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.

Thursday, June 14, 2018

The Picture Words Paint


The story has been repeated dozens of times.  A CEO at BIO last week told of a daughter of a terminally ill woman demanding a drug for her mom under Right to Try.  The picture he painted was of an uninformed, irate, unreasonable daughter.  One irate daughter has become the image of terminally ill people trying to find out how Right to Try access to investigational drugs might work for them.

It's an unfair and inaccurate picture.

This article paints it on an even bigger canvas --

https://www.forbes.com/sites/arleneweintraub/2018/06/13/biotech-executives-fret-over-hassles-and-uncertainties-of-right-to-try/#2c683ac0bc78

"...the resources they would have to pour into handling a potential flood of requests from desperate patients..."(Ah, paint them as desperate rather than terminally ill people trying to discern their options. Desperate.)
"It was just one of 10 Right to Try requests the company had already received." (Wow, 10 whole requests can bring a company to its knees? )
"...but that doesn’t make the burden of dealing with phone calls and e-mails from patients demanding access to experimental products any less burdensome."(Demanding?  Or are most simply inquiring, as would anyone in their shoes.) 
"... countless inquiries from patients..."(Perhaps that's a sign that companies need better outward communications so that countless people dealing with quickly fatal diseases don't need to inquire, one after another.) 
"...patients pestering Lebovits..."(Pestering?  Really?  Pestering?  Did the author actually listen to the call?  Pestering is what the kid behind you on the airplane does kicking incessantly on your seat back.  Asking polite, thoughtful questions about access to NurOwn on a call designed for that purpose is not pestering.  I have verified that "pestering" is not a word that Mr. Lebovits used.)

Communications-savvy companies now have a new challenge to address -- their Right to Try policies. It is not unreasonable, nor are the thousands of families dealing with ALS who simply (and urgently) need to know their options.  

That's a picture worth painting accurately.

Thursday, May 31, 2018

Here's The Deal

People with ALS hear that all the time.  Here's the deal.  Sign here.

Clinical trials have a deal.  Van purchases have a deal.  Homecare companies have a deal.  Power wheelchairs have a deal.  Pharmacies have a deal.  Hospitals have a deal.  The ramp for the front door has a deal.  The fundraiser even has a deal.

But it's never really a deal.  It's always THEIR deal.  They write the rules.  Their lawyers write the contracts. Take it or leave it.  A person with ALS has no leverage.  You sign.

The patient is at the center of everything they do except the deal.

Yesterday with the signing of Right to Try legislation in the US, we're entering the era of some important new deals.  Who is going to look out for people with ALS in these deals?

Who is going to make sure that dying people aren't handed more papers to sign that are a bad deal for them and others with ALS?

  • Who is actually going to make clinical trial deals better so that people who volunteer for clinical research aren't forced into a pay-to-play arrangement to continue an experimental therapy?  
  • Who is going to make sure that people who got stuck with placebo in a trial are at least given a free try of the therapy as part of the trial deal without having to resort to Right to Try access?
  • Who is going to keep Right to Try deals from imposing secrecy that will attempt to stifle patient rights to have and use and share their own data.
  • Who is going to make sure that Right to Try deals and monetary exchanges are transparent?
  • Who is going to stand up for the patients and make sure that they get some leverage in a deal and are not just handed somebody else's idea of a deal

Patients asserted the right to try.  They have a right to a say in the deal, too.

Code of Conduct anyone?  Now is the time.








Monday, May 28, 2018

The Corn Maze We Call ALS


I've never understood why someone would pay to enter a corn maze. Why would you want to become totally disoriented, having to learn the hard way at each decision point, receiving no clues along the way from people who navigated before you, and never sure how or when or where you will finish?  Oh, and then the next person gets to be as confused as you.  Fun?  Not for me.

Perhaps it's because I've already done the ultimate corn maze -- ALS caregiving.

Families dealing with ALS work hard and do research and discover the same things that other families discovered two years ago or two months ago or two hours ago.  They waste precious time going down disorienting wrong paths.  Sometimes they later find that others knew things that would have saved that them from the dead ends.

And the maze gets even worse when we have multiple places to look for sometimes conflicting information. Think about a registry that notifies people that they should also register in a different registry. 

Last week with the passage of the federal Right to Try legislation,, a new maze was dreadfully apparent.  Families were calling support organizations and drug companies and individuals, trying to find out what they might be able to try and how soon they could try it. Who knew fact from rumor?  Who got a real answer from someone?

