Corinth Boy Suffering From Rare Disease Gets Lung Transplant

Corinth Boy Suffering From Rare Disease Gets Lung Transplant

Web Editor: Rhonda Erskine, Online Content Producer
Created: 5/14/2008 6:13:58 PM
Updated: 5/15/2008 11:25:28 AM

CORINTH (NEWS CENTER) — Last fall, we brought you the story about a 10-year-old boy from Corinth who needed a lung transplant. Wednesday, Zachary Nickerson returned home after getting the procedure he desperately needed.

Zachary has SCIDS, Severe Combined Immuno-Defficiency Syndrome. It is often called “bubble boy disease.”

Zachary’s immune system is so weak that even the slightest cold can land him in the the hospital.

Zachary got a special ride home from the Corinth Fire Department Wednesday. An excited group of friends and family were there to greet him.

“This is pretty awesome,” said Zachary’s mother, Michele Nickerson. “Zachary is so excited. He’s been anxious to get home. There’s been times in that hospital when he would wonder if he would ever come home.”

Zachary has lived three years of his life in the hospital and has had more than two dozen operations, including two bone marrow transplants. Despite his problems, his family says he has an amazingly positive attitude.

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IDF Applauds Legislation to Improve Patient Access to Life Saving IVIG Treatment

 

IDF Applauds Legislation to Improve Patient Access to

Life Saving IVIG Treatment

{WASHINGTON, D.C., May 8, 2008} – The Immune Deficiency Foundation (IDF) applauds Senators John Kerry (D-Mass.), Lamar Alexander (R-Tenn.) and Debbie Stabenow (D-Mich.) for introducing S. 2990, “The Medicare IVIG Access Act.” The bill seeks adequate reimbursement for Intravenous Immunoglobulin (IVIG) therapy and accompanying care in the management of patients with primary immunodeficiency diseases (PIDD) and other serious conditions that require IVIG therapy.

“For our patients with primary immunodeficiency diseases who need antibody replacement, IVIG is their only treatment option,” said Marcia Boyle, IDF President and Founder. “Senators Kerry, Alexander and Stabenow have stepped forward to ensure that individuals whose lives depend on access to IVIG will not be put at risk.”

Presently, Medicare beneficiaries who require IVIG are experiencing serious access problems – an unintended result of the way Medicare determines the payment for IVIG. Many doctors and hospitals cannot purchase the products at Medicare reimbursed rates, and as a result, some patients are not getting this life saving treatment.

“The Medicare IVIG Access Act” would fix these current problems by ensuring adequate reimbursement for IVIG. It also allows for home treatments for Medicare beneficiaries with PIDD by covering related items and services excluded from the current home infusion benefit. These changes will allow these patients to lead healthy and productive lives.

Multiple patient, professional and industry organizations have joined IDF in support of this legislation.

About Primary Immunodeficiency Diseases

Primary immunodeficiency diseases are a group of over 150 rare disorders in which part of the body’s immune system is missing or does not function properly. In contrast to secondary immune deficiency disease in which the immune system is compromised by factors outside the immune system, PIDD are caused by intrinsic or genetic defects in the immune system. Approximately 250,000 people in the United States are diagnosed with PIDD and between 35,000 and 50,000 of them depend on IVIG as their life-saving treatment. Untreated primary immunodeficiencies may be characterized by frequent life-threatening infections and debilitating illnesses.

About The Immune Deficiency Foundation

The Immune Deficiency Foundation (IDF), founded in 1980, is the national non-profit patient organization dedicated to improving the diagnosis, treatment and quality of life of persons with primary immunodeficiency diseases through advocacy, education and research.

To learn more about IDF visit www.primaryimmune.org

 

 

 

SCID Conference Registration now open!

Registration for ”SCID Conference 2008″ at Wintergreen Resort in Wintergreen, VA has started. Please register now by clicking the down loadable registration form at: http://www.primaryimmune.org/pubs/SCID_Conf_2008_registration.pdf

OR credit card users can go to the link at: http://www.primaryimmune.org/conferences/scid_conference_2008.htm

Remember, the registration fee is $250 per room (each room will accommodate approximately 2 adults and 2 children).

