Instant Expert: Stem Cells

       12:09 04 September 2006

       NewScientist.com news service

       John Pickrell

Stem Cells - Learn more about the promise and the controversy in our cutting edge special report .

Fast-forward to the end of the 21^st Century: surgeons can create new organs to order, regrow crippled spines and hearts and reverse the damage of Parkinson's disease or diabetes with ease. Immune rejection and waiting lists for replacement organs are consigned to history.

Stem cells may have the potential to fulfil this promise and much more, but there are still many technical, ethical and political obstacles to overcome before real therapies are possible.

Doctors have been transplanting adult blood stem cells, in the form of bone marrow transplants, for many decades, but stem cells from human embryos were only isolated and cultured in 1998. Though research has progressed rapidly since then, we still have much to understand; not least what gives stem cells their unique properties, but also how exactly they are able to differentiate into the 300 or so different types of human cell.

Despite their medical promise, stem cells have been dogged by political and ethical controversy because some are derived from discarded human embryos, and because of fears and confusion about links with human reproductive cloning. The future of stem cell therapies was thrown deeper into doubt in late 2005, when a leader of the field - Woo Suk Hwang, South Korea's stem cell king" - was found to have forged key discoveries and flouted ethical protocols. So has the stem cell miracle been postponed?

Full of potential

Embryonic stem cells (ESCs) come from fertilised human embryos - pinhead-sized balls of cells called blastocysts - just a few days old. In the embryo, these cells go on to form all the tissues of the developing body. They have generated so much interest because they are virtually immortal in the laboratory and can also generate any tissue type from bones to brain cells - making them pluripotent.

So far, most existing ESC colonies come from human embryos leftover from infertility treatments. But women are now donating eggs specifically for stem cell research in some countries.

The cells could potentially be cultured in the lab as an unlimited source of repair tissue for many ailments. Humans might even one day regenerate missing parts, as newts do.

Besides regeneration, stem cells could also be studied to provide insights into how human bodies develop from fertilised eggs. Stem cells with genetic defects could further be used to understand how congenital diseases, such as cystic fibrosis, develop. Stem cells might also be used to test new drugs in the lab on a range of tissues, instead of on people or animals.

Adult stem cells

As well as the fetus, stem cells are also found in the placenta, amniotic fluid and umbilical cord, and they remain in many adult tissues. Cord blood is sometimes collected at birth today, and the stem cells stored.

Adult stem cells have been found in: bone marrow, blood, the cornea and retina, intestine, liver, muscles, nervous system and the brain, pancreas and skin. These "multipotent" stem cells are less flexible than ESCs and are typically only able to form cells of the tissue in which they reside. "Adult" distinguishes these cells from their embryonic equivalents, but they are present in children too.

For example, hematopoietic stem cells are blood-forming stem cells, which largely reside in bone marrow. They are responsible for replenishing all blood cell types on a continual basis. It is these stem cells that rebuild the damaged blood system of leukaemia sufferers after successful bone marrow transplants. Mesenchymal stem cells, also found in bone marrow, can go on to form cells including muscle, fat, skin and cartilage.

Though adult stem cells are less flexible than ESCs, and are not immortal in the laboratory, they sidestep the ethical quandary of destroying embryos. Furthermore, we may be able to stimulate the adult stem cells we already possess to travel to and repair damaged tissues within our bodies.

Currently stem cells of both types are being tested to treat many conditions, including: Alzheimer's disease, blood disorders, blood loss, baldness, blindness,cystic fibrosis, deafness, diabetes, heart disease, kidney failure, liver damage, lupus, motor neuron disease, multiple sclerosis, osteoporosis, Parkinson's disease, spinal cord injuries and stroke.

Stem cells have also been used to fight cancer, treat dogs and race horses, and provide tissue for human breast implants and replacement teeth.

Researchers still have much to learn about how to direct stem cells to form and repair different tissues and how they behave within a patient's body. Even identifying stem cells is difficult currently. Concern that stem cells could divide uncontrollably to form tumours called teratocarcinomas is also likely to delay major clinical trials for some years. Stem cells might also become cancerous in the lab.

The cloning connection

The most significant hurdle however, is immune rejection. As with any tissue transplant (from a donor other than an identical twin), the body will recognise ESCs as foreign and mount an attack which could destroy them. ESC recipients would have to take immune suppressant drugs for the rest of their lives.

Multiplying a patient's own adult stem cells in the lab and then reinjecting them is one way to avoid rejection. Duping the immune system is another possibility, perhaps using stem cells from the brain that somehow avoid detection.

Therapeutic cloning is a clever technique that circumvents the problem. We can make custom-made ESCs using a patient's own DNA and a donor egg. In the same way as reproductive cloning, the nucleus of a skin or muscle cell from the patient is added to an unfertilised egg that has had its own genetic material removed. This egg is then persuaded to divide as though it had been fertilised and, with luck, goes on to form the ball of cells called a blastocyst. At this point, the inner cell mass is removed and cultured in the lab to derive stem cells. These stem cells now contain the DNA of the recipient and would not be treated as foreign by the immune system.

But, in theory, the cloned embryo could be implanted into a womb where it might develop into a cloned human baby. This would be reproductive cloning, and is the same method used to produce Dolly the sheep.

Ethical minefield

Producing viable clones of many species is a lengthy and difficult problem, and has yet to be proven in humans – despite questionable claims of success.

In any case, reproductive cloning has been banned in many countries for ethical reasons and because of suspected health risks to the clone. It was banned in the UK in 2001. Despite strong opposition, it has yet to be banned in the US.

For many, the destruction of embryos for scientific purposes is unacceptable, so numerous countries – such as Germany and France - also support bans on therapeutic cloning and using embryos to derive stem cells. A total of 87 nations voted for a resolution totally banning both types of human cloning in March 2005, but it was abandoned due to failure to agree on therapeutic cloning.

For others, the medical benefits outweigh these concerns. For example, in the UK, Belgium, Sweden, Japan, China and South Korea, therapeutic cloning has been allowed, but regulated. In the UK, licenses have been granted for studies into diabetes and motor neuron disease. ESC lines have been created in the UK since 2003. The EU provides some funding for ESC research in those countries that have embraced it.

In the US, the situation has become complicated. Disagreement between the religious groups who want a total ban on cloning and an equally vociferous pro-therapeutic cloning lobby has stalled legislation. In the place of a ban, US president George W Bush introduced legislation that restricted federally funded research to 22 stem cells lines created before 2001. However, research now suggests that these lines may have been tainted with material from mouse feeder cells in the lab, rendering them useless for human therapy. New ESC colonies free of this contamination have now been created.

Some US states have taken the situation into their own hands. California agreed a plan in 2004 to provide $3 billion for stem cell research over 10 years. By contrast, the Bush administration has pledged just $25 million annually to stem cell research.

In response to these restrictions, the race is on to find an ethical stem cell source - one that does not involve destroying embryos. One method does exist, but it creates ESCs with abnormal chromosomes. Other methods extract stem cells without destroying embryos, or create embryos that could never become babies. Further possible sources are: baby teeth cells, "universal" adult stem cells umbilical cord blood and testicle cells.

Fall from grace

Politics is not the only controversy that has gripped the stem cell world.

In May 2005, one of the world's top stem cell scientists - South Korea's Woo Suk Hwang – announced that his team had used therapeutic cloning to produce 11 ESC lines tailored to individual patients. This was one of a string of remarkable achievements. In 2004 Hwang cloned human embryos for the first time, and he later produced the world's first cloned dog - an Afghan hound named Snuppy.

Then in late 2005, the research community was rocked by claims that Hwang had flouted ethical guidelines by obtaining eggs from women in his own research group. As investigations proceeded and other transgressions unfolded, it became clear that much of his research had been fabricated. There are now questions over his use of funds too.

The fall from grace has been spectacular for a man who was revered as a national hero in South Korea, and the repercussions have travelled far and wide. Collaborating researchers have been tarnished by association, other stem cell science is under doubt, investors are wary of stem cell medicine, and there are now questions about how easy it is to fabricate results.

