ALS Advocacy

ALS Advocacy
Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Wednesday, April 27, 2016

You Had To Be There

On Monday I decided to listen to the webstream of the important FDA Advisory Committee hearing
on Sarepta's eteplirsen for DMD.  You've probably read the news accounts by now, but you had to be there (or at least listening as the drama unfolded) to grasp the significance for ALS.

The FDA's Dr. Woodcock set the stage.  This hearing was about the Accelerated Approval path.  This is important, not only for DMD, but for a lot of quickly fatal diseases with no existing treatments.  She made a comment that she has made before about the significance of making a Type 2 error -- rejecting a therapy that actually works -- with this disease.

In the morning we heard well-designed and polished presentations from the drug developer and investigators.  We got the gist that this stuff was doing something good for a well-defined subset of DMD kids.  We heard about measures.  We learned about what's important in DMD kids' disease progression (a term I hate).  We learned about the logic of the drug's target.  But during question periods, there were FDA Advisory Committee comments constantly dripping into the conversation about this not being a double-blind, placebo controlled trial.  The committee was slipping in boxer's jabs regarding historical controls that really didn't land any individual wallop but were accumulating.

By the afternoon it was the FDA's turn to present, and it was anything but polished or logical to follow, but the discomfort with a trial that did not have a placebo control group kept bleeding through.  There were more jabs about endpoint measures and meaningfulness of the drug target. I kept wondering if this was really about the science and the medicine, or was it all about giving a panel a comfort level with the way they had always made decisions.  Bold they weren't.

And then there were the parents and kids and DMD physicians who got their turn to talk.  The press accounts talk about the emotion they stirred, but it wasn't all emotion.  They spoke to data.  They spoke about their experiences with the drug.  They spoke about what is important to them.  They spoke to looking at the patients in the room and seeing some results.

At the end of the day there were some votes on questions that I guess were supposed to constitute the committee's "advice."  They were difficult questions to grasp as was reflected by the number of abstentions on several.  There was a terrible quality to the voting moments, especially when we heard some of the voters' comments.

The votes reflected MDs who were not comfortable with the historical controls.  They kept saying that historical controls can work, just not these historical controls. The constant drip of discomfort with anything but a double-blind, placebo-controlled trial won.

A wise physician once said to me that you can have all the studies and data in the world, but you have to look at and treat the patient in front of you.  That was certainly missing in the Advisory Committee's comments on their votes.

I'm pretty sure that most everyone in that hearing room felt that the drug was working on some DMD kids and that it was doing no harm.  The data just weren't speaking to it well enough for the FDA to feel comfortable.

There is always a chance that any FDA decision is wrong.  That's a fact. Even the most comfortable ones for them could be wrong.  That's a fact we live with.  Why can't they?  Some there want to be perfectly comfortable in decision-making while people are dying.

The final FDA decision for Sarepta comes in the next month.  It's not looking optimistic, but just maybe some regulator will realize that the odds are that the Type 2 error was made on Monday, and that's devastating for a nasty disease with no effective therapies.

This is a big deal for the fight against ALS.  Accelerated Approval is a path that was designed for diseases like DMD and ALS, yet the FDA does not seem comfortable with it.  DMD has an FDA Guidance document.  Did that make a hill of beans of difference?  Is anyone working on the FDA Guidance draft for ALS (which is modeled closely in process and in content after the DMD document) talking to Sarepta and the DMD groups to find out how a difference in the FDA Guidance document could have changed Monday's results?

It was a difficult, draining day to listen on Monday.  We must learn from it.

Sunday, April 24, 2016

There Is Stunning Loss In 78,000 Empty Chairs

The first time I attended the ALS Advocacy Conference and Day in Washington, DC, it was 2004.  The highlight to me was on the first day.  A women with ALS named Dee Chiplock grilled a presenter from the FDA with some pointed questions and comments.  It was uncomfortable for everyone but Dee.  I was hooked.  This is how we could make a difference with people who hold the ultimate bureaucratic power to make a difference.  It was like flexing our democracy muscles.