The registration deadline is June 3, 2008 so REGISTER NOW! Space is limited and will be assigned on a first-come, first-served basis.

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How does gene therapy work?

May 13, 2008

Arthur Nienhuis, a hematologist at St. Jude Children’s Research Hospital in Memphis, Tenn., and president of the American Society of Gene Therapy, responds:

Gene therapy is the addition of new genes to a patient’s cells to replace missing or malfunctioning genes. Researchers typically do this using a virus to carry the genetic cargo into cells, because that’s what viruses evolved to do with their own genetic material.

The treatment, which was first tested in humans in 1990, can be performed inside or outside of the body. When it’s done inside the body, doctors may inject the virus carrying the gene in question directly into the part of the body that has defective cells. This is useful when only certain populations of cells need to be “fixed.” For example, researchers are using it to try to treat Parkinson’s disease, because only part of the brain must be targeted. This approach is also being used to treat eye diseases and hemophilia, an inherited disease that leads to a high risk for excess bleeding, even from minor cuts.

Early in-the-body gene therapies used a virus called adenovirus—the virus behind the common cold—but the agent can cause an immune response from the body, putting a patient at risk of further illness. Today, researchers use a virus called adeno-associated virus, which is not known to cause any disease in humans. In nature, this agent needs to hitch a ride with an adenovirus, because it lacks the genes required to spread itself on its own. To produce an adeno-associated virus that can carry a therapeutic gene and live on its own, researchers add innocuous DNA from adenovirus during preparation.

In-the-body gene therapies often take advantage of the natural tendency of viruses to infect certain organs. Adeno-associated virus, for example, goes straight for the liver when it is injected into the bloodstream. Because blood-clotting factors can be added to the blood in the liver, this virus is used in gene therapies to treat hemophilia.

In out-of-the-body gene therapy, researchers take blood or bone marrow from a patient and separate out immature cells. They then add a gene to those cells and inject them into the bloodstream of the patient; the cells travel to the bone marrow, mature and multiply rapidly, eventually replacing all of the defective cells. Doctors are working on the ability to do out-of-the-body gene therapy to replace all of a patient’s bone marrow or the entire blood system, as would be useful in sickle-cell anemia—in which red blood cells are shaped like crescents, causing them to block the flow of blood.

Out-of-the-body gene therapy has already been used to treat severe combined immunodeficiency—also referred to as SCID or boy-in-the-bubble syndrome—where patients are unable to fight infection and die in childhood. In this type of gene therapy, scientists use retroviruses, of which HIV is an example. These agents are extremely good at inserting their genes into the DNA of host cells. More than 30 patients have been treated for SCID, and more than 90 percent of those children have been cured of their disorder—an improvement over the 50 percent chance of recovery offered by bone marrow transplants.

A risk involved with retroviruses is that they may stitch their gene anywhere into DNA, disrupting other genes and causing leukemia. Unfortunately, five of the 30 children treated for SCID have experienced this complication; four of those five, however, have beaten the cancer. Researchers are now designing delivery systems that will carry a much lower risk of causing this condition.

Although there are currently no gene therapy products on the market in the U.S., recent studies in both Parkinson’s disease and Leber congenital amaurosis, a rare form of blindness, have returned very promising results. If these results are borne out, there could be literally hundreds of diseases treated with this approach.

link back url: http://www.sciam.com/article.cfm?id=experts-gene-therapy

Bone marrow drive could save 7-month-old infant’s life

Linda Russell, KY3 News

Story Published: May 5, 2008 at 5:47 PM CDT

Story Updated: May 6, 2008 at 10:44 PM CDT

By Michelle Leroux

Video

Hundreds turned out to help an Ozarks infant diagnosed with what’s often called “bubble boy disease.” Seven month old Granton Bayless of Bolivar suffers from a rare condition called Severe Combined Immuno-deficiency, or SCID. A cure may depend on a stranger.

