Little Lily Hasselman has already been through a great deal in her first five years. She was diagnosed with Cystic Fibrosis at just six weeks of age and as a result has to endure daily painful physiotherapy, take medication with every meal, and undergo constant medical checks.

Cystic Fibrosis is a life-shortening genetic condition, with no known cure. Many children with the disease used to die in early childhood, but even with treatment advances their life expectancy rarely exceeds 30 years.

However hope is on the horizon. Professor Bob Williamson of Melbourne University is behind a new Australian medical study which aims to prove that umbilical cord blood can successfully treat cystic fibrosis.

The project will use sibling cord blood to regenerate the damaged lung cells of the affected child. “Most CF siblings cord blood has a 1 in 4 chance of being a perfect match that will not be rejected by the immune system of the child living with CF.”

This is where Lily’s little brother and sister come in. Two year old Henri and six month old Zara are both CF-free and had their umbilical cord blood banked at birth with CordBank.

Professor Williamson says Lily’s siblings’ cord blood won’t be used in the study, but will be kept for when a breakthrough comes. The study will prove the safety of the treatment with donated cells from unrelated people, and lab tests are showing positive signs.

Lily’s mum, Sarah-Jayne’s advice to any parent considering storing their baby’s cord blood is, ”Just do it. You never know what might be round the corner and although as parents you hope your children will never need to use their cord blood – it’s a great insurance

Dr Wise Young answered your questions on the future of umbilical cord stem cell therapy to cure spinal cord injury when he visited New Zealand as a guest of CordBank in February 2011.

Q. ” In your opinion, how far do you think we are, realistically, from finding a cure for SCI?”

A: I believe that it is a matter of time and not a matter of if we will have a “cure” for spinal cord injury, of course, how fast the cure comes will depend on the investment by society into research. Clinical trials are going on right now

Q. “Is this cure for people who have just had an injury, or for those who have been in wheelchairs for some time?”

A: We are focussing on chronic spinal cord injury which we define as more that one year after injury and stable neurologically for at least 6 months. Our trials are for people from age 18 years through to 60 years old.

Q. “What are the side effects of Lithium in the lab rats?”

A: Lithium has been used to treat manic depression for over 50 years. In the United States, about 3% of people have the diagnosis of depression and half have taken lithium. So this is a very commonly used drug. Its side effects are relatively minor if the serum levels are carefully titrated.

Q. ” Would you inject this cord blood and lithium directly onto point of injury? or is it injected intravenously?”

A: The cells are transplanted into the spinal cord above and below the injury. The lithium is taken orally for 6 weeks.

Q. Is any of your intense rehab being done with “Project Walk”? (San Diego)

A: We are doing intensive rehabilitation for our clinical trials. I am aware of the work being done by Project Walk in San Diego.

Q. Dear Dr Young, Our little boy aged 22 months was born with Spina Bifida and we banked his cordblood at birth. In terms of spinal injuries I have always asked if someone could provide an accurate definition of injury ie. does the spinal cord have to suffer some form of trauma, or could spina bifida, although termed a birth defect also be classed as a spinal injury. I would be interested to know if any research has been done using cord blood to treat SB and if so what of any results have been collated.

A: We are not currently doing trails on spina bifida. However, I am aware of other promising work relating to nerve bridging to restore bladder function in children with spina bifida.

Q. Hi Dr Young, I have a cousin in Australia who suffered a spinal injury almost 3 years ago. He is now a quadriplegic. Would the cord blood that I have stored from my two children’s births be able to be used to help him? He is my cousin on my mothers side. He also has another cousin who is about to have twins and we were wondering if their stem cell blood would be helpful (the cousin is on his mothers side) Would these match’s be closer than using a ‘unknown’ donor?

If I did this would there still be enough stem cells remaining in the bank for our children if they needed it? We are very keen to help him out!

A: Cord blood from relatives is more likely to match but there is no guarantee, especially for distant relationships. Usually the chances of siblings matching 6/6 HLA is 1:4, so, the likelihood of a perfect match will be less. However, cord blood will often engraft even with 4/6 match. It is worthwhile, when the time comes, to do an HLA match of your children’s blood and your cousin. We will know in about 2 years time whether the cord blood plus lithium therapy is effective for spinal cord injury. I hope that is effective.

Q. Is possible for you to take stem cells from a part of the body several years after the baby born?

A: Unfortunately, no.

Q. My 11 year old son has mild spina bifida.  I did cord blood banking with his 3 year old sister.  Is this able to be used for him to assist in a bit more healing and to repair nerve damage and bring back some feeling in his leg that was affected from his lipoma surgery?

