Angel Flight to aid ailing BUGC student

11/26/2007, 10:37 am

By Mary Baskerville
mbaskerville@daily-journal.com
815-937-3304

Thirteen-year old Balei Chinski faces pain each day, suffering from a rare immune-deficiency disease that leaves her unable to fight off infections.

In December, she will travel on an Angel Flight from the Greater Kankakee Airport to Duke University in North Carolina to meet with Dr. Rebecca Buckley, one of the leading specialists in the disease in the nation. Angel Flights are offered by a network of pilots providing free air transport to places offering specialized medical treatment.

She has juvenile rheumatoid arthritis and experiences difficulty with movement. The disease is similar to the one that caused the late David Vetter, the boy that became known worldwide as the “Boy in the Bubble,” to live in a germ-free isolator, said Balei’s mother, Cheryl Chinski.

Diagnosed with undefined severe combined immunodeficiency (SCID), her condition “is not like his. His was genetic and total,” Chinski said.

Balei’s immune system “comes on and off, like a faulty switch,” her mother said. Some days are fine, others are not, she said, adding that Buckley was one of the doctors to treat the “Boy in the Bubble.”

Balei faces her health problems with grace, Chinski said. Doctors call her daughter “a brave soul and a stoic soul,” she added.

A seventh grader at Bourbonnais Upper Grade School, Balei has only been able to attend classes for a week this year. She’s experienced strokes and has been hospitalized several times. She tires easily but tries to keep up with school work. A tutor is available for only five hours a week, Chinski said.

Her week also includes trips for physical, occupational and speech therapy. Health problems were first identified when Balei was just five months old.

Friends and family members are raising funds to help pay for Balei’s upcoming trip to Duke. Chinski will fly with her daughter and is grateful for the fundraising drive organized to help offset the costs.

Friend Michelle Beaupre said that while the doctor has agreed to see Balei because of the uniqueness of her disease, insurance will not pay for any of the tests or procedures done in North Carolina.

Both Chinski’s work and education have been shaped by her daughter’s disease. She is a full-time nursing student, expecting to graduate in July. She also works as a nurses’ aide at Provena St. Mary’s Hospital in Kankakee.

Working at the hospital has been wonderful because it gives her the security of being close when her daughter is a patient there. “When she’s ill, I can be there.”

Balei’s family includes her dad, Jeff Chinski, and sisters Micalyn, Kelsei and Madesen.

Benefit planned

Friends are also planning a benefit from 1 to 11 p.m., at the Kankakee VFW, on March 1. The VFW has donated the hall rental.

Contributions to the “Balei Chinski Benefit Fund” may be sent to:

People’s Bank, 315 Main Street NW, Bourbonnais, IL 60914.

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Transplants Without Tears

By Mitch Leslie
ScienceNOW Daily News
26 November 2007

A new treatment might allow patients to avoid some of the grueling side effects of bone marrow transplants. Researchers reported in the 23 November issue of Science that they can use a specific type of antibody to clear away old marrow stem cells in mice, allowing fresh ones to take their place. The discovery could allow patients to receive bone marrow without undergoing chemotherapy and other toxic procedures. Bone marrow transplants can ameliorate diseases such as sickle cell anemia by replenishing hematopoietic stem cells (HSCs) that spawn white and red blood cells. But before they receive this marrow, patients must typically undergo conditioning, a course of chemotherapy (and sometimes radiation) that wipes out immune cells that might attack the transplants and eliminates the existing, faulty HSCs. However, conditioning also devastates stem cells throughout the body, triggering hair loss, diarrhea, mental decline, and other side effects.

Searching for a gentler approach, postdoc Deepta Bhattacharya and immunologist Irving Weissman of the Stanford University School of Medicine in Palo Alto, California, and colleagues dosed mice with an antibody that ties up c-kit, a receptor on the surface of HSCs that promotes their division and survival. The antibody sent the number of HSCs in the animals’ bone marrow plunging by more than 98% after 8 days, the researchers report. That seemed to clear space for new cells to rebuild the animals’ immune systems. Six months after a bone marrow transplant, 90% of one type of immune cell were derived from transferred HSCs, the team found.

