Cerebral Palsy: Cord Blood Stem Cell Research and Treatment in Clinical Trials – Update

For those of  you who follow our blogs, you know well that this is a topic of interest for us.  It bears repeating – our job as lawyers is to properly investigate potential claims of malpractice in areas such as cerebral palsy and seek redress for our clients when the evidence demonstrates a connection between birth injuries and medical care, but the much more important topic for our clients and victims of cerebral palsy is in the field of medical research. It is through research efforts – including clinical trials – that this dreaded condition will be ameliorated and hopefully eradicated. Trust me, after practicing law for over 35 years, I’m not worried about job security – the frailties of the human condition will more than suffice to fill our file cabinets with people to help due to the negligence of others.

We have reported previously on various topics involving cord blood and stem cell research as they relate to a number of conditions, including cerebral palsy.  It seems that months have passed since there has been any significant news about two programs underway: one at the Medical College of Georgia and the other at Duke.  Earlier this month, an update came across the social media network via a post by Singularity Hub – Cord Blood Stem Cell Treatment for Cerebral Palsy in Clinical Trial | Singularity Hub.

Here’s our encapsulated version regarding the studies and Singularity Hub’s report.

Photo provided by MSNBC

Duke University

According to the website ClincalTrials.gov, Duke began a clinical therapeutic trial – identifier: NCT00593242 – in January 2008 (estimated completion date of January 2011) whose primary purpose is listed as treatment of newborns with hypoxic ischemic encephalopathy (HIE) – inadequate oxygenation in the perinatal period for purposes of this study – through the controlled “collection, preparation and infusion of a baby’s own (autologous) umbilical cord blood in the first 14 days after birth if the baby is born with signs of brain injury.” For information concerning the inclusion and exclusion criteria for participation in this clinical trial, see the online posting. Essentially, the babies are then to be “followed for neurodevelopmental outcome at 4 – 6 and 9 – 12 months at Duke’s Special Infant Care Clinic. MRI’s will be obtained between postnatal weeks 1 and 4, and, for study purposes at 4 – 6 postnatal months.”

While other aspects of processing and administration are no doubt part of the key components of this project, it is readily apparent that the end-point goal is discovery of an effective treatment of cerebral palsy for the identified neonates in the study and then development of a second stage clinical trial to take such treatment modality to a greater number of potential beneficiaries.

Medical College of Georgia

For detailed information on this study, which began in February of this year, similar information is available through ClinicalTrials.gov under identifier NCT01072370.  This clinical trial investigation has a patient population consisting of children from ages of 2 to 12, “whose parents have saved their infant’s cord blood, who have non-progressive motor disability, and whose parents intend to have a cord blood infusion.”  Again – for full details regarding inclusion and exclusion criteria, see the full online posting.

For those parents who may be interested in determining if their child would qualify to participate, the study is still recruiting participants.  The contact information is also available at this link: Contact: James E Carroll, M.D.     706-721-3371     jcarroll@mcg.edu

Today’s report from Singularity Hub provides some encouraging – albeit anecdotal – news of potential progress.

The anecdotal evidence in support of treating cerebral palsy with cord blood stem cells is astounding. Much of it has actually been been performed at Duke University by one of the investigators in the pilot study: Joanne Kurtzberg. Among those that have been successfully treated at Duke include Ryan Schneider, Maia Friedlander, Chloe Levine, and Dallas Hextell. All had CP or CP-like symptoms and all made remarkable recoveries after cord blood stem cell treatments. Dallas Hextell, who showed improvements just 5 days after his therapy was featured on the Today show (the original report contains video compliments of MSNBC).

In addition to the early good news coming out of these projects, one other lesson is learned – for the time being, the storage of cord blood is an important component for those hoping to participate in such studies – particularly that being conducted at the Medical College of Georgia.  We have earlier reported on this topic as well.  You may want to refer to our early posting for some basic information if you are interested.