The already controversial field of stem cell research was brought further into disrepute, and it remains to be seen how much the scandal will delay the development of the miracle therapies that are so desperately desired.

 

Adult brain cells made to multiply and regenerate

       18:08 18 August 2006

       NewScientist.com news service

       Helen Thomson

Tools

Adult human brain cells can generate new tissue when implanted into in the brains of mice, new research reveals. The findings could pave the way to new therapies for a host of neurodegenerative diseases, including Alzheimer’s, the researchers say.

Furthermore, lab tests show that the mature brain cells have the versatility to divide many times in culture and develop into a wide range of specialised cell types.

Researchers at the University of Florida, US, showed for the first time that common human brain cells are adaptable and self-renewing – qualities normally associated with stem cells.

Dennis Steindler and his colleagues transplanted adult human brain cells into mice and found that they could successfully generate new neurons and incorporate themselves in a variety of brain regions.

The researchers also coaxed a single adult brain cell to divide into millions of new cells in culture. “We can, theoretically, take a single brain cell out of a human being and generate enough brain cells to replace every cell of the donor’s brain,” says Steindler.

Brain donor

The new source of human brain cells could be used to repair or replace damaged tissue in degenerative disorders such as Alzheimer’s and Parkinson’s disease, the researchers suggest.

“Anything that removes the need to use foetal or embryonic tissue [to clone new tissue] is very interesting because that’s where the controversy lies,” says David Dexter, a specialist in Parkinson’s disease at Imperial College, London, UK, who was not involved in the study.

“Now we can use adult human brain cells for research. They can be donated, like you would a heart or lung.”

Growth promotion

The brain cells were acquired from adult patients undergoing surgery for epilepsy and were extracted from grey matter, an area not known for harbouring stem cells.

When the cells were bathed in a solution containing a growth-promoting agent, the researchers noticed the emergence of neural progenitor cells. Progenitor cells are similar to stem cells but are further along in their development.

Steindler speculates that progenitor cells may pre-exist in grey matter and they simply multiplied after being bathed in growth promoter, or being transplanted into the mice.

Another possibility is that the "ageing clock" of the mature cells is actually reversed when the donor cells arrive in their new environment, returning them to their past lives as stem cells, he suggests.

Directed development

“One day we might be able to coax our own cell populations to provide us with regenerative aid for disease,” Steindler hopes.

However, although scientists can control what the brain cells develop into in culture, they may not exhibit the same control inside the brain. “Although the transplanted cells survive, we don’t yet know how to tell some cells to grow over here and others to grow over there. Until we can direct the brain to wire them in the right places, they’re going to be useless,” Dexter warns.

But, the ability to produce huge numbers of brain cells outside of the brain may serve as a useful instrument to test the safety of new drugs, Steindler says.

Journal reference: Development (vol 133, p 3671)

 

Enlisting the immune system to fix broken spines

       13:38 22 August 2006

       NewScientist.com news service

       Michael Day

 

Tools

A vaccination that stimulates immune cell production could be key to enabling people with serious spinal injuries to walk again, researchers say.

However, the study has been criticised by some experts in the neurological field who remain sceptical about the findings.

The controversial research claims come from a team at the Weizmann Institute of Science in Rehovot, Israel, who say that key immune cells can work with stem cells to mend broken spines in mice.

Their latest study involved a vaccine that increased the numbers of immune cells, known as T-helper cells, that specifically protect myelin – a protein that coats nerve cells. The vaccine encouraged and protected stem-cells in the spine as they grew and become nerve cells, to such an extent that previously crippled animals were able to resume walking, they say.

However, the new claims have reignited a major controversy in neuroscience.

Significant involvement

Traditional theory suggests that the delicate central nervous system needs to be isolated from the heavy-handed cells of the immune system in order to function properly and affect repairs.

Michal Schwartz, who led the latest study, has spent the last 10 years working on a different theory: that a significant degree of immune system involvement is needed for the central nervous system to repair itself.

In February 2006, Schwartz published a study in Nature Neuroscience demonstrating that immune cells had an important role in nerve cell regeneration.

Non-invasive treatment

Now she reports that by boosting T-cells at the same time as injecting mice with stem cells that had partially differentiated into nerve cells, she was able to reverse severe spinal damage.

Injections of the stem cells without the T-cell-stimulating vaccine had little effect. Significantly, the myelin vaccine alone had more effect than simply injecting stem cells, she says.

The findings suggest that “immuno-supressive drugs should not be used” with future stem-cell therapy for spinal injuries, she says.

The team simply injected the animals’ soft tissue, so invasive, intra-spinal injections would be unnecessary, they believe.

"No scientific basis"

Schwartz’s belief that key neurological diseases, such as multiple sclerosis, are caused by an over-active immune system has been greeted with some scepticism, however.

Geoffrey Raisman, director of University College London’s Spinal Repair Unit, was unequivocal in his denouncement. “There is no scientific basis for this paper,” he told New Scientist.

“The experiments reported do not have validity. It is beyond the bounds of possibility that this approach could improve spinal cord injury. I am surprised that it was published,” he adds.

Diverse functions

Another leading researcher in this field, Phillip Popovich at Ohio State University, US, has been less critical of Schwartz’s theories, describing them as “encouraging”. He too, however, has called on more substantial animal research to be done before tests on humans are even considered.

Schwartz cites papers she has published in recent years in reputable journals such as Nature Medicine and the Journal of Clinical Investigation as support for her theories. “I’m aware that this research is controversial. I think that neurologists are not aware of the diverse functions of the immune system,” she says.

“I think they’re locked into the concept that the immune system can be only detrimental to the central nervous system. But I think there’s clearly evidence now to say that’s not the case.”

Journal reference: Proceedings of the National Academy of Sciences (DOI: 10.1073/pnas.0603747103)

 

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Surgeon General Warning: Secondhand Smoke Puts Children At Risk

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Billionaire spends millions mapping mouse brains

 

WASHINGTON (Reuters) -- So you're a billionaire and you've bought a couple of sports teams, launched an amateur space project and spent $800 million on good causes -- what do you do with the change?

Microsoft Corp. co-founder Paul Allen decided to make a genetic atlas of the mouse brain.

The atlas, begun in 2002 with $100 million from Allen's fortune, was declared finished on Tuesday, with fine-tuned information on 3,000 active genes -- although scientists have been using it regularly for more than a year.

Allen said working with computers all his life made him appreciate the complexities of the brain. "You realize that computers take a very simplistic approach to computing things," Allen told Reuters in an interview.

"Ever since I grew up in Seattle as a kid, I was fascinated by science," he added. So he found a group of scientists and asked them what he should do with some of his money.

The result -- the first project of the Allen Institute for Brain Science -- a 3-D reference atlas of the genes that are active in the mouse brain.

Allen, who left Microsoft in 1983 and has an estimated fortune of $16 billion, makes the map freely available online at http://www.alleninstitute.org.

"Since mice and humans share more than 90 percent of genes, the Allen Brain Atlas has enormous potential for understanding human neurological diseases and disorders affecting more than 50 million Americans each year," the institute said in a statement.

These include Alzheimer's disease, which affects 4.5 million Americans, autism, which may occur in one in every 175 births, epilepsy, which affects 2.7 million Americans, schizophrenia and Parkinson's disease.

In four years, scientists working for the Atlas project have mapped more than 21,000 genes. They then checked each gene to see which ones are turned on -- expressed -- in brain tissue.

Each cell in an organism's body carries all the genes, but not all of them are expressed, or active. Gene expression is what determines each cell's type and function.

To their surprise, Allen's team found that more than 80 percent of the genes in the brain are active. They had believed that perhaps 60 or 70 percent were expressed.

The atlas was produced using in situ hybridization, a technique that uses a chemical marker such as a jellyfish fluorescence gene to show whether a gene is active.

Tissue containing cells expressing each active gene was stained, photographed and the pictures uploaded to the Web site.

That makes it easy to browse.

"It's a bit like peeling the onion," said Allan Jones, the institute's chief scientific officer.

The institute said an average of 250 scientists looked at the site a day, with more than 4 million hits monthly.

While examining the mouse brain is critical for basic scientific research, Allen also wants to look at the unique parts of the human brain.

