Brother's stem cells could be gift of life

Stephanie Payne with Tayler, left, who has cystic fibrosis,
and his younger brother Jordan. Times photo Wayne Martin.

 LITTLE Jordan Payne doesn’t know it yet, but one day he could well be the key to saving his brother’s life.


The three-year-old’s big brother Tayler has cystic fibrosis (CF), a genetic condition that thickens the body’s mucus and blocks tiny tubes in various organs.

About one in 3000-3500 children in New Zealand are born with CF and need regular physiotherapy to keep their airways clear, prevent lung damage and reduce infection.

When Jordan was born his parents decided to save his umbilical cord blood, which is full of stem cells that could help with his brother’s illness in the future.

Watching Tayler tear around the house with his best mate in tow, it is easy to forget he has a life-threatening illness.

Twice a day the four-year-old is given chest percussion by his mum Stephanie, which involves tapping his chest to shift the mucus from his lungs.

“He’s a little star,” she says. “He just sits there and takes it all.

“But he’s at the stage where he asks why he has to have physio and Jordan doesn’t.”
A hand sanitiser unit positioned at the Paynes’ front door is a telling sign of the care the family takes to safeguard Tayler’s health.

When the little boy develops a cough or cold he has to be treated with antibiotics as soon as possible to stop the infection in its tracks.

“Cystic fibrosis is a lifetime illness,” says Stephanie.

“They are born with it and they carry it through for the rest of their lives. It’s not ‘if’ he ever gets unwell, it’s ‘when’.”

However, she and her husband Brett try to resist the urge to wrap their son up in cotton wool as much as possible, so he can have a normal childhood.

“Bacteria is a big concern,” Stephanie told the Times.

“You have to be mindful, but you can only do the best you can. And we’ve had a lot of luck with him.”

A few days a week Tayler goes to Superstart Pre-school in East Tamaki, which has been incredibly accommodating of his needs.

In the past, the centre has waived the family’s childcare fees and donated the same amount from its own pocket to the Cystic Fibrosis Association.

“They are just amazing,” says Stephanie. “The kindy has been so supportive. I feel he’s in such safe hands and they have an extremely good safety policy.”

The mum-of-three is running a fundraiser at the Pakuranga Scout Hall from 10am-2pm this Saturday to put the spotlight on Cystic Fibrosis Awareness Week, which runs from August 15-21.

It will feature a white elephant sale to raise money and awareness of CF, and a gala with entertainment such as bouncy castles, face painting, raffles and a sausage sizzle.
“Everything goes to the association,” says Stephanie. “Usually the money is used to buy equipment and medication. A nebuliser is really expensive, and its maintenance is expensive too.”

Quality household items, clothes and toys are being sought for the bric-a-brac sale at 474 Pakuranga Road, and they can be dropped off at Stephanie’s home.

“For me, it’s more important to raise awareness than money, because so many know so little – if anything – about cystic fibrosis,” she says.

“The hard thing is they look so well on the outside.”

She hopes advances in medicine will one day make use of Jordan’s banked stem cells and a difference to the life of her eldest son.

“I’m so optimistic about his future now,” she says.

“There’s a lot of research now, and the possibility of Jordan saving his life one day.
“You talk with other parents and you become more hopeful.”

Source: Times Newspapers, New Zealand,  8 August, 2011

康盛人生臍帶血庫 9月份講座

萬能幹胞製人工心肌 為「人工心臟」鋪路

港大李嘉誠醫學院幹細胞及再生醫學研究組總監李登偉及其研究團隊，獲研資局撥款6000萬元，利用5年時間研發「萬能幹細胞」製造「人工心臟」技術和安全性，為未來將「人工心臟」移植至人體鋪路。（尹錦恩攝）

為「人工心臟」鋪路

【明報專訊】研資局早前撥出約2.5億元作主題研 究，其中6000萬元撥作研究「萬能幹細胞複製人類心臟」。項目統籌港大李嘉誠醫學院幹細胞及再生醫學研究組總監李登偉指出，現已可透過培育「萬能幹細 胞」，以一個月時間製造出一條直徑1毫米的心肌，期望利用5年時間加強研究，為長遠製造首個「人工心臟」並移植人體鋪路。

