Newsletter - Issue 2, April 2012
Let's Cure CP is what happens when parents refuse to accept the status quo.
Founded by two families who want to see a better life and future for their children with cerebral palsy, Let's Cure CP launched in 2010 as a 501(c)(3) non-profit organization committed to funding the support of CP research. All available funds go directly to research institutions in the United States to fund credible studies and groundbreaking clinical work that take us closer to a cure.
Co-Founders Ed Drambel and Lizette Dunay, both Atlanta parents of children afflicted with CP, explain the urgent need for and drive of Let's Cure CP:
We are so lucky to live in an era where there are so many advancements being made in regenerative medicine! Brilliant doctors and scientists are proving that neurological regeneration of damaged brain tissue is possible. However, these scientist and doctors are receiving very little funding to advance this exciting research toward a cure. That's where we come in.
As parents and advocates, we started asking questions and knocking on the doors of the leading pediatric Neurologists and Neurosurgeons in the United States. We went to them with the questions that so many parents are asking:
- What can we do to help our kids?
- We are hearing so much in the news about regenerative medicine. Why can't we sign our kids up for a study?
- Where are the studies for CP?
- Why are there so few?
It became clear that doctors want to do more and are excited about the potential outcomes... but there is limited funding available to them. We learned that, related to other conditions and the percentage of people affected, CP is vastly underfunded at the federal level.
CP affects over 900,000 Americans and growing, and we knew in our hearts that we needed to raise awareness and funding. We saw a need for a national organization whose sole mission is to raise money and advocate for the medical community working toward a cure.
As parents, we feel it is our duty to help our children and as many others as we can along the way. We want to help those scientists uncover new discoveries and help change the future of all individuals living with CP.
Overview of Clinical Trials Treating Cerebral Palsy with Cord Blood
The vast majority of the media stories about cord blood therapy for cerebral palsy (CP) have been anecdotal reports of children who were treated by the group of Joanne Kurtzberg, MD, at Duke Medical Center. However, there are now multiple trials running around the world and more planned, so it is helpful to compare and contrast some of the trials. This overview is simply meant to be informative, not a comprehensive list or a rating of trials.
This type of therapy started because Dr. Kurtzberg noticed that children who were receiving cord blood transplants for hematologic disorders were displaying remarkable improvements in neurologic skills. In 2004 her group began giving autologous (their own) cord blood stem cells to young children who were diagnosed with various "acquired neurological disorders", a label that includes CP as well as other diagnoses such as anoxic brain injury, hypoxic brain injury, periventricular leukomalacia, encephalopathy, hydrocephalus, and in-utero stroke.
By the end of 2009, 184 children had been treated at Duke and were reported in a publication. Those cases showed that the procedure is safe, but there was no control group against which the efficacy of the treatment could be measured. The following clinical trials divide the patients into "arms", and each arm of the study receives a different treatment, enabling the effect of the treatments to be measured against each other.
In Korea, Minyoung Kim, MD PhD, is the lead investigator of a trial (NCT01193660) that has used allogeneic (donated) cord blood to treat 105 children with CP. The study ran from May 2010 to April 2011 and the results have been submitted for publication. The study had 3 arms: rehabilitation only, rehabilitation plus erythropoietin (a hormone that stimulates bone marrow to produce stem cells), and rehabilitation plus erythropoietin plus a matched cord blood donation. Since the effects of cord blood stem cells and erythropoietin are expected to be similar, that study is not likely to yield conclusive results about the efficacy of the cord blood infusion.
The table below compares another 4 trials that treat CP with cord blood. In all of these trials, the patients are divided into 2 arms. In those trials which enroll children who have stored their own autologous cord blood, both groups of children receive stem cells, but the difference between the two arms is whether the children receive their cells right away, or after a time delay. Basically the 2 arms test stem cells now versus later, and the length of the time delay varies between the trials. The study in which children receive donated allogeneic cord blood tests stem cells against a control without stem cells. To keep the patients blind as to who received stem cells, most trials give a "sham" infusion to the control group. In Australia, the ethics review board does not approve of subjecting children to sham infusions, so instead that study will emphasize double blind neurological assessments.
Hopefully, by 2013 or 2014 we will see the first publications from controlled trials that can measure the efficacy of cord blood for the treatment of cerebral palsy in young children.
|Eligible Diagnosis||Cerebral Palsy|| Spastic
|Cerebral Palsy||Cerebral Palsy|
| Cord Blood
|Institution||Georgia Health Sciences University, USA||Duke University, USA||Monash University, Australia||Sung Kwang Medical Foundation, Korea|
|Investigators|| James E Carroll
Roger A Vega
Lloyd O Cook
| Joanne Kurtzberg
| Suzie Miller
| Minyoung Kim
Su Jin Jang
Myung Seo Kang
Sang Heum Kim
|Start Date||January 2010||June 2010||Pending||February 2012|
|Both arms get stem cells?||Yes||Yes||Yes||No|
|Delay between infusions||3 months||12 months||6 months||None|
Clinical Research on Stem Cells in Orthopedic Medicine
Knee arthritis is a very common problem, afflicting about 1-2 in 10 Americans and getting more common with a heavier population. For some patients with early arthritis (like tears in the meniscus due to age), a common treatment has been arthroscopic surgery. Regrettably, recent research shows that this type of surgery isn't effective. For more severe problems like holes in the cartilage, some patients try micro fracture or autologous chondrocyte implantation (ACI). The micro fracture procedure involves poking holes in the bone to stimulate a less than perfect cartilage repair. The ACI procedure uses cultured cartilage to be placed into the hole and surgically sewn into place. The problem with both procedures is that they don't apply to patients who are older or who have more extensive arthritis. For those patients, the only option is simply waiting until you're old enough to need a knee replacement.
There may be a better way. Stem cells live in all of our bodies. They can be easily collected, concentrated, or grown to bigger numbers and years of animal research has shown that they can help repair cartilage. We began using bone marrow derived stem cells in our practice in 2005. Since then we have performed thousands of stem cell based procedures. This procedure is a "needle out/needle in" procedure where the cells are harvested from the back of the hip area with a needle, either concentrated the same day or grown over several weeks to bigger numbers, and finally re-injected back into the injured area. We have developed several technologies that help us get cells to the right spot via a needle and/or that help cells stay at that spot to act on the injured tissue.
We first published "imaging case reports" or reports on patients who had impressive repair of their cartilage or where we noted a bigger meniscus after treatment. We then published large safety studies with hundreds of patients followed for years, showing that these stem cell treatments were safer than the surgical approaches they help many patients avoid. More recently we have published efficacy data for patients who were mostly knee replacement candidates that showed very good results when compared to a group of patients we didn't treat.
Who is a candidate for this kind of therapy? We have treated many different orthopedic conditions including partial rotator cuff or ligament tears, non-healing bones, and arthritis. The therapy is always done without surgery, as our long range goal is to switch many patients who now need more invasive surgeries to non-surgical methods of treatment. The treatment is not magic, in that we have patients who respond and patients who fail to respond. We believe this is both due the severity of the problem (some problems are just too severe for a stem cell injection to help) and the health of the patient's stem cells (patients on multiple medications for example have stem cells that are generally less healthy than someone who is not on medications).
In summary, we feel that the use of a stem cell injection is a viable alternative to surgery for many orthopedic patients. While the technology is still advancing, we feel that after gaining experience from several thousand stem cells procedures, we have a good sense of where this technology tends to work and where it tends to fail. The future for these therapies is very bright.