Death of 3-year-old spurs Ottawa school to hold auction to help pay medical bills

Death of 3-year-old spurs Ottawa school to hold auction to help pay medical bills

By Eric Weslander

Thursday, November 30, 2006

During her short life, 3-year-old Ottawa resident Chloe Robinson developed a love for knock-knock jokes. She told them to anyone who would listen: doctors, nurses, family members. Her favorite:

Knock, knock.

Who’s there?

Boo.

Boo who?

Don’t cry, it’s just a joke.

“We joked that she knew every knock-knock joke that you could ever know,” said her mother, Chrissy. “She was very smart for being locked up in the hospital room.”

Chloe died earlier this month at Children’s Mercy Hospital in Kansas City, Mo., after a lifelong battle with severe combined immunodeficiency, or SCID. This weekend, the Ottawa elementary school where Chloe’s father works is holding a silent auction fundraiser to help the family defray its medical costs, which haven’t yet been tallied.

“Anyone that’s seen pictures of Chloe or heard about her struggle ... you couldn’t help but fall in love with her,” said Tori Wilson, a technology aide at Garfield Elementary School in Ottawa. “That same spirit is alive in Josh and Chrissy too ... They’re part of us.”’

The auction will be from 9 a.m. to 4 p.m. Saturday at the elementary school. Items donated include autographed Kansas City Chiefs memorabilia and Kansas University men’s basketball tickets from coach Bill Self’s personal allotment, Wilson said.

If there’s any money left over from paying bills, the family plans to use it to help start a foundation in their daughter’s memory that will help other families in a similar situation.

“Rather than continue to ask why, we’re starting to decide what we can do to make sure that, even though she died early, her life was still meaningful,” said her father, Josh, a first-grade teacher.

Chloe was born in August 2003 in Lawrence. From birth, she had chronic gastrointestinal problems that were later attributed to her immune system.

She spent only a few weeks of her life in Ottawa. The rest of her time was spent under medical care in Kansas City, Nebraska, Boston, North Carolina and finally in Cincinnati, where she received a bone-marrow transplant early this year aimed at replacing T-cells in her body.

“On her transplant day, we threw a big tea party,” Josh Robinson said. “Since then, tea parties were her favorite thing to do.”

She had a way of making adults laugh, her parents said. They said that once, when she went into surgery, a hospital employee came out laughing because Chloe had been telling knock-knock jokes up until the point that she went under anesthesia.

After her bone-marrow transplant, her immune system improved. But in August of this year, her liver began to fail.

For the last weeks of her life, she left the hospital where she’d been staying in Cincinnati and returned to Children’s Mercy in Kansas City.

“We were basically sent back to Children’s Mercy with the understanding that her liver was going into failure, and we wanted her to be closer to home, wanted her to be able to spend time with her family,” Josh Robinson said. “I don’t think she knew necessarily that she was dying, but we got the sense that she knew something wasn’t right.”

Near the end, the decline in her health happened quickly. Even on her last day, when she developed internal bleeding, she had been sitting up in bed, painting, telling jokes and reading books with people, her father said. She died Nov. 11.

“Our greatest fear, knowing that she was dying, was that we were going to have to be the ones to decide to turn something off or stop something. There weren’t any choices. It was that quick,” Josh Robinson said. “She curled up with her mom and went to sleep.”

At her memorial service, the Robinsons had a photo of Chloe with room for visitors to write a knock-knock joke around the edges. They also gave away tea bags with a note that said, “With your next cup of tea, think of Chloe.”

The Robinsons said they’re grateful for the help they’ve received from the community.

“It’s been amazing,” Josh Robinson said.

Linkback URL: http://www2.ljworld.com/news/2006/nov/30/
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Duke takes strides in stem cell research

Jasten McGowan

Posted: 11/29/06

Since stem cell treatments first became a possibility in medicine during the 1960s, Duke University Medical Center researchers and physicians have played a major role in advancing their increasingly complex uses.

In recent years, DUMC has worked to create a number of unique methods to apply stem cells to the treatment of cancer and rare diseases.

Linkback URL: http://www.dukechronicle.com/media/storage/paper884/news/2006/11/29/News/
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"Stem cells were once used as a last-ditch effort," said Dr. Keith Sullivan, director of Duke's Center for Cancer Outcomes and Research. "But, with Duke's reputation for the treatment of rare and complex diseases, things are rapidly changing."

