Do the patients need to be able weight bear alone for an hour?
No, just in a standing frame. Probably achievable for most patients.
What's the difference between the kind of rehab they do in China vs. the USA?
This has been the most intense debate we've had . . . in China every patient is pushed to the point of tolerance, which is obviously not the standard. Here we think that 1 hour a day for 3 days a week qualifies as intense. They've landed for the US trial on a standard of 3 hours per day for 3 days a week. All this will be walking, walking, walking. There is no data whatsoever on this subject. In Germany they did 3 hours a day for 5 days a week, which was considered at that time to be very intense.
Q: If you have a settlement, you're golden, if you're in the VA, you're silver, if you're neither, you're screwed . . . what's the plan to get people into these expensive trials?
There will be a lot of competition in the coming years. Money will be very tight. What the sci community can do is share the wealth. I see expensive equipment sitting in peoples' homes, and we should be making maximum use of these devices. There will be studies coming out that will justify insurance payment for these devices. It's hard, because it's a very tough question to answer.
Q: Will there be immunosuppression in your trials?
It won't be necessary, because we're matching blood in 4 out of 6 HLA categories. In our animal studies the cells do not get rejected.
Q: will the lithium cause pain?
Not in our studies. That was one of my primary worries; that lithium would cause pain. we just did an 80 patient study with a randomized placebo control . . . we will have a definitive answer this fall. we don't have any data on transplant plus lithium yet;
Q: Neurotrophins have been tried in the past and have not worked out very well
No, I have not seen that in humans . .
Lithium has been shown to stimulate stem cells in the brain as well as neurotrophins. It's been used for a century in treating the brain and the cord
Q: Will the patients have to live at the centers?
During the observational phase, people will have to be at the center 3 times. The patient doesn't have to come for the whole period. we anticipate a 3 day hospital stay, then they'll go home for 12 days, then come back for an intensive 6 week period of locomotor training. Some of the centers will be able to do more patients, but this has been the source of some very intense discussion, but we have not landed on any definitive answer.
Q: Who is going to pay for this?
We estimated that the total cost for this phase 3 trial would be $32 million. But we got the company to donate the cells, which brings it down to $24 million. Then on August 12 the FDA changed the rules so that we can recover some of the money from the patients; we're doing local fundraising, we're negotiating with the centers to lower the costs. We are committed to not having MONEY be the reason someone gets turned away.
We may do it like college admissions, where everyone who qualifies to be admitted is admitted, and then the potential to pay is factored into the cost for each person.
Q: What would probably be the expense after the trial to a patient?
We're carrying clinical trial insurance, so nothing. Remember that the trial will be randomized, so a quarter of the patients will get nothing, a quarter will get only lithium, a quarter will get only cord blood cells, and a quarter will get both. We have committed to giving all the participants the full treatment, assuming of course that the full treatment is effective.
In closing -- we should do this ourselves. I'm sick of waiting. We should not be waiting for a gift from a sugar daddy, or from the NIH, or from a company. I'm tired of waiting for someone who has a conflict of interest to step up. The people who have no conflict of interest in this -- who have in fact a vested interest in this -- are you.
www.justadollarplease.org
Friday, August 28, 2009
Wise Young
Donna starts by asking if anybody in the room does not know Wise Young? No hands go up.
She talks about how important CareCure has been to all of us, and Wise's tireless advocacy and availability to people in chairs.
Wise takes the stage.
Thanks for this great meeting; organizers, you've hit another home run.
I've been asked to give a 101 on spinal cord injury. After yesterday's meeting I added a few slides. I'm going to talk about the barriers that this thing should address. I hope by the end of this, you'll feel as I do that this is not a never-never land. It's something we can conquer.
I want to bring up a couple of points. One is how tired I am of opening a newspaper and reading that someone will never walk again.
In the USA there are over a million spine injuries per year, but only 10% of them get a spinal cord injury, because we have this amazing protective system called the vertebrae. it only weighs a few pounds, which is astounding. It's been said that all of us will have back pain sooner or later . . . the pain we feel is meant to keep us from stressing that vertebrae system.
What's contusion? What's compression?
The great majority of sci's are caused by bone pressing into the cord. Contusion is when the bone presses rapidly into the cord . . .compression is when the bone presses slowly into the cord, cutting off the blood supply. The cord is more resilient than the brain. You can press on it for 20 minutes and it will still be okay; the brain gets damaged after 6.
Most injuries involve both. Early decompression after sci is critical; as recently as the 1980's neurosurgeons were saying there was no need to remove bone from damaged cords. That's how deep was the conviction that a damaged cord was not worth anything.
The cord is protected by the this membrane called the dura mater (tough mother); it floats in cerbrospinal fluid. The dura's job is to redistribute compressive forces; it's so tough that even with a very sharp knife you can barely cut through it.
The cord is stretchable -- it can be stretched by as much as 50% as long as you do it slowly. What's the speed at which axons break? half a meter per second.
Andy Blight did a study in the 80's that will never be replicated . . . contused the cords of cats and then went and counted the axons. What he found is that the myelinated axons were the weakest, because the little spaces between the bits of myelin wrapping are like weak spots in a chain of links.
Contusion damages large myelinated axons, and compression injury damages small unmyelinated axons.
Perfectly understandable image up of what a laminectomy does -- remove the bone that's near the cord. Showing sections of rat cords with injuries caused by dropping 10 grams onto them.
Secondary injury -- which people in the field did not even believe in 30 years ago -- is caused by calcium into neurons. Calcium rushes into injured neurons, and if the level of it is high enough, it will activate enzymes that kill the cell. At low levels calcium is just doing a job; at high levels it's toxic.
Methylprednisolone (think sylvester stillone for pronunciation) ... Wise gave a huge dose of the drug to patients -- a thousand times higher than was ever given before -- this was in 1981, and he says that if he asked permission from the FDA to do this today it would probably be denied. He couldn't sleep the night this was done, because many of his colleagues warned that he was going to kill these patients with such a high dose.
Okay, so should we really hate the secondary injury? Not exactly; it's there for a reason, and it's actually involved in tissue repair. Within 5 seconds after you contuse the cord, there are cytokines on the scene, and they're producing growth factors and calling for macrophages that remove toxins and myelin fragments that inhibit growth.
Talking about a colleague who uses staining to show that the cells that go to the injury site are coming from the bone marrow . . . their job is to rebuild blood vessels. There are also astrocytes that restore the blood brain barrier, which is a sort of field that protects our central nervous system. If you have a leaky blood brain barrier, you're one of the people who responds badly to certain Chinese food with a lot of glutamate.
Okay, what about the glial "scar" ? Astrocytes line the boundaries between the cns and the pns. These cells look at severely injured tissue and see it as foreign, meaning they see it as "outside" the central nervous system. Every week, he says, somebody on CC gets on to ask if they should have a surgeon cut out their scar . . . and every week he answers the same thing: you will get a worse scar if you do, because it's not really a physical scar. The act of "cutting" would introduce more astrocytes . . .
