Help at Last

Courtesy: inquirer.net

Novartis Oncology helps cancer patients have long-term access to life-extending therapies through its Glivec International Patient Assistance Program (GIPAP), one of the most comprehensive and far-reaching cancer patient access programs ever implemented on a global scale.

Since its introduction in 2002, GIPAP has provided the breakthrough treatment Glivec (imatinib) at cost to more than 35,000 cancer patients in more than 80 countries. Glivec is a proven-effective treatment for Philadelphia positive chronic myeloid leukemia (Ph+CML) and gastrointestinal stromal tumor (GIST). GIPAP and the Novartis Oncology Program (NOA) have helped provide Glivec to over 1,400 socially disadvantaged Filipino cancer patients.

From 2006 to 2008 alone, benefits provided by the NOA program to Filipino patients are valued at over P2 billion. This innovative program is being implemented in more than 65 participating centers across the archipelago. Today, GIPAP has evolved into the broader NOA program which continues to serve Filipino patients already enrolled in GIPAP and an additional 120-plus more nationwide.

Another life-saving treatment developed by Novartis Oncology is Exjade (deferasirox). Exjade is used for the treatment of chronic iron overload due to frequent blood transfusions in patients aged six and older with beta thalassemia major. This is an inherited blood disorder that causes severe anemia and bone deformities. Untreated, it can lead to death before the age of 20.

Novartis is exploring ways to increase Filipino patients’ access to thalassemia treatments.

Thalassemia Ambassador Program to be Launched

Courtesy by: tempointeractive.com

TEMPO Interactive, Jakarta: Orange for Kids, an organization in the field of education by and for people with thalassemia or hereditary blood disease, will launch the Thalassaemia Ambassador program on December 30 at the president’s home in Puri Cikeas.

The organization is cooperating with PT Novartis Indonesia, the Child Hematology Team of Cipto Mangunkusumo Hospital, Hasan Sadikin Bandung Hospital, Tangerang General Hospital, and the Indonesian Parents of Thalassemia Patients Association. The Thalassemia Ambassador Program is aimed at educating people of this disease and the importance of blood screening.

”As for Thalassemia patients, the program is aimed at sharing motivation and tips and tricks of living with thalassemia,” said Wanda Harahap, PT Novartis’ Communication Manager, contacted by Tempo via the telephone.

Cardiac Function Continues to Improve in Beta-Thalassemia Patients After 2 Years on Iron Chelation With Deferasirox

Courtesy by: docguide.com

NEW ORLEANS — December 8, 2009 — Patients diagnosed with cardiac iron overload in Beta-Thalassemia continued to show functional improvement after 2 years of treatment with the iron chelator deferasirox, researchers stated here at the American Society of Hematology (ASH) 51st Annual Meeting and Exposition.

“This is the first large prospective study to report 2-year data on cardiac iron removal for any iron chelator,” said Dudley J. Pennell, MD, Royal Brompton Hospital, London, United Kingdom, during his poster presentation on December 7.

The effect of deferasirox on cardiac function was measured using magnetic cardiac imaging, focusing on the relaxation parameter T2*. Cardiac T2* values <20 ms indicate cardiac iron overload. Values <10 on the T2* scale indicate patients have a likelihood of experiencing heart failure and arrhythmia, according to Dr. Pennell.

He reported 2-year results of the Evaluation of Patients’ Iron Chelation With Exjade (EPIC). Deferasirox is a once daily oral iron chelator. He reported that favourable changes in heart function as measured by T2* assessment that were seen after the first 12 months of treatment were continued out to 24 months.

Among the 39 Beta-Thalassemia patients whose baseline T2* scores were <10 ms, the T2* score rose from 7.3 ms at baseline to 8.1 ms at 1 year (P < .001) and reached a mean of 9.5 ms for the 29 patients who were evaluable after 2 years (P < .001).

Among the 62 patients in the study who had baseline T2* scores between 10 and 20 ms, their mean baseline value of 14.6 ms improved to 17.5 ms at 12 months (P < .001) and reached 20.4 ms after 24 months (P < .001).

The patients also showed improvement in right ventricle function. Dr. Pennell suggested that improvement “is best explained by improved left ventricle and right ventricle compliance associated with reduced cardiac iron, and may be an early marker of functional improvement.”

