Home for the Holidays

On December 1, 2002, my son suffered a C6/7 spinal cord injury when a wheel came off an oncoming truck and landed on top of his pickup.  9 years later, having graduated from law school and secured a full-time job, he came home to celebrate the holidays with his family.  Together we reflected on the remarkable journey he had taken to work his way back to physical, emotional, and financial independence.

If there is such a thing as being lucky in the wake of a spinal cord injury, it applies to my son.  Had his neck broken just a few millimeters higher, he would not have the use of his hands or arms.  He used every ounce of his energy and determination to achieve his goal of independence, but still could not have made it without the extraordinary support of his family, friends, community, and numerous strangers.  Such good fortune does not fall to many who are stricken with SCI.

While we celebrate and take pride in his accomplishments, we know that he represents the minority of those living with SCI.  According to the National SCI Statistical Center, at 20 years post-injury, only 35% of persons with a spinal cord injury are employed.  This statistic is not just a function of injury level; the employment rate for people with paraplegia is only slightly higher than for those with tetraplegia.  There are numerous social, psychological, and economic barriers that conspire to keep persons with SCI out of the work force.

The unemployment rate is but one of many “invisible” secondary complications of spinal cord injury.  While the media is full of “inspiring” stories about people living with SCI, much less is said about what goes on behind the scenes.  No one wants to start a pity party, but if we’re going to achieve “cure consciousness”, we need to speak the truth about:

• the loss of bowel, bladder & sexual function;
• psychological trauma & suicide;
• loss of time, productivity, and often independence;
• a host of other issues that accompany spinal cord injury.

For every heartwarming story in the media, there are hundreds more untold about people and the daily realities of SCI that no one ever sees.  The public is often led to believe that one can achieve recovery through hard work and determination alone; if this were true, the majority of survivors would be up and walking around.

I am extremely proud of what my son has accomplished since his injury, and at the same time appreciate that it was made possible by some good fortune and a remarkable support system that augmented his efforts.  I also know that he would readily embrace the opportunity for regenerative therapies that could give him back a healthy body.

A Revolution of Empowerment

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!

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.

The Devil is in the Details

With less than 3 weeks to go until the Working 2 Walk Science & Advocacy Symposium, my head is spinning with the endless details that need to be addressed.  Making sure everyone is registered properly, compiling and proofreading the event program, keeping the website updated, communicating with our attendees and supporters, talking with the hotel about catering and audio/visual requirements, and the list goes on – connecting all the dots keeps W2W on my mind 24/7.

Everybody should take a turn at event organizing at some point, if just to gain an appreciation of the minutiae that need attention every step of the way.  I’d venture to say there are similarities with trying to get the nervous system re-organized after a spinal cord injury.  Talk about connecting the dots!

Laboratory research has produced a number of theories about the best way to go about restoring function lost to paralysis.  Our current real-time therapies include adaptive equipment, intensive exercise, electrical stimulation (FES), and locomotor training, all of which have shown the ability to enhance recovery.  To date, though, none of these could be considered a cure.

At Working 2 Walk we will hear a variety of theories about the best way to go about achieving curative therapies, including:

• nerve bridging enhanced by chondroitinase to promote regeneration;
• the use of biomaterials as part of drug and cell therapy treatments;
• the role of neuroplasticity in repairing the nervous system;
• lab results and the first stage of clinical trials using different cell therapies.

Slowly but surely we are assembling the pieces of the puzzle that will one day fit together and provide answers to the complex problem of curing paralysis.  As one scientist said to me a few years ago, “Most people believe that cell therapies will offer solutions for people with SCI; the devil is in the details.”

It’s Business, Not Personal

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.