ALS Everyday Living: 2011

Tuesday, December 20, 2011

Part 2 - One Man's Opinion on the Diaphragmatic Pacing System

Hello again from the Pacer world. Wanted to give everyone an update on what I have additionally learned with my research and numerous conversations with Dr. Onders and his staff since September. For background, also see my original Oct. 7 blog post.

Will dispense with the bad news first. Biogen has forced me to drop out of the Dex trial to have this FDA approved procedure done. Riluzak ok, Bi-Paps ok, but no DPS. Instead of being on the open label in seven months I have to go to the back of a 36-48 month line when this might be generally available. O well. Guess I am ahead of my time on this one (more on this in a coming blog post).

Good news. Cedars Sinai Medical Center in Los Angeles moved heaven and earth to become only the 2nd or 3rd facility in the country to be able to perform this procedure on ALS patients. The Neurology - ALS Center was extremely responsive to my request to get up and running by year end. My physician and the primary investigator who put it all together was Dr. Evgeny Tsimerinov. Dr. Patrick Lyden, Dr. Abi Muthukumaran, and Nurse Practitioner Hope Gruendler also contributed significantly to the efforts internally. Some additional encouragement by the local ALSA Chapter CEO Fred Fisher probably didn't hurt either. In the end, they expedited the process to just over 10 weeks from start to finish. Beat my 6-8 month prediction by miles!

I am scheduled to be the first patient west of the Rockies to receive the device since it's approval next week. Dr. Onders himself will be assisting Dr. Robert McKenna at Cedars in delivering the device. Will follow up soon with an update about the surgery itself and my personal effectiveness with the procedure.

On a side note, this is an exciting development for pALS and offers some immediate promise in our battle with this disease. However, it is still vital for pALS to level set expectations on what this significant procedure wil and will not do going forward. Dr. Onders has been performing this for almost a decade on paralyzed vent patients with much success. Much of this literature and You Tube videos from the past presents the facts on the patients in the general SCI (Spinal Cord Injury) population and not ALS patients specifically. There are rehab opportunities for the Diaphragm with SCI patients that does not exist for pALS among other things. Make sure you get your medical information from Dr. Onders himself or your ALS Clinic to make certain your expectations match the projected results from this procedure. ALS Specific Link here.

It's important to note there were 50 ALS patients in a clinical trial that ended in 2009. Trail results here. I personally spoke with a few of them that participated at Stanford. This is not a cure, but an FDA approved treatment that enhances quality of life.

The patients who used the NeuRx DPS^® plus non-invasive ventilation (NIV) (such as BiPAP^®) survived 16 months longer (on average) than patients who just used NIV. This 16 month time frame is from the time of diagnosis. Survival time was measured until death or the need for a full time ventilator and a tube in the throat (tracheostomy).
The patients who used the NeuRx DPS^® plus NIV survived 9 months longer (on average) than patients who just used NIV. This is from the time they started using NIV.
Some patients had a feeding tube placed with the NeuRx DPS^®. All of these patients survived past 30 days. Normally, 2 to 25 patients out of 100 (up to a fourth of all patients) would not survive this long.
Some patients had their sleep tested just before their NeuRx DPS^® implant and again after using the NeuRx DPS^® for 4 months. They had better sleep with DPS, with reduced apneas and hypopneas during REM sleep, than before they received NeuRx DPS^®.

If your Phrenic Nerve is shot, or your FVC Respiratory readings are too low it is too late for you. If in doubt, get a Phrenic Nerve study and a Diaphragmatic Fluoroscopy to see if you qualify at your local ALS Clinic or Center.

Medicare and Private Insurance covers this $55,000 procedure. You can go other places than Cleveland to get it done like LA or Dallas, with more on the way. Make sure you go to an ALS authorized Hospital, not SCI approved. There is a difference. ALS Locations here.

Hope to give you update #3 shortly after New Year's Day.

Friday, December 9, 2011

Thursday, November 10, 2011

Compassionate Use Drug Availability for ALS Patients

Time to wade into the water on a controversial topic, Compassionate Use Drug authorization for ALS Patients.

This topic has been discussed extensively for some time on the message boards and social media sites. Terminally ill patients with little to lose want access to whatever is available that has shown safety and efficacy and potential upside as soon as possible. Every day and minute counts.

Most clinical trials run 12-15 years from conception to shelf. Only one drug is approved for ALS treatment today and is marginally effective. All the new announcements of discoveries we see today will ultimately yield some hypothesis in labs. A few of those will begin that 12-15 years cycle at some point noted above and the cycle continues. Some promising drugs may already be in that 12-15 year pipeline at a middle or even latter stage and the cycle continues.

On the one hand you have pALS that are ineligible for these studies. Diagnosis more than 2-3 years prior, Functional Ratings Scale not at a certain point, or Respiratory Readings below the required reading are common disqualifiers.

On other hand you have pALS that are qualified to participate in Clinical Trials. All of them are double blind placebo based, the longtime industry standard. By definition, only half of the patients actually receive the drug that do participate. The Phase 2 or 3 study finishes recruitment and runs the required 12-18 months AFTER the LAST patient is enrolled. They then usually need 6-12 months to interpret the data and at least another year to get FDA approval and something potentially to shelf. Total elapsed time estimated from start to finish for this portion is 2-5 years. (Does 2-5 years ring a bell with anyone here?).

You get the picture. It's no wonder that ANYTHING currently showing promise stirs up interest in the pALS community for Compassionate Use.

Dexpramipexole (KNS-760704) is now 14+ years from the time the drug was discovered as a theoretical treatment. As is common with many other biotechs and pharmas, current owner Biogen Idec entered the process on this particular drug only recently.

The recent ALS TDI conference was extremely informative and featured some industry insiders discussing the state of ALS Research from a business perspective. It was very topical and George Scangros (CEO of Biogen Idec) was one of the participants. During the Q&A, a caregiver speaking on behalf of a pALS who could not speak, ask a key question to Mr. Scangros. He stated that he was ineligible for the DexPra Clinical Trial currently being conducted. "Was there a way to obtain compassionate use availability of the DexPra Drug for pALS outside the trial?," he asked. George said that they had considered it. But...Biogen felt the best way to get to a final decision on whether they could move the drug forward to the FDA was to focus all their resources toward the 800 current enrollees and the 400 additional pALS they are now enrolling." BOOM. No follow up question from the audience. Only a vague timeline of 12 months from now they will have the last patients full readings, then another 6-12 months to interpret the data, then another year or so to get FDA approval. This poor guy, and any of the rest of us pALS listening basically were told TOUGH LUCK FOR AT LEAST 2-5 YEARS (2-5 Years ring a bell?).

