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.

Sunday, April 8, 2018

"Y'all Take Care Of It Yourselves"

"We don't have anything to say right now."


That's the mystifying strategy that a gaggle of large ALS organizations have taken during the most important ALS public policy crisis in decades.

We're in the final week of the comments period on the FDA ALS Draft Guidance.  So far organizations have gone into hiding and engaged in an odd abdication of leadership, telling individuals that it's up to us. They are urging us to submit comments but have not issued any comments themselves.

Keep in mind that this guidance will directly affect clinical trial designs moving forward, and the draft guidance we got does not encourage innovation in trial designs.  If made final, this guidance will in no way speed up drug development or approvals.  

But our largest ALS patient advocacy and research organizations have no official comments.

They turn to us individuals and say, "Y'all take care of it yourselves."

Mystifying.

Disappointing.

Infuriating.

Revealing.













Sunday, March 18, 2018

Guest Blogger: It's Crunch Time. Take Your Shot!

It's crunch time for comments on the FDA ALS Guidance.  Time's almost up  Here are thoughts generously provided by a knowledgeable and relentless ALS advocate.  Please read and take your shot!
________________________________________________________

Hey. Back to the “Considerations Regarding Food & Drug Administration Review and Regulation of Drugs for the Treatment of Amyotrophic Lateral Sclerosis”. We need help. It would be fantastic if we had an advocacy group taking the lead here but alas…. let’s move on. 

When it comes to drug/ therapy development we are failing. We have two approved drugs to “treat” ALS. One “may” extend life 3 months. One “may” slow down progression (while fleecing everyone involved). There is no question – ALS is a difficult disease to target. However, we are failing before we begin. We are currently using archaic protocols in drug development. The measure of success will never be met because we are using a yardstick that does not correspond to the disease. If we are ever going to make a dent in the utter havoc this disease wreaks on everyone it touches, we MUST change the way we approach it. 
We have one month left to make comments. PLEASE comment on the guidance for industry the FDA has proposed. I am including some suggestions below to get your thoughts rolling. Feel free to cut and paste any combination, should you agree with them. I am also including some complete comments. Change them up, use completely different ones. use them exactly. Whatever works for you. PLEASE speak up, share your thoughts, be heard. 
The FDA must encourage innovative clinical trial designs tailored to the population living with the disease and reflective of the heterogeneous nature of its presentation.  

This disease demands urgency. Use clinical trial designs which reflect the same. 
  • Push for the use of trial designs that realistically represent the number of people living with this disease at one time. 
  • Push for mobile travel sites in to allow a larger number of participants.
  • Push for remote data collection to make it easier on families to participate. 
  • Use historical controls from the PROACT data base vs. placebos. 
  • Widen participant eligibility criteria. 
  • Choose an endpoint other than death... a predesignated drop in FVC or specific point loss of ALSFRS. 
  • Consider taking guidance from people living with this disease.

Please do not accept this guidance as is or we will be having the same conversation in another 50 years. 
This guidance does not reflect the advances in technology, science, or data collection we have made this last century. We must encourage the industry to embrace innovate trial design that correlates to the number of people living with the disease at a given time. Use historical data, mobile trial sites and remote data collection. Change eligibility criteria so more people can participate without compromising quality of life interventions that currently exist like the feeding tube which allows people to maintain hydration and minimize the thickening of secretions. We already ask too much of these families - do NOT ask them to give up the few things we can offer. 
This proposed guidance will do nothing to accelerate the development of viable therapies to treat ALS. The status quo is NOT working and this guidance IS the status quo. We need the FDA to encourage and support innovative trial designs that make sense with a rare and terminal disease with rapid progression. Widen the base of participant eligibility, use mobile trial sites and remote data collection to include people who do not live near trial sites. Use historical controls. encourage accelerated approval. We must change the way this disease is approached because what we have done the past 50 years, what we are doing RIGHT NOW, is not working.
The guidance presented is antiquated, out of date, and demonstrates zero use of updated technology and data collection methods. This is a rare and fatal disease with no significant therapy. Trials used in drug development must reflect the number of people living with this disease at one time - the duration of the disease - the wide geographic locale of those diagnosed. There should be mobile trial sites and remote data collection, the use of historical data vs. placebo arms, eligibility criteria reflective of a savagely progressive disease. Please do not allow this blatant disrespect and disservice to the people from whom we already ask too much.

Click HERE to submit your comments.

Friday, March 16, 2018

You Are Smarter Than That

Over a decade ago we fought hard for a good ALS registry for the US -- a sound epidemiological census of people with ALS that would retain people's data and inform the science.

We fought very hard for it. We got it generous funding.  Perhaps we got too much funding for it.