Image of chronic sci -- in this case a rat with an 18 week old injury. There are macrophages sitting right on the surviving part of the cord, eating up cells.
What about regrowth? Lots of vertebrates do have the ability to regrow their cords, like lamprey, zebrafish, tadpoles, newts, lizards . . . they're all short, less than 2 cm in diameter. Until around the time of world war II, people died from sci, so there was no time for evolution to work out a way to regrow human cords.
There are some tracts in human cord that do re-grow (sorry, missed the names, but the corticospinal cord was not one of them. But you knew that.)
The human cord has about 20 million axons; cats have about 500,000. Only 10% of the cord is needed for locomotion in rat, cat, and human. Most people with sci have 5 to 10% of their cord intact. This means that adding 5% of the axons is often enough for recovery. If you have a growing tumor in the cord, and it takes up 90% of the cord, you may not notice nothing.
There's a guy up on 76th street who cuts the thinnest slices of lox you can imagine; I've seen tumor removal patients who had thinner sections than that, and they walked out of the hospital. This made me believe early that if I only had to restore 5%, I could do it.
Graph up of percent of white matter sparing versus walking score . . . the 10% rule applies. If 10% of myelinated axons survive, the rats can walk.
Okay, what about the central pattern generator? Locomotion is programmed into the cord, which is why chickens with no heads can run.
Talking about a dancer named Carey who had only one patch of sensation on one leg; he was ASIA B and it took him 2 years to regain walking. He worked for Wise as a patient liaison who signed up every patient he visited to be in a study.
-------
What are the barriers to regeneration?
The injury site lacks certain cell adhesion milecules and is surrounded by reactive gliosis.
Time. Axons grow no faster than ahair, lesthan 1 mm per day. we need years of sustained growth.
Growth Inhibitors. Nogo and chondroitin 6 sulfate proteoglycans stop some axonal growth.
Effective regenerative therapies have to take on all three of these.
You put cells into the edge of the injury, not the center, where they can form a bridge. You put in growth factors that will help the cells to grow. And you put in antibodies to the inhibitors.
What kind of cells? Umbilical cord cells combined with lithium is what Wise has chosen to use, wow, he's speeding through his slides about the effects of lithium combined with umbilical cord blood.
When they announced these trials, and said they were being done in China, lots of Americans volunteered to go to China. Speed-talking, jeez, Wise.
The criteria for the US trials is:
Injury at least 1 year old,
OKAYYY. . . I have to get the final slides from him because he did the last 12 in about 1 minute. Or less. Posting now and making another post for the QA
She talks about how important CareCure has been to all of us, and Wise's tireless advocacy and availability to people in chairs.
Wise takes the stage.
Thanks for this great meeting; organizers, you've hit another home run.
I've been asked to give a 101 on spinal cord injury. After yesterday's meeting I added a few slides. I'm going to talk about the barriers that this thing should address. I hope by the end of this, you'll feel as I do that this is not a never-never land. It's something we can conquer.
I want to bring up a couple of points. One is how tired I am of opening a newspaper and reading that someone will never walk again.
In the USA there are over a million spine injuries per year, but only 10% of them get a spinal cord injury, because we have this amazing protective system called the vertebrae. it only weighs a few pounds, which is astounding. It's been said that all of us will have back pain sooner or later . . . the pain we feel is meant to keep us from stressing that vertebrae system.
What's contusion? What's compression?
The great majority of sci's are caused by bone pressing into the cord. Contusion is when the bone presses rapidly into the cord . . .compression is when the bone presses slowly into the cord, cutting off the blood supply. The cord is more resilient than the brain. You can press on it for 20 minutes and it will still be okay; the brain gets damaged after 6.
Most injuries involve both. Early decompression after sci is critical; as recently as the 1980's neurosurgeons were saying there was no need to remove bone from damaged cords. That's how deep was the conviction that a damaged cord was not worth anything.
The cord is protected by the this membrane called the dura mater (tough mother); it floats in cerbrospinal fluid. The dura's job is to redistribute compressive forces; it's so tough that even with a very sharp knife you can barely cut through it.
The cord is stretchable -- it can be stretched by as much as 50% as long as you do it slowly. What's the speed at which axons break? half a meter per second.
Andy Blight did a study in the 80's that will never be replicated . . . contused the cords of cats and then went and counted the axons. What he found is that the myelinated axons were the weakest, because the little spaces between the bits of myelin wrapping are like weak spots in a chain of links.
Contusion damages large myelinated axons, and compression injury damages small unmyelinated axons.
Perfectly understandable image up of what a laminectomy does -- remove the bone that's near the cord. Showing sections of rat cords with injuries caused by dropping 10 grams onto them.
Secondary injury -- which people in the field did not even believe in 30 years ago -- is caused by calcium into neurons. Calcium rushes into injured neurons, and if the level of it is high enough, it will activate enzymes that kill the cell. At low levels calcium is just doing a job; at high levels it's toxic.
Methylprednisolone (think sylvester stillone for pronunciation) ... Wise gave a huge dose of the drug to patients -- a thousand times higher than was ever given before -- this was in 1981, and he says that if he asked permission from the FDA to do this today it would probably be denied. He couldn't sleep the night this was done, because many of his colleagues warned that he was going to kill these patients with such a high dose.
Okay, so should we really hate the secondary injury? Not exactly; it's there for a reason, and it's actually involved in tissue repair. Within 5 seconds after you contuse the cord, there are cytokines on the scene, and they're producing growth factors and calling for macrophages that remove toxins and myelin fragments that inhibit growth.
Talking about a colleague who uses staining to show that the cells that go to the injury site are coming from the bone marrow . . . their job is to rebuild blood vessels. There are also astrocytes that restore the blood brain barrier, which is a sort of field that protects our central nervous system. If you have a leaky blood brain barrier, you're one of the people who responds badly to certain Chinese food with a lot of glutamate.
Okay, what about the glial "scar" ? Astrocytes line the boundaries between the cns and the pns. These cells look at severely injured tissue and see it as foreign, meaning they see it as "outside" the central nervous system. Every week, he says, somebody on CC gets on to ask if they should have a surgeon cut out their scar . . . and every week he answers the same thing: you will get a worse scar if you do, because it's not really a physical scar. The act of "cutting" would introduce more astrocytes . . .
Image of chronic sci -- in this case a rat with an 18 week old injury. There are macrophages sitting right on the surviving part of the cord, eating up cells.
What about regrowth? Lots of vertebrates do have the ability to regrow their cords, like lamprey, zebrafish, tadpoles, newts, lizards . . . they're all short, less than 2 cm in diameter. Until around the time of world war II, people died from sci, so there was no time for evolution to work out a way to regrow human cords.
