Groundbreaking cp study receives fda approval;

CherieC

New member
GROUNDBREAKING CP STUDY RECEIVES FDA APPROVAL;
AWAITS FUNDING

Kids Will be Treated With Their Own Bone Marrow. No Cord Blood Required. This is why we started Let's Cure CP . A leading researcher at University of Texas Medical School at Houston has received approval from the FDA to start a groundbreaking study to treat CP kids with autologous (their own) stem cells.

Let’s Cure CP first approached Dr. Charles S. Cox, Jr., about doing this study almost two years ago, and we are overjoyed to see this dream come to fruition. This is the FIRST study of its kind in the United States that will treat CP kids with their own bone marrow. Until now, if you did not bank a child’s cord blood, there were no other options for this promising type of treatment in the U.S. That is about to change due to a powerful partnership between Let’s Cure CP and Dr. Cox.

The study will have two arms (groups). One arm is the bone marrow group and the second is the cord blood group. Dr. Cox and his team will compare the two types of stem cell lines and their efficacy.


Dr. Cox, who is the Children’s Fund, Inc. Distinguished Professor of Pediatric Surgery and directs the Pediatric Surgical Translational Laboratories and Pediatric Program in Regenerative Medicine at UT-Houston, will measure results in two ways:
1.Study baseline MRIs of the children and compare with post-treatment MRIs for any changes.
2.Compare baseline motor function and post-treatment motor function of the children.
The study will follow 30 children over a two-year period; 15 kids in each "arm." This will be a randomized, blind, crossover study, meaning that the patient and the parents will not know if they received the real infusion or placebo infusion at the start of the trial. EVERYONE will get treated in this trial; the children that received placebo at the start of the trial will receive treatment at the one-year follow-up visit. The children will be evaluated at baseline (start of the study); 6 months; 1 year after infusion; and 2 years after infusion.


Dr. Cox is ready to begin this astounding research, except he lacks one critical component: funding. That's why we NEED YOUR HELP more than ever! The study will not begin until more money is raised to fund it. To date, $300,000 has been raised; the study requires approximately $1.3 million dollars.



We at Let’s Cure CP are committed to making this trial happen! We are aggressively seeking corporate sponsors and reaching out to other foundations for help. Still, most research organizations get their donations from families and people affected by the disease and condition. That is why we are pleading to YOU, the CP community, to fight for the research and the treatments that we deserve. CP research has been underfunded and under-exposed, and it's time to change that.
 

barbara

Pioneer Founding member
CherieC - This is wonderful news. Recently, there was a similar story of patients with Parkinson's disease also self funding a clinical trial.

I certainly admire those who have taken it upon themselves to get the ball rolling. It shows that people do have power. I know that so many parents of kids with CP like you have been very disappointed because clinical trials shut them out if they did not have the cord blood saved or have access to it.

Thank you so much for posting this. I will Tweet it and urge others who may be on Facebook, Twitter or other sites to get the word out so that this trial can get the needed funding and move forward.
 

Donna

New member
I sure hope they can raise the money. After hearing about some of the silly trials that the taxpayers have to fund through the NIH, I have to say this irks me that this study doesn't receive any funding at all.
 

pink7

New member
CP research

I hope they get the funding needed.It has been so difficult to get the medical community to look at stem cell treatment for the various medical conditions.
 

Bobcat

New member
It just seems like this would be such a noble cause for a philanthropist to want to be involved in. Where are all those rich Texans?
 

CherieC

New member
Let's hope someone finds it worthy!

Someone needs to feel that it is worthy. CP needs an ambassor like Micheal J Fox, Jerry Lewis or someone famous to advance the cause. Treatments have basically stayed the same for years and hardly any funding for research. It boggles the mind since 1 in 278 children are born yearly with CP. This number in on the rise with premature infants even futher with medial advances saving babies being born.
Cerebral palsy affects more children than muscular dystrophy, multiple sclerosis, cystic fibrosis or childhood leukemia.
But CP receives only a fraction of the government research funding these other disorders receive. I wish I knew why. I only know it isn’t right.Not that these organizations deserve any less research support. I just believe cerebral palsy should warrant more attention. In the U.S., 1 in every 300 eight-year-olds has cerebral palsy. The odds are 1 in every 270 among African-American children. Yet, the cause of 80% of CP is still unknown.
As a teacher I work with families of children with cerebral palsy on a regular basis.This hits home for me personally because I am the mother of a child with CP. I know what kind of challenges she faces, and what it costs to provide children with the kinds of therapies and services they will need the rest of their lives. I am well aware that science has not found a way to prevent most cerebral palsy, or to cure it.Without more research funding, it is unlikely this will occur anytime soon, which is a travesty. Dedicated research funding is practically nonexistent, according to the Academy for Cerebral Palsy and Developmental Medicine. The Centers for Disease Control and Prevention (CDC) has no specific funding for CP, and funds dedicated for CP by the National Institutes of Health (NIH) (only $22million for this year) is not for innovative, curative research, the academy notes.
Today is National Cerebral Palsy Awareness Day. Do your part to urge Congress to dedicate more funding to preventing, curing and developing effective therapies for cerebral palsy. Contact your elected officials. The 800,000 Americans who are living with CP today are counting on you. I believe in my heart that stem cells will play a vital part in not only treating but curing CP some day. Please take the time to call your elected officals!
 

