Moderator is Kim Anderson, panel is the two people who just spoke.
Q: Okay, so InVivo is now solely dedicated to SCI … is your company dedicated to the long haul? Because we (she’s in a chair) don’t have a choice about that.
Q: How can we in the community help?
A: Ann Parr — we’re not ready to talk commercialization yet. When that day comes, we’ll have to partner with an established company or a startup. Right now look at Matt and Rob and GetupStandup and what they’ve done to become partners with us. We’ve been the beneficiaries of their work to get that bill passed. We also get money through the U of M foundation when it’s directed to our lab.
Q: in your pig model, what happened to the pig and how many trails do you think it would take?
A: What we’ve seen preclinically is still being evaluated — can’t say what happened to the pig because it was an early sacrifice.
Q: So how can we help you?
A: The open dialogue is very important in helping us understand what the endpoints we’ve chosen mean & help us defend the benefit of the clinical therapy at the end.
Q: Thanks for knowing that everything you do must be clinically relevant. How does the Rose-Bengal stuff compare to chondroitinase?
A: Rose Bengal is not cell specific, so we have to be super careful about where we put it. And it’s the case that CSPGs are not the only component in the early scar, and they’re not present in such numbers in the chronic phase.
Q: Wait .. so how do you know where to inject the Rose-Bengal?
A: We just put it right into the cavity, where it’s take up by the cells all around the cavity. Our goal has to be to understand dose and timing.
Q: In the inVivo results, you had 5 of 8 patients change their ASIA score — how do we understand that given that there is often recovery in the first days? How did you choose your patients?
A: Of course a large randomized clinical trial is the best method there is, but it’s not available to us. The regulatory pathway we’re taking involves showing safety and benefits that outweigh risks. (They’re approaching the FDA with a Humanitarian Device). Sorry, missed the answer to the 2nd q.
Q: Aren’t those ASIA scores kind of useless?
A: Yes, and we are definitely using other ways of capturing potential recovery to build a strong story of clinical good.
Q: How do you know that every injury involves a cavity? I have a vascular bleed …
A: That was a controversy in the beginning, but we’ve shown in large animal models that following a contusion injury there is spontaneous damage … we’re trying to develop imaging techniques to understand who will be a candidate for our products and procedures.
Q: Any comment from Dr Carr?
A: The issue with the insertion of the scaffold is that you can’t know how much recovery is due to decompression .. there are some interesting studies coming out of the UK now that show we maybe should be doing decompression and duroplasty after injury in the same way we do after a brain injury. However, if you can show that even if that’s the case, there could be a benefit later in avoiding problems down the line.
A: from Alex Aimetti: We have done surgical controls that show there are significant benefits in terms of changing the natural progression of the injury. We’re also looking at the downline formation of cysts and looking to know if we’ll be having
Q: Your subjects were very young … how did you talk the FDA into allowing that?
A: I don’t exactly recall that being a key discussion point … possibly the protocol is different for the Device situation.
Q: As you develop these scaffolds, are you going to develop biomarkers?
A: We’re going to be very interested in what other labs are doing, but in our trial we’re not engaged in that.
Q: Dr Parr, you said you got some motor neurons but you’re focused on OPCs … are you looking at using them and transplanting both?
A: We’ve thought about transplanting the combination, but right now we’re looking at doing them separately and understanding how that works; we’ll have to at some point figure out which should go first.
Q: Reminder that relationships are what matters, and keeping them strong. (This isn’t a question, it’s just Matt.)
Q: Have you thought about having a consumer board to guide your research?
A: We don’t have one now, but we do have very good relationships with the SCI community in MN. Come visit us and give us your feedback. it’s very hard to keep up with all the literature, and there have been times when Rob or Matt emails me information I’d missed.
A: We’re ramping up our participation in conferences like this one, and trying always to engage the right people all along the way.
Q: (InVivo CEO chiming in to address previous questions) InVivo is focused on this because for so long SCI has been the place where there are zero therapeutic options. That’s why we chose to take it up, and yes, we’re in it for the long haul. I worked with Act Up way back when, and yes we want patient advocacy groups.
Q: Dr Parr, are you worried that your transplanted OPCs might produce the “bad” kind of astrocyte or molecules?
A: One of the problems with cell transplants is that axons routinely refuse to grow out the far side of an injury, probably because there’s something they “like” inside the injury site.
Q: Dr Aimetti, possible chronic timeline?
A: Optimistically, the work can be done in 2 yrs … realistically, a ton of things could go wrong so will probably take a bit longer than that. We’ll be out in the community a lot, being as transparent as we possibly can be.