After all these years and $78,000,000, it has simply not delivered.
  • The Registry has found a maximum of 15,927 people with ALS in the US (in its recent report that reflects 2014 data).  That's a number that nobody believes.  The CDC continues to depict ALS as white guys' disease based on its data.
  • This report was published in October on registry completeness,  It's a smoking gun. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5815913/  Whoa.  The National ALS Registry only found 57% of the people with ALS that smaller studies found.  Even more concerning was that the 43% the Registry didn't find were more people of color, more Hispanic, younger.
  • Look at scientific ALS papers that cite ALS data for the US.  They simply don't use this Registry's data.
  • For over a decade the project has permitted scope creep rather than focus and deliver its primary purpose -- "to better describe the incidence and prevalence of ALS in the United States."  Shoot, it doesn't even gather the data needed to calculate incidence.
  • The CDC can brag about using the Registry as an emailing system for clinical research notification, but in reality only a tiny percentage of enrolling ALS trials are included.  Even the scope creep isn't done well.
ALSA is telling you to ask for another $10,000,000 for this project.  You are being told that it's a success.  You are smarter than that.  

There are many lesser options to ask your legislators to consider --

  • Cease funding this project
  • Use the neurological disease surveillance included in 21st Century Cures to track ALS epidemiology (provided it does not use the same failed processes of the ALS Registry)
  • Decrease funding to well under $5.000,000 to allow basic operation of the ALS Registry
Do not let the tab for this disappointment grow to $88,000,000 as part of the charade that it is a success.  You are smarter than that.  Please speak up to legislators who are drawing up appropriations now.




Friday, February 23, 2018

Dear FDA,

Mom's Favorite Letter-Writing Ink
The following comments were submitted at
https://www.regulations.gov/document?D=FDA-2013-N-0035-0273
in response to the recently issued FDA ALS Draft Guidance for Developing Drugs:



____________

Dear FDA,
We must not let the FDA issue this Guidance to drug developers!

The draft Guidance document issued on February 18, 2018, does nothing to improve or accelerate drug development for ALS. Instead it institutionalizes the status quo. We cannot afford to do that. It is the definition of insanity.

For decades we have respected the FDA's position that you do not design clinical trials. If people dealing with ALS wanted novel trial designs, a drug developer needed to bring one to the FDA. The FDA couldn't approve or suggest something that was not proposed to them. 

The FDA's draft Guidance document for ALS insures that a novel clinical trial design will not be brought to the FDA.  
"FDA strongly recommends that sponsors conduct randomized, placebo-controlled, double-blind studies. Generally, these studies are the most efficient way to demonstrate efficacy of drugs for the treatment of ALS."
...
"Historically controlled trials for ALS are strongly discouraged."

These recommendations will stifle all innovation to find trial designs that mitigate placebo concerns for dying people or that might actually improve the control groups used to show efficacy.

In addition, you completely ignore possibilities for using technology for better, continuous assessment (removing their concerns about "patient motivation and effort") and which can make the clinical study itself more patient-focused by allowing remote reporting.

If we compare and contrast the ALS Guidance to the final DMD Guidance, we see that ALS got a much less thoughtful document when it comes to measures and effects on patients.

If we compare and contrast the ALS Guidance to the BCG-Unresponsive Nonmuscle Invasive Bladder Cancer Guidance, we wonder why ALS does not raise the same concerns that the cancer document raised about placebo --
"Single-arm trials are appropriate in clinical settings where a randomized, controlled trial is either unethical or not feasible."  

Why would this not also apply to ALS? Why is there not consistency at the FDA between cancer and ALS Guidances?  Do ethics not cross FDA divisions?

“Death” is only mentioned once in the ALS document, and it is in the context of how death can confound trial results.  Death is both cruelly and quickly approaching for every individual with ALS. Why does the guidance ignore the fatal side-effect of ALS itself?
There is nothing in your Guidance draft that will improve the status quo for people with ALS and for drug developers seeking more efficient and more effective paths to drug approvals.  This Guidance would be worse than no Guidance.
Making the status quo the official guidance of the FDA is not acceptable. You could have written this document 40 years ago. Have you no thoughts related to acceptable risk, type 2 errors, trial designs now that there are two FDA approved products, identification of responder subgroups, etc?
 I participated in the FDA ALS hearing five years ago.  Were you not listening, FDA?  We deserve better than this document.
 From your mission: “"FDA is responsible for advancing the public health by helping to speed innovations that make medical products more effective, safer, and more affordable and by helping the public get the accurate, science-based information they need to use medical products and foods to maintain and improve their health.”
This draft Guidance fails to meet your mission.
Sincerely,

Thursday, February 22, 2018

You Can Pay A Consultant The Big Bucks

Or you can simply take the earmuffs off.

