After All, It's All About The Patients

In the last few days patients have continued to lead the way.

The Good News

• http://www.clinicaltrials.gov/ has traditionally been the best database of clinical trials, yet it was not completely accurate on the eligibility window for one ALS trial. Since ALS patients are seldom diagnosed quickly (that's a whole other story), they often have very little time in their clinical-trial eligibility windows by the time they know they have ALS. It's a big deal if a trial will accept patients up to 36 months (rather than 24) from onset. Patient communication to the trial sponsor resulted in an important update to an important clinical trial's listing -- Neuraltus NP-001 -- http://www.clinicaltrials.gov/ct2/show/NCT01281631?term=neuraltus+amyotrophic&rank=1



• A new location was announced for the same clinical trial and within hours, our patient mapmaker had updated his work and communicated it to dozens of ALS websites.



• Several patients chided ALSadvocacy for not being more specific when referring to an important phase III trial in a prior posting. Here you go. -- Biogen Idec Dexpramipexole http://www.clinicaltrials.gov/ct2/show/NCT01281189?term=dexpramipexole&rank=3



• The facebook commentary regarding clinical trials has increased significantly. Patients are encouraging each other to "try out." Patients may or may not qualify, depending on how much damage the ALS has already done to them. The support and information being exchanged on facebook are impressive.
  


• Patients are hearing from their own trial coordinators of an uptick in inquiries based on patient referrals.



• Some trials provide unadvertised assistance with travel expenses. Patients are learning to ask.



• Finally, there are rumblings that some ALS organizations may finally be working together to make a single best information source of clinical trial possibilities. Based on past performance, that's a refreshing new goal to prove that it really is all about the patients. If a reliable, up-to-date, easy-to-use source of ALS clinical trial information emerges, then there may still be hope that all organizations might cooperate on having one excellent source of information. Patients have been begging for inter-organizational cooperation for years. This is a chance to help patients and leverage resources.

The Bad News

• There continue to be reports of large groups of patients at support groups and clinics who are not being informed of clinical trial options by their organizations and healthcare professionals.



• The online information regarding clinical trials has been updated on some ALS organization sites but not on others, and obsolete information is worse than no information! ALSA chapter sites are not accurate at this time. There is an old saying that everything is harder with ALS, and this is an ironic source that reinforces the adage. ALSadvocacy hopes that organizations will remove the red-herring information and simply supply a link to http://www.clinicaltrials.gov/ct2/results?term=amyotrophic&recr=Open It's all about the patients, right? It seems unconscionable that a patient might miss a trial opportunity based on wrong information on a trusted organization's site.



• Not all trial locations supply email addresses in addition to phone numbers for the contact people. A disease that steals the ability to speak would be served well with more appropriate communication options for patients to inquire about clinical trials.



• Clinics that are holding trials may occasionally be forgetting to tell patients about other clinical trial options that are available elsewhere. In the same vein, are not-for-profit trial sponsors forgetting to tell patients about trials sponsored by for-profit pharmaceutical companies?

The Bottom Line

It should be all about the patients. There are some current clinical trials that actually may provide improvements to patients, but we'll never know until the trials are filled and completed. There are therapies that will be huge if they live up to their promise, but only the data will tell. Patients are leading the way in getting the information to their peers so that the seats will be filled. Our ALS and healthcare delivery and clinical trial organizations have a perfect opportunity to prove that they are all about the patients. Simply listen to your patients and act, quickly, please.

Wow ... The Patients Are Taking Matters Into Their Own Hands

And they're making a big difference.

Right now there are some very interesting clinical trials being conducted for ALS. Over the years there have been hundreds of such "promising" trials, always with the quotation marks around "promising," and the disappointments and failures have been heartbreaking. This year it seems different. There are anecdotal reports of improvements in one trial. There is another that is a Phase III trial. Both are being led by some of the finest neurologists throughout the country. After all of the recent buzz and hope of finally removing the quotation marks from "promising," one fact remains -- we'll not know if these drugs will live up to the promise until the clinical trial seats are filled and the trials are completed.

One would think that patients with an untreatable, terminal disease would be knocking down the doors to get to these clinical trials. One would think. It seems that the process for getting information to ALS patients regarding clinical trials is horribly flawed. Many rely on their neurologists for information and guidance, yet often trials aren't introduced to patients and when they ask, the physician may not be familiar with the possibilities. Many rely on their trusted ALS organizations for information and guidance, yet we find a melange of outdated and incomplete information on their websites.

