Meet The Change-Agent Brothers

There is a video at http://www.kickstarter.com/projects/1295266764/patients-finding-cures-the-drug-trial-revolution that is a "must see."

Please don't just read the text.  I'm one who prefers to read than watch, but this is worth watching.

When was the last time it was so easy to be a part of a revolution?

Let's Stop Storing Needles In Haystacks

Did you ever wonder what Dr. Benatar talked about at the May, 2012, ALSA FALS Summit?  Did you ever have trouble remembering if Dr. Scott talked at an ALSTDI conference?  Would you love to have a look at an ALS Registry presentation from 2008?  Does NEALS provide any presentations from its annual meeting?  Is there a Neuraltus presentation from last years International Symposium in Sydney?

Have you ever tried to find any ALS-related Powerpoint stack that was made available somewhere online after a workshop?

Industry groups that hold lots of conferences typically have great archives of presentations available in orderly manners online.  Members know where to go to find the presentations.  Groups share tools. They have great institutional history and know the value of access to information for their constituencies.

Unfortunately with ALS presentations, it's like trying to find needles in multiple haystacks.   Somebody stored the presentations somewhere (maybe) but it takes a relentless Google genius and countless hours to find some of them.

How about a common, co-branded, well-maintained spot that all of our ALS organizations would share to provide the Powerpoint stacks and other documents and media from their conferences and workshops?  That would make the information much more accessible and would greatly enhance its usefulness.  

Please?  It's all about the patients, right?

p.s. This also leverages resources.  Curing ALS is hard. This is not hard.

Finally, An Organization Uses Global Numbers To Describe A Global Problem

For decades our American ALS organizations have used estimated U.S. numbers of new cases and numbers of patients.  For decades we have left an impression that ALS is a "small numbers" disease.  Thanks to ALSTDI for finally speaking about ALS in global metrics.

We All Have A Lot To Learn

There have been many online discussions of some serious ethical dilemmas regarding people with ALS in two recent clinical trials.

The rules of the trials seem pretty simple, yet there are a lot of things that can happen that make the interpretation of those rules a matter of life and death for people with ALS.  They deal with the ticking clock and a future with no great choices.  It's more than troubling when a good choice unanticipated when the rules were written is taken from them.

I now realize that I have a lot of questions about how a clinical trial's rules came to be.  I'm sure there are a lot of people involved -- the pharmaceutical company, scientists, the clinical investigators, the FDA -- oh, and patients -- and I have a lot to learn about who does what.

• Who sits down and writes the rules for a clinical trial?
• How many people are involved?
• Who has input into the inclusion and exclusion criteria?
• Who approves the rules?
• Who pays for the rules of the clinical trial to be written?
• How long does it take to write the rules?
• At what point does the FDA see the rules?
• How are clinical trial sizes established?
• Can any of the rules be clarified once the trial starts?
• Who handles the interpretation of the rules when a question arises?
• Who has the final say on an interpretation?

One thing I have learned in the last few weeks is that those many people involved all have a lot to learn from one another.

When Will It Stop

Carey Hall died from ALS this morning.

It's Time For Some New Scientific Tables

In early December a large global contingent of professionals involved in the fight against ALS will hold their annual meetings in Chicago.  There are three related conferences -- one for an organization of organizations, one for allied health professionals, and one for researchers.

Football programs and golf tournaments often feature a celebrity athlete at each table for those attending banquets.  How about a some people with ALS at the tables during the many meals at these meetings?  They are our celebrity heroes.  Let's make it real, and perhaps a scientist who has been spending years on a theory will benefit from learning how to work the suction machine or feeding tube.  Food always stimulates some interesting conversation, and perhaps these people should converse more.  Oh, people with ALS often speak via technology, but they can and deserve to be heard.

We talk about breaking down barriers among patients and healthcare professionals and research scientists.  They need to speak to each other.  They need to listen to each other.  The all need a place at "the table."

Perhaps there will be some tables in Chicago where the process can start.