Obviously, the implications – if these projects prove to be successful – are far-reaching. The enthusiasm of the participants in these research projects is not limited to them alone. The words of the author, Aaron Saenz, from Singularity Hub somewhat tells it all:

So we have some exciting news for cerebral palsy, and some exciting news for those thinking about cord blood. What about the rest of us? Well the MCG and Duke work has some far reaching implications. Neurological damage, whether it’s caused by oxygen deprivation or some other injury, is one of the most difficult things to heal in the body. Work in animals (like that done by Carroll on rats) show that stem cells can not only help damaged brain cells recover, but they can also replace cells that have died. We may find that stem cells therapies have a wide range of applications for many different forms of brain damage. Kurtzberg is researching many different ways cord blood could be used (autologous or through donors) to treat a variety of conditions. In other words, today stem cells conquer cerebral palsy…tomorrow, the world.

Let’s all hope that Mr. Saenz is a prophet.

Children With Cerebral Palsy May Benefit From Cell Transplants

Since our firm at any given time handles a number of cases involving cerebral palsy, I am constantly trying to find useful information about new developments in therapy, research, procedures – anything relevant to the topic of preventing or helping those who suffer from cerebral palsy.

This morning I came across a report in Medical News Today –Children With Cerebral Palsy May Benefit From Cell Transplants – which, I must admit, I had not heard of before.  Apparently a group of Chinese researchers are now hypothesizing that a type of cell known as olfactory ensheathing cells (OECs) may hold promise for treatment of infants and those in early childhood with cerebral palsy.  

Clearly this research by the Chinese is not yet being conducted on a large scale level. Nevertheless, I thought I would report on this since the ‘science’ (in which I don’t purport to have expertise) seems logical and potentially noteworthy. With those qualifiers in mind, here is what’s behind this ‘thinking.’  Rather than try to give you the layman’s recap of OEC’s, I’ll let the article speak for itself.

Transplanted OECs, known to retain exceptional plasticity and promote olfactory blood vessel growth while offering neuroprotection, have been demonstrated to be potentially useful for a number of neurological disorders, including multiple sclerosis, spinal cord injury and amyotrophc lateral sclerosis (ALS).

So what does this mean for those who suffer from cerebral palsy? The corresponding author Dr. Hongyun Huang of the Beijing Rehabilitation Center provides the details:  “We conducted a randomized, controlled clinical trial with 33 volunteers, 14 of whom completed the six-month study, to determine if transplanted OECs were effective in treating children and adolescents with CP, given that CP shares many of the same features of other degenerative diseases.”

According to Dr. Huang, a protocol was developed from this hypothesis based on their knowledge of a key location “in the brain’s frontal lobes (defined as the “Key Point for Neural network Restoration (KPNNR)” based on previous studies) for injecting OECs and that the injected OECs would produce Schwann cell-like myelin sheaths around demyelinated axons.”  Demylinated axons are often referenced as a rather consistent finding when imaging the brains of victims of cerebral palsy.

Buoyed by the findings of this admittedly small trial group, Dr. Huang reports:

“Our results showed that transplanting OECs into CP patients could improve the neurological function of the patients and did not cause significant side effects. The procedure may be a plausible method to treat this as yet incurable disorder.”

For anyone who saw the recent 60 Minutes report (21st Century Snake Oil) on charlatans advocating a cure through injection of stem cells (for virtually any malady under the sun), caution takes center stage with all such reports.  No – I’m not suggesting that the Chinese study is voodoo medicine by any stretch of the imagination.  For those who keep up with this field of stem cell research and its potential applicability to treatment of cerebral palsy, you are aware of the recent (early February of this year) announcement by the FDA of an approved trial for stem cell research for victims of cerebral palsy. In addition to our having reported on this FDA-approved trial, I have also reported on a collaborative research program called Newborn Possibilities Program in Arizona, which is yet another collaborative effort using stem cell research concepts to improve the lives of these children.

