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.
Thursday, November 10, 2011
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!
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!
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