Sullivan is lead investigator of the trial study Scleroderma: Cyclophosphamide or Transplantation, which treats patients suffering from severe forms of the rare skin disease commonly known as systemic sclerosis.

The trial compares stem cell treatment to high-dose drug treatment using cyclophosphamide, a chemotherapy agent used to treat chronic immunosuppression.

Sullivan's SCOT trial approach, however, is merely one of many at Duke that applies variations to the traditional approach to stem cells, which once required perfect matches with varying results.

Dr. Rebecca Buckley, professor of allergy and immunology in Duke's department of pediatrics, studies treatments for severe combined immunodeficiency disease, or "bubble boy disease," which is caused by abnormal changes in the development of the T cells of the immune system.

Buckley has utilized methods that rid bone marrow of T cells, which are regenerated upon implantation.

"Unlike bone marrow [stem] cells requiring perfect matches, our approach doesn't require a perfect match," said Buckley, who has utilized stem cell transplants since the 1980s. "The 34 patients we've lost over the years were due to viral infection."

Buckley said methods of "manipulating stem cells to avoid the hindrance of the perfect match" are prevalent among Duke researchers.

"We have one of the largest stem cell reserves in the nation," Buckley said.

While scientists lobby for increased reserves of available stem cells-through increased blood donation in various forms-approaches to stem cell treatment for cancers and rare diseases are becoming increasingly complex.

Joanne Kurtzberg, director of the Pediatric Bone Marrow and Stem Cell Transplant Program and the Carolinas Cord Blood Bank at Duke, said the use of cord blood stem cells presents growing opportunities for children and some adults suffering from various cancer types and children with rare immune diseases.

Cord blood cells present alternatives to what were once "dead ends" in treatment options for some conditions, she said.

"For patients lacking sufficient bone marrow matches, this is their only choice," Kurtzberg said. Stem cells from cord blood are currently used to treat patients with cancers, certain genetic blood disorders and metabolic diseases.

Kurtzberg said that while it is likely that many types of stem cells will be used therapeutically over the next few decades, it is important to conduct research to determine the optimal ways to utilize them as therapeutic agents.

"While there are limited options for any one condition, approaches are changing," she said.

Stories of Lives Saved by Cord Blood

November 26, 2006
Stories of Lives Saved by Cord Blood - Larry Mitchell

The advancements of medicine and technology are saving lives each year that would otherwise be lost to disease or medical disorder. Each year, more and more children are living through potentially terminal diseases and battling their way back to health with help of cord blood. Cord blood is found within the umbilical cord and preserved upon the birth of the child. Since the blood contains stem cells, numerous diseases and disorders are successfully treated and even cured through transplants. Parents today are choosing to either bank cord blood for future use or donate their newborn’s cord blood so that ill children can take advantage of this life-saving blood.

An inspiring case is that of siblings Ashley and Kelvin J. of Maryland. These two children were both born with severe combined immunodeficiency syndrome that is usually terminal, since the body’s immune system cannot fend off the germs that would otherwise be harmless to a healthy body. Projected life span for children diagnosed with severe combined immunodeficiency syndrome (SCIDS) is approximately six months; however, both Ashley and Kelvin received a transplant of cord blood from anonymous donors whose cord blood was donated to public blood banks......

Linkback URL: http://cordblood.okat.us/cord-blood/
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NIH Launches 'Health Info Rx Program' On Newborn Screening And Related Genetic Disorders

NIH Launches 'Health Info Rx Program' On Newborn Screening And Related Genetic Disorders
Article Date: 26 Nov 2006 - 2:00 PST


After a doctor sees a patient, he or she often prescribes medications. But what if such a doctor also wants to direct a patient to up-to-date, reliable, consumer-friendly information about a genetic condition, or an explanation of the basics of genetic science? Under a new program launched today, practitioners are being encouraged to refer their patients to Genetics Home Reference, a free, patient-friendly Web site of the National Institutes of Health (NIH), at http://www.nlm.nih.gov/.

Under this program, doctors can request free "Information Rx" pads, which will enable them to write "prescriptions," pointing patients to the Genetics Home Reference site and to the wealth of information it contains. Obstetricians can direct their patients to the site's explanation of newborn screening, so expectant mothers will better understand why this testing will be important for their baby.

Pediatricians and family physicians who see new moms and dads often provide good advice on newborn or child care concerns. If there happens to be a problem detected in a screening, where should this doctor direct the concerned parents for reliable, easy-to-read information at a stressful time? NIH's Genetics Home Reference can be an invaluable resource.

