Cerebral palsy is a major health problem, affecting approximately 1 in 500 newborns. It is caused by damage to the brain by lack of oxygen before birth. The scientific rationale for the use of stem cells for this condition has been discussed previously in the video Stem Cell Therapy for Cerebral Palsy.

In a recent news announcement, a case of a child in Singapore with cerebral palsy that was treated with their own cord blood stem cells was discussed.
"It is quite a safe procedure. It is like a standard blood transfusion, except that you are using the cord blood cells that were stored. So there is no risk of a reaction, apart from perhaps minor hypersensitivity reactions, as in all blood transfusions," said Dr Keith Goh, neurosurgeon, Mount Elizabeth Hospital.

After the administration, the patient, 2-year-old Georgia Conn is reportedly calmer, with a decrease in constant crying an seizures. The parents, Michael and Louise Conn, previously stored Georgia’s umbilical cord blood cells. "Within two days, Georgia was noticeably happier. Just instantly more smiley, chatty and more energetic. That was the first real indication that something was going on," said Louise Conn. "And since then we all feel, and all her therapists feel, that her muscle tone has reduced, which is enabling her to achieve a lot more within her therapy sessions," she added.

Theoretically the risks of using a patient’s own cord blood stem cells are minimal since they are not manipulated, and are of the same genetic make up as the patient. However there are certain considerations, for example, "are there enough cells" to actually cause a meaningful effect? Additionally, what if the patient needs the cord blood cells later in life?

Other approaches to cord blood stem cell therapy include using cells from non-related cords, as well as expansion of the cord blood stem cells before using. The rationale for the non-related use of cord blood has been previously published (Riordan et al. Cord blood in regenerative medicine: do we need immune suppression? J Transl Med. 2007 Jan 30;5:8). Expansion of cord blood stem cells has previously been attempted by the companies Viacell and Aastrom. Although the technology is still a work in progress, some clinical trials have been performed with expanded cord blood cells in the area of hematological malignancies such as leukemias.

At a Town Hall meeting on October 6th, 2009, residents of Gilbert Arizona listened to medical advances being made in the area of adult stem cells for heart failure. Stem cell pioneer Dr. Nabil Dib, Director of Cardiovascular Research at the Mercy Gilbert Medical Center has been one of the first physicians in the United States to use a type of stem cell called myoblasts, for treatment of patients with heart failure. Since those early studies which began in Phoenix Arizona in 2000, thousands of patients have been treated with their own stem cells for heart failure.

Dr. Dib explained how after a heart attack the injured heart muscle produces chemical signals that attract stem cells from the bone marrow, as well as activate stem cells that are resident within the heart but usually not active. Unfortunately, the repair response after a heart attack is usually not very strong, and as a result, after a heart attack the heart continually loses function until heart failure can occur.

One of the techniques being performed at Mercy Gilbert is administration of stem cells within several days after the heart attack. When the stem cells are administered in the blood, they can "sense" that there is something wrong with the heart and try to repair it.

At the meeting Dr. Dib received many commendations for his excellent work. "Dr. Dib’s work for Mercy Gilbert rivals that found primarily in university and research hospitals, so we are proud and honored to have him working here in Gilbert at Mercy Gilbert Medical Center," Councilmember Joan Krueger said. The Mayor of Gilbert stated "History is being made in our town of Gilbert at Mercy Gilbert Hospital."

In addition to the advances in the use of myoblast and bone marrow stem cells, Dr. Dib explained how the hospital is opening up a public cord blood bank. Cord blood is a rich source of stem cells that have several unique properties because they come from tissue that is not mature. Traditionally, cord blood has been used for transplantation of patients with leukemias because the cord blood is capable of making a new blood system when given to patients who have been previously treated with very high doses of radiation and chemotherapy. The use of cord blood without radiation and chemotherapy was reported in many situations but until recently has not been used in the United States.

Researchers at University of Florida and Duke have started using cord blood for Type I Diabetes and Cerebral Palsy as part of clinical trials. The creation of a public cord blood bank at Gilbert will allow for researchers to conduct similar clinical trials.

