FDA Suspends Seattle Gene Therapy Study

July 27, 2007

FDA Suspends Seattle Gene Therapy Study
WASHINGTON, July 27, 2007


(AP) The government has suspended a Seattle company’s gene therapy study; and is reviewing the safety of 28 others around the country; after learning that a patient died this week.The Food and Drug Administration didn’t reveal the cause of death or any details about the patient, who had enrolled in a study of gene therapy for advanced arthritis. The agency said it was investigating what role, if any, therapy played in the death, which occurred Tuesday.

It marks the third blow since 1999 to the field of gene therapy, as scientists struggle to determine if the viruses they use to deliver new genes may themselves cause serious trouble.

Twenty-eight other gene therapy studies have been reported to the FDA that used, or are using, the same virus, called adeno-associated virus or AAV.

The FDA said Thursday that it was not aware of any serious side effects in any of those studies but that as a precaution, officials were reviewing all the ones still actively treating patients.

Targeted Genetics Corp. notified the FDA of the patient’s illness and subsequent death.

The company had enrolled more than 100 people in the study without similar problems, but this patient became ill after a second injection of the therapy directly into an arthritic joint, the FDA said.

The therapy uses AAV to deliver a gene that in turn blocks tumor necrosis factor, a substance that fuels the joint inflammation behind crippling forms of arthritis. Drugs that block TNF already are widely used to treat rheumatoid arthritis and other conditions, but a gene therapy approach is novel.

“We are deeply saddened by the death of an individual enrolled in our clinical trial,” company president H. Stewart Parker said in a statement issued Thursday.

The company didn’t immediately return a phone call seeking comment.

In 1999, 18-year-old Jesse Gelsinger died in his fourth day of a gene therapy experiment at the University of Pennsylvania. Gelsinger had suffered from an inherited disorder that blocks the body from properly processing nitrogen. The FDA concluded that the gene therapy injection intended to try to cure him instead killed him.

That gene therapy attempt used a cousin to AAV, called adenovirus, to deliver the needed gene.

The only disease ever to be cured with gene therapy is “bubble boy disease,” an immune disorder formally called severe combined immunodeficiency, or SCID. But gene therapy attempts are restricted to SCID patients who have no alternative, after doctors in 2003 discovered a few babies saved by gene therapy went on to develop cancer; again, linked to the virus used to deliver the genes.

Reflecting how seriously regulators take this latest death, the National Institutes of Health’s advisory committee on gene therapy will meet in September to discuss its potential scientific implications.

linkback url: http://www.cbsnews.com/stories/2007/07/27/health/main3104268.shtml


CSL Behring Announces FDA Approval of Privigen– First Proline-Stabilized 10 Percent Liquid IVIg

July 27, 2007

CSL Behring Announces FDA Approval of Privigen– First Proline-Stabilized 10 Percent Liquid IVIg

KING OF PRUSSIA, Pa.–(BUSINESS WIRE)–Jul 27, 2007 – CSL Behring (ASX: CSL) today announced that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Privigen(TM) (Immune Globulin Intravenous (Human), 10% Liquid), an intravenous immunoglobulin (IVIg) for treating patients diagnosed with primary immunodeficiency (PI). Privigen is also indicated for the treatment of chronic immune thrombocytopenic purpura (ITP) to rapidly raise platelet counts to prevent bleeding.

A 10 percent liquid preparation of polyvalent human immunoglobulin, Privigen offers healthcare professionals convenience and ease-of-use. It is the first and only proline-stabilized IVIg that is always ready for immediate use, requiring no refrigeration or reconstitution.

“Privigen is an exciting new offering to patients and healthcare professionals in the United States,” said Paul Perreault, Executive Vice President of Worldwide Commercial Operations at CSL Behring. “As the leader in developing safe, high-quality, effective immune globulin therapies for use around the world, CSL Behring is proud to add yet another product to our rapidly growing portfolio. We see a strong demand for Privigen and are pleased to be bringing it to patients and other valued customers.”