All states screen newborns for certain genetic disorders. These conditions are usually not apparent in the newborn, but can cause physical problems, mental retardation and, in some cases, death.

Micki Gartzke, a patient advocate from Shorewood, Wisconsin, lost her 13-month-old daughter, LeA Marie, to a rare genetic disorder, Krabbe disease, in 1987. "As a parent, of course you want every possible piece of information when you find out your child is sick. The Internet back then was in its infancy --resources were scattered and I did a lot of hunting and pecking to find things that would help us. It's so gratifying to see a resource like Genetics Home Reference, which has collected and organized a wealth of helpful materials into a one-stop shopping experience. I know that it is of great relief to parents I work with through Hunter's Hope, the foundation to help families coping with Krabbe disease, and parents around the country."

Fortunately, most babies receive a clean bill of health when tested. When test results show that a baby has a health defect, however, early diagnosis and treatment can make the difference between lifelong disabilities and optimal development.

Four of the nation's most respected medical associations, with a combined membership of over 200,000, have teamed with two NIH institutes on this groundbreaking initiative. The National Library of Medicine (NLM), the world's largest medical library, and the National Institute of Child Health and Human Development (NICHD), the research arm of NIH dedicated to ensuring that every child in the U.S. is born healthy and grows up free from disease and disability, have entered into partnerships with the American Academy of Pediatrics (AAP), the American Academy of Family Physicians (AAFP), the American College of Obstetricians and Gynecologists (ACOG) and the American College of Medical Genetics (ACMG) to encourage physicians to point patients to first-rate online health information in NLM's Genetics Home Reference database.

"Part of a physician's job is to explain illnesses, diagnoses and treatments to their patients," says Donald A.B. Lindberg, MD, Director of the National Library of Medicine. "NLM's Genetics Home Reference provides authoritative, user-friendly, and commercial-free information that doctors can use to supplement information provided in the office or clinic. We think it saves time and improves doctors' communications with patients, in addition to its obvious value in helping keep babies healthy."

"Physicians have always known that an informed patient who takes an active role is a 'better' patient," notes Duane Alexander, MD, Director of the National Institute of Child Health and Hunan Development. "We believe that both patients and their doctors will welcome this additional tool -- good medical information -- in their continuing efforts to provide good health care, for newborns and for people of all ages."

###

Genetics Home Reference includes over 500 topics on genetic conditions and related genes. The site features a richly illustrated tutorial that explains the basics of genetics, from the cellular level on up, and a glossary of genetics terms. The site is regularly updated by scientific staff and reviewed by external experts.

A similar Information Rx Project, pointing patients to NLM's MedlinePlus database (http://medlineplus.gov/), was launched in 2003. That program has been well received by doctors and their patients nationwide, helping doctors direct patients to NLM's MedlinePlus database, with information on over 700 health topics and many other resources.

Located in Bethesda, Maryland, the National Library of Medicine is the world's largest library of the health sciences. For more information, visit the Web site at http://www.nlm.nih.gov/.

The National Institutes of Health (NIH) -- The Nation's Medical Research Agency -- includes 27 Institutes and Centers and is a component of the U. S. Department of Health and Human Services. It is the primary federal agency for conducting and supporting basic, clinical, and translational medical research, and it investigates the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nlm.nih.gov/.

Contact: Robert Mehnert
NIH/National Library of Medicine

linkback URL: http://www.medicalnewstoday.com/medicalnews.php?newsid=57201

Cystic Fibrosis Added to Newborn Screening Program

Cystic Fibrosis Added to Newborn Screening Program
Josh Pringle
Thursday, November 23, 2006

Ontario is adding cystic fibrosis to the list of screening tests for new babies under the Newborn Screening Program.

Starting late next year, newborn babies in Ontario will be tested for cystic fibrosis in addition to 27 other diseases.

Health Minister George Smitherman says early detection of the disease in newborns will help ensure they receive proper care as soon as possible.

backlink url: http://www.cfra.com/headlines/index.asp?cat=1&nid=44778

Proposed Rule Changes Aim To Strengthen Newborn Screening In Minnesota

Proposed Rule Changes Aim To Strengthen Newborn Screening In Minnesota

November 21, 2006 -- The Minnesota Department of Health (MDH) is proposing changes to rules affecting the screening of all newborn infants in the state for an array of congenital and inherited diseases that can often be life-threatening or debilitating. The changes would bring the rules up-to-date with recent changes in newborn screening laws made by the Legislature and with changes in the screening process as a result of scientific and technological advances. They also would further clarify the roles of MDH, hospitals and health care providers.

