"ALS Awareness Month" in the US Is Officially Declared A Failure

We now have the most objectionable fundraising premium in the history of fundraising.

We understand that the people who run a factory in China that makes little dolls whose heads wobble when you tweak them have probably never seen the neck weakness associated with ALS.

We realize that baseball team gift shop managers who like to sell souvenirs have never had to shop for a neck brace that a person with ALS needs to prevent a heavy head from flopping painfully.

And we know that collectors on eBay have no idea of practical difficulties (from breathing to vision) of neck muscles that simply can't support a head.

These people don't know what happens to people like Lou Gehrig after they say their farewells and slip privately into a difficult journey with ALS.

But people who accept paychecks from an organization whose mission begins with "Leading..." and contains the word "compassionate" should be able to grasp that a little statue of a man with ALS whose head wobbles uncontrollably is simply offensive and disrespectful.

And they should educate.

We need an advocacy organization that stands up for the respect and civil rights of people with ALS.

It's not that hard.  You don't mess with PALS or disrespect them.  Period.

More Qs For The Right-To-Try Discussions

The op-ed in USAToday leads us to believe that Neuralstem is full-speed-ahead with a Colorado Right-To-Try stem-cell program for people with ALS.

http://www.usatoday.com/story/opinion/2014/05/28/drugs-fda-approval-right-try-legislation-column/9692735/

The discussion is making us all think.

There are some thoughts and questions that I would like to add to the discourse --

• Is anyone willing to commit to conscientious and open data collection for any people with ALS who enter a Neuralstem RTT program?  Colorado law does not have any data requirements that I am aware of.  We need to raise that bar voluntarily lest these programs be no better than the stem cell programs in Mexico and China that thrive on anecdotes rather than trustworthy data on all patients.
• It would be very enlightening for us to see a specific head-on comparison of what Neuralstem and PALS would have to do in an FDA Compassionate-Use program versus a Colorado Right-to-Try program.  Also interesting is the question of whether any people with ALS had applied for Neuralstem compassionate use prior to this Right-to-Try push.
• The USAToday op-ed again cites the truly miraculous outcome of Ted Harada who participated in the Neuralstem clinical trial.  We know that ALS is a spectrum of diseases.  Is anyone willing and able to provide a profile of Ted's ALS, e.g. known genes, onset details, pre-trial trajectory, etc.?  I would think that people with ALS that most closely resembles Ted's ALS would have the best roll of the dice in a Neuralstem Right-To-Try program. 

Thanks to all who are helping the conversation continue. I hope that it helps us figure out ways to move ahead more quickly and still advance the science for all with ALS.

Is "Right To Try" Right Right Now?

This is an extremely important discussion that affects everyone with ALS.

There are some new state laws intended to give people with untreatable, terminal conditions with a right to try experimental treatments.  The "right to try" name is awesome.  The concept makes sense to people who have a disease worse than any side-effect of any experiment.  The intentions are wonderful.

But I keep remembering my grandmother's words that the road to hell is paved with good intentions.  And if we provide experimental treatments to people with ALS, can we also advance science from their experiences?

I hope that the discussion will continue and grow in depth.  Already we have seen polarizing arguments based on emotion and lung power rather than on thoughtful consideration.

We clearly have a status quo that does not work for people with ALS.  We have failed.  We need to do better.

Here is the text of the legislation that recently passed in Arizona --
http://legiscan.com/AZ/text/HCR2005/2014

It's short and sweet.

“Right to Try” is a great name, but the devil is in the details.  Some of the details concern me a lot are --

• What constitutes an “investigational drug?”
• People with ALS are very vulnerable to hucksters, and unfortunately, some of those hucksters are even physicians.  Would this legislation permit anyone with a physician’s license (even a plastic surgeon or dermatologist) to make RTT therapy recommendations for a terminal patient with a complex neurological condition like ALS?
• Do I understand correctly that the Colorado legislation allows the drug developer to charge patients for the investigational product? How do we prevent a system that excludes dying people who happen to be of limited means.  Will it be “Right to Try” or “Right to Buy?”
• Attracting people to clinical trials is constant challenge in the fight against ALS.  How in the world would you ever get clinical trial volunteers (who have to agree to the coin-flip of a placebo)  if they had slam-dunk access to the real drug in a Right-to-Try program?
• There are Expanded Access Programs that provide investigational drugs to people with ALS, and data are not tracked or reported.  We lose valuable clues both to ALS and to these experimental treatments when programs permit data to be lost.  My impression is that there is no requirement or incentive for drug developers to retain or report data on RTT participants.  If that is correct, it seems to me that we lose yet another opportunity to advance the science.  Patients not only have a right to try, they also have a right to have their data used constructively to help the next person.

