Miami Project announcement, 10 AM Today (Tuesday)

[Jim]

Log on to watch live-

http://www.themiamiproject.org/announcement?srctid=1&erid=2351933&trid=89a8e7fb-abd2-4f46-9bb0-58972216ea90

[NoDecafPlz]

They're putting in a jacuzzi!

(sorry)

I'd like to welcome...my barber, my first baby sitter...

Schwann cells.

[Jim]

FDA approved Phase I Safety Trial of Schwann Cell transplants in 8 sub-acute patients.

[Foolish Old]

Good News! But nothing to cause any SCI to start shopping for running shoes. Nor any reason to check the news every morning.

[#LHB#]

When is it suppose to start?

[Johnnie Walked]

about 5 years lol.


Good News ! it was about time

[#LHB#]

about 5 years lol.


Good News ! it was about time



That sounds about right. The bad thing is the one's that are going to make the choice to be in the trial dont even know it yeat. There in for a rude awakening.

[Jim]

They are starting human clinical trials, a big step.

[khmorgan]

Wow, I'm surprised. I thought they were already in Phase I trials. I looked back at their website and found the following:

The Clinical Trials Initiative is The Miami Project’s efforts to carefully take new discoveries from the laboratory to clinical trials. The Clinical Trials Initiative was started in 2004 and is aimed at creating the infrastructure needed to gain approval for and to conduct clinical trials at the University of Miami affiliated hospitals.

8 years and god knows how much money to get into phase I trials with 8 sub-acute patients. I think there is a lack of urgency there. Everybody's getting paid every week and are content with status quo.

I mean, I know UM is doing good work, but I'm thinking few if any of the researchers there expect to see a cure in their lifetime.

Thank God, we have Dr. Young! He's crazy enough to try to "cure" us in our lifetimes. Even better, he's working closely with people in China who agree with him.

[Johnnie Walked]

I agree Jim ! and for this kind of guys (MP) too start clinical trials, they must be very sure of their case

[NoDecafPlz]

China scinet research will probably lap them several times over.

[rjames]

Man I hate to be so negative but I heard from a reliable source 5 years ago that this therapy won't work. I think MP felt pressured to get "Something" into trials as others have done so in less time and with less money...Hope I'm wrong

[KofQ]

China scinet research will probably lap them several times over.

Wise and his team will have results from this Phase III study completed well before MP or StemCells Inc. even finishes Phase I, and likely well before CDRF, RIRC, or anyone else even applies to the FDA with anything for chronic SCI.

I think that if the ChinaSCINet trial results show the trend for efficacy that Wise suggests, there will be a lot of hand-wringing and rending of garments at some of these big SCI organizations. At least I hope so, because it will be well-deserved.

Up until today, the big trial idea from MP was "let's put ice on it."

[0xSquidy]

26 years and a gazillion of dollars raised, to "*start*" a clinical trial... on acutes... with a one bullet therapy... okay jose!

[rjg]

A scant two-and-a-half decades after the Miami Project was started by an NFL hall of famer in a country that worships football. Please ponder the implications of that before e-pissing on Wise, you dimbulbs.

[rjg]

I'll be the lead pisser if October comes and it turns out his open-house videos were BS. I just think we owe him the benefit of the doubt.

[swh2007]

Honestly, I have spent some time at the Miami Project and met Marc Buoniconti. I think he urgently wants a cure as much as anyone. That does not mean I have to agree or any of you have to agree with everything or anything the Miami Project does. It just means that I think they are trying.

[NoDecafPlz]

A scant two-and-a-half decades after the Miami Project was started by an NFL hall of famer in a country that worships football. Please ponder the implications of that before e-pissing on Wise, you dimbulbs.

What are talking about? We're all team Wise here, or most of us at least.

Who do you perceive e-pissing on Wise?

Miami project has not shown enough return on investment. I am not the only one that feels that way.

[c473s]

They are starting human clinical trials, a big step.
Yes it is. So far they have only Jackson in Miami as a recruiting site. With inclusion criteria similar to Geron's it will take a long time (years) to recruit 8 unless they find other sites to partner with.

[lynnifer]

Saw it somewhere else last night online ... I hope it works for them. They've been studying these cells FOREVER so I hope it's a slam dunk.

Regardless, it's nice to see trials coming out of MP finally. (In emerge 27yrs ago, "Oh kid don't worry, you'll be walking and running again in five years .. there's this great place in Florida called the Miami Project ... )

ETA: Their 2004 combo that apparently no one else could replicate:
http://scienceblog.com/2757/combo-therapy-dramatically-improves-function-after-spinal-cord-injury/

[micah_d]

What are talking about? We're all team Wise here, or most of us at least.

Who do you perceive e-pissing on Wise?

Miami project has not shown enough return on investment. I am not the only one that feels that way.


what specific changes do you feel MP should make to get faster results?

[rjg]

What are talking about? We're all team Wise here, or most of us at least.

Who do you perceive e-pissing on Wise?

Miami project has not shown enough return on investment. I am not the only one that feels that way.
Most of us are on Wise's side. But there are a few. No need to name names, as I'm too classy for such behavior. Paolo. Grammy. Christopher Paddington. Jerry Silver.

[rjg]

what specific changes do you feel MP should make to get faster results?
I know you didn't ask me, but here's a suggestion: fund more than one human trial every 25 years. 2 human trials every quarter century would double the odds that the Miami Project's millions will lead to something worthwhile.

[ineedmyelin]

Most of us are on Wise's side. But there are a few. No need to name names, as I'm too classy for such behavior. Paolo. Grammy. Christopher Paddington. Jerry Silver.

true dat!

[rjames]

I know you didn't ask me, but here's a suggestion: fund more than one human trial every 25 years. 2 human trials every quarter century would double the odds that the Miami Project's millions will lead to something worthwhile.

That's a good one!!!

[lynnifer]

Most of us are on Wise's side. But there are a few. No need to name names, as I'm too classy for such behavior. Paolo. Grammy. Christopher Paddington. Jerry Silver.

I don't think that's true or appropriate in this thread, but it's obvious you are more than likely welcome to your opinion.

DA was famous for challenging Wise. I miss him a lot. I appreciate we have more who don't accept blindly, but challenge as well. That's the crux of science isn't it? Dont' forget kivi! :)

[BSgimp]

I dont understand people on this forum. Unreasonable behavior seems to be a common place here. I ask why are you looking to argue? What is the point? You are not contributing to discussion with knowledge but with pointless bickering.

We have the theory here.. based on years of research and studies. We need to understand what it would imply, and then compare the theory to the experiment and experience. This of course being the trial. We compare it directly with the observation to see if it works. If it disagrees with the experiment its wrong. In that simple statement is the key to science. Now, it does not matter who came up with theory or who did the experiment. It can be Wise, Silver or G.W.Bush for that matter. What is important is the result and this is what we need to focus on. Nobody can know for sure what the result of experiments will be .. even the person behind the theory. Picking sides like children is pointless waste of time and energy. Lets focus on science itself. Questioning is also important part of science and extraordinary claims require extraordinary evidence. This is why we wont get any info from a scientist before the claim can be verified by experiment.

