Posted tagged ‘u2fp’

Let’s Review: Part One

November 15, 2012

I’m writing this from my desk at our house near Seattle . . . everyone who came to Working2Walk is by now long since home and settled back into whatever routines normally fill their days.  As I look over the posts on this blog, it occurs to me that there are a couple of things left to do.  One is to say thanks again to the people who worked all year to bring this thing into being, mostly as volunteers, one or two as very poorly compensated staff.  We owe them.

Another is to sift through all this information and highlight a few places that stand out for follow up and further review.  Your list might be different from mine on that score, and I’d be love to hear about it if that’s the case.  So, starting from the beginning . . .

1. Marilyn Smith’s words from her opening remarksWe have three tools. EDUCATE, ORGANIZE, and TAKE ACTION.  As those with the most skin in the game, we need to invest ourselves in the process.  The most powerful force we have going for us, though, is BELIEF.  We want you to leave this conference with belief in the power of science, knowledge, and advocacy.

2. Dr. Os Steward’s excitement about his CTRP team’s success in finding a specific gene — called PTEN — that blocks regeneration of nerves.  When someone with his reputation for calm and caution and meticulous communication shows enthusiasm, it’s definitely catching.  I’ve gone back and put some links into that post so you can read the background material yourself if you’re interested.

3. Dr. Murray Blackmore’s sheer cleverness, determination, and most especially, youth.  I’ve heard for a long time now that SCI research used to be considered a big fat dead end to any serious career.  “You’ll waste your life trying to solve that!  Focus on something that’s actually achievable!”  That was once common advice.  And here was this sharp new mind attacking the problems with impressive ingenuity and determination.  I especially liked the part where he said that he went data-mining in cancer research about transcription factors and quickly discovered that there are 210 of them that seem to have a role in cancer growth.  When he cross-checked that list of 210 with the 12 that he knew were involved with axon growth, 11 out of 12 were on the list.  As he told us, “That means the other 199 suddenly become very interesting . . . ”  In his talk, Blackmore described his just-published research that shows transcription factors can make old neurons think they’re young and still able to regenerate, so this matters.

4. Oy.  This is going to be a long list, I can see that.  I’ll put this up as part one and keep working on the rest.

Q & A

November 1, 2012

(Edited for clarity; this was a panel with Dr. Steward and Dr. Blackmore, moderated by Bob Yant.  The first question was about combination therapies.)

Dr. Steward: Pten operates at the signaling end of things and regulates the synthesis of proteins that are critical for growth.

Socs 3 is another gene that regulates the transcription of a particular RNA.

Together they create a double intervention . . . deleting them both amplifies regeneration in the optic nerve by a factor of 10 . . . right now they’re growing animals to start this very project on animal model sci in a couple of months.

Emphasis from advocate Bob Yant . . . if they can get a few segments from one intervention, and the combination gets you 10 times that, you basically have the whole effing cord, which is 30 segments  (effing was my contribution, Bob Yant definitely didn’t say that).

New question . . . is it possible to use information from a recent giant project about so-called “junk dna” in this effort?

Answer . . . most of the RNA that gets transcribed doesn’t seem to be doing anything . .. there’s a sea of it floating around in every cell . . . Blackmore is saying that he thinks this is a new target.

New question . . . so this is the first time we have recovery in animal models . . . is it right that pten alone didn’t get a lot of recovery, but combined  with Gibrin, it was there?

Answer . . . yes, we think that at least in the rat setting, pten alone isn’t that functionally dramatic .  . on the question of the lesion, we use cervical models because it’s the most common injury . . . but we can’t do complete injuries because rodents can’t survive them.  As far as Fibrin — it’s a glue.  in these experiments we used fish Fibrin.

Question:  very exciting stuff . . . curious to understand a little more, what’s the path from mice to humans?  Same or similar genes involved?  How does the longterm pathway unfold?

Answer:  From the science side, these pathways –pten, etc — are ancient and conserved.  Flies have them.  Mice have them.  There’s reason to believe that they’re conserved in humans as well.  As far as how you get from a virus that works in a lab to a human, I’m deferring to people who understand the nuts and bolts and the money a lot better than I do.

Question:  How much do you guys — the field — actually talk together about these things?  Who’s in charge of collaboration?

Answer: Nobody.

Question: That’s what I thought.  And that’s a problem.

Answer:  Yeah, we know.  And all of us are struggling in our own ways to fund our own labs.  The NIH is going to lose some funding this year.

Question:  Funding is a problem, and I get that, but it doesn’t seem to me that it’s efficient to use the resources the way we do, in competition.  We need somebody globally guiding the ship.  If the ship exists and it’s not the Titanic, the money will come from the global community.  We as a community need to guide this ship.  The effort to cure SCI needs air traffic control.

Answer:  Good comment.