He is a normal boy, no problems with toileting, but does not have much feeling in the affected leg from nerve damage during his surgery at 9mths of age.  His leg has been burnt accidentally on the motorbike resulting in huge blisters and he didn’t even feel it.  The foot was always cold when he was younger, but is slightly better now.  He also has a withered calf muscle and the leg at present is about 2cm shorter than the other one.  The leg is always covered in bruises and sores.  He has problems with the knee and requires Botox to help the muscles work.  He drags the leg when tired and it slows him down quite a bit, especially with things like swimming.  The leg is turned out, but the average person would not detect this.

He wore a leg brace until the age of 5 and wore special made shoes. He has had 1 operation for a tendon lengthening, but may require more.Can still not stand on his toes as such. He is improving slowly as he matures.

I worry about the future and how his actual back will cope, as the surgeon did remove a large lipoma and bone.

Any info would be much appreciated thanks.

A: I am not sure that umbilical blood cells will do much for children with spina bifida. However, there is promising work suggesting that nerve transfer may be useful. Several groups in Sweden, Michigan (USA) and New York are running clinical trials the results of which suggest that the nerve transfers can restore bladder function in children with spina bifida. I don’t know about individual leg muscles.

Q.Can you let me know where the research is at for curing diabetes type 1 with stem cells?  Can you suggest any recent articles I can read?

A: There is anecdotal evidence that children who have had umbilical cord blood transplants to replace bone marrow for other reasons will resolve their Type 1 diabetes. This is because, as you know, Type 1 diabetes is an autoimmune disease. Replacing bone marrow will eliminate the auto immune disease. Unfortunately, there has been no large scale clinical trial confirming these findings. On the other hand, if a child has his or her own umbilical cord blood, it would be worthwhile considering a trial.

Q. What is the % of success rate for another immediate family member to have a successful cure of (for example cancer) using a siblings/sons/daughters cord blood if it was allowed?

A: The probability of a perfect 6/6 HLA match with a sibling is about 1:4, it could be higher if the mother and the father shared one or more HLA antigens. There are of course public banks providing HLA-matched cord blood should the available related cord blood units not match.

Professor Zhan-guo Li has worked with a team of researchers from Peking University People’s Hospital, China, to carry out a laboratory study in which cord blood stem cells were used to suppress inflammation and weaken the disease.

Professor Li said, “Very little is known about umbilical cord MSCs, and there has been no previous report about their use in the treatment of RA. MSCs can exert profound immunosuppression, which encourages their use in the treatment of autoimmune diseases, such as RA. At present, the most common source of MSCs has been bone marrow. However, aspirating bone marrow is an invasive procedure and the number and the differentiating potential of bone marrow MSCs decrease with age. In contrast, the collection of umbilical cord MSCs does not require any invasive procedure.”

The researchers took immune cells from RA patients and showed that the umbilical MSCs were able to suppress the cells’ proliferation, invasive behavior and inflammatory responses. Systemic infusion of the umbilical MSCs into mice was shown to significantly reduce the severity of collagen-induced arthritis.

Speaking about the results, Professor Li said, “RA imparts a massive burden on health services worldwide and none of the currently used agents reaches long term drug-free remission. Therefore, a new and more effective therapy for RA will be very welcome.”

To read more: http://www.ahealthblog.com/cord-stem-cells-a-potential-arthritis-treatment.html

Maia with mum, Jillian in the park

Maia was less than a year old when parents Jillian and Daniel realised she was developing differently to her twin sister Arielle. She’d been deprived of oxygen at birth and diagnosed with a cerebral palsy-like condition which affected both her speech and movement. She had trouble with balance and was

severely restricted in her learning and development. It was hard for her to concentrate and she had very little speech.

Maia’s parents battled hard to help her in any way they could. In 2008, they became aware of a programme at Duke University in the USA, treating children for brain injury with their own cord blood. Because Maia’s cord blood was banked at birth she was able to get this treatment.

In August 2008, Maia was reinfused with her own cord blood at Duke by Dr. Kurtzberg, a leading paediatric oncologist.

The improvements came thick and fast and Maia, now seven and a year 2 student at school in Auckland, is still developing in leaps and bounds.

Maia’s speech therapist, Ann McElwee, has seen dramatic improvements in Maia. Her class teacher, Louise Canning, who worked with Maia last year also says the changes have been remarkable.

 “When Maia first started with us she wasn’t able to work independently and needed constant one-on-one supervision to perform any tasks. Her progress has been absolutely phenomenal. She can now count, read, write and work on her own.”

Two years after her cord blood treatment, life for the Friedlanders is completely different.

“We have a real, active family which we are so thankful for.” “The two sisters interact and Arielle now has a proper playmate. They fight like most siblings which is actually wonderful to see!” says Jillian.