Let the conversations continue. Please!  And let's come up with something that has both short-term and long-term benefits.

Time To Put Away The Theatrical Fog Machine

Yesterday an alert advocate for people with ALS sent me yet another one of those things that we stumble upon online.

May 7 was the day when we sat in a large meeting room in the JW Marriott for ALSA's advocacy briefing before hundreds of us were dispatched to Capitol Hill to ask for funds for the CDC's ALS Registry.

On that very day the CDC committed almost a million more dollars to the coffers of ALSA and the MDA.  When I saw the screen shot, I could not believe the date.

Was that not worth mentioning to us?  Was that not pertinent?  Why was that not included in the explanation of the project funding and status?  Did they forget about it?  The timing is interesting.

Instead of transparency on this project it seems like we get a continuous stream from a theatrical fog machine.  We were sitting there on that very day, for Pete's sake.  Those are big contracts.  That's a lot of revenue for those charities.  Not a peep.  

Time to stop the fog and speak to advocates and taxpayers clearly.  Transparency is a serious obligation.  Fog is a bad sign.

Which Is It -- A Clueless Politician Or An Insensitive Clod?

From the Wall Street Journal Washington Wire blog --

So a politician thought it would be fun to fulfill some kind of baseball fantasy using the words of a great man dying from a disease that cut him off in his prime and for which there was no treatment.  Mr. Wolf obviously missed the beauty of the speech and the courage it embodied.

We see organizations celebrating the 75th anniversary of Lou Gehrig's farewell this year.  We also need to educate America that the outlook for people with ALS today is the same that Lou Gehrig faced in 1939.  ALS was and still is an outrageous, cruel disease.

If we ever heard the words, "How dare you?" from my mother, it was over a very serious breach of good behavior and judgement.  Mr. Wolf, how dare you?

Thanks For Asking

Last week I spent two days wearing Regina Holliday's Walking Gallery Jacket #300 in our nation's capital.  I walked on busy sidewalks, spent a half day at a conference of ALS advocates inside an upscale hotel, and called on legislators for a day on Capitol Hill with a curious painting covering the back of my yellow wool jacket.

The people who went out of their way to ask me about the jacket were interesting -- the staff at my less upscale hotel, many strangers on the Metro, people waiting to cross streets, security guards at a House office building, workers at the snack bar in the basement of a Senate office building, a lady who saw me in line at Starbuck's, several legislators who really wanted to learn about the backstory, and on and on.

Perhaps the people who didn't ask reflect something important about the fight against ALS.

The Walking Gallery has an everyman quality.  It draws interest from real people relating to other people.

How do we move clinicians and other healthcare professionals and scientists and organizational executives to have that same personal curiosity that everyman displayed last week?

The Walking Gallery makes some powerful statements in many ways.

Today There Is An Action Item for You and for You, Too

Your words and thoughts matter.  They are needed before Sunday.  Please act.  Yes, you.


We saw Medicare rules go into effect on April 1 regarding Speech Generating Devices (SGDs) that just have stupid written all over them.  They do not make sense in the 21st Century.

We have already written about how a well-intentioned move of the devices to rental status has all kinds of unintended consequences for people with ALS.  As our grandmothers taught us, the road to Hell is paved with good intentions.

You perhaps think my use of the word "stupid" is a little much?  I think not.

• The rental status means than if a person with ALS is transferred to a hospital or hospice, that rental device will not go with the person.  We are moving people into situations where communication is more important than ever, and we take away their right to communicate.  That'll keep them quiet.

