IDF Center of Excellence Dedicated at Duke University

IDF Center of Excellence Dedicated at Duke University

Oct 28 2006, 12:00 PM EST

BIOWIRE

A special dedication ceremony was held today at Duke University Medical Center for the opening of the Duke IDF Center of Excellence for Primary Immunodeficiency Diseases. The Immune Deficiency Foundation (IDF), through a generous grant from Talecris Biotherapeutics totaling more than $125,000 over two years, has collaborated with Duke to make this center available to patients and physicians with the goal of increasing early diagnosis and improving treatment and care for patients with primary immunodeficiency diseases (PID). The center, housed in the Duke University Medical Center, includes some of the world's leading experts and state-of-the-art facilities focusing on the research and clinical care of PID.

"Through the IDF Center of Excellence, we will continue to pursue our goals to offer the best available options for rapid, accurate diagnosis of primary immunodeficiency diseases, to define the molecular causes, and to ensure the optimum and most effective treatments for those patients," said Rebecca H. Buckley, M.D., center director and the J. Buren Sidbury Professor of Pediatrics and Professor of Immunology at Duke University Medical Center. "Diagnosis of a primary immunodeficiency often takes as long as nine years, primarily because of a lack of familiarity with these diseases by both health care providers and patients. Working collaboratively with IDF and Talecris, we believe this Center of Excellence will go a long way toward improving awareness of PID among referring physicians, speeding diagnosis, and providing the optimal therapy more quickly for our patients."

Along with Dr. Buckley, the following representatives of IDF, Duke, and Talecris participated in the dedication ceremony and presented their views on the importance of the new center and the opportunities it presents.

-- R. Michael Blaese, M.D., Medical Director of the Immune Deficiency Foundation

-- A. Wesley Burks, M.D., Chief, Division of Pediatric Allergy and Immunology, Duke University Medical Center

-- Joseph W. St. Geme, III, M.D., Chair, Department of Pediatrics, Duke University Medical Center

-- Ross McKinney, M.D., Vice Dean for Research, Duke University School of Medicine

-- Alberto Martinez, M.D., President and CEO of Talecris Biotherapeutics

Additionally, two patients, H. Burns Blackwell and Nicholas Schwab, who will be beneficiaries of the new center, shared their views on how the center will support efforts for those with immunodeficiencies to receive the best possible care and optimize opportunities for improved quality of life.

The new IDF Center of Excellence at Duke includes a staff of more than 21 physician-scientists, nurses, post-doctoral fellows, technicians, and other health care professionals who focus on advancing the diagnosis and therapy of PID. Distinguishing features of the center include:

-- having the largest number of faculty in the U.S. with expertise in primary immunodeficiency diseases.

-- developing sophisticated diagnostic testing utilized by many outside physicians.

-- being the largest center in the United States performing life-saving T cell-depleted haploidentical bone marrow stem cell transplants for infants with severe combined immunodeficiency (SCID).

-- being the largest center in the world performing life-saving thymic transplants for infants with complete DiGeorge syndrome.

-- treating a large number of patients with antibody deficiency with intravenous or subcutaneous immunoglobulin.

Dr. Blaese spoke about achieving IDF's continuing goals through Centers of Excellence. "This IDF Center of Excellence is an especially gratifying collaboration that brings together the strengths and commitments of IDF with those of a leading academic institution providing sophisticated diagnostic testing and therapy. Whenever we see programs that expand and improve care for the primary immunodeficient community, IDF will be there doing everything it can to facilitate the process. We are extremely grateful to Duke University Medical Center and Talecris."

Dr. Martinez represented Talecris' views on how industry should support these kinds of partnerships. "Clearly, the most important thing we can do to support the primary immunodeficient community is to provide safe, reliable supplies of therapies that effectively treat PID. But we also know we can broaden our ability to improve patient outcomes when we partner effectively with patient organizations like IDF and academic institutions like Duke."

The Role of a "Center of Excellence"

Through basic research into pathophysiological mechanisms, and clinical studies whereby new findings can be tested, Duke immunologists will continue to work to improve diagnosis and treatment and to provide patients and their families the best available information about primary immunodeficiency diseases. A Center of Excellence goes beyond what is available at the average medical facility. Because such centers offer advanced tests and procedures not yet incorporated into accepted standards of care and therefore not reimbursed by insurance companies, the cost of performing these advanced tests and procedures must be covered by other funds - funds often difficult to secure. A portion of the Talecris grant through IDF will be applied by the Duke center to support these advanced procedures with the goal of providing additional evidence needed to incorporate these tests and procedures into new standards of care.

About Duke University Medical Center

Duke University Medical Center is consistently ranked among the top ten health care organizations in the country. The youngest of the nation's leading medical centers, Duke has earned an international reputation for innovation and excellence. With hundreds of board-certified specialists and subspecialists, Duke offers expertise in every health care discipline. Duke also operates one of the country's largest clinical and biomedical research enterprises, and quickly translates advances in technology and medical knowledge into improved patient care.

