FDA Conducting a Safety Review of Deferasirox Due to Reported Adverse Events

Courtesy by: docguide.com

ROCKVILLE, Md — September 25, 2009 — The US Food and Drug Administration (FDA) is reviewing adverse event information for Exjade from a database that tracks all patients who are prescribed deferasirox (Exjade). This information suggests there may be a greater risk for adverse events such as kidney failure, gastrointestinal haemorrhage, and deaths in patients with myelodysplastic syndrome (MDS) compared with patients without these conditions.

Many of these patients are aged over 60 years and the adverse events are problems that are not uncommon in people with MDS. The number of deaths and serious adverse events seem to be fewer in younger patients with other chronic anaemias such as beta Thalassemia and Sickle Cell disease.

In reviewing the reports of adverse events and deaths, the FDA has found several factors that make it difficult for the Agency to draw conclusions without further analysis. These factors include the patients’ advanced age, the seriousness of their disease, other medical disorders they may have, and their need for blood transfusions.

Deferasirox has known adverse drug events, some of which have been fatal. These events include kidney and liver failure — particularly in patients who have other conditions that would make them more susceptible to kidney or liver problems — and gastrointestinal ulcers and/or haemorrhage.

The FDA has not determined whether or not patients with MDS or older patients treated with deferasirox are at greater risk for adverse events or death compared with patients of a similar age or diagnosis who were not treated with deferasirox, or compared with patients who are younger who have other chronic anaemias and have been treated with deferasirox.

The FDA is working with Novartis, the company that manufactures deferasirox, regarding potential revisions to the prescribing information to warn healthcare professionals about the possible risks of using deferasirox in certain patients and to ensure that the benefits of deferasirox outweigh the potential risks, particularly in older patients and patients with MDS.

The Agency will communicate its final conclusions when the analysis of these and other data are complete.

The FDA urges both healthcare professionals and patients to report side effects from the use of Exjade to the FDA’s MedWatch Adverse Event Reporting program.

Genomic Research Will Help Provide Alternative Treatments to Patients With Iron Overload

Courtesy by: marketwire.com

While most people are familiar with anemia – or lack of iron in the blood – they are less familiar with diseases of too much iron.

Excess iron in the body, if left unchecked, can form toxic deposits in major organs leading to serious conditions including heart failure, diabetes, liver cirrhosis, arthritis, and even infertility. Iron overload can be a consequence of a genetically mutated gene known as hereditary hemochromatosis, or a consequence of red blood cell transfusions, required as life saving treatments, for patients with diseases such as thalassemia.

Patients with hemochromatosis and thalassemia both absorb too much iron from their diet which either causes or exacerbates their iron overloading to levels that are toxic.

The mutated gene for hemochromatosis is carried by 1 in 9 Canadians. Currently over 100,000 Canadians and several million people in the US have hemochromatosis, with many of these patients still undiagnosed.

“Part of the problem is that this condition of hemochromatosis will present itself in disguised ways and is often unrecognized by physicians. A patient may have arthritis, or extreme fatigue or even diabetes, but often it’s not linked to the real genetic cause, which is iron overload,” says Dr. Paul Goldberg of Xenon Pharmaceuticals.

Goldberg is the lead investigator on the $7.5 million project entitled Enabling Studies for a DMT1 inhibitor – A Novel Therapeutic Approach for Treatment of Iron Overload Disorders.

The research, funded by Xenon Pharmaceuticals and Genome BC will tackle the disease, by creating a small-molecule drug to block excess iron absorption at its source: directly in the gut.

Currently, hemochromatosis patients are treated with lifelong phlebotomies – invasive and sometimes painful treatments that require regular trips to the hospital or clinic to remove about half a litre of blood, allowing the patient to produce new blood with less iron.

While phlebotomies work well in many of the patients, they can be problematic, causing recipients to feel unwell for a while following the procedure. “Patients are rendered anemic temporarily so the procedure is not suitable for people with heart conditions or needle intolerance,” says Goldberg. He also points out that patient compliance can be a problem with this form of treatment.