A high-energy advocate for those with ALS left some advice on a Facebook post.  Surely any trustee of an ALS organization could find an actionable item or two or five or ten that would improve your effectiveness.  Surely.
1. Educate the reps on Medicare & Medicaid so they can assist families in navigating the healthcare system.2. Educate HHCs (hi, Bayada) and the reps and advocate for the proper Medicare Home Health benefit to be implemented.3. Use existing technology to make filling trials easier and accessible to more people. Remote data collection, travelling nurses trained in specific trial protocols. Encourage researchers and pharmas to use innovative trial design.  
4. Financially assist families who have lost their homes in recent disasters.
5. Use a proactive approach to care in the “excellent clinics”. Teach breath stacking and respiratory strength training. Give each family an ambubag and show them how to use it. Encourage early use of the cough assist machine. Use ongoing outpatient OT/ PT services to maximize independence. Discuss specific equipment and what each piece does before it is needed. Show families more than one brand of any given item. 
6. Train the reps on communication equipment, high tech and NO TECH.
7. Do not allow a chapter to accept donations for the care of one pALS.
8. Allow reps to make home visits beyond the initial intake.
9. Stop bragging about getting Radicava pushed through the FDA until you are ready to help people actually access it.
10. Don’t allow pharmas to price gouge us just because we are desperate.
11. LEAD the fight to protect Medicaid from block grants that will directly hurt our community.  
12. Make a graphic organizer so we can understand all the consortiums.
13. Don’t block advocates from your social media accounts. That is stupid.
14. Fund the research & trials the community tells you to fund.  
 
15. BE TRANSPARENT AND ACCOUNTABLE.

Friday, February 16, 2018

If You Could Read My Mind, Love, What A Tale My Thoughts Would Tell...


Thoughts flew through my head as I read this.  In case you would like to read my mind, thoughts are in green below.


Link to Article

ALS patient group unhappy with how $115 million raised by the Ice Bucket Challenge is being spentPublished: Feb 16, 2018 8:19 a.m. ET MarketWatch

By Emma Court
The “Ice Bucket Challenge” became a global phenomenon in the summer of 2014.
Participants lined up to have a bucket of ice water poured over their heads, with videos spreading virally across social media.
Everyone from politicians to executives, athletes, rock stars and even elephants did it (sans the ice), raising awareness of the neuromuscular disease amyotrophic lateral sclerosis (ALS) and generating donations to the cause in the process.
Much of the money raised, about $115 million, went to the nonprofit ALS Association, an unprecedented amount of money for a U.S. nonprofit, of which only a small percentage bring in revenue of more than $10 million a year.  ALSA was a financially sound charity long before the ice and its revenue engine far surpassed that $10 million threshold.
But Matt Bellina, a 34-year-old Navy veteran with ALS, told MarketWatch that he and others are not benefiting from the Ice Bucket Challenge donations that poured into the ALSA.
ALS causes loss of muscle function over time and has no cure. And because most individuals with the disease are killed by it within three to five years, time is everything, he said.
The ALSA says it has committed most of the Ice Bucket Challenge funds: about $96 million of $115 million, which includes multiyear grants for research projects that are ongoing.  The bigger issue is the net assets that they have accrued.  If somebody hands you a bag with $115,000,000 and you are going to spend it all slowly, you don’t stick it under the mattress until you eventually dole it all out. I believe that ALSA issued an rfp for financial management services related to the cash before the end of 2014.  The unspent cash is a revenue engine, too.  And ALSA was not going to stop doing its annual walks and fundraising just because it had ice cash to spend.  It had been a financially sound not-for-profit and retained <$20 million in net assets for years.  Why maintain five times that now?
But a group of about 40 ALS patients and caregivers called Terminally Persistent, which formed through a private social media page and includes members from across the U.S., “would like to see a little more aggressive spending on research that’s applicable to people living with ALS today,” Bellina, who spoke on behalf of the group, said.
“Most people diagnosed with ALS tomorrow will be dead before they could spend all that money,” he said, referring to the roughly $104 million in net assets the ALSA had as of its most recent financial statement. (The group makes annual reports, independent auditor reports and IRS 990 forms available on its website.)
But the ALSA said it is spending the money, and trying to do so in as transparent a way as possible.
The ALSA’s Ice Bucket Challenge-related spending has been detailed prominently on its website and is regularly updated, a strategy that has earned praise from nonprofit experts. The nonprofit also earns high ratings from Charity Navigator, the largest evaluator and rater of nonprofits.  This honestly does not speak well for the ratings services. ALSA research spending details are more like press releases. It’s difficult to sort out annual expenditures and committed funds by project.  Cumulative numbers and multi-year totals make it next to impossible to normalize the dollars into something meaningful. I spent hours a few months ago trying to relate funding announcements to their 990 research grants.  It shouldn’t be that hard if they want to fulfill the spirit of transparency in addition to the letter of the transparency.  Clarity is what many of us seek.

“Some people expect we should have spent $100 million in one year. I don’t think that’s realistic, but I understand where it’s coming from, a place of hope,” said Calaneet Balas, ALSA president and chief executive officer. “There’s a lot of money going out the door but if you have ALS, it might not feel good enough.”  ALSA’s Chief Scientist used to have a nice development hook at the end of presentations after she had highlighted a few projects – “For every project you’ve seen tonight, we have ten more good projects that we are unable to fund.”  I’ve not heard that line lately.  Were it true, it seems to me that all the cash would have been working in labs and clinics long before today.