The department published its notice of intent to adopt changes in the newborn screening rules in the Nov. 20, 2006 State Register. The public has until Friday, Dec. 29, 2006 to comment on the proposed changes. If MDH receives 25 or more requests for a hearing, a public hearing on the proposed rules will be held on Tuesday, Jan. 23, 2007 at 9 a.m. in the Freeman Building of the Minnesota Department of Health, 625 Robert Street N, St. Paul, Minnesota 55155.

linkback url: http://www.allamericanpatriots.com/m-news+article+storyid-17139.html

Close Up: The Boy in the Bubble

Close Up: The Boy in the Bubble

James Thompson Richards


Nov 16, 2006

James Thompson Richards looks like any normal 13-year-old boy.

But he is not.

James was born with Severe Combined Immune Deficiency Syndrome - SCIDS. With no immune system he has spent most of his life in sanitised isolation.

"Just say I get the flu or I catch flu from someone in my class, I get it like four times as bad. But I feel like i've got the flu every day. Its' just like eating, It's just part of my life," James told Close Up.

Only around one in 50,000 people have this condition.

His first years were spent in isolation at Auckland's Starship Hospital, then later, alone in a sanitised room at home.

"When I was little I couldn't cuddle...or see my brothers, i'd just see them at the door they couldn't come in unless they wore gloves and masks."

James had a bone marrow transplant in Australia when he was five, but that was unsuccessful. And doctors say chemotherapy is out of the question because of his failing health, which includes chronic lung disease.

"When the professors told me in Sydney that there was nothing more they could do for him that he's never going to get better, that made me the decision at that time that I was going to tell James the truth about everything," says James' mum Jean.

James says he takes things day by day, which means alternating visits to Taranaki and Starship Hospitals every three months, where he stays for two week stints. Then there is the immunoglobulin antibiotics he injects into his belly at home, which he has done every second day, for the past six years.

Long term there is one long shot.

While researching James' condition on the internet, the family read about 11 boys in Europe with his condition, who were apparently cured using stem cell therapy.

But for James and his family, heading overseas is a huge and costly gamble.

"He's not a good candidate for it because his lungs have deteriorated too much...but these boys had a simialr if not worse prognosis," says James' dad Garth.

Taranaki Base Hospital agreed to talk to Close Up about James' condition, but pulled out of the interview after Jean and Garth said they also wanted to talk about stem cell therapy. It said it wasn't policy to talk about that subject.

Starship Hospital wouldn't talk about James' case specifically, but sent Close Up a statement about the use of stem cell therapy as treatment for people with severe combined immuno deficiency syndrome:

"A bone marrow transplant is always the preferred option for SCIDS patients. Gene therapy can help some. But because there's only small number of people with the condition in New Zealand, it's not available here."

While stem cell therapy overseas is a risky option for James - and a hugely expensive one for his family - the mere hope it could give him a better life means they are prepared to try to get him there.

They have been in touch with doctors at Great Ormond Street Hospital in England and are preparing a fundraiser to get him there.

Linkback URL: http://tvnz.co.nz/view/page/411416/894326

Newborn Screening: Complexities in Universal Genetic Testing

The following is an excerpt from:
Newborn Screening: Complexities in Universal Genetic Testing
By Green, Nancy S; Dolan, Siobhan M; Murray, Thomas H

It was originally published in The American Journal of Public Health. I found the article through RedOrbit at
http://www.redorbit.com/news/health/725943/
newborn_screening_complexities_in_universal_genetic_testing/index.html?source=r_health on November 10, 2006. You can read the complete article by following the link. The title of this article is also linked to the RedOrbit article for as a long as it's there.
One of the authors, Nancy Green, is the Medical Director, March of Dimes Birth Defect Foundation. (and I thought the March of Dimes advocated for newborn screening and the health of children; this makes you wonder for whom they really are advocating. I mean what part of "uniformly fatal if left untreated" doesn't she understand?)