“With Privigen, once again, CSL Behring has answered an important need,” said Mark Stein, M.D., Chief, Allergy Section at Good Samaritan Hospital in West Palm Beach Hospital in Florida. “The time savings and flexibility it provides to healthcare professionals is an important step forward. It will certainly be a welcome addition to currently available immune globulin therapy options in the U.S.” Dr. Stein served as lead investigator on the Privigen clinical trial.

CSL Behring plans to launch Privigen in the first quarter of 2008. In the meantime, the company is advancing the registration of Privigen in Europe. The application is currently under review by European regulatory authorities.

Clinical Studies

The FDA approved Privigen based on results of two pivotal open-label, prospective, multi-center clinical studies. One study was performed in the United States and Europe in subjects with PI, and the other study was performed in Europe in subjects with chronic immune thrombocytopenic purpura. In the PI study, 80 adult and pediatric subjects received Privigen every three or four weeks at doses ranging from 200 mg/kg to 888 mg/kg for a maximum of 12 months. The annual rate of serious bacterial infections (defined as bacterial pneumonia, meningitis, sepsis, osteomyelitis, and visceral abscesses), the primary endpoint, was 0.08 infections per subject per year. Pneumonia was reported in three subjects, and visceral abscess, osteomyelitis, and septic arthritis in one subject each. The annual rate of any infections, a secondary endpoint, was 3.55 infections per subject per year.

In the ITP study, 57 subjects with a platelet count of less than or equal to 20 x 10(9)/L received 1 g/kg Privigen twice on each of two consecutive days and were observed for 29 days. A total of 46, or 80.7% of subjects, responded to Privigen therapy with an increase of platelet count to greater than or equal to 50 x10(9)/L within seven days after the first study drug administration. Hemolysis occurred in eight subjects treated with Privigen in the ITP study. These cases all resolved uneventfully.

Important Safety Information

In clinical studies, Privigen has been shown to be safe. As with any medication, side effects may accompany treatment. The frequency of side effects was based on a review of 1,038 injections given during the clinical trial in the United States and Europe. Because Privigen is made from plasma, as are all commercial human polyvalent immunoglobulins, the risk of transmitting infectious agents, including viruses, and theoretically, the Creutzfeldt-Jakob disease (CJD) agent, cannot be completely eliminated.

Privigen is contraindicated in patients with known anaphylactic or severe hypersensitivity responses to Immune Globulin (Human). Patients with severe selective IgA deficiency (IgA less than 0.05 g/L) may develop anti-IgA antibodies that can result in a severe anaphylactic reaction. Such patients should only receive intravenous immune globulin with utmost caution and in a setting where supportive care is available for treating life-threatening reactions.

Boxed warning: Immune Globulin Intravenous Human (IGIV) products have been associated with renal dysfunction, acute renal failure, osmotic nephrosis, and death. While reports of renal dysfunction and acute renal failure have been associated with the use of many of the licensed IGIV products, those containing sucrose as a stabilizer accounted for a disproportionate share of the total number. Privigen does not contain sucrose.

For more details and complete prescribing information on Privigen, please call the CSL Behring Medical Information Department at 1-800-504-5434.

About Primary Immune Deficiencies

These are a group of predominantly genetic disorders that cause a malfunction in part or all of the immune system, keeping the patient from fighting off infections caused by everyday germs. For individuals with PI – many of them children – infections may not improve with treatment as expected, and may keep returning. As a result, patients may face repeated rounds of antibiotics or be hospitalized for treatment. Repeated infections can lead to organ damage, which, over time, can become life-threatening. In some severe cases of PI, infections may result in a patient being hospitalized repeatedly. Some infections, such as meningitis, may even result in death. Nearly 100 types of PIs exist. Most are inherited, but in some cases the cause is unknown.

No single treatment works for all of the different types of PI. Infusions of replacement antibodies (immune globulins or Ig) can help supplement the immune system to prevent infection in nearly three-quarters of those people living with PI whose disease is tied to an antibody deficiency.

