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Thread: Live from Working 2 Walk 2011!

  1. #51
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    Thanks so very much Kate!! NRF, where are you located? Would love to be able to access your gym. I want to use FES but right now that means I must purchase something for my home or travel 90 miles one way. How does one buy a bike for themselves? I have a family of 5, one in college, self employed, etc. Is there funding oppourtunites for me?

  2. #52
    Senior Member kate's Avatar
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    International Panel: Dennis Tesolat, Martin Codyre, Harvey

    Martin: Thanks to the organizers. This is my 3rd time, and I was injured 3 years ago . . . this is a unique event in the world & this is a great opportunity for us all. When I first came here, there was a father who had a child with an sci, and he said to the doctors: if I could help my daughter to regain function I'd cut off my own leg. Are you willing to do that, too? And the doctors said, no, sorry, I'm not committed at that level. Might be an apocryphal story, but it's about the level of commitment from everybody in this room.

    Here's my take, 3 years in . . . we've gone from the possibility of one trial to having competing trials, which is an incredibly positive thing to have happened in just three years, and we're looking at combination therapies. It's going to be very hard to them clinically tried . . . but I'm more positive about seeing therapies for us than I was at the beginning. CNS is in some senses the low hanging fruit. You can see outcomes very well, which is not the case with, for example, Parkinsons. Investors are interested.

    On the negative side, I've been really quite shocked to learn that there have been therapies, science in the papers, that might have helped some of us, including myself. If the Miami Project could say to me that hyperthermia doesn't work, I could take that, but just being in this situation because it was never given a rigorous trial is very hard. I'm also aware of the difficulties of bringing a product fully to market . . . $400 to 500 million to get to phase III trial in North America. That's frightening. Also the timing issues . . . 7 years to a saleable product, according the Geron representative who spoke yesterday. it's such a long, expensive road. Finally, the lack of true cohesiveness in the community has been quite shocking to me. We need to come together more at events like this.

    Harvey Sihota (from London): This is my first event and I've found it very eye opening . . . didn't sleep very much last night. Thinking about this panel and what we should say. My own background is in business and finance, so I'm daunted by the regulatory and financial barriers . . . are there creative solutions? Offshoring Phase I and II trials . . the fda is the fda is not a good answer for myself, and maybe not for you either. I'm interested in thinking outside the box.

    Dennis: This is the first time I've ever done a think like this over skype, so we'll see how it goes. I wanted to talk about our responsibility for a cure. Lots of people are wondering whose fault it is that we're still waiting. I want to talk about what we can do to support a cure. We can support the scientists, we can question organizations that talk about cure all the time. We can ask them hard questions . . . we started a campaign toward the Rick Hansen foundation, hoping they'd have a lot of things to share about how they're bringing us the cure.

    We didn't get an answer. We weren't trying to bring shame to anyone, we just wanted to know. I'm Canadian, so of course Rick Hansen was the person I looked to from the beginning. I think anybody who claims to be supporting a cure deserves our support -- but only if they're really doing it. What we need to do is

    Martin: Thanks to Dennis for raising that eloquent campaign online. We haven't really gotten a good answer yet, but we still want one. If anybody's listening, please help us out.

    Some major organizations that use the word "cure" in their promotional material:

    --Rick Hansen gets $25 million a year from federal funding and private donors, which they're using maybe 2 or 3 mill on restorative
    --CRDF raises $15 mill and spends about half on restorative research. They've got the solid brand, the best position to push for faster cures, and they're not taking full advantage of it.
    compare to MJ Fox, which raised $40, almost all of which went to Parkinsons research. Why does CRDF not do the same?
    --The Spinal Research Trust in the UK raises $3 mill per year, all of which goes to restorative research.
    --Wings for Life, basically the same kind of numbers.

    Q: from where I sit I see a lot of things wrong. Martin, you remember that 2 years ago at Wings for Life, I saw nothing for chronic injuries. I got mad. We see a lot of progress in the last few years, but it seems like about 30% of what we COULD have seen. We could be much further along toward a cure, which I define as getting out of the chair. We can't be happy just with bowel and bladder, not that we all don't want that. We have to take what we can in the short term but look toward the long. We need to push for a big change, or we'll all going to die in these chairs. Just start the clinical trials with Chase. We need to take the bull by the horn if we want to walk.

    A: Harvey -- two points. One is that everybody wants scientific rigor, but unfortunately it comes with politics and bureaucracy, which we need to get rid of. The other is the translation to the clinic. We don't see an appetite to create a product that we can use. Once in a while someone like inVivo comes along and tries to cross that valley. Our organization is interested in working with people like that.