"THE FUTURE

Newborn genetic screening has been a remarkable achievement for public health, providing populationwide detection of disorders that leads to improved clinical outcomes. Advances in medical genetics and testing technology permit the diagnosis of ever more diseases but also compel society to reconsider how NBS as a public health measure may best serve children, their families, and their communities. Powerful multiplex test technologies can identify children with anomalies that may-or may not-lead to disease. They can also find children for whom no treatment is yet available. Weighing the costs, risks, and benefits of screening in such cases is complex and will require consideration of the full range of costs and potential nonmedical benefits.33

New knowledge and NBS technologies raise additional challenges. For example, some experts advocate NBS for severe combined immunodeficiency, a uniformly fatal disorder if untreated and for which early diagnosis can lead to lifesaving bone marrow transplantation therapy.36 However, such treatments may not be within the moral compass of public health, as they are associated with risks of morbidity and mortality, expensive, and not universally available to all affected infants. Similar considerations arise with lysosomal storage disorders.37 Policy decisions will become increasingly complex and controversial regarding the use of NBS, understood as a public health measure to identify newborns with treatable diseases, when it is extended to include detecting genes with incomplete penetrance, genes for a given disease that only partially predict that disease, and disorders that cannot be successfully treated or for which treatment is available only to some newborns so identified.

In addition to the traditional single-gene disorders, conditions affected by multiple genes and gene-environment interactions raise additional possibilities for incorporating new disorders into NBS programs. Indeed, NBS for common complex diseases,38 such as asthma, is under consideration,39 with pilot screening for genetic susceptibility to diabetes40 already under way for early institution of prevention strategies for those at risk. These trends will likely push NBS programs and society to discuss the implications of revealing disease susceptibility rather than making a specific diagnosis in the first months of life.41 Some commentators have gone as far as to suggest a broad analysis of individual genomic variation from a NBS sample.42

NBS requires more resources to take advantage of current and future opportunities and challenges, including the creation of national consensus guidelines for screening and follow-up to establish universal minimal screening standards for the United States. Proposals to improve NBS, such as proposed test standards11 and key recommendations on NBS made by the American Academy of Pediatrics6 and the American College of Medical Genetics,8 are already sparking broad debate within the world of public health. These national efforts require sufficient support from the federal health system for translation to state programs and should result in identifying and standardizing best screening policies and practices across the United States; providing adequate resources for programs to incorporate long-term specialty treatment, genetic counseling and referral, and educational outreach to consumers and health providers; and increasing research capacity for well-designed prospective studies on the predictive value and clinical effectiveness of expanded screening and treatment.

As a public health program, NBS has done enormous good. New technologies and new forces are pushing NBS into unfamiliar territory, to which NBS must actively react to set its course for a sound future."

Linkback URL: http://www.redorbit.com/news/health/725943/
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Parents agonize as trip delayed

Parents agonize as trip delayed

Three-month wait for infant son's experimental gene therapy for a rare disease leaves Parker's parents fearful for his health

Nov. 7, 2006. 06:42 AM

TANYA TALAGA

HEALTH REPORTER

Nine-month-old Parker DesLauriers' trip to Italy for life-saving gene therapy has been delayed until January."We want to go as soon as we can," his mother, Tracy DesLauriers, said in an interview last week at the Hospital for Sick Children.Right now would be the optimal time since he is the picture of health. But DesLauriers is resigned to the wait, even if it means enduring the cold and flu season, which could be devastating for her only child."Everything with Parker seems to happen for a reason," his mother said.DesLauriers inherited a rare disease, called ADA-deficient severe combined immunodeficiency (SCID), from his parents, who both carried a copy of a defective gene. Parker's illness, also known as boy-in-the-bubble disease because patients have to be isolated, is so rare that Sick Kids has treated only 15 patients with it in the past two decades.The baby spent most of his first five months at the downtown Toronto hospital, but was allowed to go home to Ajax in June. Tracy and her husband Kevin were hoping to take him to Milan for experimental gene therapy in September, but the clinical trial has been delayed.Parker travels to the hospital twice a week to get an injection of an enzyme that his body can't make. Without the adenosine deaminase (ADA), he is unable to filter out toxins that would kill off his immune system and eventually attack his organs. The injections work for only about two to three years.When Parker goes to the hospital, Tracy puts him in his stroller and fastens a clear plastic weather guard over it to protect him from germs.To look at the baby, you would never guess the smiling, robust boy is seriously ill and must spend most of his days inside the house and out of contact with the general public. A simple cold could jeopardize his health. He's thriving now because of the enzyme injection he receives at the hospital, but the shot he receives in his thigh isn't a cure.In order to help Parker, Italian doctors, led by Dr. Alessandro Aiuti, will remove stem cells from his bone marrow and introduce a normal version of the ADA gene. These will be reinjected and it is hoped they will migrate to the bone marrow and begin making ADA.A cell's genetic material is stored on chromosomes in the nucleus on long molecules of deoxyribonucleic acid or DNA, which contain our genes. The genes dictate an organism's characteristics.Dr. Roderick McInnes, scientific director for the Institute of Genetics at the Canadian Institutes of Health Research, said gene therapy holds much promise for the future. So far, the one place it appears to be working well is in the treatment of ADA-deficient SCID patients. "The Italian team are amongst the world's best gene therapists," he said. Not one of the handful of children treated by the Italian team seems to have any side effects. "It appears to be a safe `cure,'" said McInnes. Since this is a new therapy and the team is dealing with children, no one quite knows if the treatment will last forever or for only five or 10 years. Gene therapy for SCID is also being done in England and Japan, noted Kevin DesLauriers. "We were kind of bummed out when they told us we weren't going until January," he said. "But we can now have Christmas at home, perhaps his first birthday here."Tracy agreed. "When you go through something like this, you learn not to question as much," she said. They've learned to enjoy every moment they have with their son. "We just have to try and keep him healthy."