"The next set of research we are going to do is focus on the neocortex -- the area where most higher function occurs," Allen said.

Allen, who owns the Seattle Seahawks football team and the Portland Trail Blazers basketball team and funds a charitable foundation and the SpaceShipOne space project, is asking for other foundations and the U.S. government to help support the institute project.

Copyright 2006 Reuters. All rights reserved.This material may not be published, broadcast, rewritten, or redistributed.

 

Neuronal differentiation of human umbilical cord blood neural stem-like cell line.

Buzanska L, Jurga M, Domanska-Janik K.

NeuroRepair Department, Medical Research Institute, Polish Academy of Sciences, Warsaw, Poland. buzanska@...

The expanding population of neural stem/progenitor cells can be selected from human cord blood nonhematopoietic (CD34-negative) mononuclear fraction. Due to repeated expansion and selection of these cells we have established the first clonogenic, nonimmortalized human umbilical cord blood neural stem-like cell (HUCB-NSC) line. This line can be maintained at different stages of neural progenitor development by the presence of trophic factors, mitogens and neuromorphogens in culture media. Neurogenic potential of HUCB-NSC was established for serum-free and low-serum cultured cells. Commitment of HUCB-NSC by serum was shown to be important for the optimal response to the signals provided by surrounding environment in vitro. Enhanced neuronal differentiation induced by dBcAMP treatment was accompanied by expression of several functional proteins including glutamatergic, GABAergic, dopamine, serotonin and acetylcholine receptors, which was shown by microarray, immunocytochemistry and electrophysiology. Electrophysiological studies, whole-cell patch-clamp recordings, revealed in differentiated HUCB-NSC two types of voltage-sensitive and several ligand-gated currents typical for neuronal cells. The above HUCB-NSC characteristic conceivably implicates that cord blood-derived progenitors could be effectively differentiated into functional neuron-like cells in vitro. Copyright (c) 2006 S. Karger AG, Basel.

 

Some may recall my soap box tirades over the past year about this clinic and others obtaining and using stem cells not meant for use in humans, well finally the Rotterdam one has been closed down!!

 

Stem cell clinic shut down

The Birmingham Post - Oct. 07, 2006
A clinic which put patients at risk of contracting HIV and CJD through its use of stem cells classified as unfit for human use has been closed down by Dutch health bosses.

The Rotterdam-based Pre-ventief Medisch Centrum (PMC), which charged one Midlands MS sufferer pounds 14,000 for treatment, has been ordered to stop trading immediately by the Netherlands Health Care Inspectorate.

The company had been using stem cells provided by UK-registered company Advanced Cell Therapeutics (ACT) to treat patients with Multiple Sclerosis and other debilitating illnesses.

During an investigation into PMC, the Inspectorate concluded it had not been providing responsible care, nor could it demonstrate the origin, suitability and safety of its stem cells.

In addition, it found patients had been exposed to the risk of infection with HIV, Creutzfeldt-Jacob Disease (CJD), acute allergic reactions, rejection reactions or malignant tumours.

The clinic's closure comes a month after The Birmingham Post revealed how a Bromsgrove MS sufferer had handed over more than pounds 14,000 for ACT's "pioneering" stem cell treatment.

Moments after undergoing the procedure abroad, 52-year-old former chartered accountant Malcolm Pear was able to walk unaided, despite having to previously rely on elbow crutches.

But less than three months later, Mr Pear's health had deteriorated.

The only contact he received from the organisation was in the form of e-mails encouraging him to pay thousands of pounds more for top-up treatment.

Mr Pear said: "Everything seems a bit more depressing now in terms of finding a cure for neurological problems.

"It was a case of people finding an opportunity to make money dishonestly and that is bad."

In a letter addressed to PMC doctor Robert Trosser, Inspector of Healthcare Dr D.C. van der Plas-Huisken said the Dutch health authority had carried out an investi-gation following media interest.

He said: "The notification and media coverage were reason for the Inspectorate to conduct a further and intensified investigation into the quality of care and the safety of the stem cell treatments you perform. A programme broadcast on 4 September showed that the cells, "were not intended for use in humans" according to the accompanying certificates.

"You stated during our telephone conversation that you had voluntarily stopped using cells from ACT and were now obtaining stem cells from Pakistan.

"At no time whatsoever did you show any concern about the fate of the patients you had already treated with cells from ACT or AllCells.

"For stem cell treatment you are required to demonstrate that the preparation you administer to your patients is suitable and safe for the intended purpose.

"If this care is not provided responsibly, it poses an immediate threat to patients' lives.

"You use stem cells to treat patients even though you are unable to demonstrate their origin, suitability and safety. Therefore, the serious situation regarding your centre's performance of this therapy makes it necessary for the Inspectorate to take immediate measures.

"With immediate effect you are ordered to discontinue use of stem cell treatment at your centre." The clinic will now remain closed for a week. The Inspectorate will then ask the Dutch Minister of Health, Welfare and Sport to renew the closure order.

It is also further considering whether to bring disciplinary charges against the doctors.

 

Mitogen-activated protein kinase signaling, oxygen sensors and hypoxic induction of neurogenesis.

Zhou L, Miller CA.

Department of Pathology, Keck School of Medicine, University of Southern California, Los Angeles, CA 90033, USA.

In the adult nervous system, neuronal subpopulations sustain a hierarchical pattern of selective vulnerability to hypoxia. Hypoxia also activates quiescent neural progenitor cells (NPCs) resulting in their amplification and subsequent differentiation into neurons and glia. Use of rat organotypic hippocampal cultures facilitates examination of early signaling events in response to hypoxia and reoxygenation that result in neurogenesis. Cultures were exposed to hypoxia for up to 6 h followed by reoxygenation. CA1 neurons showed focal nuclear condensation by 2 h of hypoxia, but CA2 and CA3 neurons were spared. JNKs and c-Jun reached peak activation by 4 h, returning to basal levels by 6 h. Expression of oxygen sensors, hemoxygenase 2 and HIF1, were elevated by 30 min and 2 h, respectively. By 24 h of reoxygenation, there was proliferation of nestin-positive NPCs. With U0126, an upstream inhibitor of ERK activation, BrdU labeling was markedly reduced immunohistochemically as well as PCNA protein expression, suggesting a role for ERKs in the proliferation response. Immunohistochemically, antinestin detected NPCs and on Western blots reached peak levels by 24-48 h of reoxygenation. Proliferation and differentiation of endogenous NPCs in the area of neuronal loss further suggests that mechanisms potentially exist in vitro for replacement with functional neurons. Copyright (c) 2006 S. Karger AG, Basel.

ALS

 

Researchers Discover Misfolded Protein Clumps Common to Dementia, Lou Gehrig’s Disease
Study Provides New Insights into Neurological Disorders

http://www.nih.gov/news/pr/oct2006/nia-05.htm

 

 

Autism

 

The U.S. Centers for Disease Control and Prevention has announced a $5.9 million study in six states to try to find the causes of autism.

http://news.yahoo.com/s/nm/20061007/sc_nm/autism_dc_1

 

A schizophrenia drug from Johnson & Johnson won U.S. approval as the first medicine to treat symptoms of autism in children and adolescents, the

http://news.yahoo.com/s/nm/20061006/sc_nm/jnj_risperdal_dc_5

 

Umbilical cord stem cells general

 

Neuronal differentiation of human umbilical cord blood neural stem-like cell line.

http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?tmpl=NoSidebarfile&db=PubMed&cmd=Retrieve&list_uids=16909032&dopt=Abstract

 

 

Washington - Scientists have used stem cells and a soup of nerve-friendly chemicals to not just bridge a damaged spinal cord but actually regrow the circuitry needed to move a muscle, helping partially paralysed rats walk.

Years of additional research is needed before such an experiment could be attempted in people.

But the work marks a tantalising new step in stem cell research that promises to one day help repair damage from nerve-destroying illnesses such as Lou Gehrig's disease, or from spinal cord injuries.

"This is an important first step, but it really is a first step, a proof of principle that ... you can rewire part of the nervous system," said Dr Douglas Kerr, a neurologist at Johns Hopkins University.

