Meet The Change-Agent Brothers

There is a video at http://www.kickstarter.com/projects/1295266764/patients-finding-cures-the-drug-trial-revolution that is a "must see."

Please don't just read the text.  I'm one who prefers to read than watch, but this is worth watching.

When was the last time it was so easy to be a part of a revolution?

Let's Stop Storing Needles In Haystacks

Did you ever wonder what Dr. Benatar talked about at the May, 2012, ALSA FALS Summit?  Did you ever have trouble remembering if Dr. Scott talked at an ALSTDI conference?  Would you love to have a look at an ALS Registry presentation from 2008?  Does NEALS provide any presentations from its annual meeting?  Is there a Neuraltus presentation from last years International Symposium in Sydney?

Have you ever tried to find any ALS-related Powerpoint stack that was made available somewhere online after a workshop?

Industry groups that hold lots of conferences typically have great archives of presentations available in orderly manners online.  Members know where to go to find the presentations.  Groups share tools. They have great institutional history and know the value of access to information for their constituencies.

Unfortunately with ALS presentations, it's like trying to find needles in multiple haystacks.   Somebody stored the presentations somewhere (maybe) but it takes a relentless Google genius and countless hours to find some of them.

How about a common, co-branded, well-maintained spot that all of our ALS organizations would share to provide the Powerpoint stacks and other documents and media from their conferences and workshops?  That would make the information much more accessible and would greatly enhance its usefulness.  

Please?  It's all about the patients, right?

p.s. This also leverages resources.  Curing ALS is hard. This is not hard.

Finally, An Organization Uses Global Numbers To Describe A Global Problem

For decades our American ALS organizations have used estimated U.S. numbers of new cases and numbers of patients.  For decades we have left an impression that ALS is a "small numbers" disease.  Thanks to ALSTDI for finally speaking about ALS in global metrics.

We All Have A Lot To Learn

There have been many online discussions of some serious ethical dilemmas regarding people with ALS in two recent clinical trials.

The rules of the trials seem pretty simple, yet there are a lot of things that can happen that make the interpretation of those rules a matter of life and death for people with ALS.  They deal with the ticking clock and a future with no great choices.  It's more than troubling when a good choice unanticipated when the rules were written is taken from them.

I now realize that I have a lot of questions about how a clinical trial's rules came to be.  I'm sure there are a lot of people involved -- the pharmaceutical company, scientists, the clinical investigators, the FDA -- oh, and patients -- and I have a lot to learn about who does what.

• Who sits down and writes the rules for a clinical trial?
• How many people are involved?
• Who has input into the inclusion and exclusion criteria?
• Who approves the rules?
• Who pays for the rules of the clinical trial to be written?
• How long does it take to write the rules?
• At what point does the FDA see the rules?
• How are clinical trial sizes established?
• Can any of the rules be clarified once the trial starts?
• Who handles the interpretation of the rules when a question arises?
• Who has the final say on an interpretation?

One thing I have learned in the last few weeks is that those many people involved all have a lot to learn from one another.

When Will It Stop

Carey Hall died from ALS this morning.

It's Time For Some New Scientific Tables

In early December a large global contingent of professionals involved in the fight against ALS will hold their annual meetings in Chicago.  There are three related conferences -- one for an organization of organizations, one for allied health professionals, and one for researchers.

Football programs and golf tournaments often feature a celebrity athlete at each table for those attending banquets.  How about a some people with ALS at the tables during the many meals at these meetings?  They are our celebrity heroes.  Let's make it real, and perhaps a scientist who has been spending years on a theory will benefit from learning how to work the suction machine or feeding tube.  Food always stimulates some interesting conversation, and perhaps these people should converse more.  Oh, people with ALS often speak via technology, but they can and deserve to be heard.

We talk about breaking down barriers among patients and healthcare professionals and research scientists.  They need to speak to each other.  They need to listen to each other.  The all need a place at "the table."

Perhaps there will be some tables in Chicago where the process can start.