There are some tracts in human cord that do re-grow (sorry, missed the names, but the corticospinal cord was not one of them. But you knew that.)
The human cord has about 20 million axons; cats have about 500,000. Only 10% of the cord is needed for locomotion in rat, cat, and human. Most people with sci have 5 to 10% of their cord intact. This means that adding 5% of the axons is often enough for recovery. If you have a growing tumor in the cord, and it takes up 90% of the cord, you may not notice nothing.
There's a guy up on 76th street who cuts the thinnest slices of lox you can imagine; I've seen tumor removal patients who had thinner sections than that, and they walked out of the hospital. This made me believe early that if I only had to restore 5%, I could do it.
Graph up of percent of white matter sparing versus walking score . . . the 10% rule applies. If 10% of myelinated axons survive, the rats can walk.
Okay, what about the central pattern generator? Locomotion is programmed into the cord, which is why chickens with no heads can run.
Talking about a dancer named Carey who had only one patch of sensation on one leg; he was ASIA B and it took him 2 years to regain walking. He worked for Wise as a patient liaison who signed up every patient he visited to be in a study.
-------
What are the barriers to regeneration?
The injury site lacks certain cell adhesion milecules and is surrounded by reactive gliosis.
Time. Axons grow no faster than ahair, lesthan 1 mm per day. we need years of sustained growth.
Growth Inhibitors. Nogo and chondroitin 6 sulfate proteoglycans stop some axonal growth.
Effective regenerative therapies have to take on all three of these.
You put cells into the edge of the injury, not the center, where they can form a bridge. You put in growth factors that will help the cells to grow. And you put in antibodies to the inhibitors.
What kind of cells? Umbilical cord cells combined with lithium is what Wise has chosen to use, wow, he's speeding through his slides about the effects of lithium combined with umbilical cord blood.
When they announced these trials, and said they were being done in China, lots of Americans volunteered to go to China. Speed-talking, jeez, Wise.
The criteria for the US trials is:
Injury at least 1 year old,
OKAYYY. . . I have to get the final slides from him because he did the last 12 in about 1 minute. Or less. Posting now and making another post for the QA
Friday morning: Marilyn Smith & Rachel Hammerman
Marilyn is at the podium talking about the agenda for today; unfortunate news is that Phil Horner will not be giving his presentation here today because of a family emergency.
The good news about that is that we get a little more time for lunch, a little more time for the presentations we do have.
Now . . what's u2fp anyway? Not a fundraising organization, just a group of people connected to sci in some way who wanted to step up and become the voice of the community. We see our job as
She introduces our public policy liaison, Rachel Hammerman, who helped us get the CDRPA passed.
Rachel is young and beautiful, standing at the microphone explaining that she's going to talk about speaking up and being visible. "Raise your hand of if you can't raise your hand have your neighbor raise two hands . . . " Almost everyone does.
So what does it mean to be an advocate? Woman says that for her it was speaking up and asking that her son get what he needs. Todd says that the most powerful thing is how you appeal to someone's emotions. Stephen Davies says that he tries to come up with effective therapies, and speaks with politicians and with the media about the fact that the research is getting somewhere. Sue Maus says that last year on Capitol hill Stephen had a 102 fever and went to the congressional briefing anyway, then agreed to go to Norm Coleman's office . . . Coleman wanted to hear from him: "Where is the science? How close are we?" She says that Stephen's presence was key to getting that vote.
Rachel: that bill was on the floor for 7 years. We were up there for 4 of those years, and if you were on our mailing list, you got those emails, you made those calls.
Advocacy means speaking out on our own behalf or on behalf of others -- what we're doing is helping people become passionate about research and cures.
If someone asks you what you did this week, you can tell them: I was learning about research for sci. That's advocacy.
What forms can advocacy take? Just about anything . . . for some people it's clicking that button when you get an email, for some it's public speaking, for some it's just talking with your neighbors. It's NOT keeping your mouth shut.
The key is persistence, and obviously every person in this room knows what that word means . . .
So, she's going to go through a list w/ more detail. Suggests that people use the blog link to show others what this conference was about. The big goal is to show others what this is. Use all the social networks you have, like facebook -- put it on your status. "I was at w2w in chicago."
Then there's the media advocate . .. before you get to that, you need a 15 second elevator pitch -- a sentence that can be said in the time it takes to ride in an elevator.
Media includes everything from old-fashioned newspapers to twitter. Yesterday Naomi Kleitman said yesterday that she was quoted in the media as saying "I don't think there's going to be a cure" when what she said was "I don't think there's going to be one single cure, it's going to be more of a combination." So, be careful.
Sean Tipton, who led the fight on Capitol Hill to pass funding for ESCR, says that you must have 4 talking points, and use the image of a prism to help you stay on them. The question comes through the tip of the prism . . . your job is to direct that question to one of the 4 faces of the prism-- your talking points. Never, ever leave them.
All of us, she says, are BOSSES in the sense that each of us has 4 employees: our representatives in congress (2 senators and 1 representative, plus the president). You're free to call them up, write to them, get their attention. The person you'll usually work with is the Health Legislative Assistant. You'll probably get their voicemail. You almost NEVER get to speak with them right away. This is where you must be persistent . .. eventually will get to speak with them.
This is why it matters so much to have some talking points -- faces of the prism -- all ready. Go ahead and use what's on the u2fp website; that's what it's there for.
Talks about the wonderful Donna Sullivan, who put the phone numbers of her own representatives in Ohio into her speed dial and called them every day until she got their attention. They all signed on to CDRPA.
No matter what your own strengths and weaknesses are, you can make a difference. Talk about why this matters so much, wherever you are.
The whole reason u2fp exists is to give each of you a LOT of ways to advocate. Call us, email us, read your handouts.
Joe White is talking about being in the Mall of America, and how he always carries cards with him . . . if someone asks a question ("How do you like your chair?") I give them a card and explain what it means.
Todd says that people should remember that the legislative office people you usually talk with are very young, and they'll be moving up the ranks; now's an easy to time educate them and motivate them to help when they have more authority. Make relationships now.
Joe says that people should call the newspapers when we get home to describe this event. You might get a little blurb, but every bit of exposure helps.
RAchel signs off, and Marilyn is back to say that although u2fp is not a fundraising organization, we do recognize that sci research is deeply underfunded, and that many people here are interested in how to do this.
The website and the materials we handed out at registration have a fundraising kit that has some guidelines about how to do this. Joe White has staged a Walk and Roll fundraiser in his home town . . . he explains:
We got together a couple of years ago . . . I left working2walk a few years ago and decided that I needed to do something. We rented a pavilion and got some local businesses to donate the cost of that, plus t shirts and food and flyers. I thought we might get 20 people who donated 100 dollars. We got 120 people and raised 16 Thousand dollars. Gave some to u2fp and some to Dr. Davies and kept some seed money for the next one. Our 3rd annual event is this October.