Patient groups like the ALSA, which aim to represent the sick, are widespread in the U.S. Though these groups provide invaluable information and services, they have become controversial because of close financial ties with industry and advocacy groups that have helped get drugs approved.  ALSA is not immune to this concern, and it does relate to research that is supported.  We have no clarity on donation amounts from ALSA’s industry “partners.”  We are glad to celebrate the philanthropy of corporate donors, but it should also be clear and public knowledge of the donation amounts made by any entity that has an interest in selling goods or services to those with ALS.  It’s simple.  Get the information out on the table.  We can’t even figure out if these “partnerships” affect which clinical trials ALSA promotes.  I wish they would be more vigorous about promoting all trials and not leave us with nagging questions about who gets the special clinical trial publicity.
The ALSA, having stumbled into a small-donations windfall, is in nearly the polar opposite situation. But its financial good fortune raises a similarly pressing, and even philosophical question: What is truly the best way to help patients?
Nonprofits typically look to donors for direction on how to prioritize spending. Should ALSA not be looking to those with ALS?  Individuals living with this beast and the ticking clock are the reason ALSA exists. Donors, as kind and generous and important as they may be, are not necessarily the best ones to come up with bold and transformational ways to invest in ALS research. Donors and not-for-profits may be among the most risk-averse folks on earth!  But here, without particularly detailed guidance, “there’s going to be this tension, a built-in conflict,” said Doug White, a philanthropic and nonprofit adviser. “If the $115 million that was raised cures ALS, everyone’s going to be happy. Lacking that very finite goal, everyone’s going to say we should do x or y.”
Spending controversy
Bellina was working as a pilot in the navy when his hand started cramping on the throttle, fingers twitching. He started having balance issues, and knew something was wrong.
Bellina now lives in Pennsylvania with his wife and three small boys. He gets around in a wheelchair or walker, and needs his wife’s help to eat, bathe and get dressed.
ALS is a progressive disease. For Bellina, it’s getting harder to talk, “and at some point I’m going to be fully paralyzed,” he said. But he doesn’t see the ALSA’s attitude reflecting that.
“There really is not a sense of urgency,” he said.
Most of the ALSA’s spending is on research, because that was the guidance the group got from donors, “to the extent we were getting any communication,” (I was blocked forever from their facebook wall for a polite 2014 post suggesting the money go to research) said Stephen Winthrop, who is chair of the ALSA’s 25-member board and currently the only board member with ALS. The nonprofit made that decision in fall 2014, as part of a five-year plan to spend the Ice Bucket Challenge donations in full, he said.  I believe that donor intent matters and that the overwhelming majority of those thousands of individual ice bucket donors intended their gifts to go to research.  Note that the first $62,000,000+ of those wonderful spontaneous donations that came in during the first weeks of the ice challenge were done so via an online donation form that offered no means to restrict a donation to research.  Also, may all the donors, including the widows who dumped ice and donated $50, see the five-year plan, please?
It is currently funding more than 150 research projects in eight countries, with projects studying how to extend lives and improve quality of life, along with looking at what causes ALS, identifying biomarkers to help treat the disease and more, Balas said.
After research, providing services to ALS patients and the community accounts for the second-largest portion of the ALSA’s spending, according to its most recent financial filing. Most of the group’s annual revenue “went back out,” said Holly Ivel, director of data services at GuideStar, a nonprofit that aims to bring more transparency to the sector, and who reviewed the ALSA’s most recent financial statement.
Moreover, though she noted that she is not a scientist, “I don’t know that accelerating the spending necessarily produces faster results.”  ALS research is risky, and we know that most projects won’t result in a meaningful treatment.  That’s a fact. We need to learn how to fail faster so that we can learn faster.  This is not a disease where we are close to homing in on a single thing.  We need to learn by failures so that we can discard the wrong paths and make sense of the rest.
The ALSA does spend on both long-term research and to relieve the suffering of individuals with ALS today, Winthrop said. But it is “a bit of a Sophie’s choice,” he said, because patients ask why more can’t be done for them. “The problem with that decision in my opinion is that the money would be very well spent, it would alleviate a lot of suffering, and we’d look up six months later, we’d have blown through all that money and we’re no closer to the cure.”  So, if they go slowly, it won’t be perceived as blowing through the money… and we may still not be any closer to a cure.