In the last few weeks, we have seen patients enrolled in trials taking matters into their own hands with some impressive results. Any patient who is enrolled in a clinical trial can be highly motivated to get the other seats in that trial filled so that it can move ahead. We see patients contacting others via social networking to encourage and inform regarding trials they are in. We see patients and caregivers asking trusted ALS organizations to speak up to patients regarding enrolling trials (and there have been some results). We see patients contributing to information online so that their peers can understand the relative advantages and disadvantages of trials. We even saw one patient who had traveled away from home for his monthly clinical trial infusion who dropped into a support group meeting in the clinical trial city. He found peers who were not aware of what was happening in their own back yard. One patient published a map online to show his peers all of the cities offering his trial. Facebook friends are posting information on walls wherever people with ALS and families might visit, and patients who do not qualify for trials are being a huge help to spread the word to others who might. Patients are posting their data at http://www.patientslikeme.com/ so that others may learn.

The system for spreading clinical trial information is broken. Some say there is no system. There is no excuse for a patient who qualifies for a clinical trial to be unaware of the possibility. Patients are leading the way right now. We hope that organizations and clinics and physicians will follow their lead and step it up a notch or ten!

Bob Is Still A Silent Leader

Matt June Knows How To Ask A Question

Barbara Brenner - Activist, Breast Cancer Survivor, Woman With ALS

Also, there is a beautifully written feature in USA Today by Liz Szabo --

http://yourlife.usatoday.com/health/medical/breastcancer/story/2011/07/Breast-cancer-activist-struggles-to-preserve-her-voice/49472752/1

A Gifted Writer With ALS Just Keeps On Teaching

Dudley Clendinen wrote "The Good Short Life" in Sunday's New York Times.

David Brooks wrote "Death and Budgets" in today's New York Times.

Both are enlightening. Thank you, Dudley Clendinen.

Oh, That More People Would Become Emotionally Attached!

Diesel Pete, Rest In Peace

Pete Duranko has died from ALS.

http://www.denverpost.com/sports/ci_18446945

Diesel Pete made this college freshman think that national championships were the normal part of a college football experience. Many years later he showed this ALS advocate the meaning of courage when facing this beast of a disease.

Another of our best and brightest has been taken. When will we stop this madness?

We Hope And We Pray

Over the last two decades there have been hundreds of clinical trials for ALS drugs that have delivered nothing but disappointment. We continue to raise funds and awareness so that research may continue.

Over the last several weeks there have been early reports of some positive results. It's way to early to know if early reports mean anything, but it is a most encouraging buzz among some of the trial participants.

If you have ALS or if you know someone with ALS, this Neuraltus NP001 trial might be worth some serious consideration. One must be within 36 months of disease onset to be eligible. If travel is required to get to a trial site, we understand that there is some assistance available.

We hope and we pray that this portends a viable treatment for ALS that can be delivered quickly. Around 500 Americans with ALS die every month.

Swim The English Channel to Defeat ALS

This is pretty cool...

I'm writing to you because I hope will will help spread the word about a remarkable man taking on a remarkable effort for the benefit of ALS research .....

In August, Doug McConnell, 53, of Barrington, Ill, will attempt to become the 48th person over the age of 50 to successfully cross the temperamental English Channel. He will be swimming more than 21 grueling miles in memory of his father who passed away from ALS (Lou Gehrig's disease). Doug is using this epic effort to educate the public about ALS and raise funds for medical research at Northwestern University ’s Feinberg School of Medicine in Chicago.

McConnell truly loves swimming and appreciates physical challenges. As a child, during a family vacation to England, he saw the Channel and thought to himself that one day he'd swim across the busy waterway. He and friend Don Macdonald committed to this endurance challenge and have been training for this event for approximately two years.

In late 2009, Doug developed a medical setback – a severely herniated disc between two cervical vertebrae, resulting in the loss of all use of his left arm. After physical therapy and other unsuccessful treatments, Doug underwent a cervical disc replacement procedure. The surgery was successful. Today, Doug has regained strength in his arm and the ability to move his neck to breathe while swimming, and he swims without pain. This was not enough to stop Doug's pursuit of his dream.

