Stem Cell Treatments for Cerebral Palsy – Mary Washington’s Story

Sarah Washington and her daughter Mary. Mary is 2 years old and was diagnosed with cerebral palsy when she was 4 months old. She was first treated at the Stem Cell Institute in Panama using human umbilical cord-derived stem cells. Mary received these stem cells intravenously over the course of 4 days. Her first treatment was in September of 2012. This interview was recorded in March 2013 at the clinic while she was there for a second round of treatments.

What kind of improvements have you noticed?

Overall, in general, what we noticed was, her upper body, she’s very stiff. She’s spastic in her legs and her arms and she has low tone in her trunk. In her upper body, we noticed a pretty significant improvement. Her range of motion, she couldn’t lift her arms over her before and now she can do that on both sides easily. And her hands, she was in tight little fists most of the time, whereas now if she is in a fist it’s very loose. But most of the time she kind of has her hands relaxed. That was a big improvement for us; small but significant for our family.

Her vision has improved, not necessarily distance but as far as like what I think as seeing details. So she would be able to tell the difference between you and me if we didn’t say anything. Whereas before, I don’t think she could do that. She would rely on smell and hearing more than her vision. Now she uses her vision a lot more. So that’s been an improvement that we’ve seen.
She is more involved in the world around her. [She] wants to be more involved in the world around her so that’s good. She has definitely, she doesn’t say, “mama, dada, ba, ba” yet but she definitely is making more vocalizations, making more sounds and trying to experiment with her voice, which is a positive thing.

She is trying different ways of communicating versus just screaming which is what she did before because that is all she knew how to do. And now, she is kind of fussing or sometimes she’ll just kind of like, yell and stop and wait for me to answer her instead of just going from “zero” to “scream”. So her communication has improved as well.

One thing that we were not expecting that we were pleased about is an improvement in her seizures. That wasn’t even something that we had even, I don’t know. I guess it just didn’t occur to us that maybe this would be helpful with that but before we came the first time in September she had been sick and we actually had to delay our trip twice because she had bronchialitis. She was just having a really hard time. And she was having a lot of seizures. We had to add a new seizure medicine to try to get them under control and she was having 6 or 7 short myotonic jerks every day. The first time we were here, by about Thursday, which was like day three of treatment, of stem cells we noticed that she hadn’t had a seizure that day. Whereas before, she was having 6 or 7 a day. And so, that was one thing. It was surprising but it was a nice surprise that those have improved since we’ve been here. She will still have breakthrough seizures every now and then when she’s sick, teething but she, we haven’t gone back to that, where she was having them on a regular basis. So that’s good. That’s been a good improvement.

Since we’ve been here this time, I haven’t really seen anything where I could say, “Oh, I think that’s the stem cells.” yet but I do know that last time it did take a couple of weeks for us to notice, you know, or to really pinpoint and say, “Yes, we think that is the stem cells. There’s no way she could have done that before.” So, and all of our therapists agree that her range of motion and her upper body for sure, and her vision would not have been the way they were without coming here.

Stem Cell Treatments for Cerebral Palsy: Hudsyn

IMG_6901If you haven’t been following our story on stem cells since the beginning, check out our previous blog posts first:

1. Exploring Stem Cell Therapy for Cerebral Palsy: This is how we came to our decision and provides lots of background research as to why we started down this path.
2. Panama City – Hudsyn’s Big Adventure: A recap of our trip and all that we experienced while there.
3. Hudsyn’s Stem Cell Therapy: 1-Month Update
4. Happy 2nd Birthday Miss H!: I wrote a letter to Hudsyn on her birthday…and near the end are a few other updates.
5. Hudsyn’s Stem Cell Therapy: 2-Month Update

So now that you’re fully caught up, I’m sure you’re anxious to hear about her most recent progress:

Improvements We’ve Seen

SeitzureTrendbyMonth - Dec 20121. Extreme Seizure Reduction: While we had nearly a week of zero seizures (huge leap from the 6-10 she was averaging before stem cells), she broke her record and has averaged 1-2 per day since that week in November. But we’re so much more hopeful that between her Keto Diet and the stem cells, we’ve got things under control…finally. It makes me teary to fully embrace this accomplishment because two years ago, we were watching our little girl have 40-50 a DAY, rendering us helpless as her brain became severely affected by them. Even medications had failed to help her. And now…to for a moment think that we might have a future without seizures is, well, just amazing…a miracle. Praise God!

