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.

Skin in the Game

One of my favorite fellow advocates, who is quadriplegic, often uses the term “skin in the game” to describe his commitment to achieving cures for spinal cord injury. No less a renowned figure than Warren Buffet coined the phrase, referring to a situation in which high-ranking insiders use their own money to buy stock in the company they are running.  The idea behind creating this situation is to ensure that corporations are managed by like-minded individuals who share a stake in the company (Investopedia).

When it comes to making policy and delegating research funds, the individuals who have the most “skin in the game”  are those directly impacted by a disease or condition.  Unfortunately, too often they are not part of the conversations that lead to decision-making.  At U2FP this issue is rising up our priority list as we watch the regulatory environment strangle medical innovation.

History does provide us with at least one outstanding example where patient advocacy led to expedited treatments – the AIDS community’s response to the 1980s epidemic and their subsequent scientific and political involvement.  Currently, the California Institute of Regenerative Medicine (CIRM) has a governing board with representation from science, industry and patients.  One of the board’s members, Duane Roth, has written a convincing argument for the participation of patient advocates in his essay, “A Third Seat at the Table”.

One of our outstanding SCI community advocates and bloggers, Don Reed, also sits on the CIRM oversight board.  In case you haven’t heard, CIRM recently awarded $25 million to Geron Corporation to support clinical development of GRNOPC1, the cell therapy being used in Geron’s Phase I clinical trial for spinal cord injury.  For a fascinating report on the behind-the-scenes negotiations that led to the decision, read Don’s blog entry, “California Versus the Valley of Death”.  The story is a testament to the power of patient advocacy.

Learning From Others

I have been on the road for the last couple of weeks.  My first stop was Washington, DC, where I attended the One Voice Advocacy Conference hosted by Parent Project Muscular Dystrophy (PPMD).  PPMD was founded 15 years ago by Pat Furlong, whose 2 sons had been diagnosed with Duchenne Muscular Dystrophy.  While she eventually lost her sons to the disease, Pat continued to build PPMD into the powerful advocacy organization that it is today.

PPMD serves as an admirable model for the spinal cord injury community to follow.  Duchenne is a rare disease that strikes boys almost exclusively, with diagnosis at age 3-5 and average survival of 10-15 years.  It is an exhausting and heartbreaking experience for families to endure, yet in the midst of this journey parents are finding the time and energy to advocate for their sons.

The conference opened with testimony from Duchenne family members.  Representatives from the NIH, FDA, and CDC were in the room to listen, offering an extraordinary opportunity for the advocate community to share a perspective “from the trenches”.  The families did an amazing job of communicating their concerns, their daily life experiences, and their sense of urgency to find therapies that will prolong and improve the lives of Duchenne patients.

Parents spoke with a passion borne from experience; at the same time they demonstrated an impressive level of knowledge about the disease, the progress of research, and the clinical trial process.  Clearly they had spent a lot of time educating themselves, and as a result spoke from a position of strength to the policy makers in the room.  They asked and answered questions with equal aplomb, and I came away with a couple of lasting impressions to inform the work of U2FP:

1. Advocacy by community members can make a HUGE difference in the progress of research, but it must be targeted and well-informed to be effective.
2. We must add the community voice when decisions are being made about where to allocate research funds, be they public or private.

For more information on PPMD’s programs and how they are making a difference, please visit their website.  I’ll write more about my travels in the next blog post.