Why Are We Still Paralyzed?

It’s a pretty basic question for a Cure Warrior.  As we ponder the multifaceted answers to that question, we begin to grasp the enormity of the task in front of us.   What are the biggest challenges?

1. Scientific: Figuring out how to regenerate the appropriate pathways that will bring about functional MOTOR recovery in the chronic injury.  As we begin to experiment with therapies in humans, we’ll then need to identify the best DELIVERY method(s).
2. Translation: Okay, we’ve had success with a couple of therapies in the laboratory.  How do we raise the money, get regulatory approval, and enroll enough patients to run a human clinical trial?  As a case in point, the Geron trial proved to be a massive and ultimately failed undertaking.  They ran out of money and couldn’t enroll sufficient patients before they could prove much of anything.
3. Marketing: Any investor looking to bring a therapy to market had better have an excellent business plan, or the therapy will never reach widespread use.  We have seen this play out with the introduction of several neurotechnology devices.  Many of these tools have proven to be beneficial for clients, but implementation is hindered by lack of awareness and/or failure of insurance companies to cover.
4. Apathy: The SCI community suffers from a lack of focus and energy when it comes to pushing for curative therapies.  We all know how living with SCI drains one’s physical, financial, and emotional resources.  And to date, we haven’t seen anything in the way of a tangible scientific breakthrough to galvanize the troops.

How can you or I address these challenges in a meaningful way?  First and foremost, and I know I’m repeating myself, STUDY THE SCIENCE!  For starters, you’ll need to understand the terminology.  The more reading you do, the more researchers you talk to, the more leaders you network with, the more you will get comfortable in the conversation.

Where to start?  Check out the U2FP Clinical Trials Table for continuous updates on what’s happening in the field and an introduction to acronyms.  Read our lay-friendly reports from recent lab visits with top researchers.  Join us at the Working 2 Walk Symposium in Irvine, California, this November.

We need people with knowledge and passion to join this fight and commit to the long-term.  If we can invent the atom bomb, if we can put humans on the moon, if we can map the human genome, surely we can repair the injured spinal cord.  It’s all about priorities.  Making a difference starts with us, and I look forward to the day when we don’t have to ask the question, “Why are we still paralyzed?”

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.

Why Working 2 Walk

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.