The first evening there was a candlelight vigil at the Jefferson Memorial.  There was no press.  There was nothing to let the nearby tourists know why these people had gathered.  We were pretty cloistered.  That seemed like a missed awareness opportunity to me.

At that time I thought how poignant it would have been to have 6,000 empty folding chairs in front of our crowd to represent the Americans lost to ALS in the past year.  Every empty chair would represent somebody's somebody -- a mother, a father, a sibling, a loved friend, a child, a grandparent,...

And the next year there could be 6,000 more including Dee Chiplock's.  And the next year 6,000 more.  In a few years the Mall would be so clogged up with empty chairs that people in Washington would have to pay attention.

There were always organizational excuses not to do anything to foster outside awareness in Washington.   One chapter actually went rogue and brought a stunning display to Washington for two years that garnered a lot of attention despite the objections of the conference organizers.

And a couple dealing with ALS arranged (completely on their own) a wreath-laying ceremony at Arlington one year to remember the veterans who have died from ALS.  That was taken over by conference organizers the next year.  They got the film in the can and dropped the ceremony after that.

Last year some people dealing with ALS who are pressing for FDA change arranged a rally in a public space outside the conference hotel.  Conference attendees were discouraged from attending.  In spite of that, there were some bold and bright Dee Chiplocks bravely flexing the muscles of democracy in lieu of their plated lunch in the hotel.

And so it goes.  The annual conference goals don't change much.  The official legislative requests are remarkably modest and similar year after year.  The faces change, but we don't get a sense of the cumulative loss that we experience from ALS year after year after year after year.

The conference hotel is fancier now and the conference content is much more scripted.  It's largely an infomercial for the achievements of past legislative priorities.  And the candlelight vigil is now being  replaced with an evening of hilarity with a comedy troupe.

This will be my 13th ALS Advocacy Day.  And there would have been 78.000 empty folding chairs on the National Mall by now.  Over 1,500 rows of 50 empty chairs.  And each of those empty chairs is somebody's somebody from the U.S. who has been lost to this stinking disease since 2004.

The emptiness of those chairs is painful.  The loss is stunning.  The loss is startling.  We need to stun and startle.  We need to flex more  democracy muscles in memory of Dee Chiplock and every somebody who has died from ALS.

Tuesday, April 19, 2016

Do Not Be Misled by the Everybody-Does-It Defense

In case you've been asking questions about the sweet government contract that the ALS Association signed for "education and outreach" for the CDC's ALS Registry, you may have heard an excuse that lots of not-for-profits have government contracts.  That's true.  And many of them are a blessing for taxpayers and the citizens they serve.  They compete to do things economically and deliver better than a government agency can.

The government contract that ALSA was handed by the CDC is a far different beast.

1. It was a no-bid contract.  There was no competition.  It was a single-source proposition.  Nobody in government purchasing seemed to catch the fact that later they handed a similar no-bid, single source contract to the MDA.  How many single sources can one task have?

2. The ALS Association has been the advocating organization for the CDC to do the ALS Registry.  They tell us how exactly how much to request every year.  We advocates go to ALSA's conference and are handed the list of precisely what to request of legislators, including an unsubstantiated amount for the CDC for the Registry.  I repeat, they tell us exactly how much to request.

3. Transparency demands that a charity be crystal clear with advocates and donors when it enters a contractual relationship with a government agency and has a financial interest in a project that we are told to support on Capitol Hill.  That didn't happen. It took a Freedom of Information Act request and two years to find out exactly what ALSA was supposed to be doing and what their contractual work standards were.  When it's that difficult, the parties obviously don't want to talk about it.

4. And then there's the contract clause prohibiting the contractor from lobbying for the project using contracted funds.  There is no doubt that ALSA has managed to fulfill the letter of the law, but have they not defied the spirit of it in the way they send us (sincere, unquestioning advocates) to Capitol Hill with a huge number for the CDC's Registry?

With two signatures on a contract, the ability to provide responsible advocacy and oversight on a big government project was compromised.

Please do not be misled by the "everybody does it" defense.  Everybody does not do it.  And if anybody else does, that does not make it right.

