Hans Keirstead

Posted November 2, 2012 by katewillette
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Roman Reed is introducing Dr. Keirstead as the best stem cell scientist in the world.  “He’s like a brother to me.  He’s a pioneer.  He’s going to win the Nobel prize one day.  We’re lucky to have him here.”

Hans:  I’m going to go through a couple of the things I’ve developed with my teams.

(Background . . . Keirstead’s lab famously was first to coax embryonic human stem cells into differentiating into the exact kind of neural cell he wanted, which was oligodendrocyte precursors (OPCs).  He tested those pure OPC cells in both acute and chronic animal models, and in the acutes ONLY, was able to show that the OPCs did their job, which is to remyelinate naked axons.  The company Geron took that work to the FDA and asked permission to do human acute trials . . . which were then delayed for years.  The trials finally did start, but then Geron backed out.)

Hans: Geron . . . we had developed highly pure oligodendrocytes and gave 5 patients 1/10th of what we thought would be an effective dose.  The regulatory bar was extremely high, and there was a very long patient followup — 15 years before release to the public.  All of it worked, which is great, but the company was burning a LOT of money for what would be a small market size.  It’s on hold for financial reasons.

I’ve been intimately involved in the analysis and it’s my belief that the program will be resurrected.

(More background . . . he then goes on to talk about the status of his human motor neuron trials, which he has been working for years to see happen in the USA with Spinal Muscular Atrophy (SMA) babies, who suffer from a terminal congenital condition that mimics in some ways the loss of motor function people with SCI have.  He’s told us in the past that it would be faster to try this therapy with SMA than with SCI because there’s much more urgency in the former.)

The high purity human motor neurons for sc disease was placed on clinical hold at the FDA.  We went to the UK with it  . . . using these cells, we’ve established a long body of evidence that it works to restore all kinds of motor function.  We expect to see it go into trial in 2013.

(So apparently the US FDA didn’t go for the idea, but its counterpart in the UK did . . . )

Hans moves on from updates on old work to new stuff . . . we have made high purity human neuronal progenitors for SCI — these cells don’t make anything but neurons — no astrocytes, no oligodendrocytes.

What I decided to do is manipulate the pten gene.  We looked at a couple of pten inhibitors. If we block pten, we get bigger, faster, better, longer axon growth — a 40-50% increase in growth.  Neurons grow, and pten-inhibited ones grow much better.  You also get a lot more secondary neurite growth.

Pten inhibition promotes outgrowth beyond cAMP levels . . we know exactly how that works, i.e., which pathway is being employed.

But, you can’t add pten to people . . .what we can do is add it to cells in a dish and then add the cells to people.  It’s a temporary thing, which is good.  You don’t want it in you for a long time.

In animal models, we’re looking at double the outgrowth in vivo.

pten is big all over the regenerative medicine world . . . many labs exploring what it means to have found a gene that controls whether or not a neuron can regenerate.

Another major effort is to reprogram the astroglial scar.  This is different from development, which is a process of differentiation.  Pluripotent reprogamming is a process of doing that in reverse, and lineage reprogramming is turning one cell into another kind altogether.

What this means is taking astroglial cells from an SCI scar and turning back the clock so that they become like they were when they were young.  In their aged, scar phase they’re inhibitors — but in their young phase they’re  promoters of growth.

They started by establishing astrocyte cultures.  Then they chose some outcome measures, which in itself took a lot of doing. (He’s saying they needed reliable ways to know if they’d succeeded in turning back the clock.)  They landed on markers of proliferation, and what happens when ROCK is added to them, and laminin expression, and dorsal root ganglion neurite outgrowth, and migration — all of which show clear differences between old and young.

They treat their old astrocytes with factors that make them think they’re young, look young, act young.  The factors work.

This is evidence that they’ve succeeded — in a dish — in the goal of taking astroglial scars backwards and turning them from regeneration blockers to regenerations supporters.