    Martin: the model of relying on companies to get us from bench to bedside is problematic. If it takes $500 million to show that a therapy works, and the job of the fda is to make sure that it's done safely . . . but what that turns out to mean is a lot of nothing.

    Dennis: I think that what we're going to do is take up our own part of the job. What is it? If you're paralyzed and you have a part time job, your full time job is moving this along. If you're paralyzed and you have a full time job, your part time job is moving this along.

    Audience: a lot of us are speaking about hope, and that's awesome, but we need to talk about action. I hear that there's a lack of collaboration. I hear that it's not a function issue . . . I don't understand, that's very confusing. I heard Jerry Silver say "I'm fine." Well, I'm happy that you are fine, but I am not fine. You said that switching to a clinical trial is another story. Does that mean that you are not the one who is going to trigger a trial? Who is going to do that? I believe you and you seem to believe in what you do . . .I'm not here to criticize, but I want to know how we get well. Let's start with a goal: we want clinical trials in two years and manage toward it. Another side would be to manage the program as a whole . . . there's much more need for collaboration and an integral approach. How can this community take this forward? What is the next step?

    Jerry Silver: I think what you've already done, bringing scientists and companies together in one meeting is a huge step. You may not realize it, but having Tony and I sitting here next to each other is enormous. Acorda might be motivated to switch its focus . . .they're making a lot of money now, and I believe they may have enough to move this along. What we need is a kind of Wise Young to move Chase into trials . . . he spends an enormous amount of time traveling, selling, educating . . . that's why he's got a trial going and we don't.

    Martin: What you said about this particular event bringing people together is very, very true. I know of scientists and other people from business who've made similar connections. To be honest, I was flabbergasted that this doesn't happen ALL the time . . this is a catalyst for the future. Every time I've come here I've seen more and more.

    Audience: It does seem like we've got a lot of things going on that are not as coordinated as they could be . . . again, thanks to the organizers of this event for making this possible. It's fantastic. We need to form a community that allows all of us, USA and internationally, to choose a path and figure out what to support. There's competition in academia, in the corporate world -- we need to figure out creatively how to overcome it. So many things don't seem to build on one another. We need to form a group around this core at u2fp that does coordinated, cohesive advocacy.

    Tansey: It's all about money when it comes to healthcare payers. One thing that's hard is the difficulty of translation. First in Human Research is something you should look at . . . it's not just the fda that's the barrier. Think about assisted suicide; the idea there is that people can and should take autonomy in terms of their own lives. You could make the case that there would be an ethical way to create that option for spinal cord injury. You'd want to proceed very cautiously and ethically, but it bears examination.

    Martin: We will take that message with us to the Capitol. And here's another thing -- people in chairs are travelling all over the world, spending many thousands of dollars and NOT advancing science at all. We're already taking our lives and putting them into the hands of charlatans, so that decision has been made.

    ----------

    Just my own opinion, but that conversation was an astonishing instance of what u2fp has been after for the last 5 years . . . you need to imagine a half a dozen people sitting in a living room in suburban Minneapolis, dreaming that somehow it might be possible to create a space where people would see that advocacy matters, that it's doable for everybody, and most of all that it's probably the ONLY way we're ever going to get anywhere. It's a long way from there to here.
    Last edited by kate; 10-20-2011 at 01:59 PM.

  3. #53
    Quote Originally Posted by kate View Post
    All the guys from this morning's presentations are taking questions from the audience . . .


    Q: In regards to FES for caudaequinal injuries . . . any new ideas? I did the FES bike for months and only got flickers.
    A: (McDonald)All CE injuries are not complete and nor are they all the same. There are things that can be done in order to move you forward, to get those muscles to contract --
    (Tansey) If you have nerve cells, you can use classic FES, if you don't, you can use another kind of stimulation -- and this is testable so we know which it is.

    Q: With regard to the locomotor plus stimulation training studies , what was the average duration of the studies?
    A: (Tansey) 3 times a week, 1 hour a day, for 3 months
    So they do not say what kind of stim is there other then FES? How do you get tested? Pretty vague answers

  4. #54
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    Heidi Marchand: FDA Ass't Commissioner for Special Heath Issues

    I'm pleased to be here this afternoon; my first time speaking to this group. I'm somewhat surprised to see that it's as large as it is. I'm looking forward to talking with you individually.