Friday, April 1, 2016

Whoa! Enough of the $10,000,000 Annual Autopay.

Enough!  We need our charge card back.

Many of us ALS advocates have worked hard for a number of years to get a national ALS Registry. The goal was to have complete and orderly data for a better scientific understanding of ALS.  Every year the ALS Association hands us a funding number that we are told to request to enable the CDC's ALS Registry to meet that goal.

For a decade this project has been on autopay.  We need to stop.  We need to look at the bills we've paid and the value of goods and services we've received.  We need to take back our taxpayer charge card and see a detailed budget and decide what should happen next.


We should not be asked to raise $10,000,000 without having a detailed budget accounting on how it will be spent.

Contractors on the project, including the ALS Association, should be crystal clear about their pieces of the budget and their work products.  Advocates should not have to file Freedom of Information Act requests and wait two years to see their contracts as we were forced to do!

And how is ALSA,  the contractor that tells us advocates how much to request for the project,  living up to the spirit of its contractual and ethical obligation not to lobby for appropriations for its project?

The secrecy of the budget and various financial interests in the project are troubling.  The annual  cycles contribute to the lack of transparency.

When the hundreds of advocates trek to Capitol Hill each May for funding, many of them are employees of the ALS Association, trustees of the ALS Association, and advocates whose travel has been subsidized by the ALS Association.  The deck is stacked to avoid questions and candid discussion on the cost and value of the registry.


After all of these tens of millions of dollars have been invested already, we have received exactly one annual report from this project.  It was in 2014 -- .  Sure there was a time-lag in the ability to report data because of the passive data mining techniques being used on old government files, but the quality of the report itself was a concern.  Here is the best evaluation of that report that I've seen --  There are serious completeness concerns.

And the second "annual" registry report on ALS did not arrive in 2015 and has still not arrived in 2016.  We taxpayers paid the autopay but the reports are not arriving as promised.

The project has been plagued with scope creep, perhaps a result of a budget that is more than is needed to deliver its core mission.  We get tweets about the wonderful resource locator at   Take a look in the lower left corner of that busy page.  Why are we taxpayers paying to maintain a directory of clinics and chapters when we could just have two simple links to those organizations' own directories?  And if you feel brave, give that registry resource locator a try. Did it work for you?  It hasn't worked for us for months.

Another of the products of the scope creep has been the clinical research notification tool.  It is the "success" story that has been used in recent years to deflect the lack of deliverables in the registry's core mission.  We hear that 70,000 emails have been sent.  Nobody has ever told us how many unique people have been notified and how many of those people actually enrolled in clinical research as a result of those emails.  Impact?  Is the registry leading people with ALS to be passive rather than active in their searches for clinical research opportunities?   These registry studies represent a tiny fraction of currently enrolling ALS trials, and these notifications are almost all for observational studies.  One is actually notifying people of another government-funded registry.   This kind of notification is not patient-centric. We have an expensive way to crank out emails that has been spun into a "success" story.


Last year a large meeting was convened by the ALS Association to get a broad spectrum of ALS organizations to "collaborate."  There were just two agenda items.  One was to agree on the number of people in the United States with ALS.  Was it collaboration or collusion?  In either case, they couldn't reach a decision.  More study was needed.  Over $60 million invested in a registry and more study is needed?

Any modicum of oversight on the registry project would seem to come from its own annual meeting group. The following is from the ALS Registry Act:

The people with ALS, family members, and other individuals are hand-picked by the ALS Association and the MDA (both paid government contractors on the project).  Those two government contractors are the only ones allowed to nominate anyone to participate per the current CDC policy.

If that annual meeting had every attendee self-identify as a government employee, a paid government contractor on the project, or the handpicked nominee of one of the contractors, there would be nobody else left in the room.

Who in that room asks some simple questions like how many unique people with ALS were seen at contractors' ALS clinics in the past year, and of those, how many are self-enrolled in the registry?  Who in that room asks simple questions like how many new people with ALS were brought into the contractors'  clinic system in the past year, and how many of them had self-enrolled in the registry?  And who in that room would come prepared to answer such simple questions?

