Posts Tagged ‘advocacy’

Our Brother’s Keeper

March 4, 2012

Several years ago I read a book titled “His Brother’s Keeper” by Jonathan Weiner.  It’s the true story of one man’s race against time to try and find a cure for his brother, who was stricken with ALS at the age of 29.  The book is subtitled, “One Family’s Journey to the Edge of Medicine”.  Indeed, this is where we must travel if we’re going to solve the neurological puzzles of paralysis from spinal cord injury.

As a writer, Weiner has an amazing ability to explain the complexities of neuroscience in terms that can be readily understood by a lay reader.  He also does a terrific job of portraying the impatience and urgency that patients and family members feel when confronted with the glacial pace of advancing therapies from bench to bedside.  The story repeatedly raises the philosophical question of how best to advance science, and towards the end quotes Art Caplan, a bioethicist at the University of Pennsylvania:

“Should the pace of medical research be determined by people desperately afflicted and their kin?  Is that the best way to move the science?  My argument would be no.  Just as it’s hard to do the best science when you’re heavily invested and have a financial interest in what’s going on, it’s very hard to interpret results when your vision is completely clouded up by love of your subject.”

Caplan’s point is well taken.  In our own SCI community we have seen many patients spend tens of thousands of dollars on unproven therapies that provide little or no benefit.  At times advocates have allowed passion to drive their research investments rather than reason.

On the other hand, to my knowledge Caplan is not living with a debilitating or life-threatening condition.  It’s frustrating and tiresome to sit in your wheelchair and listen to scientists, funders, and regulators counsel patience.  And of course we have the problem of our big-name charities losing sight of their initial purpose (see my previous post, “Chase the Vision, Not the Money”).

It’s time for the stakeholders in the SCI cure effort to keep watch over each other:

  • For researchers to share their scientific results openly and honestly;
  • For advocates to educate themselves about the science of regenerative medicine;
  • For charities and patient advocacy organizations to operate with transparency and purpose;
  • For regulators to consider the voice of the patient when evaluating risk and reward;
  • For those living with SCI to speak up and put a face on spinal cord injury.

Kudos to Dr. Keith Tansey for writing so eloquently on this subject here.  I met Dr. Tansey at Working 2 Walk in 2011, and I know that he is a compassionate professional who understands the frustrations of those living with SCI.  He, too, believes that the science will advance more quickly and safely if we as a community work together, with ongoing, open dialogue and a sense that we are “Our Brother’s Keeper”.

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A Revolution of Empowerment

November 17, 2011

In my opening remarks at Working 2 Walk, I spoke about the legacy of Justin Dart.  If you don’t know the name, Justin Dart is widely considered to be the Father of the Americans with Disabilities Act, and he was a lifelong disability and human rights activist.

Mr. Dart was stricken with polio at the age of 18, and this life-altering event set him on a remarkable life journey.  A series of encounters with other polio patients, with the writings of Mahatma Gandhi, and with 3rd world “rehabilitation” centers charted a course that eventually led to passage of the ADA.

Throughout his life of advocacy, Justin Dart embraced the principle of inclusiveness and shared his vision of a “revolution of empowerment” – a revolution that would “eliminate obsolete thoughts and systems”, and give every human being the right to develop his/her capacities to the fullest.  Shortly before his death in 2002, he published a manifesto of extraordinary wisdom and lessons for the future, “Toward a Culture of Individualized Empowerment” (it’s a worthwhile read).

One of the most humbling and frustrating aspects of paralysis is the loss of power, and in many cases, independence.  If Justin Dart were alive today, I have no doubt that he would join our effort to revolutionize the way we look at paralysis.  To teach the world that it is a curable condition, that we, the grassroots advocates, the cutting-edge research scientists, and the pro-active investors, can empower ourselves by organizing, educating and advocating until a cure is achieved.

On another note, this week’s news that Geron is dropping their clinical trial in spinal cord injury shocked and angered much of the community.  Personally, while this development is disappointing, I don’t find it surprising.  As I’ve said before, achieving marketable therapies is business, not personal.  We can take some positives out of the fact that Geron made an enormous investment to demonstrate that hESC’s could be used safely in humans, a step that has hopefully paved the way for future therapies to pass through the regulatory process more quickly.


Enough With the Rats!

October 30, 2011

Working 2 Walk in Rockville was such a rich experience, I’ve had trouble deciding what to write about first.  For today, let’s look at the message of our international advocates, all of whom traveled great distances to meet the scientists at W2W and express an urgency to make the leap from working on the spinal cords of lab rats to those of humans.

Martin Codyre (Ireland), Harvey Sihota (United Kingdom), and Dennis Tesolat (via Skype from Japan) spoke frankly about what it’s like to sit in a chair and observe:

  • Promising therapies such as cethrin and hypothermia that never make it to Phase III trials because of financial and regulatory hurdles;
  • Large SCI organizations that use the word “cure” in their promotional materials, but spend relatively little on restorative research;
  • The lack of research attention paid to chronic injuries, when arguably that population has the greatest need and potentially the greatest return on investment;
  • A perceived lack of collaboration, commitment, and coordination amongst the stakeholders to push a product to market.