Europe’s leading stem cell organization, the XCell-Center of Germany, has released results from a follow-up study in which significant improvement was seen in 67% of 45 cerebral palsy patients who were treated with their own autologous adult stem cells derived from bone marrow.

According to the press release, the most common improvement reported by the patients was improved hand and finger coordination, as well as less upper limb spasticity. Additionally, improvement in leg and foot coordination were observed in nearly half of the patients, with 40% reporting reduced lower limb spasticity, and 20% reporting improvement in walking ability. Speech improvement was also found in 40% of the patients, and 20% reported improved cognition.

As described by Mrs. Ritu Giacobbe, whose 13-year-old son was among the patients treated in the study, "Not long after the treatment, our son started speaking in full sentences. His fine motor skills have improved and he can now hold his fork and eat without help."

Other parents of children who were recently treated at the XCell-Center had nothing but praise for the therapy. According to the mother of a boy who was treated, "For Dominic, the most significant improvement has been his ability to focus his eyes." Similarly, according to the mother of another boy who received the therapy, "Some of the milestones are significant. Harrison can roll himself over now. He holds his head up without his chin sinking into his chest. His speech is clearer."

According to Dr. Ute Tamaschke, pediatric neurosurgeon at the XCell-Center, "These results confirm what we see in Germany on a weekly basis: that treating patients with their own stem cells yields positive results. Many of these children require less care and are now more independent. And this positively impacts the quality of life of the children and their caregivers. We couldn’t be more delighted."

The treatment involves harvesting a small amount of bone marrow from the patient’s hip via a procedure known as thin needle mini-puncture, from which the adult stem cells are then separated, counted, purified and readministered into the patient’s cerebrospinal fluid via a fine spinal needle between the L4 and L5 vertebrae. From the cerebrospinal fluid, the stem cells are automatically transported into the brain where they naturally target and regenerate damaged tissue.

The cost for the treatment for cerebral palsy patients starts at around 9,000 Euros.

With clinical treatment centers in both Cologne and Dusseldorf, Germany, the XCell-Center is the first privately-owned clinic in Europe to specialize in regenerative medicine using autologous adult bone marrow stem cell therapy. Since its founding in January of 2007, the XCell-Center has treated more than 1,600 patients – "safely", as stated on the website.

The XCell-Center uses therapies that are based exclusively upon autologous (in which the donor and recipient are the same person) adult stem cells derived from bone marrow. In no case are embryonic stem cells ever used. As stated clearly on their website, "Therapy with embryonic stem cells is strictly prohibited in Germany. At the XCell-Center, we only use the patient’s own stem cells for therapy."

In an article entitled, "Lack of patient access limits promising cell therapy", a number of examples are cited in which a simple lack of public awareness about umbilical cord blood is the only thing standing between many patients and adult stem cell therapy.

Such awareness begins with the birth of a child, and whether or not the parents are even informed of their options for storing the child’s umbilical cord blood. Private cord blood banks charge a fee that can be as high as several thousand dollars, while public cord blood banks offer free storage. According to recent surveys, however, nine out of ten parents choose neither option, which means that the umbilical cords – along with all the highly potent adult stem cells that are contained within the cords – are discarded as medical waste. Even when a conscious choice is made to bank umbilical cord blood, many adult patients in hospitals around the nation who could benefit from the cord-blood-derived adult stem cells are unaware that such a possibility exists.

Known to be an extremely rich source of highly potent adult stem cells, umbilical cord blood has a long and carefully recorded history as it has been used for a wide variety of clinical applications for decades, safely and effectively. Additionally, such uses have been routinely reported in the medical literature for more than half a century, predating World War II, and also predating any clear scientific understanding of a human stem cell. With the more recent development of the modern concept of a stem cell, umbilical cord and placental blood are now recognized as excellent sources of adult stem cells that can be used in the treatment of a broad range of diseases and injuries. Furthermore, when one considers the number of births that occur daily, throughout the world, umbilical cord blood represents a virtually limitless supply of versatile adult stem cells which otherwise would simply be discarded as waste. Unfortunately, most of the time, this is exactly what happens.