And do they ever talk about budgets and actual spending and the project's delivered value at that oversight meeting?

HHS Secretary Burwell and we taxpayers are not well served by the cozy quality of participant selection for this oversight group. It is anything but open. It is anything but businesslike.   It is anything but collaborative.


How did they come up with the $10,000,000 annual figure?  How is that large budget built, please?  When we asked a few years ago, the response was that $10 million was the most the legislative leaders thought they could raise for the registry.  That's no way to fund a project!  That's precisely the way to fund a government boondoggle.

A project's budget needs to be built from the need for resources to produce defined deliverables.  To build a project around a huge budget number is backwards.  It explains the scope creep.  It explains the lack of accountability.  It is anything but good stewardship of taxpayer funds.  It is anything but good stewardship of scarce resources in the fight against ALS.

So here we go again.  Legislators were approached about another $10 million by those who participated in ALSA's invitation-only March fly-in.  Hundreds of us will be sent to Capitol Hill and will be told to reinforce that request on May 10.  ALSA and the CDC will get another $10,000,000 or close to it.  The CDC says that the second "annual" report that is almost a year late is in final review.  I seriously doubt that we see it before May 10.  Then there will be another cozy oversight meeting later in the summer.  We won't get minutes from that meeting until close to the time when we will be told how much to request for another year.  It's a cycle that keeps people with vested financial interest in the project in control of messaging.  It's a cycle that keeps a bloated request for funds de-coupled from accountability for project results.

It's time to take this project off of autopay.  We've become enablers.  Enough!  We need our charge card back.  

Thursday, March 17, 2016

This Was Simply Wrong On So Many Levels -- Neuraltus NP001

This is the story of a couple of my friends and an investigational drug for ALS -- Neuraltus' NP001.  It contains many pieces of ALS history that must not be forgotten.  And they must never happen again.

Introduction -- It Started in 2010

I met Rob and Ben online as a result of their participation on some ALS message boards. They were both really smart gentlemen with ALS.  They were both scientists.  Rob was an accomplished statistician who had professionally worked for General Motors on predicting car parts failures.  He said it was excellent background for his second career in trying to outsmart ALS.  Ben was a medical physicist.  His expertise was in medical proton accelerators.  He said that he worked on equipment that took cancer patients within inches of their lives so that they could live.  He wanted the same right for himself.  Rob and Ben were also young fathers with lovely families.  We three all had dogs named Otis.  And so the story begins.  By time you finish reading this, you will know them, too.

It started in June of 2010.  People interested in ALS science kibitz on the forum at  The first inquiry about Neuraltus' NP001 came up.  Did anyone know anything about it?  The question was posed by Eric Valor, a young man with ALS and a gifted scientist. When he asks, people listen.  And he was an essential force throughout the NP001 saga that followed.  The images below are from conversations on the fora at or

The conversation began.

By August, Rob was really digging in.  He and the others on the forum do this with everything that comes up in the news or literature, trying to figure out what the stuff is. Rob found evidence that it was related to a compound already marketed in Europe for other indications - WF10.  It was some form of sodium chlorite.

The  Phase I trial was a safety/dosing trial on the surface, but there was a placebo arm.  Something else was going on.  Every ALS trial is an efficacy trial, regardless of what the phase says it is. Maybe that was it.  Or maybe they were looking for a biomarker.  You just don't think of placebos as being essential in a safety/dosing trial.

Rob scoured the literature. That Phase I trial went quickly.  By November they knew that NP001 was safe and a dose-dependent biomarker had been tracked.  Interesting.

By December, Rob had uncovered some patent applications.  The startling part was that as far back as 2005 the similar product, WF10, had been used in two people with ALS with good results.  Five years earlier there was a reason to think that this could do something.  Five years.  Nobody pursued this in five years.  That was another 600,000+ ALS deaths ago.  You can see Rob's comment below.

Chapter I -- The 2010 Symposium

Every December there is a global ALS symposium that attracts around 800 researchers and healthcare professionals.  It has never encouraged people with ALS to attend, but that didn't stop Rob. It was in Orlando that year. He was interested in the science. Nothing would discourage him from attending.  The most hyped new drug for ALS to this point was dexpramipexole.  NP001 was still in the shadows.