About the Immune Deficiency Foundation

The Immune Deficiency Foundation (IDF), founded in 1980, is the national nonprofit health organization dedicated to improving the diagnosis and treatment of primary immunodeficiency diseases through research, education and advocacy. There are more than 250,000 people in the United States who have a primary immunodeficiency disease. These individuals often find it difficult to receive specialized healthcare, proper diagnosis and effective treatment. Individuals affected by primary immunodeficiency diseases also experience difficulties financing their healthcare, finding educational materials on the disease, and locating others with whom to share their experiences. The goal of IDF is to help individuals overcome these difficulties so that they can live healthy and productive lives. For more information about the IDF, visit www.primaryimmune.org or call IDF at 1-800-296-4433. To be included in the distribution list of Primary Immune Tribune, please send a request to enewsletter@primaryimmune.org.

About Talecris Biotherapeutics

Talecris Biotherapeutics is a global biotherapeutic and biotechnology company that discovers, develops and produces critical care treatments for people with life-threatening disorders in a variety of therapeutic areas including immunology, pulmonology, and hemostasis. Talecris is proudly building upon a 60-year legacy of innovation and a commitment to improving the lives of people who rely on its therapeutic products. With an emphasis on scientific inquiry and technological excellence, Talecris is expanding its current portfolio of products, programs, and services through its own world-class product development organization as well as through strategic initiatives that leverage its strengths with those of its partners.

Talecris, which earned revenues exceeding $1 billion through the year ending June 30, 2006, is headquartered in the biotech hub Research Triangle Park, N.C., and employs more than 2,000 talented people worldwide. To learn more about Talecris and how our employees are making a difference in the lives of patients and the healthcare community, visit www.talecris.com.

Linkback URL: http://www.genengnews.com/news/bnitem.aspx?name=7697642
and http://primaryimmune.org

East Texas Baby Diagnosed With SCIDs

10/19/06-Tyler
East Texas Baby Diagnosed With SCIDs

An East Texas baby has been fighting for his life after being diagnosed with a rare disease. It's called Severe Combined Immunodeficiency also known as SCIDs. What should be a time of joy for one local family, bringing their son home from the hospital is becoming quite the challenge.

"He is beautiful, he is my little miracle baby. There were times that the doctors' couldn't tell me if he was going to make it," says Rachel Moreno, mother of one year old Diego.

Diego, was diagnosed with SCIDs. A disease that makes it difficult for his immune system to fight infection. For months the Moreno's thought they had a healthy baby.

"They told me only one in a hundred thousand kids have that. And I said why couldn't I have hit the lotto? Why did we have to have this?" asks Rachel.

Rachel works full time, in order to have health insurance. While, her husband stays at Children's Medical Center in Dallas with Diego. In July, Rachel donated her bone marrow to Diego and now he's getting stronger.

"They told me two weeks ago his cells have grown from 5% to 32% and now at present 46%," says Rachel.

Some other good news, doctors told Rachel and her husband they need to start preparing for Diego to come home, which means making the house completely germ free.

"The carpet holds dust and they don't want any carpet or curtains or all of that stuff that hold dust, particles or infections," says Rachel.

"We had some people evaluate our house because it will have to meet home inspection. He has to have a special ventilation system like a clean air type filtration system," says Rachel.

But since Rachel and her family live in a mobile home, the ventilation system is not feasible.

"We are working on trying to get a house built for him that will be environmentally safe for him and germ free. So we can have him home and once we get him home we can keep him home, which is going to be a big challenge," says Rachel.

Something Rachel says her faith will help them through.

Rachel says in addition to trying to build an environmentally safe home for Diego, they also have several million dollars in medical bills.

If you would like to help the Moreno family, the Diego Tragedy Fund has been set up at Southside Bank locations in Tyler.

Karolyn Davis, reporting. kdavis@kltv.com
linkback url: http://www.kltv.com/global/story.asp?s=5563447

Family To Leave Canada To Help Boy With Rare Immune Disease

Family To Leave Canada To Help Boy With Rare Immune Disease
Tuesday October 10, 2006
Little Parker DesLauriers sits playing easily in his room, blissfully unaware of the world around
him.

That's something his parents can't afford to do.

The child entered the world in January, but his life has been anything but normal. He was born with a condition known as SCID, or Severe Combined Immunodeficiency, a genetic disorder that leaves him unable to fight off any infection.

He's been forced to exist in a sparkling clean environment as his folks look for a way to save his life and restore his world and theirs to some sense of normalcy.

"He has the most severe form of Severe Combined Immunodeficiency, because it's not just an immune problem, it's a metabolic problem that causes damage to his other organs and tissues," explains his father, Kevin.