In 2003, Ryan and Jenny Levine chose not to bank the cord blood of their newly born daughter, deciding that the cost was too high. When another daughter was born in 2006, however, they decided to bank her cord blood, paying the $2,000 fee for a private banking facility in Tucson. When the second daughter was diagnosed a year later with cerebral palsy, she was able to receive stem cell therapy from her own cord blood stem cells.

As an infant the child would only reach for her bottle and toys with her left hand, while her right hand remained clenched to her chest in a fist. Likewise, instead of crawling, the child could only scoot along the floor, with her right leg dragging behind her. Her cerebral palsy was suspected of being caused by an in-utero stroke, which could have left the child crippled for life. Instead, when the child was treated with her own adult stem cells derived from her own umbilical cord blood, she began improving within days. Within two weeks, the stiffness on her right side that had been evident since birth was no longer detectable. She was reinfused a second time with her own stem cells in May of 2008. Today, she is able to catch a ball with both hands, and she uses all four limbs to ride a tricycle. The autologous (in which the donor and recipient are the same person) adult stem cell therapy was performed as part of an FDA-approved clinical trial conducted at Duke University in North Carolina. According to the child’s mother, Jenny, "I tell anyone and everyone who is expecting a child that this is something that they need to at least consider."

Similarly, when Al Copeland of Phoenix was suffering from leukemia in 2007, he faced certain death within days after the bone marrow transplant that he had been awaiting fell through. Although he had never heard of cord-blood-derived adult stem cells, his physicians turned to a public cord blood bank in order to obtain the regenerative cells that saved his life. According to his physician, Dr. Jeff Schriber, who is also medical director of the Banner’s Blood and Marrow Transplant Program, "He didn’t have much time. Fortunately, we were able to get the cord blood quickly." Now that Mr. Copeland has been cancer-free for 18 months, he is a regular, voluntary visitor at Banner’s oncology unit, where he visits and encourages transplant patients, offering a sympathetic ear to their fears and concerns. As Mr. Copeland puts it, "If a poor kid from south Texas can do this, so can you. It’s going to be hard. It’s going to be a struggle. But you can pull it off, man. You can." As 57-year-old Graig Stones listens attentively, Mr. Copeland explains that the stem cells saved his life, adding, "It made me realize there’s a lot more to living than just getting up in the morning, putting on your shoes and socks and going to work each day."

Adult stem cells from umbilical cord blood have already been used to treat more than 70 illnesses over the past two decades, though most people are unaware of such facts. According to the National Marrow Donor Program, the number of units stored in their public network of cord-blood banks, which is now at 150,000, has more than tripled over the past five years, although this represents only 3% of the 4 million births that occur annually in the United States.

In addition to a severe lack of public education on the topic, experts cite two main obstacles that limit the availability of cord-blood stem cells, namely, cost, and an inadequate number of collection sites. The private Cord Blood Registry, based in San Bruno, California, for example, charges a $2,000 collection fee in addition to an annual $125 storage fee, though this guarantees the customer direct and exclusive access to his or her individual adult stem cells throughout the future, whenever necessary. Public cord blood banks, by contrast, allow neither direct nor exclusive accessibility to one’s donated cord blood stem cells, though donation is free, and the stem cells are made available to anyone in the general public who may need adult stem cell treatment. At the time of this writing there are currently only 19 public cord blood banks throughout the U.S., most of which have agreements with hospitals on the east and west coasts but nowhere in between; consequently, availability of their services is virtually nonexistent in other parts of the country. Additionally, the cumbersome amount of paperwork that is required for donation to a public bank is often a strong disincentive for many parents. Among other things, expectant mothers must submit a complete medical history prior to the third trimester of pregnancy and make their own arrangements for mailing the cord blood to the storage facility immediately after birth. Clearly, a donation process such as this would be more efficacious if it were simplified. According to Dr. Jordan Perlow, "Every day I have patients who say, if there’s an easy way I could donate, I’d love to do it," but an easy way does not yet exist. Although in recent years the U.S. federal government has initiated programs to increase public awareness of cord blood banking, in actuality the accessibility of such banking services still lags behind expectations. Since only 17 states have passed legislation requiring expectant mothers to be informed of cord blood preservation options, most new parents are still unaware that such possibilities even exist.