Below are some links to information for the meetings.  It's thoroughly confusing and not particularly welcoming, but perhaps that's all the more sign that it's time for people with their own skin in this game at the table.

http://www.mndassociation.org/research/International+Symposium/Symposium+Registration+page

https://www.mndcommunity.org/sslpage.aspx?pid=416

http://www.lesturnerals.org/files/3113/4401/1886/Alliance_reg_form_2012_Chicago_fillable.pdf

Are We There Yet? Are We There Yet?

As we saw last year as people with ALS swarmed over social media and support group meetings to recruit peers in a promising Neuraltus clinical trial, a day saved recruiting patients is as valuable as a day waiting for data analysis or an approval later.

It would be interesting if subjects (and others) had three simple time lines for every clinical trial --

1. An optimistic version if every step were to go swimmingly well
2. An expected version
3. A pessimistic version featuring some worst-case timing

Sometimes we have visions of data sitting somewhere waiting for a meeting to happen on a certain date.  Sometimes we have visions of stacks of papers growing yellow with age at the FDA.

If we had a much clearer picture of what can be expected and who does what and what patients can affect themselves, would it not make for a better process for everyone?

It's Nuts

Imagine going to a stock broker who gives you two kinds of reports --

1. An occasional list of the things you bought (without prices)
2. An occasional report on one of your investments that did well

That's it.  You don't get dollar amounts on your individual investments.  You don't get a list of the investments that went bust.  You don't get information in a format that makes it easy to track individual investments over time.

Nobody would use that broker, right?  You would have to be nuts.

Much of ALS research is funded by donors and taxpayers.  We are the investors in a portfolio, yet we have information scattered here and there without a good, businesslike trail of projects, grantees, start dates, end dates, and outcomes.  We get occasional feel-good lists of what we bought.  We get occasional feel-good reports on research projects that did well. That's it.

As investors, it's time that we got better information about our portfolio.  It's time for organizations and our government to supply us data in a format that permits us to see the who, what, where, when, how, how much, and outcome.  We need information in a format that lets us look back before we invest again.

It's nuts to be such blind investors when surely grantmaking organizations hold the data that the investors deserve to see.  It's just nuts.

Holding Breath Is Not An Option

The following was submitted by a reader of www.alsadvocacy.com:

_____________________________________________________

Of course when I was a newbie ALS inductee, when it really didn't affect our lives that much, I was sure there would be a treatment soon, when our family became more affected by the disease, I was still sure there was going to be a treatment. Now that we face life and death decisions, I am sure there is NOT going to be a treatment, in time, for our family.

We've been fighting this disease for years, doing everything on every front possible that a family can do to help bring treatment to fruition. We've walked, we've ridden, and we've hounded out friends for years to help support ALS research. I know exciting research news gives people with ALS hope, and yes, can even brings in donations.

But now, when I hear news about THIS ALS research and THAT ALS research, I want to stifle back a scream or an eye roll, or at times even a laugh...most of this research write up news always ends with, "this discovery will lead to new ways to study ALS."

To me, I have learned over the years that these research news blurbs means is, "yeah, we think we found something, but you know, IF it gets from the research lab to a clinical trial, it's still YEARS away to a treatment." Oh yes, yes, I KNOW ALS is a tough disease to research, there are many aspects and nuances, and it seems to affect every person differently. I have been told that many times in ways that are compassionate, in ways that are condescending, and yes even completely ignored at times when I tried to question research.

How much more studying do you have to do? Can't you do something instead of studying the disease?

But you know what, I'm guessing research is not as tough as living with it, day in, day out, week in, week out, year in year out. The research is frustrating I'm sure,

ALS is a disgusting, vile disease all the way around; there is nothing easy about any of it. I think that is one thing that both researchers and ALS families can agree on.

But after living with the disease for these years, I'm not impressed. I'm sick and tired of hearing about research breakthroughs, that are years away from producing any kind of treatment. I suppose you could say I'm a bit jaded in my thoughts because like I said, when I was an ALS newbie, I knew there would be a treatment. However, as an old-timer in this stupid disease, I wish they would keep their news to themselves until they can come out and say, "WE HAVE A TREATMENT that works RIGHT NOW for EVERYONE." Until then, I'm not holding my breath...because hey someone has to keep breathing here.

Four Years Ago Who Would Have Known?

During the London 2012 Olympics around 340 people will die daily from ALS.  That's well over 5,000 funerals during the 16 days while we watch Bob Costas tell us of athletes who achieve greatness and others who must look ahead to Rio.

Most of those 5,000 were feeling just fine watching the Beijing games just four years ago.

Wham.  ALS changes everything and it hits quickly.

Looking forward to Rio?

Wouldn't This Be Interesting?

Would clinical trial design be different if a few people with ALS were at the table when the exclusion criteria were written?

Certainly nobody wants to compromise good science, but are today's trial designs stuck in a rut from the past?

They Did It

Yesterday I think we witnessed the most impressive thing ever done to raise funds for ALS research.  

It was a record in many ways.  It was exciting and fun to follow. It was inspiring.

Thank you, www.channelforALS.org .

Go, Go, Go!

The Channel for ALS team is on the home stretch back to the white cliffs of Dover.  Tweets indicate that they are on a world record pace.  This is the most exciting ALS event ever!

http://www.channelforals.org/

You can watch their twitter stream there and get a link to track their escort boat live, or you can get updates and pictures on facebook.

These are amazing women doing a remarkable feat for ALS research.

If you've not done so already, please consider tossing in a few dollars for the cause at http://www.channelforals.org/ . Be a part of something special. I was impressed that they even made a note that none of the donations will go to their travel expenses.  It's for ALS research.  Class.

Let The Data Speak

Let the patients speak.  Better yet, encourage the patients to speak.

Yesterday we heard of a "promising" new treatment that is being tested in a clinical trial for safety on 12 patients.  We heard words like "exciting" and "compelling," but we didn't get any specifics.  It will be years (if ever) before we see the efficacy data to support those words of promise.  The words were music to the ears of those of us who are not scientists, but how much more meaningful would they have been if we could have seen some data voluntarily supplied by people in the trial.

Whether people with ALS are in a formal clinical trial or in a do-it-yourself trial, why not encourage them all to share their data in public place where others can benefit from insights into the data?  I know, it's just not done that way.

Why not?

• In a double-blind, placebo-controlled trial, it's really a purer environment if the patients are clueless.  I get that. But this is ALS, a disease like no other.  Do both the patients in the trial and those on the sidelines not have an ethical right to know more?

• If a couple of patients start to feel better, that could cause a placebo effect of lots of patients feeling better.  I get that, but this is ALS, a disease like no other.  If placebo can save these folks' lives, then we'll be quite happy.

• If a couple of patients get a rash, that could cause everybody to start itching.  I get that, but this is ALS, a disease like no other.  People who put up with the things that ALS throws at them aren't likely to be deterred by side effects if the end goal of life is being met.

• If the trial data aren't living up to the promise, it could scare people (including investors) away.  I get that, but this is ALS, a disease like no other.  It's the ultimate in knowing when to hold 'em and knowing when to fold 'em... in many ways.  

• Lay people don't know how to interpret the data. I get that, but this is ALS, a disease like no other.  There are some very smart people who get ALS. 

• There are all kinds of privacy concerns. I get that, but this is ALS, a disease like no other.  Data can be shared online ( http://www.patientslikeme.com/ comes to mind) under a creative screen name.  Encourage each person with ALS to share data and let each decide whether there are personal privacy concerns.

We struggle with regulatory systems that are slow and very protective.  Perhaps one of the keys to helping people with ALS is already in the hands of the people.  Perhaps all stakeholders should encourage the data to speak by encouraging the patients to share data.

Please let the conversation begin.

Be Sure To Follow @ChannelForALS on Twitter

Watching their tweets is the next best thing to swimming the English Channel with them.  Go for the record, ladies, and thank you!