While hope reigns eternal that meaningful research will lead to meaningful cure or improvement in the quality of life for cerebral palsy victims, Cell Transplantation associate editor Dr. Cesar V. Borlongan offers the following words of caution concerning the Chinese (and other) stem cell clinical trial:

In parallel with recently FDA-approved US clinical trials of cell therapy for adult stroke and cerebral palsy, this clinical study in China advances the use of stem cells for treating brain disorders, but a very careful assessment of this experimental treatment needs to be exercised in order to gauge its safety and efficacy.

Through social networking media such as LinkedIn and Twitter, I have come across some fascinating people who have devoted their lives to stem cell research.  If perchance any of my ‘connections’ and ‘followers’ out there have further information about this Chinese research and other stem cell research programs involving cerebral palsy, I would greatly appreciate your sharing your information with the Nash Law Firm community of readers by posting helpful comments to this blog.

Cerebral Palsy: new research to improve dexterity by home therapy using modified gaming instruments.

Today I came across yet another interesting and common sense concept in the quest to help those with cerebral palsy for performing important dexterity exercises in the comfort of their home.  The article – Daily Targum – Researchers spawn new therapy for cerebral palsy patients – recounts a small study (3 teenage patients) taking place over the last year at Rutgers University and Indiana University using a modified Sony PlayStation 3 gaming glove to improve dexterity for victims of cerebral palsy.  

One of the keys in this research project is to find a way to move therapy into the patient’s home utilizing an activity that all kids enjoy – gaming.  The basic goal is to not only move important therapy into the home but to provide a method for young cerebral palsy patients to perform this therapy without the need of costly and time-contrained supervision.

The program is the product of a collaborative effort of these universities headed by Grigore Burdea, a University professor of electrical and computer engineering, and Moustafa Abdelbaky, an electrical and computer engineering graduate student.  Another key player in this endeavor is Meredith Golomb, an associate professor of neurology at Indiana University School of Medicine.  She found out about Burdea’s work through the Internet and said the combination of her skills with Burdea’s was perfect.

“I’m a pediatric neurologist and know how to assess the kids medically,” Golomb said. “He is an engineer and knows how to get the systems working — it has been a great collaboration so far.”

Some weeks ago, I posted a story about research underway at the University of Michigan in the use of a treadmill to help improve the neuromotor development of younger children with cerebral palsy.

It is through the work of such researchers and many others devoted to helping discover the causes of cerebral palsy that key progress in making the lives of these people with special needs better will be made.  We will  keep you posted on similar studies and research efforts.  Hopefully, if you are the parent of a child with cerebral palsy, you will find one of these techniques of interest and potentially useful in maximizing the chances of a better life for your child.

CDC Features – Data Show 1 in 303 Children Have Cerebral Palsy

Cerebral palsy – how common is it? A recent “Features” posting by the CDC (Centers for Disease Control and Prevention) provides some answers and gives parents some ‘early signs’ of which to be aware.

While everyone knows that cerebral palsy (CP) can be a devastating condition, it is less well-known how common CP actually is. The Center for Disease Control recently released new data on the frequency of CP. In its study, it found that CP affects 3.3 per 1,000 eight-year-old children, or 1 in 303 children. This data was collected from select communities in Georgia, Alabama and Wisconsin, not the nation overall. Rates may differ slightly in other localities. However, the CDC pointed out that its most current findings on CP frequency were similar to previous studies which showed that CP affected 3.6 per 1,000, or 1 in 278 children.  

In reporting the data, the CDC also advised parents what to look out for in terms of signs of CP, based on the age of the child. Parents should consult a physician if they notice any of the following signs:

A child over 2 months with cerebral palsy might have difficulty controlling head when picked up, or have stiff legs that cross or “scissor” when picked up;

A child over 6 months with cerebral palsy might continue to have a hard time controlling head when picked up, or reach with only one hand while keeping the other in a fist;

A child over 10 months with cerebral palsy might crawl by pushing off with one hand and leg while dragging the opposite hand and leg, or not sit by himself or herself;

A child over 12 months with cerebral palsy might not crawl, or not be able to stand with support;

A child over 24 months with cerebral palsy might not be able to walk, or not be able to push a toy with wheels.`

Parents, be aware of these early signs!  If you are not sure what to do or to whom you can turn, the CDC offers the following information:

To find out who to speak to in your area, contact the National Dissemination Center for Children with Disabilities by calling 1-800-695-0285 or visiting the Center’s Web site

Of course, you always have your child’s pediatrician as a starting point.