李登偉說，人體器官各由不同細胞組成，其中腦部和心臟細胞無法增生。以心臟為例，若病人患上中風、心血管閉塞等疾病，將導致大量心臟細胞壞死，令其他細胞的工作量大增導致心衰竭，理論上只有器官移植才能根治問題，因此近年科學家致力研究「人工心臟」。

獲6000萬元研發

過 去10年，科學界利用病人的皮膚抽取細胞，培育出人工誘導的「萬能幹細胞」，並將之培育成不同器官的細胞。李登偉說，最新技術已成功將1000萬個心肌細 胞，利用生物工程技術，將細胞放入預先準備的「生物框架」，將細胞整齊排列並固定位置，砌成一條長幾毫米、直徑1毫米的心肌，並與正常心肌一樣可收縮心跳 及傳送心電；連同培育細胞的時間，約需1個月才能製造一條心肌。

他表示，現時成功在實驗室製造心肌，不等於已可造出「人工心臟」，以及立即移植至人體，「今次希望能解決製造『人工心臟』的一連串問題，包括研究如何確保人工心臟移植入人體後不會出現排斥問題，以至『人工心臟』的耐用性、安全可靠度等」。

研究團隊正着手設計「人工心臟」的藍圖，包括如何大規模培育不同器官的細胞，以至將細胞排列成器官亦是一大難題，研究團隊將在研究過程中改善現有儀器，甚至研發新儀器解決大量生產的問題。

現時幹細胞治療法只有骨髓移植一種，其他所謂幹細胞仍未廣為醫學界認同。李登偉估計，單是通過美國食品及藥物管理局審批，可能需時5至10年，現階段難以評估實際何時有望應用在人體身上。

資料來源: 明報, 新浪網 (2011年7月27日)

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Lab-made organ implanted for first time

This is the artificial trachea, covered in the patient's cells.

(CNN) -- For the first time, a patient has received a synthetic windpipe that was created in a lab with the patient's own stem cells and without using human donor tissue, researchers said Thursday.

Previous lab-generated transplants either used a segment of donor windpipe or involved tissue only, not an organ.

In a laboratory in London, scientists created a trachea, which is a tube-like airway that connects at the voice box and branches into both lungs.
On June 9, doctors implanted this synthetic windpipe into a 36-year-old man with late-stage tracheal cancer at Karolinska University Hospital in Stockholm. The patient is doing well and is expected to be released from the hospital Friday, said Dr. Paolo Macchiarini, professor of regenerative medicine there.

Tracheal cancers are extremely rare, accounting for less than 1% of all cancers.
After the patient's initial diagnosis in 2008, he had exhausted every treatment available, including chemotherapy, radiation and surgery, Macchiarini said. The patient, an Eritrean who had been studying in Iceland, is the subject of a BBC documentary airing Thursday in Sweden.
His tumor had almost blocked his windpipe, doctors said.

Rather than waiting for a transplant, his doctors suggested growing an organ. Scientists created a Y-shaped framework for the new trachea, modeling it after the specific shape of the patient's windpipe.

The form was made of polymers that had a spongy and flexible texture. Stiff rings around the tube mimicked the structure of a human trachea.

The form was then bathed in a solution containing the patient's stem cells "to get the cells to grow on the sponge material," said David Green, president of Harvard Bioscience. Stem cells can divide and turn into a range of cell types, including those in organs.

His company worked on the stem cell solution, which is seen as a pink liquid in the photo at left. The purpose was to "seed" the synthetic windpipe -- as you would seed a new lawn -- to grow on the structure.

"Stem cells from the own patient were growing inside and outside," Macchiarini said. "This structure was becoming a living structure."

The stem cells were given physical or chemical cues to create the desired type, Green said.
Once the cells were thriving on the form, the artificial trachea was implanted into the patient.
His body accepted the new trachea, and he even had a cough reflex two days after the surgery, Macchiarini said.
Three years ago, Macchiarini made headlines by implanting an artificial trachea created from donor tissue combined with stem cells from the recipient, Claudia Castillo, whose windpipe had been damaged by tuberculosis.

"The results were quite good, but unfortunately we were still dependent" on organ donation, which can take months, Macchiarini said.

Creating the synthetic structure for the trachea in the current case took 10 to 12 days, compared with waiting months for an organ donor, Macchiarini said.