(Next would be taking those newly rejuvenated astrocytes and seeing what happens when they’re given to animals with SCI.)

Mark Tuszynski

Posted November 2, 2012 by katewillette
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Axonal Sprouting and Regeneration in the Central Nervous System

Going to talk with you about neural stem cells, which we hadn’t worked with much until recently.  My colleague Paul Lu came to me with a plan to look at neural stem cells . . . I discouraged him, but he was right, and his work has changed the direction of what we do in our lab.

Giving credit to funders, including the VA, CIRM,  and the Roman Reed foundation.

Showing a 9-year-old injured cord. What keeps it broken?

No permissive substrates in the lesion cavity, not enough neurotrophic support, and inhibition.  (My translation of this for the non-scientists would be: nothing for the axons to lie on as they grow, not enough fertilizer to help them grow, and too much toxic shit in the way.)

Inhibitors include myelin-asociated inhibitory proteins, like nogo, mag, omgp, semaphorin, netrin, and ephrin. (That’s some of the toxic shit.)

Then there are neuron-intrinsic factors like regeneration-related gene expression and or production of proteins (this isn’t really toxic shit . . . it’s shit that relates to the way your genes magically know when they’re allowed to help neurons regenerate and when they’re forbidden to do that.  Neurons in an embryo can regenerate, but neurons in an infant can’t.  Same cells, different rules coming from your genes.)

This talk is going to be about that last item . . . if you take an embryonic neural stem cell that IS growing and put into the adult spinal cord injury, will it grow?

Many years of efforts say, not really.  BUT, if you take E14 (E14 is shorthand for a 14-day-old embryo) cells from a developing rat and isolate the stem cells, they’re pluripotent in terms of neural cell types. (Meaning, they can still be turned into all three kinds of  neural cells: neurons, astrocytes, and glial cells.)  Hans Keirstead was able to take human stem cells and grow them in such a way that he got pure oligodendrocytes, but we’re not doing that .  . we’re taking them before they’re differentiated and putting them into the site.

They put trackers on their cells so they could see what happened to them once they were transplanted.

We grafted them two weeks post, because we wanted as much clinical relevance as possible. (“Clinical relevance” means being able to actually do this in human beings.)  We spent more than a year figuring out how to include a gel — a fibrin/thrombin matrix — that would let the cells survive and stick together, and we added some growth factors. (So, they had a good surface for the axons to lie on while they were growing across empty space, and they added some good fertilizer along with their undifferentiated, embryo-style neural cells.)

We used a T3 complete transection . . . six weeks later, the transplanted cells had completely filled the injury site.  Our tissue stains let us see what the cells had become.  A lot of them were neurons.  (Showing a slide with the section lit up)  Some cells turned into astrocytes, and others into oligodendrocytes.  All 3 types were there . . .

The truly remarkable thing was that axons emerged at very high densities over extremely long distances.  Until this experiment, the best we’d ever seen was about a hundred.  We have a thousand times more than that.

Not only that, but they’re growing much further than anything we’d ever seen, all the way up to c4 and down to lumbar.  When we did c5 hemisection models, the cells went all the way to the brain stem and then back down to the t8 level.  Some axons even grew out of the roots to exit the cord and head into the nerve roots.

We could follow the rate of growth over time . . . growth started right away and went at a rate of 1-2 millimeters per day, same rate as peripheral nerves.

This wasn’t just a few extra good results — it’s 80% of the time.  The stem-cell derived axons form connections with host motor neurons.  And they’re being remyelinated as they go.

So . . . a lot of axons grow out and grow for long distances — something I never thought I’d see.

But that’s only half the story.  You need axons to grow into the site as well, and that’s what we saw.  The host axons from the brain stem can penetrate the graft.  This means they could be forming new relays, new connections between the brain stem and the motor neurons.

We measure whether that’s happening, and it is.  Take a t3 graft, stimulate at c7, measure at t6 . . . and got a measurable electrical response, not quite normal, but real.  (To make sure they weren’t fooling themselves, they cut the cord again, just above the graft — and saw what would be expected, namely that the response vanished.)