Zarontin – The First-Line of Treatment for Children with Absence Epilepsy

According to an article published in WebMD, researchers found that Zarontin, one of the oldest drugs used to treat epilepsy, is also one of the most effective drugs to treat this condition. The research study was published in the New England Journal of Medicine.

As many as 17% of children with epilepsy have absence seizures, also known as petit mal seizures, which involve brief but frequent staring spells that can occur dozens or even hundreds of times a day. These children do not have the convulsions typically associated with epilepsy, but they are at high risk for developing them later in life. During the 10- to 15-second seizure episodes, people with absence epilepsy become unresponsive and may stop walking or talking in mid-sentence. Children with these seizures often perform poorly in school.

Zarontin is the trade name of the anticonvulsant drug Ethosuximide. Ethosuximide is also known as Emeside. In addition to Ethosuximide, a physician’s other options include Valproic Acid and Lamotrigine. The study examined the efficacy of these drugs in treating absence epilepsy.

Lamictal was found to be significantly less effective than Zarontin or Depakote for preventing absence seizures, while treatment with Depakote was more likely to result in concentration problems than treatment with the other two drugs.

Moreover, Zarontin was found to have less of an impact on a child’s concentration and attention. If your child has absence epilepsy, be mindful that the newest and most expensive anticonvulsant on the market may not be your best option. Study co-author Shlomo Shinnar, MD, PhD, of New York’s Montefiore Medical Center noted:

Unless there are reasons not to use it, Zarontin would certainly be my first-line choice.

Contributing author: Jon Stefanuca

Cerebral Palsy Verdict: $23.3 Million – Minnesota – birth injury – delay in performing C-Section

This is a report from AboutLawSuits.com:  

The family of a girl who was diagnosed with cerebral palsy as a result of a birth injury has been awarded $23.3 million by a Minnesota jury after suing a hospital for waiting too long to perform a Cesarean section.

The Minnesota cerebral palsy lawsuit was filed against Rice Memorial Hospital and Affiliated Medical Community Center by Elise Rodgers, as a result of alleged negligence during the birth of her daughter, Kylie, in June 2007. According to a report by Minnesota Public Radio, Rodgers claimed that negligent medical care caused the girl to suffer severe brain damage because doctors failed to act quickly once the fetal monitor warned the child was being deprived of oxygen during labor.

The family argued that Kylie’s umbilical cord was compromised, and that doctors should have performed a Cesarean before she suffered permanent brain damage. Kylie, who now has cerebral palsy as a result of the negligent care, requires constant suctioning of her airway, sometimes as often as every three to five minutes, in order to survive, according to the lawsuit.

In a verdict handed down earlier this month by a Kandiyohi County jury, Rodgers was awarded $10 million for the child’s future medical expenses, $1.7 million for past medical expenses, $1.5 million for future lost earnings and $10 million for disability, emotional distress and pain.

Many research studies are underway and new therapies are being developed for infants with cerebral palsy.  Nevertheless, many children are still born with this condition and its spectrum of disabilities.  When these injuries are the result of medical negligence by physicians, hospitals and/or other health care providers, the civil litigation system is there to provide for these special needs children.  Contrary to politically-charged statements by those on the right, juries do understand when these life-altering injuries are caused by negligence.  When they do and the care needs are properly presented, verdicts such as this in Minnesota are the proper result.

New Research : Treadmill may help tots with neuromotor delays (such as cerebral palsy) walk

While the study group was a small one (15), researchers at the University of Michigan’s School of Kinesiology have reported supervised treadmill walking may help children with neuromotor delays.   This study was reported yesterday in an article published in The Hindu.