Weissman envisions that an HSC-removing antibody will be part of a two-pronged attack on illnesses such as sickle cell anemia, severe combined immunodeficiency, aplastic anemia, and thalassemia. First, patients would receive antibodies to suppress immune cells that might reject a bone marrow transplant; such antibodies are already in use, although they can cause flulike symptoms and other side effects. Then, an HSC-deleting antibody would make room for new stem cells. Weissman cautions, however, that researchers need to find a human antibody that performs as well as the mouse version. But if successful, the strategy could eliminate the need for chemotherapy and radiation and allow transplants for diseases, such as type 1 diabetes, multiple sclerosis, and lupus, in which traditional conditioning was considered too drastic.

“It’s an intriguing new approach,” says stem cell biologist and clinician David Scadden of Harvard Medical School in Boston, Massachusetts. But stem cell biologist Kateri Moore of Mount Sinai School of Medicine in New York City questions whether the antibody removes all HSCs. She notes that even without a transplant, HSC numbers rebound in mice within about 3 weeks of an antibody dose. Any HSCs spared by the antibody, she warns, could compete with newcomers for space or even produce T cells that attack the transplants.

linkback url: http://sciencenow.sciencemag.org/cgi/content/full/2007/1126/2

SCID takes top billing on October 2007 JACI

This is the cover of the October 2007 Journal of Allergy and Clinical Immunology. It features 5 SCID boys with a picture taken at the IDF Patient and Family Conference in 2001.

Bubble Baby Logan recovering well

“BUBBLE baby” Logan Wilkieson is recovering after a vital stem-cell transplant and blood transfusion which could save his life.

Eight-month-old Logan, from Horwich, was born with the ultra-rare condition, Severe Combined Immunodeficiency, which affects just one in 100,000 babies.

It weakens the immune system to the extent that any infection could prove deadly.

Little Logan first spent six weeks at Newcastle General Infirmary living in a bubble of clean air with everything having to be sterilised before he touched it to stop him picking up infections.

Then he underwent 12 days of chemotherapy.

This Wednesday he received his stem-cell transplant and transfusion. It will give him an 85 per cent chance of survival.

Mum Ruth Lawrie, aged 20, said: “He is all right. He has had the operation and transfusion and has picked up so well that he is playing all the time now.

“Now it’s just a case of waiting to see if the cells grow properly in the body, which can take anything from six weeks to two years.”

Ruth and her partner, Gareth Wilkieson, aged 22, had to give up their rented house in Horwich after Logan was admitted to hospital in Newcastle.

Ruth is now living in a specially provided flat near the hospital, while Gareth is sleeping at friends and his parents while continuing his job as a shutter-door fitter. He drives to Newcastle at weekends.

Logan was born on March 21. He was two weeks overdue and delivered by Caesarean section.

There did not initially appear to be any difficulties, but he started with a cough and was given an inhaler and medicine.

Later he was admitted to the Royal Bolton Hospital with sickness and diarrhoea. Then blood tests discovered Severe Combined Immunodeficiency Syndrome and Logan was transferred to the specialist unit in Newcastle, one of only two in the country.

If the stem cell transplant, from an umbilical cord - which doctors say is a perfect match - works, Logan should make a complete recovery and be able to live a normal life.

When Logan reaches a certain point in his recovery, he will be taken to a halfway house, where he will be allowed outside in a buggy fitted with a protective hood, before heading home.

He will still be vulnerable to infection and he will be kept away from large groups of people or pets for a few months.

Ruth said: “He will have to go for blood tests every so often just to make sure the bone marrow that was transplanted is growing.

“But eventually he should be able to live a completely normal life.”

Logan’s progress is being detailed in a blog created by Ruth’s sister, Katie, which is on the website www.littlelogansblog.blogspot.com.