The BBB score went from about 2 to about 7 with the grafts.  All of which supports the idea that you’re getting functional new relays across the injury.

These were the most potent results we’d ever seen.  So we looked at two human stem cell lines, one from neural stem cells and another from neuronal cells grown at Harvard from human stem cells.

(okay, to be clear . . . he’s saying that they started by putting rat embryo neural cells into transected rat cords, and then did the same experiment two more times, once with human embryo-derived neural cells put into transected rat cords, and then with human neural cells grown in the lab from human stem cells, again placed in rats.)

When we did the human neural cells, it looked a lot like what we saw with the rat neural cells when we put those cells into transected rats.  I have to say that for someone who has worked in this field for a very long time, these results are simply astonishing.

When we did the human stem-cell-based neural cells, we again got axons growing over very long distances, and lots of them.

As part of the CA consortium about 10 years ago, we set up a primate lab, so that was in place for the next phase of testing.

We did C7 hemisection lesion on the primates, and again delayed the graft for 2 weeks.  This time we used the human neural stem cells and have shown — again — axons growing robustly through white matter and gray matter.

This work with the monkeys is just beginning; it’s very important to do it.  The gels we used in the rats didn’t work in the monkeys . . . we have to devise a whole new matrix to fill that much larger cavity.  95% of rat-to-human models fail.  (95%!)

We need to see this work if (when!) we get to human trials.  We’re seeing many scientists from other labs come to see what we’re doing in the hope that it can be extended.

Progress with Neural Stem Cells

Posted November 2, 2012 by katewillette
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This afternoon there are just two speakers — but they’re stars.

First will be Dr. Mark Tuszynski, from UC San Diego and next an old friend of w2w, Dr. Hans Keirstead, from the RIRC.

We’re all kind of excited after listening to the morning group, so it’s a little hard to get people settled down and ready to listen up.

Mark’s talk is called “Neural Stem Cells for Severe Spinal Cord Injury”

Hans’ is Stem Cell-based Approaches to Treat SCI

Justin Brown, MD: Peripheral Nerve Surgery

Posted November 2, 2012 by katewillette
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Slide show is called Building Bridges to Restore Function

When you cut a peripheral nerve, Schwann cells go to work, attracting axons like a magnet.  How do you repair nerves that are too damaged?  You can do grafting by building an effective bridge, usually with nerve cells swiped from a place on the body where you don’t care much about sensation (side of the foot, e.g.)  it works pretty well.

Obstacles are distance, time, complexity, and age.  Closer is better.

The traditional way is to try to recreate what it used to look like . . . results are often mediocre. And sometimes you pop the nerve right out of the spinal cord (this is called avulsion).

A new idea was to do nerve transfer, which is a way of redistributing nerves that doesn’t try to reproduce what used to be there but rather invents a new system.  The peripheral nerve system is just as plastic as the CNS . . . showing a guy who survived a bad motorcycle crash and ended up with a fully paralyzed arm.  They did a graft . . . video from 3 months later, and he’s got some function back, 2 years later he’s got a normal arm with full range of motion, and you can’t tell which of those arms was injured.

You can take nerves that used to have a whole other destination and put them where you want them . . . and make them work.  And it doesn’t always take high end rehab.

Another example — guy got shot in the arm and lost use of his hand.  After the graft, video of his hand working normally while running off completely new nerve set.

So what about SCI?  You have a similar situation to a peripheral nerve loss.  Even years after injury, you should be able to take nerves still connected to the brain and use them in exactly the same way.  You’re transferring axons.

New video of a hand that was rewired.  Can you redistribute axons and restore grip?  Worked with a typical c5 patient, regrafted some nerves that were connected to the brain to replace those that had lost that connection . . . (Arghhhh, showing the surgery itself.  Okay.  Very bloody.)