    Today's objectives:
    1. understand the basic structure of the fda and the centers responsible for human therapeutic products

    2. learn about fda's office of special health issues as a point of contact for patients and patient advocacy organizations

    3. discover opportunities for patients and patient advocacy organizations to engage with fda

    Health and Human Services is a cabinet-level department under the white house; the president appoints the head, who is confirmed by the congress and the senate. Within HHS is CDC, FDA, NIH, and Centers for medicare and Medicaid Services.

    The FDA has 13,586 full time employees and a budget of $4B ($1.58 of that is in user fees)

    NIH has 18, 784 FTEs and a budget of $32B

    CDC has 9,753 FTEs and a budget of a measly $735M

    Centers for Medicare and Medicaid has 4869 FTEs and a budget of $511B

    The FDA's current leader is Obama appointee Margaret Hamburg, MD
    She has 8 major offices reporting to her, one of which is the office of external affiars, which contains the office of special health issues.

    Things get approved in one of 5 centers for drug development. Slide up of early '80s AIDS advocates in a big rally, holding a sign that said TIME ISN'T THE ONLY THING THE FDA IS KILLING. It got their attention, apparently, and let to the creation of the Office of Special Health Issues. This woman leads that group.

    They have a group of patient liaisons and another of healthcare professional liaisons . . . what are the mechanisms for public input?

    There are public advisory committee meetings, other public hearings, and individual communications. They get involved in trying to understand what the messages are with respect to biologics, drugs, and devices. They take what they learn and communicate with patient groups of all kinds . . . we could get on an email list that would feed us information about new drugs, safety, or initiatives the fda might be undertaking. The fda can't speak specifically about any individual product or company, because these matters are confidential.

    They can't say why something is on hold, but they can say what sorts of things reviewers consider before something gets put on hold.

    Drug development process . . . before humans can take it:
    1. Quality assurance -- synthesis, purification, and stability
    2. Non-Clinical -- two species (rodent plus something else) testing on starting dose, carcinogenicity

    That part of things takes 2 or 3 years. Now you're into human trials, which is what the IND is for.

    3. Phase 1,
    4. Phase 2
    5. Phase 3

    Should have 1,500 patients, unless it's designated orphan or fast track. She's seen one trial that had only 150 patients. This takes 5 to 7 years. Then if everything goes well, you get an NDA filed to get FDA approval, which can take up to 20 years. And only 1 in 5 drugs makes it.

    That last sentence seems wrong, but I'll check with her when we get to the panel. Even without it, this is pretty much blech.

  5. #55
    Senior Member kate's Avatar
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    Naha Bora, DOD, Director of Congressionally Directed medical Research Programs

    CDMRP is part of a DOD group that targets research as directed by congress; since 1992, they've administered more than $6B in congressional appropriations.

    Their goal is to foster high-risk, high-impact types of research. They assess the current state of science to accommodate rapid change. They use a two-tier review, where every proposal that passes scientific review gets a second review for mission relevance.

    Grants have been given for several kinds of cancer, ms, and . . . sci.

    The have partnerships with advocates, congress, researchers, and the DOD. There's a program cycle that goes like this

    vision setting
    Release of Program announcement
    Pre application receipt
    Pre application screening
    invitation to submit
    application receipt
    peer review
    programmatic review
    commanding general approval
    candidate award list
    negotiations, contracting, regulatory, program issues
    grant award

    This guy oversees this entire process, and it's not just giving awards in the usa; 23 countries have been given grants from all over the planet.

    We support awards at differing stages of careers
    Support individuals and teams
    Support innovation
    Support Translation Research

    They got started because of breast cancer advocates and have made extensive use of consumer reviewers.

    How do consumers participate? They're in most of the steps I just named above. If you want to do this, there's an entire section dedicated to you at the website . . . which I'll get a link to in a minute.

    The sci research program is relatively new. The mission is to fund innovative and interdisciplinary research and foster collaboration . . . getting to the clinic.

    In 2011 they have 17 million dollars and plan to use it on about 11 awards.

    Last year they gave grants for implementation research -- development of methods or approaches that would get things to the clinic, and to developing guidelines.

    He's showing slides that detail how much money, where it's going or has gone, what area it's focused on. The peer review for the current grants will happen in January, a couple of months from now . . . you can volunteer to do this.

    $750k for clinical trial in rehab
    500k for investigaor initiated
    400k for qualitative research award to help create studies that get researchers to understand issues better
    750k for 2 or 3 principal investigators to colloborate with a t least 1 clinician and1 researcher

    Kenneth Curley is the chair of the org, and he has an sci.
    link

  6. #56
    Senior Member kate's Avatar
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    Panel: Heidi Marchand, Naba Bora

    Q: What's fast track at the fda?
    A: It's for those conditions that are life-threatening and have no other alternative therapies.
    Q: Does it actually make things faster?
    A: Well . . . the theory is that it would lower the development time, but not necessarily testing time. You would also want to request a priority review.