Now, the OP came with amazing news. New trial means we can verify if this therapy works. Even if it does not it mean we can scratch it of the list and learn from it.

[dan_nc]

From the FAQ about the study,
http://www.themiamiproject.org/page.aspx?pid=1047

Is this a first step in the cure for paralysis?
No - this is a Phase 1 safety study.


So there you have it. In their own words, this is not the first step in the cure for paralysis.

[Fly_Pelican_Fly]

As I understand it MP have leveraged lessons learned from the abandoned Geron study, including the device used for administering their hESC cells. To complete a safety study with any cell type being injected into the spinal cord under US FDA regulations would be a significant milestone. Many other organisations see this as a crucial step.

Do I think Schwann cells will cure chronic SCI? No. Do MP think that Schwann cells will cure chronic SCI - I hope not otherwise I will seriously doubt their credibility as a research group. I think and hope that this is just a first step towards realising a combinatory approach later with a number of other lines of science (namely Ch'ase). And if not, then we go after them ;)

As for their spending to date - well, that is another story. Put it this way, they are further ahead than Rick Hansen and CDRF in terms of getting a trial going in North America. They are not my favourite organisation, but there is no point bashing them now they have stuck their neck out and started a trial. That would be destructive.

One thing that MP can shout about is that they can run the trial within their big shiny facilities without worrying about other centres.

[swh2007]

As I understand it MP have leveraged lessons learned from the abandoned Geron study, including the device used for administering their hESC cells. To complete a safety study with any cell type being injected into the spinal cord under US FDA regulations would be a significant milestone. Many other organisations see this as a crucial step.

Do I think Schwann cells will cure chronic SCI? No. Do MP think that Schwann cells will cure chronic SCI - I hope not otherwise I will seriously doubt their credibility as a research group. I think and hope that this is just a first step towards realising a combinatory approach later with a number of other lines of science (namely Ch'ase). And if not, then we go after them ;)

As for their spending to date - well, that is another story. Put it this way, they are further ahead than Rick Hansen and CDRF in terms of getting a trial going in North America. They are not my favourite organisation, but there is no point bashing them now they have stuck their neck out and started a trial. That would be destructive.

One thing that MP can shout about is that they can run the trial within their big shiny facilities without worrying about other centres.


Their so called big shiny facility is just another medium (at best) sized medical building in a huge sea of medical buildings at the University of Miami Hospital. It does not seem big at all when you tour it. I am a big Dr Young supporter but I appreciate whoever is trying to find a cure. Just a comment about something I see as a misconception.

[Fly_Pelican_Fly]

Their so called big shiny facility is just another medium (at best) sized medical building in a huge sea of medical buildings at the University of Miami Hospital. It does not seem big at all when you tour it. I am a big Dr Young supporter but I appreciate whoever is trying to find a cure. Just a comment about something I see as a misconception.

I make no distinction between The Miami Project and University of Miami Hospital when we are talking about clinical trials. The clinical trial will take place at the hospital facilities no doubt.

[manouli]

more details.



Aug-01-2012


UM's Miami Project gets FDA approval for clinical paralysis trial


The Miami Project to Cure Paralysis received permission from the FDA to begin a Phase I clinical trial to evaluate the safety of transplanting human Schwann cells to treat patients with recent spinal cord injuries (SCIs). Found mainly in the peripheral nervous system, Schwann cells are essential to sending appropriate electrical signals through the nervous system, and Miami Project scientists and supporters believe they are key to finding cures for paralysis. [1] His father, Nick Buoniconti, is a Hall of Famer who played football for the Miami Dolphins and Boston Patriots. He has worked tirelessly since his son was injured to find a cure for spinal paralysis. The two are the heads of the Miami Project to Cure Paralysis that tries to help the thousands suffering from spinal cord injuries in the U.S. The Buoniconti's finally heard good news when the U.S. Food and Drug Administration approved a phase one trial to evaluate the safety of making transplants of human Schwann cells as a treatment for those with paralysis. It will be the world's first trial if its kind. One neurosurgeon on the team said the announcement today was as important for their field of study as was the first step taken on the moon to the NASA space program. [2] THE MIAMI PROJECT: In 1985, Barth A. Green, M.D., and NFL Hall of Fame linebacker Nick Buoniconti helped found The Miami Project to Cure Paralysis after Nick's son, Marc, sustained a spinal cord injury during a college football game. The Miami Project's Christine E. Lynn Clinical Trials Initiative will take discoveries found to be successful in laboratory studies and fast track them to human studies with the approval of the FDA. The Miami Project is well positioned and confident that we have the expertise, knowledge, and drive to navigate through the process and continue to initiate new clinical trials in the future. Since its inception, The Miami Project has worked carefully and diligently toward these goals and the results show that the time is right to make these important steps into humans. [3] "When we started The Miami Project, the short-term goal was to improve the quality of life of people living with paralysis, but the long-term goal remains re-establishing function and finding a cure. Today the scientists, clinicians, and technicians who have made this day possible have taken a giant leap forward. The hundreds of millions of dollars and incalculable hours of bench and clinical work invested in this goal have been well spent." "The FDA decision validates the commitment of a family who has turned their own tragedy into hope, and the vision of scientists who have never wavered in their quest to reverse the catastrophic consequences of spinal cord injury." Nick Buoniconti, Co-Founder of The Miami Project, said, "It has been 26 years since my family made a promise to Marc and all those living with paralysis that nothing will stand in the way of us finding a cure, absolutely nothing. FDA approval of this clinical trial is allowing us to follow through on this incredibly important commitment that impacts millions of families each day. This validation of the tireless efforts being undertaken at The Miami Project offers real hope and shows we are closer than ever to curing paralysis." [3]

read...

http://newsfeedresearcher.com/data/articles_m31_2/miami-cells-project.html


Spinal-Cord Injury Therapy OK'd by FDA Could Lead to Cures

Marc Buoniconti, got older like all of us.

http://abcnews.go.com/Health/spinal-cord-injuries-fda-approves-cell-regeneration-therapy/story?id=16889554

[swh2007]

I make no distinction between The Miami Project and University of Miami Hospital when we are talking about clinical trials. The clinical trial will take place at the hospital facilities no doubt.

There should be a distinction, I think. The University of Miami Hospital is a huge, urban health system and teaching hospital serving the entire Miami metro area and MP is just a small sliver of it. For example, the Diabetes Research Institute (aimed at tType I Diabetes research) has a similiar research facility right next to MP. Both the DRI and MP are basically privately funded by donors but are affiliated with the Health System, which as a teaching hospital I assume provides a source of professional researchers.

I have this little SCI and my teenage daughter is totally insulin dependent as a result of Type I diabetes. I have chosen to support others in my giving to find a cure for SCI and Type I Diabetes, but at the same time I appreciate the efforts of MP and DRI and their donors, especially since I know both of them are rather small organizations founded and in part funded by families and people with SCI and Type I, respectively. I know they are real stakeholders and the folks running them actually have the injury/disease. They care.