Ha ha, Jerry Silver says from the audience, “You know, 2 shots of ChABC would really help!”

Justin says, “Let’s do it!”

They find the target with electronics, and — the guy can extend his fingers.  He has brain connection to fingers– connection that didn’t exist before this surgery.  “You can’t come into this game passive.  You have to work to get what you get.  It’s really dependent on how hard you work.”

What about incomplete injuries . . . There are classifications of EMG activity that show level of spasticity.  Turns out you can do peripheral neurotomies to deal with that.  Showing a video of man who had this treatment and went from needing a walker to being able to free-walk, and that was 15 years post.

The thing is, once these stem cell and other strategies get up and running, there’s going to be a tuneup phase.  Things won’t go back to the way they were; there will probably be a need for these kinds of targets.

We’re also working now on restoring the bladder.  With dogs, we’ve already been able to get that working.

Dr. Jerry Silver, Talk #2

Posted November 2, 2012 by katewillette
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Note: Matt Roderick is going to introduce the late morning speakers.  He has a son in a chair; first came to w2w in Phoenix 2 years ago.  He’s been a loud and persistent voice in Minnesota, where he lives, on behalf of the cure.

About Jerry Silver . . . he did a little for us in MN about 6 weeks ago.  He came to speak at the UM stem cell institute, where we’re trying to get a bill passed.  While he was there he talked with a researcher who I’ve been lobbying to get a chronic model going . . . she told me after listening to him that she’s on board for it now.

Here’s Jerry.

This is going to be about bridging a complete transection model — at first about an acute model and then at the end about the work they’ve done toward getting a chronic model going.

What we know about why axons don’t generate . .   The lesion.  Bleeding.  Macrophages rush in.  Axons die back.  Astrocytes move away and surround the entire area of inflammation with a scar, which keeps the inflammation from spreading.  Inside the core of the lesion are bunches of oligodendrocytes, but they’re not myelinating.

We decided to either build a bridge through this or just bypass the whole damn thing.  How do you get axons to cross?  You can create a conditioning response, give them some high test fuel . . . and get rid of the inhibitory molecules, like with chondroitinase (ChABC).  High test fuel and no guard rails.  Zoom!

In the 1800s they knew that central nervous system (CNS) nerve cells could be bridged across multiple centimeters to peripheral nerves.  BUT once they got to the end of the bridge, they couldn’t get out.  Why not?  25 years of research, and we learn that there’s a little scar filled with proteoglycans, which seemed to be like a little cul-de-sac for axons.  We solved that.  We published that last year, which is how I got invited to be here.

Now we’ve gone further.  The lesions we worked on were hemisections, and we wanted to try to do a bridge for a complete transection, the hardest bridge to build.  (He’s very enthusiastically giving credit to Yu Shang Lee, who is a bladder expert and a brilliant surgeon, along with one of his own cohorts in Ohio, Marc DePaul.)

They tried to use their strategies to all ow paralyzed animals (and hopefully humans) to urinate normally after complete transection SCI . . . can this be done?

In 1996, Chang published a paper claiming that with peripheral nerve grafting (PNG) and FGF, they could restore some walking to animal models.  Word got out that this was not good research because it couldn’t be reproduced.  Jerry says he asked Yu Shang Lee (who knew Chang) if the animals could really walk . . . answer was NO . . . but they sure could pee.

So interesting!  So Jerry’s lab did an experiment with PNG and FGF and ChABC (chondroitinase) . . . the FGF is crucial.  In their acute model, they labeled the nerve fibers, looking for the path of the axons relative to the spot in the brain where urination is controlled.

Showing a movie of these propriospinal neurons having crossed the cavity and keeping on going, 7 millimeters past the other side.  You can see hundreds of them, passing the gap, and going all the way to the lumbar sacral levels, the terminal point.

