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.

Sunday, November 30, 2014

What Should I Talk About?

That's every speaker's quandry.

There is a wonderful old story about the young, new preacher who arrives in a small town in Indiana. He is replacing a long-time fixture and is worried about his first sermon. He goes around to the barber and the banker and the grocer and asks, "What should I talk about?" He gets much guidance. Then he goes to the little old lady who is known to be the most opinionated person in town. "What should I talk about?" She gives him the wisest advice of all, "You should talk about God, and you should talk about five minutes."

So often at ALS MND meetings speakers prepare all kinds of intricate material when there are hundreds of questions that those with ALS and their caregivers would like to ask and have discussed.  There is never enough time for Q and A at the end of presentations, and many questions go unanswered.  Maybe they should just talk about ALS and talk about five minutes and let the questions lead the way.

This week prior to the big ALS MND Symposium in Brussels, there will be a two-hour "Ask the Experts" session for patients and caregivers to ask questions.  We asked, and they are accepting submitted questions via .  Some patients and caregivers who participate in the research forum at asked me to submit the following list of varied questions:

  1. Do you have any ideas why the developers of Generveron are not participating in the Symposium? As a most exciting announcement this year of halting or slowing progression in 7 of 8 PALS in a trial, it seems incredible that the subject does not appear to be anywhere on the agenda.
  2. The idea of a GUID for de-identified patient-level data in ALS data repositories seems like the ultimate common sense to me, especially with the proliferation of new, similar iPS projects. Please share your thoughts on what it will take to implement a Global Unique Identifier in ALS project databases.
  3. What can we learn about speeding up drug development from the Ebola crisis? Bill Gates recently made some comments about needing better global disease surveillance. He and others have suggested that there are ways to gather powerful clinical evidence without placebo arms. It seems to bother people a lot to suggest a placebo for Ebola patients but yet placebo is just fine for someone with ALS. Your thoughts, please?
  4. Has it become customary practice in clinical trials to sequence the DNA of the participants? If so, whole genome or just for specific known and suspected genetic markers? And if so, what is the customary practice regarding providing participants with those results? And what is the customary practice with respect to releasing the data to ALS genetic databases?
  5. Regarding the Cytokinetics… It is frustrating to me when a trial fails and then gets redesigned for a costly and time-consuming do-over. Have any other trials used SVC as a primary endpoint? Are you allowed to go into a trial with a premise that a drug could positively A or B (alternative primary endpoints)?
  6. If standard-of-care were not an issue, should every clinical trial have a branch without riluzole? Is it possible or likely that riluzole adversely effects efficacy of other treatments?
  7. If you had ALS, what would you be trying?
  8. For years ALSA has cited multiple studies of a much higher incidence of ALS among those who have served in the US military.  Have there been such studies of those who have served in other countries’ military?
  9. What ideas do you have for the next year’s equivalent of this year’s IBC?
  10. If an MND patient is getting a balanced diet, are vitamin and mineral supplements a waste of time and money?
  11. How important are large-animal MND studies. Have any large animals with ALS-related conditions ever been successfully treated?
  12. How important will bioinformatics become? Will Terry Speed be helping with MND-related research in the near future?
  13. Why is it so difficult for any research lab to win the $1 million Prize4Life prize for extending the lives of SOD1mice by 25%
  14. A $1 million Prize4Life prize was awarded for a Biomarker Tool that does not seem to be very useful. Do you have any updates on this tool or other biomarkers in the pipeline?
  15. Do you think that there’s any future in exoskeletons (and 3D printers to make them cheaply) for MND patients?
  16. In the panel of experts suddenly developed MND, what would be the one main question that they would ask? And how would the others respond?

We look forward to the discussion of the answers.

Saturday, November 22, 2014

We Need An Air-Quotes Dictionary For The Fight Against ALS

From the day a neurologist made air-quotes with his hands when he used the word promising to
describe experimental clinical trials for ALS, I knew we were in trouble.

Over many years we hear words over and over used to generate promise and hope and donations.  The words have become devalued.  They don't mean diddle without specifics.  They all need air-quotes.

We have to press for what they really mean.  Words are cheap.  We need substance.