As such, Goldberg’s research is focused on creating a safe and effective oral therapy as an alternative or adjunctive treatment for those affected by iron overload.

“Patients with hemochromatosis are hyper-absorbing iron he says. The drug that we are developing would block the key transporter for this excess iron uptake, known as DMT1.”

Thalassemia patients may also directly benefit from the DMT1 blockers. These patients become severely iron overloaded at a young age due to the requirement for regular life saving blood transfusions and the additive complication of excessive iron absorption from their diet.

Patients who chronically receive blood transfusions are currently treated with iron chelators to control their iron levels. However chelators have severe dose limiting side effects and it remains challenging for clinicians to maintain normal iron balance in these patients. In addition, chelation therapy is extremely expensive, sometimes costing up to $50,000 per patient and may require for some patients frequent and prolonged intravenous administration.

“The Xenon drug provides an oral alternative and is being optimized for patient safety,” says Dr. Simon Pimstone, President and CEO of Xenon Pharmaceuticals. “Our DMT1 blockers could lower the dosage of iron chelator drugs making them safer or could make them more effective, either way, improving patient outcomes. Our goal is to test this drug in iron overload patients within two to three years.”

“Genome BC is pleased to co-support this innovative research, which may develop an innovative, safe and valuable treatment alternative to patients suffering from iron overload disorders,” says Dr. Alan Winter, President and CEO of Genome BC.

Bloodlines: What you don’t know about sickle cell disease can cause a family of hurt

Courtesy by: stlamerican.com

Informing others about sickle cell anemia is a commitment sealed in blood at Sickle Cell Disease Community Advocates (SCDCA) in St. Louis. It is run and operated by women on a mission to assist families with sickle cell disease and to support those who are working on a cure.

What is sickle cell disease?

Sickle cell anemia is an inherited blood disease that affects one in 500 African Americans in the U.S. and millions of people worldwide. The sickle cell gene causes the body to make abnormal hemoglobin, the iron-rich protein that gives blood its red color and carries oxygen from the lungs to other parts of the body.

What causes sickle cell anemia?

If a person inherits one sickle cell gene from a parent, he or she will have a sickle cell trait. Persons with the trait usually live normally, but can pass the trait to their children. If someone inherits two sickle cell genes, they will have sickle cell anemia. Genetic counseling can help families and individuals understand the risk of having a child with the disease.

Who is at risk for sickle cell disease?

Although it is more common in darker-skinned ethnic groups, sickle cell disease can occur in people of all races.

What happens during a sickle cell crisis?

With sickle cell disease, there is not enough oxygen in red blood cells. Instead of being round, healthy red blood cells, they become rigid and deformed into the crescent sickle shape, which do not move easily throughout the body. The sickle cells can clump together and cause blockages in the blood vessels, depriving tissues of needed oxygen. This episode, known as a sickle cell crisis, can cause mild to excruciating pain, swelling and other difficulties for a period of minutes, hours or days, making it difficult to breathe, function or move about.

What are complications from sickle cell disease?

Leg ulcers, infections, acute chest syndrome, gallstones, high blood pressure in the lungs, an enlarged spleen, stroke, and organ failure are other complications that can occur with sickle cell. Many sickle cell patients require repeated hospitalizations, strong pain medication and blood transfusion therapy.

Sickle cell anemia has different names, including Hemoglobin SS disease, Hemoglobin S disease, Hemoglobin SC, HbS disease, sickle beta thalassemia, but they all are some form of sickle cell anemia.

“I have two children with sickle cell SS, which is considered to be the severe form of sickle cell anemia, but I have two different experiences between Hakeem [age11] and Haniyah [age 6],” said their mother, Tamneca Reid, who also has sickle cell disease.

“Hakeem suffers ? his cognitive skills are low, he’s deaf in one ear. He suffers emotionally. He aches pretty much every day, from mild to severe. And Haniyah, on the other hand ? she suffers none.”

At least that’s what she thought, until Haniyah received an MRI of the brain.

“Haniyah doesn’t suffer (with pain) from sickle cell disease as of now… and the testing came out that she did have a silent stroke… so you could have the same disease and suffer two different ways.”