Okay, so some neurons can grow?  We just heard that this can’t happen the other day . . . so who are these special guys who can grow?  It takes them six months to get all the way from the brain stem down, but they can do it.  We haven’t even knocked out pten.  These are the primitive brain stem neurons that control urination, not the ones that control voluntary movement.

Showing graphs of rat peeing behavior.  They pee like crazy, all night long, dozens of times, over and over.  After injury, their bladders stay full all the time . .  they only pee five or six times.   If they get the treatment, they gradually start peeing more normally over a six-month period.  This is highly quantified, dense data that shows the rats have become physiologically normal.

So . . . is it regeneration or plasticity?  They transect and find that it’s regeneration.  They confirm that result with pharmacology, by blocking functions with drugs and watching the behaviors change. Proven beyond a shadow of a doubt that regeneration is critical for return of function in the bladder.


All brand new data . . . we started with a contusion injury.  Bridged with the triple combo, nerve graft, FGF, and ChABC.  And wait.  And wait.  And wait.

Oh, no . . . the animals got worse, far worse.  Depressing.

New strategy.  So they added an intermediate phase, in which those sleepy axons got waked up.  They went in and added FGF and Fibrin and ChABC, and wait, and then did the graft.  And that time it worked!

I just got to see the anatomy before I got on a plane to come here . . . there are thousands of axons in the graft and many of them are exiting.  At least at two months, we know that we can get regeneration.

Now we have a strategy.  We have to remove the scar surgically, but there may be other ways.  We can do any creative attempt to alter the wound.  We can add peptides, we can alter pten, we can add neurotrophins, we can do anything we can think of, and we hope a lot of people will get excited about this and JOIN US.

You’re hearing it from me, Jerry Silver, working2walk 2012:

After complete cord transection even at chronic stages long-distance regeneration is possible.

Q and A: Dr. Huhn & Dr. Anderson

Posted November 2, 2012 by katewillette
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Q: Will there be rehab?


Q: Congratulations!  Can you speculate about the anatomical substrate and the improvement in light touch?  Are you targeting the dorsal columns and the dorsal horn in humans?

One thing to consider is that these cells are incredibly migratory — in the mouse you can’t find the injection locus by density of cells.  They travel centimeters.  We think the cells won’t stay localized.  It may be that “complete” patients, that’s where we have detection sensitivity.  We’ll know a lot more when we get to the incompletes.

Q: To the mouse study people: Was there anything when you treated the acutes and the chronics that you did differently?

No, and we’ve seen recovery everywhere BUT in the immediate transplants.  We think it may be possible to extend the transplant time even further.  What’s going to be hard is doing combinations — you’re going to need a TON of safety information about how those combinations play out.

It’s likely that cells aren’t going to be the only thing, and that we’ll need to work very hard at creating designs for efficient trials.

Q: Are you working with inVivo to take advantage of their scaffolding?

We’re tracking it & trying to understand it  . . . this is an example of combinations we need to understand.

Q: Would it not make more sense to do a primate model instead of guessing with humans?

Primates are very interesting in terms of looking at SCI . . . one of our issues is immunorejection; when we work with mice, we’re going human to rodent.  (She’s making the case that it’s impossible to immunosuppress primates.)

When I look at the SCI trials that have been done in all kinds of animal models that have then failed at human levels, ultimately what we’re trying measure is what happens in humans.

Q: Could you measure myelin re-growth after the trial?

The hardware in those cords makes that very difficult . . . we can see it above and below, though.

Q: What to do if you’re interested in being in the clinical trial?

Here’s the link

Dr. Stephen Huhn, Stem Cell Inc.

Posted November 2, 2012 by katewillette
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Phase 1/2 Clinical Trial of HuCNS-SC Cells in Chronic SCI

As a neurosurgeon I have a different perspective when it comes to translating . . . what I know is that it takes a village — basic science, academic institutions, regulatory bodies, advocacy groups, (and others I missed) to get this done.  Clinically, there are transplants you can’t do.  You can’t do a brain transplant.  But there’s a long history of transplantations beginning with the kidney in 1954 and going through hearts, livers, lungs, and to a full face in 2010.

