Bolivar ‘bubble boy’ leaves Children’s Mercy Hospital

September 11, 2008

Bolivar ‘bubble boy’ leaves Children’s Mercy Hospital
By DEBRA SKODACK
The Kansas City Star
Despite the dreary gray weather, Thursday turned into one of the brightest days so far in the life of tiny Granton Bayless.

Eleven-month-old Granton rolled out Children’s Mercy Hospital’s front door in a bright blue wagon wearing the cowboy boots his parents bought months ago in hopes that this day would happen.

Granton’s blue eyes widened as he peered over a protective mask to a sky he hadn’t seen since being admitted to Children’s Mercy on March 27.

The Bolivar, Mo., infant was born with a rare condition — severe combined immunodeficiency — that prevents his body from producing enough T cells to fight off disease.

The condition often is referred to as the “bubble boy” disease because its victims are so vulnerable to infectious diseases that some must live in protective bubbles. Experts estimate that it occurs once in every 500,000 births. The cure is either a bone-marrow or a cord-blood transplant.

What made Granton’s recovery remarkable was how gravely ill he was when he underwent a cord-blood transplant June 10. At the time, the baby was on a ventilator fighting multiple infections.

There were no medical statistics, no success rates on whether Granton would live.

“We’re not talking percentages, more like we have heard (success) stories,” said Doug Myers, a pediatric oncologist and one of a dozen or so doctors and nurses who came to say goodbye to Granton on Thursday.

In fact, for most of the time, Daniel and Jenni Bayless faced the possibility of their only child dying while at Children’s Mercy.

One of the most critical periods came in late May, when doctors put Granton on a ventilator. They gave him medicine that paralyzed him so the equipment wouldn’t hurt him if he moved and so he wouldn’t burn energy or oxygen.

The Baylesses considered taking Granton off the ventilator and having a tracheotomy performed.

“I was thinking about not being able to see him wake up before he dies,” Jenni Bayless said Thursday of that time. “I wanted to see him awake and look into his eyes.”

Devout Baptists, the Baylesses prayed for guidance and decided to wait. Granton rallied enough to try the cord-blood transplant.

A bone-marrow transplant was considered too risky because Granton was fighting a respiratory syncytial virus. But before that was ruled out, about 2,200 people in five towns in Missouri and Kansas registered as bone-marrow donors in hopes of helping Granton or someone else.

Already, two people from those drives have been matched to awaiting patients, including a 55-year-old woman fighting leukemia.

The Baylesses had hoped to take Granton home to Bolivar for his first birthday on Oct. 2. But his doctors want them to stay at a sanitized apartment at the Ronald McDonald House for two months to allow his immune cells to mature more.

“It is kind of discouraging not to be going home but this will help protect him from other kids,” said Jenni Bayless, adding she would be tempted to have friends over or to take Granton to church.

“This is extra security and protection so he isn’t in places he shouldn’t be,” she said. “That’s good, because I don’t want to go through this again.”

The first year after a transplant is most risky for Granton, said Jignesh Dalal, a member of Granton’s transplant team. But, Dalal said Thursday, once Granton’s immunity system develops, he can live a normal life.

Shortly after 5 p.m. Thursday, Jenni Bayless stood at the door of her family’s apartment at the Ronald McDonald House. Granton snuggled in her arms, a feeding and medicine tube coming out of his left side — a reminder of the challenges still ahead.

“It’s feels wonderful,” Jenni Bayless said. “It feels awesome.”

linkback url: http://www.kansascity.com/105/story/793245.html

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Enzon Pharmaceuticals might sell Indy facility

September 10, 2008

Does anyone know what affect this will have on those patients who need PEG-ADA to survive?

Enzon Pharmaceuticals might sell Indy facility

Mon. August 11 – 2008
Norm Heikens –  nheikens@ibj.com
IBJ staff

By Norm Heikens

An Indianapolis plant that manufactures specialty drugs might be sold, its parent announced yesterday.

Enzon Pharmaceuticals Inc., based in Bridgewater, N.J., said it might sell the plant or one or all of its products as part of a strategic review of its specialty pharmaceutical business.