• Devices are locked from internet access.  The bureaucrats say that's an old rule that they should have been enforcing all along.  Rental just makes enforcement all the easier.  Peachy.  Let's unhook PALS from the best communication network known to mankind.  That'll keep those patients quiet.

• But wait, there's more!  We hear that on September 1, a government bean-counter somewhere will celebrate.  There are indications that devices that include word processing, email, or remote control will not be covered at all.  That will really keep those patients quiet.

• There have been recent denials of eye-gaze devices.  Evidently some Medicare reviewers say that this is not needed and people use them to play games and surf the internet. Is your blood boiling yet?

Please consider a quick action item if you have ALS, know someone with ALS, or simply care about government getting in the way of common sense.

Please write a quick message telling the importance of Speech Generating Devices to those with ALS. Your letters will be delivered to the Medicare powers that be on Monday by executives from Tobii, the company that has developed much of this enabling technology. The following is from Tobii:

 Here are the kinds of things you should write about. Remember this is Medicare. Use terms like "medically necessary."


How has a communication device changed your life?
What impact has eye tracking had?  How would you function without eye-tracking?
Has eye tracking been instrumental to have them work longer? Stop abuse? Communicate an emergency? Able to communicate with their children? (any other examples?)
What should Capitol Hill be aware of if patients going forward will be denied eye gaze across the board ? What if all claims would have to be appealed on all eye tracking? (CMS appeals right now at three year wait, yes that is not a typo)
How would losing the ability to use skype , email, text, phone , teleconference with their doctors, and internet impact you?
Have you been denied access to a communication device with eye tracking what has been the trail or timeline for process and if possible how have your symptoms have changed.

Please sign your name, town and state and if willing, connection to pALS, age and disability. 
These are just quick thoughts/ideas. You need to think like the government. Why should they care? We need the patients & families to remind them who they were before the disease, who they have been able to be after and why communication is so so critical. 
We are asking for the letters by Sunday and from people throughout the country. They will be hand delivered to CMS. Please email your letters to: stephan.floyd@tobiiati.com

Please submit something.  It doesn't have to be elegant.  Direct is always better. Please act.  It's up to you and you and you and me.  Let's send them to Washington with a flood of messages, many of which will not be possible to write after September 1.

Thanks, all.  Please help spread the word.  Friends and their friends can speak to how important it is to be able to interact with those with ALS.  Any American taxpayer should speak up. Yes, you.

Today Is Brought To You By The Letter "Q"

Click here for the ALSA legislative priorities.

We hope that the following questions will be addressed before we are asked to advocate for more funds on Thursday:

·         How have the Registry funds been spent in the past few years and what are the plans for the $10 million being requested?  Approximately how much is attributable to the cost of data files, basic data file mining, website, risk-factor surveys, patient support, analysis and reporting, travel and meetings, clinical trial matching, promotional materials, special studies, local studies, biorepository feasibility, etc. ?  

 ·         Who have been the government contractors on the project and what are (or were) their roles and deliverables?

 ·         Has anyone re-measured the Rob Tison metrics?  If so, what were the results?

 ·         Do you know what percent of the identified cases of ALS were found in data files only, in both data files and self-enrollment, and in self-enrollment only?

 ·         Do you know how many newly diagnosed cases of ALS enter the ALSA clinic system annually?  Do you know how many of those have self-enrolled in the Registry?

 ·         How many clinical trial enrollees has the Registry notification produced?

 ·         How are the participants for the Registry annual meeting, for the DOD ALSRP Integration Panel, and for the DOD ALSRP Peer Review group chosen?

Today is brought to you by the letter "Q."

We hope that tomorrow will be presented by the letter "A."

It's Time For Some Three-Point Shots

For over a year I've been scratching my head about the MODDERN Cures Act.

http://als-advocacy.blogspot.com/2013/04/these-are-my-questions-du-jour-on.html

Recently an outstanding advocate/tweeter/patient/person @SDFatPhRMA put me in touch with the folks at the Campaign for Modern Medicines modernmedicines.com who have gone above and beyond the call of duty to help me understand MODDERN Cures and exactly what's in it for people with ALS.  I am grateful.

As we perceived years ago, the dormant therapy features of MODDERN Cures are the most interesting for the fight against ALS.