11:13am Saturday 24th November 2007

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Utah Medicare patients denied life-saving treatment

Utah Medicare patients denied life-saving treatment
As the head of the section for clinical immunology/ immunodeficiency diseases at the University of Utah, I treat patients with primary (or genetically determined) immune deficiency diseases. Recently, an unintended consequence of federal Medicare legislation has had a very disturbing effect on PIDD patients and the health-care professionals who treat them.
PIDD is characterized by a weakened or absent immune system. As a result, the patients are extremely vulnerable to infection. For a patient with PIDD, a respiratory infection, for instance, could lead to pneumonia and an emergency room visit and possible hospitalization.

Many patients rely on a treatment call intravenous immune globulin. Without regular infusions of IVIG, these patients face the risk of serious, overwhelming infection and even death.
In 2005, there was a change in Medicare reimbursement for IVIG. It created a treatment access problem for patients by changing the way Medicare pays for IVIG therapy. This same legislation changed the policy for home health-care coverage as well, leaving many of these patients unable to afford treatment in their homes - the safest and least contaminated environment for patients with compromised immune systems.
Almost two years subsequent to this well-intentioned legislation, the IVIG access and reimbursement problem persists. Though I have eventually been able to help most of my patients find IVIG treatment, too often the patients struggle with serious infections and debilitating illnesses as a result of missing their regular treatments.
Gaps in treatment are not only dangerous to the health of these patients, but also result in severe emotional distress in many instances. IVIG is crucial to my patients, providing them with their only chance at living a normal and productive life. Needless to say, they often become distraught when they learn that Medicare’s inadequate reimbursement policies limit their access to this life-saving treatment.
It is my understanding that patients and physicians nationwide are facing these same challenges. In April 2007, the Office of the Inspector General surveyed physicians regarding IVIG treatment procedures. More than 40 percent of physicians surveyed said Medicare payments are not enough to cover the actual cost of purchasing IVIG for their patients.
Because of this, many physicians, I am told, have been forced to discontinue IVIG infusions to Medicare beneficiaries altogether.
A recent patient survey sponsored by the Immune Deficiency Foundation reported many patients had to postpone and/or reduce frequency of treatment because of these reimbursement problems. As a result, they are at a higher risk of hospitalization with serious infections, which, ironically, almost always becomes more costly to Medicare.
But there is hope. A bill called the Medicare IVIG Access Act, recently introduced in Congress, provides a solution to this crisis by establishing a process for reviewing IVIG reimbursement and adjusting rates appropriately. The bill, HR2914, also authorizes Medicare to pay for the costs of administering IVIG in the home, giving patients a safer, more convenient option for treatment location, which is also less expensive and probably even safer.
Similar legislation has not yet been introduced in the Senate, but it’s important that senators be made aware of the issue before they act on Medicare measures this fall. I ask, on behalf of the patients, families and physicians in our community, that Utah’s Sen. Orrin Hatch and Sen. Bob Bennett take a leadership role in ensuring that there is proper access to treatment and medication for patients with primary immune-deficiency diseases.
These patients can’t afford to wait. They need your help now.
—
* HARRY HILL is a professor of pathology, pediatrics and medicine at the University of Utah.

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Help for Adam; Naples baby has rare disease

ONLY ON WINK: Help for Adam; Naples baby has rare disease

By Nick Spinetto, WINK News

Story Created: Nov 18, 2007 at 10:33 PM EST

Story Updated: Nov 19, 2007 at 1:51 PM EST

Naples - A Naples family needs your help.

Their 7 month old baby is fighting a rare disease that could take his life.

For months Adam Saada literally lived in a bubble.

Now wearing latex gloves and a surgical mask are a requirement just to see him.

“He was diagnosed with S.C.I.D, where he has no immune system,” said Amy Saada, Adam’s mother.

No immune system means you have to wear masks and gloves whenever you’re around Adam, so you won’t spread germs.

A sign on the front door warns guests: you must wear gloves and a mask in order to come inside.