Right ventricle ejection fraction increased from 66% at baseline to 68.8% at 1 year and 69.9% after 2 years (P < .001 of both scores compared with baseline values).

No drug-related serious adverse events were reported in the 1-year extension. Two serious adverse events seen in the first year of treatment — an episode of renal failure and renal tubular disorder — resolved following drug discontinuation.

About 30% of the participants in the study were aged under 16 years; the rest were aged less than 50 years.

“Results show that continued therapy with deferasirox for up to 2 years at doses of 30 to 40 mg/kg/day was effective in removing iron from the heart in Thalassemia major patients with mild, moderate and severe cardiac siderosis,” Dr. Pennell said.

Funding for this study was provided by Novartis Pharma AG.

He’s here to heal

Courtesy by: statesville

When nine-year-old Khai arrived in America 10 weeks ago, he had little idea that up to three years of his life might be spent in Mooresville with a family giving their all to see the child return to Afghanistan healthy.

Khai was one of nearly three dozen kids from the war-torn country of Afghanistan to come to the Mooresville area June 23 through the nonprofit Solace for the Children. With a motto of “Building peace on a foundation of health,” the organization aims to bring children to the area for various medical treatments that aren’t widely available or affordable in their home countries.

And although many Afghan children came to North Carolina this summer with vision problems or hearing difficulties that could easily be aided in the six-week timeframe, several others arrived with more serious ailments requiring extensive care.

Khai, who has Beta Thalassemia Major and iron overload, was among the latter. But his diagnosis was too life-threatening to send him home in early August with the rest of the Afghan children.

Rather, he remains with his American foster family – the Ayris family of Mooresville, whom he met for the first time in June – for at least the next year to help extend his chances of survival.

Up to three years, however, could be needed before sending Khai back to his family in Afghanistan.

“We realized that his returning home would be detrimental to his health,” said Heather Ayris, who — with her husband, Aaron, and two sons — have welcomed Khai into their family.

Beta Thalassemia Major is a genetic blood disorder that causes the hemoglobin to become unhealthy, requiring frequent blood transfusions, which Khai has received since he was a baby, said Ayris.

“Due to the many transfusions needed to keep him alive, his iron levels are now dangerously high,” she said. “If they remain high, it will cause damage to his organs and the risk for cardiac arrest is the greatest concern.”

Ayris said Khai is unable to excrete the excess iron and is experiencing metal poisoning within his body as a result.

Three of Khai’s siblings have died from the same disorder. A younger sister is also afflicted.

“It’s so much larger scale that a traditional anemia,” said Ayris, adding that Khai ultimately requires a bone marrow transplant to help save his life. The family should find out by October if the nine-year-old is a candidate for the transplant. If he is, the length of his stay in Mooresville will extend to three years, at the least.

Until then, Khai is undergoing blood transfusions every three weeks – the next is scheduled for Tuesday – and receives nightly chelation therapy to remove the toxic iron from his body, Ayris said.

Despite knowing the extreme expense these treatments and a potential bone marrow transplant would cost – approximately $270,000 in all – Ayris said she and her family were committed from the start to helping the youngster as much as they could when they offered their home, hearts and money toward making him healthy.

And less than 48 hours before Khai was scheduled to return to Afghanistan with the other kids from Solace for the Children, the official word came from the embassy approving his stay in the United States.

“He thought he was going home. We were packing his suitcase and everything because we didn’t know,” said Ayris, adding that an interpreter had to tell Khai he’d be remaining with the family for another year.

“Khai has become close to our family in such a short time and we could no sooner send him back home to die than we could one of our own biological children,” Ayris said in a letter to friends seeking prayers and monetary donations when their journey began in early August.

With at least a $270,000 bill looming for Khai’s medical care, the Ayris’ reached out to members of the community and other agencies who might offer their assistance.

“Every time we’ve needed something, a door has been opened to us,” she said, noting that the $70,000 for Khai’s treatments over the next year have been paid for by the Patient Assistance Program of Novartis, the company that makes one of Khai’s medications.

“At this point, our attention is turning to try to get him the bone marrow transplant that he needs to cure him, finding the funding and doctors and hospitals to get that.”