Perhaps most importantly, the experiment illustrates that if stem cells eventually live up to their promise, treatment won't be simple - they can't just be injected into a diseased body and repair it on their own.

Instead, the new research details a complex recipe of growth factors and other chemicals that entice the delicate cells to form correctly and make the right connections.

Miss a single ingredient, and the cells kind of wander aimlessly, unable to reach the muscle and make it move.

The study may bring "the appropriate tempering of expectations of stem cells", said Kerr, considered a leader in the field.

"Some of my patients say, 'Oh, I'm going to pull into the stem-cell station and get my infusion of stem cells,' and it's never going to be that."

Stem cells are building blocks that turn into different types of tissue. Embryonic stem cells in particular have made headlines, as scientists attempt to harness them to regenerate damaged organs or other body parts.

They're essentially a blank slate, able to turn into any tissue given the right biochemical instructions. But human embryonic stem cell research is politically controversial, because culling the cells destroys embryos.

The Hopkins experiment isn't the first to use stem cells to help paralysed rodents move. But previous work bridged damage inside the spinal cord that blocked nerve cells from delivering their "move" messages to muscles, sort of like fixing the circuit that brings electricity to a fan.

The new work essentially installs new wiring: replacing motor neurons - specialised nerve cells for movement - that have died to make a new circuit that grows neuronal connections out of the spinal cord and down to a leg muscle.

"They did something that people have been trying to do for at least 30 years and literally hit a brick wall until now," said Dr Naomi Keitman of the National Institutes of Health's neurology division.

 

This is a nice read as it is from a 17 year old boy with CP who is doing stem cell treatments.  It makes me wonder about what the younger children and babies would say if they could talk to us about how they feel.

 

http://www.stemcellschina.com/blogs/Gabor%20cp/

 

My Miracle Son: A Gift From God By dorothysweeney

This story is not about me. It is about my 23 year old son, Matthew. Little did I know all those years ago when I named him, that it meant "Gift from God" -- and that he is.

I lost Matthew's twin in my third month and he was born a preemie with cerebral palsy. My world fell apart when he was diagnosed at the age of 10 months. We were told we could simply put him in a home. I don't think so -- he was the survivor -- and so he stayed with us.

We enrolled him in an early intervention school by the age of 13 months and within three weeks, the absolutely wonderful teachers there had him sipping from a cup and sitting up. By the age of 3, he was walking -- a miracle.

Matthew is always smiling and always friendly. He is truly the light of my life. When he was 11, he took a seizure and was diagnosed with epilepsy but through it all, he always smiled. He was on meds for four years and is now free of epilepsy. But at the age of 16, he developed manic depression and we thought we would never get his "mind" back again. God surely does smile on my son -- he was put on very strong meds and he did come out of it.

My son was a commencement speaker at the Overbrook School for the Blind here in Philly (he is not blind, but visually impaired). To watch my son on that stage proves to me that God continues to smile down on him.

He works now at a WaWa store. No matter how bad I might look, he always tells me I'm beautiful. He is loving and caring and would be a friend to all, if he could. All those who know him, think he's the best and my husband has been calling him the "Rock" ever since he came out of that manic depression.

I have been living a shattering life with my oldest son but all I have to do is look at Matt and just the sight of him pulls me out of my own depression.

I also have a teenage daughter who is the light of my life but Matt (he's not disabled and he's not handicapped) is Heaven's Special Child.

 

We cannot change anything unless we accept it. Condemnation does not liberate, it oppresses.
-Carl Jung

My Disabilities Do Not Define My Life
By Robin J. Titterington

I was born with spina bifida (think Baby Noor!) When I was 19, I became deafened from an antibiotic given to save my life from severe kidney infections. When I was 33, my kidneys failed and I began dialysis. I had a transplant and have been on dialysis for nine years. I am not eligible for another transplant due to my worsening scoliosis. Yet I hesitate to submit my story.  Why? Because my disabilities do not define my life. Our culture is such that if you do not look like people in magazines and in movies, it is assumed you have a sad life. I am a Christian, a woman, a sister, an aunt, mom to my pets "the fabulous furries," a devout Braves fan, and a tree-hugger. I can think of many more adjectives before I would arrive at “person with a disability.”

My life is not so different from other single women: I graduated from college (with honors), I hold a Master’s degree from New York University, I am a certified public manager, and I have 20 years of full-time work experience in rehabilitation administration. I own my house (which I share with the fabulous furries), I drive my car. I have traveled to most of the 50 states, Mexico, Canada and Austria. In fact, during both trips to Austria I received dialysis treatments.

Why do I feel I have a happy, satisfied and peaceful life? No doubt most of it is due to my faith. I know I am never alone. That is not to say I never have a bad day, but knowing I am not alone and that I am loved by my Lord gets me through the rough times. I am a sister in the Daughter of the King lay order and my sisters are wonderful spiritual role models. I feel challenged by learning from them to continue to grow in faith.

Of course, none of this would be possible without my parents. Sadly, they both passed away when I was relatively young, my mom died when I was 14, my dad when I was 24. I am now 51 and grew up in a time when expectations were not high for someone with a severe disability. Yet there was never a question that I might not go to college, just like my older brothers did. I am sure they must have had to fight a lot to get treatments and education for me but their expectations of me were high. (And my family laughs a lot too!) We are now on the second generation of “Give me a ride, Aunt Robin!” I hate to brag but I have been told I am more fun than Six Flags!

Lastly, there are my friends. Some are close by in location, some are not, but all are close by in thought and prayer. I have a group on my email listing of “earth angels” and one email to that group and I know I am lifted in prayer by many.

 

 

Rat success could lead to human treatment
(China Daily)
Updated: 2006-03-30 06:50

Scientists eased the paralysis of rats with spinal cord injury by transplanting cells taken from the brains of adult mice, an encouraging sign for developing a human treatment, researchers report.

Someday, such cells might be taken from the brains of patients with spinal cord injuries for their own treatment, said researcher Dr Michael Fehlings.

In addition, similar cells are found in the spinal cord, so perhaps researchers may find a way to activate them to improve a person's mobility, he said.

Fehlings, of the University of Toronto and the Toronto Western Research Institute in Canada, and his colleagues, report the rodent experiment in yesterday's issue of the Journal of Neuroscience.

The experiment used 97 rats. Spinal injuries were created in the lab, and the mouse brain cells were implanted two weeks or eight weeks later. While the animals didn't start walking normally, those treated at the two-week mark did gain in coordination and ability to bear weight on their hind limbs.

Those treated eight weeks after the injury weren't helped, which Fehlings and other experts said illustrated a hurdle in treating spinal cord patients long after their injury.

Previous studies have also reported improvement in paralyzed lab animals with transplanted cells. But experts said the new work was notable because the cells were taken from adult animals rather than foetuses or embryos, and they produced an effect even when implanted two weeks after the injury.

"It's an important step forward," said Dr John McDonald, director of the International Centre for Spinal Cord Injury at the Kennedy Krieger Institute in Baltimore.

The transplanted cells, called neural precursor cells, are not as versatile as embryonic stem cells because they can give rise only to cells of the nervous system, Fehlings said.

Once implanted, they formed cells that can create a sheath around nerve fibres that resembles insulation around wires. Such sheaths are disrupted in spinal cord injury and restoring them produced the therapeutic effect in the rats, he said.

 

China regulates stem cell collection, transplant
(Xinhua)
Updated: 2006-07-24 20:45

China's Ministry of Health on Monday outlined new requirements concerning stem cell collection and transplants, banning profits from illegal stem cell transplantation.

Two sets of the regulations issued by the ministry said that the sources of peripheral blood stem cells (PBSC), or stem cells, must be legal, registered sources that can be traced.

The regulations aim to strengthen the management of stem cell collection and medical security, the ministry said.

The regulations also forbid disclosing stem cell donor data.

Stem cells, which are the body's master cells, circulate in the blood and in tissues. Stem cell transplants have proved effective in treating blood diseases like sickle-cell anemia, leukemia and other blood disorders.