I am not naive. George runs a large biotech company that answers to shareholders. Biogen is a $4 Billion Fortune 500 company that threw off over $1 Billion in Net Income in 2010. They have stated they are committed to ALS Research. God knows we need more profitable companies like this supporting finding treatments and a potential cure. The capitalistic nature of drug development absolutely motivated these guys to make this investment after Knopp's trials showed promise. Biogen wants to bring this to market on a broadscale level to maximize their Return on Investment. I GET THAT.

Biogen's website states "To our partners and investors, we are a profitable company with a rich and diverse pipeline. We have the resources, financial strength and vision to successfully discover, develop, manufacture and commercialize new products. And to our friends, neighbors and the world at large, we are a responsible corporate citizen with robust programs that make our communities better places to live and work and address critical issues like diversity and sustainability. Biogen Idec is all these companies. We are dedicated to serving all our constituents, because Biogen Idec is in business to make a difference for all those we serve. Addressing patient needs is our core responsibility and defines our corporate citizenship. We updated our Code of Business Conduct to include additional guidance on critical issues, such as workplace health, responsible marketing and ethics in clinical research."

Two things need to occur with Dex, Fast Track Status Application and Compassionate Use Application. I assume the former is in process, and the latter has not occurred.

Regarding Fast Track Status, the FDA Division of Drug Information says to be eligible for the fast track program, a sponsor must first submit a request for fast track designation with supporting documentation for the product and its proposed use. Only when such a request has been formally submitted can FDA consider whether the conditions for fast track designation have been met (in brief, fast track designation can be granted for a drug to treat a serious or life-threatening disease if it is considered to fulfill an unmet medical need). If fast track designation is granted, the development of the drug may be expedited in certain ways. For a drug to be FDA approved, the sponsor must submit a New Drug Application (NDA) with data from clinical trials supporting the safety and effectiveness of the drug. For those drugs granted fast track designation, the Agency works closely with the sponsor to determine the most efficient development path for the drug, given that all drugs must be found to be safe and effective before they can be approved. It is sometimes also possible that drugs not yet approved may be made available to patients under special provisions of the law while the review of an NDA is on-going. Whether these provisions will apply in a given case will depend upon the data submitted by the sponsor, as well as other circumstances.

Regarding Compassionate Use, it's a common misperception in the ALS community that the FDA decides who gets compassionate use exceptions in totality. That is only part true. The first thing that needs to occur is the drug company has to APPLY. I would like to ask Biogen to review the statements italicized above and call for them to at least APPLY. Apply to the FDA for a compassionate use exception for pALS like my friend in the room at the ALS TDI conference that could not speak for himself. The data they have espoused over the past year to gain pALS, Investor and Industry support states this a safe, promising and potentially effective treatment option for pALS in the offing. While I am sure it would cost them some money to provide the drug to the pALS on a Compassionate Use level, it would in no way jeopardize their ability to complete the clinical trials and move through the required process.

In the near term, there are other hopefully effective treatments in the pipeline that pALS should encourage to do the same. To my knowledge Cephalon, with the drug Myotrophin (IGF-1), was about the only company in the past (over 15 years ago) that I am aware of that has even bothered to apply for a compassionate use exception with the FDA. These companies all walk a tightrope on bringing tomorrow's promise to patients today, ahead of the established curve. I GET THAT. BUT WE PATIENTS HAVE NOTHING TO LOSE AND TIME IS OUR ENEMY. Many of them, like Cephalon, are companies with shoestring budgets that are barely holding on to be able to run the existing trials in process. Money will almost certainly play a big role in their ability to offer Compassionate Use in the future. If they go out of business, it will not matter how promising the drug unless someone else acquires or invest in them. That's an unfortunate reality within the system today.

The system also continually asks pALS to participate in Clinical trials today that have almost zero probability to help us as individuals. But we do it to help others because it's the right thing to do.

Biogen is different from almost all the other companies noted above because they have the ability to help patients now. They have the money ($1 Billion in Net Income last year alone) and resources to step up and provide compassionate use of the drug today. If we are to believe all the hype, and the investors are to believe all the hype, then this is a no brainer. BIOGEN PLEASE STEP UP AND APPLY FOR COMPASSIONATE USE FOR DEXPRAMIPEXOLE TODAY.

BE A RESPONSIBLE CORPORATE CITIZEN. MAKE A DIFFERENCE. ADDRESS PATIENT NEEDS. SHOW ETHICS IN CLINICAL RESEARCH. ITS THE RIGHT THING TO DO.

Tuesday, November 1, 2011

Attitude is the One Thing an ALS Patient Can Control

As many of my blog followers already know, I have had three different and distinctive 10 year careers. Started in College Athletics Fund Raising/ Marketing, then CPG Sales/ Marketing, and lastly Technology Sales/ Marketing. Like to to write about lessons learned and how they relate to my battle with ALS.

Have already written about the last two a few times, so today will switch gears and talk about College Athletics. I have been extremely fortunate to have been around four Hall of Fame coaches while an athlete, student, and co-worker. Two are indisputably the best of all time in their sport, Dan Gable (Iowa Wrestling) and Augie Garrido (Cal State Fullerton/ Texas Baseball). Two others are widely recognized as winners, leaders, and trailblazers in Hayden Fry (Iowa/ N Texas/ SMU Football) and Lute Olsen (Arizona/ Iowa Basketball). Today we'll look at Augie Garrido and the lessons I learned in the three years working alongside him.

Augie Garrido is now the winningest coach in the history of College Baseball with 1800 wins and counting. He has won five National Championships, three at CSF and two at UT. He has sent close to 100 players to the big leagues and touched the lives of 1000's more over the last four decades. He brings a swagger to a team, an edge, has incredible charisma, and is a top notch recruiter and evaluator of talent. More importantly, he is just flat out an "it" guy. Could have done anything he wanted to, anytime, anywhere, including managing in MLB numerous times, but he chose College Baseball.