This is all I am saying. I am not really talking to you FPF.

[JimmyMack]

true dat!
Double Tru-dat

[ResidentBio]

This is what Kimberly Anderson had to say about this step.

The Miami Project to Cure Paralysis, a Center of Excellence at the University of Miami Miller School of Medicine, has received permission from the Food and Drug Administration to begin a Phase 1 clinical trial to evaluate the safety of transplanting human Schwann cells to treat patients with recent spinal cord injuries.

We are not recruiting anybody until we obtain approval from our Institutional Review Board.

This trial is just 1 brick in the wall. We will continue working with our scientific colleagues to test other “bricks” in the wall to ultimately develop a strong defense to prevent or reverse the many effects of paralysis.

The approval to move forward with this trial should represent hope for all people living with SCI. There are so many scientists dedicated to developing safe and effective treatments for SCI; we at the Miami Project are but a few. We are all determined, however, to collaborate and move promising treatments forward in a safe and effective manner to ultimately benefit everyone with SCI.

What is a brief overview of the trial?
A potential subject would have to agree to participate within 5 days after his/her injury, which is considered the acute phase. At that point, he/she would have a biopsy of a nerve in one leg to obtain his or her own Schwann cells. The Schwann cells then need to grow in a culturing facility for 3 to 5 weeks to multiply in number and undergo purification. By the time the Schwann cells are actually transplanted into the site of spinal cord injury, the participant would actually be 26-40 days post-injury, which is considered the sub-acute phase.

All procedures would be conducted in Miami at The Miami Project, the University of Miami Hospital, and at Jackson Memorial Hospital. We will be following participants for 1 year after the transplantation surgery and evaluating their neurologic status, medical status, pain symptoms, and muscle spasticity very closely.

What is the goal of the clinical trial?
Safety. The goal of every phase I clinical trial is to demonstrate safety. We have designed a clinical trial that will minimize risk and maximize evaluation of safety.

Is this a cure for paralysis?
No.

The spinal cord is a very complex organ, after all it, in combination with the brain, controls every single aspect of our body. Hence, when the spinal cord sustains damage people lose more than just the ability to walk. Aside from the loss of movement and sensation, almost every single SCI results in the loss of bladder and bowel control. Depending on where in the spinal cord the injury occurs, there may be additional impairments in sexual function, loss of the ability to regulate body temperature and blood pressure, reduced breathing and coughing capacity, and inefficient metabolism of food.

Inside the spinal cord, there is a multitude of damage. First, there is primary damage. In regard to traumatic injury, the primary damage is a direct result of the impact of whatever caused the injury (ex. fall, gunshot, car crash, etc.). Second, waves of biological events are triggered by the primary damage that ends up causing secondary damage. The main contributor to the secondary damage is the immune system. The end result is that spinal cord tissue that was not damaged by the primary impact ends up dying. Third, cells die in and around the injury site. These include motor nerve cells (motoneurons), sensory nerve cells (sensory neurons), relay nerve cells (interneurons), and multiple types of support cells called “glia” (astrocytes, oligodendrocytes). Fourth, individual nerve fibers (axons) running up and down the spinal cord get severed. The cell bodies of these severed axons are located away from the injury site and often remain alive; however, they can no longer communicate. Fifth, inhibitory scar tissue develops around the injured area.

So, it does not make sense then, to expect one intervention to be able to repair all the different types of damage that occur and restore all of the different functions that are impaired. Schwann cells are one component of a multi-faceted cure.

What age groups are included in the trial?
18-50 years old at the time of spinal cord injury

What injury levels are included in the trial?
T3 to T11, ASIA Impairment Scale grade A

Will the trial need to include both men and women, or the first 8 who meet the criteria and consent?
Men and women will be eligible for the trial, however, the first 8 people who meet all of the inclusion criteria, agree to participate, and undergo transplantation of their Schwann cells will be the participants.

Will the people included in this trial be able to receive hypothermia?
No, that would confound the interpretation of the results of this trial.

What do we hope to learn from this study?
Safety. First, we hope to demonstrate that we can insert a needle into the center of the spinal cord injury site without causing any damage. Second, we hope to demonstrate that transplanting one’s own Schwann cells into the thoracic cord does not cause additional damage.

How long will it take to complete this trial?
It could take 2-3 years from the time we enroll the first subject until the final subject is 1 year post-transplantation.

Why does it take so long to complete the trial?
There are many reasons. 1) Only about 25% of all spinal cord injuries are between T3-T11 and, of those, many sustain additional trauma during the accident, which will exclude them from this particular trial. 2) For the first 2 subjects, there is a 1 month waiting period between the time of transplantation surgery and enrollment of the next subject; this leads to about 2 ½ - 3 months between enrollments. Realistically, it could take 7 ½ - 9 months to enroll and transplant the first 3 subjects in the trial. After that, we can enroll 1 subject each month. So, best case scenario, it could take 12 – 15 months to get all 8 subjects enrolled and transplanted. 3) Each subject will be followed for 1 year following the transplantation procedure to analyze safety. Therefore, it could take 2 – 2 ½ years from enrolling the first subject to collecting the final data from the last subject. The data will then need to be analyzed and submitted to the FDA.

In light of Geron shutting down their trial, what are the guarantees that the same will not happen to this trial?
The Miami Project is committed to moving basic science discoveries forward into clinical trials for people living with spinal cord injury. Being based within an academic institution, the University of Miami Miller School of Medicine, we are driven by factors different from corporations in the business world. Our goal is to translate safe, promising discoveries to clinical trials where they can be thoroughly evaluated and ultimately approved by the FDA for medical use.

Why does this trial include only acutely injured people?
The main reason we are doing new injuries first is because we had more animal data in that setting and it allowed us to seek FDA approval faster. Getting faster FDA approval means going into people faster, which means finding out about human safety faster.

Will the trial eventually include chronically injured people?
This specific clinical trial will not include chronically injured people. We are currently conducting more animal chronic transplantation preclinical studies and we will soon being a combined exercise and locomotor rehabilitation study for chronically injured people to determine the minimum exercise and rehabilitation needed to prepare people for transplantation surgery and to ensure that they are neurologically stable. We will take all of that data and design another clinical trial specifically for people with a chronic injury. We will submit that in addition to safety data generated by the acute trial to the FDA for approval.


How can I get my loved one in this trial?
First, we are actively seeking ethical approval by the University of Miami Institutional Review Board. Once we receive that we will begin recruitment for the trial.

If you have a loved one who has had a thoracic spinal cord injury less than 5 days ago, call the Miami Project Education Department at 305-243-7108 for information and preliminary screening.

If you or your loved one has a spinal cord injury that is more than 5 days old, we recommend staying healthy, being active, and participating in the studies that are currently approved, such as exercise, rehabilitation, spasticity, pain, etc.