2. Physical Therapy/Occupational Therapy: I attended one of Hudsyn’s therapy sessions at RIKC Friday, Dec. 7. She bore weight on all fours (with the help of arm braces of course) for over 8 minutes without complaint…a new record! We really enjoy our relationship with the team of therapists at RIKC. Erin (her OT) and April (her speech therapist) adore Miss H….and both are often caught between appointments coming to talk to or hold Hudsyn before it’s her time to work with each of them. We hope we will be able to continue her regular sessions here throughout this year. It truly is one of the best, most prominent therapy centers we’ve worked with thus far.

Here, you can see Erin holding her iPad, working from an app called Ladybird Baby Touch. The physical therapist is bracing Hudsyn’s head here so she doesn’t move her body to get the penguin on the screen and instead has to use her arms independently. This is a lot of work…but she DOES it:

3. Increase in Responsiveness & Smiling: This is the fun one. I can’t tell you how much joy it brings to see her interacting with more people now. We’d see this every so often (maybe once a day) before stem cells. Now…it’s on demand. If you talk, hold, sing, read, etc to Miss H…you’ll likely get a big smile or some type of physical response from her. There’s a big personality inside that little body…and we are completely in love with it.

4. Social Interaction: Hudsyn continues to enjoy preschool at CCVI.

IMG_6930She participated in her first school Christmas program last month. I must admit, I was pretty emotional watching the event. Not only was the setup and organization behind something like this incredible, but I realized deep down that I didn’t think I’d ever get to be in the audience watching my 2 year-old daughter shake jingle bells and be on stage with all the other kids in preschool. I had really come to a place where I’d accepted that she wasn’t going to be normal…that she wouldn’t be able to give us the joy of watching a holiday music program. But there she was, sitting on one of her therapist’s lap while thrusting her whole upper body forward, working hard to shake her bells as everyone sang the familiar songs.

Santa and Hudsyn 2012Yeah, I bawled like a baby. I’m sure the folks sitting next to me understood – either they wrote me off as a 9-month pregnant woman being emotional or they were teary right along with me. There were several wet faces in the audience that night. It was a wonderful holiday gift from our little girl. And, after the program, Hudsyn got to meet Santa for the first time!

Some Backsliding
Her frequent vomiting is back. But we really believe this is a result of the winter weather, thus contributing to some nasal mucous that is building up in the back of her throat and causing her to gag/vomit during key times of the day. These times are usually in the morning and then again in the evening. The vomiting isn’t the same as it was before, though. It’s not projectile, but instead a real puke…like you and I would do if our stomach was upset or we had something caught in our throat. She gives warning signals before doing it, so that’s been helpful in getting her to the bathroom sink on many occasions.

Another thought Dan had was these scheduled pukes are happening about 30-45 minutes after we give her meds. You can time it almost exactly every morning and evening. We discussed this with Dr. A (her neurologist) last week at her regular Keto Diet follow up appointment. Dan wondered if maybe the meds are causing this reaction whereas before her brain was more in need of them than after stem cells. So we were hoping to begin eliminating this drug, especially since the sharp decline in her seizures. Keppra in particular causes dizziness and nauseousness as side effects – even in small doses.

Dr. A agreed to begin slowly weaning her off Keppra and we’re happy to report that in one short month, she will be rid of it and all its side effects. We’ve also eliminated her Citicholine as it had some similar side effects and we weren’t seeing much benefit to this supplement any longer.