Here is a blog from the Campaign for Modern Medicines that answers most our questions --

https://modernmedicines.com/entry.php?id=486

And here is a recap of some salient points for those interested in the fight against ALS --

Are these "dormant therapies" only things that never made it to market for another indication?

That is correct.  Think about things that didn't get through FDA approval for their original intention.  Those things that are sitting on shelves in labs somewhere could have their patent "clocks" reset so that there would be incentives for drug developers to try them on ALS.  MODDERN Cures would not apply to a lithium or a ceftriaxone, but it would apply to something that never made it through FDA approval before. 

How does it work when something is already off patent?  

There are two things that are really important in this case -- patent protection and data protection. 

The MODDERN Cures Act aligns patent protection and data protection at the date of FDA approval. 

Even if there is no patent protection left, the drug receives data protection.  A generic or biosimilar manufacturer cannot generally receive FDA approval to launch a competing product during that protection period.  If a competing manufacturer started clinical development of an off-patent molecule before the first manufacturer received FDA approval, then competing products could also be approved.

Can anybody pick up a potential drug and run with it, or does it have to be the company that owned the rights when it was on patent?

If it still has any patent life, an interested scientist would have to work with the patent holder on a license agreement; otherwise, scientists, please go for it!

OK, what happens when something has just a little patent time left?

The MODDERN Cures Act allows the patent clock to be reset during the development process and run from the date of FDA approval. This is a big incentive.

We know that it's going to take a lot of shots on goal to finally find some therapies for ALS.

We need for drug developers to make smart (and long) shots.  If something is sitting on a shelf with little or no patent protection, we owe it to people with ALS to make it viable for drug developers to pick up one of those oldies and shoot.

Many thanks to the folks at modernmedicines.com for their time and help.  If I got anything wrong, I'm all ears.  

Finally I think we have some clarity on what's in it for people with ALS.

Why?

That's the best title I can come up with for a post I never expected to have to write.

Last year some of us stumbled across some large no-bid government contracts given to ALSA and then to MDA from the CDC ATSDR ALS Registry funding.  The MDA's contract appeared in September and the amount, $2,695,642, floored some of us.

On October 18 I emailed a question directly to the head of the project at ATSDR who is a frequent speaker to groups on the Registry project.

Dr. Horton, Are you able to tell me what MDA is doing for this sum, please?

 https://www.fbo.gov/index?s=opportunity&mode=form&id=c7856e6375ef7f0fcc2c4cb47ae176a0&tab=core&_cview=0  

I have a bad case of sticker shock.  

Thanks very much for any insights.

On October 21, I received a reply from Abby Martin of the CDC.

Neither Mr. Horton or I have the authority to respond to requests for public information not in the public domain.  (This includes any request for information via social media.)  All such requests should be directed to the CDC/ATSDR Freedom of Information Officer for a response 

Okeydokey.  I learned how to submit a Freedom of Information Act request.  The following two requests were submitted and accepted on October 22.

Request 1 I would like to know what the work product, deliverables, and performance standards are for the work in the MDA contract for “ALS Outreach and Education Activities to Support the National ALS Registry.”

Contract Award Number:
200-2013-57122
Contract Award Dollar Amount:
$2,695,642.00

https://www.fbo.gov/index?s=opportunity&mode=form&id=c7856e6375ef7f0fcc2c4cb47ae176a0&tab=core&_cview=0 

Request 2 I would like to know what the work product, deliverables, and performance standards are for the work in the ALS Association contract for “Education and Outreach for the National ALS Registry.”

Dollars Obligated	$319,465
Current Contract Value	$319,465
Identifying Agency ID	7523
Procurement Instrument Number	HHSD200201141609C
Modification Number	6
Transaction Number	0
Fiscal Year	2013
IDV Agency ID
IDV Procurement Instrument ID
IDV Modification Number	0
Solicitation ID	2011N13315

 http://www.usaspending.gov/explore?fiscal_year=all&comingfrom=searchresults&piid=HHSD200201141609C&modification=6&typeofview=complete 

There is a nice online status tool that they provide to track the status of your request.  The status of both requests went from and initial  "Pending Program Search" to "Pending Final Review" within a few weeks.  Great!  I honestly expected to have answers by the end of the year.  Silly me.