Think of this as our building block to developing the most effective treatments for people living with SCI. The best way for anyone to get access to these treatments fastest is to keep healthy and in very good condition so that he/she can qualify for clinical trials as they become available. For treatments to go all the way through multiple clinical trials and become mainstream medical practice in the “clinic”, it could take 10-15 years or more depending on the intervention being tested. Participating in clinical trials not only helps us move the field forward faster, but may also benefit the clinical trial participants faster than waiting.



Kim Anderson-Erisman, Ph.D.
Research Associate Professor, Department of Neurological Surgery
Director of Education, The Miami Project to Cure Paralysis

[ResidentBio]

I have participated in some trials at the miami project and i think i will continue to do so in the future, (hoping to do it for wise trails too if posible). I don't think anyone got the answers yet, but I do believe most people looking for a cure have the right intentions. Volunteering to their trials is for me a way to help the community.

[Sue Pendleton]

If they aren't adding hypothermia are they treating the participants with methyprednisolone? I find it hard to believe they got FDA approval before IRR.

[ineedmyelin]

Nothing for chronics!

How can they look Mark in the face and tell him that they aren't doing chronic trials any time soon. All that money, all these years and still nothing for chronics. Makes you appreciate even more what Dr. Young is doing on a shoe-string budget.

[GRAMMY]

Yes it is. So far they have only Jackson in Miami as a recruiting site. With inclusion criteria similar to Geron's it will take a long time (years) to recruit 8 unless they find other sites to partner with.

You're right. Geron was only able to get the 5 and they had multiple sites available. The financial drain over such a long period helped bring the trial to a close.

[Buck503]

Nothing for chronics!

How can they look Mark in the face and tell him that they aren't doing chronic trials any time soon.

I don't think he could abandon his support for MP after so many
years, regardless of where they're at compared to other research.

MP could still be in a safety phase for treadmill studies while a
treatment for chronic sci is available, and they'd still probably
receive support from the Buonicontis.

I couldn't care less if MP has been misunderstood and they're
sincerely working toward a cure for chronic sci or not. The fact
remains that they're way behind and a treatment won't come to
fruition on just good intentions.

[paolocipolla]

This is what Kimberly Anderson had to say about this step.

The Miami Project to Cure Paralysis, a Center of Excellence at the University of Miami Miller School of Medicine, has received permission from the Food and Drug Administration to begin a Phase 1 clinical trial to evaluate the safety of transplanting human Schwann cells to treat patients with recent spinal cord injuries.

We are not recruiting anybody until we obtain approval from our Institutional Review Board.

This trial is just 1 brick in the wall. We will continue working with our scientific colleagues to test other “bricks” in the wall to ultimately develop a strong defense to prevent or reverse the many effects of paralysis.

The approval to move forward with this trial should represent hope for all people living with SCI. There are so many scientists dedicated to developing safe and effective treatments for SCI; we at the Miami Project are but a few. We are all determined, however, to collaborate and move promising treatments forward in a safe and effective manner to ultimately benefit everyone with SCI.

What is a brief overview of the trial?
A potential subject would have to agree to participate within 5 days after his/her injury, which is considered the acute phase. At that point, he/she would have a biopsy of a nerve in one leg to obtain his or her own Schwann cells. The Schwann cells then need to grow in a culturing facility for 3 to 5 weeks to multiply in number and undergo purification. By the time the Schwann cells are actually transplanted into the site of spinal cord injury, the participant would actually be 26-40 days post-injury, which is considered the sub-acute phase.

All procedures would be conducted in Miami at The Miami Project, the University of Miami Hospital, and at Jackson Memorial Hospital. We will be following participants for 1 year after the transplantation surgery and evaluating their neurologic status, medical status, pain symptoms, and muscle spasticity very closely.

What is the goal of the clinical trial?
Safety. The goal of every phase I clinical trial is to demonstrate safety. We have designed a clinical trial that will minimize risk and maximize evaluation of safety.

Is this a cure for paralysis?
No.

The spinal cord is a very complex organ, after all it, in combination with the brain, controls every single aspect of our body. Hence, when the spinal cord sustains damage people lose more than just the ability to walk. Aside from the loss of movement and sensation, almost every single SCI results in the loss of bladder and bowel control. Depending on where in the spinal cord the injury occurs, there may be additional impairments in sexual function, loss of the ability to regulate body temperature and blood pressure, reduced breathing and coughing capacity, and inefficient metabolism of food.

Inside the spinal cord, there is a multitude of damage. First, there is primary damage. In regard to traumatic injury, the primary damage is a direct result of the impact of whatever caused the injury (ex. fall, gunshot, car crash, etc.). Second, waves of biological events are triggered by the primary damage that ends up causing secondary damage. The main contributor to the secondary damage is the immune system. The end result is that spinal cord tissue that was not damaged by the primary impact ends up dying. Third, cells die in and around the injury site. These include motor nerve cells (motoneurons), sensory nerve cells (sensory neurons), relay nerve cells (interneurons), and multiple types of support cells called “glia” (astrocytes, oligodendrocytes). Fourth, individual nerve fibers (axons) running up and down the spinal cord get severed. The cell bodies of these severed axons are located away from the injury site and often remain alive; however, they can no longer communicate. Fifth, inhibitory scar tissue develops around the injured area.

So, it does not make sense then, to expect one intervention to be able to repair all the different types of damage that occur and restore all of the different functions that are impaired. Schwann cells are one component of a multi-faceted cure.

What age groups are included in the trial?
18-50 years old at the time of spinal cord injury

What injury levels are included in the trial?
T3 to T11, ASIA Impairment Scale grade A

Will the trial need to include both men and women, or the first 8 who meet the criteria and consent?
Men and women will be eligible for the trial, however, the first 8 people who meet all of the inclusion criteria, agree to participate, and undergo transplantation of their Schwann cells will be the participants.

Will the people included in this trial be able to receive hypothermia?
No, that would confound the interpretation of the results of this trial.

What do we hope to learn from this study?
Safety. First, we hope to demonstrate that we can insert a needle into the center of the spinal cord injury site without causing any damage. Second, we hope to demonstrate that transplanting one’s own Schwann cells into the thoracic cord does not cause additional damage.

How long will it take to complete this trial?
It could take 2-3 years from the time we enroll the first subject until the final subject is 1 year post-transplantation.

Why does it take so long to complete the trial?
There are many reasons. 1) Only about 25% of all spinal cord injuries are between T3-T11 and, of those, many sustain additional trauma during the accident, which will exclude them from this particular trial. 2) For the first 2 subjects, there is a 1 month waiting period between the time of transplantation surgery and enrollment of the next subject; this leads to about 2 ½ - 3 months between enrollments. Realistically, it could take 7 ½ - 9 months to enroll and transplant the first 3 subjects in the trial. After that, we can enroll 1 subject each month. So, best case scenario, it could take 12 – 15 months to get all 8 subjects enrolled and transplanted. 3) Each subject will be followed for 1 year following the transplantation procedure to analyze safety. Therefore, it could take 2 – 2 ½ years from enrolling the first subject to collecting the final data from the last subject. The data will then need to be analyzed and submitted to the FDA.