The ministry will review the capabilities of stem cell collecting and transplant in medical institutions. The roster of the approved institutions to carry out stem cell collection and transplants will be sent to the Chinese Marrow Donor Program, which serves as a databank.

Under the regulations, medical institutions must obtain letters of consent from donors before collecting stem cells and must test for diseases transmitted by blood transmission, including AIDS, hepatitis, syphilis and malignant tumors. Stem cells of donors with positive test results must not be used for stem cell transplants.

 

The Chinese alternative to the stem cell research debate
By Sam Crane(China Daily)
Updated: 2006-07-28 09:24

Believe it or not, ancient Chinese philosophy can add to our understanding of the ethics of embryonic stem cell research. Hear me out.

In the US, debate on stem cell research centres on the question of whether destroying an embryo is tantamount to killing an individual person. Opponents believe that, since an embryo has the potential to become a person, it should be treated as a person and not be subjected to scientific experiments that might cause its destruction. Supporters argue that embryos are not yet fully formed persons and thus can be used to harvest stem cells for scientific study. In addition, proponents of stem cell research would add that the social benefits of science outweigh the destruction of individual embryos.

The US controversy pits devout Christians, whose religious beliefs lead them to see embryos as persons, against utilitarian liberals, whose definition of an "individual" does not include fetuses before the third trimester of pregnancy. The issue has become politicised of late, with the US Congress ready to pass legislation supporting embryonic stem cell research and the President threatening a veto.

Unsurprisingly, the US debate has called upon various strands of Western philosophy and religion. But Daoism and Confucianism can add something to the conversation.

A modern-day philosophical Taoist would likely be sceptical of the entire scientific research enterprise. The Dao De Jing has this to say in passage 29: "Longing to take hold of all beneath heaven and improve it; I have seen such dreams invariably fail. All beneath heaven is a sacred vessel, something beyond improvement. Try to improve it and you ruin it. Try to hold it and you lose it."

Religious Daoists (dao jiao) are famous for their search for a death-defying elixir of life. Philosophical Daoists (dao jia), however, are more accepting of the inevitable demise of the human body. Zhuang Zi is marvellously free of anxiety and resentment about death. Indeed, the notion that purposive human activity can overcome the natural aging process is contrary to the general Daoist attitude to do nothing (wu wei) that might get in the way of Way.

If confronted with the question of stem cell research, therefore, a philosophical Daoist might say: "why bother?" It may help a few people with certain maladies, but it will not fundamentally transform the human condition. Such Daoists would generally dissent, not because embryos might be persons, but because science cannot define destiny.

Contemporary Confucians, on the other hand, would probably find themselves aligned with supporters of stem cell research, but for somewhat different reasons.

For a Confucian, persons are defined socially. Our identities are shaped through our daily cultivation of our closest social relationships. The question of whether an embryo is a person is, therefore, nonsensical: how could it be a person if it was not yet actively engaged in social relations. A person becomes a person at birth.

Furthermore, if stem cell research helped to cure disease, allowing people to better perform their social roles and duties, then the science would be advancing the cause of Humanity (ren), the highest Confucian virtue. Confucians would emphasize, even more than Western liberals, these sorts of social benefits. It is less about individual rights and accomplishments for a Confucian, and more about the mutual realization of individual and social morality.

We learn more when we consider the widest possible range of ideas in any debate. Adding Confucian and Daoist perspectives to the American discussion of embryonic stem cell research gives us insights into the issues, and into ourselves, that we might otherwise overlook.

Contact the author at scrane@...

Sam Crane teaches Chinese philosophy and politics at Williams College in Massachusetts, USA, and is the author of Aidan's Way.

(China Daily

 

 

 

 

The Cord Blood Cure

Rich in stem cells, umbilical cord blood has become a viable alternative to bone marrow transplantation in adults.

By Kristina Fiore, posted September 27th, 2006.

It was hot that summer in 1996, Gayle Serls recalled, so she had blamed her constant fatigue on a sweltering Southern July. It wasn’t until August that the pain and symptoms—the nausea, the fainting, the vomiting—became unbearable. At that point, the athletic Durham, N.C., native who exercised frequently and ate healthfully became a patient at Duke University Medical Center. She was diagnosed with acute lymphocytic leukemia, a fast-growing cancer of the white blood cells.

Serls suffered through weeks of chemotherapy, and was able to beat the disease into remission within a month. But that wasn’t the end of her treatment. Follow-up tests revealed Serls’s leukemia was a rare kind that would not stay in remission. That’s when her doctor started talking about a bone marrow transplant.

Since Duke didn’t have an adult bone marrow transplant program at the time, Serls was sent to Johns Hopkins University instead. There, doctors told the 45-year-old that she was too old for a regular bone marrow transplant. They could perform an autologous bone marrow transplant instead, which would “clean up” her own cells and put them back into her body.

But before she could begin her treatment at Hopkins, Serls relapsed, literally on the medical center’s doorstep. She couldn’t go through with the bone marrow treatment, and was immediately flown back to Duke for chemo again.

Just days before her daughter’s severe relapse, Serls’s mother watched a television news report on a new leukemia treatment. It was called an umbilical cord blood transplant, and it was very experimental, with a low survival rate. Serls didn’t care—she was desperate.

Though it wasn’t common during Serls’s treatment 10 years ago, adult cord blood transplantation has now gained popularity as an alternative to bone marrow transplantation in diseases such as leukemia and lymphoma. In 2005, there were more than 1,400 cord blood transplantations in adults, according to NETCORD, an international network that coordinates umbilical cord blood banks. This year, journals have published reviews of the state-of-the-art of the procedure. Their consensus: cord blood transplantation in adults is a viable alternative to bone marrow transplant, is often easier to obtain and enables more efficient donor-recipient coordination.

This wasn’t the case in 1996, when Serls was dying from acute lymphocytic leukemia. The procedure was largely experimental, and though she didn’t have much to lose, she also didn’t have much of a choice.

Finding hope in an experimental procedure

Cord blood is the blood leftover in a baby’s umbilical cord immediately after birth. It is usually thrown away. Lately, its collection has gained popularity because of the value of the stem cells found in the blood. These are cells in a state of “identity crisis,” which have not morphed into specific cell types (such as liver or heart tissue), giving them excellent potential to adapt to another person’s body. In patients with leukemia, a cancer that prevents the body from creating healthy blood cells, transplanted stem cells can become the blood-making cells the body lacks.

The first recipient of cord blood transplantation was a six-year-old boy suffering from Fanconi anemia. His 1988 procedure was a success, and cord blood transplantation has since become a popular treatment for childhood leukemias.

Over the years, researchers have also been experimenting with the use of cord blood transplantation in adults. Claudio Brunstein, a researcher at the University of Minnesota Cancer Center’s cord blood transplantation program, focuses on using cord blood to treat adult leukemias. He was an author of the June 2006 cord blood transplantation review published in the journal Bone Marrow Transplantation, a sister publication of the journal Nature.

In the article, he notes some of the limitations of the treatment—such as survival rates and the amount of cord blood needed to treat an adult patient—but also notes its many benefits, which have increased over the years.

At the time Serls underwent treatment, many kinks in the procedure hadn’t yet been worked out. As soon as her mother told her about cord blood transplantation, Serls tracked down Mary Laughlin, then an assistant professor of medicine at Duke University, who was conducting a clinical trial on cord blood transplants in adults. Laughlin was a pioneer; the treatment had been common only in children at the time.

 

Serls became the third of Laughlin’s adult cord blood transplant patients.

“She came to see me in my [hospital] room, and I wanted to know, ‘could I be a candidate?’” Serls recalled. “But she was talking to me like, ‘OK, we are going to do this.’”

Laughlin’s expertise is in the field of innovative leukemia therapies. She began testing cord blood as a stem cell source because many of her patients could not find a matching marrow donor in time to treat their disease. She was tired of facing patients with life-threatening leukemias and being forced to advise them to just go home.

“Testing cord blood stem cell transplant in adults … allowed me an opportunity to offer a potentially life-saving therapy for my adult leukemia patients,” said Laughlin, who is now a professor of medicine and pathology at Case Western Reserve University’s School of Medicine.