Now the family has been forced to make an agonizing choice - literally go for broke to travel to Milan, Italy for experimental therapy that could cure him or stay here and wait for a miracle.

In this case, the choice is no choice, and the couple has already booked their plane tickets. But it comes with a stiff price.

The two teachers have both taken leaves of absence from their jobs and been forced to dip into their savings to afford a trip and a stay that doesn't have an apparent end in sight.

"If untreated, he wouldn't live probably past two," his mother Traci relates about what doctors have told her.

While Parker's life is about to undergo a sea change, it's already been anything but normal.

The charming little boy lives in a dirt free environment, with his mother completely cleaning the house from top to bottom every single day.

He can go out of the house, but only with the protection of a plastic shield that prevents him from feeling the touch so many other youngsters crave and take for granted.

"His aunts and uncles have never held him," laments Kevin as he contemplates the coming journey.

His family is hopeful donations will help aid them in their desperate search for a cure. But whether they get them or not, their plan to head to an unknown land is a done deal.

How do they keep going on? "We get a lot of strength from family and friends," Kevin admits. "We also get a lot of strength from Parker."

If you'd like to help the family in their quest for a cure, click here.

Linkback URL: http://www.citynews.ca/news/features_4272.aspx?&categoryurl=http://www.citynews.ca/news
/features_3736.aspx

IMMUNE DEFICIENCIES LEAVE BABY DEFENSELESS

IMMUNE DEFICIENCIES LEAVE BABY DEFENSELESS
By RHIANNON MEYERS, Staff Writer

(Courtesy Photos)
Diego Moreno's baby pictures are almost unbelievable.

Photos of the smiling newborn show he was well on his way to becoming an early crawler.

A year later, pictures of the Tyler toddler are taken only in his 12th-floor hospital room in the intensive care unit at Children's Medical Center of Dallas.

His first birthday will arrive Tuesday to find him hooked to life-support machines.

"It's very painful looking at him from the way he was from the get-go," said Diego's 28-year-old mother, Rachel. "I have pictures that would just freak you out. He was trying to crawl at four months. He was alert, looking around, looking at colors, smiling, 'goo-ing' and 'gah-ing.' Now, you can't hear him make any noise."

Diego has SCID (severe combined immunodeficiency). Most people were made aware of this disease by David Vetter, who lived for 12 years in a plastic bubble.

SCID is a group of rare immune deficiencies that leaves a child unable to fight infections, according to information from Children's Hospital in Boston. Most children are diagnosed only after they become ill with pneumonia, meningitis and chicken pox. Many die in their first year of life, before SCID can even be diagnosed.

For those who are able to survive, life is tough. Normal environments, such as schools, movie theaters and grocery stores, can prove to be fatal.

Diego will never be able to fight off infection from viruses, bacteria and fungi. So, although his release from Children's Medical Center is several months away, Rachel is already planning for the future of her son, who can never be exposed to germs.

DIAGNOSIS

The pages and pages of photographs in Rachel Moreno's album document Diego's quick deterioration.

"I brought pictures (to Diego's doctors). They couldn't believe it. Look, look at my baby. He was so happy. He was crawling. And when he was first admitted, he looked like he was dead. He wasn't even moving because there was so much stuff attacking his system," Rachel said.

At four months, Diego developed cold and flu symptoms and recurring ear infections. At six months, he was flown to a hospital when Rachel noticed he was panting heavily. Doctors at Children's Medical Center diagnosed him with pneumonia, gave him breathing treatments and sent him home.

Two and a half weeks later, Rachel called 911 because Diego stopped breathing. The doctors at the Dallas hospital did special blood work, ran all sorts of tests and sent him through a magnetic resonance imaging machine, Rachel said.

At first, doctors weren't sure what to believe, she said. They found that Diego's lymphocytes were too low, which could have indicated cancer. They discovered that Diego had cerebral atrophy (his brain was regressing) which could have indicated abuse. Rachel said some questioned whether Diego had been dropped or mistreated.

"I was very angry and hurt by that ... We love our baby. If I heard him cry five times in my life, I'd be surprised," she said.

When the SCID diagnosis came, Rachel and her husband Luis were shocked.

Approximately one in 100,000 children is born with the inherited disease, according to hospital information. Rachel points out that she had a better chance of winning the lottery.

Even though the diagnosis came early enough to save Diego's life, the viruses that attacked his system had already taken their toll. His ventilator and feeding tube will probably be permanent. The virus that diminished his brain could prevent him from ever walking or talking again, Rachel said.

For 72 days, Luis and Rachel couldn't hold him. Even now, they must wear gloves, masks and gowns before touching their baby so they don't infect him.

"It started with a cold and now it's this," she said.

