Thursday, August 27, 2009

Welcome to Chicago!

We've just had opening remarks by Marilyn Smith from u2fp and Ida Cahill from the Sam Schmidt Paralysis Foundation . . . I typed them up and then the blog I was trying to use ate them -- I'll put em in later.

Right now Susan Maus is speaking from her chair, giving props to Marilyn for being the rock star that she is, and recognizing the speakers and sponsors. She's going to move right along with the program.

First up will Dr. Doug Kerr, who has not spoken at w2w before. He's director of the transverse myelitis group at Johns Hopkins . . . investigates neural stem cells and -- yikes, she's reading from a little bio and it's VERY full of jargon.

Here's Doug . .. 40 ish guy with a low voice, leaning with both arms on the podium.

title of talk is emerging restorative approaches in neurologic disorders

my task is to describe some science to you in a way that is understandable to the educated advocate . . . you're going to see the data . . . i'm going to tell you 3 small stories.

stem cells for demyelinating disorders -- challenges are differentiation, delivery, and immune rejection. Opportunities are non-cell replacement trophic support and early clinical trials will be coming soon.

next story is stem cells for motor neuron disorders

next will be novel restorative approaches based on functional electrical stimulation

---

first slide is up image of glial restricted precursors, which is could for demyleinating disorders.

basically what you want is a cell that can become one of the cells needed to fix things in the brain.

steve goldman published data in 2004 that showed that glial restricted precursors that were derived from human stem cells produced healthy myelin in a mouse.

big bright images that show tight wraps of myelin wrapping around an axon -- very impressive demonstration . . .

in another study, steve goldman showed that by 9 months after transplantation, the glial cells had filled the territory. these transplants were given to this strain of mouse called shiverer, which normally dies after a very short time. they lived normal spans after the transplants.

so, how do you get gmp (good manufacturing practices) grade cells?
we're moving forward and trying to get to a clinical trial as fast as we can.

the problem is that most of the precursor cells don't become oligodendrocytes (myelinating cells) . . . but we figured out how to tell if they were . . . specifically we figured out a way to make them glow (the screen shows hot green on a mouse's head) if they were oligodendrocytes.

cool.

showing a hugely magnified section of a damaged mouse cord. shows the migration of human glial restricted precursors to fill up the whole damaged space.

more evidence (graph-form) to show that the cells did indeed fill up the injury site.

so, do the cells help the axons to conduct signals more efficiently? they measured N1 latency and showed that they do -- sometimes all the way to normal.

what was the functional return? the transplanted animals had more grip strength than the untransplanted animals.

here's the weird thing -- the cells went in, they occupied the injured region, but they did not generally turn into oligodendrocytes. so, why did they work? that's a question that we need to investigate a lot more.

you're going to see a lot of clinical trials coming very soon

we didn't do a particularly good job of differentiating these cells in this first go-round . . .

____

conclusion -- myelin precursors do sirvive and migrate, cell replacement and remyelination is HARD but not impossible

they're going to try to get a "hit" -- a very successful trial, which means one that will show functional return right off the bat. for this purpose, the best choice for a trial subject is tranverse myelitis, because it's a singal focal lesion, it has the right pathology of demyelination and axonal loss, it's an easy surgery with a small trget, and thre are surrogate makers (which I don't understand and will try to ask about)

we hope to get these studies off the ground in early 2011

image on the screen of a demyelinated multipel sclerosis cord. it's pocketed with holes. so the problem is how to deal with all those lesions? can't be sticking a needle into a cord or brain 40 times.

so they have developed a protein that makes cells traveling through the blood bind to the kind of receptors that will get them to into the nervous system.

this is very cool. no surgery!

what about the problem of immune rejection? do we really have to put every patient on cyclosporin? it's very dangerous to make people susceptible to infection . . .

our approach is to find a way to teach the immune system not to freak out over the foreign cells . . .

some options - block the microglial activation, which leaves the immune system intact.
or, do a single tolerizing approach (which he doesn't explain, but says that the idea came from the kidney transplant folks) -- this has been done successfully in that realm.

Ann Neurol 2006

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