“I had hope again,” Serls said about meeting Laughlin and trying the experimental therapy. “She explained that it was probably only a 10 to 20 percent chance of survival. But when you’re told there’s NO hope, then 10 to 20 percent might as well have been 100 percent as far as I was concerned.”

The first step was to find Serls a matching donor.

Easier matchmaking

The biggest advantage cord blood transplantation has over bone marrow transplantation is that it is much easier to match patient and donor, Brunstein said. While bone marrow requires an exact match, cord blood does not. Optimally, the cord blood donor and recipient should match six out of six blood antigens, in the same way that a blood transfusion must be matched by type. However, the transplant can be successful with as few as four matching antigens.

Because these requirements are less restrictive, Brunstein said, it is often easier to find a match. At the University of Minnesota, matches have often been made within one day—a significant advantage over bone marrow transplantation, which can take three to four months, according to Brunstein’s review.

“Such rapid availability can be particularly useful for patients with high-risk malignancy or rapidly progressive non-malignant diseases,” the review states.

This is especially good news for minority patients because there aren’t many minority donors on the national bone marrow donation list.

It was also good news for Serls. She found a four out of six antigen match—the lowest number of matching antigens acceptable for the transplantation. Her only worry, then, was graft-versus-host-disease, a condition in which the transplant recipient’s T-cells attack new tissue they perceive as foreign.

Brunstein said, however, that GVHD is less common than expected considering that most cord blood transplants are not fully matched.

“We believe it is because the immune cells in [cord blood transplantation] are naive and less prone to attack the recipient,” he said.

Serls analyzed the risks: she had an average donor match and a chance of graft-versus-host-disease. Those risks looked better than her other alternative: death.

Policymakers focus on cord blood

Kristine Gebbie, director of the Center for Health Policy at Columbia University, had heard about cord blood transplantation but didn’t know much about it when she was selected to head the Congressional committee investigating cord blood banking in 2004.

The committee’s goal, Gebbie said, was not to establish if cord blood was a good treatment as well as a stem cell source—that was a given. Instead, the committee worked for eight months, hiring economic professionals, researchers and other advisors, to come up with a system of accreditation and standards for cord blood banks.

By April 2005, Gebbie’s committee had settled on recommendations for establishing a banking process and submitted them to Congress. The report concluded that a national center to coordinate the network of collection agencies needed to be set up.

“Public banking [of cord blood] needs to be more strongly coordinated through central process linking donors and stored blood with transplantation sites and transplants,” she said. “Also, standards need to be enforced through accreditation.”

Following the committee’s report, the federal government realized the growing demand for cord blood transplants. In December 2005, President Bush signed into law the Stem Cell Therapeutic and Research Act, which set aside $10 million to get the cord blood center up and running.

The legislation also mandated the need for the collection of 150,000 units of donated cord blood to stock the new center. Currently, there are 14 public cord blood centers, which communicate via an international network foundation called NETCORD—not a government-sponsored registry.

Two main competitors—New York Blood Center and National Marrow Donor Program—are in the running for control of the national registry. The federal government is still in the request-for-proposal phase for the center’s creation, but the Health Resources and Services Administration under the U.S. Department of Health and Human Services has set goals for collecting the requested number of units.

According to David Bowman, spokesperson for HRSA, the agency will spend $9 million in 2006 on 6,900 units of cord blood, followed by another $9 million worth in 2007. The agency, however, did not request funds for 2007, and is using excess money from fiscal years 2004 to 2006 to purchase the units.

A ‘tiny bag’ of ‘important cells’

If it was up to Gayle Serls, cord blood would receive all the funding necessary to make it a popular and affordable treatment. Ten years ago, her treatment cost $250,000, but costs much more now, she said.

Even though her insurance paid for almost all of the procedure, Serls said the price didn’t matter. She was going to have the treatment, no matter what.

Two weeks after she began seeing Laughlin, Serls had her donor in place and was ready for the procedure. She was nervous, but she knew a cord blood transplant would be her best hope.

It was May 1997, and during that day at the hospital, Serls remembers being hooked up a pint-sized bag—“a tiny bag full of very important cells,” she said—that would hopefully save her life.

The procedure was like any blood transfusion, she said. She was conscious the entire time, watching the liquid enter her veins, and hoping her body would accept it. Though she can’t remember exactly how long it took, she knows the transplant was quick. But as she was wheeled back to her hospital room, waiting for her body’s reaction seemed like an eternity.

All she could do was wait—wait for new cells to grow. Wait to see if she would develop GVHD. Wait to see if she would live.

Serls recalls being tired all the time during the following weeks as she lay in her hospital room. She couldn’t eat, since her stomach couldn’t hold food down.

A few weeks after the transplant, Serls did develop GVHD.

Luckily, she says, it was so minor that doctors were able to treat it.

After that, things started looking up. By mid-July, about nine weeks after her treatment, Serls was able to go home. Eating was still a task, and fatigue was a plague. But slowly, she healed.

It is now 10 years since her treatment, and Serls couldn’t be happier with the choice she made. Though she hasn’t felt as healthy as she did when she was young and active before the disease, she says she’s doing well. She gets tired more easily, but that’s her only complaint.

Serls believes so strongly in the power of adult cord blood transplantation that she now works as a cord blood unit coordinator in Duke University Medical Center’s Pediatric Blood and Marrow Transplant program. Despite its title, Serls said, adult patients were frequently seen at the facility. Duke now has an Adult Bone Marrow and Stem Cell Transplant Program.

Sometimes Serls will comfort patients going in for the transplant, sharing her own story of survival.

“I’m one of the longest-surviving adults who have had the transplant in the world,” she tells patients enthusiastically. “The longer I live from that moment, the more it means to me to have had that opportunity. It was just so serendipitous.”

 

 

Stem cell trial to combat childhood brain disease

       12:13 25 September 2006

       NewScientist.com news service

The first clinical safety trial of a purified human fetal stem cell product is about to begin in the US for a rare and fatal childhood brain disease. The trial could pave the way for neural stem cell transplants to treat a range of brain and spinal cord disorders.

A team from the Oregon Health and Science University Doernbecher Children’s Hospital plan to treat six children suffering from the inherited neurodegenerative condition, Batten’s disease – also known as neuronal ceroid lipofuscinosis (NCL). There is currently no alternative treatment for the disease.

The team expect to treat the first child before the end of 2006. The children will receive injections of neural stem cells that have been purified – isolated from other cell types – and grown from donated human fetal tissue. The stem cell product and isolation technique was developed by StemCells Inc, of Palo Alto, California, which is sponsoring the trial.

Children with Batten’s disease suffer seizures, motor control disturbances, blindness and communication problems. As many as 600 children in the US are currently diagnosed with the condition – death can occur in children as young as 8 years old.

The children lack an enzyme for breaking down complex fat and protein compounds in the brain, explains Robert Steiner, vice chair of paediatric research at the hospital. The material accumulates and interferes with tissue function, ultimately causing brain cells to die.

Neuron support

Previous tests on animals demonstrated that stem cells injected into the brain secreted the missing enzyme. And the stem cells were found to survive well in the rodent brain.

Once injected, the purified neural cells may develop into neurons or other nervous system tissue, including oligodendrocytes, or glial cells, which support the neurons, say the researchers. Steiner is hopeful that the treatment will work for the 25 or so other hereditary brain diseases related to Batten’s disease.

In addition to secreting enzymes, Steiner says these cells can become the type of nerve cells found in spinal cord, and so they could potentially help after spinal cord injury. The stem cells can form into neural cells found in the brain or nerve cells found elsewhere in the central nervous system, he explains.

However, Stephen Minger, director of the stem cell biology laboratory at Kings’ College London, believes that despite Steiner’s claims about the versatility of the new purified cells, their use is limited to Batten’s disease. “The cells in question have little clinical relevance to other neurological disorders,” he says.

"Groundbreaking process"

The researchers hope that the treatment will offer some clinical benefit to the children suffering from Batten's disease, but they stress that the primary purpose of the trial is to assess the safety of the product. It is the first such safety trial to be approved by the FDA.