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Q & A about the FluMist™ Influenza Vaccine

Get your Questions Answered about FluMist™
See our Q & A about the FluMist™ Influenza Vaccine
From the IDF Newsletter, Primary Immune Tribune, Volume 1 Issue 3

Q: What is FluMist™?
A: FluMist™ is a live virus influenza vaccine. It was approved by the U.S. Food and Drug Administration in June 2003 and is the first nasally administered vaccine to be marketed in the United States. FluMist™ is approved to prevent influenza illness due to influenza A and B viruses in healthy children and adolescents, ages 5-17 and healthy adults, aged 18-49. FluMist™ is produced by MedImmune Vaccines and distributed by Wyeth.

Q: How is FluMist™different from the traditional flu shot?
A: FluMist™ is a live virus vaccine and administered through a nasal spray. The flu shot is an inactivated (killed virus) vaccine. Killed virus vaccines can be taken by individuals with primary immune deficiencies and their families.

Q: What is influenza?
A: Influenza, or "the flu", can cause serious respiratory illnesses in normal people. Those with primary immune deficiency diseases may be at an increased risk for the flu and experience more serious complications. There are two types of influenza virus that cause human disease, Type A and Type B. Each year these types undergo changes, which make individuals susceptible to infection even though they may have antibodies to other strains of influenza from prior infections or immunizations.

Q: How is influenza spread and what are its symptoms?
A: Influenza is spread by coughing and sneezing. After infection, there is an incubation period of one to four days. Infected individuals can infect others before symptoms begin and for about five days after symptoms begin. Symptoms include fever, cough, muscle aches, headache, sore throat, runny nose, and fatigue. The cough and fatigue can last a few weeks. Pneumonia and other complications can be very severe.

Q: Who should not receive the FluMist™ vaccine?
A: FluMist™ should not be given for any reason to the following:

* People with primary immune deficiency diseases or their close contacts
* People with weakened immune systems due to HIV infection, certain cancers, or immunosuppressive agents used to treat cancer or organ transplantation
* People with asthma or other reactive airway diseases
* Children younger than 5 years and adults older than 50 years
* People with a history of Guillan-Barre syndrome, chronic diseases of the cardiovascular or pulmonary system, or allergies to eggs
* Pregnant women

Q: What are the potential complications of the FluMist™ vaccine?
A: The most common adverse events associated with the vaccine in normal individuals were nasal congestion, runny nose, sore throat and a cough. Although there is no specific information available, it is anticipated that if a person with a primary immune deficiency receives FluMist™ (s)he would be more likely to develop complications. If a close contact is vaccinated, the resulting viral shedding could cause a person with a primary immune deficiency disease to become infected with the flu vaccine strains. (See A7 below)

Q: What is the risk to individuals with primary immune deficiency disorders if a close contact is vaccinated with FluMist™?
A: During a clinical trial with FluMist™ in a day care center, there was documented transmission from vaccinated children to unvaccinated children. Viral shedding following the administration of FluMist™ typically continues for about a week on the average, but may be as long as three weeks. The risk of transmission in the day care center setting was estimated at 2.4% or one in approximately 42 children. The risk could be higher if different children in the center receive the vaccine at different times over the fall.

