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, January 29, 2015

Place Your Bet

Ask a person with ALS to put all his or her remaining chips on a bet.

Choice "A" is against ALS.

Choice "B" is for safety.

The chips land on "A" every time.

Friday, January 23, 2015

Will The ALS Ice Bucket Challenge Have A Surprising Legacy?

Perhaps the real legacy of the ALS Ice Bucket Challenge won't be the buckets of money.  Perhaps it will be that individuals found voice in the fight against ALS.

This week there have been incredible examples, all with roots in individuals who spoke and acted --

The meeting of Steve Gleason directly with HHS Secretary Burwell and key Senators and government officials. 
An online petition of the FDA that now has 85,000 signatures. 
And this eloquent piece from a man with ALS.

We are our stories and so much more.  There are thoughtful individual voices in the fight against ALS.  Yes, sometimes one voice is all it takes.

Thanks to all.

Sunday, January 18, 2015

Let's Change The Subject

Over the past few years, many interested in patient-centricity in clinical design and processes have banished the old word "subject" to describe those who volunteer as trial participants.

Words matter.  Nobody likes being called a "subject."  It conjures up visions of royal underlings or targets of police actions or objects.  It's simply not patient-centric.  It does not reflect the valuable role that people who volunteer for clinical trials have in these projects.

In the fight against ALS, many healthcare professionals and researchers question why it is so difficult to fill clinical trials.  What can we do?  Using words more respectful of the most important people in the projects might be a good start.


Friday, January 16, 2015

It's Time To Think Out Loud

When I testified to a very stoic FDA panel at the ALS hearing two years ago, I envisioned thought-clouds above a lot of heads.  When people with ALS, their caregivers, drug developers, and organizations asked for faster approvals, I imagined FDA scientists facing us thinking, "Fine. Bring us something."

There was a lot that was unspoken in that room.

It's time to speak.  It's time to get a proposal on the table and work out the good, the bad, the ugly, the impossible, and, yes, the possible.  There is a willing drug developer.  There are people with ALS who are more than willing to accept risk beyond a normal clinical trial in order to advance the science and have a chance at a possible therapy.  There is an FDA that understands the urgency of ALS within the context of its serious responsibilities.

Let's think out loud this time, candidly.

The Genervon GM6 conversation has its roots (literally grassroots) on the forum at www.als.net .  To view the forum requires registration, but it is well worth it if you are interested in the science and challenges.

In addition here are some pertinent background links --

This blog's first post on the subject including a letter from Genervon.
This blog's second post on the subject about choices.
The change.org petition started by a gentleman with ALS with 19K signatures as of this morning.
An important blog from another gentleman with ALS who has taken GM6 and is working with Genervon.

Silence is easy.  It's time for some difficult conversations.  Out loud.


Wednesday, January 14, 2015

We Have Reached A Fork In The Road

People with ALS face choices every day.  Most of their options are poor, but they make the best
decisions they can.

The FDA faces choices every day.  They strive for excellence as we consumers and taxpayers trust and expect.

Drug developers face choices every day.  They answer to their investors who expect financial viability while they try to answer to the needs of people with ALS and the FDA.

It's difficult.  Today we have a choice that involves all three groups.  It's especially difficult, but that's good.

Two years ago many of us made trips to the FDA to offer testimonies to those who are trusted to regulate ALS drug approvals in the United States.  Over and over, the people with ALS (who have to pick from lousy choices every day) asked to be allowed to assume more risk in the drug development process.

Genervon is going to ask the FDA for "conditional approval" of their GM6 drug candidate.  Truly, that's a shortcut from the perfect path, but can we let the perfect get in the way of something that might be good?  There are people with ALS more than willing to roll the dice with eyes wide open.  There have been enough disappointing clinical trials for them to know that "promising" does not mean "will work" and "perfectly safe."  There is a drug developer willing to make product with a good safety profile more accessible to people with ALS in a closely monitored situation.  Data will be reported to the FDA.  Nobody is trying to sell a secret magic potion.  It's an option within the FDA regulatory environment that could give people living with ALS today some access to GM6.  If it works, awesome.  If it doesn't work, valuable lessons will be learned.

Perfect?  No.  Good?  Perhaps.

If we don't seriously discuss the options and design a responsible, creative solution, we will have let down all of those people who trekked (many with great difficulty) to Washington to testify to the FDA two years ago.

Your thoughts today to the FDA will be appreciated.  Click here for contact information (contact form or email address are at the top of that page).

In addition here is an online petition provided by a gentleman with ALS for your consideration.

Thank you.




Sunday, January 11, 2015

"The FDA Will Listen To Your Voice If You Choose To Express It"


The letter below was received by an ALS caregiver/widower/advocate. He had inquired for information from Genervon about their GM6 drug candidate.  GM6 was also in the news again this week.  There is an interesting conversation on the forum at als.net that includes the gentleman with ALS treated in the recent news item.

Please read and consider and act.  It has been two years since the FDA ALS hearing.  We have a chance to speak again on something immediately actionable. If there is one thing I hope we can especially appreciate this week, it is our freedom of expression. Thank you.

______________________________

Dear All,

Thank you for writing to Genervon about your interest in GM6. Attached are our two latest press release of our trials results. Sorry there is no giddy up option to Tijuana clinic or several variation of it as suggested by a few of you.