In light of Geron shutting down their trial, what are the guarantees that the same will not happen to this trial?
The Miami Project is committed to moving basic science discoveries forward into clinical trials for people living with spinal cord injury. Being based within an academic institution, the University of Miami Miller School of Medicine, we are driven by factors different from corporations in the business world. Our goal is to translate safe, promising discoveries to clinical trials where they can be thoroughly evaluated and ultimately approved by the FDA for medical use.

Why does this trial include only acutely injured people?
The main reason we are doing new injuries first is because we had more animal data in that setting and it allowed us to seek FDA approval faster. Getting faster FDA approval means going into people faster, which means finding out about human safety faster.

Will the trial eventually include chronically injured people?
This specific clinical trial will not include chronically injured people. We are currently conducting more animal chronic transplantation preclinical studies and we will soon being a combined exercise and locomotor rehabilitation study for chronically injured people to determine the minimum exercise and rehabilitation needed to prepare people for transplantation surgery and to ensure that they are neurologically stable. We will take all of that data and design another clinical trial specifically for people with a chronic injury. We will submit that in addition to safety data generated by the acute trial to the FDA for approval.


How can I get my loved one in this trial?
First, we are actively seeking ethical approval by the University of Miami Institutional Review Board. Once we receive that we will begin recruitment for the trial.

If you have a loved one who has had a thoracic spinal cord injury less than 5 days ago, call the Miami Project Education Department at 305-243-7108 for information and preliminary screening.

If you or your loved one has a spinal cord injury that is more than 5 days old, we recommend staying healthy, being active, and participating in the studies that are currently approved, such as exercise, rehabilitation, spasticity, pain, etc.

Think of this as our building block to developing the most effective treatments for people living with SCI. The best way for anyone to get access to these treatments fastest is to keep healthy and in very good condition so that he/she can qualify for clinical trials as they become available. For treatments to go all the way through multiple clinical trials and become mainstream medical practice in the “clinic”, it could take 10-15 years or more depending on the intervention being tested. Participating in clinical trials not only helps us move the field forward faster, but may also benefit the clinical trial participants faster than waiting.



Kim Anderson-Erisman, Ph.D.
Research Associate Professor, Department of Neurological Surgery
Director of Education, The Miami Project to Cure Paralysis

I think this is rather clear and honest. No cure here.
A learning step for the cure process, probably.
I wonder why the FDA took so long to give them a green light?
Schwan cells have been around for so long and should have gone to trials years ago.
We must take an active role in the cure process like the AIDS community did to speed up things. We can't be happy with this slow progress.

Paolo

[Sue Pendleton]

Nothing for chronics!

How can they look Mark in the face and tell him that they aren't doing chronic trials any time soon. All that money, all these years and still nothing for chronics. Makes you appreciate even more what Dr. Young is doing on a shoe-string budget.

Considering the Buonoconti's ARE the funding arm for TMP maybe they should have said chronic cure only science using their funds from the start?

If I remember correctly, about 20 years ago both Richard and Mary Bungee were working on Schwann Cells at TMP. After Richard died Mary began working with another scientist and they found 2 types of cells were needed for any growth. I think this trial is a desperate attempt to keep a certain institution and a certain person on salary especially with the news coming from so many other labs and businesses. Any why restrict the range solely to thorasic when quads to C5 or 6 could also benefit? Or is that why no endpoint or goal is stated other than to safely stick a needle into someones' spinal cord? That has been a lot of research years if that is the main goal.

[GRAMMY]

Considering the Buonoconti's ARE the funding arm for TMP maybe they should have said chronic cure only science using their funds from the start?

If I remember correctly, about 20 years ago both Richard and Mary Bungee were working on Schwann Cells at TMP. After Richard died Mary began working with another scientist and they found 2 types of cells were needed for any growth. I think this trial is a desperate attempt to keep a certain institution and a certain person on salary especially with the news coming from so many other labs and businesses. Any why restrict the range solely to thorasic when quads to C5 or 6 could also benefit? Or is that why no endpoint or goal is stated other than to safely stick a needle into someones' spinal cord? That has been a lot of research years if that is the main goal.

Keep in mind that InVivo needed a safety study on schwann cells to add them to their acute scaffold they are proposing. That collaboration was announced quite some time ago.

[Sue Pendleton]

"If you or your loved one has a spinal cord injury that is more than 5 days old, we recommend staying healthy, being active, and participating in the studies that are currently approved, such as exercise, rehabilitation, spasticity, pain, etc."

I'm not so sure of this. The more we support these studies the more NIH funding will go toward these kind of studies. Not to mention you only get into an exercise or rehab study for so long and Medicare will be cutting PT amounts again come October first. I can see a study for the use of lithium for irretractable neurogenic pain on the horizon after the final results of the SCIChinaNet studies are published. The only question here is which generic lithium producer will jump first?

[paolocipolla]

Considering the Buonoconti's ARE the funding arm for TMP maybe they should have said chronic cure only science using their funds from the start?



Yes I think so.

The SCI community must wake up and dig in what is really happening in SCI research and take action to put things on track of common sense.

Paolo

[Sue Pendleton]

Keep in mind that InVivo needed a safety study on schwann cells to add them to their acute scaffold they are proposing. That collaboration was announced quite some time ago.

But again, we're discussing acutes. Marc and company are serious chronics. And as Paolo pointed out, schwann cells have been studied for decades now and this is their first study in humans? Either we need to redefine safety for conditions with no current treatment at the FDA or find other ways to speed up this process besides skipping acutes because by the time most care about paralysis it's too late for them or their loved ones. I would apply a militarism to the research and publish system to speed it up: "Lead, follow or get the hell out of the way!"

[c473s]

One thing that MP can shout about is that they can run the trial within their big shiny facilities without worrying about other centres.
They do not see the numbers of SCI to do it with any speed. A multi center trial is nearly a requirement to recruit the numbers needed to enroll 8.

[GRAMMY]

But again, we're discussing acutes. Marc and company are serious chronics. And as Paolo pointed out, schwann cells have been studied for decades now and this is their first study in humans? Either we need to redefine safety for conditions with no current treatment at the FDA or find other ways to speed up this process besides skipping acutes because by the time most care about paralysis it's too late for them or their loved ones. I would apply a militarism to the research and publish system to speed it up: "Lead, follow or get the hell out of the way!"

Sue, I agree. Dr. Keith Tansey brought up this great discussion last year and thoughts about the FDA being more lenient with sci therapies and allowing for dignity of risk. Would enough people actually waiver liability and risk to enter under those circumstances? http://spinalcordresearchandadvocacy.wordpress.com/2012/05/11/108/ (http://spinalcordresearchandadvocacy.wordpress.com/2012/05/11/108/) An interesting presentation by Naomi Kleitman PhD concerning some of the challenges in translating sci therapies. They will need to file an IRB with the FDA before recruiting.