Rosa Angulo-Barroso, Associate Professor of movement science  at the School, and her colleagues followed 15 infants at risk for neuromotor delays over a period of two years.  They tested the changes in physical activity and treadmill-stepping performed with parental supervision in the children’s  homes.   Six of these children had been diagnosed with cerebral palsy.

“We found that in those with neuromotor delays, the pattern of development through time was parallel (but less) than normal kids.” said Angulo-Barroso. “We also found less toe-walking, so foot placement improved.” Angulo-Barroso added. The study also suggests a critical intervention window. Both children without a diagnosis and kids with cerebral palsy improved the most between 10 months and 18 months.

As The Hindu article notes, “The study, “Treadmill Responses and Physical Activity Levels of Infants at Risk for Neuromotor Delay” appears in the journal Pediatric Physical Therapy.

It would be most interesting to see if a larger, multi-center, double-blind  study could produce the same or similar results.  I haven’t personally seen the ‘tot’ version of a treadmill, but a quick search on Google images does show some interesting devices (see picture on left).  As researchers in the etiology and treatment of cerebral palsy keep working, the efforts of others such as reported here will potentially provide a wonderful interim measure toward helping these kids with special needs.  We’ll try to keep an eye on this story and will report should more information become available.

Announcement: Newborn Possibilities Program underway in AZ – stem cell research related to cerebral palsy

A posting yesterday in PRNewswire tells of a collaborative research effort called “Newborn Possibilities Program” in Tuscon, Arizona, for preservation of cord blood stem cells “so that if a child is diagnosed with the condition, he or she may be eligible to receive a new treatment being researched under approval from the FDA using the child’s own cord blood stem cells.”

This research initiative is being done as a collaborative effort by the Cord Blood Registry, the world’s largest stem cell bank; Save the Cord Foundation, a nonprofit advocacy organization based in Tucson; Tucson Medical Center, the region’s leading birthing hospital; and Watching Over Mothers and Babies Foundation (WOMB), a local non-profit research foundation.

A statement by the CEO of the Cord Blood Registry, Tom Moore, best describes the goal of this new program:

This program is a way for us to provide a safety net to children at-risk for cerebral palsy and other forms of neonatal brain injury while helping to advance the science of regenerative medicine.  CBR and our collaborators in this unique program share a commitment to advance cord blood stem cell research, expand education about the value of cord blood banking, and improve outcomes for children with neurological disabilities.

In a statement by the founders of one of the program’s collaborating members, Save the Cord Foundation, Anne Sarabia and Charis Ober,  we are provided further insight into the present and future goals of the program:

We believe that cord blood will play a very important role in future medicine and will benefit so many children in our community at risk for cerebral palsy.   Our dream is that this program will inspire other medical institutions to follow and make preserving cord blood the standard of care for all newborns.

Dr. Hugh Miller, head of WOMB and a specialist in Maternal-Fetal Medicine, who will be the program’s director at Tuscon Medical Center, said in the interview:  

The Newborn Possibilities Program is laying the groundwork for potential new treatment options for cerebral palsy and neurological disorders by connecting patients who may be at risk for these conditions and who have access to their cord blood stem cells with FDA-approved research studies. We believe this program will be instrumental in accelerating the pace of research and potentially helping many children.

As those of you who follow our blog postings and/or the recent news concerning stem cell research relating to cerebral palsy are aware, the Food and Drug Administration recently approved  a human clinical trial evaluating the safety and efficacy of using a child’s own newborn blood stem cells to treat cerebral palsy.

Having been involved in handling cases of children with cerebral palsy for many years, I have seen the issues of preservation of tissue, blood and medical records (i.e. fetal monitor strips) go the full gamut.  For years, placental pathologists were begging medical institutions to preserve placentas for further research into perinatal  birth injuries (among other concerns).  There was a time when one institution, which shall remain nameless, had a suspected policy of discarding fetal monitor strips (gee – wonder why that was!) and so on and so on.  It is really heartening to see that this new collaborative program is putting the interests of science, children’s health and knowledge far ahead of any conjured concerns relating to fear of litigation.    Hopefully, this program, in conjunction with all the other research regarding cerebral palsy now going on, will get to the bottom of this horrible disorder and provide new meaningful care options for at least some of the victims of cerebral palsy.

