianese describes meeting some of them here: http://www.speed4sarah.com/notes-on-a-party/ If you are not diligently reading her blog, you need to start. Now.)

As we were flying home, I was thinking about all of the great people we had met over the weekend. I was smiling thinking about having turned my Internet friends in to real friends. So many great people facing a similar future to ours with positivity and determination. Everyone had a spark. People who love life and won’t let this disease change that. People who I can’t wait to see again. But then it hit me. By next year’s gala, we will all look different. Some of us may lose our ability to walk, for others the ability to talk, for others the ability to breathe. Science is moving at an incredible pace, but so is the disease. The urgency is still there. Like Deb said, we can never be silent, we must continue to keep ALS in the spotlight, and to make sure organizations like TDI have the resources they need to move forward as fast as possible.

However, it is comforting to know we are in this with such great peers, families, advocates, and scientists, all working to end ALS.

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Renewed Hope, Renewed Urgency For ALS Organizations

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% 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.

ALS Ice Bucket Challenge

As many people have pointed out, dumping a bucket of ice on your head isn’t going to cure ALS and it doesn’t do anything directly for my family. But it still matters.

Most people in the ALS community believe that if more people were aware of the horrible nature of this disease, then much more resources would be devoted to finding a treatment or a cure. I personally knew nothing about ALS until I was going through the process of receiving my diagnosis. So anything that shines a light on what people currently living with the disease and their loved ones are going through is a positive.

Sure in some of the videos it isn’t clear whether or not people truly get it or just want to see themselves on Facebook. It is a vanity project for many. Lots of people who watch Justin Timerlake or Jimmy Fallon or Aaron Rodgers just laugh at the spectacle and move on. However, in the majority of the ones I have seen, people are thinking about ALS. ALS was the fourth most searched topic on Google this weekend. Maybe they just click on a link to see who Pat Quinn or Pete Frates is. Well, it only takes a second for the reality of ALS to hit you when you see Pete Frates. An expectant father and charismatic former college baseball star who now is losing all of his physical abilities. He lives in Boston close to some of the most preeminent ALS researchers in the world, but right now there is little they can do for him. When he was diagnosed he got the same prognosis that Lou Gerhig did 75 years ago. 2 to 5 year average life expectancy. No real treatments. No cure. He is left to fight on his own, with his family, and with anyone that will join him. He has inspired thousands of people to get involved. If pouring ice water on your head and making silly videos on Facebook brings more people into that fight, then we as a community win.

With something like this, you also see how many people are supporting other families facing this disease. You realize that the tent is growing. It is similar to the feeling I got when I went down to New Orleans. Everyone in that city is behind Steve Gleason. That means they are behind me as well. Seeing the outpouring of support for other people living with ALS through a something like the ice bucket challenge gives me hope that someone has my families’ back, that the ALS community will receive the resources it needs to find a treatment or a cure as quickly as possible. And it is all playing out. Awareness is leading to actions. Many ALS organizations, like ALS TDI (www.als.net) have reported tenfold increases in fund raising over the past couple of weeks.

So I completely agree with the skeptics, pouring a bucket of ice over your head will not cure ALS and does not help families living with the disease. But people learning about the horrors of the disease and the desperate need for treatments, people reaching out to support those of us affected, people choosing to generously donate ALS charities like www.als.net and www.teamgleason.org, this makes a difference. This gives hope to me and my family.

An Economist and pALS’s Response to “Prevalence of Amyotrophic Lateral Sclerosis —  United States, 2010–2011”

(As published on July 25, 2014 in the Huffington Post http://www.huffingtonpost.com/stephen-finger/national-als-registry_b_5620631.html)

Yesterday the first paper related to the National ALS Registry (http://www.cdc.gov/mmwr/preview/mmwrhtml/ss6307a1.htm?s_cid=ss6307a1_e or http://www.cdc.gov/mmwr/pdf/ss/ss6307.pdf ) was released.  The ALS Registry is a very difficult project. Because ALS is not a nationally notifiable condition, it is difficult to accurately identify every patient. This study aims to get an idea of the population of ALS patients by collecting data through to two different means.  Both datasets are worthwhile. However, both are also imperfect. My concerns are not related to the long-term value of the Registry. My concern is that this study’s conclusions are based on the idea that the combination of the two imperfect studies captures the entire population. There is absolutely nothing in the data that suggests this is the case. Any results based on this assumption are at best flawed, and at worst disingenuous and reckless. It is counterproductive to disseminate statistics that we know are wrong. The results are already being quoted by ALS organizations and the media. I cannot understand how the CDC or the ALS organizations can justify pretending that these results are representative of the US ALS population.

The database portion of the project began in 2006. It includes administrative data from four major sources. These sources combined cover 90 million patients. There are well over 300 million people in the US. Therefore, we know these databases will not cover every patient.

The registry was launched in 2010 and requires patients to self-enroll on the website. Again, this is imperfect system for trying to capture data from every ALS patient. Not everyone has access to the web or is willing to fill out these forms, especially after being given a diagnosis of this magnitude.

Both of these approaches are useful and should contribute to our knowledge of US patients. However, it is unrealistic to think that combined they have captured every single patient.  Therefore, using them to calculate prevalence in an unadjusted manner is incorrect.  In addition, neither source is a random sample, so any demographic findings are as likely to be the result of the selection methodology as they are to be of the result of the true underlying characteristics of the entire ALS population.

For example, there are 10,261 cases identified in the databases. The registry identifies 3,715 patients. If we believe either one of these databases was doing a good job capturing all of the patients, we would expect there to be substantial overlap. This is not the case. Less than one half of the registry patients also appear in the database. This suggests not only shortfalls in the databases to capture all patients, but it also suggests that if we had other means of identifying patients, we would likely be able to find many more patients who have not appeared in either source.  The article shows us that we have been able to identify 12,187 cases using the registry and four government databases. It does not identify how many patients are likely to be living in the US at a given time. The prevalence calculation is at best a back of the envelope estimate. It should have been presented as such.

US ALS Population as Presented.                                True US ALS Population?

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The databases also cover a much older sample. They include Medicare, Veterans Benefits Administration and Social Security Administration data. Each of these sources is highly skewed to an older population. Using this data to calculate the age distribution of ALS patients in the country is absurd.  Less than one-third of registry respondents under the age of 50 are in the database. Restricting the data to patients under 40, only one-fourth of the registry patients appear in the database. The registry says almost 10% of patients are under 40. The database says only 3%. One or both of these sources is obviously biased. Simply averaging two incorrect figures isn’t necessarily going to give us something better. The database obviously does a bad job identifying younger patients, so unless the registry is capturing all of those missed patients, the demographic data is going to be wildly incorrect. Again, why are we presenting this data?

The paper also reports some education and employment statistics from the registry. Without considering who is likely to appear in the registry, these numbers must be taken with a grain of salt. I do not think anyone believes that college graduates are 50% more likely to get ALS. To the authors’ credit they note the limitations of these sections though they are still being picked up in the press.

I still have hope that the registry will be a valuable tool for researchers. I have personally taken some of the surveys through the registry and believe they ask intelligent questions. The registry has the potential to help identify things like environmental risks. However without knowing more about who is in the registry, it is grossly premature to try to use this information to calculate representative figures for the entire US population. I am highly discouraged that this initial paper tries to make those claims. There is a shortage of good data related to ALS, so any data that is published becomes “commonly accepted”. Therefore, we have the utmost responsibility to make sure we are not providing misinformation.

I respectfully ask the article’s authors, as well as the organizations publicizing these results, to clarify the shortcomings of their conclusions before these results become taken as fact.