Here are some that set off bells after you've been around the fight against ALS for a few years --
• Breakthrough
• Collaboration
• Consortium
• Exciting
• Game-changing
• Groundbreaking
• Hopeful
• Leading
• Partnership
• Promising
• Transformative

They make us all feel good. We need to press for details from those who use them.

As an ALS advocate once said, "If something is called 'exciting' in a press release, it probably isn't."

Or maybe there's a drinking game here...

Thursday, November 20, 2014

Please Speak Up So That People With ALS Are Not Silenced

Medicare CMS has a public comments period that ends on December 6.  It involves their rules on so-called Speech Generating Devices and related technology.  We must submit comments on behalf of all those who can't.  We must make it clear that technology that enables people with ALS to communicate in any manner and, therefore, participate in their healthcare and lives is not a luxury.  It is a necessity.

Here is the issue we need to address as stated by CMS:

Effective January 1, 2001, speech generating devices are considered to fall within the durable medical equipment (DME) benefit category defined in section 1861(n) of the Social Security Act (the Act).   Speech generating devices are covered if the patient suffers from severe speech impairment and the medical condition warrants the use of a device. Speech generating devices are defined for Medicare coverage purposes to include dedicated speech devices used solely by the individual who has severe speech impairment.   Speech generating devices also include software that allows a laptop computer, desktop computer or personal digital assistant (PDA) to function as a speech generating device.  Devices that are not considered speech generating devices include devices that are not dedicated speech devices, or devices that are capable of running software for purposes other than for speech generation.  The national coverage determination (NCD) is available in section 50.1 of the Medicare NCD Manual (Pub. No. 100-3, Ch. 1, Part 1).
Since 2001, the technology of devices that generate speech and the ways in which the devices are used by patients to meet their medical needs has changed significantly.  For example, patients now use speech devices to generate text and email messages for the purpose of communicating with their caregivers and physicians.  Therefore, in light of the changes in technology and use of devices that generate speech, we are internally generating a reconsideration of this NCD to address coverage of devices that generate speech as well as other forms of communication.

Here are the comments that I just submitted.

When Mom was dealing with ALS in 1996, there was a huge lesson in our lives -- we take so much for granted. 
This morning I sat down in a comfortable recliner and am typing on a laptop to participate as a citizen in communicating with my government.  I can reach to the left and turn on a light switch.  I can get up and turn up the thermostat when the room gets cold.  If I need someone else in the house, I can yell (or better yet, text).  If, God forbid, I have an emergency, I can dial 911 and get some help. 
It was not so easy for Mom.  We worried about her needing to call 911 in an emergency and not being understood or being dismissed as a drunk. There was some technology that helped.  She had a little Franklin talking dictionary that we programmed with some emergency information.  The Clapper let her triumphantly control the lights... until she could no longer clap.  Communicating with her healthcare providers was difficult to impossible.  
Fortunately technology has leapt forward from the days of talking dictionaries and The Clapper.  There has been a remarkable convergence of technologies that can let people with ALS lead fuller lives more safely and be in charge of their own healthcare decisions.  Thank heavens, they can be enabled to communicate clearly and directly with healthcare providers from home without relying on speech.  Technology today can let them turn on the lights even when they can't clap.  They can control the temperature in the room or beckon a caregiver downstairs doing laundry.  They can tell a power-wheelchair to adjust to avoid the dreaded pressure sores.  They can even advise a commode when it's time to start cleansing.  Yes, we healthy people take so much for granted that technology can now assist.  Consumer devices are getting amazingly smart thanks to two-way communications.  
I feel strongly that CMS needs rules that will let CMS make smart decisions as technologies advance and converge.  We saw Steve Gleason in a Super Bowl commercial demonstrating some empowering software that is pretty mainstream today.  As consumer technology gets better and more affordable, CMS needs to be able to adapt its policies to take advantage of that.  It's good for those with ALS and it's good for the taxpayers.  As our world gets smarter, we need to make smarter policy decisions.  Communications today are so much more than speech. 
Thank you.  We take so much for granted, and technology can help give back some of life's basics that are stolen from those with ALS.

Please add yours.  We must be heard so that people with ALS are not silenced.  Thank you.