By January 23, I felt that surely something had gotten lost somewhere in the "Final Review."  I emailed the CDC FOIA officer who had helped me with the requests.

The online status for both of these has been “pending final review” for over two months. Is there a way to check to make sure that something didn’t get lost in the process? Thanks very much for your help.  

I immediately got a reply that floored me again.

This is in response to the email below concerning your Centers for Disease Control and Prevention and Agency for Toxic Substances and Disease Registry (CDC/ATSDR) Freedom of Information Act (FOIA) requests #14-00039 and 14-00043.
 As we advised in our acknowledgement letters dated October 22, 2013, CDC processes all FOIA requests on a first-in, first-out basis, which is the court-approved method for agencies operating under a backlog. Program staff have completed their search for the records you requested, and your case is currently in this office awaiting final review. A thorough document review will be conducted by the CDC FOIA staff, and all releasable records will be provided. Processing time is contingent upon the number of requests ahead of yours and their complexity and volume. Therefore, we are unable to give you an exact timeframe for completion of your request but estimate that it may take up to 36 months for your request to rise to the top of the complex queue based on current workloads. Please be assured, however, that a response will be sent to you as quickly as possible.
 You may continue to check on the status of your case by going to our FOIA webpage at www2a.cdc.gov/od/foiastatus and entering your request number. The fiscal year is the first two numbers and the request ID is the second set of numbers.

Hmmmmm.  Why is this so hard?  Thirty-six months?  Does someone not want to talk about this?

My response, still on January 23, was --

I appreciate the insights.With matters ALS, the clock ticks quickly.  One of the advocates who was interested in the information has died already.I’ll keep watching the status and hope that it finds the top soon.Thanks.  Have a good afternoon.

The status is still "Pending Final Review."  That must be some fancy review.

This started as a very simple question.  The answer should be pretty simple.  It just gets curiouser and curiouser when people make everything difficult.  This is information that should be very transparent, especially since the contracts required no bidding and since we advocates go to our legislators to ask for the project's funding every year.

It should be simple, but it's not.  Such is the way with matters ALS.

Why?

Help! Someone, Please Help!

Eight years of advocating for a national ALS Registry.

I felt and still feel strongly that we need a census of cases of ALS.

$40 million taxpayer dollars spent.

This is the spring when we finally expect to see some numbers.

And so far we see a flock of poster sessions from this week's AAN meeting.  Here are a couple.

I feel like I've been spun around a few times and the blindfold has been partially removed.  Now I certainly need some help with some questions.  My comments and questions are in hot pink below.  These are things that we need to understand.  I think we've earned that right.


http://www.neurology.org/content/82/10_Supplement/P5.077.short?sid=85093e2b-8d66-4dd4-9722-dfa4458c5825

Using the National Amyotrophic Lateral Sclerosis Registry as a Subject Recruitment Tool for Research (P5.077)