Of course it’s our goal at Working 2 Walk to motivate members of the SCI community to move beyond observation and into action, which became the focus of a lively exchange between the panel members and audience.

A few of the more powerful comments:

“I think that what we’re going to do is take up our own part of the job. What is it? If you’re paralyzed and you have a part time job, your full time job is moving this along. If you’re paralyzed and you have a full time job, your part time job is moving this along.”

“A lot of us are speaking about hope, and that’s awesome, but we need to talk about action.  I heard Jerry Silver say ‘I’m fine.’  Well, I’m happy that you are fine, but I am not fine.  I’m not here to criticize, but I want to know how we get well. Let’s start with a goal: we want clinical trials in two years and manage toward it.”

“We could be much further along toward a cure, which I define as getting out of the chair. We can’t be happy just with bowel and bladder, not that we all don’t want that. We have to take what we can in the short term but look toward the long. We need to push for a big change, or we’re all going to die in these chairs.”

Working 2 Walk itself is an effective catalyst for change, bringing together scientists who in the presence of consumers may be more motivated to collaborate, building the knowledge base of advocates, and creating unique networking opportunities.  But more needs to be done on a year-round basis.

Translation of therapies from the laboratory to the clinic is often called “The Valley of Death”.  In a recent Science Daily article about stem cell treatments for spinal cord injuries, Dr. Michael Fehlings noted that “At this time, a strong patient advocacy base would likely help provide momentum to help translate current research into clinical applications.”  Let’s get going.

It’s Business, Not Personal

August 25, 2011

Let’s face it.  Finding curative therapies for paralysis is a business every step of the way.  It starts in the laboratory, where a research team has to:

  1. Sell an idea that will earn a grant to secure funding for lab equipment and personnel;
  2. Sell the progress of their laboratory research to raise more money until a hoped-for breakthrough is achieved;
  3. Sell the story of a unique discovery to a peer-reviewed journal for publication;
  4. Sell the science to an investor who will start the process of bringing it to market.
If and when a potential therapy goes to an investor, another cycle begins.  The investor has to:
  1. Raise the money to fund a clinical trial (millions of $$$);
  2. Write a comprehensive proposal to meet FDA standards for approval;
  3. In many cases negotiate with the FDA for months to years over therapy details – dosages, method of delivery, patients accepted, etc.  In the eyes of the FDA, many of the newer cell therapies are moving into uncharted territory, and they are acting with extreme caution;
  4. If the first phases of the trial are successful, continue to raise money to fund later phases.

All of the above has taken several years and an enormous amount of time, effort and money to achieve.  And at this stage the therapy is still not available to patients.  If in fact it has shown clear benefit and the FDA approves, what’s it going to cost the consumer?  Will insurance pay for it?

Seattle Genetics just made the headlines by setting the cost of its new drug for lymphoma at $100,000 for one course of treatment.  Bone Marrow stem cell transplants, which have been available for decades, can cost anywhere from $50,000 to $500,000 with many variables in insurance coverage.  Regenerative therapies for spinal cord injury will not come cheap.

When you’re living with paralysis on a daily basis, it’s easy to get frustrated with the slow pace of progress.  At the same time it’s important to understand that moving a therapy from bench to bedside can be just as complex a problem as repairing the spinal cord.  Once you grasp the process, it’s easier to decide where you want to direct your fundraising and advocacy efforts.

Why Working 2 Walk

July 29, 2011


Last week U2FP announced our lineup of confirmed speakers for this year’s Working 2 Walk Science & Advocacy Symposium.  It’s an impressive group and features the names of scientists and business leaders who have been making news in the world of SCI cure research over the past year.

Back in 2006 when we organized our first conference, we had no idea whether anybody would come.  But as we started planning, we gave a lot of thought to picking an appropriate name.  We didn’t call it “Wishing 2 Walk” or “Hoping 2 Walk” or “Dreaming 2 Walk”; we chose “Working 2 Walk”, because we believed that people living with a spinal cord injury could roll up their sleeves and contribute to the cure effort.

Our purpose at W2W is to bring together all of the stakeholders in the march toward cures and create opportunities to exchange knowledge, strategies, and priorities.  Our research scientists continue to work hard at solving neurological problems at a molecular level.  Biotechs have begun to invest in promising science and the clinical trial process that will bring it to market.  A handful of advocates are opening the doors of communication that will hopefully lead to enlightened decisions on the part of policymakers.

If you’re sitting in your wheelchair wanting your healthy body back, you may be thinking that none of this is happening fast enough.  You’re right, but unfortunately, bringing cures to reality is not just about the science.  It’s just as much about the financial and regulatory hurdles that must be overcome, and I think this is where patient advocates have a real opportunity to make a difference.  We’ll talk about how at Working 2 Walk.


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