Granton Bayless was a healthy, bouncing, baby boy- until a couple months ago, when he started to get sick. First, Granton was diagnosed with pneumonia and the dangerous respiratory virus- RSV.
“They couldn’t figure out why he wasn’t getting better,” explains Granton’s grandpa, Todd Schrader.

Granton spent a few days at St. John’s in Springfield, before he was transferred to Children’s Mercy in Kansas City. Finally, doctors discovered he has the rare condition- Severe Combined Immunodeficiency (SCID). “His body won’t produce the T-cells, which are part of your white blood cell count, and he can’t fight off disease,” Schrader explains.

The only known cures for SCID are a cord blood or bone marrow transplant. Neither of Granton’s parents, Daniel or Jenni, are a close enough marrow match. So friends organized a bone marrow drive at Bolivar High School. ” We ordered 1000 kits, and we hope we’re gonna run out of them,” says drive organizer, Clayton McCullah.

To get screened at the bone marrow drive, people simply fill out an information form and swab their mouth, but if they’re a match, the donation process isn’t quite as easy. If someone is a match, there are two ways to donate- one is similar to donating plasma, drawing blood from the arm, which goes though a machine that strains out all the needed cells. The other is more invasive. A hollow needle is used to take liquid bone marrow from the pelvis bones.

“I’ve heard it’s painful, but if I can save someone’s life, it’s worth it,” says Jennifer Whitlock, who came to get screened. “They may not be an exact match for Granton, but there are so many other people that’ll be helped through this process,” says McCullah.

But family and friends are holding out hope that Granton will find his cure. “I keep picturing him in the back yard swinging a baseball bat, so we just gotta have faith, gotta believe- he’s a fighter,” says Schrader.

To see how Granton’s doing, go to http://www.grantonbayless.blogspot.com/

Granton’s family members have also organized five other bone marrow drives in neighboring states. The Bolivar High School drive goes until 7:00 PM Monday. If you missed your chance and would like to become part of the bone marrow registry, go to the link below to have a kit mailed to you.

For more information, visit National Marrow Donor Program.

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Extraordinary outpouring for baby with rare condition

Posted on Thu, May. 08, 2008

By DEBRA SKODACK
The Kansas City Star

Daniel and Jennifer Bayless always knew there were great things about living in a small town such as Bolivar, Mo.

Everyone knows you at the grocery. They wave when they see your truck drive through town. They pack the high school stadium on autumn Friday nights to root on the football team.

“And they take care of their own,” Daniel Bayless said Thursday.

And for this young couple, there is no more important time to care than now.

The Baylesses’ 7-month-old son, Granton Lee, is in Children Mercy’s Hospital’s Pediatric Intensive Care Unit. He has a rare condition — severe combined immunodeficiency. His body isn’t producing enough of the T-cells needed to fight off disease. The cure is either a bone-marrow or a cord-blood transplant.

On Monday, three towns with ties to the Bayless family — Bolivar; Dearing, Kan.; and Yukon, Okla. — held bone-marrow registry drives in hopes of providing a match for Granton or some of the other estimated 6,000 people across the country awaiting transplants.

Across the country, 12,000 such drives are held each year, with an average of from 30 to 40 people turning out to be registered.

Monday’s drives for Granton registered 1,792 people.

“The number of people being recruited at Granton’s drives makes them among the largest that we’ve seen as an organization,” said Steve Lovelace, director of recruitment and community development for the National Marrow Donor Program in Minneapolis.

“These recruitment numbers show an extraordinary outpouring of community support, not only for the Bayless family, but also for thousands of searching patients across the country.”

Donnie Bigby is registering himself Tuesday in Miami, Okla., where he works with one of Daniel Bayless’ sisters at Northeastern Oklahoma A&M College.

“I am scared to death,” Bigby said.

He has no fondness for the needles that would come into play if his bone marrow were a match.