Earlier this year, regenerative medicine scientists at Wake Forest University School of Medicine reported that they had engineered five urethras between March 2004 and July 2007.

They had used a small piece of each patient's own tissue from the bladder, then grew the cells in a lab onto a mesh scaffold shaped like a urethra.

This area of research remains somewhat controversial in medicine, because critics say this could lead to human cloning.

But Macchiarini said making these first artificial organs viable in patients opens doors for future transplants through the relatively new field of regenerative medicine.

"It's a beautiful international collaboration," he said about the recent effort that involved doctors and researches in Sweden, the United Kingdom and the United States. "If scientists and clinicians work together, we can help humanity."

Source: CNN, 7th July 2011

Study gives hope to patients with genetic disease

Aussie study brings hope to a Templeton family
AMY CLASS

Anahera Inns, 4, with baby brother Tiaki aged
4 months. Blood from his umbilical cord
may one day help Anahera'scystic fibrosis.

Templeton toddler Anahera Inns endures a daily fight to rid her lungs of thick, sticky mucus.

Anahera has cystic fibrosis (CF), a genetic lung disease that cannot be cured.

However, a University of Melbourne faculty of medicine study aims to prove that stem-cell-rich umbilical cord blood can treat cystic fibrosis by regenerating lung cells.

Melbourne University medical genetics professor Bob Williamson said the project would eventually use

sibling's cord blood to treat children with cystic fibrosis (CF).

"Sibling cord blood has a one-in-four chance of being a perfect match that will not be rejected by the immune system of the child living with CF," he said.

Progress was slow, since it was essential to prove cells were safe to use in the lung, but the research was "positive", he said. However, a breakthrough on the research could be several years away.

New Zealand cord-blood stem-cell banking facility CordBank had since offered free sibling cord-blood banking to families who had a child with CF, he said.

"Because CordBank has come on board with the project, we are ensuring that affected families are banking precious sibling cord blood now, so they are ready when a breakthrough comes."

With the assistance of CordBank, the Inns family was able to collect the cord blood from Anahara's youngest brother, Tiaki, who was born in February.

The family were given free collection and storage for the next 18 years.

The blood is collected after a baby is born, before the mother's placenta is delivered. It must be transported to Auckland quickly for processing and storage.

So far, seven New Zealand families have taken up the CordBank offer.

Cystic Fibrosis Association of New Zealand chief executive Kate Russell said cystic fibrosis "is an expensive illness and families with a CF child can be placed under severe financial strain".

"People would be surprised at what the Government does not fund in terms of the most basic medical equipment and support for these families," she said.

Cecileah Inns said her daughter had "as normal a life as possible".
The toddler endured vigorous physiotherapy and was nebulised daily to help with lung drainage, she said.

"She's articulate and brave - a right little character. She doesn't know any different."
Anahera enjoyed being a "bossy" big sister to her brothers Mika, 3, and Tiaki, four months, neither of whom has cystic fibrosis.

 Source: The Press, 4th July, 2011

 

Jun 16, 2011

First patients enroll in US stem cell trials on blindness

WASHINGTON - THE first clinical trials that examine the use of stem cells to treat two forms of blindness are ready to begin now that patients have been enrolled, a US company announced on Thursday.

A total of 24 patients have entered two separate trials at an eye institute in California, said representatives from the Massachusetts-based Advanced Cell Technology.

ACT was cleared by the US Food and Drug Administration several months ago to begin clinical trials of human embryonic stem cells to treat a form of juvenile blindness known as Stargardt's disease and dry age-related macular degeneration.

Now that patients have been enrolled, the trials aim to check the safety of the treatment before moving on to see whether the therapy can help stop vision loss.

'These trials mark a significant step toward addressing what is one of the largest unmet medical needs of our time, treatments for otherwise untreatable and common forms of legal blindness,' said lead investigator Steven Schwartz at University of California Los Angeles Jules Stein Eye Institute.

Dry age-related macular degeneration is the most common form of irreversible vision loss in people over age 55. There is currently no cure for the disease, which affects around 10 to 15 million Americans and another 10 million people in Europe, the company said. -- AFP

 Source: The Strait Times, 16 Jun 2011