Click here for the link to submit your comments.  There are only 51 comments posted as of today.  Please spread the word so that there will be hundreds or thousands!  We must be heard.

Friday, November 14, 2014

Please, Let The Conversations Begin

The abstracts for the big annual ALS MND Symposium have been posted --

There's something for everyone interested in defeating ALS.

I hope that next year they will post those abstracts in a framework that would permit some public
conversations.  Every study is extremely important both to the authors and to all dealing with ALS.  How energizing it would be if patients, caregivers, authors, and others could comment and ask questions on the important "so what" that goes with every one of those research findings!

In the meantime, we have been advised that people can submit their questions to . That's an appreciated resource, but if we learned one thing about ALS and buckets of ice, it's that there are millions of people interested in ALS who are active in our world of social media, and many have invested heavily in these projects.  Please give them ways to comment, ask questions, and share the good news publicly.

Please, let the conversations begin.

Monday, November 10, 2014

Finally, A Pearl

There are a few people who get a case of ALS rage after helping a loved one through the difficult journey.  Nothing is easy with ALS, and it is an outrageous disease.

One such person fights relentlessly on behalf of those with ALS.  It's not her job.  It's her passion.  She is quiet about what she does.  She knows who she is.

She's the one who first pointed out the change in Medicare CMS administration of Speech Generating Devices that was a disaster for those with ALS.  When told by organizations that they had it covered, she kept pressing.  When told by professionals that these things take time, she pushed and prodded because time is the one thing that those with ALS don't have.  When one advocacy organization sloughed her off or offered feckless statements, she pressed others.  When told to stand down, she stood up and fought.

She was an irritant to many.  Too bad.  You can't be afraid to irritate a few on the way to fixing a
problem for those with ALS.

Last week, all of that irritation resulted in a pearl.

As she said to me last week, "It's so rare to have positive news."  Amen.  Finally people with ALS got some positive news with the announcement from Medicare CMS that the Speech Generating Device rules were being fixed.  Were it not for that one advocate who pushed and prodded and irritated and found individuals who could be effective, I'm sure we would still be in the bureaucratic morass.

Here's an article on the announcement.

And you can see that there is more to do.

Special thanks to this remarkable advocate and to Team Gleason and the Center for Medicare Advocacy and the other amazing irritants who joined forces.

It was a pearl of a week.  Finally.

Sunday, November 2, 2014

T Minus 29 Days And Counting

A solution regarding the Medicare CMS Speech Generating Device problem is due by December 1.

The following message is from The Center for Medicare Advocacy.  Note that they are considering action if CMS does not reach an acceptable decision.

Please help them know of people with ALS who have been affected, and please help spread the word.

Thank you.


We Need Your Speech Generating Device (SGD) Stories!

Thank you again for taking action to preserve the voices of those in need of Speech Generating Devices. Because of your advocacy, CMS is currently reviewing its SGD policies. We are hopeful that CMS will revise these policies to allow SGD users to keep their speech when they’re admitted into health care facilities and be able to use their SGDs to communicate with the outside world.
However, in the event CMS does not reach an acceptable decision about SGDs, we are considering action to help people who will lose access to SGD communications.
If you, or someone you know, have been harmed by these policies, please contact us at SGD@MedicareAdvocacy.orgWe are gathering information and data from anyone who wants to be heard, but we are particularly interested in those who can share stories because they have been affected by any of the following:
  • They experienced problems keeping their SGD since capped rental payments went into effect.
  • They experienced problems getting or keeping non-speech communication functions on their SGD in 2014.
  • They experienced problems getting or keeping environmental control functions on their SGD in 2014.
  • CMS denied their request for eye-tracking, or other SGD attachments in 2013 or 2014.
If you or someone you know can answer "yes" to any of the questions above, please contact us at SGD@MedicareAdvocacy.orgInclude the name of the SGD user, email address, phone number, street address, and a summary of the experience.  We will not use any of the information you share without your express permission.

Every story we receive helps us advocate to maintain the voices of our most vulnerable citizens. Thank you for your continued support of all those affected by CMS’s SGD policies.

Kathy Holt
Associate Director/Attorney
Center for Medicare Advocacy

For more information see: