Gene therapy gives hope to ‘boy in bubble’

Gene therapy gives hope to ‘boy in bubble’ TheStar.com – GTA – Gene therapy gives hope to ‘boy in bubble’

Parker DesLauriers and his parents return from Italy with renewed hope for the little boy with rare immune deficiency

September 28, 2007

Theresa Boyle
STAFF REPORTER
When Parker DesLauriers’ mother pushed his stroller through the arrivals gate at Pearson International Airport yesterday, throngs of adoring relatives and friends crowded around the tot, whom they smiled and waved at, but could not touch.

Blowing kisses and laughing from behind a plastic shield around his stroller, the chubby-cheeked 20-month-old boy looked the picture of health despite having a seriously compromised immune system.

Parker had just returned from Italy, where he spent the last six months undergoing experimental gene therapy, which his parents say has given him a fighting chance to beat the rare condition with which he was born, dubbed “boy-in-the-bubble” disease.

“We’re definitely on our way to success,” exclaimed his mother, Tracy Kulas-DesLauriers, 32.

“I totally believe it will work out in the end. And if for some reason it doesn’t, then we’ll do whatever we have to do to make sure it works later,” she added.

The Ajax lad was born with ADA-deficient severe combined immunodeficiency. Each of his parents unknowingly carried a copy of a defective gene, which they passed to him. So rare is the disease that the Hospital for Sick Children has treated only 15 patients with it in the last two decades.

Without treatment, Parker would probably die before age 2.

The therapy he received in Milan is not available in Canada. It involved harvesting his bone marrow, extracting stem cells and genetically altering them. The cells were then re-injected into the boy.

The treatment is Parker’s best hope for long-term survival.

“It’s successful so far. It’s a long-term thing and it’s going to take a while for us to figure out how well it worked,” explained Parker’s father, Kevin DesLauriers, 35.

The gene therapy trial is being conducted by Dr. Alessandro Aiuti and the results will eventually be published in a medical journal. In the meantime, Parker’s family is not at liberty to discuss some details of his treatment.

They said Parker suffered a setback shortly after the genetically altered stem cells were re-injected and at one point it was feared the treatment had failed.

“Even though he didn’t have the greatest (initial) response, he had a lot of cells given back to him, which is definitely an advantage. He has engrafted,” his mother said.

Parker will have to visit the Hospital for Sick Children every two weeks for blood tests and next spring, he will return to Italy for additional testing.

At Terminal 1 yesterday, the family’s relatives and friends welcomed them home with balloons, flowers and toys.

“You can see it. He’s so loved. He’s never played with his cousins,” remarked his mother, noting the boy is allowed very limited contact with others because of his compromised immune system.

“He’s not out of the woods yet,” said a teary-eyed Roy Kulas, Parker’s grandfather.

Kulas scrubbed his daughter’s home from top to bottom with Lysol in preparation for Parker’s return. The boy has to live in extremely sanitary conditions with few visitors.

“It’s probably the cleanest house in the whole of Toronto. You don’t want to take a chance,” Kulas said.

Only Parker’s grandparents are allowed to pick him up.

His parents always carry a bottle of sanitizer with them and there are other bottles in every room of their home.

They got nervous during the nine-hour trip home whenever they heard someone cough or sneeze.

But they were full of praise for Air Canada, for giving them free flights.

DesLauriers and Kulas-DesLauriers have turned their lives upside down for their son.

Both teachers, Parker’s parents took leaves from their jobs – his mother two years ago and his father 18 months ago.

Kulas-DesLauriers plans to stay home with Parker, and her husband is considering a new career in the computer field.

“That’s the stress now,” she said. “It’s a financial stress.”

Italian fundraising covered the cost of the gene therapy and the family’s accommodation. But they’ve still had to make their monthly mortgage payments and cover their own expenses.

The family has a blog documenting Parker’s journey and providing information on making financial donations. Visit the blog at: www.scidada.blogspot.com

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Family holds medical fundraiser for grandson with ‘bubble boy’ disease

Family holds medical fundraiser for grandson with ‘bubble boy’ disease

By Mary Gottschalk MediaNews San Jose Mercury News

Article Launched:09/27/2007 03:11:09 PM PDT

Relatives of Sebastian Westfall are rallying around the 5-year-old once again to host another fundraiser to defray costs of a new medical treatment that could extend his life. The event tonight will take place at his grandfather’s office on the sixth floor of 1900 The Alameda.Sebastian has a rare genetic disease called severe combined immunodeficiency (SCID), commonly known as “bubble boy disease.” He lacks an enzyme necessary for normal immune system function, making him vulnerable to infections that other children can easily fight off. Unlike David Vetter, another child born with SCID and the inspiration for the 1976 made-for-TV film “The Boy in the Plastic Bubble,” Sebastian doesn’t live in a bubble. However, he does live in a very controlled environment, and his contact with others is also carefully controlled. Born eight weeks prematurely on April 6, 2002, Sebastian spent the first three months of his life in hospitals. An early diagnosis identified Sebastian’s condition, allowing doctors to begin treatments that have prolonged his life. “When Sebastian was first born, we couldn’t take him out of the incubator. When he was in isolation, we had to scrub in and put on a mask. We couldn’t be sick. Sometimes my husband and I could just look through the window,” Lynette Westfall says. Sebastian’s SCID is linked to an adenosine deaminase enzyme deficiency. Currently he receives two shots a week in his thighs, and every four weeks he must have an IV infusion of antibodies. Additionally, he takes multiple antibiotics. In an effort to have Sebastian live to adulthood and beyond, his parents are constantly investigating treatments and seeking help everywhere they can. Their best hope seems to be a gene therapy clinical trial in Milan, Italy. After Sebastian was accepted into the trial, he and his parents flew to Milan on April 30. Although he passed all the pre-tests, there was a problem with the quality control on the batch of medicine that he was expected to use. As it takes weeks to months to make the medicine, which is spread throughout the body much like a virus, and to test it before use, the Westfalls returned to their Bay Area home on June 5. Now they are waiting to hear if the new medicine is good. If it is, they will return to Italy, perhaps by the end of September. The next phase of treatment in Milan is expected to be a minimum of four months. The cost of the therapy is expected to be at least $60,000, which is why Sebastian’s grandparents and other relatives and friends have been raising money for months. Trudy Jones initially crocheted scarves she sold for $25 to raise money for her grandson. Then her husband Doug opened up his Mortgage Magic office on The Alameda last March for a fundraiser that brought in $20,000. Grandmother Karlene Westfall organized a golf tournament, which brought in another $5,000. Now they are all working on the next fundraiser. “We have a good support team and they’re saying, let’s just keep going and raise money,” Karlene says. “If we get to the point where we have too much, we’ll give it to someone in this same situation that can use it.” Karlene says it’s important for people to know “all the good that is coming from this.” She points toward donations from people who don’t know Sebastian, but still give. “I want people to know a lot of good is going on in this world.”   The Sebastian Westfall Fundraiser is 5 to 8 p.m. tonight at Mortgage Magic, Suite 900, 1625 The Alameda. The event will include live music, hors d’oeuvres, wine tasting and a silent auction. Tickets are $45 per person. For additional information on Sebastian, visit http://www.sebastiansspace.blogspot.com. Contributions can also be made to the Sebastian Westfall Fund, c/o The First Republic Bank, 1625 The Alameda, Suite 100, San Jose, 95126.

Singapore joins global cord blood network

Sep 24, 2007
Singapore joins global cord blood network
This gives patients here another 60,0000 donor cords.
By Judith Tan

PATIENTS here suffering from several blood diseases will get a better chance in finding a perfect match for a life-saving transplant using stem cells from cord blood.

Singapore’s first public cord blood bank has become a member of the National Marrow Donor Program (NMDP), a global network of cord blood banks, opening up patients here to another 60,000 donor cords.

At the same time, ethnic Asians in the West and other parts of the world can seek a match from the more than 2,500 cords banked here.

Dr Jeff Chell, the Chief Executive Officer of NMDP revealed that already there has been a request from an Asian American suffering from leukaemia to the Singapore Cord Blood Bank for a match.

SCBB is the third international cord blood bank outside of the US to become a member of NMDP, after that in Taiwan and Israel.

Blood in the umbilical cord is a rich source of immature cells that can develop into a wide range of different blood cells and can be used effectively to replace diseased ones.

Since the SCBB opened its doors two years ago, there have been two recipients of stem cell transplant from unrelated cord blood donations. The first was Hoh Sin Jun, two, who was diagnosed with severe combined immunodeficiency, sometimes referred to as ‘bubble boy disease’. His transplant operation took place in May last year at KK Women’s and Children’s Hospital.

The second recipient was Jayjay, five. He was diagnosed with Hyper-IgM Syndrome, a different type of immunodeficiency that causes frequent infections of the ears, eyes, sinuses, lungs, skin, respiratory tract and other areas of the body. He went through his transplant at the National University Hospital last month.

linkback url: http://www.straitstimes.com/Latest+News/Singapore/STIStory_160834.html

Mother gives son new life with bone marrow transplant

Mother gives son new life with bone marrow transplant

Naplesnews.com

 By Liz Freeman

Thursday, September 20, 2007

He’s only 5 months old and already he’s experiencing a new start in life.

Adam Saada, who was diagnosed in August with a severe immune deficiency when he was 3 months old, underwent a bone marrow transplant earlier this week with his mother, Amy Saada, as his donor.

The first 30 to 90 days are critical in whether he will accept the stem cells in the bone marrow to develop a new immune system and save his life. Adam’s family lives in Naples.

So far, so good, said Steve Tozier, Adam’s grandfather.

“Adam is doing very well,” Tozier said earlier this week, following the transplant that began Tuesday and wrapped up early Wednesday at Jackson Memorial Hospital in Miami.

Adam’s mother was not a perfect donor match but the DNA and other factors were close enough, he said.

She was being discharged from the hospital to an apartment nearby and Adam must stay in the hospital for at least 30 days and possibly up to 90 days, said Sue Buck, Adam’s great aunt.

“Everything seems to be going well,” Buck said. “We’re just keeping our fingers crossed.”

The family is insured but the cost of the transplant, aftercare treatment and medications, along with disruption to the family’s life is a big financial hardship. Donations have been steadily coming in and fundraisers are scheduled to help toward the goal of raising $250,000.

The community already has been generous with $41,000, said Sharon Ardrey, a family friend who has been handling a trust account toward Adam’s medical expenses.

Donations have been coming in from as far as California and Ontario, along with great outpouring locally, Ardrey said.

“It’s been wonderful,” she said.

Adam was born April 3 and was fine until early August when he became sick and was taken to North Naples Hospital. He was flown to Joe DiMaggio Children’s Center in Hollywood, where he was diagnosed with a rare type of pneumonia that only occurs in people with low immune systems.

Shortly afterward, he was diagnosed with Severe Combined Immunodeficiency, SCID, and was transferred to Jackson Memorial in Miami. His rare medical disorder is the same as that of David Vetter, the boy in the 1970s who lived in a plastic germ-free bubble for 12 years and became known as “the Bubble Boy.”

The Saada family has linked up with the Children’s Organ Transplant Association (COTA), a national charity dedicated to helping communities organize fundraisers for hometown children who need life-saving transplants.

Adam’s father, Hussam Saada, works at the Pine Ridge Road branch of Wachovia bank and the branch is matching for every $25 that’s donated, Tozier said. His own employer, the Wyndemere Home Owners Association, and its residents have donated at least $15,000, he said. Ardrey said she’s been receiving donation checks nearly every day from Wyndemere residents and they may have donated as much as half of what’s been raised so far.

Amy Saada works for Collier County’s Department of Human Services and has taken a leave of absence Adam’s illness.

A fundraiser will be held in the parking lot of Gina’s Cafe, 11000 Commercial Blvd., on Sunday, Oct. 21, from 2 to 6 p.m.

“They are donating all of the food,” Ardrey said of the cafe, where one of the owner’s sisters has a son who was hospitalized at Jackson Memorial.

Tickets for the buffet are $10 per person and there will be a live band, a clown, crafts and a silent auction, Ardrey said.

Another event is an online Pampered Chef party, where proceeds will go to Adam’s care. The online event is http://www.pamperedchef.biz/melskitchen and enter Sharon Ardrey in Trust for Adam Saada in the space for “organization name.”

Since the family has linked up with COTA, the nonprofit charity is handling donations for him. Tax-deductible checks can be sent to “COTA for Adam S.” at 2051 Cota Drive, Bloomington, Ind., 47403. All the proceeds goes to Adam’s medical expenses, Ardrey said.

To keep up with how he is progressing, visit his Web site, http://www.cotaforadams.com.

© 2007 Naples Daily News and NDN Productions. Published in Naples, Florida, USA by the E.W. Scripps Co.

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A first in home treatment

A first in home treatment
Children’s hospital leading the way. New infusion product means fewer side effects, less fatigue for kids with rare genetic disorder
AARON DERFEL, The Gazette
Published: Friday, September 14

Since he was stricken with severe pneumonia at age 4, Akeel Knight-Lee has been very careful not to catch the slightest infection.

That’s because the boy, now 10, has a rare genetic disorder called X-linked agammaglobulinemia. The disease has robbed his immune system of the ability to make antibodies that fight infection.

For six years, Akeel has had to spend one day a month at the Montreal Children’s Hospital, hooked up to an intravenous line so he can be infused with immunoglobulin – a solution of pooled antibodies from blood donors.
Katherine Knight with her son Akeel at their Dollard des Ormeaux home. Akeel Knight-Lee has a rare immunodeficiency and used to have to go to the Children’s Hospital for treatment that left him weakened. Now, he receives treatment at home and has more energy.View Larger Image View Larger Image
Katherine Knight with her son Akeel at their Dollard des Ormeaux home. Akeel Knight-Lee has a rare immunodeficiency and used to have to go to the Children’s Hospital for treatment that left him weakened. Now, he receives treatment at home and has more energy.

The treatment is lifesaving, but it can cause side effects like seizure-inducing headaches and nausea in about 25 per cent of cases. And everyone who receives the treatment – regardless whether they suffer from the side effects – feels sapped of energy for about three weeks after the infusion.

“Akeel would say to me, ‘Mommy, when do I have to go to the hospital?’ ” Katherine Knight recounted. “And I would say, ‘Next week.’

“Oh,” Akeel would respond dejectedly. “I’m feeling so tired.”

But now, thanks to the pioneering efforts of the Children’s, Akeel and others with similar disorders now can infuse themselves in the comfort of their homes. And since the treatment is weekly, their energy levels never drop.

“We’re so happy,” Akeel’s mother said. “His energy level is high. He played soccer in the summer.”

Akeel is one of about 25 children with primary immuno-deficiency who are followed by the Children’s, the first hospital in Canada to set up the at-home treatment.

Primary immunodeficiency is the umbrella name for a range of 20 to 30 genetic disorders. The disorders are not like AIDS, which is a secondary immuno-deficiency. Since the mid-1980s, people with primary immuno-deficiency have been treated with replacement immunoglobulin or antibodies.

The driving force behind the at-home treatment is Christine McCusker, a pediatric immunologist. In an interview, she said she wasn’t satisfied with the in-hospital treatment, not only because it could produce side effects, but because it proved to be a major inconvenience for the children and their parents.

“Can you imagine a 4-year-old having to come into the hospital, get picked (with a needle) three times to have his IV started and then sit for four hours while he gets his infusion?” she asked.

“Mummy has to take time off work and the child has to be off from his usual activities. And if he’s older, he has to miss school for that day.”

So McCusker made some inquiries and discovered nearly three years ago that a group of physicians in Britain had switched to a new infusion product that could be used at home. The treatment, Vivaglobin, does not require an intravenous line, but is delivered subcutaneously – that is, just under the skin.

A child’s mother can inject the needle into the belly and, over the course of an hour, deliver an ounce (about 28 grams) of the antibodies. The only side effects are redness at the site of the injection and a bulge that goes away after a few hours.

At first, Health Canada approved the treatment on a case-by-case basis. But last year, Vivaglobin was approved for use without special authorization.

Children’s hospital leading the way. New infusion product means fewer side effects, less fatigue for kids with rare genetic disorder

And since the Children’s developed expertise with the treatment, it’s now a training centre for nurses from hospitals across the country.

“When you can make a small change that improves a child’s life, it’s the greatest feeling in the world,” McCusker said.

“Now, not only do these children not have to come to hospital, with all the attendant problems, but they become independent sooner and it’s better for their self-esteem and their social development.”

Katherine Knight said she’s greatly relieved that her son no longer misses school exams and field trips.

For Akeel, who wants to be a doctor, the at-home treatment is simply fun.

“I can just relax,” he said, “and play games like Transformer.”

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MIT Researchers Develop Better Way to Deliver Gene Therapy

MIT Researchers Develop Better Way to Deliver Gene Therapy

Researchers at MIT have taken a big step toward developing safe and effective methods for gene therapy. They have found a way to refine the ability of biodegradable polymersto deliver the genes

Gene therapy involves the inserting of new genes into the cells of patients. It is used to combat diseases like cancer and it great promise, but in order to reach its full potential the researchers need to be able to set aside the concerns of the safety level of using viruses to transport the genes.

This research focused on creating a new type of carrier for the gene from synthetic, non-viral material.

Their aim was to take a safe biocompatible, degradable polymer and make it more effective instead of starting out with a virus and trying to make it safer.

Even thought gene therapy had been researched for close to 20 years, and there have been more than 1,000 clinical trials there still are no FDA approved gene therapies. In most of the trials viruses are used as carriers-vectors- to deliver the genes. But there are risks that are associated with using the viruses. Researchers have been working on the development of non viral delivery methods.

In this particular study the scientists at MIT focused their attention on three chains of alternating amine- an organic compound- and diacrylate- crosslinking agent between the molecular chains of polymers- groups. These groups had previously shown potential to be gene carriers. They hope to make them even more efficient by modifying them at the very ends of the chains.

When these polymer are mixed together, they can spontaneously mix with DNA to make nanoparticles. These can act in certain ways like an artificial virus and thereby deliver functional DNA when it is injected either right into or near the target. They have already been shown safe in mice and the next step will be to run clinical trials.

No viral vectors like these could turn out to be not only safer than the viruses are but they may be able to avoid the immune system , which would allow for multiple applications.

One of the specific line of research that MIT is pursuing along with Janet Sawicki at the Lankenau Institute for Medical Research involves ovarian cancer. With this, they have found that these polymer-DNA nanoparticles are able deliver DNA at high levels to ovarian tumors without harming healthy tissue.

The team is led by Daniel Anderson, research associate in MIT’s Center for Cancer Research. First co author is Jordan Green, a graduate student in biological engineering as is Gregory Zugates, a former graduate student in chemical engineering now at WMR Biomedical, Inc. The rest of the team are Nathan Tedford, a former graduate student in biological engineering now at Epitome Biosystems; Linda Griffith, professor of biological engineering; Douglas Lauffenberger, head of biological engineering, and Institute Professor Robert Langer. Sawicki and Yu-Hung Huang of the Lankenau Institute.

The research was funded by the National Institutes of Health, the Department of Defense and the National Science Foundation.

Sosurce: MIT http://web.mit.edu/

linkback url: http://www.associatedcontent.com/article/373012/
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Midlander hopes to see residents at bone marrow registration Tuesday

Midlander hopes to see residents at bone marrow registration Tuesday
By Tony Lascari
09/10/2007
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It might be easier to help save a life than most people think.

