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I saved my sons cord blood with viacord. I called the company and unfortunately they are not working to cure muscular dystrophy.
However if the cord blood can assist in anyway to researchers I would like to know where.
When you are visiting with Doctors across the country please ask them if cord blood is of any interest to them.
My thinking is that maybe they can use the stem cells to figure out how to correct the mutation on the cord blood. He has a deletion of exon 13 in frame. If anyone knows of any clinics using cord blood to research md, please let me know.
I have already been to Harvard Boston Childrens and they told me that the cord blood could be useful for research, but they don't have enough NIH funding.
Stem Cell Breakthrough: Mass-Production Of 'Embryonic' Stem Cells From A Human Hair
Concentrated stem cells directly injected into patient's heart in new clinical trial

Washington, Nov 21 : For the first time, surgeons at the Methodist Hospital in Houston have injected highly-concentrated stem cells directly into a patient's heart, providing an intense, direct hit on damaged heart tissue.

The promising new technique can turn out to be more effective in regenerating healthy heart tissue than current methods that use a catheter to put standard stem cells through the bloodstream into the heart.

The patient, who was a 58-year-old veteran and businessman, is resting comfortably and may be discharged this weekend.

"Some patients have such severe heart failure that their only current option is a heart transplant. We hope that stem cells will stimulate angiogenesis, the growth of new blood vessels, restore mechanical function in diseased heart tissue, and return patients to a much better quality of life without a transplant," said Dr. Brian Bruckner, cardiac surgeon at the Methodist DeBakey Heart & Vascular Center in Houston.

The new process requires the patient's strongest and most robust stem and progenitor cells, derived from the patient's own bone marrow, to be amplified up to 1,000 times before they're injected back into the patient's heart.

In the procedure, Dr. Bruckner made a small incision in the left side of the patient's chest and administered approximately 25 injections of concentrated stem cells into the patient's heart. All patients in the trial will be followed for 12 months after the injections.

A large number of people are suffering from chronic heart failure, and some of these individuals have dilated cardiomyopathy (DCM), a chronic heart disease in which the patient's heart can not pump effectively enough to deliver blood and oxygen to the vital organs in the body.

Patients with DCM typically experience severe limitations to physical activity and shortness of breath.

"Without a new approach to treatment of these patients, they will continue to decline and less than 40 percent will survive five years. We hope this trial will provide a completely new and viable treatment for them," said Bruckner, principal investigator for the trial.

The IMPACT-DCM trial is a randomized, controlled, prospective, open-label, Phase II study that will seek to enroll 20 patients with ischemic dilated cardiomyopathy (DCM) and 20 patients with non-ischemic DCM at five clinical sites in the U.S.

Participants must have a left ventricular ejection fraction of less than or equal to 30 percent (60-75 percent is typical for a healthy person) and meet certain other eligibility criteria.

All patients in each group will receive standard medical care and 75 percent of the patients will be treated with cardiac repair cells (CRC), a mixture of stem cells and progenitor cells derived from the patient's own blood marrow, through direct injection into the heart muscle during a minimally-invasive procedure in the operating room.

While the primary objective of this study is to assess the safety of CRCs in patients with DCM, efficacy measures including left ventricular ejection fraction and other cardiac function parameters as well as heart failure stage will be monitored.

Patients will be followed for 12 months post treatment.
Patient, heal thyself: Grow your own new organ

The idea of growing your own new organ is now a reality. The possibilities are endless, say Steve Connor and Jeremy Laurance

Saturday, 22 November 2008
British scientists claimed a world first last April after they helped a blind man to see with an injection into the back of the eye.

Surgeons crossed another medical frontier this week with the transplant of a windpipe grown from stem cells – the "mother" cells of the body capable of developing into specialised tissue. The success of the operation in Barcelona on Claudia Castillo, a 30-year-old mother-of-two, proved what scientists have long promised – that stem cells can now be used to fashion replacement body parts.

The success of the medical procedure has opened the door to other possible breakthroughs in regenerative medicine – which is when damaged body parts are repaired in situ rather than being removed and replaced by whole organ transplants. Medical scientists believe that few body parts will be left untouched by the technical, genetic and surgical breakthroughs that could revolutionise medicine in the 21st century.