You see, there is urgency here. People are dying. 

We need a central clearing house for objective, accurate, and timely information on the three paths that people with ALS now have to access experimental treatments, and we need it immediately.

For every candidate drug (having already passed a Phase 1 trial and currently in a Phase 2 or 3 trial), families need to know at a minimum --

  • Is that drug developer currently seeking clinical trial volunteers?
  • Will the company consider an Expanded Access Program?
  • Will the company consider Right to Try access?

Think of how efficient it would be for families dealing with ALS, support people, healthcare professionals, and therapy developers to know even these basics.

Maybe start with a simple public spreadsheet.  Grow to a good database as it gets traction. Add transparent information about how programs work (criteria to participate, locations, costs), and we would actually have refreshingly efficient information for people to deal with ALS and to take advantage of appropriate paths to experimental treatments.

Until then, step right up to the entrance of yet another corn maze that will waste the precious time of everyone involved in a disease that makes you pay a terrible price for wasting time.


Thursday, May 24, 2018

Give Them A Moment At The Controls

The day a family member gets an ALS diagnosis, you become a passenger on a terrible, out-of-control, speeding, downhill train.  It is a nightmare ride and there is little you can do to change what is happening.  Somebody is in the locomotive doing ineffective things, and the whole family is stuck riding in a passenger car.

"Try" is a word that has a different meaning when you are stuck in coach.  It's not particularly the transitive verb that the rest of the world uses -- try escargot, try a Chevy, try a new tennis racket, try asparagus.  It's simply that you want to try.  You want to take a swing at life.  You want to take a nasty swipe at the disease that put you on this train.  You want a moment at the controls.

Regardless of where you are on the Right to Try opinion spectrum, we all owe it to every family dealing with ALS to give them a better journey.  Yesterday there was pandemonium with families trying to decipher what concrete options may have opened up to them.  They don't need more pandemonium.  They need help.

ALS advocates and advocacy organizations owe them that help.

This isn't a moment for "I told you so."  This isn't a moment to conveniently hide behind agnosticism.  This is a moment when families need organized information on all options (including candid policies from drug developers).  This is a moment when we can encourage people with ALS to openly share data about whatever paths they find and take.  This is a moment when we can fix the problems of trial and access processes that we didn't deal with for years.

People with ALS and their families deserve a fighting moment in that locomotive.

Monday, May 14, 2018

The $88,000,000 Question



It undercounts prevalence.

It can't even do incidence.

It has burned through $78,000,000.

Do we really want to spend another $10,000,000 on this?

Read more here.

Wednesday, May 2, 2018

Every 90, 85, 80, 75 Minutes

"Every 90 Minutes" has been a tagline that organizations have used for over a decade to describe how often someone dies from ALS in the US.

Take a look at CDC death certificate data  --

The number has been steadily increasing.  Based on an average of 7000 ALS deaths per year in the US in recent years, the tagline is missing 3 American funerals every day.

It's every 75 minutes, and that number is quickly approaching one funeral per hour.

The tagline should be "Every 75 Minutes and Not Getting Any Better."




Saturday, April 28, 2018

Do You Really Want To Be A Silent Partner In This?

Words of concern have clouded the CDC ALS Registry for a decade -- disappointing, incomplete, expensive, opaque.  The concerns have never been enough to be faced seriously by those who set funding priorities. The project gets a generous annual autopay of $10,000,000 that is a recurring top ALS Association priority. Hundreds of sincere advocates just do what they're told every year and support what must be a noble goal.

The project has now crossed a line that demands action. The goal may be noble, but the deliverable is doing harm. Yes, it is doing harm.

In October a report was quietly published that confirmed and quantified the completeness problem, and it showed data that should concern (if not outrage) every ALS advocate. Please read it.  The CDC Registry missed almost as many people with ALS as it found, and the people it missed don't exactly look like the people it counted.

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5815913/



Bias (implicit or explicit) is a big issue in clinical research, and this Registry is a source of bias.

CDC annual reports on ALS assert general disease demographics based on the people with ALS they found, not on the missing 43%.  Think about that.  In a poorly diagnosed disease, this can even be self-fulfilling.

CDC clinical research notification goes to the people with ALS they found, not the overlooked 43%.  So much for diversity in clinical trials.

Do you want to be a party to this?  Will we all look back in a decade and cringe at what this generous funding was enabling?

First, do no harm.  This Registry is doing harm.

Do you want to be a silent partner in this?

Please speak up.  This issue matters.  The 43% matter.



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.