Lyn Jakeman will moderate again. This time she’s joined by Brian Kwon, Wolfram Tetzlaff, and Karim Fouad
She starts by talking about how the scientific community — having studied regeneration for decades — is in a strange place relative to that focus. Lots of work, lots of time, lots of careers … and not much to show for it. Sometimes it seems like we got ahead of ourselves when it came to what cells can and can’t do. Turns it over to Brian Kwon.
Brian says he wants to give us the perspective of an orthopedic surgeon, for the sake of shedding some light on challenges in SCI. Way back when the Egyptians were laying down the news that spinal cord injury was permanent — way back then, they were able to set bones and see them healed.
He’s showing a picture of a fence with green grass on one side and brown grass on the other. The grass on the bone side of the fence is a lot greener than on the SCI side. Brian’s a bone surgeon, so his focus is on how to use the naturally occurring bone healing in the context of the spine. (If only.) They’ve done all this work about getting bone regeneration in the spine. The seminal paper in this area was published in 1965 by Marshall R. Urist. Then in 1979, another paper about what was happening in bones, followed by another one in 1988 …. that’s how that path worked.
He poses a question: If you were making $70k per year and got a tip about a biotech company with a lot of promise but no chance of making a profit for 10 years, and you were being sued at the same time, how much would you invest in that biotech company? Choices go from nothing to $50,000.
The metaphor is about a real situation, in which Medtronic had to make that kind of choice. They had $70M in annual revenues. They had 3000 lawsuits pending. And they invested $50M in a new product. In July of 2002 they got FDA approval. So, the timeline: 1965 paper to 2002 FDA approval. That’s 37 years. And all they had to show was that this thing worked as well as natural healing.
So … what evidence of efficacy is needed in preclinical studies to justify moving to a clinical trial of human SCI? The translation model is …. Animal species, injury paradigm, time window, demonstration of clinically meaningful efficacy (whatever that means) ….
He’s laying this out so that we have perspective, and to show how tough it is and how long it takes.
Lyn asks for responses to what Brian just said. Wolfram says that he’s still a believer that we’ll one day get neuroprotection under control, with help from the work Brian has been doing in biomarkers. Karim says that there’s a lot of frustration in the field that we still have no treatments. We have to understand that sometimes research takes us down a dead end. There are a lot of treatments that have been dismissed as failures, but maybe that’s because we didn’t properly combine them – with rehab or with other kinds of therapy.
Lyn says we’re right at the cusp here, which is why the NIH is eager (for the first time!) to sponsor a conference.
Matt: I was in Columbus OH this spring, sitting in a coffeeshop with a researcher. He was talking about the acute rat model he was working on. I asked him if that had implications for a chronic model. And he said that he was interested in the work he can do, which happens right after an injury. And Matt replied that he was interested in what could be done for his son, Gabe, who was living with an injury. Important moment for them both. (My formulation of this kind of moment is: I am not you. Which means, I don’t know what you know. I may also not want what you want.)
Okay, on to the table discussion.
(In which I was talking and not typing!) Here are some comments from the general session following the table discussions …
We have a balanced table here, and we had a debate about acute v chronic, and we agreed that if you can fix a chronic injury, the acute will just follow along. Brian Kwon says that one of the important things about progress is that acute injuries get the very best care right from the jump. That’s how they get set up to optimize what’s coming down the pike for chronic injuries. Person from the table says that yeah, they recognize that the immediate trauma phase must get optimal care.
Matt says that almost every year someone raises the question about acute v. chronic strategies. Someone always says, “Why does acute injury strategy matter to me?” (This is two things, though … caring for the acute cord is different from trying to repair the acute cord.) Sue says that of course it makes sense to talk about neuroprotective strategies for acute injuries, but everybody here is concerned about chronic injuries. (Sigh. This conversation is so old …. it’s about allocation of scarce resources in research.) Matt says again, this ain’t either/or.
FYI, something like 20 minutes have now passed, during which we’re still all talking about the first question on a list of four. We’re now moving into the time period for the next item on the agenda, which is a talk from Rick Hansen himself. Someone is now saying that SCI needs a “win.” We need something that shows this is really doable. In the USA, we’re going to have to change the whole system we’re up against when it comes to post injury rehab. Kelsey says she wants to move on to Question #2! Which is, how does the SCI community get involved in the process of translation … Kelsey says there’s a really deep importance in looking inward to figure out where your own talents and energy lie. We can make a difference.
This is feeling very tiring right now … so strange, because after all the years of there being nothing at all that was even close to ready for humans, much less working in many, many trials with humans, we have that. We have epistim and transcutaneous stim. We have lots of clinical trials with chronic subjects, something that has seemed so far out of reach that it might never happen. And it seems very odd that instead of having some kind of party, we’re just saying the same old things to each other. Time for Rick Hansen!