Advocates of public cord blood banking point out that the services have already saved numerous lives, even though the system is far from perfect. The highly potent mesenchymal stem cells that are found in cord blood are "immune privileged", meaning that they do not require an identical matching to the recipient, as bone marrow does, and therefore even one donated cord blood unit can potentially treat a large number of people, for a wide variety of illnesses and injuries.

In 2008, the National Marrow Donor Program, a Minnesota-based non-profit organization that handles requests for all publicly available cord blood, provided cord blood units for the treatment of nearly 900 people, signifying a 40% increase over 2007. Still, public cord blood banking remains significantly less popular than private banking, despite the difference in cost. New business models have been proposed for the public banking systems, which have set a goal of doubling their number of units, to 300,000, by 2015. Because the public banks bear the entire cost of the services, however, the question of funding is a serious one. By contrast, the Cord Blood Registry, which is the oldest and largest private cord blood bank in the world, already has over 270,000 cord blood samples in its inventory, which is 80% more than the number of samples in the "Be the Match" public-donor network. According to Tom Moore, CEO of the Cord Blood Registry, there has been a consistent 30% annual growth at the Registry over the past several years.

Nevertheless, private cord blood banks are increasingly attracting criticism for their exclusivity, as critics are increasingly encouraging a wider use of the more open services offered by public banking companies. In a policy statement issued in January of 2007 by the American Academy of Pediatrics, for example, it was estimated that the odds of any particular child actually needing to be treated with his or her own cord blood ranged from 1 in 1,000 to 1 in 200,000. There are therefore many physicians who agree with the Academy in urging greater use of public banks over private banks, since it is only the units stored in public banks which are made available to any patient anywhere who may be in need of adult stem cell treatment.

Umbilical cord blood has been documented in the peer-reviewed medical literature for its broad range of clinical therapeutic applications for over half a century, long before the concept of a stem cell became a topic of general interest. Today, a number of clinical trials are being conducted in which cord-blood-derived stem cells are being studied as a treatment for a wide variety of diseases and injuries, as these highly versatile adult stem cells are proving to hold greater, more concrete and more tangible therapeutic value than embryonic or iPS (induced pluripotent stem) cells, both of which are still in the experimental stages.

Unfortunately, neither the popular media nor, therefore, the general public, seem to be aware of such facts.

A multinational group of scientists has developed a type of conditioned media from adipose stromal cells which they have utilized to protect the brain against hypoxia- and ischemia-induced brain damage in neonatal rats.

Led by Dr. Xing Wei of the Department of Neurology at the Indiana University School of Medicine, the scientists used a neonatal Sprague-Dawley rat model of cerebral palsy to assess the protective properties of the adipose stem cell conditioned media on neurological tissue, from which they found that the conditioned media has a protective effect on brain cells when the media is administered either one hour before, or 24 hours after, the induction of ischemic injury. Specifically, the scientists observed protection against a loss of brain volume in the hippocampal and cortical regions of the brain. Additionally, the conditioned media was also found to preserve and protect mental function as measured according to the Morris water maze test. Possible mediators that were identified in the protective mechanism of the media included IGF-1 (insulinlike growth factor 1) and BDNF (brain derived neurotrophic factor).