[swh2007]

Yes I think so.

The SCI community must wake up and dig in what is really happening in SCI research and take action to put things on track of common sense.

Paolo

A good first step would be to try to get 80 percent of the top 50 posters on the Cure Forum to agree on how to do that and then to get those 40 people or a close friend or family member to follow up with consistent long term action.

[KofQ]

Keep in mind that InVivo needed a safety study on schwann cells to add them to their acute scaffold they are proposing. That collaboration was announced quite some time ago.

I don't think InVivo can afford to wait 3-5 years for MP to complete this trial. Their balance sheet suggests that they can't last more than another six months without some tangible progress. They have been talking about the scaffold approval, which was supposed to happen a couple of months ago at the latest (huge red flag). I think they announced a collaboration with MP just to signal to shareholders that they have a cash cow to go to for trial funds without having to dilute the share price or take on usurious financing terms to remain in operation.

So far they have only Jackson in Miami as a recruiting site. With inclusion criteria similar to Geron's it will take a long time (years) to recruit 8 unless they find other sites to partner with.

I think that's the idea. Keep the checks coming in from uninformed donors to keep the lights on and the 401k plans fully funded.

It's the same situation at CDRF. They have an acute trial of Riluzole that isn't doing anybody any good.

This trial is about saying "Look! We have a trial going, too." while other groups leapfrog past them on budgets one-tenth the size.

[adrien]

hello all
I just give my opinion ...
for me this therapy will not work at all, not a finger move over, I really hope I am wrong because I'm like you on the lookout for a therapy for me to walk again!
I understand why this therapy is so much noise on this forum, while the is to Gregoire Courtine much more exciting .. http://www.youtube.com/watch?v=kuDkkAK5tZ4

What do you think of the therapy to Gregoire Courtine?
Mr Wise Young, you have not commented on the therapy to Gregoire Courtine.
Can you make one?

And other, what do you think?

[GRAMMY]

I think they announced a collaboration with MP just to signal to shareholders that they have a cash cow to go to for trial funds without having to dilute the share price or take on usurious financing terms to remain in operation.

http://www.massdevice.com/news/invivo-and-university-miami-team-spinal-injury-research

http://www.masshightech.com/stories/2011/05/02/daily37-InVivo-Therapeutics-Miami-Project-to-co-develop-spinal-cord-injury-treatments.html

I noticed these articles from 15 months ago.

[KyleP2112]

I really wish there were more research news on chronic spinal cord injuries...

Anyone else with chronic SCI feel frustrated by the lack of progress? It would be great to have a reason for optimism about a "cure" or at least a therapy/surgery that improves function/independence for those of us with chronic SCI.

Kyle, C4/5...17 years post injury.

[Patton57]

I really wish there were more research news on chronic spinal cord injuries...

Anyone else with chronic SCI feel frustrated by the lack of progress? It would be great to have a reason for optimism about a "cure" or at least a therapy/surgery that improves function/independence for those of us with chronic SCI.

Kyle, C4/5...17 years post injury. I just think it shows how challenging the problem of finding a cure for scis really is. I wonder if there are enough scientists doing research (enough perspectives) to really make any progress.

[NoDecafPlz]

And the truth will set you free!

It's all finger puppets against the cave wall down in Miami.

I don't think InVivo can afford to wait 3-5 years for MP to complete this trial. Their balance sheet suggests that they can't last more than another six months without some tangible progress. They have been talking about the scaffold approval, which was supposed to happen a couple of months ago at the latest (huge red flag). I think they announced a collaboration with MP just to signal to shareholders that they have a cash cow to go to for trial funds without having to dilute the share price or take on usurious financing terms to remain in operation.



I think that's the idea. Keep the checks coming in from uninformed donors to keep the lights on and the 401k plans fully funded.

It's the same situation at CDRF. They have an acute trial of Riluzole that isn't doing anybody any good.

This trial is about saying "Look! We have a trial going, too." while other groups leapfrog past them on budgets one-tenth the size.

[ineedmyelin]

I really wish there were more research news on chronic spinal cord injuries...

Anyone else with chronic SCI feel frustrated by the lack of progress? It would be great to have a reason for optimism about a "cure" or at least a therapy/surgery that improves function/independence for those of us with chronic SCI.

Kyle, C4/5...17 years post injury.

I'm very frustrated. 22yrs chronic and counting. I'm a c4-c5 walking incomplete and I'm starting to have issues with my health. My strength, balance and energy levels are all starting to give me problems. I'm getting old and my window opportunity closes a bit more every year.

[smirking1]

I am going to play devil's advocate here. The timing of all this smells a little bit like politics (or b.s. same odor) 4 years ago about this time we were a priority. Then every 8-10 months a news story about rats walking or chimps walking, other than that lots of crickets chirping.
Now after 25 years and enough money to send every person in a wheelchair to the moon and back......finally MP is approved phase I trials? on something that nobody believes works. Something they have had on the shelf for years.
I am willing to bet this will be mentioned by a presidential candidate in the next 2-3 weeks.

P.S. I do not wear an aluminum foil hat or believe in conspiracy theories. I am no more or less for Obama than Mitt , in fact I am ashamed that all our country can come up with for choices is these 2. dont care if you think i am right wing or left wing. I am just saying we are at the mercy of politics. Why is Dr.Young in China and not New Jersey?

[nrf]

I am going to play devil's advocate here. The timing of all this smells a little bit like politics (or b.s. same odor) 4 years ago about this time we were a priority. Then every 8-10 months a news story about rats walking or chimps walking, other than that lots of crickets chirping.
Now after 25 years and enough money to send every person in a wheelchair to the moon and back......finally MP is approved phase I trials? on something that nobody believes works. Something they have had on the shelf for years.
I am willing to bet this will be mentioned by a presidential candidate in the next 2-3 weeks.

P.S. I do not wear an aluminum foil hat or believe in conspiracy theories. I am no more or less for Obama than Mitt , in fact I am ashamed that all our country can come up with for choices is these 2. dont care if you think i am right wing or left wing. I am just saying we are at the mercy of politics. Why is Dr.Young in China and not New Jersey?

I think I'd vote for you for POTUS.

[StemCells&AtomBombs]

"recent spinal cord injuries"

and we're excited about???

[jcalix]

in my opinion be patience, be positive and keep in good health, please remember there is a protocol to follow, this is just the begining of this trial, hope the results are ok then they will proceed with the effectiviness in acute & cronics as well, thank God there are other trials in progress and thanks to Wise for working so hard to find us a CURE, God Speed

best regards

Jose C6-C7, 7 years post injury

[paolocipolla]

"If you or your loved one has a spinal cord injury that is more than 5 days old, we recommend staying healthy, being active, and participating in the studies that are currently approved, such as exercise, rehabilitation, spasticity, pain, etc."

I'm not so sure of this. The more we support these studies the more NIH funding will go toward these kind of studies.
.....


I agree!

Paolo

[paolocipolla]

....
..
I would apply a militarism to the research and publish system to speed it up: "Lead, follow or get the hell out of the way!"