IMG_6958Treating the mucous is a bit harder. She can’t have regular expectorants or decongestants like you and I because of the Keto Diet. So, we’ve upped her water intake significantly which will hopefully help to thin it out. I’m glad we did it when we did because the next few days after her neuro appointment were awful. Miss H contracted the flu and a nasty cough coupled with a 102 degree fever. Tis the season! Thankfully, this time we were able to avoid being inpatient, mainly because of her G-tube and that we could force water, food and meds in her without putting them in her mouth. Without it, she would’ve been in the hospital. This time last year we were fretting about whether to do the G-tube surgery or not…and now…we don’t regret it one bit.

Miss H is still battling the cold/cough, but we’re happy to report her fever broke and the vomiting has decreased enough that she’s on full feeds again.

The last “backslide” that we’ve seen is one we’re actually happy about. Miss H has taken to some behavior much like that of a typical 2-year old in that bedtime is a huge fight. We used to be able to lay her down and many nights she’d go to sleep quietly. It’s predictable now that she’ll cry 30-90 minutes straight while attempting to go to sleep. She’s also learned during the day to manipulate anyone nearby (but especially her parents & grandparents) when/if she’s bored, she’ll fuss or fake coughing to get attention. The most recent incident of this was running an errand to Children’s Mercy, Dan captured this:

While most parents would complain or be annoyed by this behavior, we’re THRILLED. This is just more of her personality and desire coming out to communicate with us…and we couldn’t be happier as a result.

Overall Thoughts on Stem Cell Therapy for Cerebral Palsy
IMG_6905We’re now over halfway into our post-stem cell therapy journey with this being month 4 of 6. After six months from her first visit, Miss H is eligible to go back to Panama for another round of stem cell IV injections. Many of her fans, like you, have wondered if we’re planning to go again. We’ve been discussing this extensively with Dr. A, who has several other patients curious about the treatment. In addition to sending him journal articles and published academic studies from the Panama doctors we’ve showed him several of Hudsyn’s videos, her seizure tracking chart (above) and how she responds to our voices with smiling and interaction. He’s been amazed at her progress and obviously is very pleased. His only question is whether these improvements are a result of the stem cells or of normal development she would’ve had anyway. This exact question is the primary point of the clinical phase II trial Duke University is doing right now.

As her parents, we have to force ourselves to look at this objectively. Because in our heart of hearts, and after everything we’ve been through as a family…and everything Hudsyn has had to endure medically, we really just want this to work. With that perspective in mind, we’d love nothing more than to be able to say that umbilical cord blood stem cells are a “cure.” But we feel strongly that the science needs more research and development before we’re to that claim.

We will, however, say with the below evidence, Miss H would’ve never seen this kind of development in such a short period of time without stem cells.

  • Her field of vision went from 20% to 100% in less than 3 weeks.
  • Seizures became nearly non-existent after two months. As a result, anti-seizure medications (and all their “fun” side effects) are being discontinued.
  • Vocalization, eye contact and general responsiveness improved significantly within 1 month.
  • PT, OT, Speech and vision therapists documented large enough gains during her 3-month therapy visits that they claim are “remarkable” for a child with Hudsyn’s brain injury. With no other changes to her treatment, we must look at stem cells as helping with these gains.

To say that stem cells are a complete cure is false. But to say they have improved her quality of life is certainly true. We believe the combination of intense PT, OT, speech and vision therapy (with stem cells being at the primary level of the treatment plan) have not only helped Miss H repair some of her original brain damage, we know they’re allowing her to make exponential leaps in shorter periods of time.

As a parent who knows this treatment works…and knows its available for those who seek it out, the question remains…

Will we go to Panama again? We can faithfully and loudly declare: Hell yes.