Many stories resonate with me and are not widely known outside of Cal State Fullerton, but should be. They also have alot to do with the way I have approached obstacles in my career and personal life, including my battle with ALS. From 1958-1978, USC Baseball had won ten (10) National Championships. In 1975, they were coming off an unprecedented four (4) consecutive titles as well. CSF was an Orange County based commuter school just transitioning from a very poor Division II baseball program to it's first year in Division 1. Brand new coach Augie Garrido, in his first year with no facilities, a bunch of JUCO castoff players, and a $5000 budget, won a West Regional at USC and kept them from even being able to try and defend in Omaha! That was just the beginning of a legendary run. He gradually built up the program in the years following and ultimately won his first of three CSF National Titles only four years later in 1979. Won again in 1984 and 1995 also. Although he left in 1997 to go to Texas, the Titan program still reigns as a national power.

So how did a 26 year old guy with only two years of prior experience at Division II Cal Poly SLO build Cal State Fullerton from absolutely Ground Zero? How did he take a school with no resources, no tradition, and no money to a perennial Top 10 powerhouse that has stayed relevant even after his departure? Why did four of his former players and former coaches choose to continue the tradition and follow in his footsteps as the Head Coach when they could have easily gone somewhere else where it would be much easier to win along the way?

Attitude.

"Skip" as Augie was widely known, had a saying, "It just doesn't matter." IJDM there was no home facility. IJDM there was no budget. IJDM SC was only 30 miles down the road with all the resources and tradition in the world and CSF would have to go through them every year to get to Omaha.....

What did matter was attitude. Never let himself, his coaches, or his players dwell on what they didn't have.
He sold what he had. Great weather, fertile area for high school baseball prospects that could stay close to home, free education, a chance to play against the heavily recruited blue bloods at SC, Stanford, UCLA, etc. A chance to be on the ground floor of something big, maybe even a chance to maybe play professional baseball someday. By the time I came to Cal State Fullerton in the mid-80's, they had already arrived. The facility was still sub-standard and the budget still shoddy, but it didn't matter. IJDM. The Titans had a swagger and they intimidated everyone they played against before the first pitch was even thrown. The players and coaches to this day still view themselves as the blue collar overachievers. It's them against the world, and the world doesn't stand a chance. They don't win it all every season, but they believe they can win and compete for the title every year.

As an ALS Patient, I have adopted "It just doesn't matter" as my mantra as well. IJDM we have no timetable on how we will progress, IJDM that there is only one barely effective treatment available to patients. IJDM that the Neuros, Hospitals and Clinics after diagnosis can really only help make us comfortable with temporary treatments. IJDM that most people outside our immediate family and close friends are so busy with their own lives that they don't understand or feel our emotional pain. And the list goes on. All of those things are completely out of my control.

Like Augie, we should try to focus on the things we do have and not worry about those things we don't have or cannot control. We have our minds. We have our family. We have our friends. We have other pALS and cALS in the ALS Community, whose willingness to help others is unlike any other group of disease stakeholders in the world. We have many organizations and individuals willing to support us to the extent they can. We have some groundbreaking discoveries beginning to come our way with hope of new advancements for the future. We have some promising Clinical Trials in place and more on the way. And we have today.

Anybody can have a winning attitude if they are playing football today at Alabama or hoops at Duke. Having an ALS diagnosis is kind of like if you were with Fullerton baseball almost 40 years ago, Boise State Football ten years ago when they were playing in the 1-AA Big Sky, or Butler Basketball 15 years ago when they were a perennial loser and it all looked hopeless. What makes all three of these programs winners today? ATTITUDE!

If these programs above had stayed at the same level they were before, they would be the equivalent of Cal Poly Pomona, Northern Arizona, and Youngstown State on today's sports map. But they didn't worry about USC, BYU, or Indiana being in their backyard. They took it day by day, brick by brick, and made themselves something special. Any one of those days on their particular journeys I can promise you it would have been much easier to focus on the negative, listen to the naysayers, and not have tried at all.

Let's not worry about other larger diseases getting much more notoriety. Let's not lose sleep over not having that single national spokesperson that can make us all famous. Let's not worry about what has happened in the past. If we focus on the things we do have and those areas we can control, and make the absolute best of each of them each day, we can make ourselves into something special too. IT'S ALL ABOUT ATTITUDE!

Monday, October 24, 2011

One Man's Opinion on Technology Transforming ALS Research - Great Things to Come

It's extremely difficult as a patient to watch the limited progress made against ALS by the medical community. Almost 150 years since the disease was identified and only one lousy, very expensive medication (Riluzole) that may extend life only 60-90 days on average in some patients. If you believe the past predicts the future, we are doomed. If you think linearly and look at the experts constant reference to how puzzling and difficult ALS is to understand, you become disheartened. If you understand it takes 12-15 years on average for a drug to make it from concept to shelf, that is many times the average ALS patients lifespan after diagnosis.

Although an individual like a scientist or a researcher is an expert in his or her field of ALS Research, that does not mean they are an expert at technology-based forecasting. The common wisdom is to think linearly, and assume the pace of development and change will continue based on his or her expert knowledge of what is known "today." I am not a scientist, but am a technologist and a mathematician. Also like to explore new ways of thinking and challenge conventional wisdom. Thankfully in the past, some overcame the linear thinking that the world was flat, that blood letting was a great medical treatment, or even that women should not be treated equal or even vote. I believe that technology is just beginning to revolutionize modern medicine, and we are at the cusp of some major breakthroughs in the months and year to come. There is now almost monthly news of new discoveries, advances in Stem Cell research, promising new clinical trials, a recent FDA approval of the Diaphragmatic Pacer, an attempt to better mine data via the new ALS CDC Registry, etc. All cause for hope.

