Working 2 Walk

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Congressional Briefing

Posted by katewillette on April 15, 2008

Here’s what it’s like . . . you reserve a room in one of the office buildings near the capitol.  You make sure there’s coffee and tea plus the usual tray of cut-up fruit – maybe some muffins or sliced bagels.  Neat rows of bottled water and juice.

Your presenters show up with their laptops, wearing dark suits and ties, carrying their notes in their pockets.  (Dark suits and ties– or heels, for those of us who are not male –are required attire here.  It’s very obvious who the regulars are in any room.)  You hope for some congressional aides and maybe even the press.  We have 6 scientists here this morning, plus several people in chairs and more than a dozen activists.

We start on time, more or less, although there are almost no guests from the congress or the media.  Possibly the problem is that this is the day the pope is in town . . . possibly we’re too early in the day . . . possibly –as usual—there are too many other priorities.

Wise Young gets up to introduce the session and name the speakers . . . he’s talking as if there were a crowd of aides here.  First to speak is Dr. Pearse from the Miami Project; he explains what MP is – an organization devoted to curing spinal cord injury.  He’s just said this is a very good time to be working in this field because there are so many extremely promising therapies.  The issue, he says, is that we need funding to bring the research forward . . . many young scientists are considering leaving not just sci research but science itself because they can’t do their work.

Stephen Davies gets up to tell us about two new and exciting therapies.  He’s showing his slide that has the axons straggling toward the injury site and being stopped by a scar.  His strategy is twofold – to get rid of the scar and to encourage the axons to grow past it.  His lab has discovered that there’s a substance called Decorin that breaks down the scar tissue in an injured cord.  What about the business of getting axons to grow?  An NIH-funded group from the University of Rochester isolated a type of cell called a glial-restricted precursor that can be coaxed into forming another cell called an astrocyte.  The combination of astrocytes and Decorin is an extremely promising therapy, and Dr. Davies wants very much to bring it clinical trials, hopefully (since he’s an academic and not a businessman) through the NIH.

Less than 10% of SCI grant requests are being funded.  The breakthrough that Dr. Davies achieved with his astrocyte cells was originally funded by the NIH . . . but it’s likely that under current conditions his application would not have been funded.  We must assume that there are plenty of grant applications currently sitting in drawers, unfunded.  This is science that could be done but isn’t.

Dr. Simon Archibald gets up to say that we’re NOT looking now for the way to restore function through therapies, because we have therapies that have been shown to work –but instead how to choose the best combination of those therapies.  We’re at a critical point, where the process of getting from labs to patients is finally underway.  It must be funded.

Dr. Mark Hurtt, (who is a businessman) wishes us a good morning and addresses the clinical trials and the fragmented research infrastructure.  “My job is to find technologies, pull them out of the labs, do the work to meet regulatory requirements, and then get them to the patients.”  His company’s product is called Cethrin . . . it’s the drug that’s furthest along in terms of getting it to patients.  This was given in a Phase I trial to patients with complete spinal cord injuries, and the results were very encouraging.  Two of their patients went all the way from ASIA A to ASIA D.  The company is called Alseres, and it’s based in Boston; they have a whole suite of pre-clinical projects.  Dr. Hurtt is the one who chose to work with Cethrin.  There is no turnkey research structure they can turn to, as there would be in cardiovascular research, or almost any other kind.  (More on this later.)

Mark says: “I need one clinical trial network.  I need one budget with that network.  I need one institutional review that measures the efficacy of my trials.

“We own Cethrin.  We have a Phase IIB trial starting in 2008, and we’ll be doing a Phase III trial.”  (I get excited all over again listening to him; this drug works.)

Wise gets up again and makes concluding remarks . . . The CDRPA is made to provide the infrastructure – it takes years to build and small biotech companies don’t have the means to do it.  He brings Melissa Pitts up to talk. She’s a PT with twin 7-year-old sons, one of whom is paralyzed due to a C6-C7 injury sustained during birth.  She thought she understood what it meant to live with a disability; she’d been working disabled patients for years.  She learned.  She learned both patience and hope.  There are treatments within our reach; she’s been following the progress carefully, as a scientist and as a mother, for all these years.  Here’s what she’s learned:  the scientists are just like her old PT patients in the sense that they are often stopped dead in their tracks because of money.  Your insurance has run out, your funding has run out, you’re done.

She says, through tears:  I believe I will see my sons walk together.