Marilyn: anybody who's ever done anything like this will tell you that it's a lot of fun. Image is up of Karen Miner's group, Research for Cure, which has raised $340,000 for Hans Keirstead's lab in Irvine over the last decade or so.
Okay, so what if you don't have the time or interest to organize a fundraiser? There are ways to participate . . . we know of a few people who did the Rock and Roll marathon, or the Team Reeve marathon and raised money through them.
There are also matching gift programs that many employers use. And, think about micro-funding. You don't need to go raise $500,000 . . . you can raise $5 from 100,000 people, or from 50 people. Whatever. It all helps, and whatever you do, even the old tried and true things like car washes and bake sales, is using what you have to make a difference.
Woman is describing how after her son's best friend was injured in a crash 16 years, her daughter had the idea of raising money through a car wash and bake sale -- that was a success and it grew into a statewide organization.
Image of Jennifer Longdon, who did the Rock and Roll marathon in Phoenix and raised a pile of money for the Keirstead lab.
Marilyn describes a Chicago guy who was here earlier but had to work -- 2 year old injury and dove right into fundraising. Has so far raised $100,000.
Turns it over to Donna, who will introduce Wise.
The good news about that is that we get a little more time for lunch, a little more time for the presentations we do have.
Now . . what's u2fp anyway? Not a fundraising organization, just a group of people connected to sci in some way who wanted to step up and become the voice of the community. We see our job as
She introduces our public policy liaison, Rachel Hammerman, who helped us get the CDRPA passed.
Rachel is young and beautiful, standing at the microphone explaining that she's going to talk about speaking up and being visible. "Raise your hand of if you can't raise your hand have your neighbor raise two hands . . . " Almost everyone does.
So what does it mean to be an advocate? Woman says that for her it was speaking up and asking that her son get what he needs. Todd says that the most powerful thing is how you appeal to someone's emotions. Stephen Davies says that he tries to come up with effective therapies, and speaks with politicians and with the media about the fact that the research is getting somewhere. Sue Maus says that last year on Capitol hill Stephen had a 102 fever and went to the congressional briefing anyway, then agreed to go to Norm Coleman's office . . . Coleman wanted to hear from him: "Where is the science? How close are we?" She says that Stephen's presence was key to getting that vote.
Rachel: that bill was on the floor for 7 years. We were up there for 4 of those years, and if you were on our mailing list, you got those emails, you made those calls.
Advocacy means speaking out on our own behalf or on behalf of others -- what we're doing is helping people become passionate about research and cures.
If someone asks you what you did this week, you can tell them: I was learning about research for sci. That's advocacy.
What forms can advocacy take? Just about anything . . . for some people it's clicking that button when you get an email, for some it's public speaking, for some it's just talking with your neighbors. It's NOT keeping your mouth shut.
The key is persistence, and obviously every person in this room knows what that word means . . .
So, she's going to go through a list w/ more detail. Suggests that people use the blog link to show others what this conference was about. The big goal is to show others what this is. Use all the social networks you have, like facebook -- put it on your status. "I was at w2w in chicago."
Then there's the media advocate . .. before you get to that, you need a 15 second elevator pitch -- a sentence that can be said in the time it takes to ride in an elevator.
Media includes everything from old-fashioned newspapers to twitter. Yesterday Naomi Kleitman said yesterday that she was quoted in the media as saying "I don't think there's going to be a cure" when what she said was "I don't think there's going to be one single cure, it's going to be more of a combination." So, be careful.
Sean Tipton, who led the fight on Capitol Hill to pass funding for ESCR, says that you must have 4 talking points, and use the image of a prism to help you stay on them. The question comes through the tip of the prism . . . your job is to direct that question to one of the 4 faces of the prism-- your talking points. Never, ever leave them.
All of us, she says, are BOSSES in the sense that each of us has 4 employees: our representatives in congress (2 senators and 1 representative, plus the president). You're free to call them up, write to them, get their attention. The person you'll usually work with is the Health Legislative Assistant. You'll probably get their voicemail. You almost NEVER get to speak with them right away. This is where you must be persistent . .. eventually will get to speak with them.
This is why it matters so much to have some talking points -- faces of the prism -- all ready. Go ahead and use what's on the u2fp website; that's what it's there for.
Talks about the wonderful Donna Sullivan, who put the phone numbers of her own representatives in Ohio into her speed dial and called them every day until she got their attention. They all signed on to CDRPA.
No matter what your own strengths and weaknesses are, you can make a difference. Talk about why this matters so much, wherever you are.
The whole reason u2fp exists is to give each of you a LOT of ways to advocate. Call us, email us, read your handouts.
Joe White is talking about being in the Mall of America, and how he always carries cards with him . . . if someone asks a question ("How do you like your chair?") I give them a card and explain what it means.
Todd says that people should remember that the legislative office people you usually talk with are very young, and they'll be moving up the ranks; now's an easy to time educate them and motivate them to help when they have more authority. Make relationships now.
Joe says that people should call the newspapers when we get home to describe this event. You might get a little blurb, but every bit of exposure helps.
RAchel signs off, and Marilyn is back to say that although u2fp is not a fundraising organization, we do recognize that sci research is deeply underfunded, and that many people here are interested in how to do this.
The website and the materials we handed out at registration have a fundraising kit that has some guidelines about how to do this. Joe White has staged a Walk and Roll fundraiser in his home town . . . he explains:
We got together a couple of years ago . . . I left working2walk a few years ago and decided that I needed to do something. We rented a pavilion and got some local businesses to donate the cost of that, plus t shirts and food and flyers. I thought we might get 20 people who donated 100 dollars. We got 120 people and raised 16 Thousand dollars. Gave some to u2fp and some to Dr. Davies and kept some seed money for the next one. Our 3rd annual event is this October.
Marilyn: anybody who's ever done anything like this will tell you that it's a lot of fun. Image is up of Karen Miner's group, Research for Cure, which has raised $340,000 for Hans Keirstead's lab in Irvine over the last decade or so.
Okay, so what if you don't have the time or interest to organize a fundraiser? There are ways to participate . . . we know of a few people who did the Rock and Roll marathon, or the Team Reeve marathon and raised money through them.
There are also matching gift programs that many employers use. And, think about micro-funding. You don't need to go raise $500,000 . . . you can raise $5 from 100,000 people, or from 50 people. Whatever. It all helps, and whatever you do, even the old tried and true things like car washes and bake sales, is using what you have to make a difference.
Woman is describing how after her son's best friend was injured in a crash 16 years, her daughter had the idea of raising money through a car wash and bake sale -- that was a success and it grew into a statewide organization.
Image of Jennifer Longdon, who did the Rock and Roll marathon in Phoenix and raised a pile of money for the Keirstead lab.
Marilyn describes a Chicago guy who was here earlier but had to work -- 2 year old injury and dove right into fundraising. Has so far raised $100,000.
Turns it over to Donna, who will introduce Wise.