“This is a very important first step – a groundbreaking process to bring this technology to patients,” says Nathan Selden, head of the Division of Paediatric Neurological Surgery at the hospital, who will perform the transplants.

Human fetal stem cell transplants have been performed before on adult patients with neurodegenerative conditions including Parkinson’s and Huntington’s diseases, and also for spinal cord injury. But these used mixtures of various, unpurified fetal stem cells. Results have been mixed.

The researchers plan to follow the children’s progress over the course of a year.

 

Woman plans China trip for treatment

By Justin Boulmay
Rocky Mount Telegram

Saturday, September 23, 2006

SPRING HOPE – Dawn Smith knows what she wants to do when she's able to walk again: go upstairs for the first time.

Smith, 28, of Spring Hope has never been on the second story of her house.

"We've been here almost three years," she said. "I don't know what it looks like up there."

Smith, who suffered from a spinal aneurism almost eight years ago, is looking for ways to raise money for an upcoming trip to China. She found a research group called the Beike Biotechnology Co. Ltd. in the Chinese town of Shenzhen. The group uses stem cells from umbilical cords to try and improvea people's health conditions.

When she was 20 years old, Smith suffered from an arterial venous malformation of the spine, or a spinal aneurism. She said it happened because one of the veins at the base of her neck weakened and broke. At that time, she was paralyzed from the neck down.

Over the last several years, Smith has regained some ability and movement in her limbs, but she still walks with the assistance of a walker.

"I don't want to walk with the walker for the rest of my life," she said. "A cane I can take, but a walker, I don't necessarily want."

If all goes well, she might get her wish.

Smith discovered Beike after doing research on various medical techniques. She also talked to people who had been through the program, and has read the Internet blog of one of the patients.

"You're sort of skeptical at first to find out what's going on, what all's involved, and then you get a little more interested," she said. "You just try and hold your composure until you find out."

Smith said she hopes she can leave by the end of October. Her mother will go with her.

"I just told her I was going along for the ride," Smith's mother, Phyllis Joyner, said. "She's making all her decisions, and I'm pretty much there for support."

When she arrives, Smith will stay at the facility for a month. During her stay, doctors will give Smith four to five injections of stem cells in the injured area. Her time will also include physical and massage therapy.

While Smith is excited about the trip, she also said the injections are not a cure.

But at least it's something, she said.

"It's a hopeful start," she said. "It's more than you had."

While excited and nervous, Smith also has another obstacle to overcome. The trip, with the injections, therapy and residence included, comes out to $20,000.

Smith and her family are trying to hold an auction and charity event to raise money for the trip. Also, Smith said her best friend sold Krispy Kreme coupons to help out.

People have offered to donate items, and even live animals, for the auction.

"Somebody said we could have a horse," she said.

Some members of Smith's family were concerned about the trip, but they also said they hope she finds what she's looking for.

"When she gets her mind set on something, that's what she's going to do," said Bill Joyner, Smith's stepfather.

When Smith returns to the United States, her work will be far from over. She'll have to keep up her therapy and work on building muscles.

"I think as long as you come home and push your therapy and keep working at it and trying to strengthen and build muscles, that you're only getting better," she said.

Smith said anyone interested in more information can send her an e-mail at dawn78787@....

 

Cord Stem Cells: Results, Not Hype

 

The stem cells likely to yield the quickest, least expensive, and largest clinical benefit are readily available and present no ethical dilemma. They are umbilical cord blood stem cells.

The controversy surrounding President Bush's decision to fund research involving existing lines of embryonic stem cells has dominated discussions of stem cell research. Yet in the background, umbilical cord blood cells have been steadily delivering verifiable clinical results. The only limit on their availability is the technologic expertise required to separate and store the cells from the umbilicus.

Use of cord stem cells is rapidly expanding in bone marrow transplantation, where the immunologically naive cord blood cells carry a much lower risk of graft-vs.-host disease (GVHD) than occurs with traditional transplants .of adult, marrow-derived stem cells. Investigators also speculate that cord blood stem cells could be used to revitalize a damaged immune system, making them nearly as versatile as fetal stem cells for treating such immune disorders as type 1 diabetes and rheumatoid arthritis.

In the earliest stages of type 1 diabetes, for example, cord blood cell transplants could virtually replace the patient's immune system, preventing complete destruction of the pancreatic islet cells. This approach becomes feasible if the risk to the patient of developing GVHD is low, as it appears to be with cord blood cells.

In hematologic cancers, umbilical cord blood has all the advantages, observed Dr. John E. Wagner Jr., scientific director of clinical research for the blood and marrow transplant program at Fairview-University Medical Center, Minneapolis. Comparisons of matched marrow transplants and unmatched cord blood transplants suggest that the effectiveness is equivalent, but the rate of GVHD is far lower with cord blood.

Until recently, cord blood transplants were reserved for use in children with malignancies, explained Dr. Wagner, director of clinical research at the Stem Cell Institute at the University of Minnesota, Minneapolis. The reasons are largely historical--most of the researchers were pediatricians--but there also was the question of whether a cord blood specimen would contain enough stem cells to reconstitute an adult system.

That concern was put to rest by a recently published study of 68 adults, 54 with malignancies, who received grafts of umbilical cord blood from unrelated donors. Myeloid hematopoiesis was reconstituted in 90%. The mean time to a successful neutrophil count was 27 days, slightly longer than the 20 days seen with marrow-derived grafts from human-leukocyte-antigen (HLA)-matched, adult donors. Survival rate was low; 19 patients were alive at 22 months. A high percentage of patients had advanced disease, but the delay in immune reconstitution could have been a factor. At 40 months after transplant, 18 of the 19 patients are disease-free.

Even though 66 patients received cord-blood that was not a perfect HLA match, 20% of recipients in the cord blood group had severe acute GVHD. The rate typically seen with HLA-matched bone marrow from unrelated donors is 30%-50%.

The experience with cord blood in adults has been so successful in Dr. Wagner's program that it is now routinely done. They automatically begin searching cord-blood banks for a donor specimen for patients who cannot wait as well as those for whom the alternative is a somewhat mismatched graft.

Dr. Wagner's group is trying to circumvent the delay to reconstitution by performing two separation infusions on subsequent days from two different cord blood donors. They also are experimenting with adult mesenchymal cells, which provide the environment for stem cells and hematopoietic maturation, in patients whose own mesenchymal cells have been destroyed by ablative therapy The first three patients treated in this fashion "had the fastest recovery I have ever seen," Dr. Wagner said.

In children with hematopoietic disorders, long-term results of cord blood transplants are available for those with Fanconi's anemia, including some who are 8 years post transplant.

Cord blood is just beginning to be used in sickle cell disease. A nonmyeloablative approach with immune suppression is used. It is this nonmyeloablative approach that could be exploited to treat autoimmune disorders like diabetes. The transplanted cells may prove to exist side by side with the patients' original stem cells, but there also is reason to believe that--with immunosuppression--the transplanted cells can gradually replace the genetically aberrant cells.

So far, five patients with sickle cell anemia have been treated at Dr. Wagner's center using cord blood from a sibling. Follow-up time has been short, but engraftment has occurred, he said

COPYRIGHT 2001 International Medical News Group
COPYRIGHT 2001 Gale Group

 

 

 

 

 

Ill girl home after China therapy
Sacha received four stem cell injections in China A five-year-old girl from Sussex has returned home after undergoing weeks of stem cell treatment in China in an attempt to halt a degenerative disease. Sacha Skinner, from Brighton, suffers from Batten's Disease - a rare disorder inherited through her genes - which affects her speech and movement. Annette Dacosta, Sacha's mother, said she was hopeful that the treatment may have brought some benefit. She said Sacha was now eating better and using her mouth muscles more. Sacha had four injections taken from umbilical cords after her family raised £20,000 for the trip, as the treatment is illegal in the UK. It is claimed in other similar cases stem cells have slowed the progress of Batten's disease. She was treated at a clinic in Shenyang in northern China.