Question: NASA was funded because scientists got together.  Isn’t it time for all the scientists to get together and create something?  Couldn’t they get enough money together to get it done?  Bob, you’re the perfect guy to put that together.  Friends of mine within a week gave $18.5 million, willingly.  The money is out there.  People do want to see a profit, but it seems like if there was a common leadership, it could be done.  United.  United to fight paralysis.

Answer:  (Bob Yant) — for myself, this is still at a basic research level.  The biotech world is way different from 15 years ago; today investors are more savvy and cautious.  Many of them call this kind of project the valley of death.  I’ve been to maybe 100 conferences, and this moment is almost surreal, to have been in this room hearing two different scientists talk about regeneration in published work that led to functional recovery.

Gahhhh.  Time for this session is over.  Holy smokes.

Why We Fight

November 1, 2012

Why we fight is the name of the talk that u2fp Executive Director Marilyn Smith is about to give, as soon as people are settled and the audio-visual-tech guys have all systems running.

Wow, 8 am turns out to be EARLY.  Just sayin’.

The scene right now: big carpeted ballroom, not too offensive low lighting, people getting fruit and bagels and coffee, scientists (they’re the ones in dark suits) and advocates, many of them in chairs, mostly not wearing suits. 🙂  I myself went for the jeans and tee-shirt option; this is California, after all.

The program this morning is focused on genetic therapies, and the first speaker after Marilyn will be Dr. Os Steward, who runs the Reeve-Irvine Research Center.  (You can see the whole agenda here.)

Lights down, music up, here we go!

I want to thank you all for coming; I know it was a long journey & we’re especially sad that so many people from the east coast couldn’t get away . . . next year we’ll be in Boston, so hopefully they’ll all see us then.

Thanks the many sponsors of this event, along with the speakers who take time out to show up and share what they’re doing.  Talk to them!  There’s a huge beneficial exchange of knowledge that always happens here.  Take advantage of it!  Thanks the staff, the people behind the scenes who make this all work, mostly volunteers.  Thanks the Reeve Irvine Research Center people who have also volunteered lots of time to us.  Thanks Succinct Productions for making our new video.  BIG thanks to Chris Powell and Donna Sullivan, her partners in pulling this together.

Recognizes people from Europe and from the Japan Spinal Cord Foundation, and from Nepal, and from Australia, all terrific advocates and all SO welcome here.


So why u2fp?  Why 6 people who met on the internet deciding to organize a WA DC rally?  Why did a couple of them decide after that to found u2fp?  Today is 7 years to the day since that beginning.  

For the people here, for the people in chairs, the people who support them, the researchers . . . for the people who aren’t here this year, the first time they’ve had to miss the conference (photos of David and Sue on the screen . . .blub).

Reading from a letter:  I’ve attended w2w twice now, and I can’t begin to tell you what a difference it made to me.  I told my sister that I hoped before I die I would see my baby boy walk again.  Unfortunately it didn’t work out that way. Sepsis, fever of 108, irreversible brain damage. . . we fight so that people will not have to endure what we did.  

As everybody knows, in spite of the name of the conference, it’s not just about the walking.  She’s asking how long it takes for people to get up and out the door in the morning . . . 15 minutes?  30?  an hour?  two hours?

Lots of people do of course figure out how to live well, even with paralysis.  They even make it look easy.  It’s not.

We’re here to fix it.  We have three tools. EDUCATE, ORGANIZE, and TAKE ACTION.  As those with the most skin in the game, we need to invest ourselves in the process.  The most powerful force we have going for us, though, is BELIEF.  We want you to leave this conference with belief in the power of science, knowledge, and advocacy.

She’s talking about Bob Yant . . . who has spent 30+ years living with SCI.  He’s educated himself about science, like a human encyclopedia.  He’s raised over $10 million to support that research.  He’s going to introduce our morning speakers.

Yant rolls up to the podium in his power chair.  Marilyn sits.

Yant:  Thank you for the very kind introduction and the wonderful passionate words . . . it’s so important to keep passion alive.  I want to give you a brief bio of Os Steward . . . gives his academic creds, then 1999 to now director of RIRC.

Woot!  Here he is.




Tuesday afternoon and the tribes are gathering!

October 30, 2012

I’m writing this from the 9th floor of the Hilton Hotel in Irvine, CA . . . which is right across the street from the John Wayne Airport and not very far from the ocean. Had lunch an hour ago with old friends Marilyn, Chris, and Donna who are part of the genius Unite2FightParalysis team responsible for this gathering.

Who are we?  People who want to fight for a faster cure.  People in chairs, people who love people in chairs, people who have some skin in this game.  People from all over the world.  Some of us are over 60, and some of us are teenagers.

What are we doing?  We’re going to hang out together and meet a bunch of scientists who are working on a faster cure and let them tell us more about it.  This year the focus is on regeneration, and many of the scientists are working here in California  We’re going to ask them hard questions; we’ve been at this for awhile now and our sense of urgency is only growing.  We need their sense of urgency to grow, too.

Some of us having fun at last year’s gathering