Dylan’s hope (Stem Cell Therapy for Cerebral Palsy)

The possibility of using stem cells to treat cerebral palsy
has been suggested by several scientists based on the ability of these cells to:
a) stimulate regeneration of damaged nervous system tissue; b) to prevent
ongoing death of neurons; and c) to directly turn into, or "differentiate" into
neurons. This is explained in the video

One type of stem cell therapy that is currently under
investigation for cerebral palsy involves administration of cells from the
umbilical cord blood. This treatment has been the subject of much interest
because of the possibility of using cord blood from other patients. Routinely
performed outside of the United States, Dr. Joanne Kurtzburg from Duke
University has been the first to perform this treatment under the regulations of
the FDA. This recent story provides a personal description of one of the
patients treated.

In May 2009 5-year old Dylan Cain could only speak about 30
words and could not interact with family and friends. Subsequent to receiving a
cord blood transplant Dylan had a "miraculous" recovery according to parents.

"They told us at Duke that it might be months before we saw
any sign of improvement," Mother Jinger Cain said. "Just six weeks after we
returned home, he started to answer questions. His right leg straightened out a
bit, and his vocabulary has expanded amazingly."

"The speech therapist found that Dylan had progressed
5-plus months in the 3 1/2-month period of time, which means he is progressing
faster than his peers," Jinger said. "What is even more impressive is that
before the stem cells and hyperbaric treatments, he was progressing at a rate of
one month for every four months that went by, or three to four months of
development in a year, so he was consistently falling behind his peers. Now he
is progressing five times faster than before, and that has blown away his
teacher and therapists at the school he attends, as well as his doctors."

Jinger said that Dylan’s teacher in Bend told her she has
never seen a child make such gains in her 20 years of teaching.

Successes such as this case have prompted other doctors to
performed clinical trials assessing in a standardized fashion whether stem cells
actually impact cerebral palsy. Dr. James E Carroll, (706) 721-3371, of The Medical College of Georgia has recently announced initiation of a 40 patient placebo controlled trial in patients with cerebral palsy between 2-12. For more information please see the link below.

2010-05-16T17:19:51+00:00May 16th, 2010|Adult Stem Cells, Cerebral Palsy, News, Stem Cell Research|

Cord Blood Stem Cell Therapy for Cerebral Palsy in Clinical Trial

Cerebral palsy is characterized by hypoxia/reperfusion
induced damaged to the brain in the perinatal period. It is manifested in four main types: a)
Spastic, which occurs in 70-80% of cases and is associated with damage to the
corticospinal tract or the motor cortex; b) Ataxic, occurs in 10%, is
associated with damage to the cerebrum, and causes deficiencies in walks, hearing
and speech; c) Athetoid/dyskinetic is caused by injury to the to the
extrapyramidal motor system and/or pyramidal tract and to the basal ganglia, it
occurs in approximately 20% of cases. Cerebral
palsy is a non-progressive disorder in which recovery does not occur and
treatments revolve around addressing symptomology. The possibility of stem cell therapy for
cerebral palsy was proposed by Cellmedicine several years ago and is discussed
in this video

One type of stem cell that has been used for cerebral palsy
comes from the cord blood. Usually cord
blood stem cells are used for treatment of hematological (blood) disorders such
as leukemias or genetic metabolic conditions. Cellmedicine proposed the use of cord blood for conditions such as
cerebral palsy
because of: a) its superior growth factor producing ability to other types of
adult stem cells; b) the possibility of using cord blood with minimal matching;
and c) the ability of cord blood stem cells to directly differentiate into
other types of cells relevant to cerebral palsy such as neurons and glial

In order to test validity of the possibility that cord blood
may be useful for such a condition, the developmental cycle that occurs with
drugs has to be applied. That is,
firstly animal data needs to support the possibility of efficacy, as well as
the safety of the intervention. Secondly, pilot human studies are needed to determine if it is feasible
to administer the cells in patients with the particular disease without
possibility of adverse effects. Thirdly,
formal clinical trials need to be initiated. These usually begin with Phase I trials that assess safety and maximally
tolerated dose, Phase II trials that assess efficacy in a non-blinded manner,
and Phase III trials that seek efficacy in a
double-blind placebo-controlled manner.