Delores Rucker didn’t know she carries the sickle cell trait until her toddler son became ill a few years ago –right after the birth of her daughter.

They were both tested and confirmed with sickle cell anemia.

Pain treatment and pain management for sickle cell patients is crucial.

Dayvin is now 6 and Dyamond, her daughter, has experienced a lot of pain in her 4 years.

“My daughter was just in the hospital twice at Cardinal Glennon, and my son, he is receiving blood therapy,” Rucker said. The blood therapy has decreased the number of painful episodes for her son.

“As far as pain goes, for my daughter … I try Tylenol with codeine. At times, it doesn’t work, and then we’ll switch it to oxycodone. When that didn’t work, we had to send her into the hospital and they had her on morphine ? a constant-drip morphine. And after a while – after a few days, it tends to work.”

Rucker said when the intravenous morphine doesn’t work – her health care providers will give her a blood transfusion.

She avoids extreme weather but any day without a sickle cell crisis is considered a good day.

“My son was actually just in the hospital and when we were leaving out with him, she was fine … running through the hallway fine and she slept the whole way to the house and the moment she woke up, she was in pain, so I turned around and took her right back. It’s just hard.”

Every day is a challenge with sickle cell anemia.

Social worker Tanjila Bolden is in pain every time she walks because of what sickle cell anemia has done to her hips. Her doctor told her she needed a hip replacement, but the graduate school student is trying to avoid that surgery as long as possible. Bolden is the mother one child. Her 10-year-old son has the sickle cell trait rather than the full-blown disease, and thankfully, she was able to bare her son before sickle cell anemia took its toll on her body.

“Every time I had a menstrual cycle, I had severe cramps and the cramps would start in my lower abdomen and lead into my back and down into my legs and I would go into a full blown crisis every time I had a cycle,” Bolden said.

Fluids, pain medicine – and sometimes oxygen were used to treat these monthly crises, but Bolden said they did not provide adequate relief.

“I got to a point where they put me on Depo [Provera] shot–the birth control to stop the periods … it stopped my cycles, which stopped the amount of crises that I had. Well, the Depo caused osteoporosis in the lower part of my spine, so I had to stop taking that. They were looking at burning the lining of my uterus to stop the cycles. That didn’t work… so they did a partial hysterectomy.”

A sickle cell anemia crisis can strike any part of the body, even your sexual organs. For 10 to 40 percent of males with sickle cell, priapism can occur, a condition causing painful, elongated erections.

Living through these terrible ordeals of both her son and her sister are the reasons Caroline Douglas founded SCDCA.

“There is nothing like an amputated spirit – many of these families suffer with that,” Douglas said.

The SCDCA Camp Moon Light debuted in August for children with sickle cell anemia and their siblings to learn and understand what they are going through.

“Sickle cell disease is not just one person’s condition – it affects the entire family. What we want to do is not just educate about sickle cell disease, but about sickle cell trait,” Douglas said. As a community with sickle cell, it is important for us to network with one another. What we try to do is make sure they are aware of the nature of their condition and then we educate them about what their condition is and how to live a better quality of life.”

Reid’s son attended Camp Moon Light and Camp Crescent, which is for children with sickle cell disease.

“Both camps were really good for him and his spirit. On one hand, Camp Crescent –it supplied friends and peers that suffer with the same condition. One the other hand, Camp Moon Light brought him out of his element,” Reid described. “My son – he’s nervously shy … but at the Camp Moonlight, he showed personality, he was dancing and doing things he normally don’t do around other people and built his confidence up to the ceiling.”

Last week, SCDCA held its Moon Light Walk to help fund Camp Moonlight and other programs and activities. The organization is looking for assistance to expand the camp into from a two-day to a five to six-week enrichment session.

Carriers of anemia gene are denied civil servant jobs

Courtesy by: chinadaily

GUANGZHOU: Some applicants here have been denied civil service jobs by local government authorities because they failed “unnecessary” physical checkups, sources said yesterday.