Autologous (in which the donor and recipient are the same person) adult stem cells of several varieties have already been widely documented for their ability to mediate neural protection subsequent to brain insults such as stroke, after which it is already known that bone marrow stem cells, for example, are naturally mobilized, and the extent of a patient’s bone marrow stem cell mobilization is directly related to the extent of his or her post-stroke recovery. Additionally, autologous adult stem cells derived from umbilical cord blood have been widely and repeatedly demonstrated to have therapeutic effects in children with cerebral palsy, as reported especially by Dr. Joanne Kurtzberg at Duke University. Now Dr. Wei’s study sheds further light on the phenomenon by identifying with greater specificity the mechanisms of action that are involved in such therapeutic recovery.

Dr. Wei and his colleagues in Indiana conducted the study in collaboration with researchers in Germany, Ireland, and at the Rockefeller University in New York. The publication appeared in the IFATS Series, of the International Federation for Adipose Therapeutics and Science.

Caring for babies suffering from cerebral palsy or those who suffer a stroke may soon follow a protocol similar to what Dr. Tadashi Masuda and Dr. Mina Maki are performing today. They are preparing for a transplant, the first step: placing two syringes inside an ice packed plastic foam box.

Protocols that could be followed in any clinic in the country are being developed by Cesar Borlongan and his team inside the laboratory at the Augusta Department of Veterans Affairs Medical Centers. While establishing a systematic method, they hope to also prove that adult bone marrow-derived stem cells are effective in helping repair the brain damage.

This particular stem cell therapy is being studied in animal models by Dr. Borlongan, also an associate professor at the Medical College of Georgia, and Dr. David Hess, MCG chairman of neurology. The research is being facilitated by a $4.2 million dollar grant which will span five years. The same therapy will be studied for use in babies who, due to lack of blood or lack of oxygen around the time of birth, suffer brain damage. 10 percent of the cases of cerebral palsy can be attributed to this this circumstance. This second study will funded by an additional $1.8 million dollar grant spanning three years, and the research will be carried out with James Carroll, chief of pediatric neurology at MCG.

The research on cerebral palsy may be approved for human trials sooner since the procedure has already been proven to be relatively safe and there is a lack of existing therapies for the condition as well.

“I would hope we would get a start on this in a year,” Dr. Carroll said.

The researchers were looking for methods that could be duplicated easily, such as shipping the cells in a liquid nitrogen container to thawing them and loading them into syringes for injection into an IV. Just as if they were trying to get the FDA to approve a new drug, the approach for the stroke model has been standardized said Dr. Borlongan.

“This is the same way we’re going to do it in the clinic,” he said. “Once the cells arrive in the clinic, all the clinicians will do is thaw the cells and then inject them into the patient.”

Dr. Borlongan said that they now believe the benefit to stroke victims might be from growth factors secreted by the cells as opposed to the former theory that the benefit was derived from the replacement of damaged cells.

“Once they release these beneficial substances, they help rescue the dying cells from the host tissue,” he said. “And they also increase the production of new cells.”

Dr. Carroll said that neonatal patients could also benefit from the treatment.

“We think it may have some additive effect in terms of new brain cells, but the main effect has to do with assisting with brain repair,” he said.

Many stroke patients arrive at the hospital too late to meet the deadline of a three-hour window in which a clot-busting drug must be given. The possibility of creating a treatment that could provide benefits past this point of no return has sparked the interest of many. Even days after stroke, observations from animal models showed that the treatment made a difference according to MCG researchers.

“What we have seen with the stem cells, even after seven days post-stroke, you can get functional benefit,” Dr. Borlongan said. “But because most stroke patients are discharged after a few days, they chose a two-day time frame to deliver the stem cells. And because these are adults cells, and not the embryonic stem cells that have generated much controversy, they can sidestep some of those ethical concerns,” Dr. Borlongan said.

Dr. Borlongan said that prior to using human cells, rodent cells will be the focus in order to prove safety and effectiveness. Human trials could follow in the fifth year, when the team plans to apply to the Food and Drug Administration.

“Hopefully, after the five-year project, we’ll have something to give to the patient,” he said.