At a cost of $200,000, Ayris said her family not only needs to seek donations, but has to hope and pray Khai is both a candidate and a transplant match can be found to save his life.

In the meantime, Khai’s life in America resembles that of a typical nine-year-old.

Enrolled in the third grade at Lake Norman Elementary, Khai attends classes with his new “brother,” eight-year-old AJ Ayris. The two kids are in the same class and Khai daily works through some English as a Second Language courses as he swiftly picks up the language.

“He understands probably 95 percent of what we say to him now,” Heather Ayris said, noting that Khai knew very little English when he first arrived. “He’s just a sponge. He’s been able to learn so much very quickly.”

Like AJ and seven-year-old Cade, Khai has taken up Tae Kwon Do.

“He’s watched AJ and Cade train over the past several weeks and we could tell he was ‘itching’ to join in on the fun,” Ayris said in an online update to family and friends through Caring Bridge, which offers “free, personalized Web sites that support and connect loved ones during critical illness, treatment and recovery.”

And as Ayris began registration for Khai, she discovered his membership for an entire year had already been anonymously paid for.

Calling the Afghan youngster “a part of the family,” Ayris said her two sons and Khai have become brothers since he arrived, which has made the transition much easier for all of them. But, unfortunately, it will also make his eventual departure extremely difficult.

“We’ll have to cross that bridge if and when we get to it. I would think he’d miss us just as much as we’ll miss him.”

She added, “It will definitely be a bittersweet moment because he has a family that loves him. He’s not going back to people that don’t care for him.”

She said Khai has been able to speak to his father about once a week and the two families have begun exchanging emails with updates on Khai’s health and daily life.

“He’s such a brave boy that sometimes you forget that he’s just a nine-year-old child,” Ayris added. “He’s surrounded by war (at home).”

And although he will eventually return to Afghanistan, Ayris said his immediate medical needs remain the primary concern.

With a variety of hurdles already overcome, Ayris said she and her family believe “the grace of God” has allowed each small miracle in this process to occur.

“If we just had 200,000 people give $1, that would save his life,” she mentioned. “He’s a child of God and we truly feel very called that god has called us to help.”

Want to help?

To donate, visit http://www.solaceforthechildren.org and utilize the “add special instructions to the seller” tab to write Khai’s name and ensure the donation helps his specific needs.

Also, cash donations or checks – with Khai’s name in the memo line – can be mailed to SOLACE for the Children, PO Box 65, Davidson, NC 28036.

(PR)Exjade* benefits chronically transfused patients with rare anemias by significantly reducing toxic iron that can damage key organs, according to landmark trial

Courtesy by: cnw.ca
– First prospective, multicentre study to show Exjade(*) removes iron from the heart in beta-thalassemia patients with mild to severe cardiac iron overload.
– In a subgroup analysis of 341 patients with myelodysplastic syndromes (MDS), Exjade(*) significantly reduced levels of toxic iron.
– These results are part of the largest prospective trial in iron chelation, which includes more than 1,700 patients with various transfusion-dependent anemias, including other rare anemias

Dorval, QC, Dec. 9 /CNW/ – New data from the largest prospective trial in iron chelation demonstrate the efficacy and safety of Exjade(*) (deferasirox) in treating chronic transfusional iron overload, a potentially life-threatening condition for patients who have had multiple blood transfusions to treat
underlying anemias, including beta-thalassemia, myelodysplastic syndromes (MDS) and other rare anemias.

Data from this landmark trial, known as EPIC, were presented today at the 50th American Society of Hematology (ASH) Annual Meeting and Exposition in San Francisco, California.

The EPIC cardiac substudy showed that Exjade(*) removed iron from the heart in beta-thalassemia patients, based on a statistically significant improvement in T2(*) magnetic resonance imaging, a validated technique to assess cardiac iron content (P less than 0.0001). The one-year substudy included 114 beta-thalassemia patients with cardiac iron overload, the leading cause of
death in these patients.

“These data clearly demonstrate that deferasirox significantly reduces cardiac iron in beta-thalassemia patients with iron overload, which is a critical goal of treatment for these patients,” said Dudley Pennell, MD, Professor of Cardiology, Royal Brompton and Harefield NHS Trust and Imperial College, London. “Cardiac complications caused by the buildup of toxic iron in the heart can be life-threatening for people living with thalassemia.”