“But if there is any chance we can help that little feller, it’s time to suck up the pain.”

At a registry drive, the inside of a person’s cheek is swabbed so the tissue can be typed. People identified as potential donors have additional blood tests to confirm a match.

Bone marrow is donated two ways: 80 percent is given through a blood donation in which marrow-producing blood cells are taken, and 20 percent is given through a 30-minute outpatient surgery that extracts bone marrow from the rear pelvic area. Either procedure will leave people sore for a few days.

Now is a particularly good time to register: Through May 19, the National Marrow Donor Program is waiving the $52 fee that potential donors pay to cover the cost of tissue typing.

The community outpouring for Granton didn’t surprise his father, who has taught English at Bolivar High School for eight years.

“We never asked for help, but Bolivar is the kind of a town that they are there if you need help,” he said.

Daniel Bayless moved to Bolivar to finish college at Southwest Baptist University. He stayed on to teach and coach football, wrestling and track. And he fell in love with the school district athletic director’s daughter, Jennifer, an elementary school art teacher. They married in 2003.

Granton, their first child, was born Oct. 2 at 9 pounds, 2 ounces. The nurses called him “Brutus.”

At about three months, Granton began to lose weight, something his parents initially thought was baby fat. But then Granton developed a persistent cough that doctors couldn’t explain.

In March, relatives noticed how much weight Granton had lost since the last time they had seen him, sending Daniel and Jennifer Bayless into emergency mode.

The child was admitted March 27 to Children’s Mercy.

Granton is battling pneumonia and respiratory syncytial virus (RSV). Daniel Bayless said his son would need to get stronger before a bone-marrow transplant was possible.

Meantime, family, friends and strangers are trying to make it possible.

People also are trying to help the family financially. Jennifer Bayless is a stay-at-home mom, and Daniel Bayless is on sick leave through the school year. They are staying at the Ronald McDonald House in Kansas City.

One of Daniel’s Bayless’ sisters was approached in a Coffeyville, Kan., grocery by someone selling a coupon book as a fundraiser for a family in need. It was for her brother’s family, and she bought one without revealing her connection.

A friend is holding a garage sale today and Saturday in Olathe. Another friend is selling 300 T-shirts with Granton’s photo and the phrase “Don’t Worry, Pray,” something Daniel Bayless was told by a consoling relative of another patient at Children’s Mercy.

Daniel Bayless returned Monday to Bolivar because although he had been tested as a bone-marrow donor for Granton, he hadn’t registered with the national program.

“I never expected so many people to come out and get their cheeks swabbed based on the fact that my son is in the hospital,” he said. “It is humbling that so many people care about my family.

“The whole thing has made me realize all the great people in our country. They reach out with no ulterior motive to do something good.”

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On the Web
To see a video describing the marrow donation process, go to KansasCity.com.

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Registration drives
The National Marrow Donor Program is in the middle of its annual “Thanks Mom” program. Through May 19, the program is waiving the $52 fee that potential donors pay to cover the cost of tissue typing.

Kansas City “Thanks Mom” drives:

•11 a.m. to 2 p.m. Saturday at Brush Creek Community Center, 3801 Emanuel Cleaver II Blvd.

•10 a.m. to 2 p.m. Monday at Children’s Mercy Hospital, 2401 Gillham Road.

•2 to 6 p.m. Thursday at St. Charles Borromeo Parish, 900 N.E. Shady Lane Drive in the Northland.

•9 a.m. to noon May 18 at St. Therese School, 7277 N.W. Missouri 9 in the Northland.

•8:30 to 11 a.m. May 18 at Temple B’nai Jehudah, 12320 Nall Ave., Overland Park.

•9 a.m. to 1 p.m. May 18 at Christ Church, 5500 W. 91st St. Overland Park.

•Noon to 8 p.m. May 19 at Argosy Casino, 777 Argosy Parkway, Riverside.

You can also register online by going to www.marrow.org. The program will mail a cheek-swabbing kit.