I have written elsewhere in this blog about the many technological advances that we have seen just in my lifetime. We math guys believe in Moore's Law, which says essentially that processing power doubles every 12-18 months. This makes the progression not linear (2/4/6/8/10), but exponential (2/4/8/16/32/64). It has been the basis for the incredible advances in Computers, Communications, and other areas too numerous to list. But it has not seemed to touch Medicine as quickly as some other areas. I believe that many factors, not the least of which will be the convergence of technology with economic potential, will conspire to ramp up discoveries and ultimately cure disease very rapidly in the future. It's been less than a decade since the Human Genome Project, and many believe that it will ultimately open the door to some incredible advances to come.

When you have been diagnosed with a terminal illness that runs heavily in your family, like me, it obviously colors your view. You are consumed with three things: 1) Trying to live every day to the fullest because literally some day in the not too distant future could be your last. 2) Hoping there will be a treatment and/ or cure that will be help you personally. 3) Hoping there will be a prevention, treatment or cure that can impact your immediate family and any relative that might get the disease down the road.

On the subject of hope, I recently finished reading The Singularity Is Near, by Ray Kurzweil, probably technology's most credibly hyperbolic optimist. Link Here. His first book, The Age of Intelligent Machines, written from 1986 to 1990, put forth his theories on the results of the increasing use of technology and very notably accurately foresaw the explosive growth in the Internet, among other predictions. He also forecasted the demise of the USSR due to new technologies, predicted computer chess software performance would allow a computer to beat the best human chess player by 1998. It occurred in May 1997.

Two decades later, many will view his newest book as wild speculation. Others will be consumed with the potential for negative outcomes to outweigh the positive. I view it as a potential road map of how technology and biology with intersect to cure all disease within the next three decades. By a guy that is a renowned futurist with a track record of amazingly accurate predictions.

In Singularity, Kurzweil says that humankind is at the threshold of an epoch ("the Singularity," a reference to the theoretical limitlessness of exponential expansion). Moore's Law is frequently quoted, and at the core of all discussion. We will soon see the merging of our biology with the staggering achievements of "GNR" (genetics, nanotechnology and robotics) to create unrecognizably high intelligence, durability, comprehension, memory and so on. The word "unrecognizable" is not chosen lightly. Kurzweil's argument is twofold. He argues that there will be virtually no constraints on our capacity, and he also tries to convince readers that such developments are desirable. In less capable hands, his speculative and bewildering variety of charts, quotations, playful Socratic dialogues and sidebars, would be easier to dismiss. But Kurzweil is a true scientist—a large-minded one at that—and gives due space both to "the panoply of existential risks" as he sees them, and the many presumed lines of attack he was certain to entertain. What's exciting and intellectually stimulating at the same time isn't the degree to which Kurzweil's heady and bracing vision fails to convince—given the scope of his projections, that's inevitable. It's the degree to which it all seems downright plausible.

Believe Mr. Kurzweil or don't. Agree with my views, or don't. Choose to be optimistic, or choose to be cynical. Be futuristic and hopeful, or linear and fatalistic in your views. Be optimistic and challenge conventional wisdom, or be dismissive and tell all that will listen that history repeats itself. It's all up to you and you are absolutely entitled to your opinion. I choose hope.

Just one man's opinion.

Wednesday, October 12, 2011

One Man's Opinion on Media Fragmentation and ALS - Do We Need a National Spokesperson?

All organizations are looking for a panacea, a message that resonates across the populace and draws them to your product or service in droves. They are also searching for that ideal spokesperson that reaches their targeted consumer and delivers that message in an emotional way to tie their brand to your heart, and ultimately your wallet.

I currently work in the Custom Analytics space in the Consumer Packaged Goods Industry, and help Fortune 100 companies decide how to market, advertise, price and promote their products and services. Billions of $$ are spent annually on TV, Radio, Price Promotion, Coupons, Billboards, Print, Sampling, Direct Mail, Sponsorships, Loyalty Cards, Viral Campaigns, Rebates, Digital, Mobile, Social Media and the list goes on.

Does all that advertising work? Yes and No.

The challenge - Media Fragmention. As recently as 1980, almost all U.S. homes received only four TV stations via their roof top antennas - ABC, NBC, CBS, PBS. PBS did not advertise. You probably read a morning and maybe even an afternoon local newspaper. Subscribed to a weekly magazine like Time, Newsweek, Sports Illustrated, or Good Housekeeping, saw an occasional billboard while driving to work, and listened to AM Radio or FM if you were really adventurous and your car received the signal.

The advertiser needed only to drop a network spot or two, or a print ad, and could reach almost the entire target audience very cost effectively. Many brands had celebrity spokespeople that had broad appeal and reached across demographic differences to influence almost everyone. You would be surprised as to how many of today's Top 20 US Brands were on a similar list thirty years ago. Unless you have an incredibly compelling product or service with mass appeal it's very difficult to move into this group today.

Circa 2011 and the landscape is vastly different. 500 TV channels are distributed without regard to geography via cable, satellite and the web. Content specific to almost any interest is able to be accessed live, via DVR, or on demand. Newspapers and other publications as we knew them are almost dead, etc....

This means the consumer is in almost complete control and can now go his or her entire life and only watch, read, and listen to what they want to see and hear. A Midwestern born sports fan like myself living in LA can ignore the Dodgers, Angels, USC, and UCLA. I now receive the same local media content delivered on the Twins, Vikings, Hawkeyes, and Wildcats as guy living in Minneapolis, Iowa City or Kansas City. I can also watch only Fox or watch only MSNBC and never have my political views questioned. With all the messaging and information being delivered the brain cannot process everything. We survive by selectively processing only a fraction of all the noise.

What does this mean for overall messaging and ALS Awareness and how do we reach a broader audience?

Regarding messaging, there are many ALS organizations out there but no one that singularly controls the message overall. Good news is that almost every group has a message related to Finding a Cure. As well they should. It's what all PALS demand, what we want to hear, and it has a broad appeal. Patient Care, Patient Services, and Public Policy are also all incredibly important as well. Especially if you are a PALS or a CALS. Just not as sexy and saleable to outsiders.