IMPROVING

For a long time, neither Rachel nor Luis wanted children.

Rachel was 14 when she had her first child, Jordan Jett. Back then, she mowed lawns and cleaned houses to survive.

Years later, Rachel met Luis in Tyler. They married, bought a home and waited seven years before Diego was born.

In the Moreno family photo album, there are photographs of a very pregnant Rachel at a baby shower thrown by her co-workers at The University of Texas Health Center at Tyler. She laughs and compares bellies with a male coworker.

"I was so happy. I was like a queen the whole time," she said.

A year later, Rachel is happy for much different reasons.

Diego is still on life support, but seems to be improving. Rachel and Luis met with doctors on Friday who told them that Diego may soon be moved out of the ICU to another floor. And from there, they will begin preparations to bring Diego home.

"That's a huge step," Rachel said.

Part of Diego's improvement is due to a bone marrow transplant from Rachel. Transplants of stem cells are now considered the best treatment for SCID, according to Children's Hospital Boston. Stem cells develop into red blood cells to carry oxygen, white blood cells to fight disease and infection and platelets to aid in blood clotting. Bone marrow transplants help children with SCID rebuild their immune systems.

Rachel said her donation of bone marrow was painful and scary, but she would have done it a million times over to help Diego.

FUTURE

Rachel said Diego's prognosis is good and she expects him to live a long time. The next step in her life is to begin preparations to bring Diego home in the next three to six months. She plans to home-school him.

The Morenos' home is not equipped to maintain a germ-free environment, she said. A germ-free house must be free of anything that collects dust, including carpet, plants and animals. The house must have a filtration system equipped for an immune-compromised child, and enough outlets to support Diego's ventilator and feeding machines.

Her home must also be wheelchair accessible, she said. Rachel said that she believes it would be impossible to renovate her mobile home to fit Diego's requirements - and that she needs to build a house.

Diego's illness has left the Moreno family financially strapped, Rachel said, with medical bills totaling $3 million. Luis quit his job to stay with Diego 24/7. Rachel still works at UTHCT so she can keep her insurance, but she travels back and forth to Dallas each week.

Rachel said that anyone wishing to help can contact her on her cell phone, 903-570-2814; or call her stepmother, Tammy Bratusek, at 903-858-1506 or her mother, Sherry Gilliam, at 903-526-3332.

A fund has been set up in Diego's name at all Southside Bank branches in Tyler.

Linkback URL: http://www.tylerpaper.com/site/news.cfm?newsid=17299428&BRD=1994&PAG=461&dept_
id=226369&rfi=6

‘Help save my son’

‘Help save my son’

The mother of a five-year-old boy who will die unless he has a bone marrow transplant has issued a plea to readers of the Borehamwood & Elstree Times.

Tommy Cash-Hutchin, of St Paul's Close, Boreham- wood, has a rare condition which means his immune system cannot fight even the smallest of infections. He needs a bone marrow transplant is he is to lead a normal life and survive into adulthood.

But there are currently no matches for him on the donor register, so his only hope is for a suitable donor to come forward soon.

His mother, Kelly Hutchin, 23, discovered Tommy had severe combined immunodeficiency (SCID) when he was three months old after he suffered from repeated coughs, colds and pneumonia.

The same condition, which is hereditary, killed Tommy's five-year-old cousin.

Miss Hutchin said: "You wouldn't think Tommy's got anything wrong with him to look at him, but if he doesn't have a bone marrow transplant, he won't live.

"The doctors want to do it as soon as possible because it's easier when they're younger."

The pair travel to Great Ormond Street Hospital every Wednesday for life-saving treatment to ward off infections by giving him essential white blood cells.

Miss Hutchin added: "He's started asking me questions about why he has to go to hospital and his cousin doesn't. I try to explain to him that he hasn't got any white blood cells, but he's still a bit too young. We need more people to come forward to be donors - not just for Tommy, there are other people as well who need it."

News of Tommy's plight has spread and a group of fundraisers who work at Cardif Pinnacle in Borehamwood have set about raising enough money to send him and his mum on a holiday.

Heather Walsh, who works at The Community Shop in Leeming Road, met Tommy and his mother at a young parenting group. She plans to put on an awareness-raising event after Christmas in conjunction with the Anthony Nolan Trust, which will provide blood-testing equipment for people to join their donor register. Mrs Walsh said: "I think Kelly does a tremendous job."

* To sign up to the Anthony Nolan Trust register of potential bone marrow donors, you need to be aged between 18 and 40. The charity particularly needs young men to join. Call the trust on 020 7284 1234 or visit www.anthonynolan.org.uk

6:18pm Thursday 5th October 2006

By Emma Hutchings

Linkback URL: http://www.thisishertfordshire.co.uk/news/borehamwood/
display.var.955970.0.help_save_my_son.php%20