And while some in the ALS community disagree with the five-year plan, “that’s not what everyone in the ALS community is saying,” Winthrop noted.
This type of conflict is fundamentally the purview of a nonprofit’s board, said Larry Lieberman, chief operating officer at Charity Navigator, who noted that the ALSA is among the highest-rated organizations that Charity Navigator evaluates.
“Beneficiaries are not always donors. And both deserve a voice,” Lieberman said.  This statement troubles me a lot.  Beneficiaries are families dealing with ALS.  Theirs are THE voices that really matter.  They deserve better than a pay-to-play system where high net-worth donors who are vested in an organization set an agenda for them. It’s difficult for major donors to admit that the organization has been wrong and needs to change. They are not the ones who change paradigms.
Other ALS organizations have spent Ice Bucket Challenge donations more quickly, though they received far more limited funds.
The ALS Therapy Development Institute, a nonprofit biotech that conducts ALS research, spent “every single cent” of its $4 million in Ice Bucket Challenge donations within 15 months, primarily to expand participation in a new program and to advance a drug in clinical trials, said ALS TDI’s Rob Goldstein, vice president of ALS community engagement and chief marketing officer. (The ALS TDI has been a recipient of ALSA grants.)
His organization did so because “we thought we could apply that money in a quick amount of time to advance the mission,” and many other ALS groups did too, Goldstein said. “Urgency has to be at the core of everything you do.”
But “other nonprofits may have a different way of allocating their resources, and that’s up to them,” he said. “And it’s up to donors and the general public to decide if that’s what they want to see.”
This isn’t the first time that the ALSA has been criticized in conjunction with Ice Bucket Challenge donations. Back in 2014, the group was slammed for trying to patent “ice bucket challenge” and “ALS ice bucket challenge.”
More recently, in mid-2016, the nonprofit said that a new ALS gene had been found thanks to proceeds from the Ice Bucket Challenge.

But experts questioned whether it was much of a discovery at all.
Two doctors told the publication HealthNewsReview that the gene, called NEK1, had already been of interest in ALS, and that the finding was merely an association with ALS, which is the case with many genes for many diseases.
But Balas, who became CEO and president of the ALSA last December, defended the research in an interview with MarketWatch late last week.   We have red flag words that constantly come up in ALS research announcements – “breakthrough,” “exciting,” “promising.”  The more we discover, the less we really know about the disease.  The task ahead is enormous.  That is the reason why we must not break our arms patting ourselves on the back. We must move boldly with more and novel research.
“What it highlighted was we were able to show progress in a very short period of time from the Ice Bucket dollars. In less than 24 months, we had four new genetic discoveries, and that helps us create targets for therapies,” she said.  So?
Clinical trial conflict
Terminally Persistent also objects to the ALSA not providing funding to a biotech called BrainStorm Cell Therapeutics BCLI, -1.92% The biotech’s stem cell treatment is the only ALS therapy currently in phase 3 trials, it told MarketWatch.
BrainStorm and the ALSA discussed funding for the phase 3 trial, but the ALSA said it does not fund that stage of clinical trials, both parties told MarketWatch.  Fine, but if that’s a new policy, they should have said so.  It appears that they’ve used a high-tech eraser since the BCLI question came up. The title of the Cytokinetics Phase 3 grant they made in 2015-2017 has been tweaked to look less like a Phase 3 grant.  The fact that they would retitle an old announcement is revealing.
Phase 3 trials are too high-risk and high-cost, Balas told MarketWatch.
But Bellina, who owns about $1,000 in BrainStorm stock, said the nonprofit was too set on a paradigm that “we’re way out from finding a cure, so we have to keep the momentum going and hopefully generations later we’ll find a cure.” (Bellina said he bought the stock a while back based on research that impressed him.)  Ok, we know that Bellina invested $1000 in Brainstorm.  We know generally what ALSA has invested in grants to  therapy developers.  We know nothing about what ALSA has accepted from therapy developers who are investing in ALSA.  How about if everybody gets all investments out on the table as clearly as Bellina did with his $1000 and let’s continue the conversation.
“As a result, when we have these new and exciting treatments coming up, we don’t have the ability to jump up and get involved, because that’s not the way their business has been structured,” he said.
This is a fairly common conflict for any kind of organization, but especially a nonprofit focused on disease, White told MarketWatch.
“Any disease charity has a tough road, because they have to balance the short term and long term,” he said. “That’s especially true with disease organizations, because you want to end the disease.”
In an interview with MarketWatch, Balas said she didn’t know whether there was a connection between BrainStorm and Terminally Persistent, but suggested that there was one.  This reflects the disrespect and marginalization that many have experienced when they have challenged decisions at ALSA.  It’s not new. There seems to be no sense there that individuals who seek change can be independent and altruistic and well-informed when they disagree with ALSA. There’s not a quid pro quo behind every group or individual. I don't know why they think that way.
Bellina, for his part, said there is no connection, though he has spoken with BrainStorm, which BrainStorm Chief Executive Chaim Lebovits confirmed.
As for Bellina, he still thinks the ALSA does good work, especially his local chapter, which he’s involved with.
But he says he’s willing to fight for change, even if it makes him look like the bad guy, criticizing a nonprofit that works on behalf of patients.
“I think they’re doing more good than harm, but I think allowing them to get away with things that we know are wrong is going to ultimately do more harm,” he said. “In other words, the damage we’re doing to the organization is overall worth the trouble, in order to push them to real transparency.”
BrainStorm shares have fallen 5.2% in the last 12 months, while the S&P 500SPX, +0.54%  has gained 14% and the Dow Jones Industrial Average DJIA, +0.82% has gained 20%.