There’s a long list of central nervous system diseases that might be cured with stem cell transplants.  What gets everyone excited about stem cells is that they’re self-renewing and able to differentiate in ways that you can define .  . seems like sound footing for a potential therapy.  SCI has captured our imagination for a long time, partly because the injuries occur in young people, partly because the target is just a few inches.

I work at publicly traded biotech company. They have four ongoing trials. The first is when they put up to a billion cells into the brains of children with a fatal disease.  Three of the children have passed, and their parents have allowed his group to examine their brains and find out that the cells survived and did no harm.

The 2nd trial was another fatal myelination disorder that happens in children and can be diagnosed within hours of birth.  They used MRI techniques to find signs of myelination, (which matters to us because de-myelination is a problem for SCI).

The 3rd trial is for chronic SCI, being done at the University of Zurich.

It’s open label, they get a single dose of 20 million cells and do immunosuppression for 9 months.  They have 12 patients with T2 – T11 injuries.  The patients were 3 – 12 months post-injury.  They have 3 Asia A, 4 Asia B, and 5 Asia C.  They do a super fine sensory/motor test before the transplants to be sure they’re measuring meaningful change.

They did transplants last fall on all three of the Asia A patients.  Cells introduced at the margins of the injury, above and below with a very tiny needle, two places each.

Safety profile at 6 months:  All good.  No increased pain.

Any gains in sensory?  In 2 of them, yes.

1st patient was a t-8, 23 yr old male. Asia A . . . there was no sensory change in him.

2nd patient regained significant sensation as measured by electric and heat.

3rd patient 45 yr old male ASIA A, got back sensation.  Interesting that they’re not depending on patient reporting of sensation, but on measurable electrophysiological results.

So . . . no safety issues, which was the point. How to interpret this data?  It’s encouraging. They’re going ahead with the Asia B cohort, and the trial will be open to patients not just from Europe.

When we look back 20 years from now, this will probably seem very crude — but it’s very important that we’re doing it.  With each trial that we do, we’ll learn more.

And you can learn more about this trial, track its outcomes, and consider enrolling here.

Aileen Anderson

Posted November 2, 2012 by katewillette
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(Note: video of Dr. Anderson here, made a few weeks before the conference) Her agenda this morning is Stem Cell 101, Nervous System 101, SCI 101, and is timing really everything?

Stem cell 101 . . . these are undifferentiated cells that divide via mitosis, and their daughter cells can either differentiate into adult cells, or remain as stem cells.  When she was in grad school 20 years ago, people didn’t even know that there were stem cells existing in the adult human body.  Big change since then . . .

You can imagine stem cells as being like at the very base of a tree, with more and more differentiation as you move up the trunk and out into the branches.

The nervous system is 3 types of cells: neurons plus oligodendrocytes plus astrocytes.  The neural stem cells they work with can become each of these.

SCI 101 . . . signals not flowing in either direction.  (Yup.)  Getting injured is a longterm and dynamic process.  The injury isn’t the same after day one as it is after day 14, or six months later.

Early thinking about stem cells was to just make new cells and replace the ones that were lost. But there are other tasks they might be good for, like changing the ones that survive — activating them to do their job.  If we assume that we need replacements, what do we need from them?

First, they need to live, survive.  In SCI there’s an epicenter, but there’s also damage far away. Our transplants need to be able to migrate.  Then they need to do only what we want them to do and nothing else.  This is why we do safety trials (Phase 1 trials) — to make sure we’re doing no harm.

They work on the nature of cells, the nurturing of those cells, and the niche into which the cells go.

Their neural stem cells are derived from 16 – 20 weeks gestated human fetal brain.  They sort them based on cell surface markers.

Is timing everything? in 2003 someone published a review of historical literature that suggested there was no point doing transplants right after injury or a long time after injury; the subacute phase was best.  For years after that paper, everyone took it for granted that he was right.  It was logical.  (Okay, she just moved right on from that set up . . . maybe she’ll revisit it later.)