This Can't Be Rocket Science

Google can alert me when almost anything that interests me happens. Ebay can alert me instantly when somebody bids. Amazon can alert me when a new book is available. Airlines can alert me when it's time to check in. Any Walk to Defeat ALS can alert me the second anybody donates to my team.

Why in the world can't clinicaltrials.gov alert me the moment an ALS clinical trial opens for enrollment? 

Clinicians' and Patients' Perceptions Differ In So Many Ways

Here is a very interesting article on the perception gap related to ALS treatments.

The article cites patients' perceptions and included data shared by patients at www.patientslikeme.com.

Conclusions were that both perspectives need to be taken into consideration.  For this we needed a study?

It shouldn't be a news flash that there are many perception differences between patients and physicians; however, we seldom talk about them frankly.

Decades ago in the pre-mall age when I was in junior high, my best friend and I would take the bus downtown for shopping or lunch or a movie or to buy records (remember those?).  She was the daughter of a prominent surgeon.  She was also diabetic and had to have regular blood tests.  On one of our shopping jaunts, she had to stop at the big medical building downtown where most of the docs in our city had offices.

I had spent so many hours in that building's waiting rooms as a child that I can still remember its smell and terrazzo floors.  The eye doctor always had Readers Digests that fell apart from wear or  Highlights that had been spoiled with someone else's crayons.  The orthopedic surgeon had the constant sound-effects of typewriters.  One typist would click-clack so fast, only to have long pauses for the eraser. That would make some of the waiting patients smile.  The internist had little to read but that wasn't an omen of quick service. He managed to have a ritual waiting period even if you were the first patient in the morning.  Time went quickly because there was often a booster shot waiting.

Shortly before lunch time that day, I followed my friend into the endocrinologist's office where she was to get her blood test.  There were people in the chairs in the wood-paneled waiting room.  Ugh. I had visions of a terribly late lunch.  She checked in and was welcomed like a long-lost friend.  She went straight through the magic door that requires the dreaded waiting-room initiation for mere mortals.  She was out in five minutes and there wasn't that added step where you had to hand a check to the bookkeeper.  It was a very pleasant and efficient experience.

That was my first clue that physicians and their families do not experience the same healthcare delivery system that the rest of us do.  They have access.  They receive professional courtesies.  They certainly have earned those things.  In any profession, there is a peer respect that oils some wheels.

Do our physicians admit that they've not really experienced the healthcare delivery that their patients have?  Do they try to understand the differences?

Fortunately with the perception gaps related to treatments described in the article, physicians and allied professionals have ways to look online and try to understand those gaps... but do they?

Walking a mile in the other person's shoes is always good.  Are any neurologists up for spending a day in the other person's wheelchair during a grueling morning at the ALS clinic?  Of if you want to stick with walking in another's shoes, the caregiver gig at the doctor's office isn't a walk in the park.

How Far Would You Go?

These women are amazing.

http://vimeo.com/palindromevideo/life-cycles

Dear Seth MacFarlane, You Give Us Way Too Much Credit

ABCnews.com carries a response from Seth MacFarlane over the brouhaha around his movie Ted and ALS --

"I lost my mother to cancer, yet there is a joke in the film which contains the word cancer," he said. "I urge analysis of context, lest the 'outrage industry' get the better of us."

You give us too much credit when you suggest that we're part of some kind of "outrage industry."  The fight against ALS has not exactly been a well-oiled machine.  It has been fragmented for many years with numerous organizations following good intentions rather than common goals.  There has never been one voice.  It would make a poster-study in having none of the earmarks of an industry.

You did give us some industrial-strength glue last weekend when your fictional John uttered the words, "From one man to another, I hope you get Lou Gehrig's Disease," to your fictional villain Rex.  Perhaps you should have picked a fictional disease for your fictional pox.

You see, we are simply angry and frustrated with you.  That is our common voice.  That is our glue. The social media that make movie lines legendary also make it possible for a grass-roots bunch of people affected by ALS to speak out.