Thursday, August 27, 2009
Panel: Naomi Kleitman, Barry McKay, Peter Gorman, and Tony Caggiano
Tony is speaking about his role as manager of the clinical trial process at Acorda.
Naomi asks if people were in clinical trials, and several explain what they were involved in.
One did the fampridine trial and is eagerly waiting for the release of the drug next fall. One used a device that was not yet approved, but the company that was manufacturing it went under. She now gets it from England at a very high cost. The panel says that it's a big issue; companies go under and there you are. A couple of people went to Gainesville for the suspended gait training, and both got benefit.
Naomi wants to know if any minds were changed today about the lack of clinical trials ~ nobody answers her question, but someone asks another one about the 4sci trials. It seems to be about repeating animal tests that seemed promising. Theyve now repeated about a dozen of these trials and so far failed to replicate what was published.
(This sucks, of course, but here's how finely tuned the spinal cord is . . .when they tried to replicate studies from Europe, one issue was that the strain of the animals being tested was a little different in the USA. Small physiological change => different outcome.)
Man in the front row is asking about off-label usage of approved drugs. They tell him that doctors are free to prescribe drugs . . . but does the NIH care about this? Naomi says that there are 26 funding centers at the NIH. The entry criteria for getting money from NIH is about a million $ a year.
Ida Cahill says that she gets phone calls from people who are thinking of going outside the USA. She knows of 2 women who went to Portugal, and 2 more who went to India. They spent $70k each plus travel to go get who knows what done to them.
Some talk about this, and how to know if a trial is legit. Basically, it's just a very risky undertaking to leave the country, especially if the therapy you're getting involves cells. Naomi gets up to talk about what red flags look like, refers people again to the iCord website where the full question set is available.
Man asks about the existence of a national database . . . if you get cancer your name automatically gets put into a giant list that keeps track of everyone; the answer is that hiipa regulations prevent this from being done -- that can't be right, can it? Then why do cancer patients do it?
A question about emergencies -- how the heck to doctors and scientists go about inviting people with new injuries (who are devastated physically and personally) to give their consent to a trial. Naomi is describing something called community consent that made it possible to include minorities, who as it turned out, were being underserved.
A question about whether or not the trials that involve cells are going to have controls. The answer is no, at least not in Phase I. What will happen in Phase II?
In some studies (ProChord) they've hidden the information from the evaluators. It's always a tension between the need to do the science right and the need to treat patients ethically.
Woman says that the VA hospitals are looking for study subjects.
Man says that the Portugal trial has been through the EU version of the FDA, and that an IRB does exist there, and at least one paper has been published about the results. He's a rehab doctor at Wayne state university. Panel member replies that the paper had flawed data, as would be clear to anybody with a statistical background. He says they also have claimed no adverse effects, but he personally knows of 6 very adverse effects. He's worked with people who got the Lisbon treatment, came back for intense PT and compared them with people who just got the PT . . . no difference. NADA.
Another panelist says that, you know, here we are with 2 qualified people looking at the same data and coming to a different conclusion. (That is what I hate.)
Woman whose daughter has been injured for 18 years says that she's seen rats walking, for heaven's sake. How on earth do you all come together? Panelist says that his favorite annual meeting is the ASIA meeting. They do learn from one another there. Also the journal Spinal Cord Injury. Also the peer review process. Also the NIH funding process itself, which is a marker to show that this work is new, promising, and original.
Woman asks the assessment measures guy what's being done with his work . . . he says that it will let small changes be documented and approaches be validated.
She says, could this assessment tool be used to convince insurance companies to pay for treatments like rehab?
He says he can't imagine that it wouldn't . . . and people should understand that to bring a group of combined treatments to the FDA -- oh, wait. Naomi says all they need is to show that their is safety in each of the individual components.
Oy, long day, people. Marilyn says it's time for a little socializing. Yay! Plus we get a buffet -- a Mexican buffet! Woo hoo, seriously.
Naomi asks if people were in clinical trials, and several explain what they were involved in.
One did the fampridine trial and is eagerly waiting for the release of the drug next fall. One used a device that was not yet approved, but the company that was manufacturing it went under. She now gets it from England at a very high cost. The panel says that it's a big issue; companies go under and there you are. A couple of people went to Gainesville for the suspended gait training, and both got benefit.
Naomi wants to know if any minds were changed today about the lack of clinical trials ~ nobody answers her question, but someone asks another one about the 4sci trials. It seems to be about repeating animal tests that seemed promising. Theyve now repeated about a dozen of these trials and so far failed to replicate what was published.
(This sucks, of course, but here's how finely tuned the spinal cord is . . .when they tried to replicate studies from Europe, one issue was that the strain of the animals being tested was a little different in the USA. Small physiological change => different outcome.)
Man in the front row is asking about off-label usage of approved drugs. They tell him that doctors are free to prescribe drugs . . . but does the NIH care about this? Naomi says that there are 26 funding centers at the NIH. The entry criteria for getting money from NIH is about a million $ a year.
Ida Cahill says that she gets phone calls from people who are thinking of going outside the USA. She knows of 2 women who went to Portugal, and 2 more who went to India. They spent $70k each plus travel to go get who knows what done to them.
Some talk about this, and how to know if a trial is legit. Basically, it's just a very risky undertaking to leave the country, especially if the therapy you're getting involves cells. Naomi gets up to talk about what red flags look like, refers people again to the iCord website where the full question set is available.
Man asks about the existence of a national database . . . if you get cancer your name automatically gets put into a giant list that keeps track of everyone; the answer is that hiipa regulations prevent this from being done -- that can't be right, can it? Then why do cancer patients do it?
A question about emergencies -- how the heck to doctors and scientists go about inviting people with new injuries (who are devastated physically and personally) to give their consent to a trial. Naomi is describing something called community consent that made it possible to include minorities, who as it turned out, were being underserved.
A question about whether or not the trials that involve cells are going to have controls. The answer is no, at least not in Phase I. What will happen in Phase II?
In some studies (ProChord) they've hidden the information from the evaluators. It's always a tension between the need to do the science right and the need to treat patients ethically.
Woman says that the VA hospitals are looking for study subjects.
Man says that the Portugal trial has been through the EU version of the FDA, and that an IRB does exist there, and at least one paper has been published about the results. He's a rehab doctor at Wayne state university. Panel member replies that the paper had flawed data, as would be clear to anybody with a statistical background. He says they also have claimed no adverse effects, but he personally knows of 6 very adverse effects. He's worked with people who got the Lisbon treatment, came back for intense PT and compared them with people who just got the PT . . . no difference. NADA.
Another panelist says that, you know, here we are with 2 qualified people looking at the same data and coming to a different conclusion. (That is what I hate.)