The Eye

British scientists claimed a world first last April after they helped a blind man to see with an injection into the back of the eye. Steven Howarth, 18, from Bolton, who had a rare inherited eye disorder which left him unable to see in low light, improved sufficiently after the treatment to be able to navigate a maze in conditions similar to street lighting at night.

He was one of the first three patients with eye disorders to be treated with gene therapy – an injection of normal versions of the defective gene that caused his condition, Leber's congenital amaurosis. Scientists at Moorfield hospital, London, said it would lead to other eye treatments.

The Windpipe

Claudia Castillo made history as the first patient to receive a whole organ transplant grown using her own stem cells and without the need for powerful anti-rejection drugs.

Surgeons used a windpipe from a donor which they stripped of all living cells and re-seeded with cells grown in the laboratory from Ms Castillo's bone marrow. The transplant was carried out last June and blood tests have shown no sign of rejection.

The Bladder

American specialists announced a breakthrough in 2006 by growing parts of new bladders in the laboratory from patients' own stem cells and successfully implanting them. Seven patients given the new bladders had functioning organs that performed as well as those conventionally repaired.

The breakthrough demonstrated the potential of regenerative medicine – growing your own tissues and organs.

Scientists at the Institute for Regenerative Medicine in North Carolina, where the operations were carried out, are working on 20 tissues and organs, including blood vessels and hearts.

The procedure involved taking cells from the patient's bladder, which was grown on a biodegradable "scaffold"After eight weeks, it was used to patch the damaged organ. The repaired bladder was working well up to seven years later, when the breakthrough was announced.

The Skin

Chronic wounds are already treated by grafting pieces of the patient's own skin, which is usually taken from the thigh, onto the affected area. However, this can result in unsightly scarring at two places on the body.

Another technique being developed is to grow skin – dermal tissue – in the laboratory using adult stem cells extracted from the roots of hairs plucked from the back of the scalp. Scientists can grow numerous small patches of skin using this technique. These skin patches can then be transplanted on to the wounded area to regrow the skin over the damaged tissue without scarring or the risk of tissue rejection, which occurs when donor skin is used.

Scientists at the Fraunhofer Institute for Cell Therapy and Immunology in Leipzig, Germany, have been given a licence to grow skin in this way from patients with conditions that result in open wounds, such as leg ulcers.

The Immune System

The body's defensive system against invading diseases can be regenerated or replaced in a number of ways. One of the most successful is by a transplant using donated umbilical cord blood for children with severe combined immune deficiency (Scid), when they lack the white blood cells of the immune cells produced by the bone marrow.

The transplanted stem cells repopulate the patient's bone marrow. An alternative is to create embryonic stem cells from a patient's own skin cells and convert them into the stem cells of the immune system, in which case there would be no need for immunosuppressant drugs.

The Brain & Nerves

In one technique, stem cells are extracted from cloned embryos created from a patient's skin cells and used to create mature nerve cells that can be transplanted into damaged areas of the brain. Another strategy is to block production of the protein inhibitors within nerve cells that are thought to prevent regeneration.

The Face

The world's first face transplant carried out by French surgeons on Isabel Dinoire in 2005 made headlines around the world. It marked a breakthrough because of the intricate surgery required to reconnect the scores of nerves and blood vessels which supply the face. She had been savaged by her dog after taking a cocktail of drugs in an apparent suicide attempt.

Mme Dinoire, 38, was given a new mouth, nose and chin and the success of the operation was judged in part on how effectively her new face regenerated to restore her sense of identity. Three years on, she admitted she had still not come to terms with her new face which was "not hers, it's somebody else's".

Doctors at the Royal Free Hospital, London, have been given ethical permission to carry out a further face transplant and hope to refine the technique.

The Heart

French doctors unveiled the latest version of the mechanical heart last month, which is claimed to beat almost exactly like the real thing. Made of titanium and animal tissue, the device uses electronic sensors to regulate the heart rate and blood flow and is even said to fool cardiologists when they are shown its ECG trace.

Existing artificial hearts are designed as a stop-gap, to help people over transplant operations or while they are waiting for a new organ. In some cases they are used to "rest" the patient's own heart, to give it time to recover from infection or disease until it is ready to take over its function as the body's main pump circulating the blood.