barbara

Pioneer Founding member
I think private funding is far more likely to get more immediate results. As we are finding out there are a lot of self interested researchers and institutions vying for government grants and funding. The process is lengthy and the research endless. You have an immediate prospect of getting something accomplished with Dr. Cox.

I am spreading the word about the funding that is needed for this stem cell trial. I urge others to do so as well. There has to be a guardian angel or angels out there. The amount needed is not that much in comparison to what some celebrities and successful people give to other causes.
 

paigie

New member
updates

Hi everyone, its been a long time since ive been on here. I read this about the CP trial and wanted to no where i can go to look into this. Where exactly is it being done and is it any closer. I have my sons cord blood frozen, for my daughter but i no they are a few years away from using that yet. wonder if this will come up quicker. :D:eek:
 

mkosel0

New member
The last time I checked in with Dr. Cox they were still not further down the path. Still lacking some funding... but it is on the horizon!
 

CherieC

New member
Study

The people from "Let's Cure CP" is the group behind this study and you can find them on their website. From what I have read they will only take 40 children and my worry would be they already know which kids (the groups) who will be in this study.
I have chosen to do the same treatment as this group is going to do but paying for it myself. We have a few things to get in order but my daughter Bella will have her treatment in May of 2014 or I should say her first treatment. We are using donated cord blood as we were unable to save her cord blood. This is through Dr. Thermos in Mission Viejo in Cal but I am sure one of those places would allow you to use your daughters stored cord blood for her brother.
 

CherieC

New member
Trial is starting

The people from "Let's Cure CP" is the group behind this study and you can find them on their website. From what I have read they will only take 40 children and my worry would be they already know which kids (the groups) who will be in this study.
I have chosen to do the same treatment as this group is going to do but paying for it myself. We have a few things to get in order but my daughter Bella will have her treatment in May of 2014 or I should say her first treatment. We are using donated cord blood as we were unable to save her cord blood. This is through Dr. Thermos in Mission Viejo in Cal but I am sure one of those places would allow you to use your daughters stored cord blood for her brother.
I think you could see from the most recent post this trial is starting soon and has the total of 30 children in it. They must have decided to go with a lower number and push through!
 

barbara

Pioneer Founding member
Maybe there were other parents who also were against putting their children through a placebo based trial.
 

LLL6521

New member
I have spoken to one of the main founders, and they have my deepest respect as a fellow parent with CP. These parents took it upon themselves to organize and raise money as parents' of children with CP. I admire their tenacity and drive to get closer to find a cure, and they have my deepest respect. The organization has grown tremendously, however, the organization is deeply rooted into helping fund FDA approved clinical trials. Most of the money raised goes to these researchers. As these researchers dismiss and discourage any treatment outside the FDA world, the FDA route just prolongs the goal which is finding more treatments and an ultimate cure. As a parent of a child with CP, I find it more encouraging having that responsibility taken away from the FDA regulations and shifting to where it has always belonged in the field of medical practice. I am sure by now, if the treatments have started this way, the options would have been 100 times more available than what we have now. With that being said, my hat comes off for "Let's Cure CP's" efforts. They try under the extraordinary limited options.
I for one like to focus my energy in pointing out the FDA’s draconian efforts to categorize autologous stem cells as a drug, and having children with CP wait years and years for treatments. This goes for the 100’s of other ailments stem cell treatments could/would help, but instead they wait in the sidelines crippled and dying.
 
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barbara

Pioneer Founding member
I agree LLL6521. The onerous regulations that the FDA has imposed is impeding progress and preventing millions of patients from getting potentially life saving treatment. There are many experts in the field who agree with this. Recently on a LinkedIn discussion a researcher from a well known facility here in the U.S. commented that he had spoken to some people from the FDA who said they are quite satisfied with the regulation of our own stem cells as drugs and have no intention of changing the regulations unless Congress mandates them to do so. Seeing that Big Pharma has a very powerful lobby and many in Congress enjoy Big Pharma's contributions to their campaign funds, can anyone imagine that Congress will mandate change before hell freezes over? The only thing that can bring that change will be a public outcry; a public that little understands the issue.
 