Rob met researchers working on several clinical trial candidate drugs.  He came home with a particular interest in NP001.  It was based on data (albeit a very small sample) and science.  And he wanted to find out if his patent research was correct.

Chapter II -- The Phase II Trial Starts

Keep in mind that dexpramipexole Phase II trial enrollment was being hyped by a lot of organizations.  NP001 was simply not on the radar.  Rob was concerned about the NP001 exclusion criteria that would keep him out of that trial.  He was quickly approaching 24 months post-onset, a traditional barrier to ALS trial participation.

NEALS (referenced in Rob's message above) is a large Clinical Research Organization (CRO) that is considered to be the leading such organization for ALS clinical trial design and administration.  Neuraltus was using a different CRO.

People online were starting to get interested in NP001, and a man with ALS made a telling comment on a forum.  There were no contingencies for access to NP001 after the trial or for those with ALS who did not qualify for the trial.

Yes, people with ALS can interact intelligently with scientists and business executives, yet they are never called to the table for input before things are designed. The importance of trial design will become even more apparent as you read on.  In the meantime, Rob kept working trying to get the inclusion criteria for the NP001 trial expanded.

Rob was placing the investment of himself in medical research with NP001.  He enrolled in the trial and didn't say too much publicly about his experience.

The schedule slippage began.  Take a look at the last paragraph.

Rob didn't say much at all publicly, but he told me that he was noticing things.  He was still driving and one day realized that he was able to work some controls in the car that he had not been able to work before.  ALS trials always try to determine if people are getting worse less quickly than they were before.  Rob was actually seeing improvements. He didn't want to say much but was tracking data religiously and taking videos of what was happening.

Ben had indicated that he was going to join the dexpramipexole trial. It seemed most promising to him.  After some correspondence with Rob, he decided to try the NP001 trial even though it was less convenient for him.  The two scientists wanted to be human lab rats with something that  perhaps was actually doing something.  It may not have been a long-term solution, but it was doing something.

Ben enrolled and started NP001 infusions on June 20, 2011.  And his tagline reflected his belief that the data must be captured publicly if the science is to advance.

The trial was designed to require 21 weeks of infusions with a placebo, a low dose, or a high dose.  Each participant would then be monitored every four weeks during a washout period to watch the activity of a proposed biomarker.  No drug would be available during that washout period, and there would also be further monitoring through about 9 months with a final interview at 49 weeks.

Chapter III -- Fill the Trial!

Ben was on a mission as you see below.  Both Ben and Rob believed that if there were participants who were actually improving that the Data Safety Monitoring Board (DSMB) for the trial would step in and not withhold product from those who were experiencing improvements.  Surely.

As the realization that the trial design wasn't ready for patients experiencing good things was sinking in, Rob asked people to reach out to Neuraltus.

Ben loved coffee.  The ALS had made it impossible for him to drink it without choking.  He emailed me that he inadvertently had taken a cup of coffee at one of his infusion appointments in Lexington and suddenly realized that he had finished a cup of coffee.  Something was going on.

Rob expressed frustration at NP001 not being on organizations' promoted trials lists.  

They started speculating on possibilities for approval, yet they knew that the only thing they could control was filling that trial quickly.

The reality of the trial design was sinking in.  The biomarker was going to be tracked come hell or high water, regardless of what that meant for the people in the trial.  Read Rob's last sentence below.

And Ben questions whether we have developed an obsession with biomarkers and have lost sight of ethical ways to find them.

And Rob agrees.

And the statistician Rob pleas with researchers to look at what is going on here.  It's different.

Ben continued to fill seats in the trial.  He traveled from Indiana to Lexington for his infusions.  He searched for ALSA support group meetings in Lexington and showed up one night.  The people there were not aware that in their own backyard there was a trial seeking volunteers.  That's a telling indictment of the effectiveness of organizations at getting information to patients regarding clinical research.  If they wonder why people don't enroll in trials, they should look in the mirror.  Ben and Rob filled a trial in record time through their own efforts.

And Rob was giving interested people information that they were not getting from the trial sites. 

And their efforts continued.  And ALS organizations should have been embarrassed.

Both Ben and Rob were keeping meticulous records in their profiles at  They were encouraging all in the trial to post their data, too.

And some things are just not captured by ALSFRS-R.

And they continued to recruit relentlessly.

There was still a confidence that the DSMB could and would see that some patients were getting actual improvements and would act.

The term "Accelerated Approval" enters the conversation.

Chapter IV -- The Horrible Premonition Sets In

The reality of an inflexible system is starting to set in to Rob who is approaching the end of his time on NP001.  The DSMB won't learn about their improvements.  There will be no decisions to modify the plan.  And there is a long period staring at him when he will no access to the drug so that a biomarker can be tracked.

They are capturing and watching data.  Statistician Rob has figured out some subgroups (high side effects, low side effects, no side effects), and responders seem to be in the high side effects group.  Rob's words below are a terrible portent of what is to come -- "This has me really concerned for what will happen at the end of the trial. I am convinced that I wlll again decline, just like those of the patent..."

Ben comments on the trial design and his pessimism about what will happen next is setting in, too.

Ben will fulfill his commitment to the trial, but he is planning ahead.  And his words in his last paragraph below about his work and cancer patients should stick with us all.

And Ben continued to monitor his grip strength, an outcome that this trial did not measure.  And he was clearly measuring improvement.

And see Ben's words below. Surely the world would watch in horror as those in the study begin to decline again. Surely.

And Rob speculates and says that Neuraltus must lead.  Surely they will. Surely.

Rob speaks to what we already knew about the lessons from the old data on WF10.  Is Neuraltus going to withhold drug to watch a biomarker crash while the trial participants crash?  Why is this trial designed this way?

Neuraltus knows the patients are going to crash.  Why are they doing this?

And Rob notices that the patient advocacy organizations are conveniently failing to pay attention to their plight.

We hear more from Rob on his condition and the trial's endpoint selection.

And again, Rob speaks to what is within the control of Neuraltus. Surely they will act. Surely.

Chapter V -- Neuraltus and Others Fiddle, Patients Burn

Clearly the search for the worshipped biomarker is at odds with urgency and humane treatment of participants with this trial design.

Rob and Ben finished their commitments to the trial.  They endured the washout period and crashed, each losing the gains and wondering where they ended up relative to where they would have been without the trial.  Neither posted much about how poorly they were doing without the NP001 while their data were monitored, but their emails reflected their frustrations and disappointment, not only with Neuraltus, but also with organizations that they thought would advocate on their behalf.

Both tried some do-it-yourself  NP001 options after that washout period.  Ben was featured in the Wall Street Journal for his efforts.  Both men tracked their data religiously at PatientsLikeMe so that others might learn.

They weren't the only ones who wished the trial had been designed differently.  I met a grandmother at the ALSA Advocacy Conference that year.  She had terrible bulbar symptons.  She said that she had been in the trial and had done so well, and since the washout period when they were observing her without NP001, she had just gotten so much worse.  And there were others on the message boards who felt the same way.

Below is Rob's ALS Functional Rating Scale R (ALSFRS R) chart from PatientsLikeMe.  ALSFRS R is a summary measure that misses many details.  You can see the uptick during the period when he was on NP001.  There was an immediate and steep decline while Neuraltus watched his data decline relative to their biomarkers.

And below is Rob's weight chart. 

Here is Ben's ALSFRS-R chart.  ALSFRS-R wasn't granular enough to pick up many of his gains

And Ben, too, had the precipitous weight loss.

They did not have easy deaths.