About ITP

Immune Thrombocytopenic Purpura, or ITP, is an autoimmune disease in which the immune system attacks and destroys the body’s own platelets, the cells that prevent bleeding in blood vessels and facilitate clotting. There are two forms of ITP: acute ITP, which resolves within six months, and chronic ITP, which most often occurs in adults and by definition lasts six months or longer. The annual incidence of ITP is 100 to 115 in every one million people. In the U.S., approximately 200,000 people have the disorder.

ITP is characterized by a low number of platelets (less than30 x 10(9)/L), usually caused by the body’s production of substances (antibodies) that coat the platelets and signal their elimination from the blood. Diagnosis of ITP is often made by excluding other possible causes of the low platelet count and bleeding. People with the disorder often have purple bruises on the skin called purpura, a sign that bleeding has occurred in small blood vessels under the skin. They can also have petechiae, small red splotches on the skin that resemble a rash.

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 disease in newborns, 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 http://www.CSLBehring.com.

Contact

CSL Behring
Sheila A. Burke, 610-878-4209
Director Public Relations & Communications
or
Weber Shandwick
Nicole Symon, 212-445-8175
Account Supervisor

linkback url: http://www.pharmalive.com/News/index.cfm?articleid=462787&categoryid=29#


The Kindness of Strangers

July 19, 2007

The Kindness of Strangers

From the blog: Uppity Rib

Open letter to the person who saved my nephew’s life:

I don’t know who you are, what you look like, or where you live. I don’t know if you are a he or a she. I don’t even know whether you are still alive today.

Ray.jpg

But the kid in this photo is very much alive, and that’s because of you.

Ten years ago, when he was three months old, my nephew Ray was diagnosed with Severe Combined Immunodeficiency (SCID), a genetic disease that affects 1 in every 100,000 live births. If untreated, most babies with SCID usually die from infection before their first birthday.*

At four months, Ray got his first infection. His body was covered head to toe in thrush that wouldn’t go away; with his skin flaking off everywhere, he looked like a burn victim. He was tiny, not growing – “failing to thrive,” as they say in the medical field.

He cried a lot, and so did we, watching helplessly as the disease began to sap his life away.

The only treatment for Ray’s SCID was a bone marrow transplant, and for it to work, we had to find a donor with the same marrow type as Ray. Marrow matches are most commonly found in members of the patient’s family, but none of us could help.

Thus we were forced to rely on the kindness of strangers for the life-saving marrow. Unfortunately, the chronic shortage of registered donors made finding a match difficult. Ray was already half into his life expectancy; we were racing against time.

Then we got the news we’d been praying to hear. Because you took a moment to register as an organ donor, our little boy survived.

Ray’s body accepted your marrow, recovered from SCID and hit the ground running. He’s our radiant Miracle Child.

Ray is smart, generous and kind. He likes school, Warner Brothers cartoons, Disney movies, and Harry Potter – that fellow “Boy Who Lived,” saved by love.

We in Ray’s family will probably never get to thank you in person, so we post this letter in hopes that it inspires Rib Readers to register as organ donors in your honor.

We hope your story convinces them of how much they have to give, and how five minutes of their time at OrganDonor.gov could mean a lifetime for someone like Ray.

With love,

Uppity & Family

——————————–

linkback url: http://www.uppityrib.com/archives/512


Stem cells raise hell

July 19, 2007

Stem cells raise hell
From the roots of life stem many controversies: the history, hype, and hope surrounding a potential real-life fountain of youth
By: Dan Rios
Posted: 7/16/07
The cells and their function

Stem cells are remarkable for the fact that they can both renew themselves and give rise to a huge range of cell types. Through mitosis (when a single cell divides itself equally in two), a stem cell is able to replicate itself and, when the time is right, differentiate into whatever specialized cell type is needed. This absolute flexibility is necessary to produce complex, multi-cellular organisms such as humans. After all, we start as a single fused sperm and egg cell and end up as a collection of trillions of different cells.

Keeping our origins in mind, it makes sense that stem cells are found inside embryos. These cells, true jacks-of all- trades, are the basis of embryonic development. A little more surprising is the presence of stem cells in various tissues of adults. There, they act as inhouse mechanics, repairing damaged tissues and replacing dying cells.