I would too, but it's unlikely we can make it happen... maybe we can have some positive effect by constantly controlling how the money are spent in the field.
Cosider that the money of the SCI orgs are controlled by the scientific advisory boards & the reviewers (which all know each other). Most of them think that chronic SCI is too difficult to deal with so they just do acute as it's much easier to have some sort of positive results and therefore nice publications... that's how it works, not always, but most of the times.

So in my opinion WE need to set the guide lines on how money should be spent to make sure chronic cure research is actually done.

As an alternative there is always that crazy idea of an X-Prize

Paolo

[paolocipolla]

They do not see the numbers of SCI to do it with any speed. A multi center trial is nearly a requirement to recruit the numbers needed to enroll 8.

c473s,

would you agree that if it were a trial for chronic SCI the Miami Project could enroll all the 8 patients in about a year or maybe even less than a year?

Paolo

[paolocipolla]

A good first step would be to try to get 80 percent of the top 50 posters on the Cure Forum to agree on how to do that and then to get those 40 people or a close friend or family member to follow up with consistent long term action.

That's an intersting proposal (I am sure StemCells&AtomBombs likes it too).
One problem I have found in the past is that we all have a relatively limited/different understanding of what's going on so that makes it hard to agree on actions.
Then consider also that even is we all had a good enough understanding of the field likely we'll have different visions on how to tackle the problems as we belong to different cultures, have different educations, have been in chair for differt number of years etc..
So far it looks to me that all the attempts to unite a "critical mass" of the community have failed in short period of time, but I don't want to say we shouldn't try again, I am just pointing out some difficulties to keep in mind to have some possibilities of success.

Paolo

[paolocipolla]

Quote:
Originally Posted by GRAMMY http://sci.rutgers.edu/forum/images/buttons/viewpost.gif (http://sci.rutgers.edu/forum/showthread.php?p=1563005#post1563005)
Keep in mind that InVivo needed a safety study on schwann cells to add them to their acute scaffold they are proposing. That collaboration was announced quite some time ago.

I don't think InVivo can afford to wait 3-5 years for MP to complete this trial. Their balance sheet suggests that they can't last more than another six months without some tangible progress. They have been talking about the scaffold approval, which was supposed to happen a couple of months ago at the latest (huge red flag). I think they announced a collaboration with MP just to signal to shareholders that they have a cash cow to go to for trial funds without having to dilute the share price or take on usurious financing terms to remain in operation.

Quote:
Originally Posted by c473s http://sci.rutgers.edu/forum/images/buttons/viewpost.gif (http://sci.rutgers.edu/forum/showthread.php?p=1562582#post1562582)
So far they have only Jackson in Miami as a recruiting site. With inclusion criteria similar to Geron's it will take a long time (years) to recruit 8 unless they find other sites to partner with.

I think that's the idea. Keep the checks coming in from uninformed donors to keep the lights on and the 401k plans fully funded.

It's the same situation at CDRF. They have an acute trial of Riluzole that isn't doing anybody any good.

This trial is about saying "Look! We have a trial going, too." while other groups leapfrog past them on budgets one-tenth the size.


Very likely this is all correct IMO.

Paolo

[GRAMMY]

Either we need to redefine safety for conditions with no current treatment at the FDA or find other ways to speed up this process besides skipping acutes because by the time most care about paralysis it's too late for them or their loved ones. I would apply a militarism to the research and publish system to speed it up: "Lead, follow or get the hell out of the way!"

The House has passed it, but lets hope the Senate passes legislation to establish the new user fees act for the FDA. It's not all of the answer, but it could help.
http://spinalcordresearchandadvocacy.wordpress.com/2012/08/03/spinal-cord-injury-innovation-measured-in-decades-not-headlines/

[swh2007]

That's an intersting proposal (I am sure StemCells&AtomBombs likes it too).
One problem I have found in the past is that we all have a relatively limited/different understanding of what's going on so that makes it hard to agree on actions.
Then consider also that even is we all had a good enough understanding of the field likely we'll have different visions on how to tackle the problems as we belong to different cultures, have different educations, have been in chair for differt number of years etc..
So far it looks to me that all the attempts to unite a "critical mass" of the community have failed in short period of time, but I don't want to say we shouldn't try again, I am just pointing out some difficulties to keep in mind to have some possibilities of success.

Paolo


Then you might as well give up the idea of the SCI community having any impact on SCI research. Do not expect more from the researchers than you expect from your own community.

[c473s]

c473s,

would you agree that if it were a trial for chronic SCI the Miami Project could enroll all the 8 patients in about a year or maybe even less than a year?

Paolo
A chronic trial would fill quickly "IF" the inclusion/exclusion criteria were much looser than what it is in this or the Geron trial. It would by default eliminate several criteria. The challenge then becomes the money for rehabilitation. In acute trials the "acute rehab" is funded by insurance or government and in smaller numbers by charity allowances.

[malthouse]

Everyone knows that all Wise needs is a Billionaire to give him the financial independence to do what he needs to do on this great humanitarian effort.

Most people on this site may remember my "rant" here of a few months ago about my pet peev, Billionairs dreaming up hairbrained tax avoidance schemes ie man made floating tax havens etc.

Well, Clive Palmer, a particularly divisive billionaire here in Australia has come up with the grand plan of building a replica titanic!
Of all the things this world needs, a replica titanic ego trip for this Oger would come some way down the list I would have thought. He has declared third class, yes that's third class passengers will be banned from entering first class areas!!! When asked at a press conference what the budget would be he said what ever the cost " I have the money".

His very good friend is fellow Australian and worlds richest woman Gina
Rhinehart, who he fully supports in her efforts to avoid paying her fare share of tax.
I dispair at what people in our community of the stature of Wise young could do for humanity with the support of these people rather than wasting there money on ego trips? My dad used to say "you can tell God doesn't like money by the people he gives it to" Well said dad, couldn't agree more!!

[malthouse]

Oh and BTW, I neglected to mention Gina Rhinehart, who was born into wealth, she has not created one cent in wealth herself has publicly stated she "does not donate to charitable causes.

[paolocipolla]

Then you might as well give up the idea of the SCI community having any impact on SCI research. Do not expect more from the researchers than you expect from your own community.

I agree..
...one thing we miss also is a leader of the community. It could be a person like it was Christopher Reeve or it could be an SCI org. with a clear plan & a good governance.
Probably most of us now agree not to support the Rick Hansen Foundation when it comes to a cure for chronic SCI.
On the other SCI orgs the discussion seems still open.
Probably it is necessary to have more than one SCI org to cover complementary parts of the whole SCI problem.
I think things are evolving, I try to put my two cents to agitate the situation, so that good things can emerge at the end.

Paolo

[paolocipolla]

A chronic trial would fill quickly "IF" the inclusion/exclusion criteria were much looser than what it is in this or the Geron trial. It would by default eliminate several criteria. The challenge then becomes the money for rehabilitation. In acute trials the "acute rehab" is funded by insurance or government and in smaller numbers by charity allowances.