I applaud Angela Lansbury and Nancy O'Dell, and of course Jerry Lewis, for what they are doing and what they have done to contribute to the cause. No question there. Do they bring people to the cause that otherwise would not have participated? Absolutely. Do they make as big an impact today as they did 30 years ago? Not even close. Should the ALS organizations continue to solicit celebrity involvement? Of course. In the future, will some more well known celebrities or their spouses, parents or children contract ALS? Not many, but unfortunately, some will. Will those that do participate in ALS Awareness campaigns? Maybe, but it's a very personal decision and completely up to them. As much as any PALS or any ALS organization wishes they would, it's up to them. If they choose to support only local or regional ALS clinics or organizations, is that OK? Absolutely, with the power of the web and social media there are no organizational and geographic boundaries regarding the expansion of ALS Awareness. It's all additive, it's all good, and now it's all national. Any news is good news and keep it coming!

I am in my early 50's and can say that outside of the MDA Telethon, I have never watched one television show that Angela Lansbury or Nancy O'Dell has appeared in. Nor Jerry Lewis either since I was 10 years old either. No interest. On the other hand, Ex- Iowa Hawkeye and Minnesota Viking Wally Hilgenberg's story on HBO, or former Minnesota Twin Kent Hrbek's commitment to his father's legacy means more to me than any television star ever could because they were players on my favorite teams growing up. It takes a village and everyone is drawn to something different. Never have watched American Idol, Dancing with the Stars, or any Reality TV show for that matter. Just doesn't appeal to me. If you were advertising with ESPN, Big Ten Network, Fox Sports, NFL Network, 24, Law & Order and a handful of others you might reach me (if I did not zip through it). If not, it wasn't happening. Even if you are able to reach me, I better have a need for your brand, or a personal interest in your cause, or it will be tuned out and selectively processed.

It may be the top rated show of the ratings period, or a story about the saddest and nastiest disease or illness a person could have. Unless the show is of interest, or the disease was close to myself, my immediate family or a close friend it gets tuned out and selectively processed. Could be an incredible and heart-warming story to some and not matter at all to others. There are so very few brands, less causes, and even fewer celebrity figures today that resonate across all consumers that you can count them on one hand.

The traditional media landscape is costly and archaic for all but the most sophisticated advertisers. A top rated TV show today produces a much lower Nielsen rating share number than even ten years ago. With the possible exception of the MDA Telethon, there is not one ALS media related event that continually resonates with a broad audience. And we share that with 42 other, albeit very important, neuro-muscular diseases. Breast Cancer and HIV/ AIDS, among the diseases, have done by far the best job in gaining support and awareness. Both also have an exponentially higher current or potentially affected population than ALS, and an audience pre-disposed to hear the message through life experiences related to themselves, a relative, or a close friend. ALSA, MDA, the research organizations, and other various ALS groups are not sophisticated advertisers or marketers. A strong argument could be made that hiring outsiders with that expertise and finding an agency to help could be a great investment that would grow the pie and reach a broader audience. However, if they aspired to do so, it would require spending 20-40% of revenue on those efforts. To do it well (with no guarantee of success) would also necessitate a long term, multi-year investment to "build the brand." This would undoubtedly bring scorn from the PALS community who demand the lion's share of monies go to finding a cure and not "PR" or "Marketing." That leaves grass roots activities, networking, and publicity via social media as the best use of scarce resources.

If you believe the numbers, there are 30,000 in the US living with ALS at any given time. Only 50% - 75% of these folks are aware that they have the disease in the US and assuredly far less worldwide. Average onset age of 45-65, so maybe a third to half of those are domestic PALS are internet-savvy. But their kids are all web-savvy and act as a proxy for the other 1/2 - 2/3. The US is only 5% of the world's population so there may be as many as 600,000 living PALS worldwide. Let's say 33% of those know they have the disease (probably very high estimate but humor me). Internet penetration in all but Western Europe is probably spotty, but conservatively add another 30,000 to the mix. Now we have 50,000+ PALS with web access, another 250,000 caregivers, families, and friends, and now we are at 300,000 with access to the above. As many in these groups (unfortunately) turn over every 2-5 years, and internet and social media penetration continues to grow worldwide, the ALS communities penetration and reach will continue to expand.

This is good news. In fact, for a fairly uncommon disease like ALS, there is great news. The Internet, and as importantly Social Media, transcends geography, citizenship, race, gender, and almost every other defining characteristic of an individual. Content doesn't have to appeal to a broad audience or project huge ratings to make it online. It's free and allows those families with ALS to reach out to their networks of family, friends, and even acquaintances with news, updates, and requests for support. It allows sharing of research news, provides patients information and resources, and informs us of upcoming events at the push of a button.

It allows a home bound PALS in Europe to watch and participate in an Ask The Experts webcast, AND ask questions via chat streamed live across the globe from the campus of UCLA. It allows a person in California to be in a fALS Facebook group hosted by a fALS Advocate in Kansas City, along with fellow patients and family members across the nation the world. It allows the inspirational stories of former NFL players from Baltimore and New Orleans to go viral and become national and international stories overnight. It allows an individual PALS from North Carolina to lead an industry-wide discussion on bettering the new ALS/ CDC Registry and make it more rich and meaningful. It gives an Indianapolis PALS a forum to promote clinical trail participation for an exciting new study. It gives a notable Washington DC based fALS Advocate a vehicle to help families explore their ancestry and better understand known and unknown family connections with the disease. It allows the story of long-time PALS fighter and contributor, published in a local Santa Cruz, CA newspaper, to become widely known across the entire PALS community. I wish I had more time and space to single out the hundreds more like them that do these things not for publicity, but because they can, and it's the right thing to do. These PALS represent us and come from all walks of life every day to inspire and lead by doing. Let's continue to share stories and celebrate their achievements. After all, they are our celebrities and they are our spokespeople. And many of us wouldn't have it any other way!

The attached is only my opinion, and a record of my own experiences. Like any other post on this blog it is meant to share information. It's up to you to agree or disagree.

Monday, October 10, 2011

Driving with a Disability and Buying a Wheelchair Van - Trials and Tribulations

Had an interesting experience purchasing a wheelchair van a few months ago and thought I would share my experiences.

It had become extremely difficult for me to continue to drive on my own. I still had good use of my upper body, decent use of my left leg, and no use of my right leg with the exception of being able to slightly move the foot. Early in my diagnosis had only foot drop in the right foot. Got a carbon orthotic device to stabilize the ankle but that made accelerating and braking very difficult with a stiff foot. Tried to stay ahead of the game and looked into hand controls for my 2007 Honda Accord. Hand Control Tips.