Jillian and Daniel are adamant Maia would not be making this progress without the cord blood re-infusion. “To every person who is pregnant I say bank their baby’s cord blood as you never know when you might need it.”

• Dr Young’s trials are being undertaken by the China Spinal Cord Injury Network (China SCINET) which he helped established in 2003. It is the largest spinal cord injury clinical trial network.

• ChinaSCINet has completed three clinical trials to date. The first is an observational trial of 500 subjects with spinal cord injury (SCI) to show that the 24 ChinSCINet centers in mainland China, Hong Kong, and Taiwan can collect follow up data on people with SCI.

The second is a phase 1 trial carried out at Queen Mary Hospital of Hong Kong University showing the safety and feasibility giving a 6-week course of oral lithium to 20 people with chronic SCI.

The third is a double-blind randomized placebo control trial to assess the safety and efficacy of a 6-week course of oral lithium. The fourth trial is currently being carried out.

This trial is testing increasing doses of umbilical cord blood mononuclear cells (UCBMC) without and then combined with a 6 week course of lithium on 40 patients. If this trial shows promising results, testing the treatment in phase 3 trials in China, United States, India, and Norway will commence.

• As many US patients wanted to take part in the China-based trial, Dr Wise established a US-based group (called SCINetUSA). Several US-based spinal cord injury centres have expressed interest in joining this group including Mt Sinai Medical School (NY); UMDNJ/Kessler Institute of Rehabilitation Medicine and the University of Colorado at Denver. The flagship institution in SCINetUSA is University Medical Center Brackenridge in Austin, Texas.

July has been designated “Cord Blood Awareness Month” by the American Hospital Association’s Society for Healthcare Strategy & Market Development (SHSMD), with the goals of raising awareness about cord blood stem cells and helping empower expectant parents to make informed choices regarding their family’s future health.

Here in New Zealand you can help raise awareness by telling expectant friends and family members about cord blood banking. For everyone who then registers with CordBank – you will both get 1 year’s free storage. For more information click here

Three more kiwi children are set to benefit from a cord blood lung study currently underway in Melbourne.

Led by Professor Bob Williamson at the University of Melbourne’s Faculty of Medicine, the study is using umbilical cord blood to treat Cystic Fibrosis an inherited disease in which a thick mucus clogs the lungs, making it very hard to breathe and digest food.

To help support this ground breaking study, CordBank has agreed to collect, process and store cord blood from families, who already have one child with CF, for free.

The latest families to benefit from this support are the Legge family from Queenstown, the Conlands from Wellington and the Hasselmans from Napier.

Bob Williamson, Professor of Medical Genetics at the University of Melbourne, has a research group trying to take cord blood stem cells and push them down the path towards lung cells.  “So far, we have been able to get cells from cord blood to make some lung proteins, such as surfactant, but the CF protein still seems to be present only at low levels.  We have, however, been able to correct gene defects in cord blood stem cells, and groups are trying to put a healthy copy of the CF gene into stem cells and get it to function normally.” 

Professor Williamson also noted that other groups, including one in Perth, Australia, have used similar cells from the placenta to repair lung injury.  “While this research is aimed at helping premature infants, rather than those with CF, it is valuable to know that stem cell science is advancing quickly towards effective treatments of many diseases.”

Professor Williamson noted that this cord blood could be very valuable in future, if a way can be found to get the stem cells to express the CF gene.  “This is a real challenge”, he said, “but there is now a great deal of stem cell science throughout the world aimed at getting this result, and progress is occurring rapidly.”

Australian researchers have just found a way to use stem cells to reduce injury and scarring in the lung and even generate new lung cells.

Lead researcher, associated professor Yuben Moodley from the University of Western Australia, said the investigation could provide hope for patients suffering from lung damage.

In Australia, one in four are affected by serious lung conditions. Globally, lung diseases cause many deaths and disabilities, with most lung conditions responding poorly to traditional medicines. “Cellular therapies, although in the early stages of development, may form a vital part of future life-saving treatments.” Moodley said.  To read the full news story click here

In February 2010, a study reporting the successful treatment of spinal cord injury in dogs using cord blood was released – with researchers confident that the this technique has many potential applications in the treatment of human spinal cord injury.

While previous research on spinal cord injury has typically used rats, this study used dogs which have a spinal cord structure more similar to humans. Within the test group, those dogs that received cellular transplants exhibited gradual improvement in hind limb locomotion two to three weeks after the transplant and also demonstrated reduced cyst and injury size.

The study was a collaborative effort between a team of doctors from the Department of Veterinary Science and Veterinary Anatomy at Konkuk University in Seoul Korea.

Dr Han, President and founder of Histostem, said the research demonstrated a new less invasive method of transplanting cord blood stem cells which avoids surgical exposure and allows the cells to be more precisely transplanted into the spinal cord.