1. D. Kevin Horton₁,₂, 
2. Vinicius Antao₁,₂ and 
3. Paul Mehta₁,₂

+SHOW AFFILIATIONS

1. Neurology April 8, 2014 vol. 82 no. 10 Supplement P5.077

ABSTRACT

Objective: To describe how the National ALS Registry links persons with ALS (PALS) to scientists who are conducting ALS research.Background: Subject recruitment for research is critical for understanding inherently fatal diseases like ALS, however, linking patients with researchers is not so straight-forward. Researchers often face challenges in timely recruitment, obtaining sufficient sample sizes, and/or in enlisting patients that meet certain eligibility requirements. Similarly, patients can face difficulties in finding specific research studies for which they are eligible.Design/Methods: The federal Agency for Toxic Substances and Disease Registry’s (ATSDR) National ALSRegistry now links Registry-enrolled PALS with external scientists who are conducting ALS research. TheRegistry’s new online Research Notification Mechanism allows ALS researchers to upload an application that briefly describes their research study and objectives, while PALS can elect to be notified about these opportunities.If the application is approved, ATSDR then queries the Registry for PALS meeting the study’s specific eligibility criteria (e.g., age, sex), Does the registry have onset date? and then distributes the researcher’s study material and contact information to PALS via email. PALS have to directly contact the researcher to take part in any research.Results: Since the Research Notification Mechanism’s May 2012 deployment, approximately 96% of Registry enrollees have elected to be notified about ALSresearch opportunities. Additionally, ATSDR has approved a number Do we know what the number is? of institutions to use the Registry for research recruitment, sending out thousands Do we know how many thousands? of e-mail notifications to PALS Do we know how many PALS have been targeted? on behalf of the recruiting And do we know what percentage of the open recruiting clinical trial seats were represented? institutions. And do we know the most important metric of all, how many clinical trial seats have been filled because of this mechanism? Conclusions: The National ALS Registry’s Research Notification Mechanism is an effective ??? tool for linking PALS with ALS researchers who are conducting epidemiologic studies or clinical trials. The Mechanism benefits PALS by conveniently delivering timely and tailored research opportunities via email. It also benefits researchers by helping to speed-up the recruitment process, increasing the study sample size, and efficiently identifying PALS meeting specific eligibility requirements.  Do we know it actually sped or increased sample size in any studies?

Disclosure: Dr. Horton has nothing to disclose. Dr. Antao has nothing to disclose. Dr. Mehta has nothing to disclose.

Wednesday, April 30 2014, 3:00 pm-6:30 pm

http://www.neurology.org/content/82/10_Supplement/P2.072.short?sid=e107f57f-3dcb-4ff4-a777-7245f9f289f7

Amyotrophic Lateral Sclerosis (ALS) Estimates from National Databases in the United States - 2001-2010 (P2.072)

1. Marchelle Sanchez₁,₂, 
2. Paul Mehta₁,₂, 
3. Wendy Kaye₃, 
4. Vinicius Antao₁,₂ and 
5. D. Kevin Horton₁,₂

+SHOW AFFILIATIONS

1. Neurology April 8, 2014 vol. 82 no. 10 Supplement P2.072

ABSTRACT

Objective - To present preliminary ALS estimates from national databases in the United States.Background - The uncertainty about the incidence and prevalence of amyotrophic lateral sclerosis (ALS) in the United States, as well as lack of knowledge about the role of environmental exposures in the etiology of ALS, have created a need for data collection through a national ALS registry. In 2008, the Agency for Toxic Substances and Disease Registry (ATSDR) conducted four pilot projects to determine the feasibility of creating a national registry. It showed that approximately 80% of ALS patients can be found through national databases. The first approach utilizes existing national administrative databases to identify prevalent cases. The second approach uses a secure web portal to identify cases not included in the national databases. This latter approach allows patients to self-identify and enroll in the ALSregistry and take risk factor surveys. Design/Methods - We sought to identify U.S. residents with ALS in Medicare, Medicaid, Veterans Health Administration (VHA), and Veterans Benefits Administration (VBA) databases for the years 2001-2010. Records were searched and identified in these databases for ALS and Motor Neuron Disease (MND) codes.Results - A total of 147,889 individuals, with any MND code Are those distinct individuals? So over a period of nine years, 147,889 distinct MND individuals were identified?, were identified across the four national databases. Medicare contributed the largest number of individuals. The total number of individuals identified having ALS was 36,547, which is 24.7% of the total number of individuals identified with any MND. I always thought that ALS was by far the largest subset of Motor Neuroon Diseases.  Am I wrong?  Do they really think that over 75% of the MND cases are not ALS... or did they just fail to identify a lot of MND cases as ALS?  Conclusions - This is the first effort to identify ALS cases for a national registry. The number of individuals identified via the national administrative databases is not a prevalence estimate Why not?; however, it does indicate that a large portion Based on what assumption? of ALS individuals can be identified for the National ALS Registry by using the national databases selected. How do you arrive at that conclusion?  It sure seems like a jump to me!  

Disclosure: Dr. Sanchez has nothing to disclose. Dr. Mehta has nothing to disclose. Dr. Kayehas received personal compensation for activities with McKing Consulting Corporation as an employee. Dr. Antao has nothing to disclose. Dr. Horton has nothing to disclose.

Tuesday, April 29 2014, 7:30 am-11:00 am