“As far as I know, more than 10 people like me have been refused civil servant jobs although we all passed written tests and interviews,” said an applicant surnamed Xie.

Xie was asked by the personnel department in Foshan of Guangdong province to undergo a physical examination after he passed tests and interviews in April.

Xie did not pass the physical tests because he is a carrier of the thalassemia gene.

“But the country’s regulation for civil servant jobs does not specify that thalassemia gene carriers cannot pass the physical test,” Xie said.

Under the general physical examination standard of civil servant enrollment, an applicant will be denied the job if he or she develops anemia, a condition caused by various mineral and vitamin deficiencies. Symptoms can include fatigue, shortness of breath, dizziness and difficulty concentrating.

According to the standard, a man will be diagnosed with thalassemia if his hemoglobin (HGB) is lower than120 g/L.

“How can I be seen as a thalassemia patient? Yes, I am a thalassemia carrier but my HGB is 136 g/L, which is shown on the physical report,” Xie asked.

In Guangdong province, one in nine people, or about 12 percent of its population, have developed thalassemia, a form of chronic anemia, according to the Guangzhou-based Southern Hospital.

“They are no different from others in terms of working and living. It is absolutely wrong to deny their job offers because of their light thalassemia,” said Li Chunfu, a doctor with the hospital. Thalassemia gene carriers or those with light symptoms usually do not develop physical signs, Li said. Denying thalassemia gene carriers jobs as public servants is employment discrimination, he said.

Guangdong and the neighboring autonomous region of Guangxi have reported a rising number of thalassemia cases in recent years, Li said.

An official with the Foshan personnel bureau, who declined to be named, told China Daily yesterday that traditionally, thalassemia gene carriers have been denied jobs as public servants. He declined to elaborate.

Cord blood stem cell transplantation cures minor girl

Courtesy by: samaylive

Chennai, Sept 16 An eight-year-old girl suffering from Thalassemia has been cured by ‘cord blood stem cell transplantation’ at a hospital here.

The doctors used her younger brother’s cord blood stem cells for transplantation and the hospital authorities claimed it was the first time this method was practised.

Thalassemia is a genetic blood disorder in which the body makes an abnormal form of hemoglobin that results in excessive destruction of these cells and causes severe anaemia that can occur months after the birth. The patient then has to undergo monthly blood transfusion.

Mayur Abhaya, president and executive director of LifeCell International, a leading stem cell bank, told reporters here that Thamirabhurani was suffering from Thalassemia for the last six and a half years.

She went through painful blood transfusion and medication until the stem cell Cord blood stem cell transplantation cures minor girl, he said.

“The stem cell transplantation was recently done by Dr Revathi Raj at Apollo Hospital and it helped the girl get rid of Thalassemia,” he said, adding doctors had recommended that the patient start on iron reducing medication initially.

“The doctors then asked her parents to consider another pregnancy and go for umbilical cord blood stem cell banking”, he added.

Raj said a pre-natal test confirmed that the foetus was not affected with Thalaseemia.

Stem cell bank to start in July next year

Courtesy by: thepeninsulaqatar

DOHA: Bringing renewed hope in the treatment of various medical conditions in the Eastern Mediterranean region, the Virgin Health Bank (VHB)-QSTP, will start its operations here in July 2010. The bank which collects, processes and stores cord blood, taken from the umbilical cord of newborns, was launched here in March as a partnership between Qatar Science and Technology Park (QSTP) and the UK-based VHB.

With the bank headquarters opening here, Qatar will be at the forefront of using regenerative medicine.

“We aim to transform lives through the use of cord blood stem cells in the region. This will be by providing information and championing new standards of high quality ethical banking services,” said Andrew Glen, Commercial Director, VHB-QSTP.

The stem cells, taken from the blood in the umbilical cord, can renew themselves through cell division and differentiating into specialised cell types. Today, 85 diseases can be treated using cord blood. Sickle cell anemia and thalassemia can be completely cured using cell therapy. The facility opening here will help spur cell therapy for various disorders including genetic ones.