A pre-planned analysis of 341 MDS patients enrolled in the study showed that Exjade(*) significantly reduced levels of serum ferritin (SF), a key measure of iron in the body, by 253.0 ng/mL from baseline (P=0.0019). Of the 171 MDS patients whose SF was measured at one year, the decrease from baseline
was 606 ng/mL.

“Many MDS patients receive regular blood transfusions as part of their ongoing treatment, which puts them at risk for iron overload,” said Norbert Gattermann, MD, PhD, Hematology, Oncology and Clinical Immunology, Heinrich Heine University Medical Center, Dusseldorf, Germany. “This study, which includes the largest number of MDS patients of any iron chelation study, shows deferasirox can effectively reduce iron burden and is generally well tolerated when used appropriately to treat these patients.”

Iron toxicity can lead to permanent damage of the liver, heart and endocrine glands, leading to an increased risk of serious health problems and early death. Previous studies of transfusion-dependent MDS patients have found that increased levels of SF are associated with shortened overall survival.

About the EPIC trial
The EPIC trial was a one-year, open-label, prospective, multicentre trial. EPIC studied the efficacy and safety of a fixed starting dose of Exjade(*) based on transfusional iron intake, with subsequent dose titration at 3-monthly intervals based on serum ferritin (SF) trends. With 1,744 patients, this trial is the largest ever conducted for an iron chelator and included the largest cohorts of underlying anemias in a single trial, including patients with beta-thalassemia, MDS, aplastic anemia and other rare anemias. Twelve abstracts from EPIC are being presented at ASH.

Study details
The EPIC cardiac substudy evaluated the cardiac efficacy of Exjade(*) in 114 beta-thalassemia patients with myocardial siderosis (T2(*) less than 20 ms). Baseline myocardial T2 was less than 10 milliseconds (ms) in 47 (41%) patients (considered severe cardiac iron overload) and 10-20 ms in 67 (59%) patients
(considered mild to moderate). Mean baseline liver iron concentration (LIC) was 28.2 +/-10.0 mg Fe/g dry weight (dw), median SF was 5235 ng/mL, and the mean amount of transfused blood in the year prior to study entry was 185 mL/kg.

Patients experienced a statistically significant increase in myocardial T2(*) indicating a decrease in myocardial iron content. Based on a geometric mean +/- coefficient of variation, change from baseline (11.2 ms +/-40.5%) to 12.9 ms +/-49.5% represents an increase by a factor of 1.16 from baseline (P
less than 0.0001). Overall, 69.5% of patients taking Exjade(*) had an improvement in T2(*) (greater than 4% increase); there was no change in 14.3%; and worsening (greater than 4% decrease) in 16.2% of patients. Left ventricular ejection fraction remained stable throughout the study.

Additionally, LIC and SF levels (both indicators of total body iron) were significantly reduced from baseline by -6.6 +/-9.9 mg Fe/g dw and -1257 ng/mL, respectively (P less than 0.0001). Four patients discontinued treatment due to adverse events. Most investigator-assessed drug-related adverse events were mild to moderate in severity; rash was the most common (13.2%). There is an ongoing one-year extension of this substudy.

The pre-planned subgroup analysis of the EPIC study included 341 patients with transfusion-dependent MDS and SF levels greater than or equal to 1000 ng/mL, or SF less than 1000 ng/mL, but with a history of multiple transfusions (greater than 20 transfusions or 100 mL/kg of red blood cells) and an R2
MRI-confirmed LIC greater than 2 mg Fe/g dw. Overall, mean actual dose of Exjade(*) over one year of treatment was 19.2 +/-5.4 mg/kg/day. Based on the last observation carried forward statistical method, at one year, there was a significant reduction in median SF from baseline (-253.0 ng/mL; P=0.0019,
n=341). Of the 171 MDS patients whose SF was measured at one year, the decrease from baseline was 606 ng/mL. Overall, 48.7% of pts (n=166) discontinued therapy. Most common investigator-assessed drug-related adverse events were mild to moderate in severity and included diarrhea (n=110, 32%),
nausea (n=45, 13%), vomiting (n =26, 8%), abdominal pain (n=26, 8%), upper abdominal pain (n=25, 7%), rash (n=23, 7%) and constipation (n=21, 6%).

