Working 2 Walk

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Breakout Session 3: Hans Keirstead

Posted by katewillette on April 13, 2008

He says that in his lab they focus on acute, sub-acute, and chronic.  (Hans Keirstead, for those who haven’t met him, is kind of Robert Redford type.  I’m just sayin’.)  He’s describing an animal whose cord had been cut exactly 50%; within days it has lost a lot of tissue because of a “bad guy” molecule.  A single injection — of a restorative molecule –that could be given by any nurse — gets rid of a lot of the secondary degeneration.

There’s a video taken from underneath a glass table top on which a rat is dragging itself along — a single dose of the molecule prevents a LOT of the secondary damage.

The technology to do this is in clinical testing –Medarex began  clinical trials in 2006 for ulcerative colitis . . . the Keirstead approach is to find other (more profitable) diseases that studies can get funded for, then piggyback onto what’s learned and done in the process to bring the therapies to sci.

If Hans can get 1.5 million dollars, Medarex might provide him with $3 million worth of product to run a trial of his own

Subacutes— different beast completely from the acute.  Wow, he puts up a picture of a human egg sitting in a fallopian with a tiny blue speck of sperm heading its way. Wow.

We’re going to talk about how to grow human embryonic stem cell lines just for clinical use. The job is to be able to do that with certainty that they’re pure, and then be able to grow them into whatever you wanted.  Aborted fetuses are not a source for this for ethical, political, and practical reasons.

What if we had limitless sources of something like human heart tissue,not to grow new hearts, but to try out things that might

2 guys and a rat show up at the FDA and say, we’ve made a rat walk again . . . the FDA says, so what.  Do it again, under our regulations.

OEG’s are the cells that make myelin, the conductor that makes transmission of information through the cord possible.

He cooked up a soup to make pure OEG’s . . . it’s cooking time is 42 days.  They did this by simulating the media in which our own brains create these things.  This is the only place in the world where a pure and inexhaustible source of any kind of cell has been developed.

Hans just radiates confidence.

Geron is going to submit their IND to the feds this summer and they plan to go to clinical trial right away.  They went to the FDA and said they’d treated thoracic injuries in rats and the FDA said, well you can’t test humans with cervical injuries until you repeat the work with cervical injuries.  Jeebus.

About safety  . . . think about inserting a bolus of oeg’s into a cord . . . could be a problem.  So they did a test of a very, very tiny, light little injury and did their transplant on that one.  The injury was so small that it didn’t even cause deficit in function, and the insertion of oeg’s didn’t hurt anything.

Geron’s safety studies have done very well . . . except we only know this because the CEO of the company stood up in front of Reuters to say that he’s going after an IND (federal permission) to begin trials.  (Wahoo! No wonder Hans is so smiley.)

The clinical trials for this will happen this year.

Chronics.

Is it possible to create an inexhaustible source of motor neurons out of human embryonic stem cells?  He’s talking about repairing muscle groups one at a time . . . the thumb-mover, for example.

“We can grow one to 10 billion cells a week of these things. . . any one human is only going to take a few million.”

After 6 weeks, the cells display markers just like they should — meaning just as they would display if they were motor neurons that came into being in the usual way.

The true test, though, is to see if your motor neuron does anything with muscle.  Myoblasts can be cultured from a biopsy . . . a hunk of your own muscle.  We culture them together and omg I just saw a video of a hunk of myoblast  throbbing under the influence of motor neurons grown from stem cells.  “It doesn’t  just look like a motor neuron, or smell like a neuron, or talk like a neuron . . . it is a motor neuron.”

How do you get those motor neurons to grow fast?  They’ve created a substance that works. He doesn’t name it, just refers to it as an “attractant.”

Two months after transplantation . . . here’s a slide that shows –2 months after transplation — that the motor neurons are bundling themselves together and heading out to get to work. And–holy freakin’ smokes– he’s got rats that were 6 months post injury . . . he gives them the “attractant” shots into their non-functional limbs, leaving one limb untreated.  The video shows the rats clearly able to use the treated limbs while the untreated one drags along.

The strategy to use this treatment starts with type 1 infantile SMA, followed by terminal ALS, followed by chronic sci.  The reason to test other populations first is that the window is very, very quick (the patients are terminal); if he began with chronic sci, it would take a lot longer.

The list of challenges to clinical translation has 10 items on it, and the last one is the isolation of academics — we need to engage industry, the FDA, the government.  Whose job is it?

We’re way thrilled here, people.

He’s saying that he’s learned a lot from the sci population about what it wants.  Well, obviously complete reversal is the goal in the end.  But should we ignore what could be accomplished along the way?  They’re going to use their “attractant with motor neuron” therapy to target a single muscle . . . then move on to another one.

The oeg’s and motor neurons that will be part of the treatment do not come from our own bodies . . . which means that those who use those treatments will have to be on lifelong immunosuppression– which carries some issues and is undeniably a detriment.  Which is why they’re working ways to use our own tissues to create the cells that can fix what’s wrong with us.

Yikes, I hope I captured the gist of that, because it was quite amazing and delivered at warp speed.

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