The plant at 6925 Guion Road on the far-northwest side makes Oncaspar, for leukemia; DepoCyt, for meningitis; Abelcet, for fungal infection; and Adegen, for the “bubble boy disease” immune disorder.

Enzon did not return calls seeking comment. In early 2007, Indy Partnership, the organization that markets Indianapolis to expansion-minded companies, said the operation employed about 100 workers.

The company said it has received preliminary interest from companies considering buying all or part of the business.

This summer, Enzon moved to spin off its biotechnology business as a separate publicly traded company under the name Evivrus Inc.

Billionaire shareholder Carl Icahn has pressed the company to unload assets or sell itself in order to increase value to shareholders.

Enzon stock has languished below $10 a share since 2006. The shares traded at $8.74 today.

linkback url: http://www.ibj.com/html/detail_page_Full.asp?content=18520


Resistance of mature T cells to oncogene transformation

September 9, 2008

Resistance of mature T cells to oncogene transformation

Sebastian Newrzela1, Kerstin Cornils2, Zhixiong Li3, Christopher Baum3, Martijn H. Brugman3, Marianne Hartmann1, Johann Meyer3, Sylvia Hartmann4, Martin-Leo Hansmann4, Boris Fehse2, and Dorothee von Laer1

1 Georg-Speyer-Haus, Institute for Biomedical Research, Frankfurt; 2 University Hospital of the Johann Wolfgang Goethe-University, Experimental Pediatric Oncology and Hematology, Frankfurt am Main; 3 Hannover Medical School, Department of Experimental Hematology, Hannover; and 4 University of Frankfurt, Department of Pathology, Frankfurt, Germany

Leukemia caused by retroviral insertional mutagenesis after stem cell gene transfer has been reported in several experimental animals and in patients treated for X-linked severe combined immunodeficiency. Here, we analyzed whether gene transfer into mature T cells bears the same genotoxic risk. To address this issue in an experimental “worst case scenario,” we transduced mature T cells and hematopoietic progenitor cells from C57BL/6 (Ly5.1) donor mice with high copy numbers of gamma retroviral vectors encoding the potent T-cell oncogenes LMO2, TCL1, or {Delta}TrkA, a constitutively active mutant of TrkA. After transplantation into RAG-1–deficient recipients (Ly5.2), animals that received stem cell transplants developed T-cell lymphoma/leukemia for all investigated oncogenes with a characteristic phenotype and after characteristic latency periods. Ligation-mediated polymerase chain reaction analysis revealed monoclonality or oligoclonality of the malignancies. In striking contrast, none of the mice that received T-cell transplants transduced with the same vectors developed leukemia/lymphoma despite persistence of gene-modified cells. Thus, our data provide direct evidence that mature T cells are less prone to transformation than hematopoietic progenitor cells.

linkback url: http://bloodjournal.hematologylibrary.org/cgi/content/short/112/6/2278?rss=1


Cognitive and behavioural abnormalities in children following haematopoietic stem cell transplantation for severe congenital immunodeficiencies

September 4, 2008

Cognitive and behavioural abnormalities in children following haematopoietic stem cell transplantation for severe congenital immunodeficiencies

Penny Titman*, Elizabeth Pink, Emily Skucek, Katherine O’Hanlon, Tim J Cole, Jane Gaspar, JinHua Xu-Bayford, Alison Jones, Adrian J. Thrasher, E Graham Davies, Paul A. Veys, and H. Bobby Gaspar

Psychosocial Services, Great Ormond Street Hospital, London, United Kingdom
Centre for Paediatric Epidemiology and Biostatistics, UCL Institute of Child Health, London, United Kingdom
Immunology Department, Great Ormond Street Hospital, London, United Kingdom
Molecular Immunology Unit, UCL Institute of Child Health, London, United Kingdom
Dept of Bone Marrow Transplant, GOSH, London, United Kingdom