Healthcare workers who receive FluMist™ may also present a possible way for a person with a primary immune deficiency disease to become infected with the flu vaccine strains. Although there is no data about transmission of the live vaccine virus from vacinees to immune compromised contacts and subsequent development of disease, the Centers for Disease Control and Prevention have stated that the inactivated vaccine (flu shot) is preferred over live, intranasal influenza vaccine (FluMist™) for physicians, nurses, family members, or anyone else coming in close contact with anyone with a weakened immune system.

Q: Is the killed virus flu vaccine (flu shot) recommended for those with primary immune deficiencies or their close contacts?
A: People with primary immune deficiency diseases may choose to receive the inactivated influenza vaccine shot. This is the killed version of the vaccine and will not cause the flu! The only risks of receiving the shot are soreness at the injection site, and less often fever, tiredness, muscle aches, and headache. Those allergic to eggs should not receive the vaccine shot, as there is a risk for more serious reactions. Even if you do not develop antibody titers high enough to prevent influenza, you still might benefit from receiving the shot every year. Receiving the shot may reduce your risk for hospitalization, pneumonia, and other complications. Your family members should seriously consider receiving the killed version of the flu vaccine to reduce the risk of bringing the flu virus home to their family member with primary immune deficiency.

Q: How can you reduce your risk of complications from the FluMist™ vaccine?
A: To reduce the risk of contracting the flu vaccine strains, the Immune Deficiency Foundation's Medical Advisory Committee has made the following recommendations:

* Because it is a live virus vaccine, people with primary immune deficiency diseases should NOT receive the FluMist™ vaccine.
* The FluMist™ vaccine is not recommended for close contacts of primary immune deficient patients.
* Primary immune deficient patients should talk to their doctors to see if it may be advisable to receive preventive medicine to avoid becoming infected with the FluMist™ strains of the flu.
* Primary immune deficient patients exposed through close contact to FluMist™, should see their doctor immediately, as (s)he may advise a treatment medicine.
* School authorities may want to advise their immune deficient pupils if FluMist™ is being administered in the school system. This information may be especially useful to those with T cell or combined T and B cell immune deficiencies.
* Family members and healthcare workers in close contact with immune deficient patients should be advised to receive the killed virus flu shot, rather than the FluMist™ vaccine.

Q: How do you evaluate the risk of FluMist™ vaccine complications for you or your family member?
A: The FluMist™ vaccine is not recommended for individuals with primary immune deficiency disorders and/or any of the above listed medical conditions (A5). It is best to consult with your physician when evaluating the risk of complications for you or your family members.

Q: Is treatment available for individuals who develop complications from the FluMist™ vaccine?
A: One antiviral agent, Tamiflu (Roche) has been approved by the FDA for prevention of Types A and B Influenza in those 13 years of age and older. It is given once daily for up to 42 days and may have benefit if given within 2 days of exposure to influenza strains. However, there are no studies of the use of Tamiflu in primary immune deficient patients exposed to FluMist™ influenza strains. If exposed to FluMist™ strains of influenza, a primary immune deficient patient should contact his or her physician immediately.

Q: How can I find out if FluMist™ is being given in my local community?
A: For information about FluMist™, contact your state or local health department and/or school system.

Q: Where can I find more information?
A: Please utilize the following websites:

* Centers for Disease Control and Prevention, National Immunization Program: www.cdc.gov/nip.
* Immune Deficiency Foundation: www.primaryimmune.org
* Clinical Focus on Primary Immune Deficiency Diseases: Immunization of the Immunocompromised Host: www.primaryimmune.org . Go to "Publications" and then to "Clinical Focus". It is the October 1998 issue.
* U.S. Food and Drug Administration, Center for Biologics Evaluation and Research: www.fda.gov/cber.

Original Article from the IDF Newsletter:
Linkback URL:
http://www.imakenews.com/idf/e_article000704177.cfm?x=b8y4wp9,b5TSrMPk,w

IDF Survey Helps Persuade CMS to Temporarily Stop Reductions for IVIG Medicare Payments

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FOR
IMMEDIATE
RELEASE
November 1, 2006
IDF Survey Helps Persuade CMS to Temporarily Stop Reductions for
IVIG Medicare Payments
TOWSON, MDThe Immune Deficiency Foundation (IDF) patient survey helped persuade the Centers for Medicare
and Medicaid Services (CMS) to issue final rules that grant a critical reprieve for patients with primary immune
deficiency diseases (PIDD). On November 1, CMS issued a ruling that will continue the temporary
"preadministration-related services" add-on for IVIG in 2007 for both physician services and hospital outpatient
department (OPD) reimbursements for IVIG. CMS had established this additional payment for IVIG in 2006 to
compensate physicians and hospital OPDs for extra resources needed for locating and obtaining appropriate IVIG
products. It was also used for scheduling patient infusions during a period "where there may be temporary instability."
CMS also withdrew another separate proposal to reduce Medicare's payment for IVIG in the hospital OPD setting.