    Q: Who has won the DOD grants in the past, and how do you follow up with them?
    A: Everyone who gets an award must give an annual update, and those things are posted online. We track their spending quarterly. We now have every one of the 70-odd projects listed on our website.

    Q: Heidi, I'm really disappointed that we didn't get to finish your presentation because a lot of us need to hear more about the fda . . . is there someone in your office who is focused on sci, and if so how do we track?
    A: The person in my office is David Banks; he would be the one who could answer questions about topics unique to spinal cord. We have a patient representative program and we have 165 patient representatives, and that program is on our website if you want to apply. One of the things they would evaluate is your potential conflict of interest. You'd have to mess with a lot of paperwork, but it's a very worthwhile thing to do.

    Q: Heidi, one thing we're always talking about is that a cure for sci will most likely involve combination therapies. How is the fda going to deal with that?
    A: fda is not doing science, we're just evaluating it . . . one criteria that's always a point of some discussion is this: what's the endpoint that demonstrates success? What's a complete win, what's a partial win? The other question is how to design trials that involve more than one drug . . . patients do need to push and show willingness to see such things happen. There's also statistical testing; if you're going to talk to congress, ask what's in the prescription drug user re act 5

    Q: As I see it today, we waste a lot of time trying to guess what will pass . . . can't we work together since the beginning, so that we don't have to have this back and forth, hold and release, wasted time.
    A: Well, (she goes through the process as it now stands)
    Q: No, no, the point is that what I've seen, for example, is the way the Geron thing went . . .
    A: Well, here's the problem, there's information that goes back and forth as you go along. You're still learning in the preclinical and that information has to feed into what happens next. (she's saying that the process does kind of work that way now -- the structure is a set of gates, yes, but it's a collaborative set of gates)

    Q: spilled my coffee, missed it . . . something about lost time due to employee turnover . . .
    A: Naba says not in my group; Heidi says that regulatory affairs work is not on any college curriculum. It's always learn on the job, which means that people who know how to do this are rare. At DOD, they always hire outside consultants from universities so turnover isn't an issue. At fda, once you're on an IND, you're going to stay on the same project for the NDA.

    Q: Naba, I'm one of the fundees, so I appreciate that you're here. I had a question about the IRB . . . we had to do that twice, once at our facility and once again in your organization.
    A: We do that because it's the DOD and we do our own research. It's policy, and we know it's a burden of 2 or 3 months, but we're trying to streamline it.

    Q: How does the fda sci specialist (David Banks) stay abreast of developments as they happen?
    A: These are well-trained scientific, medical doctor, Ph.D component, go to lots of conferences . . . they're exposed to lots of information . . . I know that you can't always keep up with every single thing, but they're very well prepared for what they do.

    Time for a break, then the last workshops. Oh, good.

  7. #57
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    Quote Originally Posted by momo3 View Post
    Thanks so very much Kate!! NRF, where are you located? Would love to be able to access your gym. I want to use FES but right now that means I must purchase something for my home or travel 90 miles one way. How does one buy a bike for themselves? I have a family of 5, one in college, self employed, etc. Is there funding oppourtunites for me?
    Sorry momo3 we're in Pennsylvania. RTI will sell direct, their phone # is 800-609-9166 but they're not cheap. I have heard of insurance coverage paying for them, the people at RTI can help try to get coverage.

  8. #58
    Jerry Silver: I think what you've already done, bringing scientists and companies together in one meeting is a huge step. You may not realize it, but having Tony and I sitting here next to each other is enormous. Acorda might be motivated to switch its focus . . .they're making a lot of money now, and I believe they may have enough to move this along. What we need is a kind of Wise Young to move Chase into trials . . . he spends an enormous amount of time traveling, selling, educating . . . that's why he's got a trial going and we don't.
    Why not a collaboration? China and the other SCI Networks were born like a platform to test therapies for chronic SCI, no?
    -Ramps in buildings are necessary, but it would be usefull to have another ones for people (mind/heart).....

    -Hoc non pereo habebo fortior me

  9. #59
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    Thanks nrf! My insurance is out. They pay for nothing! I guess I need to check back with RTI. FWI, didn't Dr. MacDonald help start this?

  10. #60
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    He was and is on the leading edge.

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