Rob died on September 10, 2012.
ARDEN - Robert Wayne Tison, 42, passed away peacefully on September 10, 2012 in his home in Arden, after a brave battle with Lou Gehrig's disease (ALS). He was a loving and devoted husband to his wife of 17 years, Kelly and beloved father to Tyler (15) and Sydne (12) and his best buddy Otis.
He is survived by his wife, Kelly Renea Tison and two children, Tyler and Sydne; his parents, Barbara and Darryl Tison; a brother, Lynn Tison (Lisa) and a sister, Cassie Jerore (Duane) of Michigan; in-laws, Grandpa George Thatcher, Rich and Sue Thatcher of Asheville, sister-in-law, Amy Herren of Asheville, and many nieces and nephews who loved him very much (Matthew, Megan, Michael, Mitchell, Madison, Taylor, Luke, Cody and Jordan).
Rob grew up in Commerce Township, Michigan and was a 1988 graduate of Walled Lake Central High School and 1993 graduate of General Motors Institute. He has worked as a mechanical engineer for Rockwell, Con-Met (Oregon), American Axle, Cane Creek Cycling, Borg Warner Cooling and Turbo systems. He was a licensed Realtor, and most recently a project manager for Glennwood Custom Builders.
He was an avid outdoorsman and loved cycling, running, hiking, geocaching, kayaking and camping. After being diagnosed with ALS on March 8, 2010 he focused his skills on finding a cure for ALS. He was a patient representative for the CDC National ALS Registry, ambassador for the Northeastern ALS Consortium, and advocated on Capitol Hill three times for money to fund ALS research. He received many awards for his advocacy, including the ALS Association 2012 Rasmussen Advocate of the Year award.
He could be found regularly on giving advice and input with graphs to back up his analysis. A Facebook Page was created in honor of his advocacy for ALS (Persevering - You are a game changer).
A gathering for the Celebration of Life service will be at Zephyr Hills Baptist Church, 283 Shelburne Road, Asheville, NC 28806 at 10:30 AM Wednesday, September 12, 2012 with the service starting at 11:00. The family graciously asks that in lieu of flowers, memorial donations be made in Rob's memory to the college fund savings account for Tyler and Sydne Tison at any local First Citizens Bank.

Ben died on August 15, 2013.
Ben Harris, 46
JAN. 2, 1967 — AUG. 15, 2013
BLOOMINGTON — Ben William Harris, 46, passed away peacefully at his home in Bloomington on Thursday, August 15. Ben was born on January 2, 1967, in Menomonee Falls, Wisconsin, to William and Claire Harris.
Ben attended high school at Northfield Mount Hermon boarding school and received his BS in physics from Columbia University. He went on to receive Masters degrees in both philosophy and physics from the University of California, Riverside, and subsequently worked as a dosimetrist and later calibration physicist at Loma Linda University Medical Center. He was granted certification by the American Board of Radiology in 2005. He continued to work with protons when he moved to Bloomington, Indiana, where he was employed as Director of Medical Physics by ProCure Treatment Centers.
Since first learning of his ALS in January 2011, Ben showed incredible courage and indomitable will to make a difference in the face of something that most of us would have succumb to just out of hopelessness. He has shown us again, it’s not when you go, it’s how you go. Ben is survived by his wife, Rebecca; son, Rawden; parents, William and Claire; siblings, Michael, Daniel, Kathy, Amy and Jason; as well as four nieces and seven nephews. Ben was a gentle soul, a devoted husband and father, and a brother without peer.
A memorial service will be at the Unitarian Universalist Church in Bloomington at 3 p.m. on Sunday, August 18. Friends are welcome to visit with the family in their home after 5 p.m. In lieu of flowers, donations may be made to American Funds for an account set up in the name of his son, Rawden.

Chapter VI -- So What?

This isn't meant to be about a particular drug.  This is about real people and a process that failed them.  This is about two beloved husbands and fathers who volunteered to advance the science who were stuck with a clinical trial design that was inhumane.  This is about all those who weren't paying attention.

This must never happen again. 

Every time we hear about the important search for biomarkers, we should ask whether that will be allowed to impede an efficacy trial.

Every time a drug developer proposes a Phase II trial on the cheap (and, yes, I realize that they are all expensive), we should ask whether it should wait until they can afford to treat the volunteers humanely.

Every time a person with ALS is handed informed-consent documents, he or she should ask, "What will happen to me if this stuff actually does something good?"

We must learn from all that we lost in these two fine people.

Dad always told us that there is a right way and a wrong way to do everything.  This was so terribly wrong.