In 2008 the Reeve foundation did a big survey of the SCI community and gathered a lot of information — the most surprising thing was that there were MANY more people living with sci (1.3 million vs. 250,000), which meant that curing it would save a lot more money than had been previously known.

They use “sort of like boy in a bubble” rodents for their experiments to eliminate problems associated with human to mouse transplants.  They put 75,000 cells above and below the injury site . .. in the first week, at 30 days, and at 60 days.  Then they look at what happened to the cells.  Showing a slide that’s a colored cross section of an injured mouse cord that got cells in the chronic phase . . . the cells took on the characteristics of surrounding cells, which is what you want.

In the subacute environment, most of the cells became oligodendrocytes, some were neurons, and a very few were astrocytes.  The cells reach out and integrate into their surroundings.

Video of a little white mouse dragging herself along — she’s untreated.  Second mouse motoring along.  The tests they did later showed that the mouse cells were getting myelinated, and that axons were creating synapses.  They figured out a way to eliminate the human cells they’d put into those mouses — using a substance that was a thousand times more toxic to human cells than to mouse cells.  When they gave that to the treated mice, they lost the recovery they’d gained.

All that was about a subacute phase . . . what about a chronic phase?  Can the timeline be pushed later.  Actually, the cells at chronic phases survived just fine, the majority of them became oligodendrocytes, and very few of them became astrocytes.  Did they help with function?  Yes.

Fast forward . . . you take your findings and do more work, changing variables like crazy in order to show that it wasn’t a fluke.  There are many considerations for human trials:

Timing, dosage, neuropathic pain, multiple models, locus of transplantaion, migration, long term safety, scaling to primates, recover endpoints, molecular switching, AAANNNNDDDD

She stops because now we’re going to hear about clinical trials.

Jonathan Thomas, California Institute of Regenerative Medicine (CIRM)

Posted November 2, 2012 by katewillette
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Thanking the organizers, admiring the agenda, hoping to add to it in a good way . . . starting off by telling the story of a couple of recent meetings that have to do with the general field of regenerative medicine.

A month or so ago, Mike Milken hosted a group designed to move research faster — Faster Cures — at this moment in DC they’re talking about cutting everything.  The threat of sequestration is real and would mean 8% cut at NIH, which would be a very big deal.

Milken wanted to talk about organizing an event on the doorstep of the Capitol.  The Celebration of Science was 1000 people for 3 days, pulling together leaders of gov’t interested in health care, university presidents, philanthropists, last few heads of NIH and the FDA . . . on the middle day of this even they were at NIH, and there were a host of speakers talking about what NIH is up to, what kind of cutting edge work was being done.  There was a satellite feed with scientists talking live about their work, including SCI and regeneration.

The most striking thing to me was that regenerative medicine seems to mean stem cell research and genomics (DNA sequencing) . . . these two things combine to become what will be personalized medicine.

On the theme of personalized medicine, there’s a family in San Diego called the Beery family, who had twins, a boy and a girl.  They were diagnosed at age 2 with cerebral palsy.  The mother noticed that the condition of the children was deteriorating, which isn’t usual for CP . . . she went looking for anything she could find that the doctors had missed.  She found an article about something called Dopa Responsive Dystopia and brought it to her doctor.

He said it was worth a shot.  There’s a treatment for that, and the kids got it.  Picture the parents standing on the stage at NIH . . . with images of the kids being healthy and normal through grade school.  But at about age 12 they started to deterioriate for reasons nobody understood.  Back to the internet goes Mom.  She gets a doctor at Baylor U. to do a full genome sequencing on the kids, and there it was, a gene that caused serotonin deficiency in their bodies.

They got the treatment, and today are track athletes in high school in San Diego . . . and at that point in the talk the kids came bounding onto the stage.  The point of all this is that this kind of personalized medicine is what’s coming.