While plentiful in supply, the required regulations make them very costly for an ALS Patient with no idea of how long they will be used as I progress. California law dictates that a "Certified Trainer" meet with you if you decide you want to proceed. They decide what you will need and are also required to "train" you on how to use the new device(s) after you obtain them from a "Certified Dealer" and have them installed. Estimated around $500 - $1,000 for a simple accelerator/ braking device mounted on your wheel, another $500 - $1,000 for the training and evaluation. The PALS is left to decide whether or not to spend the $1,000 - $2,000 on this based on guessing how many months you will be able to use it before arms and hands give out and render this useless. If you need to modify this again as you progress later on, you most likely will have to repeat the steps and costs noted above again. Finally, and if you chose to move forward, additional considerations include transferring in and out, when someone else utilizes the same vehicle the ease of temporary dis-engagement for them and re-engagement for you, and the cost of returning the vehicle to it's original condition after you can no longer utilize it and/or it's time to re-sell.

Ultimately, I decided to pass on the hand controls and kept driving on my own until very recently based on the ROI vs the unknown costs and timing. Extremely difficult transfers in and out of the drivers seat and a few falls convinced me to purchase a handicapped equipped wheelchair van a couple of months prior to absolute need. Glad that I didn't wait, and my wife is even happier.

My father worked in the automobile care industry for years and I grew very comfortable buying and selling vehicles over time. Many people grow very anxious during this process but fortunately I was never one of them. Relished doing my research (and the internet has made that even more easy recently), walking out (or sometimes pretending to walk out) of a few dealers, and feeling like I was in control of the process. Buying a wheelchair van is different. Since there is no Kelly Blue Book or Edmunds.com for information, you are pretty much on your own as to defining what is a good deal. There are also many model options.

Price Value Decision, New vs Used - Factors to weigh include ROI on a $5,000 to $60,000+ investment with timing unknown as to how many months or years you will benefit. Add in the fact are probably less than two years into the whole money pit that is ALS, and cash flow is not normally at its peak. I cannot tell you how many PALS I have seen buy the $50,000 brand new van and then are able to use it for only a year or two or even a few months. Your spouse or heirs have to then go through the same transaction anxiety of not knowing what the vehicle is really worth when selling it. Buying used does not diminish these anxieties but at least reduces the dollars at stake. Of course, buying new and under warranty for 60,000 miles pretty much gives you full piece of mind regarding potentially costly adaptive modification repairs. It's up to you which way to go as both options have many pros and cons.

How to Deal on a New or Used Van - As previously noted, there is no KBB or Edmunds to research price. You have to get down to pre-internet basics (even though you can use the internet to get them) and study up. Look online or visit dealers around your area or region and see what the going price is for comparable new or used units. Take any used vehicle you are serious about to an independent mechanic for evaluation. If the seller refuses, walk away. Most independent mechanics will evaluate a used vehicle in 30 minutes for a nominal charge, or even agree to do it for free if you do any subsequent repairs there. Ask fellow PALS, and also ask the dealer for referrals. When buying new, ask to see the dealer invoice, but be prepared to allow them a 10-15% margin over cost (vs 5-10% on a normal vehicle). These vehicles are pretty basic chassis but the adaptive equipment upgrades are specialized and costly so comparisons to a non-handicap version of the same vehicle is not applicable. A dealer has a reputation at stake and you should as ask for and check with past PALS referrals. See how the manufacturer warranties compare and the price of an extended warranty as well. When buying used, focus on the remaining warranty and look into the cost of an extended warranty if needed. These add re-sale value after the fact when transferable. And don't be afraid to let Dealer A know you are talking to Dealer B, C and D, it always works when played properly.

Model Options, Adaptive Equipment and Accessories - Toyota, Honda and Dodge are the main brands today to research. Do your research on available options and talk to current and former users before making any decisions. Take into account what you need today and also what you will need tomorrow as you progress. Buying one you can test drive vs shipping one in from afar, Side vs. Rear Entry, standard belt straps vs EZ Lock devices, base unit vs multiple accessories, actually replace the driver and/or passenger seat vs positioning in the middle, and the list goes on.

Sales Tax - Please beware (at least in California) that the sales tax is only applicable to the base chassis. That may be only $5,000 on a $20,000 used van or $20,000 on a $50,000 new unit. Don't get swindled.

Insurance - Many insurance agents are not used to handling handicap vans. Be careful that they understand the value of the adaptive equipment vs. the standard model chassis and will be able to measure depreciation and cost of replacement on those in case of claim. Insurance Tips.

Tax Deductions - After exhausting the 7.5% AGI standard some or all of a purchase may be tax deductible. See your CPA for details. Tax Tips.

Veterans Benefits - On a side note, I am not a veteran but wanted to make those who are aware that Congress recently raised the handicap vehicle grant to $18,000 vs $11,000. Veterans Tips. Depending on the PALS VA rating, the veteran can be eligible for this grant up to twice every four years. It covers $18,000 on the vehicle itself and much more regarding disability adaptive upgrades. One vet told me he could get a brand new $55,000 Toyota van for around $12,000 out of pocket. You will need to file an "Application for Automobile or other Conveyance and Adaptive Equipment (Under 38 U.S.C 3901-3904)." Please contact either the Paralyzed Veterans Association or the VA.

At the end of the day, I decided on a clean used 2003 Dodge Caravan base unit with 90,000 miles, no warranty, and very few options. Cost me about $20,000 including tax, delivery charge, license and fees. Has a raised hydraulic suspension, an automatic push button Braun Hydraulic side entry lift, a removed middle seat, standard straps, and that's about it. Has worked very well so far, knock on wood. Gets me from Point A to Point B comfortably and safely. Not the fastest unit in the lot, not the biggest, and definitely not the newest. Crossing my fingers on potential repairs going forward. Guess this vehicle buying decision mirrored almost every other that I have made over the years. Practicality, safety and operating cost have almost always led me to a bargain. After all, like my father always said, "since they are all used the minute you drive them off the lot, why not let the former owner pay for most of the depreciation." Why should I change now?