Medtronic, the manufacturer of the disc that was implanted into Doug's body, has pledged up to $50,000 in support of Doug's efforts. He is well on his way to raising a significant amount of money.

We hope you will post the press release on your blog. Please feel free to contact me with any questions or if you need additional information.

Regards,

Beth Richman
Les Turner ALS Foundation
773-252-3069

Here Is More On The Theme, "Let Patients Help!"

Please read the article at this link!

http://fastercures.blogspot.com/2011/07/building-bridges-across-valley-of-death.html

...He talked about how CFF’s successes – more than 30 drugs in the development pipeline, four of them already FDA-approved – have been a result of holding its partners’ feet to the fire, and keeping patients at the table every step of the way to create the urgency Feigal talked about.

But it goes beyond just having a seat at the table. While patient presence is critical to humanizing research and speeding progress, patient voices can’t just be loud, they also have to be smart on the science and knowledgeable about the regulatory environment. The recently released paper Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change, co-authored by FasterCures and HCM Strategists, distills lessons learned from the HIV/AIDS movement that can be replicated to address today’s medical research advocacy challenges. HIV/AIDS activists were successful because they refused to accept that the system was unchangeable, and instead took the time to figure out what it should be and how it should work.

In case you didn't click on that Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change link, please do so. Perhaps there really is a roadmap after all.

Here's A Wonderful July 4 Article

Thank you, Fay Vincent!

http://www.tcpalm.com/news/2011/jul/04/fay-vincent-fourth-of-july-a-time-to-remember/

We'll Never Hit A Home Run If We Don't Swing The Bat

Two years ago this weekend something happened in the United States that was wonderful. As we approached the Independence Day celebrations, traffic on this site was astronomical. People were searching for information on ALS. Each major network was doing news features on ALS, Lou Gehrig, or the 4 ALS celebrations being held on July 4 in all of our Major League Baseball parks. It was a good buzz that permeated our nation on a weekend when we feel good about being a nation that can do good things. It raised awareness of Lou Gehrig's Disease in a generation of baseball fans who weren't all that familiar with Lou Gehrig. It was a weekend when people talked to each other about ALS.

Michael Goldsmith was a remarkable man who made all of this happen with the wonderful help of Major League Baseball.

http://www.nytimes.com/2009/11/02/sports/baseball/02mgoldsmith.html

http://en.wikipedia.org/wiki/Michael_Goldsmith

We need to rekindle the spirit delivery of Michael Goldsmith and make ALS an important part of every July 4 in our country. It's not about short-term fundraising. It's about raising ALS on America's radar on an important day when baseball and good works are as important as fireworks. It takes media savvy and interorganizational cooperation and hard work.

It's interesting. People like Michael Goldsmith with ALS understand the "how" and "why" of ALS awareness. Unfortunately they die all too quickly. Michael Goldsmith pitched a nice fastball right down the center for us to hit out of the park. Our ALS organizations need to get the bats off the shoulders and learn how to hit a home run like Michael Goldsmith.

With ALS, Patients Have To Take The Healthcare Bull By The Horns

Interesting perspectives from an empowered cancer patient...

"Let Patients Help!"

What If Major Organizations Declared A Global Awareness Day...

...and nobody actually did anything?

http://alsspreadtheword.blogspot.com/2011/06/calling-day-global-awareness-day-isnt.html

It's Good To Remember Lou on His Birthday

And it will be great if we can rid the earth of this beast of a disease before his next birthday!

Eight Years... 50,000 American Funerals Later

Remember June 19, 2003? It was Lou Gehrig's 100th birthday. We passed out baseball cards and gathered donations for nice commemorative pins. We started to realize that there are a lot of young baseball fans who don't realize who Lou was. We raised awareness the way we always raised awareness -- with modest and diligent and sporadic efforts at small events.

We have lost around 16 Americans to Lou Gehrig's Diseae every day (seven days a week) in the last eight years. We still try to raise awareness in very modest ways, the way we always have.

ALS is a big problem. It's time for some big, effective, continuous efforts to bring the ALS problem into serious focus. If most people don't know ALS is still around, we certainly can't blame them for ignoring it.

Shame on us if we keep raising awareness the way we always raised awareness.