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Ron Main of Midland got his miracle in 2001, when a donor he didn’t know offered bone marrow for a transplant used in treating Main’s cancer. Main had waited three months to find a match willing to help, and now works to spread the word that donors are needed, and it’s easy to sign up.

“It’s hard to find someone to become a donor when push comes to shove, and really, you need them to save your life,” he said. “The more people you have in the worldwide bank, the chances are better you’re going to find the help you need or for a family member in need.”

Main, 62, will be at a Michigan Community Blood Centers’ blood drive Tuesday at the Midland Mall to talk about his experiences. The drive is scheduled from 12:30 to 7:30 p.m., and will include the chance to sign up on the bone marrow registry.

“People don’t understand that there’s nothing else they have to do but sign a card,” he said.

His story took a twist when a grandson, 2-year-old Jacob Main, also needed a transplant. Jacob had Severe Combined Immunodeficiency Syndrome and also found a donor willing to help.

“My little guy, my grandson, is doing extremely well and he’s going to live a normal life by all indications,” Main said.

He said research advanced treatments between his transplant and his grandson’s five years later.

While Main’s cancer cannot be cured, it has become under control because of his treatment.

“Fortunately in my case, one person was available and was willing to be my donor,” he said.

He was surprised to learn that most donors wish to remain anonymous. His did not, and the two eventually met and have stayed in contact.

“It was just utterly amazing to get to know him, his wife and his two sons, and they’ve since adopted a daughter,” he said of the Rockford resident who gave to help him continue living his life.

Main has spent time talking with people about the need for donors and how the process works, hoping to decrease people’s wait for assistance. He’s also been involved in the Midland County Relay for Life and the Midland Community Cancer Services golf tournament.

“It’s been really rewarding,” he said. “Part of the idea of my involvement (on Tuesday) is you can in fact save someone’s life.”

linkback url: http://www.ourmidland.com/site/
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Baxter and Halozyme Announce Collaboration for Development of Subcutaneous GAMMAGARD LIQUID(

Baxter and Halozyme Announce Collaboration for Development of Subcutaneous GAMMAGARD LIQUID(TM) Administration Using Enhanze(TM) Technology
PR Newswire
Development of New Route to Administer GAMMAGARD LIQUID(TM) 10% Could Make Home Therapy More Accessible; Collaboration Valued at Up to $47 Million
September 10, 2007: 08:00 AM EST

DEERFIELD, Ill. and SAN DIEGO, Sept. 10 /PRNewswire-FirstCall/ — Baxter International Inc. and Halozyme Therapeutics, Inc. announced today they have entered into an agreement to apply Halozyme’s proprietary Enhanze(TM) Technology to the development of a subcutaneous route of administration for Baxter’s GAMMAGARD LIQUID(TM) 10% [Immune Globulin Intravenous (Human)] (IGIV) (known as KIOVIG(TM) in Europe). For patients using GAMMAGARD LIQUID 10% — currently administered intravenously — subcutaneous administration with Enhanze Technology may increase overall convenience and improve the dispersion of the therapy.

Under the terms of the agreement, Baxter will provide Halozyme an initial upfront payment of $10 million. Pending successful completion of a series of regulatory and sales milestones, Baxter may make further milestone payments totaling $37 million to Halozyme. Baxter also will assume development, manufacturing, clinical, regulatory, sales and marketing costs. Halozyme will be responsible for the supply of the recombinant human hyaluronidase (rHuPH20) enzyme that provides the basis for Enhanze Technology. Upon regulatory approval, Halozyme will also receive royalties on GAMMAGARD LIQUID 10% administered subcutaneously using Enhanze Technology both in kits and in co- formulations.

Baxter’s GAMMAGARD LIQUID 10% is a large molecule therapy made from human plasma that is indicated for the treatment of primary immunodeficiency disorders associated with defects in immune system. Enhanze Technology is Halozyme’s proprietary drug delivery technology based on rHuPH20. rHuPH20 is a form of human enzyme that temporarily clears space in the matrix of tissues underlying the two outer layers of the skin to increase absorption and spreading of injected drugs.

“By using Enhanze Technology to facilitate a subcutaneous route of administration for GAMMAGARD LIQUID, patients with immunodeficiency disorders could benefit from access to home therapy that may potentially allow full IV dosing with the convenience of subcutaneous delivery,” said Hartmut J. Ehrlich, MD, vice president of global research and development for Baxter’s BioScience business. “This partnership represents Baxter’s ongoing scientific innovation and investment in enhancing administration of GAMMAGARD LIQUID therapy.”

“We believe our technology can enhance the clinical benefits that biologics have already been shown to provide. We are very excited to expand our relationship with Baxter so that they may make this important product available to as many people as possible worldwide as a way to simplify the delivery of medications and fluids and, ultimately, provide a more comfortable patient experience,” said Jonathan Lim, MD, president and CEO of Halozyme.

In February 2007, Baxter and Halozyme expanded their ongoing collaboration to commercialize product combinations of HYLENEX(TM) recombinant (hyaluronidase human injection) with certain Baxter proprietary and non- proprietary small molecule therapies. HYLENEX is a formulation of rHuPH20, supplied by Halozyme, indicated to facilitate the absorption and dispersion of other injected drugs or fluids. HYLENEX is manufactured by Baxter Healthcare Corporation for Halozyme.

About Enhanze(TM) Technology

Enhanze Technology is Halozyme’s proprietary drug delivery technology based on rHuPH20, a recombinant form of the naturally occurring human enzyme approved by FDA for its ability to break down hyaluronic acid, the space- filling “gel”-like substance that is a major component of tissues throughout the body. When combined or co-formulated with certain injectable drugs, Enhanze Technology can facilitate the penetration and dispersion of these drugs by temporarily opening flow channels under the skin. Molecules as large as 200 nanometers may pass freely through the perforated extracellular matrix, which recovers its normal density within approximately 24 hours, leading to a drug delivery platform that does not permanently alter the architecture of the skin.

About Baxter’s GAMMAGARD LIQUID(TM) 10%

GAMMAGARD LIQUID 10% (known as KIOVIG(TM) in Europe) is indicated for the treatment of primary immunodeficiency disorders associated with defects in immune system. These include but are not limited to congenital X-linked agammaglobulinemia, common variable immunodeficiency, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies.

Important Safety Information for the United States

GAMMAGARD LIQUID 10% is contraindicated in patients with known anaphylactic or severe hypersensitivity responses to Immune Globulin (Human). Patients with severe selective IgA deficiency (IgA < 0.05 g/L) may develop anti-IgA antibodies that can result in a severe anaphylactic reaction.

Immune Globulin Intravenous (Human) products have been reported to be associated with renal dysfunction, acute renal failure, osmotic nephrosis, and death. Patients predisposed to acute renal failure include patients with any degree of pre-existing renal insufficiency, diabetes mellitus, age greater than 65, volume depletion, sepsis, paraproteinemia, or patients receiving known nephrotoxic drugs. Especially in such patients, IGIV products should be administered at the minimum concentration available and the minimum rate of infusion practicable. While these 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. Glycine, an amino acid, is used as a stabilizer. GAMMAGARD LIQUID 10% does not contain sucrose.

Components used in the packaging of this product are latex-free.

GAMMAGARD LIQUID 10% is made from human plasma. It may carry a risk of transmitting infectious agents, e.g. viruses, and theoretically, the Creutzfeldt-Jakob disease (CJD) agent.

Thrombotic events have been reported in association with IGIV. Patients at risk may include those with a history of atherosclerosis, multiple cardiovascular risk factors, advanced age, impaired cardiac output, and/or known or suspected hyperviscosity, hypercoagulable disorders, and prolonged periods of immobilization.

IGIV products can contain blood group antibodies that may cause a positive direct antiglobulin reaction and, rarely, hemolysis.

Aseptic meningitis syndrome (AMS) has been reported to occur infrequently in association with IGIV treatment. Discontinuation of IGIV treatment has resulted in remission of AMS within several days without sequelae.