Below are some links to information for the meetings.  It's thoroughly confusing and not particularly welcoming, but perhaps that's all the more sign that it's time for people with their own skin in this game at the table.

http://www.mndassociation.org/research/International+Symposium/Symposium+Registration+page

https://www.mndcommunity.org/sslpage.aspx?pid=416

http://www.lesturnerals.org/files/3113/4401/1886/Alliance_reg_form_2012_Chicago_fillable.pdf

Are We There Yet? Are We There Yet?

As we saw last year as people with ALS swarmed over social media and support group meetings to recruit peers in a promising Neuraltus clinical trial, a day saved recruiting patients is as valuable as a day waiting for data analysis or an approval later.

It would be interesting if subjects (and others) had three simple time lines for every clinical trial --

1. An optimistic version if every step were to go swimmingly well
2. An expected version
3. A pessimistic version featuring some worst-case timing

Sometimes we have visions of data sitting somewhere waiting for a meeting to happen on a certain date.  Sometimes we have visions of stacks of papers growing yellow with age at the FDA.

If we had a much clearer picture of what can be expected and who does what and what patients can affect themselves, would it not make for a better process for everyone?

It's Nuts

Imagine going to a stock broker who gives you two kinds of reports --

1. An occasional list of the things you bought (without prices)
2. An occasional report on one of your investments that did well

That's it.  You don't get dollar amounts on your individual investments.  You don't get a list of the investments that went bust.  You don't get information in a format that makes it easy to track individual investments over time.

Nobody would use that broker, right?  You would have to be nuts.

Much of ALS research is funded by donors and taxpayers.  We are the investors in a portfolio, yet we have information scattered here and there without a good, businesslike trail of projects, grantees, start dates, end dates, and outcomes.  We get occasional feel-good lists of what we bought.  We get occasional feel-good reports on research projects that did well. That's it.

As investors, it's time that we got better information about our portfolio.  It's time for organizations and our government to supply us data in a format that permits us to see the who, what, where, when, how, how much, and outcome.  We need information in a format that lets us look back before we invest again.

It's nuts to be such blind investors when surely grantmaking organizations hold the data that the investors deserve to see.  It's just nuts.

Holding Breath Is Not An Option

The following was submitted by a reader of www.alsadvocacy.com:

_____________________________________________________

Of course when I was a newbie ALS inductee, when it really didn't affect our lives that much, I was sure there would be a treatment soon, when our family became more affected by the disease, I was still sure there was going to be a treatment. Now that we face life and death decisions, I am sure there is NOT going to be a treatment, in time, for our family.

We've been fighting this disease for years, doing everything on every front possible that a family can do to help bring treatment to fruition. We've walked, we've ridden, and we've hounded out friends for years to help support ALS research. I know exciting research news gives people with ALS hope, and yes, can even brings in donations.

But now, when I hear news about THIS ALS research and THAT ALS research, I want to stifle back a scream or an eye roll, or at times even a laugh...most of this research write up news always ends with, "this discovery will lead to new ways to study ALS."

To me, I have learned over the years that these research news blurbs means is, "yeah, we think we found something, but you know, IF it gets from the research lab to a clinical trial, it's still YEARS away to a treatment." Oh yes, yes, I KNOW ALS is a tough disease to research, there are many aspects and nuances, and it seems to affect every person differently. I have been told that many times in ways that are compassionate, in ways that are condescending, and yes even completely ignored at times when I tried to question research.

How much more studying do you have to do? Can't you do something instead of studying the disease?

But you know what, I'm guessing research is not as tough as living with it, day in, day out, week in, week out, year in year out. The research is frustrating I'm sure,

ALS is a disgusting, vile disease all the way around; there is nothing easy about any of it. I think that is one thing that both researchers and ALS families can agree on.

But after living with the disease for these years, I'm not impressed. I'm sick and tired of hearing about research breakthroughs, that are years away from producing any kind of treatment. I suppose you could say I'm a bit jaded in my thoughts because like I said, when I was an ALS newbie, I knew there would be a treatment. However, as an old-timer in this stupid disease, I wish they would keep their news to themselves until they can come out and say, "WE HAVE A TREATMENT that works RIGHT NOW for EVERYONE." Until then, I'm not holding my breath...because hey someone has to keep breathing here.