Woman whose daughter has been injured for 18 years says that she's seen rats walking, for heaven's sake. How on earth do you all come together? Panelist says that his favorite annual meeting is the ASIA meeting. They do learn from one another there. Also the journal Spinal Cord Injury. Also the peer review process. Also the NIH funding process itself, which is a marker to show that this work is new, promising, and original.
Woman asks the assessment measures guy what's being done with his work . . . he says that it will let small changes be documented and approaches be validated.
She says, could this assessment tool be used to convince insurance companies to pay for treatments like rehab?
He says he can't imagine that it wouldn't . . . and people should understand that to bring a group of combined treatments to the FDA -- oh, wait. Naomi says all they need is to show that their is safety in each of the individual components.
Oy, long day, people. Marilyn says it's time for a little socializing. Yay! Plus we get a buffet -- a Mexican buffet! Woo hoo, seriously.
Peter Gorman, University of Maryland
Sorry, I missed some information trying to solve a computer issue. Peter is talking about the need to publish every result, positive or negative.
Specifically, the HP184 Phase IIb Proof of concept study was not published because it did not meet the primary goal of improving the ASIA score.
(So, in the light of what Dr. McKay said, should we understand that perhaps if there had been a better, more precise, scale of measurement the outcome might have been positive?)
Another example . . . the ProChord Trial was stopped in Phase II abruptly (over the weekend!) in 2006 because PreNeuron was depending on money from Big Pharma -- Big Pharma pulled the plug quite suddenly and that was the end of that. Mid-study, and nobody knows exactly what they were finding out. Seems like that would be good information to have, eh?
Talking now about a couple of orphan products -- not enough market to keep these companies viable. The free market system adds a level of complexity. Could other companies be at risk? The one that makes the Lokomat, for example?
What are we supposed to do about this? How do we deal with complexity?
1. continue basic research
2. publish ALL results, even the negative ones
3. concinue to reevaluate outcome measures
4. create new outcome measures
5. expand the market; look at collateral disease states
6. continue funding from the government, foundations and industry
7. stick to good science
8. do multicenter collaboration
9. get on a regulatory fasttrack
10 focus on realistic hope
donna is introducing panel that will include a man from Acorda, who I hope will explain where time-release 4AP is!
Specifically, the HP184 Phase IIb Proof of concept study was not published because it did not meet the primary goal of improving the ASIA score.
(So, in the light of what Dr. McKay said, should we understand that perhaps if there had been a better, more precise, scale of measurement the outcome might have been positive?)
Another example . . . the ProChord Trial was stopped in Phase II abruptly (over the weekend!) in 2006 because PreNeuron was depending on money from Big Pharma -- Big Pharma pulled the plug quite suddenly and that was the end of that. Mid-study, and nobody knows exactly what they were finding out. Seems like that would be good information to have, eh?
Talking now about a couple of orphan products -- not enough market to keep these companies viable. The free market system adds a level of complexity. Could other companies be at risk? The one that makes the Lokomat, for example?
What are we supposed to do about this? How do we deal with complexity?
1. continue basic research
2. publish ALL results, even the negative ones
3. concinue to reevaluate outcome measures
4. create new outcome measures
5. expand the market; look at collateral disease states
6. continue funding from the government, foundations and industry
7. stick to good science
8. do multicenter collaboration
9. get on a regulatory fasttrack
10 focus on realistic hope
donna is introducing panel that will include a man from Acorda, who I hope will explain where time-release 4AP is!
Dr. Naomi Kleitman from the NIH
Title of her talk is Getting On With Clinical Trials
What's the process? She's trained in basic science . .. we heard this morning that there are lots of potential targets. Slide is showing all the good targets, including neuroprogection, replacement of lost cells, repair of axons to promote regeneration and remove inhibition, remyelination, and retrain remaining circuits through rehab.
So many good targets!
And it's not care vs. cure anymore . . .they have to go together because rehab will be part of whatever works.
So, is walking the realistic target? Go look at clinicaltrials.gov -- of the 238 trials listed in sci at that website, what are they addressing?
sensory, bladder and bowel, sexual function, spastiticy, brething, pain, pressure ulcers, cardiovascular conditions. Her point is that we're somewhere along the path -- we're not het able to translate.
Ack! Image of a rat spinal cord and a human one, both kind of hemp-colored; against a purple background (image came fromDr. Bruce Dobkin, going to try to find it so you can see.)
The point is that what workds in the skinny little rat cord might be worthless in the human cord. Issues include the length of the cord, the size of the lesion, anatomy of each, and functional things, like walking on 2 leg vs. on 4.
Some fundamental questions -- neurological disorders are complex, and we need complex strategies to deal with them. The thing that researchers have to confront is, how much information is enough???
And there is disagreement about that among scientists themselves. There's no gold standard for cellular therapies and very little consensus about what that would be. How much recovery in animals justifies tests in humans?
Okay, so what is translation? It's changing one thing to another (thought to action) or carrying something from one place to another (bench to bedside)
It's not about doing a trial that isn't anything like what happened in the lab, and not using a therapy without some kind of rigorous assessment of what you knew.
What are the targets again?
neuroprotective strategies, neurorepair strategies (anti-nogo, NgR, cethrin, rolipram) -- you have to know what the outcomes mean.
The basic flow from thought to trial is:
ID the target, do an assay, do screening, establish proof of concept with animal models, do preclinical testing, do safety studies, take it to the clinic
she's talking about the difference between clinical reearch and clinical trials. Clinical research is basic science done with humans or human tissues, like epidemiology, physiology, pathology. Clinical trials test treatments in people for safety, efficacy and compared to other treatments.
What the FDA does is define three phases in the testing process. 1 is for safety, 2 is for safety and bioactivity, and 3 is for efficacy.
There was a spinal cord injury locomotor trial done recently on people with relatively new injuries. The trial "failed" because everybody walked and everybody in the control group walked, too!
Joking about how dumb some trials are with a phony randomized trial to see if it's dangerous to jump out of a plane with no parachute.
When will clinical trials begin?
They already have. There are currently 118 sci trials recruiting at clinicaltrials.gov
She says she can't talk about anything she knows about pending grants -- and asks us not to believe rumors that nothing is ever going to be tested.
There's a lot going on right now in sci -- more than in brain or stroke. the key is to figure out how to measure outcomes.
ICCP, ASIA/ISCoS, SCOPE, SCIQOL, EM-SCI, China SCINet, CDRF NOA, NETT . . . these alphabet soup nuggets are about planning collaborations. When I have some time I'll go find em and put the hyperlinks in.
Don't be fooled by clinical trials with bad designs. The problem is that when a design is bad you won't be able to tell if the drug failed or the trial failed. What you want from trials is reliable information.
She's talking about the icord document that tells people how to know whether or not they should think about enrolling.
Why does the FDA delay Phase I trials? Safety, safety, safety.
Getting to translation is hard. But we know much more than we ever have before, and my grad school advisor was wrong when he said that "spinal cord is boring."
