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.

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in response to the recently issued FDA ALS Draft Guidance for Developing Drugs: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The draft Guidance document issued on February 18, 2018, does nothing to improve or accelerate drug development for ALS.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The FDA's draft Guidance document for ALS insures that a novel clinical trial design will not be brought to the FDA.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.