The second story has to do with a conference sponsored by CIRM, which we called Meeting on the Mesa — an aggregation of companies involved in stem cell research.  It’s a very interesting annual event, where each org. gets 15 minutes to describe what they’re working on.  One of them was the Stem Cells Inc presentation, which you’re about to hear.

There were others, too.  One is called RINO Site, which is about to ask for human clinical trials, along with others working on remyelinating nerves .  . . . a number of them that are making real progress.  We know that things are happening, & we’ve already funded a great many of them, as have others.  You don’t always hear about these things in the press, which tends to cover ethical issues and not progress.

We’re very happy to be able to fund stem cell research, thanks to Prop 71, which 8 years ago passed by 57% of the populace.  It was a grand experiment . . . $3 billion to fund companies in the state of CA doing stem cell research.  What that has done is turn CA into the epicenter of stem cell research in the USA.  We have a continuing stream of scientists from around the world moving here just to work in this environment.  At last count 135 of them, along with their staffs . . . it’s a tremendous pool of talent, which has led to a tremendous acceleration in medical research.

To date we’ve funded $1.8B, aimed at the most prevalent incurable conditions, one of which is spinal cord injury.  We’re aiming very high.  We’re looking for cures.  One of the problems we have with the press is that their stories go like this:  Gee.  We gave you all that money and where are the cures?  You were supposed to get this done in a month or two weeks or whatever.

When I sit down with reporters trying to figure out what we actually do, they can be a little intimidated . . . I sit down with them and they ask me why there are no cures, and I say: if your grandfather had been sitting with Jonas Salk in 1954 and asking him how come money raised since 1938 was just going down the hole, what could Salk have said?

The very next year he found the vaccine for polio, and that was 18 years after the effort started.  And that has arguably saved millions of lives.

That’s the level of difference we plan to make, and specifically in SCI.  We’ve funded 38 to 40 million so far, from basic research to clinical trials.  We funded Geron when they were working on SCI, and were very badly disappointed when they chose to discontinue.  At this point Mike West and Tom Okarma have bought the SCI portion of Geron’s portfolio, which all of us find very interesting and are watching closely.

As you know, six patients were treated with the Geron cells in a Phase I safety trial; they’re still being carefully monitored and have shown no ill effects from the cells.  I met patient #6, a young woman in northern CA, who was tremendously enthusiastic about the trials and full of hope that the trials would eventually deliver recovery for her and for the others.  She and the other 5 are eager to see those trials go forward.  Geron was the first company in the world to get FDA approval for using human stem cells in a product.

They did us all a considerable service by working through the FDA to break this ground; the FDA is very deliberate and slow-moving when it comes to new therapies.  Geron hung in long enough to get that part of it done.

At CIRM, we have some projects that we look to see heading toward successful conclusions.  One is about macular degeneration, another is Type 1 diabetes, HIV, heart disease . . .

All that said, nothing is closer to our hearts than SCI; Christopher Reeve and Roman Reed were hugely important in the effort to pass Prop 71, and we’re determined to carry on.

(Marilyn gets back up to say that CIRM funded the video-taping of this conference, and to introduce our friend, the amazing Donna Sullivan.)

Marilyn Day Two

Posted November 2, 2012 by katewillette
Categories: Uncategorized

Oh, it’s early!  I think a lot of folks stayed up enjoying the opportunity to socialize last night, but most of them are up and here for what’s going to be a terrific day.

She’s talking about the survey, talking about how seriously the organizers take feedback, talking about how much attention they pay to what can be improved.

There are funding envelopes too . . . in the past 7 years u2fp has done “a lot with very little” (and I know this to be an absolute fact).

This morning the keynote speaker is chair of the governing board at the California Institute of Regenerative Medicine (CIRM).  We had a lot of questions yesterday about who’s actually steering the ship . . . CIRM has a model for that, and we’re about to learn their methods.  

Please welcome Dr. Jonathan Thomas!