What's the process? She's trained in basic science . .. we heard this morning that there are lots of potential targets. Slide is showing all the good targets, including neuroprogection, replacement of lost cells, repair of axons to promote regeneration and remove inhibition, remyelination, and retrain remaining circuits through rehab.
So many good targets!
And it's not care vs. cure anymore . . .they have to go together because rehab will be part of whatever works.
So, is walking the realistic target? Go look at clinicaltrials.gov -- of the 238 trials listed in sci at that website, what are they addressing?
sensory, bladder and bowel, sexual function, spastiticy, brething, pain, pressure ulcers, cardiovascular conditions. Her point is that we're somewhere along the path -- we're not het able to translate.
Ack! Image of a rat spinal cord and a human one, both kind of hemp-colored; against a purple background (image came fromDr. Bruce Dobkin, going to try to find it so you can see.)
The point is that what workds in the skinny little rat cord might be worthless in the human cord. Issues include the length of the cord, the size of the lesion, anatomy of each, and functional things, like walking on 2 leg vs. on 4.
Some fundamental questions -- neurological disorders are complex, and we need complex strategies to deal with them. The thing that researchers have to confront is, how much information is enough???
And there is disagreement about that among scientists themselves. There's no gold standard for cellular therapies and very little consensus about what that would be. How much recovery in animals justifies tests in humans?
Okay, so what is translation? It's changing one thing to another (thought to action) or carrying something from one place to another (bench to bedside)
It's not about doing a trial that isn't anything like what happened in the lab, and not using a therapy without some kind of rigorous assessment of what you knew.
What are the targets again?
neuroprotective strategies, neurorepair strategies (anti-nogo, NgR, cethrin, rolipram) -- you have to know what the outcomes mean.
The basic flow from thought to trial is:
ID the target, do an assay, do screening, establish proof of concept with animal models, do preclinical testing, do safety studies, take it to the clinic
she's talking about the difference between clinical reearch and clinical trials. Clinical research is basic science done with humans or human tissues, like epidemiology, physiology, pathology. Clinical trials test treatments in people for safety, efficacy and compared to other treatments.
What the FDA does is define three phases in the testing process. 1 is for safety, 2 is for safety and bioactivity, and 3 is for efficacy.
There was a spinal cord injury locomotor trial done recently on people with relatively new injuries. The trial "failed" because everybody walked and everybody in the control group walked, too!
Joking about how dumb some trials are with a phony randomized trial to see if it's dangerous to jump out of a plane with no parachute.
When will clinical trials begin?
They already have. There are currently 118 sci trials recruiting at clinicaltrials.gov
She says she can't talk about anything she knows about pending grants -- and asks us not to believe rumors that nothing is ever going to be tested.
There's a lot going on right now in sci -- more than in brain or stroke. the key is to figure out how to measure outcomes.
ICCP, ASIA/ISCoS, SCOPE, SCIQOL, EM-SCI, China SCINet, CDRF NOA, NETT . . . these alphabet soup nuggets are about planning collaborations. When I have some time I'll go find em and put the hyperlinks in.
Don't be fooled by clinical trials with bad designs. The problem is that when a design is bad you won't be able to tell if the drug failed or the trial failed. What you want from trials is reliable information.
She's talking about the icord document that tells people how to know whether or not they should think about enrolling.
Why does the FDA delay Phase I trials? Safety, safety, safety.
Getting to translation is hard. But we know much more than we ever have before, and my grad school advisor was wrong when he said that "spinal cord is boring."
Peter Wilderotter and Susan Howley from CDRPF
Peter says the big new thing is the establishment of CR foundation satellites around the USA. It will start in here in Chicago.
Our knowledge manager Sam Maddox is even wearing a suit, so we know that this must be an important occasion. He's acknowledging lots of local people here in the room who are already part of the program . . . also recognizing Marilyn, Sue, and Betheny. (Hey, Peter, you must get to know Donna!)
The path that got us here is not going to get us were we need to go . .. there's a quote up on the screen: "For everyone who thought I couldn't do it, for everyone who thought I shouldn't do it,for everyone who said I didn't ahve it in me, see you at the finish line!"
Dana said that having sci enter your family was landing on the moon without a map.
Mike Mangianella was in charge of sorting the mail, and there were 3 bins. The normal people, the people who might be crazy, and the people who were definitely crazy. (Not exaggerating here. People sent him kryptonite so he could be healed.) One of the middle bins got the letter from Joan Irvine Smith offering to send a million dollars because she was so pleased that he didn't shoot the horse that threw him. Her money started what became the RIRC.
Slide up about the history:
1982 was the beginning. Scientific dogma was that the cord could not be repaired. Patients were seen as a tiny isolated who had to stay on the sidelines. There was no awareness, and no collaboration.
Their thought at CDRF was that we can't get to the finish line on the same path that got us here. The path to the future has to be strategic.
Who is our community? 1,275,00 Americans living SCI, plus the newly injured, the families, caregivers, and friends, the health care providers, the scientists, and society at large.
He's introducing Susan Howley. The head of the ACLU is not a lawyer. How can that be? The constitution is too important to be left to the lawyers. And sometimes the science is too important to be left to the scientists. Susan has become the person in charge of not leaving it to the scientists.
She gets up to thank Peter and says that she just left the most gorgeous beach with the best weather ( a joke about the rain we have here) to be with us. She started working in this field in 1983 for a small family research foundation that started in New Jersey.
They gave $25,000 each to a couple of Columbia scientists who were into brain research.
So, while we're still an infant field, we've come an extraordinary distance in the last 26 years.
She wants to tell us how the Reeve foundation funds scientists; she's a herder of cats.
There are a couple of board members who have got their fingerprints all over the systems we have in place. It's a stepwise, dynamic continuum.
First is individual grants, which go to new knowledge and new talents. They funded Stephen Davies' original work on astrocytes, for example.
Next is an international research consortium, which is a multi-disciplinary collaborative approach to SCI repair.
Next is the NeruoRecovery Network, which delivers activity-based rehabilitation interventions to SCI patients
Finally, the North American Clinical Trials Network.
How do individual grants work? the mission is to cast a wide net and fund the development of treatments for paralysis and the other dysfunctions that result from sci
since 1982: a total of 610 labs funded worldwide with $47 million
since 2002: 202 projects with $25,000,000
They look for quantifiable ROI, including publications, subsequent funding, trainees, patents
Their grants cover the whole spectrum. neuroprotection, regeneration, growth inhibition, remyelination, axon guidance, rehabilitation, dysfunctions like bowel, bladder, pain and so on.
They hold a spinal cord symposium to promote dialgue between Reeve'sindividual grantees and members of the sci community, foster exchanges of data and ideas between the two groups, and facilitate collaborations among the researchres.
They also give small network collaboration grants, which are highly leveraged funding that supports collaboration.