New BPA Research – Is Your Plastic Water Bottle Toxic?

According to an article published in WebMD Health News, two recent studies suggest that the plastic chemical bisphenol A (BPA) is not toxic to the brain and that it does not inhibit the development of the human reproductive system. One of the studies was funded by the plastics industry. Both studies were originally published in Toxicological Sciences.

BPA is an organic chemical compound, which is used as an additive or building block in several plastics. Its primary purpose is to harden plastic. For this reason, BPA is found in a wide variety of consumer products such as plastic bottles, cups, and even baby milk bottles.

“Some experts are concerned that exposure to BPA and its weak estrogen-like effects, especially during critical periods of development, may be linked to a range of health hazards, including behavioral effects, reproductive problems, cancers, heart disease, and diabetes.”

The two studies in question were designed to specifically study the impact of BPA on the brain and the reproductive system. Both studies used animal models.  The study that focused on neurotoxicity found that female rats and their litters did not develop neurological defects from exposure to BPA.  The study that focused on BPA’s impact on the reproductive system found that rats exposed to low doses of BPA in utero and throughout the breastfeeding period maintaind healthy reproductive systems.

Notwithstanding these new findings, many remain concerned about the toxicity of BPA. Even the FDA reversed its position regarding the safety of BPA in January 2010 and called for additional research on the subject. Previous studies appear to suggest that BPA is toxic. For example, in an article published in Reproductive Toxicology in 2007, 38 experts agreed that average levels of BPA in humans are above those that appear to cause harm in animals.  In 2009, an article publushed in Molecular and Cellular Endocrinology, sumarized past BPA research and concluded that:

“The potential for BPA to influence body weight is suggested by in vitro studies demonstrating effects of BPA on adipocyte differentiation, lipid accumulation, glucose transport and adiponectin secretion. Data from in vivo studies have revealed dose-dependent and sex dependent effects on body weight in rodents exposed perinatally to BPA. The mechanisms through which perinatal BPA exposure acts to exert persistent effects on body weight and adiposity remain to be determined.”

Another study published in the Journal of American Medical Association specifically addressed the impact of BPA on humans.  The study concluded that there was a strong positive correlation between the amount of  BPA in a person’s urine and the incidence of heart disease, diabetes, and enzyme abnormalities.  These are just a few of the many research studies suggesting a link between BPA and varous medical complications.

Contributing Author:  Jon Stefanuca

First FDA-approved stem cell trial in pediatric cerebral palsy

This is very exciting news – First FDA-approved stem cell trial in pediatric cerebral palsy.

Medical College of Georgia researchers are conducting the first FDA-approved clinical trial to determine whether an infusion of stem cells from umbilical cord blood can improve the quality of life for children with cerebral palsy.

While no controlled clinical trials have been conducted to date, previous studies have shown marked improvement in children with cerebral palsy about three months after an initial infusion of cord blood.

“Evidence up to this point has been purely anecdotal,” Dr. Carroll said. “While a variety of cord blood stem cell therapies have been used successfully for more than 20 years, this study is breaking new ground in advancing therapies for brain injury — a condition for which there is currently no cure.”

Here’s the protocol for the study:

Children will begin the study with a neurological exam by MCG pediatric neurologists Elizabeth Sekul and Nicole Brockway. Then, half of the study participants will receive an infusion of their own cord blood while the other half receive a placebo. Three months later, the children will be evaluated without physicians knowing which group received the stem cell infusion. Afterward, children who didn’t get the cord blood initially will receive an infusion. Children will return three and six months later for evaluation.

While this is exciting news for those of us who represent families with children having special needs, including children with cerebral palsy, I cannot even imagine what great news this is for the parents of such children.  Granted, this is perhaps just a first step BUT what a first step.  Here’s hoping it all goes extremely well.  We’ll try to stay up with this story.  Good luck to ALL involved in the project!