We have lived through a week when your movie line has become a tagline for your fans on twitter.  I suspect that few of them know who Lou Gehrig was, let alone what is implied when his disease is wished on someone.  Thousands of times the line has been robotically and mindlessly repeated on twitter.  That is troubling.

I have a feeling that you didn't write what you know when you crafted that line.  If you had ever met anyone with ALS, I am certain that you would never have written it.

And your response reinforces the fact that you don't understand ALS or the people who are dying from it or the people who are left behind.  Please stop arguing like an NBA player who never committed a foul.  Please talk to some people with ALS and try to understand what's different about these people.  You will be a better writer for it.

As a writer, you choose words carefully.  Why did you choose "Lou Gehrig's Disease?"  There must have been a reason.

Wahlberg Says, "Take It Up With Seth."

http://947freshfm.cbslocal.com/2012/07/05/podcast-mark-wahlberg-on-als-take-it-up-with-seth/

Dude?

And Herein Lies The Problem, Seth MacFarlane and Mark Wahlberg

A tweet just came through from some folks at USA Today @kimpainter @lifelinelive that points out the reason why ALS advocates are outraged with your ALS reference in the new movie Ted .

Here is the problem --
https://twitter.com/#!/search/realtime/From%20one%20man%20to%20another,%20I%20hope%20you%20get%20Lou%20Gehrig's%20disease

Go ahead and click on that link.  Sure looks like a line from the movie caught on.

Perhaps it's time for you to meet some people with ALS so that you can understand why seeing a list of flippant tweets like that is so wrong.

Thanks For Your Leadership, ALS Therapy Alliance

For the last 48 hours there has been internet buzz regarding Seth MacFarlane's use of ALS as a point of attempted humor in his new movie, "Ted."

Finally an ALS organization has stepped up with a statement.

http://www.marketwatch.com/story/seth-macfarlanes-movie-ted-causes-punch-line-about-lou-gehrigs-disease-to-become-pop-culture-chatter-on-twitter-facebook-2012-07-03

Now if only a few others would show that they have the backs of those with ALS by speaking up in the media.

Organizational backlash works.  This is an especially important week for the world to understand that there is still no known cause, there is no cure, and ALS isn't a disease to joke around about.

We All Know Poor Signage When We See It

And when the signs aren't clear, do we pay attention to any of them?

Please take a moment to read this report which was presented by a gentleman with ALS to some of the leaders of the U.S. National ALS Registry this week.  It contains important data and perspectives related to the awareness of the ALS Registry.

Every person with ALS needs to know the "what, when, why" of the National ALS Registry.  There are three important action items for each person diagnosed with ALS --

1. Self-enroll at http://www.cdc.gov/als
2. Complete the six additional Risk-factor Surveys
3. Complete the Ouality of Life updates at regular intervals

That's it. 1,2,3.  Repeat 2,3 as needed.

The message has been lost in the muddle of stuff that families hear in the first weeks after diagnosis.  Or has the message been delivered at all?

Taxpayers have spent millions and millions and millions of dollars on this project.  It's an important improvement over the decades when clues from people with ALS were buried with them.  The least we can do is get the message to people diagnosed with ALS clearly so that their data will be retained.

It's a matter of getting the right message to the right people at the right time.

And you don't fix poor signage by buying more signs to add to the message clutter.

What Comes To Mind When You Hear "Duke Blue Devils?"

Coach K?
Nah. As great as Coack K is, the definitive response is Dr. B.



This is well worth sitting back and listening for a few minutes.

Thank You, American Academy of Neurology

It was a wonderful surprise to see a tweet come through from @AANmember to urge neurologists to encourage their patients with ALS to self-enroll in the National ALS Registry.  Here's a new article on the AAN website --
http://www.aan.com/news/?event=read&article_id=10607

Immediately after diagnosis, the neurologist has a big impact on the actions that a person with ALS and the family take (or don't take).  Many patients have reported not hearing anything about the National ALS Registry from their physicians, therefore, they had been hesitant to self-enroll.  As of today, we see that changing.

Thank you, AAN!