The member labs of the consortium include the Miami Project, UCLA, Cambridge, The Salk Institute, State U of New York at Stony Brook, University of Zurich -- the core labs are The Salk Institute and UC Irvine.
In the Neuro REcovery Network, they create standardized, evidence based, activity based therapies. There are centers in Louisville KY, Philadelphia, Houston, Atlanta, Columbus, OH, Boston, and WEst Orange, NJ. They're also adding what they call Facilities (as opposed to centers), and one of them is even west of the Mississippi!
The clinical trials network is about getting to the bedside. This morning someone asked why there aren't more clinical trials . . . she says there have been trials, but they haven't worked. One problem with these earlier trials was that we had very clunky outcome measures -- they may have been effective therapies, but the outcomes were not measured very well.
The centers are in Texas (4), Virginia, Maryland, Ontario, Kentucky, Florida, Pennsylvania, Washington DC
Showing a complicated bar graph that measures number of research projects against project start date, with each bar broken into 9 possible segments. What jumps out is a huge drop in projects funded in the year 2006, along with the disturbing size of the segments that indicate "complications" in projects. (I'd love to see the data behind this graph.)
Peter is back . . . so, how do we get to the future?
1. The power of one (alliance-building, collaborations, elimination of duplicative efforts)
2. The power of the truth (striaght talk, expectation setting, replacing hype with hope)
3. The eternal mystery of the world is its comprehensibility (Albert Einstein) We need rigorous science and clinical trialing, along with a multi-faceted approach to repair. There's no magic bullet, no magical scientist or doctor, and no quick fix. We need strategic, science-driven decision-making.
He's quoting Ted Kennedy: The dream still lives . . .
He says that Sue and Marilyn and Betheny told him about going to meetings with people who were in the community but not interested in the word "cure" -- gives the example of early 1900's cancer situation. People used to be ashamed to use the word.
Wrapping is up, time for lunch.
Our knowledge manager Sam Maddox is even wearing a suit, so we know that this must be an important occasion. He's acknowledging lots of local people here in the room who are already part of the program . . . also recognizing Marilyn, Sue, and Betheny. (Hey, Peter, you must get to know Donna!)
The path that got us here is not going to get us were we need to go . .. there's a quote up on the screen: "For everyone who thought I couldn't do it, for everyone who thought I shouldn't do it,for everyone who said I didn't ahve it in me, see you at the finish line!"
Dana said that having sci enter your family was landing on the moon without a map.
Mike Mangianella was in charge of sorting the mail, and there were 3 bins. The normal people, the people who might be crazy, and the people who were definitely crazy. (Not exaggerating here. People sent him kryptonite so he could be healed.) One of the middle bins got the letter from Joan Irvine Smith offering to send a million dollars because she was so pleased that he didn't shoot the horse that threw him. Her money started what became the RIRC.
Slide up about the history:
1982 was the beginning. Scientific dogma was that the cord could not be repaired. Patients were seen as a tiny isolated who had to stay on the sidelines. There was no awareness, and no collaboration.
Their thought at CDRF was that we can't get to the finish line on the same path that got us here. The path to the future has to be strategic.
Who is our community? 1,275,00 Americans living SCI, plus the newly injured, the families, caregivers, and friends, the health care providers, the scientists, and society at large.
He's introducing Susan Howley. The head of the ACLU is not a lawyer. How can that be? The constitution is too important to be left to the lawyers. And sometimes the science is too important to be left to the scientists. Susan has become the person in charge of not leaving it to the scientists.
She gets up to thank Peter and says that she just left the most gorgeous beach with the best weather ( a joke about the rain we have here) to be with us. She started working in this field in 1983 for a small family research foundation that started in New Jersey.
They gave $25,000 each to a couple of Columbia scientists who were into brain research.
So, while we're still an infant field, we've come an extraordinary distance in the last 26 years.
She wants to tell us how the Reeve foundation funds scientists; she's a herder of cats.
There are a couple of board members who have got their fingerprints all over the systems we have in place. It's a stepwise, dynamic continuum.
First is individual grants, which go to new knowledge and new talents. They funded Stephen Davies' original work on astrocytes, for example.
Next is an international research consortium, which is a multi-disciplinary collaborative approach to SCI repair.
Next is the NeruoRecovery Network, which delivers activity-based rehabilitation interventions to SCI patients
Finally, the North American Clinical Trials Network.
How do individual grants work? the mission is to cast a wide net and fund the development of treatments for paralysis and the other dysfunctions that result from sci
since 1982: a total of 610 labs funded worldwide with $47 million
since 2002: 202 projects with $25,000,000
They look for quantifiable ROI, including publications, subsequent funding, trainees, patents
Their grants cover the whole spectrum. neuroprotection, regeneration, growth inhibition, remyelination, axon guidance, rehabilitation, dysfunctions like bowel, bladder, pain and so on.
They hold a spinal cord symposium to promote dialgue between Reeve'sindividual grantees and members of the sci community, foster exchanges of data and ideas between the two groups, and facilitate collaborations among the researchres.
They also give small network collaboration grants, which are highly leveraged funding that supports collaboration.
The member labs of the consortium include the Miami Project, UCLA, Cambridge, The Salk Institute, State U of New York at Stony Brook, University of Zurich -- the core labs are The Salk Institute and UC Irvine.
In the Neuro REcovery Network, they create standardized, evidence based, activity based therapies. There are centers in Louisville KY, Philadelphia, Houston, Atlanta, Columbus, OH, Boston, and WEst Orange, NJ. They're also adding what they call Facilities (as opposed to centers), and one of them is even west of the Mississippi!
The clinical trials network is about getting to the bedside. This morning someone asked why there aren't more clinical trials . . . she says there have been trials, but they haven't worked. One problem with these earlier trials was that we had very clunky outcome measures -- they may have been effective therapies, but the outcomes were not measured very well.
The centers are in Texas (4), Virginia, Maryland, Ontario, Kentucky, Florida, Pennsylvania, Washington DC
Showing a complicated bar graph that measures number of research projects against project start date, with each bar broken into 9 possible segments. What jumps out is a huge drop in projects funded in the year 2006, along with the disturbing size of the segments that indicate "complications" in projects. (I'd love to see the data behind this graph.)
Peter is back . . . so, how do we get to the future?
1. The power of one (alliance-building, collaborations, elimination of duplicative efforts)
2. The power of the truth (striaght talk, expectation setting, replacing hype with hope)
3. The eternal mystery of the world is its comprehensibility (Albert Einstein) We need rigorous science and clinical trialing, along with a multi-faceted approach to repair. There's no magic bullet, no magical scientist or doctor, and no quick fix. We need strategic, science-driven decision-making.
He's quoting Ted Kennedy: The dream still lives . . .
He says that Sue and Marilyn and Betheny told him about going to meetings with people who were in the community but not interested in the word "cure" -- gives the example of early 1900's cancer situation. People used to be ashamed to use the word.
Wrapping is up, time for lunch.
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