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Bone Marrow Match Needed for Six-Month Old in Polk County

Bone Marrow Match Needed for Six-Month Old in Polk County

Reported by: David Oliver

Monday, May 5, 2008 @08:31pm CST

bone12008-05-05-1210037938.jpg

When a child is sick most people will do anything to help. That’s exactly what happened in Bolivar where perfect strangers spent the day swabbing their mouths to see if they might be the bone marrow match that a little boy needs.

“We’ve been getting the word out for the last couple of weeks, people have been responding. It’s been a real neat blessing” says event organizer Clayton McCullah.

Seated at tables and swabbing their mouths, these people are trying to make life better for six-month-old Granton Bayless.

“We found out his little boy has SCIDS. It’s severe combined immunodeficiency syndrome. And he basically needs a bone marrow transplant because he can’t produce his own white blood cells to fight off infection” says McCullah.

Granton’s dad is a teacher and coach at Bolivar High School. Several current and former students are among those who are trying to see if their marrow might be a match.

“I went to high school here. Heard my old coach’s kid needed some help. Thought I’d come try to help” says former student Scott Crain.bone22008-05-05-1210038034.jpg

“And I have four children of my own and I would do anything for them and I’m sure everyone in this community would do everything they could to help baby Granton” says Shelly Brown.

Organizers ordered 1000 kits for the drive. They’re surprised at how many people showed up to embrace the effort. Even if they aren’t a match for little Granton, they might be able to save the life of a perfect stranger far away.

“People need to realize they may not be an exact match for Granton. It’s kind of like finding a needle in a haystack. But they could be that needle for someone else. They could be contacted for someone who’s looking for a bone marrow transplant” says McCullah.

Normally it costs $52.00 to be tested and register to be a bone marrow donor. But the kits were free for the Bolivar drive because of a nationwide push to find more donors. Little Granton Bayless is being treated right now in Kansas City for pneumonia and RSV.

To learn more about Granton Bayless go to: www.grantonbayless.blogspot.com.

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Bursting the bubble

A newborn was diagnosed with what’s called the “bubble boy” disease and his mother is hoping that a bone marrow transplant will save his life.

Crystal Schelle

POSTED: May 4, 2008

MARTINSBURG —Rebecca Manor is still smarting from the accusation that she wasn’t feeding her 3-month-old son Keithan.

It was Easter Sunday when she got the call from her pediatrician asking why her son was gaining weight at the hospital, but losing weight at home. She figured it was because he was hooked to an IV and pumping fluids through his body. Thinking it might be neglect, her doctor told her that she was being reported to Child Protective Services.

“I couldn’t believe they thought I wasn’t feeding him,” she says.

It would take a transfer to three hospitals before doctors discovered the problem was actually Severe Combined Immunodeificiency, which according to the SCID Foundation’s Web site is a a primary immune deficiency. According to several Web sites, SCID happens in 1 out of every 100,000 live births.

SCID is commonly referred to as the “bubble boy” disease, named for the large plastic-encased room that David Vetter had to live in for 12 years during the 1970s and 1980s. Vetter’s story loosely inspired the 1976 made-for-TV movie “The Boy in the Plastic Bubble,” which starred John Travolta.

Manor says her pregnancy with Keithan was fine. He came five weeks early, but it wasn’t a cause for concern because her other four sons —twins Bailey and Jayson, 4; Justin, 2; and Brayden, 16 months —had all arrived prematurely. “Everything seemed fine,” she says.

Keithan was born at 10:15 p.m. Jan. 24 in City Hospital. He weighed 5 pounds, 6 ounces and was 18 1/2 inches long. He had 10 little fingers, 10 little toes. He was perfect.

She had brought him home to his brothers and he seemed to be doing fine. But he wasn’t gaining weight, causing Manor concern.

On March 23, Keithan was admitted to City Hospital because he wasn’t gaining any weight. By March 25, he was transferred to Ruby Memorial Hospital in Morgantown. “After three or four days, he was shipped to D.C.,” she says.