“He’s been through a lot,” said Hussam Saada, Adam’s father.

Inside the home, Adam roams free, but it’s been months since he’s been touched without gloves or kissed without a mask.

In August, Adam was hospitalized and diagnosed with Severe Combined Immunodeficiency, or S.C.I.D.

His parents were told he had 30 days to get a bone marrow transplant or he could die.

“So far it’s been successful,” Amy said.

Amy was the donor.

After spending months in the hospital, Adam was able to come home November 1st.

“I’m smiling under my mask,” Amy said.

But his body could still reject the bone marrow.

He’s currently on a dozen different medications, including steroids, which cause his cheeks to swell.

A section of the refrigerator is reserved just for his medications.

With medications and the transplant, the family has accrued almost $600,000 in expenses.

Insurance will only pay so much, so the community is rallying behind the family.

“I’m thanking the community for everything they’ve done. This is going to be one special Thanksgiving,” Amy said.

So far people all over Southwest Florida have donated almost $70,000 dollars to help Adam.

“There’s something special about him,” Amy said.

The family has to wait until the end of December to see if Adam’s transplant will be successful.

If not, they’ll be back at square one and Adam will have to be hospitalized again.

To lend some support, the community is hosting another fund-raiser for Adam on December 7th.

It’s a spaghetti dinner.

It will be at East Naples United Methodist Church.

Tickets are $15 per person.

For a time and directions, you can call Marian Tosher at 239-793-4274.

linkback url: http://www.winknews.com/news/local/11564006.html

Cellectis Announces Delivery of a Meganuclease Targeting the RAG1 Gene

Cellectis Announces Delivery of a Meganuclease Targeting the RAG1 Gene (Responsible for a Severe Immunodeficiency) to Professor LuigiNotarangelo’s research group at the Children’s Hospital Boston (USA)

BIOCITECH, France - November 15 2007 - Cellectis SA, the rational genome engineering company specializing in the production of meganuclease recombination systems and in meganuclease engineering, today announced the delivery of a meganuclease targeting the RAG1 gene to Professor Luigi Notarangelo’s research group at the Children’s Hospital Boston (USA).

The RAG1 gene is mutated in certain severe combined immunodeficiency (SCID) patients. To date, the only possible therapeutic response for these patients has been a bone marrow transplant, for reconstitution of their entire immune system. In 2000, new hope arose for patients with X-SCID (a type of SCID syndrome, see below), following promising gene therapy trials performed in France. However, several of the treated patients developed leukemia. These severe adverse events appear to be linked to insertion of the gene therapy vector in the vicinity of a particular gene. Meganucleases enable an alternative approach based on correction of the defective gene itself, rather than random insertion of a “gene drug”. The meganuclease technology should enable avoidance of the above-mentioned adverse event and should help make the gene therapy approach safer.

In August, Cellectis had already delivered a meganuclease to Professor Alain Fischer’s group (INSERM Unit U768 at the Necker Children’s Hospital in Paris) in order to test its potential ability to repair the defective gene in cell lines carrying a X-SCID mutation. X-SCID patients represent another class of SCID patients, in whom the disease is linked to a mutation in the IL2RG gene. Meganucleases targeting the RAG1 gene address another type of patient but this new collaboration demonstrated Cellectis’ emphasis on SCID syndromes in general. Indeed, Cellectis is strengthening its commitment to finding a treatment for this type of disease and, in the short- to mid-term, intends to evidence clinical proof of concept for its genome surgery approach in humans.