* Corresponding author; email: titmap@gosh.nhs.uk

Haematopoietic stem cell transplantation (HSCT) is a highly successful treatment for severe congenital immunodeficiencies. However, some studies have suggested that children may experience cognitive difficulties following HSCT. This large scale study assessed cognitive and behavioural function for the cohort of children treated by HSCT at one centre between 1979 – 2003 to determine the frequency and severity of problems and to identify risk factors. 105 patients were assessed on standardized measures of cognitive and emotional and behavioural function together with a control group of unaffected siblings. The average IQ for the cohort was 85 (95 percent confidence interval 81 – 90), significantly lower than both the population average of 100 (p<0.001) and unaffected siblings. Multivariate analysis indicated that the underlying genetic defect, diagnosis of ADA deficient severe combined immunodeficiency and consanguinity were associated with worse outcome, but that age at transplant and chemotherapy conditioning were not. Children treated by HSCT for severe immunodeficiency have an increased risk of long term cognitive difficulties and associated emotional and behavioural difficulties. The specific genetic diagnosis, consanguinity and severe clinical course are associated with poor outcome. Long term follow up of these patients should include screening to identify and manage these problems more effectively.

linkback url: http://bloodjournal.hematologylibrary.org/cgi/content/short/blood-2008-04-151332v1?rss=1


Local Habitat for Humanity group turns sod on new home for Centreville family, with hopes to finish construction by the holidays

September 2, 2008

Local Habitat for Humanity group turns sod on new home for Centreville family, with hopes to finish construction by the holidays

A dream home is one step closer to becoming a reality for a Centreville family.

Last month Daisy Gallant and Johnathan DeMerchant found out they were the recipients of a new home thanks to the Carleton County Chapter of Habitat for Humanity.

Last Thursday evening, surrounded by friends and family at a vacant lot along Hawkins Road in Centreville, the couple took the first step towards completing their new home with a special sod-turning ceremony.

The kindness shown to her family has been remarkable, Gallant said, and has left her speechless.

“It is hard to put into words,” said Gallant. “It seems completely unreal an organization like Habitat for Humanity can do something like this for a family. This means a lot, and we are extremely grateful.”

The news is extra special for Gallant and DeMerchant as Habitat for Humanity will also create a safe environment for the couple’s young daughter Katlyn.

Nearly two years ago, the youngster was diagnosed with a severe combined immunodeficiency (SCID) called adenosine deaminase (ADA) deficiency, a rare genetic disorder which forced the little one to live in an isolation room for the early part of her life.

Through gene therapy treatment sessions, the two-year-old was able to return home for Christmas last year and has since enjoyed a happy childhood, though she still has to be careful.

Katlyn’s new home will be equipped to protect her from infection, like installation of water filters on all of the taps in the home, so she can take a bath or drink the water from the tap without any worries.

“It is going to allow us to provide her with the safest environment possible, which is essential for her health,” added Gallant. “We are incredibly grateful for that.”

The young couple has struggled to get out of its one-bedroom apartment, said DeMerchant, due to numerous medical and travel expenses with young Katlyn.

“We travel so much and so often, it is hard to keep ahead,” DeMerchant said. “It is nice to have a chance like this.”

He added he is truly appreciative to see the community rally behind the project.

Even little Katlyn said she is excited about her new home and having her own room.

Gallant and DeMerchant’s home is the second one on the Carleton County Chapter of Habitat for Humanity list. Earlier this summer, the volunteer group finished its first project in Woodstock for Trent and Karen Davenport.

“It is our goal, if we can do it, to build a home a year somewhere in Carleton County,” said John Young, chairperson for the Carleton County projects. “There is lots of need, and we have lots of wonderful volunteers. Carleton County is so giving, it is making this all possible for us. It is fantastic.”

With the sod turning now complete, Young said construction is slated to begin on Tuesday morning, Sept. 2. With the number of volunteers and local companies pledging their support, he expects the three-bedroom home will be complete fairly quickly.

“This house will go up fairly fast, which will be wonderful,” Young said. “We would like to have the family in by Christmas.”

Being able to move in by Christmas is music to the family’s ears, said Gallant, and it certainly would make for a memorable holiday.

Last year, Gallant said, Katlyn was able to come home and celebrate Christmas for the first time with her family.

It would be wonderful to celebrate this year in the family’s new home, she added.

“It would be a great Christmas present for sure,” Gallant said.

linkback url: http://bugleobserver.canadaeast.com/news/article/402688


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