Thanks c473s,

do you think the MP could do 8 people alone or a multicenter trial would still be needed even if it were a chronic SCI trial?

Paolo

[paolocipolla]

hello all
I just give my opinion ...
for me this therapy will not work at all, not a finger move over, I really hope I am wrong because I'm like you on the lookout for a therapy for me to walk again!
I understand why this therapy is so much noise on this forum, while the is to Gregoire Courtine much more exciting .. http://www.youtube.com/watch?v=kuDkkAK5tZ4

What do you think of the therapy to Gregoire Courtine?
Mr Wise Young, you have not commented on the therapy to Gregoire Courtine.
Can you make one?

And other, what do you think?

Adrien,

I agree that schwann cells alone probably will not work at all.

About Gregoire Courtine it's looks much better then what it is. He is exellent in marketing & communication for sure.
If you ck the model they have used you see that is very far from reality. Then they have not regenerated axons which is what we really need.
I still think this line of research is usefull, but is not my favorite.

Just my opinion for what it worth.

Paolo

[c473s]

Thanks c473s,

do you think the MP could do 8 people alone or a multicenter trial would still be needed even if it were a chronic SCI trial?

Paolo
Just my opinion but I believe a multi center trial is required to get 8 in any reasonable time frame.

[paolocipolla]

Just my opinion but I believe a multi center trial is required to get 8 in any reasonable time frame.

c473s,

thanks for your opinion, which I respect, but could you say what are the bases of your opinion?

To organize a multicenter trial to do "just" 8 patients with chronic SCI for a phase I/II (that was the hypothesis here) it seems a waste of time and money to me, in fact to set up a multicenter clinical trial you'll need to allocate pesonnel for recruting clinical trial centers, training principal investigators and clinicians, develop a consensum on a clinical trial protocol etc...
I have discussed this issue with clinicians who had the experience to run/be involved - in multicenter clinical trials for SCI and they cleraly told me that is much better to avoid to have a multicenter trial when ever is possible as it can easily lead to the failure of a trial just because of the comlications I have mentioned above to set up a multicenter trial. A failure happened already at least once in an acute trial.

Then consisder the Stem Cell Inc trial, it is a phase I/II palnning to recute 12 patients (recuting is open to patiants from US, Canada & Europe... because people with chronic SCI can travel relatively easy, right? ;)) and they use just one center.
Why didn't they go for a multicenter trial?
Then consider also that Stem Cell Inc. is actually doing 3 pase I/II at the same time, infact they have planned to recrute patients ASIA A, B & C.

That is a very efficent way to run a trial IMO.

Paolo

[c473s]

c473s,

thanks for your opinion, which I respect, but could you say what are the bases of your opinion?

To organize a multicenter trial to do "just" 8 patients with chronic SCI for a phase I/II (that was the hypothesis here) it seems a waste of time and money to me, in fact to set up a multicenter clinical trial you'll need to allocate pesonnel for recruting clinical trial centers, training principal investigators and clinicians, develop a consensum on a clinical trial protocol etc...
I have discussed this issue with clinicians who had the experience to run/be involved - in multicenter clinical trials for SCI and they cleraly told me that is much better to avoid to have a multicenter trial when ever is possible as it can easily lead to the failure of a trial just because of the comlications I have mentioned above to set up a multicenter trial. A failure happened already at least once in an acute trial.

Then consisder the Stem Cell Inc trial, it is a phase I/II palnning to recute 12 patients (recuting is open to patiants from US, Canada & Europe... because people with chronic SCI can travel relatively easy, right? ;)) and they use just one center.
Why didn't they go for a multicenter trial?
Then consider also that Stem Cell Inc. is actually doing 3 pase I/II at the same time, infact they have planned to recrute patients ASIA A, B & C.

That is a very efficent way to run a trial IMO.

Paolo
Paolo, No question chronics will be willing. I simply believe recruitment is dependent on inclusion/exclusion criteria. I was part of the Israeli macrophage and the Geron trials through an institution. The numbers turned down because of our FDA criteria were astounding and frustrating. There are several centers in the U.S. already trained and staffed to recruit. For me the desire is to see large enough numbers brought in before the company funding the research gets tired of watching their expenses go too long for their appetite.

[paolocipolla]

Paolo, No question chronics will be willing. I simply believe recruitment is dependent on inclusion/exclusion criteria. I was part of the Israeli macrophage and the Geron trials through an institution. The numbers turned down because of our FDA criteria were astounding and frustrating. There are several centers in the U.S. already trained and staffed to recruit. For me the desire is to see large enough numbers brought in before the company funding the research gets tired of watching their expenses go too long for their appetite.

Thanks for sharing your experience.

Paolo

[Sue Pendleton]

The House has passed it, but lets hope the Senate passes legislation to establish the new user fees act for the FDA. It's not all of the answer, but it could help.
http://spinalcordresearchandadvocacy.wordpress.com/2012/08/03/spinal-cord-injury-innovation-measured-in-decades-not-headlines/

Thanks for the link. Time to get back up to speed.

Sue

[cpaul]

Their Intake person called me the other day to update their files on me and my interest in participating in any clinical trials @ their facility. The girl on the phone said they are going take 4 years on phase 1 to follow the sub-acute patients they get Huh!!! So if this is their timeline, and this therapy actually works magic for SCI. it will be another 15-20 years before anyone actually gets on a gurney to have something administered. If you are over 50 like me and 12 yrs chronic, it ain't gonna happen from MP. No more contributions from to this monolith.

[paolocipolla]

Their Intake person called me the other day to update their files on me and my interest in participating in any clinical trials @ their facility. The girl on the phone said they are going take 4 years on phase 1 to follow the sub-acute patients they get Huh!!! So if this is their timeline, and this therapy actually works magic for SCI. it will be another 15-20 years before anyone actually gets on a gurney to have something administered. If you are over 50 like me and 12 yrs chronic, it ain't gonna happen from MP. No more contributions from to this monolith.

I agree with your conclusion.
Still we can't give up, I am a little younger than you but I want people like you to have chance to walk again.

Which SCI orgs &/or research groups would take the challenge to get someone like you out of w/c soon enough to let you enjoy a walk on a beach?

Rick Hansen Foundation - NO
Christopher and Dana Reeve Foundation - NO
Miami Project - NO

We should do a screening of all SCI orgs & research groups to figure out who deserve our donations & advocacy effort.

Perhaps we should just put together a survey and send it around... Questions needs to be smart!

Paolo

[cpaul]

that's a great idea. There are enough Hawks on this forum that know who's doing what. BTW: CRPPF is throwing money @ Dr. Harkema's thing. This is a nogo as well. I spent a year in her lab back in 2001 participating in a human locomotion study. They are still jacking around with the same thing 11 years later. This one is not going to solve it either so put it on the list. OBTW: throw HBOT in there as well. Been there, done that. Might be good if your in a tube within hours of injury but for a Chronic. forget it!

[tvot]

I like chicken.