The attached is only my opinion, and a record of my own experiences. Like any other post on this blog it is meant to share information. It's up to you to use it or not, and how to proceed. Good Luck!

Friday, October 7, 2011

One Man's Opinion on the New FDA Approved Diaphragm Pacing System - Timing Challenges Abound

Many PALS were excited to hear about the NeuRx Diaphragm Pacing System by Synapse that was very recently FDA approved for ALS patients. The FDA granted a Humanitarian Device Exception (HDE). Will definitely help those with stimulatable diaphragms and experiencing chronic hyperventilation. Synapse estimates around 3,300 living PALS with both respiratory and intact phrenic nerves could benefit. Patient Q&A document can be seen here.

This typically outpatient procedure allows the patient to breathe using his or her own muscles for a longer period of time. Dr. Raymond Onders, co founder of Synapse Biomedical, started working with patients 15 years ago in Cleveland at University Hospitals Case Western. Early patients included Christopher Reeve. It has been used in partially and totally paralyzed non-ALS patients for quite some time with good success. A pair of clinical trials were conducted beginning in 2004 with ALS patients and produced the approval noted above. Link here.
Bottom line is I called Dr. Onders to learn more and inquire about my potential for qualification and the potential timeline to get an ALS Clinic affiliated hospital in Southern California to participate. Link here. He sent me a very nice note back and connected me with his staff. Wanted to share what I have learned from a practical, medical, and insurance standpoint at this time.

Practically, there is only one certified place to have this procedure done for ALS Patients today and that is with Dr. Onders' team at University Hospitals in Cleveland. See the current nationwide approved hospital and clinic map here. "The DPS is a Humanitarian Use Device approved by the FDA as an HDE the institution is required to obtain a local IRB review to treat patients with the NeuRx DPS(r) technology. This does not mean it is experimental or it will not be reimbursed by health insurance, it's just an FDA requirement as a HUD/HDE. "

What that means related to time is that before a new clinic can go forward with any surgery they need to have a full review by an individual clinics' IRB. The average time for a study to be approved is 90 days. That is 90 days from when the application is finalized and submitted to have it reviewed, revised, and presented to several committees, etc. As this study is related to a surgical procedure they anticipate it may be a more involved review and take longer than 90 days. Add these 90-180 days in as the earliest probability of any new clinic to be up and running and we are looking practically at Spring 2012 for the initial roll out and probably end of 2012 for reasonably strong geographic coverage. (On a side note I am unclear as to the timing of the half dozen or so clinics that previously were involved in the clinical trials. In theory, their approval times should be quicker but I have been unable to confirm).

Tough call as a patient that is progressing as to whether to wait to do this locally or travel. Plan on 5 days in Cleveland as an outpatient. Travel by air or car, hotel rooms, food, local transportation if you fly, etc. Add in a caregiver for some. Total cost for me (not counting the surgery) coming from LA with a caregiver would be around $5,000. Then if there are complications or follow up the costs and inconvenience get even larger. Leaning heavily on waiting to do this locally and crossing my fingers on timing and qualification, but may not have that option.

Medically there are a few things they test to see if you can benefit. Goal is to keep the patient breathing with his or her own muscles for as long as possible. Augie Nieto went on this device shortly after diagnosis and made it six years before getting a trache. The tests run to evaluate whether the diaphragm is stimulatable are a Fluoroscopy of the diaphragm (simple scan) and a Phrenic Nerve Conduction Test (not for the faint of heart). It is important to determine if the diaphragm is stimulatable prior to beginning the procedure. The PCNT shows diaphragm movement and positive response. For Diaphragm Pacing to help, the diaphragm must respond to the electronic stimulation.

Insurance-wise, I was told even prior to FDA approval, the hospital has performed many these procedures on ALS patients that were approved by Medicare/ Private Insurance. At least 50% were covered. Now that it is FDA approved, they anticipate the coverage success rate should grow significantly. Link here.

I spoke to the caregiver of an ALS patient that participated in the clinical trial at Stanford University and she highly recommends the procedure. Felt good to confirm my thoughts on this by speaking with an actual patient. Highly recommend anyone considering this procedure (or any other of significance for that matter) to do the same. There are HIPPA laws that prevent the clinics, MDA or ALSA from giving you patient information directly. But they can call the patient or caregiver on your behalf and ask if they would be willing to speak with you and answer some questions. Almost never has a patient turned me down and I am always willing to return the favor when asked as well.

I aggressively approached the lead ALS Neurologists at both ALSA certified Cedars Sinai and MDA certified UC Irvine. Ask both of them to look into it and they agreed. If you are interested in having your local clinic participate, suggest you do the same. Make sure to include the potential surgical team leader at the clinic as well. Tell them the contact person at Synapse Biomedical is Steve Annunziato. He can be reached at (440) 774-2488 or sannunziato@synapsebiomedical.com.

University Hospitals in Cleveland contacts:
MaryJo Elmo
Nurse Practitioner
Case Western Reserve University
University Hospitals of Cleveland
Cleveland, OH
United States
Phone: (216) 844-8594
maryjo.elmo@uhhs.com

Raymond P Onders MD
Director of Minimally Invasive Surgery
Case Western Reserve University
University Hospitals of Cleveland
11100 Euclid Avenue
Cleveland, OH 44106
United States
Phone: (216) 844-5797
raymond.onders@uhhs.com

You Tube Video (10 parts) with Dr. Onders presenting his findings from November, 2009.

My intent is not to criticize this procedure in any way, nor to criticize the "system." I'll leave that to the many others that undoubtedly will weigh in with their negative or positive opinion, as they should. It's simply to provide neutral, important and practical information from a patients point or view. Like all data or information, it's up to you how to use it or not use it. It's your body, your life, and it's completely up to you.

Just One Man's Opinion.

Thursday, October 6, 2011

"Innovate or Die." ALS Research and the Tech Sector Have Many Things in Common.

With the passing of Steve Jobs yesterday, it gives pause for me to reflect on my own 30 year career. There have been some amazing advancements in technology that have enriched people's lives over that time. And I was lucky to be in on the ground floor of a few things that are now accepted as required and necessary.