LLL6521

New member
I agree Barb. As long as the FDA continues to regulate autologous stem cells as a drug, it would be those overseas countries that will progress much quicker. Sadly, the U.S. has the best medical technology and innovations in the world, and this is what makes it frustrating, because while we witness a decade go by with the same autologous cord blood treatments still in FDA clinical trial, the innovations in the U.S. medical practice field would have made huge advances by probably treating 1000's of children with CP. Who knows, possibly by leaps and bounds in treating children with CP - I am pretty sure of it.
 

barbara

Pioneer Founding member
Cord Blood Registry Clinical Trial at UTHSC Evaluates Stem Cells for Cerebral Palsy

Cord Blood Registry Clinical Trial at UTHSC Evaluates Stem Cells for Cerebral Palsy Therapy


BioNews Texas
Maureen Newman

Cord Blood Registry is conducting a Phase 2 clinical trial at University of Texas Health Science Center to evaluate the safety and efficacy of delivering stem cells intravenously to cerebral palsy patients. The goal is to recruit 30 children with cerebral palsy, aged two to ten years, and administer either autologous cord blood stem cells (collected at the time of birth) or autologous bone marrow stem cells (collected from a bone marrow harvest). Five patients from either group will receive a placebo at baseline but will be given the option to receive stem cell treatment after one year. Follow-ups at 6, 12, and 24 months will be conducted at the University of Texas Health Medical School and Children’s Memorial Hermann Hospital.

During follow-ups, patients will be evaluated for treatment safety through pulmonary and hepatic function, neurological status, and hemorrhagic or ischemic lesions; they will be evaluated for treatment efficacy through MRI analysis of white matter tracts correlated with motor function studies. The study was initiated in November 2013 and is projected to end in November 2015. As of January 21, enrollment for the bone marrow harvest group was met.

This trial, like all trials, began in the laboratory. “There is preclinical data indicating that the ongoing neuroinflammatory response is a driver of further injury in cerebral palsy so the hope is to reduce this neuroinflammation,” stated Dr. Charles Cox, principal investigator of the study and a professor of Pediatric Surgery at UTHMS. The inflammation seen in cerebral palsy is caused by abnormal brain development and damage to the motor nervous system. It is thought that infusing a patient’s own stem cells may trigger repair mechanisms to alleviate the damage. “Our goal is to break the cycle of inflammation and injury,” said Dr. Cox in material from Cord Blood Registry. This goal is good news for the 1 in every 323 children in the United States who has been diagnosed with cerebral palsy, as the prevalence of the disease has increased over time.


Here's what was posted on the CIRM blog in response to the above press release: (My question is why hasn't CIRM done any clinical trials for CP? Also, I love how this blogger assumes to know what parents like to hear and what they don't like to hear as if they are mindless idiots - that's my take of his comments anyway).

4-15-14

http://cirmresearch.blogspot.com/2014/04/a-placebo-controlled-trial-in-cerebral.html

A placebo-controlled trial in cerebral palsy might unlock some answers for parents

The parents of children with cerebral palsy (CP) rank high in number among the desperate calls that come to CIRM wanting to know about stem cell therapies offered on the internet. They don’t like to hear that we have very little information suggesting benefit from stem cells in these kids and that there is little reason to believe the types of cells being offered could grow new brain tissue to repair the abnormal brain development seen in CP.

They like hearing that there is some evidence that the type of stem cells being used might be able to tamp down any inflammatory process that is hampering brain function, and that it might be possible for these cells to trigger some sort of innate repair mechanism within the child. But the bottom line is that we really don’t know, and we certainly don’t know what is the best cell type to use and how, when and where to deliver the cells to get the maximum benefit if there is any.

So, it was heartening to see that a clinical trial registered with the Food and Drug Administration is enrolling patients in a study designed to answer some of these questions. Sponsored by Cord Blood Registry and conducted at the University of Texas Health Sciences Center at Houston the trial will compare two types of stem cells that came from the children themselves. They will compare stem cells from some children’s own stored cord blood with stem cells from other children’s bone marrow.

The research team plans to recruit 15 children into each group and 10 in each will receive the stem cells and five will receive a placebo injection, containing no cells. The parents will not know which injection their children received but at the end of one year, the parents of those who received the placebo will be told and given the option of a stem cell injection.

The Los Angeles Business Journal was one of many outlets that picked up the company’s press release. In it, the lead researcher, UT’s Charles Cox, explained the effect they hoped to see: "There is preclinical data indicating that the ongoing neuro-inflammatory response is a driver of further injury in cerebral palsy so the hope is to reduce this neuro-inflammation. Our goal is to break the cycle of inflammation and injury." They plan to evaluate the children as six months, one year and 24 months. So, anxious parents may start to get a few answers in a year or so. CIRM convened a workshop on how stem cell science could impact CP and that cerebral palsy report is available online.

Don Gibbons
 
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