British patients have been implanted with artificial hearts for up to two years, before having them removed and allowing their own hearts to pump again.

The Pancreas

Doctors have been seeking a treatment for diabetes for decades that avoids the need for daily injections of insulin. Since 2000, a dozen patients have received transplanted islet cells, the insulin-producing cells in the pancreas, from dead donors which have worked with varying degrees of success. The Department of Health agreed this year to fund further research, involving 80 patients annually.

Japanese doctors took the treatment a stage further by carrying out a live transplant, from a mother to her 27-year-old daughter. A section of the mother's pancreas was removed, the islet cells isolated and washed and infused into her daughter's liver, where they began functioning normally, producing insulin.

Scientists at Akron University in Ohio are experimenting with a "bio-artificial" pancreas – the size of a cigarette coated with a membrane that holds islet cells and promotes the exchange of insulin and glucose between the cells and the blood.

The Ovaries and Testes

The reproductive organs present unique problems. In men, sperm is produced profusely and continuously and can be easily frozen and stored.

Women, however, only produce one or two mature egg cells each month and their reproductive life ends at the menopause. On top of this, egg cells are difficult to freeze. For infertile men and women who cannot produce any sperm or eggs, there is the possibility of creating the sex cells – gametes – in the laboratory from skin cells. The idea is to use the skin cells to create cloned embryos by inserting the cell nucleus into donor egg cells that have had their own nucleus removed.

After extracting embryonic stem cells from the three-day-old cloned embryos, scientists hope to stimulate them with nutrients and messenger chemicals to develop into mature sperm or eggs which crucially have half the number of chromosomes as the skin cells from which they were derived. Several research groups have achieved this stage of successful "haploidisation" in animals and are hoping to do the same with human skin cells to produce viable sperm or eggs.


Duchenne muscular dystrophy is caused by a person's inability to produce a muscle protein called dystrophin. Scientists know how to make healthy versions of the dystrophin gene, and hope to find ways of inserting it into the affected muscles, and thus getting these damaged cells to behave normally. Gene therapy research is also looking at ways of boosting muscle size and strength.
They are doing stem cell transplants in other countries. Have any other parents looked into this option? I know it is expensive, but is is an option? PLs let me know.
Researchers at Children's Hospital of Pittsburgh of UPMC have been able to effectively repair damaged heart muscle in an animal model using a novel population of stem cells they discovered that is derived from human skeletal muscle tissue.

The research team — led by Johnny Huard, PhD — transplanted stem cells purified from human muscle-derived blood vessels into the hearts of mice that had heart damage similar to that which would occur in people who had suffered a heart attack.

These transplanted myoendothelial cells repaired the injured muscle, stimulated the growth of new blood vessels in the heart and reduced scar tissue from the injury, thereby dramatically improving the function of the injured left ventricle, said Dr. Huard, director of the Stem Cell Research Center at Children's Hospital's John G. Rangos Sr. Research Center.

"This study confirms our belief that this novel population of stem cells discovered in our laboratory holds tremendous promise for the future of regenerative medicine. Specifically, myoendothelial cells show potential as a therapy for people who have suffered a myocardial infarction," said Dr. Huard, also the Henry J. Mankin Professor and vice chair for research in the Department of Orthopaedic Surgery, University of Pittsburgh School of Medicine. "The important benefit of our approach is that as a therapy, it would be an autologous transplant. This means that for a patient who suffers a heart attack, we would take a muscle biopsy from his or her muscle, isolate and purify the myoendothelial cells, and re-inject them into the injured heart muscle, thereby avoiding any risk of rejection by introducing foreign cells."

Results of this study are published in the Dec. 2 issue of the Journal of the American College of Cardiology.

The myoendothelial cells used in this study were more effective at repairing the injured cardiac muscle and reducing scar tissue than previous approaches that have used muscle cells known as myoblasts, according to Dr. Huard. At six weeks after injection, the myoendothelial cell-injected hearts functioned at 40 to 50 percent more effectively compared with hearts that had been injected with myogenic cells (myoblasts).