What exactly, then, is a stem cell? Provided that the cell in question is self renewing and has unlimited potency (the ability to differentiate into any cell type), it can be termed a stem cell.

The history

In light of the ever-growing media coverage over stem cell research, it is interesting to note that they were almost completely ignored when first discovered. This inauspicious start occurred in the 1960s, when Joseph Altman and Gopal Das found evidence that a new type of cell creates neurons during adulthood. At this point in time, it was firmly believed that no new neurons were created after birth.

Fortunately, in 1963, Canadian researchers Dr. Ernest Armstrong Mc- Culloch and Dr. James Edgar Till conclusively proved that stem cells existed. They demonstrated that these cells were capable of self-renewal in the bone marrow of the mice they were studying. Their work directly led to the use of bone marrow transplants to treat diseases such as severe combined immunodeficiency disorder, which the famous “bubble boy” David Vetter suffered from.

With each passing year, researchers uncovered more about the initially mysterious stem cells. Hematopoietic stem cells-the progenitors of all blood cells, including platelets and white blood cells-were uncovered in 1978. Embryonic stem cells were isolated in mice in 1981. A technique to isolate and grow these same cells from a human blastocyst (the ball of cells that develops into the fetus) was developed in 1998 by Dr. James Thomson’s group at the University of Wisconsin-Madison. Embryonic stem cells have been the focus of continued research- and much controversy-ever since. The rate of progress has only increased since these initial findings and shows no signs of stopping, despite some politicians’ efforts to curb certain types of stem cell research.

The controversy

Evoking numerous fundamental bioethical issues, stem cell research may be the most controversial topic in science. Most concerns are focused on human embryonic stem cell research, which requires the destruction of a human embryo or the cloning of cells to create stem cells. Not surprisingly, many pro-life groups denounce the practice, arguing that destroying an embryo is technically abortion, and devalues life because these embryos have the potential to eventually produce a human life.

In 1995, the Clinton administration agreed to federally fund research using leftover embryos from fertility treatments, but declined to fund research that involved the creation of embryos for this research based on moral grounds. In response, Congress created and passed the Dickey Amendment in the same year, prohibiting any federal funding for stem cell research that involved the destruction of embryos, regardless of their source.

After human embryonic stem cells were isolated in 1998, the issue of federal funding had to be re-examined. In August 2001, President Bush announced that this research would receive federal funding for the first time, but only if existing stem cell lines were used. This sidestepped the moral dilemma of destroying more embryos for the sake of research, as funding was given only to projects where “the life-and-death decision has already been made.”

Not content with this limited decision, Senate continued to pressure the president to expand funding for research by allowing the use of excess embryos from fertility treatments. On July 18, 2006, Senate passed the first bill that would allow federal money to be spent on this type of research. Bush vetoed this bill the next day.

Cord-blood-derived, embryonic-like stem cells and a new type of stem cell found in amniotic fluid are the latest developments in this rapidly evolving field of study. Researchers are hoping that these potentially powerful cells can eliminate the use of embryonic stem cells and the accompanying ethical complications.

A Canadian contribution

Recent findings from McMaster University researchers describe how the cells surrounding an embryonic stem cell can affect its function. Termed “niche” cells, they are created by the parent stem cell and, through the use of protein “growth factors,” are able to direct the parent cell’s growth. This new approach to manipulating stem cells that are obtained by working with the cells directly surrounding them, could lead to novel ways of controlling their behaviour and literally “programming” them to become whatever cell type is desired. Clearly, figuring out the mechanism underlying the specialization of stem cells is key to harnessing their restorative power.

The implications

When Korean researcher Hwang Woo- Suk announced that he used unfertilized human oocytes to create embryonic stem cells in 2004, he sparked media frenzies and rampant speculation as to the future of stem cells and medicine. Woo-suk’s results, revealed to be fictitious, were an unpleasant reminder of the lengths that unscrupulous scientists can go to in order to claim a key scientific breakthrough as their own.