Wednesday, February 7, 2018

NOTICE - Stay On Job Till Whistle Blows


And the whistle isn't blowing soon!

We're in the midst of more Right to Try (RTT) controversy as the House version of the federal bill is under consideration.  There are valid concerns on both sides of the issue.  The perspective of those dealing with ALS, a quickly fatal disease with no effective treatment options, adds more fuel to the idea of giving people broader access to drugs which have passed safety trials but are still in efficacy trials.

A "yes" to RTT isn't perfect.

A "no" to RTT isn't perfect.

Some health advocacy organizations have taken stands against RTT.

Some health advocacy organizations won't touch it with a 10-foot pole.  Most ALS organizations fall into this category.

Some health advocacy organizations endorse RTT.

My concern today is that health advocacy organizations have taken one of the three positions, breathe a sigh of relief, and think that they are finished with this issue.  You are not.  Step up and get back to work!

If you think that RTT isn't the answer or if you are conveniently agnostic on the topic, get to work.  If you think RTT is the answer but are aware of its flaws, get to work.  There are dozens of things you all can and should do today to make experimental treatments more accessible to the patients you are supposed to be working for.
  • Get clinical current clinical research opportunities to your patients aggressively.  Can your employees even name the five closest enrolling clinical trials?  I didn't think so.  They should be talking about them constantly. 
  • Fight for broader access to clinical trials.  ALS researchers have said that the two-year window for eligibility is arbitrary and question whether it even makes a difference, but trial designs don't change.  Step up.  Demand trials that are more inclusive of people who have often wasted over a year from onset getting a diagnosis.  
  • Move quickly to eliminate the distance barriers that prevent people from being able to enroll in clinical trials.  There are large populations of people with ALS who are not within 100 miles of any enrolling interventional trial.  There are people in Iowa and Indiana who would love to have access to a try at a clinical trial. 
  • Stop letting clinical trials be cheap with clinical trial participants.  They should be kept whole for their investment in clinical research.  A billion dollars for a new drug and nobody can pay for an Uber/Lyft ride for someone to get to a trial appointment?  Can't anybody pop for a room at a Holiday Inn Express for a patient traveling to a site?  Cmon.

This stuff isn't that hard.  It will require effort. It will require aggressive advocacy on behalf of the people you are paid to help. You are not finished just because your board voted on your position (or non-position) on RTT.  Your patients have a Right to Better now.  You aren't anywhere nearly finished.  Get to work.  We'll let you know when the whistle blows.

Monday, January 15, 2018

We Need A Simple Standard

Lest we lose our way in a fog of "partnerships," let this be the standard by which all ALS organizations provide transparency, clarity, and a celebration of generosity --


ALS organizations will publicly and promptly reveal exact donation amounts from any benefactor with an interest in selling goods or services to people with ALS.



It's simple.  Why not?  Really, why not?

Monday, January 8, 2018

Please Join Some New ALS Advocacy Grass Roots

Do you have ideas for better government policies related to ALS?

Would you find it energizing to engage in substantive discussions with other advocates?

Do you want to speak to your legislators from YOUR heart about priorities that YOU think are most important?

Here is a new gathering for you.

www.MoreThanOurStories.org

This is truly grassroots. It's about YOU and YOUR ideas and YOUR personal calls to action.

Please join the conversations on February 13-14.

Everyone is welcome.  Everyone is included.

Thursday, December 21, 2017

My Random Walk Through #ALSSYMP

This was my fourth International Symposium on ALS MND.

After a couple of weeks, a few things have stood out to me to distinguish this one... and a few other things that remind me of the same old same old that needs to change.

The Symposium itself has some preliminary undercards to get us warmed up for the three days of hard-core science.

Ask the _______

They call it "Ask the Experts.  That name needs to change.  Actually, the event format itself could use some change.  Last year in Dublin the three neuroscientists on the panel gave crisp, timely, short presentations followed by time for Q and A.  This year the format reverted to that of my first two experiences where the neuroscientists repurposed longer presentations and talked too long, questions that were pre-submitted via email were never addressed, and the session finished with unasked questions still in the room.  It happens on a day that is convenient for the meeting schedule, but most inconvenient for people with ALS and caregivers who want to attend the three days of scientific symposium, too.

One of my questions made it to the panel -- What would it take to get fairly inexpensive potential biomarkers like serum creatinine or retina thickness (that have been suggested for years) to a definitive "yes it is or no it isn't" decision?  The answer was very vague.  Again, later in the scientific symposium, creatinine came up, Vitamin D came up, TSH came up.  These are not expensive things to test, but nobody is stepping up to come up with a definitive answer.