Wise gets up to take questions.  Tricia asks why doubling the NIH funding is not enough.  He replies that in 1995, sci research had $48 million, and it now receives $68 million.  This is not doubling. Only 10% of grant applications are funded. Also because the population is small, funding from companies is small.  And, there is the huge barrier of no infrastructure.  If you get cancer, you go to a cancer institute and you’re immediately randomized to take part in experimental therapies that quickly enroll you.  The work of research proceeds more or less smoothly and automatically, because the systems are in place to allow that to happen.

Another issue: less than 5% of people who got investor-initiated NIH grants were under the age of 42; this means that experienced scientists are spending more and more time writing grants and less and less time doing research.  And younger scientists are saying the hell with it.

Todd Adams (wearing a dark suit, naturally) staffs representative Jim Langevin’s caucus on health and disabilities.  He introduces himself and talks about the CDRPA and the importance of getting it into general awareness.  He asks a question about Wise’s 10% figure, and Wise says it’s even worse than that . . . scientists who do make it through the grant process get halfway into projects and then have to stop and re-apply, and often lose funding just as they’re getting somewhere.

Wise asks Todd to address the chances of the CDRPA this year.  Todd says it’s a very tough budget year, and that in the area of increased NIH research, we need to press.  As far as the CDRPA, he thinks there’s little understanding of the need for this bill.  Members don’t get why this is important.  Many things are up in the air when it comes to funding . . . he personally wrote letters, but there was no response at all until ACTIVISTS called, wrote, and faxed their representatives.  He’s encouraging us to nag, nag, nag.  Staffers are used to this; they expect it.  It doesn’t trouble them.

A woman asks for a legislative summary . . . Marilyn says there’s a packet prepared.  The woman wants to know if there’s a time frame.  Todd says it’s urgent and ongoing.

A woman asks a question about young scientists.  Dr. Davies gets up to say that in his lab there are many graduate students who see what hurdles he has to jump through to get funding and they –understandably—say never mind.  Dr. Pearse repeats the message that at the Miami Project many scientists have given up and left the field for the same reason.  General discussion that there are labs across the USA that are in crisis because there is no money to fund the work.  The last 8 years have been terrible for scientists.  Wise is saying that he could not have imagined that 9-11 could have had such a catastrophic effect on the field.  In his lab, too, young scientists are looking at the big picture and shaking their heads.  (This is criminal when there are therapies that work.  Makes my head explode.)

Wise says that it’s unusual for scientists to show up at an activist event like this one; they’re here because they care desperately, and because they want to do their work.

Question: What exactly do you mean by the term “infrastructure”?
Dr. Hurtt:  What we mean is not bricks and mortar, but rather management.  When we need to launch a trial in sci, we start by locating qualified investigators.  What makes them qualified is not their ability to do the science, but their capacity to manage information and regulatory affairs.  There are rules by which they must play, and not all of them know those rules.  They need a research coordinator.  These people are like hen’s teeth.  It doesn’t matter if you have 5000 neurosurgeons . . . you need a huge rolodex to discover who the neurosurgeons are that have research coordinator.  It takes us a year to find the 100 out of 5000 who know how to do this.

These guys are also like cats, unherdable . . . they don’t trust one another.  They all want their own contract, their own budget, their own protocols.  Mark says that he has 50 ethics boards to deal with.  Every single step takes months and months.  In other fields this stuff is turnkey.  Everybody knows who the principal investigators are.  It’s all so EASY.  Even though there are many very qualified high end investigators in sci cure research, the field has not jelled to the point where you can just stick in the key, turn it and make things roll.

My company has done all the grunt labor (and wasted YEARS in the process) on behalf of its product Cethrin, but you MUST grease the skids for these people (gesturing at the academics in the room).

Dr Pearse:  It takes 15-20 million dollars to take a therapy into trial.  When there’s a group of advocates inside the NIH and the FDA, the process is extraordinarly simple.  The CDRPA will create the situation where there’s somebody on the other side of the fence when you toss them the ball  . . I had to toss the ball, climb over the fence, and catch it myself.

The meeting breaks up after Wise thanks everybody, and we mill around for a bit trying to figure out the logistics of who is going where and when.  The 3 Senate office buildings are about 4 blocks away, so the people in chairs need to plan around all kinds of physical issues.

I leave this meeting feeling – just so determined.  We can do this.  We can go get those other sponsors for CDRPA, and when it’s passed the networking infrastructure will be put into place.  The scientists will be able to stay at their benches and do the basic work. My first stop is going to be at Maria Cantwell’s office.

One Response to “Congressional Briefing”

  1. Klaimr said

    katewill thanks for great posts on congressional hearings and staying with it

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