The excitement surrounding stem cell research is understandable. If scientists harness the self-renewing abilities of stem cells, diabetics could conceivably be cured with regenerated pancreas cells. Paraplegics could have their spinal cords repaired and perhaps walk again, and cancer could become a simple matter to deal with. A theoretical real-life fountain of youth, stem cells may completely change the way medicine is practiced. So little is known, however, that it is still too early to suggest any possible timelines or exact future scenarios for the application of stem cell research.

Already, stem cells are being used in novel ways. A new procedure using fat taken from a woman’s stomach and adult stem cells enables women to grow new breasts. After the combination is injected into the chest, the stem cells nurture the surrounding fat tissue, allowing it to grow over a period of many months. Seen as a promising treatment for breast cancer patients who have undergone mastectomies, this simple procedure takes only an hour and could become commonplace in a few years.

linkback url: http://media.www.thevarsity.ca/media/storage/paper285/news/2007/07/16/Science/
Stem-Cells.Raise.Hell-2924630.shtml


Viral vs. non-viral: The debate

July 18, 2007

Viral vs. non-viral: The debate

By Anna Lewcock
18/07/2007- At a packed out session at last week’s Controlled Release Society meeting in the US, two experts battled it out to win one of the key debates in gene delivery: which is superior – viral or non-viral delivery?

The opposing sides were represented by John Chiorini of the National Institutes of Health fighting for viral delivery, and Leaf Huang of the University of North Carolina who spoke in support of non-viral delivery.

An initial vote showed that Chiorini had a lot of work on his hands to convince the audience of his argument, with the majority of his listeners already siding with the opposition.  However, he pushed on, laying the foundations of his argument and outlining some of the factors that are key in gene transfer vectors and how viral vectors satisfied these requirements more satisfactorily than non-viral agents.

“There’s going to be no one ideal vector for every application, and I think you’re going to have to tailor it, “ he cautioned, “but I think there’s going to be some common themes in all the different vectors.”

Critical features such as tolerability, stability, efficiency, targeting, how regulatable it is, long term expression, the ability to modify and ease of manufacture were all highlighted by Chiorini as requisite in an ideal vector.

Viral vectors do well in fulfilling these criteria, according to Chiorini, in particular achieving the crucial requirements of very efficient gene transfer and good tolerability – “some viruses are actually considered non-pathogenic,” he argued – as well as long-term gene transfer without eliciting an immune response.

Chiorini was keen to stress that viruses are essentially “nature’s gene delivery service,” having specifically evolved over millions of years to be able to deliver nucleic acid into a cell in a manner that is tolerated by the host, and as such are perfectly designed for use in gene therapies.

However, Chiorini had a fight on his hands, and Leaf Huang took to the podium to defend non-viral gene delivery and present his case to the audience.

“The biggest advantage of non-viral systems is that they are non-toxic,” claimed Huang in his opening statement.

“Even the safest viral vectors, like adeno-associated viral vectors and lentiviral vectors, are still immunogenic.”

To further drive home his argument, Huang made the point that in all gene therapy clinical trials where death has occurred, the viral vector itself was the cause of death.

Huang went on to describe a particular method of non-viral gene transfer, hydrodynamic injection of naked DNA, which leads to very high and prolonged transgene expression in the liver.  After injection into the tail vein of a mouse, this technique resulted in hundreds of days’ of gene expression at an extremely high level, according to Huang.

“I challenge any viral vector to do this with one single injection,” he said.

Huang also went on to describe an interesting alternative method of non-viral delivery, known as mechanical massage.  Although eliciting some amused responses from the assembled crowd, the technique involves injecting into a mouse tail vein, then pressing down four times on the stomach of the mouse – this method also resulted in a high level of gene expression in the liver, according to Huang.

Huang also argued that the targeting ability and flexibility of non-viral vectors was a key point in the favour of the delivery system, again showing an example from his research in which 70-80 per cent of the injected dose reached the site of the targeted tumour.

One point of contention between the two sides focused on the efficiency of the two techniques, specifically in regard to the increase volume of DNA that has to be injected when employing non-viral techniques.