ENCALS Meeting

I appreciate that this meeting of European researchers the afternoon before the start of the scientific symposium was open for anyone to register and there was no charge.

It was enlightening.  One presentation from Dr. Tang took old Rilultek data and mapped it to the Kings ALS Staging.  Contrary to my intuition, the responders were in the Stage IV group (most advanced).  That seems to me to be a big deal as people with ALS choose among a very sparse set of treatment options.

ALS Patient Fellows Dinner

This year a program was started thanks to the help of ALSTDI and PatientsLikeMe and some motivated individuals to offer fellowships to the scientific symposium to people with ALS and caregivers.  They submitted applications showing their willingness and ability to participate in the science and move some patient voice closer to the scientists actually working in labs and clinics.  The patient fellows were fabulous -- smart, informed, insightful, challenging.  Our dinner was a highlight as we got acquainted in person and talked about the symposium and ALS in general.  Special thanks to Rob Goldstein and Paul Wicks.  I think that their later interactions and questions at the symposium proved the value of reducing barriers (such as the substantial fee for patients and caregivers) to have them be part of the fabric of the symposium.

28th International Symposium on ALS MND

The abstracts are all available online for those who want to dig in.  https://www.mndassociation.org/symposium/programme/abstracts-online/

The keynote from Dr. Rosenfeld was a refreshing dose of cold water in our faces.  He suggested some significant paradigm changes at defining ALS.  He even suggested that we have failed in the past, so it's time to change.   Unfortunately the whole paradigm shifting idea seemed to be lost after the next coffee break.  We fell back into our same old same old ruts.

We started seeing the use of the word "subjects" for people participating in ALS research in the first prize-winning presentation.  Oops.  It was better than in the past, but we still see that word used by far too many neuroscientists who should know to have more respect for the most important people on their clinical research teams.

A few of the typical acknowledgement slides at the ends of presentations included people with ALS.  Even fewer had them at the top where they belong.  Something so simple, so respectful, so appropriate is so difficult with this group.

During a panel on telemedicine and precision medicine, we heard again of the silos in precision medicine programs.  If this were a corporate shareholders' meeting, the crowd would have risen to say, "Fix the silos. Now."  This crowd nods and moves on.  I had my hand up for the "Fix the silos now" suggestion but time for questions ran out right before my moment.

A neurologist asked about how they would get paid for telemedicine.  Surely a practical question, but certainly if more people with ALS were present, it may have not been on the front burner.

At a breakout session (I was not present), one of the patient fellows evidently asked a mic-drop question to a panel about the cost/value proposition in comparing supportive care for vented people and expensive meh drugs for ALS.  You can consider his thoughts on this and another important topic, placebo groups  here.

And speaking of placebo trials, we saw to instances where placebo groups seemed to do relatively well.  Overachieving placebo groups spoil the spotlight for the drug.  So... live by the placebo sword, die by the placebo sword?  I wonder if randomization put the real drug in the hands of those who were in the overachieving placebo group if a meh drug would have been found to be wildly effective.

I learned the term "enrichment strategy."  It's a way of picking trial participants to make the drug more likely to show that (or look like) it works.

In the session on recent clinical trial results, it was a lot of "more study needed."  To my eye, either the drugs aren't working or the trial designs aren't working.

There was a chippy moment when a well-known neurologist suggested that reproducibility of trial results was needed for edaravone.  The drug company doc didn't like the suggestion.  Chippy moments are a lot more constructive when we have good discourse and not indignation.

There was a panel on Right to Try, Expanded Access, etc. that included an ethicist, an entrepreneur,

and a neurologist.  Does anyone notice who is missing in this discussion?  Dr. Bateman-House from NYU was quite good, and I appreciate her tweeting answers back to me while she sat on the panel on the big stage.

I saw one presentation that I didn't think was very solid science, and it was an area where I feel I have some background.  A neurologist who is vested in the project got the mic at the end and said, "Very nice work..." before his question.  That spoke volumes to me.

An ALS advocate at the symposium mentioned more than once that she got the best information from symposium participants at the hotel bar.  That's true sometimes.  I learned of a study on lithium that showed actual efficacy in a specific subgroup.  I saw a communications technology expert help one of the patient fellows who had been led down the wrong technology path.  Much goes on outside the sessions here.  I even learned on an elevator that my tweets about the word "subjects" are useful in educating students to avoid that term.

There was another mic-drop moment in a large session when a man with ALS asked a presenter if people with ALS were included in the study design process.  Nope.  Awkward.

There was much, much more.  Poster sessions were not as accessible as they had been at past symposia, and I felt like I missed a lot of material and good conversation there.

I realize that it takes far too much time and effort to implement even the simplest changes in the fight against ALS. I feel like as much as we rely on placebo trials, they aren't so stellar that we should stop looking for alternatives.  I feel now more than ever we need to embrace people with ALS and caregivers who are interested in the science at these symposia.  And I am especially grateful for the inaugural Patient Fellows who made the trip and added to some important conversations.