In its final rules, CMS cited the IDF patient survey as offering evidence of the hardships that many PIDD patients have
had to deal with because of reductions in Medicare payments for IVIG. Some of the findings from the survey include
patients reporting that they have had to change the site of their infusions, postpone infusions, increase intervals
between infusions, and reduce dosage of IVIG as the result of changes in Medicare reimbursement methodology for
IVIG.

IDF wishes to thank the over one thousand PIDD patients who took the time to respond to our survey this year. We
believe that the survey's results made the difference in providing hard, empirical data that CMS needed to withdraw its
proposed reductions. IDF also acknowledges the hundreds of patients and family members from the PIDD community
who have contacted their Members of Congress about this critical issue through our online advocacy program, Action
Alert. These personal contacts and our patient survey demonstrate that an individual's input can make a difference on
policy making in Washington.

While this reprieve from reductions is vital to patients with PIDD, IDF does not believe that it goes far enough to bring
stability for Medicare beneficiaries. We are taking the same patient survey findings to Congressional Committees and
Members of Congress to show them the kinds of dislocations Medicare IVIG users are experiencing as a result of
changes in reimbursement policies enacted in 2003. We believe that a permanent adjustment needs to be made in the
way Medicare pays for IVIG to assure access to care and choice of provider. Congress may need to take legislative
action to make sure this happens.

We celebrate this temporary reprieve for the PIDD community, but recognize that it is not a time to become
complacent. IDF urges all of you to again visit our Web site and make your voice heard. Log on to IDF Action Alert
at
www.primaryimmune.org
to contact your Members of Congress and let them know how important access to IVIG is
to your health.
# # #

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Ex vivo gene therapy with lentiviral vectors

GENE THERAPY
Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)–deficient mice and corrects their immune and metabolic defects
Alessandra Mortellaro, Raisa Jofra Hernandez, Matteo M. Guerrini, Filippo Carlucci, Antonella Tabucchi, Maurilio Ponzoni, Francesca Sanvito, Claudio Doglioni, Clelia Di Serio, Luca Biasco, Antonia Follenzi, Luigi Naldini, Claudio Bordignon, Maria Grazia Roncarolo, and Alessandro Aiuti

From the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), Milan, Italy; Department of Medicina Interna, Scienze Endocrino Metaboliche e Biochimica, University of Siena, Italy; Pathology Unit, Department of Oncology, San Raffaele H Scientific Institute, Milan, Italy; University Statistics Centre for Biomedical Sciences, Università Vita-Salute San Raffaele, Milan, Italy; and Università Vita-Salute San Raffaele, Milan, Italy.

Adenosine deaminase (ADA) deficiency is caused by a purine metabolic dysfunction, leading to severe combined immunodeficiency (SCID) and multiple organ damage. To investigate the efficacy of ex vivo gene therapy with self-inactivating lentiviral vectors (LVs) in correcting this complex phenotype, we used an ADA–/– mouse model characterized by early postnatal lethality. LV-mediated ADA gene transfer into bone marrow cells combined with low-dose irradiation rescued mice from lethality and restored their growth, as did transplantation of wild-type bone marrow. Mixed chimerism with multilineage engraftment of transduced cells was detected in the long term in animals that underwent transplantation. ADA activity was normalized in lymphocytes and partially corrected in red blood cells (RBCs), resulting in full metabolic detoxification and prevention of severe pulmonary insufficiency. Moreover, gene therapy restored normal lymphoid differentiation and immune functions, including antigen-specific antibody production. Similar degrees of detoxification and immune reconstitution were obtained in mice treated early after birth or after 1 month of enzyme-replacement therapy, mimicking 2 potential applications for ADA-SCID. Overall, this study demonstrates the efficacy of LV gene transfer in correcting both the immunological and metabolic phenotypes of ADA-SCID and supports the future clinical use of this approach.

Prepublished online as a Blood First Edition Paper on July 11, 2006; DOI 10.1182/blood-2006-05-023507.

Linkback URL: http://www.bloodjournal.org/cgi/content/short/108/9/2979?rss=1