While Huang argued that as long as the dose is affordable and non-toxic it is perfectably acceptable to inject a higher volume than common with viral vectors, Chiorini claimed that any peptides that are in non-viral vectors are going to be immunogenic in some way, and that large amounts of material are simply going to be more immunogenic than less material – “less is better in this case,” he said.

As to the risk of insertional mutagenesis with viral vectors and the cases of the x-Severe Combined Immunodeficiency syndrome (x-SCID) trials in France that were found to cause leukaemia in some children, Chiorini pointed out that only a small number of subjects were affected, and that as soon as the observation was made the trial design was modified and no further occurrences have been reported.

Despite the fact that Chirorini noted that there are now trials starting using viral vectors for the treatment of blindness and haemophilia, and viral vectors being the treatment of choice in patients for whom a matching bone marrow transplant cannot be found, the audience appeared reluctant to share his level of confidence in the viral technique.

With both opponents admitting that each other’s approach had significant points in its favour, and going as far as to say that they both in fact work to some extent using the opposition’s vectors, the session ended on a light-hearted note with the debate having generated significant discussion and involvement from the assembled audience.

Despite a valiant effort, however, Chiorini was unable to win over the crowd, with non-viral vectors emerging as the favourite in the future of gene delivery.

linkback url:http://www.in-pharmatechnologist.com/news/ng.asp?
n=78290-viral-gene-delivery-non-viral-gene-delivery-gene-therapy-x-scid


CSL Behring Completes Enrollment for Phase III Registration Trial of Next Generation of Subcutaneous Immunoglobulin

July 13, 2007

CSL Behring Completes Enrollment for Phase III Registration Trial of Next Generation of Subcutaneous Immunoglobulin

-Clinical trial of IgPro20 for primary immunodeficiency moves ahead-

KING OF PRUSSIA, Pa., July 13 /PRNewswire/ — CSL Behring announced today that it has completed patient enrollment for a Phase III clinical trial of a 20 percent formulation of subcutaneous immunoglobulin (SCIg) to treat patients with primary immunodeficiency (PI) who require immune globulin replacement therapy. The study will assess the efficacy, tolerability, safety and pharmacokinetics of SCIg stabilized with proline (IgPro20) in subjects with PI. The open-label registration trial incorporates 13 sites across the United States, with 54 patients now enrolled.

“We are pleased to have completed enrollment in this important trial,” said Paul Perreault, Executive Vice President of Worldwide Commercial Operations at CSL Behring. “With this achievement, CSL Behring further secures its leadership position in advancing life-saving subcutaneous immune globulin therapies.”

Data from the trial will support a market application submission to the U.S. Food and Drug Administration.

Study Design

The IgPro20 study will evaluate the rate of serious bacterial infections (SBIs) in male and female subjects diagnosed with PI, ranging in age from 2 to 75 years old. SBIs are defined to include bacterial pneumonia, bacteremia and septicemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess. The trial will also aim to show non-inferiority in steady-state area under the curve (AUC) of immunoglobulin G (IgG) levels of weekly subcutaneous infusions of IgG with proline (IgPro20) compared to the previous 3 or 4 weekly intravenous treatments.

About Subcutaneous Immunoglobulin

In January 2006, CSL Behring introduced Vivaglobin(R) (Immune Globulin Subcutaneous [Human]) to patients in the US. Vivaglobin is the first and only FDA-approved subcutaneous IgG replacement therapy for treating patients with PI. With Vivaglobin, treatment is delivered directly under the skin (subcutaneously), offering a safe and effective alternative to intravenous infusions of immunoglobulin.

This treatment option is especially important for patients who do not easily tolerate administration by intravenous infusion because they have poor venous access or experience serious side effects. With physician approval, Vivaglobin is also appropriate for patients who want the freedom and convenience of safe home self-administration of IgG replacement therapy.

About Primary Immunodeficiency (PI)

Primary immunodeficiency is a usually genetic group of disorders affecting an estimated 50,000 Americans. These disorders compromise the immune system, leaving patients vulnerable to recurrent, life-threatening infections.