 

Stem cells: Chemistry paves way toward promising therapies

SAN FRANCISCO, Sept. 14 -- Chemists are developing new insights and techniques in an effort to expand the therapeutic potential of stem cells, which includes possible treatments for Parkinson's disease, diabetes, spinal cord injury and other devastating conditions. The American Chemical Society will explore some of these latest developments, including new findings on the transformation potential of adult stem cells, during a special symposium, "Emerging Technologies: Stem Cells," on Thursday, Sept. 14, in San Francisco during the Society's 232nd national meeting. All papers in this symposium, which begins at 1:30 p.m., will be presented at the Hilton San Francisco, Yosemite B.

Shown below are selected papers from this symposium:

Adult stem cells show wider potential than previously thought -- Embryonic stem cells are the most versatile stem cells, capable of being transformed into any other cell type, depending on their desired therapeutic use. Now, researchers at Northwestern University have found new evidence that hematopoietic stem cells, a type of adult stem cell derived from the bone marrow that gives rise to blood cells, is capable of undergoing more diverse transformations than previously thought and could be transformed into a wide variety of tissue types, not just blood cells. In recent laboratory tests, human megakaryocytes (bone marrow cells that produce blood platelets that are responsible for blood clotting) derived from adult hematopoietic stem cells were, for the first time, reprogrammed into neutrophil-like cells similar to the white blood cells that are responsible for fighting infections, according to study leader E. Terry Papoutsakis, Ph.D., a chemical engineer at the University. Insights from this study could help guide similar adult stem cell transformations in other cell types in the future, he says. (BIOT 459, Thursday, Sept. 14, 1:30 p.m.)

Elasticity of tissue environment plays role in determining stem cell growth --Researchers at the University of Pennsylvania have shown that the elasticity of a stem cell's environment is a major determinant of what type of tissue the stem cell becomes. In laboratory tests, Dennis Discher, Ph.D., and Adam Engler, Ph.D., grew mesenchymal stem cells (derived from adult bone marrow) in polymer hydrogels with either soft, medium or rigid elasticity. Based on resulting cell shapes as well as messenger RNA and protein markers, stem cells grown in softer environments -- such as brain tissue -- tended to produce nerve-like cells; those grown in environments with medium elasticity -- similar to muscle -- produced muscle-like cells; and stem cells grown in more rigid environments -- like bone -- produced bone-like cells. The study provides new clues on how chemical and mechanical factors interact to influence stem cell growth, the researchers say. (BIOT 463, Thursday, Sept. 14, 3:10 p.m.)

'Stretched' stem cells have potential to be transformed into blood vessel cells -- Scientists have searched for years for a renewable cell source to craft blood vessels that can be used for heart bypass surgery and perform more like natural arteries. Now, researchers at the University of California, Berkeley, have shown that mesenchymal stem cells from adult bone marrow can be repeatedly and mechanically stretched -- in a manner similar to a taffy pull -- into patterns that could potentially transform them into smooth muscle cells similar to blood vessel tissue. These newly-formed smooth muscle cells, which can expand and contract, could be used as a component of a tissue-engineered graft that may provide superior performance over conventional grafts that are used for bypass surgery, says study leader Kyle Kurpinkski, a doctoral candidate in the University's Department of Bioengineering. (BIOT 464,

 

U. Arizona: U. Arizona scientists' tissue regeneration techniques bypass stem
cell debate

U-Wire - Sep. 06, 2006

U-WIRE-09/06/2006-U. Arizona: U. Arizona scientists' tissue regeneration
techniques bypass stem cell debate (C) 2006 Arizona Daily Wildcat Via U-WIRE

By Tarah Hackman, Arizona Daily Wildcat (U. Arizona)

TUCSON, Ariz. -- From the world's first human cornea transplant to the growth of
a pancreas, researchers at the University of Arizona are using stem cells to
make medical breakthroughs, despite the controversy surrounding stem cell
research.

Rather than fighting governmental restrictions on embryonic stem cell
experimentation, UA scientists avoided the taboo research altogether by working
primarily with stem cells found in umbilical cord blood, said David T. Harris, a
professor of microbiology and immunology.

Although researchers from Advanced Cell Technology, a biotechnology company in
California, said they recently discovered a way to extract stem cells from
embryos without harming the potential fetus, the discovery won't change the way
university labs conduct research.

"It's a great political debate, but it has become neither side providing
important information to the public," said Harris.

Within one year, Harris said UA labs expect to successfully perform the world's
first human cornea transplant from stem cells found in umbilical cord blood.

In addition, nerve and heart tissue regeneration in animals, specifically mice,
have proven highly successful in these labs, Harris said.

Harris said stem cells found in umbilical cord blood appear to be the equivalent
of stem cells found in embryos.

However, Jonathan Flax, an assistant professor of neurology, said the difference
between adult stem cells and embryonic is highly debatable.

Flax said as a general rule, embryonic stem cells have the capability to
differentiate into any cell, such as a germ cell, T-cell or those found in
organs, but adult stem cells have not been proven to differentiate in the same
manner.

"Maybe we don't see it because it's such a rare cell, but we haven't seen the
appropriate cues (for unlimited differentiation)," said Flax, who studies the
mechanisms that restrict potential stem cells' lineages using neural stem cells.

Still, researchers are making progress with umbilical cord stem cells.

The congestive heart failure lab at the University Medical Center, which uses
stem cells in umbilical cord blood, hopes to become the first to carry out a
successful regeneration of heart tissue in a human suffering from heart disease,
said Elise Furfaro, a graduate student in pathobiology who is a lab manager and
research specialist.

Michael Badowski, a graduate student in microbiology and immunology and research
specialist, said his lab is starting a project where cartilage tissue is grown
from stem cells using umbilical cord blood.

Badowski said tissue rejection is a major problem for organ transplants today,
and through this type of research, stem cells can be isolated and made into any
cell desired because of their vital role in tissue repair.

Experiments with mouse stem cells are being used to grow new pancreases, a
potential breakthrough for those suffering from diabetes.

"Why do I have to give you my kidney if you can grow your own?" Badowski said.

Harris is the lab director of Cord Blood Registry, the world's first and largest
cord blood bank, which is located in Tucson, Ariz., and started in UA labs.

Furfaro said cord blood is collected from mothers who sign a release, and the
blood is stored in the bank, ready to treat children if necessary.

Cord blood is especially valuable in regenerative medicine, spinal cord or brain
injury or if the family has a history of cancer, Harris said.

"Many people don't know that you have to use your own stem cells for it to
work," said Harris.

Extracting cord blood at birth can be vital for an individual who develops a
disorder like cerebral palsy, aplastic anemia or various forms of leukemia.

Whether or not President Bush accepts this new method of embryonic stem cell
research, UA labs intend to continue their research with umbilical cord blood.

"It's very exciting, but this change isn't going to really affect us," Furfaro
said. "We are going to keep working with our project using the alternative
(umbilical cord blood)."

 

Identification of stem cells from human umbilical cord blood
with embryonic and hematopoietic characteristics

Yong Zhao⁎, Honglan Wang, Theodore Mazzone
Section of Endocrinology, Diabetes and Metabolism, Department of
Medicine, University of Illinois at Chicago,
1819 W. Polk Street, Chicago, IL 60612, USA

26 April 2006

We identified stem cells from the umbilical cord blood, designated
cord blood–stem cells (CB–SC). CB–SC displayed important embryonic
stem (ES) cell characteristics including expression of ES-cell-
specific molecular markers including transcription factors OCT-4 and
Nanog, along with stage-specific embryonic antigen (SSEA)-3 and SSEA-
4. CB–SC also expressed hematopoietic cell antigens including CD9,
CD45 and CD117, but were negative for CD34. CB–SC displayed very low
immunogenicity as indicated by expression of a very low level of
major histocompatibility complex (MHC) antigens and failure to
stimulate the proliferation of allogeneic lymphocytes. CB–SC could
give rise to cells with endothelial-like and neuronal-like
haracteristics in vitro, as demonstrated by expression of
lineageassociated markers. Notably, CB–SC could be stimulated to
differentiate into functional insulin-producing cells in vivo and
eliminated hyperglycemia after transplantation into a streptozotocin-
induced diabetic mouse model. These findings may have significant
potential to advance stem-cell-based therapeutics.