Late addition:  Oh, and take your Vitamin D.  One study it as a possible biomarker to disease progression.  More study needed.  A neurologist asked a refreshing, common-sense question -- "Why not test people with ALS for Vitamin D deficiency?  It's not expensive.  Vitamin D supplement is low risk.  Just do it."  At times like this, if all neurologists exercised such common sense and reported findings, we could figure out if it's a helpful biomarker or not (and perhaps help some people with ALS along the way).








Sunday, December 3, 2017

Meet The ALS Clinical Research Swear Jar

Last week on a webinar, I heard a clinical investigator puzzled at the low enrollment in a particular trial.  How about dropping the word "subjects" for the most important people on your clinical research team, Doc?

Nobody aspires to be called "subject."  It's not respectful.  It's objectifying.  It conjures up images of royal underlings.  People who put their lives on the line to advance science deserve respect.  Words matter.

Reference to "subjects" has long been banished in many disease areas and industries.  ALS researchers don't seem to have gotten the message.  Maybe this will help.  Let's have a jar where ALS scientists have to drop a Benjamin every time they slip with the "s" word for a valued clinical research participant?

ALS MND Symposium presenters, use a more respectful word or have your wallets ready.

Tuesday, November 21, 2017

We Can Multitask. We Must!

You never know when a new, hot-button ALS advocacy issue will pop up.  ALS policy issues don't happen in an orderly set of three things that can be addressed every May.

Things can happen quickly, especially the bad things, and we need to jump on them and speak up to our legislators.

For the last three months, the Orphan Drug Tax Credit has been on the chopping block as part of the tax-reform proposals.  If you're not familiar with the Orphan Drug Tax Credit, Google is your friend.  It has been effective in stimulating development for low-prevalence diseases like ALS and dozens of others.  It's an odd tax credit to eliminate since the research is valuable and the orphan diseases are expensive in many ways.  There have been tax-credit abuses, but you don't throw the baby out with the bathwater.  We all need to make sure our legislators know that this tax credit must not be eliminated in the name of tax reform.

We need to jump on this.  We need to email our legislators now.  This is urgent.

Other things pop up constantly -- inconsistencies in Medicare coverage, loss of Medicaid, changes in technology access, drug approval processes, SSDI rules, CDC Registry dysfunction, etc. -- that we need to speak out about.  Our elected officials and government employees need to know our concerns and expectations.

Where are our calls to action?  It's frustrating (and wasteful) when our large advocacy organizations are slow to mobilize their large bases of advocates on issues of immense and urgent importance.  If only their passion for SoMe fundraising also applied to public policy issues.

ALS develops the world's greatest multitaskers.  Once you've been a hands-on ALS caregiver, you can juggle and complete dozens of tasks simultaneously.  You must.  We advocates can multitask.  We can handle more than a neat list of three priorities in May.

A concerned, capable base of advocates that wants to be effective and engaged is a terrible thing to waste.

Monday, October 23, 2017

Which People With ALS Count?

Dear CDC,

You published your first prevalence report from your ALS Registry in 2014 and asserted that ALS was white guys' disease.



The next day you were warned of publishing assumptions based on incomplete data in a thoughtful paper written by a man with ALS.  You obviously didn't pay attention.


You kept misinforming in the next annual report published in 2016.


We persisted to try to point out that we don't know what we don't know.  Which dots was the CDC collecting?

And here we are today with your new report that should cause outrage among all who have been paying for this Registry.  You finally admitted that you don't know what you don't know.


We repeat, the CDC ALS Registry found only 2720 people with ALS in areas where other studies identified 4767 people in a corresponding time period.  Of the 2047 who were missed, they were more likely to be people of color and younger than the group that the CDC ALS Registry found.

This is more than an "oops" moment.  This is serious.

Our data represent us.  With a poorly understood and poorly diagnosed disease, the Registry's failure to count some demographic groups has real consequences.

  1. We don't know how many cases of ALS are not diagnosed before people die.  Saying that ALS is white guys' disease can be a self-fulfilling assertion.  How many doctors miss the ALS diagnosis in people of color?  After all, the CDC said it was mostly white guys' disease. 
  2. Good epidemiological data should inform science and priorities.  Your misleading data are perhaps steering precious research funds down some wrong paths.
  3. Finally, you brag of your Registry's old-fashioned emailing system to inform some people with ALS of some clinical trials.  Now we know it actually contributes to the problem of lack of diversity in trial participation.

Every person with ALS counts.  We must stop the madness of publishing data that suggest that all people with ALS look like the incomplete subset of people that the CDC's ALS Registry found.

This should be the end of the CDC's ALS Registry.  An algorithm, process, and reporting that systematically discriminate are just wrong.

Sincerely,

A Disappointed ALS Advocate and Citizen and Taxpayer