About Cellectis

Cellectis SA is a world-leading company in genome engineering and genome surgery. The company is focused on developing and producing custom meganucleases for in vivo DNA surgery and also provides new tools for rational reverse genetics and targeted recombination. Cellectis’ products induce unique, site-directed, double-strand DNA breaks in a living cell and can be used in a wide range of biotechnological and therapeutic applications. To date, Cellectis has entered into more than 48 deals on its genome engineering technologies with major players in the pharma, biotech and agrobiotech industries. Cellectis is listed on the NYSE Euronext Alternext market (ticker code: ALCLS). For more information on Cellectis, visit our web site: www.cellectis.com

About Cellectis’ technology

A meganuclease is a (protein) molecule that cuts DNA at a highly precise site on a chromosome. Once DNA is broken, it has to be repaired by the cell’s natural endogenous maintenance systems. By providing a specifically engineered DNA molecule (called a repair matrix) which will be used as a template to repair the break, one can channel the repair pathway into an insertion, deletion or correction process. Thus, meganucleases can be used to trigger precise modification of specific genes in a variety of cells and organisms. By combining the meganuclease’s capacity to cut DNA and DNA’s ability to undergo repair, Cellectis is creating new generations of products for a wide spectrum of applications - including human health, since many genetic diseases result from a single mutation in a specific gene. Meganucleases can specifically target this same gene. In parallel, a DNA repair matrix (prepared by Cellectis and including a non-mutated copy of this gene) will be introduced into the cell.

Upon cleavage by the meganuclease, the repair matrix will be used as a template to restore a correct gene. By erasing the mutation, gene correction addresses the very cause of the disease, rather than its consequences.

About Cellectis’ collaboration policy

Cellectis’ policy is to foster research excellence in order to offer new solutions for genome engineering. To date, the major outcome of this effort has been the production of custom meganucleases that cleave genes of interest; this capability vastly expands the range of potential applications and is a prerequisite for therapeutic use. Whereas the core activity (i.e. the protein engineering itself) is usually conducted solely by Cellectis, upstream studies are often performed in collaboration with other major players in this field.

Cellectis’ Forward-Looking Statements

This communication expressly or implicitly contains certain forward-looking statements concerning Cellectis and its business. Such statements are based on assumptions and assessments made by Cellectis’ management in light of their experience and their perception of historical trends, current conditions, expected future developments and other factors they believe to be appropriate. They are not guarantees of Cellectis’ future performance and involve certain known and unknown risks, uncertainties and other factors, which could cause the actual results, financial condition, performance or achievements of Cellectis SA to be materially different from any future results, financial condition, performance or achievements expressed or implied by such forward-looking statements.

Cellectis is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise. Potential risks and uncertainties which could cause actual results, financial condition, performance or achievements of Cellectis SA to differ from those contained in the forward-looking statements include, without limitation, the risks and uncertainties discussed in the Risk Factors (”facteurs de risques”) sections of the prospectus prepared by Cellectis approved by the French Autorité des Marchés Financiers (“AMF”) on January 22 2007 under visa number 07-023, available on the websites of the AMF (http://www.amf-france.org) [and Cellectis (http://www.cellectis.com)].

For further information, please contact:

Cellectis S.A. Scientific Director
Frédéric Pâques, PhD.
e-mail: sciences@cellectis.com
Tel.: + 33 (0)1 41 83 99 00

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BioRx Offers Vivaglobin® to Patients with Primary Immune Deficiency Disorders

CINCINNATI–(BUSINESS WIRE)–BioRx, a specialty pharmaceutical company, announces the availability of Vivaglobin® (immune globulin subcutaneous [human]), an immunoglobulin (ig) replacement therapy for treating patients with primary immune deficiency disorder (PIDD). PIDD is a group of usually-genetic disorders that compromise the immune system, leaving people vulnerable to recurrent, life-threatening infections. An estimated 50,000 Americans have PIDD.

BioRx is one of a small number of specialty pharmacies currently approved to dispense Vivaglobin®, which is manufactured and marketed by CSL Behring. Vivaglobin® is the first and only subcutaneous immunoglobulin replacement therapy approved by the FDA in the United States. Vivaglobin® delivers treatment directly under the skin (subcutaneously), offering a safe and effective alternative to intravenous infusions of immunoglobulin.

Mark Kestler, General Manager of BioRx, says “Recognition by CSL Behring as an approved Vivaglobin® provider validates BioRx as a leading quality provider of specialized pharmaceutical services. We are excited to be able to offer this valuable treatment option to our PIDD patients.”