Now, Keithan is at Children’s Hospital in Washington, where Keithan’s new doctor helped to pin-point the reason for lack of weight gain. The doctor told her that he suspected SCID, or the “bubble boy” disease. “I didn’t even know what it was. I was stunned,” she says. “I sat for a moment and was quiet.”

Manor says the reason he suspected that Keithan might have SCID was because his T cell production in his blood was low. According to the SCID Foundation Web site, “the defining characteristic of SCID is the absence of T cells and, as a result, lack of B cell function as well.”

According to the National Human Genome Research Institute, it’s rare for SCID to be diagnosed. Children often exhibit the following symptoms: eight or more ear infections; two or more cases of pneumonia infections that do not resolve with antibiotic treatment for two or more months; failure to gain weight or grow normally; infections that require intravenous antibiotic treatment; deep-seated infections, such as pneumonia that affects an entire lung or an abscess in the liver; persistent thrush in the mouth or throat or a family history of immune deficiency or infant deaths because of infections.

After a blood test confirmed her doctor’s suspicions, he called the next day to tell her that it was indeed SCID. The diagnosis was bittersweet because at last, she had a name for what was causing her son’s medical problems, but it also meant a journey for her child.

According to the SCID Foundation, unless the defects in the blood are corrected infection would be Keithan’s biggest obstacle, which would most certainly mean death before his 2nd birthday.

There are at least 10 forms of SCID, but Manor says there are two main types — X-SCID and ADA deficiency.

In the case of the X-SCID, the female is a silent carrier. “It said if I was a carrier then I had 50 percent chance that any male child would have it, but none of my other four boys have it,” she says.

In the case of ADA deficiency is inherited from both of the baby’s parents, each one carrying a normal copy of the gene and one abnormal copy of the gene.

Manor, who says Keithan’s father really isn’t the picture, isn’t sure if SCID was inherited by her or by her and Keithan’s father. She says she’s waiting on test results to find out which kind of SCID her son has and what’s the next step for him.

Most likely, Manor says, he will have to have a bone marrow transplant. She says she’s already thought about the hard decision if one of her other young sons are a match. “I know some people won’t agree with it, but I would allow them to be a donor,” she says. “It might be painful that day, but when they get older they will know why they did it.”

She says her other sons have been asking about their younger brother. “They keep asking when he’s coming home from the hospital,” she says, as her boys play behind her. “I tell them that he’s sick and he’s there to make him better.”

They also ask about his helicopter ride. Keithan had to be airlifted from Morgantown to Washington, D.C.

It’s that trip that Manor says she remembers the most. “It was the first time he smiled — really smiled — at me,” she says.

Currently unemployed and in the middle of moving, Manor says she can’t stand that she’s not able to spend time every day at Children’s Hospital with her son. “It just kills me,” she says.

She’s scheduled to go down today to see him, thanks to her family driving her there. She says when son Jayson had to have open heart surgery, she slept in the waiting room for days. With Keithan, that’s not an option.

“But I’m on the phone with his doctor every day to check to see if his weight is up,” she says, looking at the photos of Keithan.

At this time, Manor says it’s a waiting game to see what’s the next step. If he does get a bone marrow transplant he might still have to undergo gene therapy. She hasn’t even thought about the bubble.

She’s also not even thinking about the mountain of medical bills she’ll be facing. Instead, she says, she’s thinking about other mothers who might face the same diagnosis of their child.

“They can do a simple test at birth,” she says.

In fact, researches at the National Human Genome Research Institute, part of the National Institutes of Health, have developed a new method to detect the immune deficiency. It’s a process that Manor says will one day be a part of the regular blood work-up for newborns.

As for the pediatrician who accused her of neglect, Manor says she received an apology. But right now, it doesn’t matter. “It kills me that I’m not able to hold him,” she says.

—Staff writer Crystal Schelle can be reached at (304) 263-8931, ext. 213, or cschelle@journal-news.net

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