Spent three years at Philips Consumer Electronics in the very early 90's. HDTV was being developed in the research lab and we salespeople were evangelizing how it would revolutionize how consumers watched TV. A few early adopters moved heaven and earth to get their hands on a $10,000+ unit before there was much programming or even a delivery mechanism for content. Today there are units for under $250 with sparkling quality.

We introduced the world's first DVD Player to the consumer market, and I was lucky enough to be one of the four guys that headed up the retailer roll out. For you fellow geeks and semi-geeks it was called Compact - Disc Interactive (CD-I) and was 5-10 years ahead of it's time. We had invented the CD and that had just started to take hold so it was time to evolve into the DVD. CD-I cost $1000, had very little software, Photo CD capability, and a high end audio CD player. Today there are quality units for well under $100 available and most people have 2-3 in their home.

Philips asked me to help them sell a new product in 1990 that only Sony and a couple of other Japanese companies had on the commercial market. Large screen 330 pixel CRT video "replay" boards then only existed in a handful of sports venues around the world and cost $5 - $10 million to install. Spent 18 months flying all over god's creation evangelizing the need for more of these. The New York Islanders, Meadowlands Racetrack, National Aquarium in Baltimore, Crystal Cathedral, Tennessee Volunteers, and Miami Heat became customers. 20 years later there are dozens of manufacturers, installations can be had for well south of $1,000,000, and they have incredible resolution and clarity. Every college and pro sports venue of any significance have installations, and even some high schools.

Philips owned a Canadian-based company called Head Start. This group was one of the 4-5 largest PC manufacturers for the consumer market at the time. IBM and Packard Bell were one and two I believe. Anyway, the units we were selling when I got there had a 186 processor, no windows operating system, a 4.8 baud fax modem, floppy disks, and we used a software called PROFS for e-mail. If you were lucky enough with this $5,000 unit to not have your phone line drop, you could e-mail the word "Hello" to someone in under 10 minutes beginning to end. Today my I-Phone has exponentially more features, speed and power and costs a fraction.

ALS Research today is much like the Consumer Electronics industry was in the late 80's and early 90's. They have to sell the sizzle before the steak to get investor and donor dollars. Press releases on new discoveries are shepherded almost monthly. Promising stem cell discoveries are in the news continually. Researchers that never before even spoke are sharing information (anyone ever think Microsoft would have partnered with Apple or Philips with Sony?). Private Equity and Big Pharma paying more attention to ALS Research is mandatory and this is all part of the game that the industry is adapting to. Never before has the phrase "Innovate or Die" had more meaning.

The last 20+ years prior have been like black and white TV (Riluzole) with three channels, the same shows year after year, and not much change. However, I have seen more activity in the past two years than in the last 20. Many of us are frustrated that it takes 10-15 years from concept to market with today's drugs in the marketplace. Innovate or Die.

For a current ALS patient like me, our best hope lies in drugs already in Phase 2 and 3 Clinical trials. Much like the early adopters in the Consumer Electronics industry drove innovation, we PALS have to participate in the these Clinical Trials to move the timeline forward as well. The system never produces things as quickly as those in need want them to. But we have no choice but to do what we can and allow it to innovate. It is impossible to connect the dots looking forward, it only works in retrospect looking back. You have to have faith and believe that these brilliant and dedicated researchers will ultimately succeed. Innovate or Die.

Somewhere very soon tomorrow, today, or maybe 5-10 years ago, some researcher may well discover, or will have already discovered, the cure for ALS. He or she doesn't know it yet, we PALS don't know it, and the investment community hasn't figured it out yet. But it's there...trust me...it's there!

Someday my son and daughter (and maybe even me if I am incredibly lucky), as we celebrate the cure, will look back at this blog entry and see that these comparisons really did prove to be prophetic.

In closing, here is a link to Steve Jobs delivering his commencement speech to the graduates of Stanford University in 2005. In it he talks about getting fired from Apple in 1985, life & death. Truly inspirational!

Innovate or Die....We choose to innovate!

Tuesday, October 4, 2011

fALS National Summit To Be Held March 13, 2012 in Washington D.C.

ALSA just announced the establishment of a new fALS Summit for 2012. This half day meeting will take place immediately prior to the 2012 ALSA National Advocacy and Public Policy Conference in Washington D.C. on May 13 beginning in the morning.

It's going to be great to be able to interact with our peers, and not having to choose to attend a fALS breakout during the larger meeting over another important topic.

They are soliciting topics and comments for meeting content by e-mailing fals@alsa-national.org. Just a few that were suggested at the UCLA fALS Meeting a few days ago were the following: Clinical Trials 101, Clinical Trials for fALS, Fast Tracking Successful Drugs/ Compassionate Use, Discovered Genes, Auto Dominant vs. Recessive Genes, Genetic Testing and Counseling, Ancestry Research, Discussions with Children/ Relatives/ Co-Workers/ Friends after Diagnosis/ Financial Planning, Health and Life Insurance Disclosures and Planning, Volunteer and Advocacy Opportunities, Living with ALS. This list is by no means all inclusive, just a starting point. Please weigh in!

My personal view is this is a very positive step and way overdue. I am awed by the contributions from people like Augie Nieto with Augie's Quest and the MDA, and Dean Rasmussen, the father of ALSA Advocacy. These individuals are not from Familial ALS Families and have still made huge, long-term impacts on the fight for a cure.

Their actions should motivate those of us that are in the fALS community. We all need to step up and participate in the cause in anyway possible. That may mean participating in clinical trial studies from our healthy relatives, volunteerism, fund raising, advocacy, or any other contribution you can muster. After all, it's our parents, children, siblings and relatives!

Pick an organization, any organization. Maybe it's one of the two national full service organizations - ALSA or MDA. Possibly one of the many researchers like ALS TDI, Packard, Northwestern, Mass General, Emory, Methodist, or UCSF. Groups that raise money for one or two research entities like Augie's Quest, Project ALS, Les Turner and the list goes on. Many are very worthy and even though there is some redundancy in their efforts I have come to view their overall contributions as cumulative. When you are part of a family with a hereditary terminal illness that is very prevalent it is very easy to be negative. Let's challenge these organizations to improve in a positive way as well and be as efficient as possible, but focus as individuals on things we can control.

E-mail 2012 fALS Summit suggestions to fals@alsa-national.org.