Immunoglobulin is a life-sustaining blood product that has become standard immune replacement therapy for most people diagnosed with PI; nearly 70% of PI patients receive Ig replacement therapy. Since the 1980s, the first-line therapy for most PI patients in the US has been intravenous immunoglobulin (IVIg), in which a pump delivers the immunoglobulin through a needle into the vein. Experience has shown IVIg to be an effective, safe, life-saving treatment. However, many patients do not easily tolerate intravenous infusions because they have poor venous access or they experience serious side effects. Vivaglobin allows patients to use a small, portable pump to self-administer their weekly subcutaneous infusions. Weekly administration of Vivaglobin provides greater stability steady-state serum Ig levels, with lower peak serum Ig levels and higher trough levels than those achieved with IVIg. The serum Ig profile for Vivaglobin is representative of that seen in a normal population.

Important Safety Information About Vivaglobin

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 immunoglobulin A 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 (Ig) 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. For more details and complete prescribing information on Vivaglobin, please visit http://www.vivaglobin.com/, or call the CSL Behring Medical Information Department at 1-800-504-5434.

About CSL Behring

CSL Behring is a global leader in the plasma protein biotherapeutics industry. Passionate about saving and 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 rare diseases such as 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 http://www.cslbehring.com/.

  Contacts: Sheila A. Burke

  Director, Communications & Public Relations

  Worldwide Commercial Operations, CSL Behring

  +1-610-878-4209

  Sheila.Burke@cslbehring.com

Website: http://www.cslbehring.com/
Website: http://www.vivaglobin.com/

linkback url: http://sev.prnewswire.com/health-care-hospitals/20070713/NYF05213072007-1.html


Calgary ‘Bubble boy’ loses battle

July 1, 2007

Calgary ‘Bubble boy’ loses battle

Paula Beauchamp, CanWest News Service; Calgary Herald
Published: Sunday, July 01, 2007

CALGARY — Courageous Calgary toddler Evan Pogubila has lost his battle against the rare SCIDs disorder, dying in the arms of his parents on Tuesday night.

Thirteen-month-old Evan, the only child in Western Canada with the rare immune system disorder known as “bubble boy disease,” had been plagued by infections, graft-versus-host disease and a cancer diagnosis.

The complications arose following a transplant operation at the Toronto Sick Kids Hospital in March.

Evan’s mom Melanie Pogubila on Saturday said the family felt devastated, but knew they were “blessed to have Evan for the short time he was with us”.

“Evan’s name means ‘little warrior’, and that’s exactly what he was,” she said.

“You’d go into Evan’s room and he’d have a smile for you, even when things were rough at the end.

Pogubila, who returned to Calgary this week with Evan’s twin sister Jordan and husband Frank, said the couple’s daughter is providing the emotional medicine the family needs.

“She’s a Godsend. She makes us smile, she’s still here and right now, that’s the best medicine for us,” Pogubila said.

Severe combined immunodeficiency syndrome is a genetic illness that leaves the body with little or no immune system to fight infection and makes simple colds as deadly as pneumonia.

Diagnosed in November, Evan lived for four months in a special germ-free room at the Alberta Children’s Hospital, isolated from the outside world.

His mother could not touch him with bare hands or let him play with her hair.

In March, Evan was transferred to the Toronto Sick Kids Hospital to undergo a rare bone marrow transplant after a perfect donor match was found.

The operation was Evan’s best hope of living a normal life and for the first 50 days he seemed to be recovering well as the bone marrow grew and engrafted.

But shortly before Evan’s first birthday, an infection set in.

In her heart-filled journal on http://www.caringbridge.org, Evan’s mom wrote in May, “Our little Evan is again proving what a strong little man he is.

“How I wish I could take his pain and make him all better.”

Evan’s grandfather, Rick Oncescu, said his grandson’s memory would always be alive for the family, especially through Jordan.

“She will always be here and we’ll always remember them as two,” he said.

Jen James, a critical care nurse who cared for Evan, said, “I wish we could have done more. It was a pleasure to look after Evan. Evan was a sweet baby and I wish I had known him when he was well.”

A memorial service will be held for Evan in Calgary on Tuesday.

pbeauchamp@theherald.canwest.com

Calgary Herald
© CanWest News Service 2007

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