About Exjade(*)
Exjade(*) has been granted a Notice of Compliance with Conditions by Health Canada for the management of chronic iron overload in patients with transfusion-dependent anemias aged six years or older. Exjade(*) is also indicated in patients aged two to five who cannot be adequately treated with deferoxamine.

The results of key clinical trials have shown that Exjade(*), at doses of 20 to 30 mg/kg reduces liver iron concentration (LIC) and serum ferritin. With its simple administration, Exjade(*) has the potential to significantly improve patient compliance and quality of life.

Exjade(*) important safety information
The most frequent reactions reported during chronic treatment with Exjade(*) in adult and pediatric patients include gastrointestinal disturbances in about 26% of patients (mainly nausea, vomiting, diarrhea, or abdominal pain), and skin rash in about 7% of patients. Mild, non-progressive, dose-dependent increases in serum creatinine occurred in 34% of patients.

Elevations of liver transaminases as suspected drug-related adverse events were reported in about 2% of patients. The increases in liver transaminases were not dose-dependent. Forty percent of these patients had elevated levels (above the upper limit of normal) prior to receiving Exjade(*).

Elevations of transaminases greater than 10 times the upper limit of the normal range, suggestive of hepatitis, were uncommon (0.3%). High frequency hearing loss and lenticular opacities (early cataracts) have been observed in less than 1% of patients treated with Exjade(*).

Cases of acute renal failure (some with fatal outcome) have been reported following the post-marketing use of Exjade(*).

The use of Exjade(*) (deferasirox) is contraindicated in patients with hypersensitivity to the active substance, deferasirox, or to any of the excipients. Exjade(*) is contraindicated in patients with estimated creatinine clearance less than 60 mL/min. It is recommended that Exjade(*) should not be
used during pregnancy.

Disclaimer
The foregoing release contains forward-looking statements that can be identified by terminology such as “potentially,” “can,” “risk,” “will,” “may,” or similar expressions, or by express or implied discussions regarding potential new indications or labeling for Exjade(*) or regarding potential
future revenues from Exjade(*). You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of management regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results with Exjade(*) to
be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that Exjade(*) will be approved for any additional indications or labeling in any market. Nor can there be any guarantee that Exjade(*) will achieve any
particular levels of revenue in the future. In particular, management’s expectations regarding Exjade(*) could be affected by, among other things, unexpected clinical trial results, including unexpected new clinical data and unexpected additional analysis of existing clinical data; unexpected regulatory actions or delays or government regulation generally; the company’s ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry and general public pricing pressures; the impact that the foregoing factors could have on the values attributed to the Novartis Group’s assets and liabilities as recorded in the Group’s consolidated balance sheet, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking
statements contained in this press release as a result of new information, future events or otherwise.

About Novartis Canada
Novartis Pharmaceuticals Canada Inc., a leader in the healthcare field,is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2007, the Company invested over $86 million in research and development. Novartis Pharmaceuticals Canada Inc. employs approximately 800 people in Canada and its headquarters are located in Dorval, Quebec. It was named one of the “50 Best Employers in Canada” in 2008. For further information, please consult
http://www.novartis.ca.

About Novartis
Novartis AG provides healthcare solutions that address the evolving needs of patients and societies. Focused solely on healthcare, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic pharmaceuticals, preventive vaccines, diagnostic tools and consumer health products. Novartis is the only company with leading positions in these areas. In 2007, the Group’s continuing operations (excluding divestments in 2007) achieved net sales of USD 38.1 billion and net income of USD 6.5 billion. Approximately USD 6.4 billion was invested in R&D activities
throughout the Group. Headquartered in Basel, Switzerland, Novartis Group companies employ approximately 97,000 full-time associates and operate in over 140 countries around the world. For more information, please visit http://www.novartis.com.

(*) Exjade is a registered trademark
For further information: Novartis Media Relations, Sabrina Tremblay, Novartis Pharmaceuticals Canada Inc., Communications, (514) 633-7880 ext.2254, (514) 880-9766 (mobile), sabrina.tremblay@novartis.com, e-mail: media.relations@novartis.com