Michael Bloomberg Gets It

Take Ten Minutes to Think About ALS

Advocacy Action Requested

Following is from an email received from ALSA:

Earlier this year, we requested that you contact your Members of Congress to urge them not to eliminate the ALS Research Program at the Department of Defense. Thanks to your outreach, Congress provided $8 million for the program this year! You made a difference. But we need your help again.

The House Appropriations Committee is scheduled to vote on the FY 2012 Department of Defense Appropriations Act on Tuesday, June 14 and we have learned that an amendment may be offered that would eliminate the ALSRP next year. We cannot let this amendment pass. If it passes, at least $8 million that is so urgently needed for research no longer would be available. It would mean that research specifically designed to find a treatment would not take place.

Please contact your Representative TODAY! Tell them to save the ALSRP and work with the Appropriations Committee to make sure our government continues to support the fight to find a treatment and cure as soon as possible!

You can email your Representative directly from our website, here. Do it today. And tell your friends, family and everyone else you know to do the same. Your outreach made a difference earlier this year and we are confident that it can again -- but only if you take action today.

Thank you!

Let's Stop Shooting Our Cause In The Foot

Years ago a really smart person came up with the tagline, "Every 90 minutes someone is diagnosed with ALS. Every 90 minutes someone else dies from ALS." That's a great, concrete, attention-grabber. Unfortunately it doesn't tell the whole story.

That "90 minutes" is based on U.S. statistics only. Around 6000 people in the U.S. are diagnosed with ALS every year and a like number die from ALS. That's around 16 U.S. diagnoses and deaths per day, or one every 90 minutes. Great. But it's U.S. only!

ALS is a global problem.

The United States has around 4.5% of the world's population according to the U.S. Census Bureau.

So... assuming that the incidence of ALS is similar globally, let's try some arithmetic...

6000 / .045 = 133,333 new cases and funerals annually worldwide. Whoa.

133,333 / 365 = 365 new cases and funerals daily worldwide. Wow.

365 / 24 = 15 new cases and funerals per hour worldwide. Holy moley.

60 / 15 means that every 4 minutes someone is diagnosed with ALS. Every 4 minutes someone else dies from ALS. Yikes.

So let's get it right. Either "Every 90 minutes someone in the U.S. is diagnosed with ALS. Every 90 minutes someone else in the U.S. dies from ALS," or "Every 4 minutes someone is diagnosed with ALS. Every 4 minutes someone else dies from ALS."

Tick, tick, tick.

Or maybe, "There are well over 100 thousand new cases of ALS annually. There are well over 100 thousand others who died from ALS last year."

p.s. If my arithmetic is off, I'm all ears.

p.p.s. Check out the first paragraph http://lillypad.lilly.com/corporate-responsibility/fighting-hunger-bold-goals-moral-obligation They made a problem come to life!

p.p.p.s. 1pm EDT Fixed the arith. Got carried away with zeroes. Sorry about that. Phew!

We Need More Of This Attitude!

It Doesn't Take Long

Two years ago this coming July 4 we celebrated the 70th anniversary of Lou Gehrig's farewell speech.

Today is the 70th anniversary of Lou's death.

It didn't take long for a man to go from baseball mega-superstar to the grave. This disease persists. The outcome today is much the same that Lou Gehrig experienced.

Perhaps our tagline should be, "ALS -- I'll bet you thought someone had cured it by now."

In the time since 1941 think of the advances in science and medicine and life. Polio and smallpox were conquered. Cancers are often treatable. MS patients have a long life expectancy. We have tiny cellphones and make long distance calls at will. We have an internet. We have dishwashers and wonderful television pictures and trash bags. Why have we not figured out ALS?

These Are Not Sickly People

They are hale and hardy and playing golf and sailing one month, and suddenly ALS hits. We need more ways to show this. Here is one created by a gentleman with ALS.




Every healthy person needs to squirm a little. Nobody is immune.

We Hope Scott Pelley Remembers Steve and ALS

If you saw the 60 Minutes piece on the 21st Century Snake Oil , then you saw Steve Watters. He was an ALS patient who went undercover with Scott Pelley to expose the con man with a bogus stem cell cure for a price. Steve was always a thoughtful contributor on ALS message boards, and he turned out to be a huge contributor to the awareness of ALS in his role on that 60 Minutes segment.

Steve Watters has died.

Another nice person. Another achiever. Another contributor. Another funeral.