Until recently, PIDD patients needed to schedule appointments with a hospital, physician or homecare company to receive intravenous immunoglobulin treatment. Immunoglobulin is a life-sustaining blood product that is standard immune replacement therapy for most people living with PIDD. Approximately 70 percent of PIDD patients receive Ig replacement therapy, primarily intravenous immunoglobulin (IVIg), in which a pump delivers the immunoglobulin through a needle into a vein.

With Vivaglobin®, PIDD patients in the United States can now self-administer the treatment at home, with training from and the approval by their physician. The drug represents another treatment option for patients who may not easily tolerate the traditional intravenous method, because they have poor venous access or experience serious side effects from that method. Vivaglobin® also is appropriate for those who want the freedom and convenience of safe home self-administration of Ig replacement therapy.

This convenient treatment option will require education to help patients properly manage their own therapy. BioRx will provide patient training, as well as the drug, infusion pump, needles, tubing and any other supplies needed for treatment. All patients who start on Vivaglobin® receive a patient starter kit to help manage their therapy. The kit includes helpful tools, such as administration guides, product information and a treatment journal. Typically, a patient requires approximately 3 or 4 educational sessions with a specially trained nurse before beginning self-administration independently.

More information about Vivaglobin® at BioRx is available by calling 866-442-4679, or via email at info@biorx.net.

Important Safety Information

As with all immune globulin products, Vivaglobin is contraindicated in individuals with a history of anaphylactic or severe systemic response to immune globulin preparations and in persons with selective IgA deficiency who have known antibody against IgA. If anaphylactic or anaphylactoid reactions are suspected, discontinue administration immediately and treat as medically appropriate.

Vivaglobin is derived from human plasma. As with all plasma –derived products, the risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, cannot be completely eliminated.

In clinical trials, the most frequent adverse event was injection-site reaction, consisting of mild or moderate swelling, redness, and itching. No serious local site reactions were observed, and reactions tended to decrease substantially after repeated use. Other adverse events irrespective of causality included headache, gastrointestinal disorder, fever, nausea, sore throat, and rash.

As with all immune globulin products, patients receiving Ig therapy for the first time, receiving a new product, or not having received Ig therapy within the preceding eight weeks may be at risk for developing reactions including fever, chills, nausea, and vomiting. On rare occasions, these reactions may lead to shock. Such patients should be monitored in a clinical setting during the initial administration.

Ig administration can transiently impair the efficacy of live attenuated virus vaccines, such as measles, mumps and rubella.

In clinical studies, administration of Vivaglobin has been shown to be safe and well tolerated in both adult and pediatric subjects. No pediatric-specific dose requirements were necessary to achieve the desired serum IgG levels. Safety and efficacy were not studied in pediatric subjects under two years of age.

About CSL Behring

CSL Behring is a global leader in the plasma protein biotherapeutics industry. Passionate about improving the quality of patients’ lives, CSL Behring manufactures and markets a range of safe and effective plasma-derived and recombinant products and related services. The company’s therapies are used in the treatment of immune deficiency disorders, hemophilia, von Willebrand disease, other bleeding disorders and inherited emphysema. Other products are used for the prevention of hemolytic diseases in the newborn, in cardiac surgery, organ transplantation and in the treatment of burns. The company also operates one of the world’s largest plasma collection networks, ZLB Plasma. CSL Behring is a subsidiary of CSL Limited, a biopharmaceutical company with headquarters in Melbourne, Australia. For more information, visit www.cslbehring.com.

About BioRx

Based in Cincinnati Ohio, BioRx is a national pharmacy specializing in highly customized care for the primary immunodeficiency diseases (PIDD) community. As one of the nation’s fastest growing providers of IgG and other specialty pharmaceuticals, the company’s clinical staff reaches patients and physicians in all 50 states. To learn more about BioRx and its products and services visit www.biorx.net.

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