Highlighting the increasing therapeutic use of autologous (one’s own) cord blood stem cells for transplant and regenerative medicine, including treatments for neurological repair, juvenile diabetes, and blood and immune disorders, two separate data abstracts were displayed today at the annual scientific meeting of the AABB. The advancement of these treatments was part of the focus as well, and the important role of family (or private) cord blood banks was demonstrated. The international association of medical professionals and institutions focused on transfusion, transplantation and cellular therapy.

In order to treat aplastic anemia, an individual’s own cord blood stem cells were released in the first study which analyzed four cases. The transplants were conducted at three different institutions: Children’s Hospital in Seattle, City of Hope (Los Angeles), and the University of Minnesota. Cord Blood Registry was responsible for processing and storing the cord blood prior to treatment.

The cases demonstrate that this approach is amenable to use at different treatment centers across the United States and also suggests that autologous cord blood transplantation for aplastic anemia is a safe and effective treatment protocol.

“Aplastic anemia is a life-threatening disease with no known cause that can be acquired at any time in life and is difficult to treat,” said lead study investigator Dr. David T. Harris, Ph.D., professor of immunology at the University of Arizona and scientific director of Cord Blood Registry. “This study offers evidence that transplant physicians have a safe and effective weapon for combating this disease for patients who have access to their own cord blood stem cells.”

The analysis also showed that:

— The average time to engraftment (the point at which the stem cells start to generate new blood cells) for cord blood is between 21 and 35 days according to the National Marrow Donor Program (NMDP). Engraftment averaged 22 days across the four cases and in one patient, occurred as early as two day after transplantation.

— Marking the longest period of time a family-banked cord blood sample has been stored prior to use, one of the autologous samples used was stored for 9.5 years.

Use in both regenerative and traditional medicine applications was documented in a second report involving 13 cases of autologous cord blood stem cell use. An increase in samples requested for regenerative medicine applications was indicated by the data and a rising demand for autologous cord blood over the last 10 years was also suggested.

The report documented nine samples released for regenerative therapies in addition to the four cases of aplastic anemia (reviewed in detail in the first study).

— Stem cell infusions were conducted at Duke University and Children’s Memorial Hospital in Chicago. Six samples were released to treat neurological conditions, including traumatic brain injury (one sample), anoxic brain injury (one sample), and cerebral palsy (four samples). Some anecdotal reports of improvement in quality of life, and anecdotal evidence by physicians involved with these cases suggests that the treatments were safe. This was because these six samples were not released as part of any specific clinical trial. Two more samples were released for treatment of cerebral palsy since the study period ended.

— As part of an ongoing clinical trial at the University of Florida, two client samples were released for type 1 diabetes. Possibly slowing the destruction of their insulin-producing cells and resetting the immune system, preliminary data from the first seven patients in the trial show the stem cell infusion appears to have reduced their disease severity.

— To treat a diagnosis of a rare immune disorder, one additional sample was released for an experimental autologous stem cell infusion.

“Cord blood stem cells are increasingly being used by transplant physicians in regenerative medicine because of their demonstrated ability to produce almost all of the cell types of the body,” said Harris. “These cases provide physicians and researchers with additional insight into how cord blood stem cells may be used to treat more conditions and ultimately benefit more patients.”

Regenerative medicine therapies could provide benefits to approximately one in three Americans according to current estimates. Currently, the U.S. alone is conducting more than 200 National Institutes of Health (NIH) funded clinical trials with cord blood.

Existing as the most recommended cord blood bank by obstetricians for eventual familial use in transplantation and regenerative medicine, Cord Blood Registry(R) (CBR(R)) is the largest cord blood stem cell processing and cryopreservation service. For more than 190,000 newborns throughout the world, Cord Blood Registry preserves cord blood stem cells. Amounting to a number eclipsing any other family cord blood bank, the AABB accredited CBR has released more than 55 client cord blood units for specific therapeutic use. Focusing on the collection, processing and storage technologies to optimize quality and cell yield, the company

To provide their eight-year-old daughter a revolutionary new treatment, a Bournemouth couple is trying to raise