Genervon is meeting FDA next month for accelerated approval of GM6 for all ALS patients. Our plea is that ALS patients cannot wait and GM6 is very safe with no significant adverse events in any treatment group patients, plus incidentally GM6 is efficacious including Genervon trial programs for other neurodegenerative diseases. Genervon argued that GM6 should be advanced to conditional approval in a post-marketing surveillance program based on the following:
1. Demonstrated safety

2. Certain death for thousands of ALS patients during the time necessary for a Phase 3

3. Statistically-significant efficacy between treated and placebo groups in biomarker data

4. Statistically significant efficacy between treated and historical control groups in clinical data

5. Demonstrated ability to regulate homeostasis


As the FDA undoubtedly appreciates, an entire generation of newly diagnosed ALS patients likely would die in the time it would take Genervon to conduct another round of clinical trials before GM604 could be approved and made available to treat ALS patients. Although Genervon acknowledges the paramount importance of public safety in the FDA’s drug approval process, it also believes that ALS presents a compelling case for an exception to the usual process. This view is of course shared by many sufferers of ALS.

However, the chance of a quick approval by FDA is very small. FDA has the mandate and mindset to know as much as possible about the disease and the new drug no matter how long it would take. But all of you being an important constituent FDA will liTsten to your voice if you choose to express it. Your expression should be appropriate and appreciative. The best contact would be CDER 

http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ContactCDER/default.htm

You can also express your support through social media, ALS non-profit associations or other channels. 

A sample of a patient's voice: 
"It is horrifying to be told that you will die very quickly and along the way be reduced from a vibrant adult to less than an infantile state, all while retaining your cognition but losing the ability to express it. I live this way every day. Every waking moment is a fierce battle between iron determination and utter despair. For the first time in history we have a treatment with major effect and an excellent safety profile. PALS need treatment options RIGHT NOW. The risk of GM6 at this point is extremely acceptable to us, especially given that without treatment the risk is 100% fatal. We cannot wait another few more years while tens of thousands of us die. Please allow GM6 preliminary approval and market surveillance for final determination. Doing so would very likely mean rescue from ALS for many tens of thousands and certain hope for all."

INFO@genervon.com
Genervon Biopharmaceuticals LLC
1055 E. Colorado Blvd. Suite 500
Pasadena, CA 91106
Tel: (323)-721-5500
Fax: (323)-721-9131

This email is sent on behalf of Genervon Biopharmaceuticals, LLC. It may be privileged and contains confidential information intended only for the use of the recipient(s) named above. Use by anyone else is strictly prohibited. If you have received this communication in error, please notify us immediately. 


______





Friday, January 9, 2015

Follow The Buckets

It's not easy.

Already, only a few months after so many people dumped ice and donated so generously, we're thoroughly confused by press releases and announcements, especially those related to new research grants.

We have an information problem.

It should be easy.  It's 2015.

Individuals investors with even the most modest brokerage accounts can look up their portfolio components and see totals and allocations and results very easily.  It's 2015.

Individual donors with investments in ALS research should be able to do the same with every research grant made with their donations.  That's their portfolio.

  • What ever happened with the 2013 grant made the XYZ University Stem Cell Lab?
  • What's the total year-to-date new money granted?
  • How much in total has been invested in ABC Labs in the last three years?
  • What was the outcome of the ZZZ clinical trial that we helped fund?
  • What percentage of 2014 grants was related to DEF technologies?
  • How much is budgeted for research in 2015 and not yet committed?
It should be easy.  After all, it's 2015.  Today for some inexplicable reason, it's impossible.

Monday, January 5, 2015

It's 2015. We Fight Smart. Usually.

Everything we fight these days benefits from data.  And our access to data gets better every year.

When the Colts battle the Broncos on Sunday, I'll have read a lot about playoff statistics that may affect the outcome.  Sports statisticians thrive these days and make us smarter fans.

When I get my electric bill, I'll have data to help me figure out how to fight the high cost of the low temperatures here.  Smarter meters and their data let customers be a lot smarter.

When I do my laundry and fight stains, I have an appliance that helps smarten up the water usage and cycles to match the load, and it's all based on data that washing machine gathers.

And then there's ALS.  It's a life-and-death fight for thousands of people, yet we fail to capture and share data.  And the ALS data situation seems to be getting less smart every day.

People with ALS try things.  They all do.  Some things are pretty simple while others are risky and expensive.  In 1996, my 78-year-old mother read that vitamin E might help with ALS. She tried it.  Little did she know that Lou Gehrig had tried it in 1939 with the same results that she was about to have. It made no difference.  Neither Lou nor Mom left the breadcrumbs to help the next person make a smarter decision about vitamin E.  The tools just weren't there.  But the tools are there today.

ALS has a cruel way of generating a new crop of rookies every year, and today they are stuck with anecdotes and trusted advisors (some more worthy than others) to help them make decisions.  They get more real data about their football teams or electric usage than they do about what they might try to fight ALS.

PatientsLikeMe.com offers a nice framework for people with ALS to track their individual case trajectories and interventions.  It can supply others with some reference points when they try to make smarter decisions.  If patients would maintain data profiles there, it would provide those bread crumbs for the next person.

Some neurologists and ALS organizations have discouraged people with ALS from participating there.  That puzzles me.  Patients will try things with or without data.  Please, all, let the data speak.  We're not talking about rigorous clinical trial data, but we're talking about clues and bread crumbs that will help every person with ALS and caregiver be smarter.

Privacy need not be an issue.  You can use a profile name like "Lefty" even if you're a right-handed ballerina.  You can be as vague about your identity as you please.  You don't have to participate in the Forum (or any other forum) if you don't want to.  Today, there is a senseless waste of information among people who participate on message boards or social media with anecdotes and don't provide orderly data about their disease trajectories before and after intervention.

And anyone promoting an experimental protocol or other intervention owes it to everyone to ask all people trying it to post their data publicly and completely.  Yes, let the data speak.

Data waste is not smart.  We need to help every person with ALS fight smarter.  That requires data.  It's 2015.