Groups like Cellmedicine have been involved in treatment of
patients with cord blood. Additionally,
Dr. Joanne Kurtzburg from Duke has been using the patient’s own cord blood in
treatment of patients with cerebral palsy Both
of these treatments were considered part of the "practice of medicine" and may
be comparable to "pilot investigations" in that safety data was generated and
the medical procedure for physically administering the cells was

Today a group at the Medical College of Georgia announced
initiation of Phase I/II Placebo-Controlled, Observer-Blinded, Crossover Study
to Evaluate the Safety and Effectiveness of a Single, Autologous, Cord Blood
Stem Cell Infusion for the Treatment of Cerebral Palsy in Children.

The trial involves 40 patients between ages 2-12 who are
seizure-free and have clinical evidence
of a non-progressive motor disability due to brain dysfunction. The subjects recruited
will not have the ability to sit independently by one year of age or the
ability to walk by 18 months of age.

Patients will be
divided into 2 groups, with the first group receiving red-cell depleted, mononuclear
cell enriched cord blood unit prepared for infusion (treatment) and the second
being administered saline combined with the inert stem cell administration
solution lacking stem cells. The
observer and patient will not know who is receiving cells from which

The main observation endpoints of the trial will be safety of autologous (patient’s own)
cord blood infusion in children with cerebral palsy by repeated follow-up over
one year with clinical and laboratory evaluations. The secondary endpoint will
be determination of whether a beneficial effect has occurred in the
recipients. This will be measured using
a patient questionnaire and standardized Gross Motor Function Measure
evaluation with effects anticipated to be seen within 3-4 months.

Conceptually this study is a very safe one because it is the
patient’s own cord blood stem cells that are being used. This however could also be a negative
issue. There is some evidence that when
stem cells from another individual (allogeneic) are used, it is the reaction
between the recipient and donor that gives rise to production of numerous
growth factors. Since this current
treatment is only using the patient’s own cells, it may be similar to simply
adding your own blood back into you. The
animal studies previously performed involved using human cord blood cells in
mice lacking part of the immune system. Additionally they used much higher concentration of cord blood cells per
kilogram of body weight. Regardless, it
is very important to state that this study lays the groundwork for translation
of numerous stem cell approaches that have previously been used for patient
treatment outside of the US, for US approval.

Parents of patients interested in trial participation should
contact James E Carroll, M.D. the Principle Investigator of the study at 706-721-3371

2010-04-08T18:38:23+00:00April 8th, 2010|Adult Stem Cells, Cerebral Palsy, News, Stem Cell Research|

Stem cells from umbilical cord used for cerebral palsy

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.

2009-12-01T00:00:00+00:00December 1st, 2009|Cerebral Palsy, News, Stem Cell Research, Stem Cells, Uncategorized|

Gilbert Stem Cell Research Programs are Making Healthcare History

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.

2009-10-08T00:00:00+00:00October 8th, 2009|Cerebral Palsy, News, Stem Cell Research, Stem Cells, Uncategorized|

Adult Stem Cells Treat Cerebral Palsy

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."

2009-09-24T00:00:00+00:00September 24th, 2009|Cerebral Palsy, News, Stem Cell Research, Stem Cells, Uncategorized|

Public Awareness of Cord Blood Opportunities Lagging

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.

2009-08-30T00:00:00+00:00August 30th, 2009|Cerebral Palsy, News, Stem Cell Research, Stem Cells, Uncategorized|

Adult Stem Cells from Fat Protect the Brain Against Injury

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.

2008-11-20T00:00:00+00:00November 20th, 2008|Cerebral Palsy, News, Stem Cell Research, Stem Cells, Uncategorized|

Systematic Approach to Stem Cell Treatment for Brain Damage Being Developed

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.

2007-11-22T00:00:00+00:00November 22nd, 2007|Cerebral Palsy, News, Stem Cell Research, Stem Cells, Uncategorized|