Various mild and moderate reactions, such as headache, fever, fatigue, chills, flushing, dizziness, urticaria, wheezing or chest tightness, nausea, vomiting, rigors, back pain, chest pain, muscle cramps, and changes in blood pressure may occur with infusions of Immune Globulin Intravenous (Human).

For full prescribing information, please go to http://www.gammagardliquid.com.

Important Safety Information for HYLENEX(TM) recombinant

Hypersensitivity to hyaluronidase or any other ingredient in the formulation is a contraindication to the use off this product. Discontinue HYLENEX recombinant (hyaluronidase human injection) if sensitization occurs. Hyaluronidase should not be used to enhance the absorption and dispersion of dopamine and/or alpha agonist drugs. Hyaluronidase should not be injected into or around an infected or acutely inflamed area because of the danger of spreading a localized infection. Hyaluronidase should not be used to reduce the swelling of bites or stings. Hyaluronidase should not be applied directly to the cornea. Hyaluronidase should not be used for intravenous injections because the enzyme is rapidly inactivated.

Furosemide, the benzodiazepines and phenytoin have been found to be incompatible with hyaluronidase. The most frequently reported adverse experiences have been local injection site reactions. Hyaluronidase has been reported to enhance the adverse events associated with co-administered drug product. Allergic reactions (urticaria or angioedema) have been reported in less than 0.1% of patients receiving hyaluronidase. Anaphylactic-like reactions following retrobulbar block or intravenous injections have occurred, rarely. When hyaluronidase is added to a local anesthetic agent, it hastens the onset of analgesia and tends to reduce the swelling caused by local infiltration, but the wider spread of the local anesthetic solution increases its absorption; this shortens its duration of action and tends to increase the incidence of systemic reaction. Edema has been reported most frequently in association with hypodermoclysis.

The full prescribing information for HYLENEX should be consulted prior to prescription or administration. For full HYLENEX prescribing information, visit http://www.HYLENEX.com.

About Baxter International Inc.

Baxter International Inc., through its subsidiaries, assists healthcare professionals and their patients with the treatment of complex medical conditions, including cancer, hemophilia, immune disorders, kidney disease and trauma. The company applies its expertise in medical devices, pharmaceuticals and biotechnology to make a meaningful difference in patients’ lives.

About Halozyme Therapeutics, Inc.

Halozyme is a biopharmaceutical company developing and commercializing products based on the extracellular matrix for the drug delivery, oncology, and dermatology markets. The company’s portfolio of products is based on intellectual property covering the family of human enzymes known as hyaluronidases. The company’s Enhanze Technology is a novel drug delivery platform designed to increase the absorption and dispersion of biologics. In addition, the company received FDA approval for two products: Cumulase(R) and HYLENEX, for use as an adjuvant to increase the absorption and dispersion of other injected drugs and fluids. The company also has a number of different enzymes in its portfolio that are targeting significant areas of unmet need.

This release includes forward-looking statements concerning the companies’ expectations related to the expanded relationship between Halozyme and Baxter. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: the anticipated timing of regulatory filings and the success of certain regulatory events regarding gaining regulatory approval for the products described in this release including those products under development; the effectiveness of the products described in this release including those products under development; the ability of Baxter to effectively capitalize on drug-delivery platform opportunities in existing and new markets; demand for and market acceptance for the products described in this release including those products under development; the impact of competitive products and pricing, including generic competition, drug re-importation and disruptive technologies; internal and external factors that could impact commercialization; and other risks discussed in each company’s filings with the Securities and Exchange Commission (SEC) that could cause actual results to differ materially from those in the forward-looking statements. Each company’s SEC filings are available on its respective website. The companies do not undertake any obligation to update any forward-looking statements as a result of new information, future events, changes assumptions or otherwise, and all forward- looking statements speak only as of the time when made. Actual results or experience could differ materially from the expectations contained in the forward-looking statements.

HYLENEX recombinant, GAMMAGARD LIQUID and KIOVIG are trademarks of Baxter Healthcare Corporation.

Cumulase and Enhanze are trademarks of Halozyme Therapeutics, Inc.

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