Dr. Huard and colleagues in the Stem Cell Research Center are researching and developing numerous therapeutic uses for the population of muscle stem cells the team identified. One of the most promising uses could be for the treatment of Duchenne muscular dystrophy (DMD), a genetic disease that affects one in every 3,500 boys. Patients with DMD lack dystrophin, a protein that gives muscle cells structure.

Dr. Huard is an internationally recognized cell biologist conducting laboratory research into the therapeutic use of stem cells to treat a variety of musculoskeletal and orthopaedic diseases and injuries. In the lab, Dr. Huard is developing cutting-edge therapies to regenerate bone, cartilage and peripheral nerve and to repair damaged skeletal muscle after sports and military injuries.
Single adult stem cell can self renew, repair tissue damage in live mammal
Research will be presented at American Society for Cell Biology conference
The first demonstration that a single adult stem cell can self-renew in a mammal was reported at the American Society for Cell Biology (ASCB) 48th Annual Meeting, Dec. 13-17, 2008 in San Francisco.

The transplanted adult stem cell and its differentiated descendants restored lost function to mice with hind limb muscle tissue damage.

The adult stem cells used in the study, conducted at Stanford University, were isolated from a mixed population of satellite cells in the skeletal muscle of mice.

The skeletal adult muscle stem cells (MusSC), which live just under the membrane that surrounds muscle fibers, normally respond to tissue damage by giving rise to progenitor cells that become myoblasts, fusing into myofibers to repair the tissue damage.

The scientists transplanted the MusSC into special immune-suppressed "nude" mice whose muscle satellite cells had been wiped out in a hind limb by irradiation.

The mice would only be able to repair injury if the transplanted MuSC "took." The scientists, Alessandra Sacco and Helen Blau, had genetically engineered the transplanted MusSC to express Pax7 and luciferase proteins. As a result, every transplanted cell glowed under ultraviolet light and was easy to trace.

"To be able to detect the presence of the cells by bioluminescence was really a breakthrough," says Blau. "It taught us so much more. We could see how the cells were responding, and really monitor their dynamics."

Through luminescent imaging as well as quantitative and kinetic analyses, Sacco and Blau tracked each transplanted stem cell as it rapidly proliferated and engrafted its progeny into the irradiated muscle tissue.

The scientists then injured the regenerated tissue, setting off massive waves of muscle cell growth and repair, and subsequently showed that the MuSC and descendents rescued the second animal's lost muscle healing function.

After isolating the luciferase-glowing muscle stem cells from the transplanted animal, the scientists duplicated, or cloned, the cells in the lab. Like the original MuSC, the cloned copies were intact and capable of self-renewal.

"We are thrilled with the results," says Sacco. "It's been known that these satellite cells are crucial for the regeneration of muscle tissue, but this is the first demonstration of self-renewal of a single cell."

The ability to isolate and then transplant skeletal adult muscle stems cells could have a wide impact in treating not only a variety of muscle wasting diseases such as muscular dystrophy but also severe muscle injuries or loss of function from aging and disuse.

In other experiments, the researchers transplanted between 10 and 500 luciferase-tagged MuSC into the leg muscles of mice.

These cells also proliferated and engrafted, forming new myofibers and fusing with injured fibers.

Unlike tumor cells, the transplanted stem cells achieved homeostasis, growing to a stable, constant level and ceasing replication.

After demonstrating that the transplanted stem cells proliferated and fully restored the animal's lost function, Sacco and Blau recovered new stem cells from the transplant with full stem cell potency, meeting the final "gold standard" test for adult multipotent stem cells.


For more information: Contact: John Fleischman, ASCB science writer,, (513) 929-4635, (513) 706-0212 or Cathy Yarbrough, freelance ASCB annual meeting media manager, or, (858) 243-1814.


Alessandra Sacco
Stanford University
(650) 736-0081

The lead author will present, "Self-renewal and expansion of single transplanted muscle stem cells," Sunday, Dec. 14,1:30 pm, Stem Cells I, Program #628, Board #B590, Halls A-C, Moscone Center.

Authors: A. Sacco, R. Doyonnas, P. Kraft, H.M. Blau, Microbiology and Immunology, Stanford University, Stanford, CA.
Thanks for posting this up.
Its very exciting news :)
This sounds like the holy grail. The press release does not say whether there's an accompanying peer-reviewed journal article. Any word on when that'll be published?

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