“The bank will open as a private one, where families can pay to store the cord blood. This is the major step before venturing into public banking which can ensure that the blood stored can be utilised for community needs. Talks are in progress for public banking.”

When public banking starts, VHB-QSTP will play a major role in Qatar’s much coveted national public health programme to create the world’s first comprehensive source of stem cells for the Middle Eastern population and make it easier to source a matched tissue typed stem cell unit when a transplant is needed.

The VHB project has already commenced in Qatar as a pilot programme with over a dozen cord blood samples being cryogenically stored in the UK. “With the cooperation of Ministry of Interior and Qatar Airways, we have been able to study the various issues in transportation of the samples to UK. To get the best result out of the cells, every thing from collecting to temperature and transportation time matters,” said Dr Magnus Nicolson.

However, by 2010, the bank will have a storage and processing unit in Qatar.

The VHB presence here will catalyse the national health programme which calls for a public bank, as people will get to know the benefits.

The bank has been conducting training for health professionals in Qatar on the management, collection and preservation of the cord blood as part of the public health programme.

Need to tap potential of wonder stem cells

Courtesy by: timesofindia

VARANASI: Stem cells are the wonder cells and there is a growing need to spread awareness about the importance of these cells to realise their

potential, says Dr CV Nerikar. Nerikar is the former blood transfusion officer, Nagpur Medical College, and the chief executive officer of the country’s only stem cell bank. He was speaking at a continuing medical education (CME) programme in the city on Sunday evening.

The programme was organised by Varanasi Obstetrics and Gynaecologists Society (VOGSI). As he stressed on the need to promote stem cell banking in the country, its advantages were also emphasised. “These cells are preserved at very low temperature (-196 degree Celsius) and can be used for nearly 130 years (two generations),” he said on the occasion.

“The stem cells have inherent capacity to multiply and regenerate into all specialised cells of the body and could be used in multiple life threatening diseases, including genetic disorders,” informed Dr Neriker. Stem cell transplant has successfully treated complicated and life threatening diseases including blood cancer (acute leukemia), thalassemia, blood disorders and other immune deficiencies. “Currently, stem cell research is also going on a number of diseases including spinal cord injury, cardiac diseases, diabetes and stroke,” he added.

He also said the umbilical cord blood is the easiest source of retrieving stem cells and the blood that is usually thrown as wastage should be preserved for complex diseases, especially for treating genetic disorders in the family.

President, VOGSI, Dr Amod Prakash and other senior obstetricians and gynaecologists were also present on the occasion.

5000 children born with thalassemia annually in Pak

Courtesy by: geo.tv

PESHAWAR: Over 5000 children are born with transfusion dependent thalassemia annually in Pakistan and 65 percent of them belonge to NWFP.

According to the three yearly report of Hamza Welfare Foundation in Peshawar the NGO has provided 14,543 patients with healthy blood.

Presently 550 patients are registered with the organisation. Out of which 455 patients are suffering from thalassemia; 58 from haemophilia and 37 from blood cancer. The majority of the patients include minors.

The Foundation is making efforts for facilitating free medical services to the poor and deserving; and a large number of people are supporting the organisation in this regard.

NFCTR Receives $1.6 Million Grant for Cell Transplant Research

Courtesy by: marketwatch

LOUISVILLE, Ky., Sep 9, 2009 (GlobeNewswire via COMTEX) — The National Foundation to Support Cell Transplant Research (NFCTR) today announced that it has received $1.6 million in federal support for research and clinical trials it is funding at the University of Louisville. Congressman John Yarmuth (D-3rd District) secured the funds to support cell transplant technologies developed by Dr. Suzanne Ildstad and the Institute for Cellular Therapeutics at U of L as part of the 2009 U.S. Department of Defense Appropriations Bill.

Dr. Ildstad’s research, now in clinical trials at the University of Louisville, Duke University and Northwestern University, may make it possible to perform complex tissue transplants without the need for anti-rejection medication in the treatment of catastrophic burn and blast injuries. The research is also being applied in new treatments for autoimmune diseases such as multiple sclerosis and inherited disorders such as sickle cell disease and thalassemia.

“The NFCTR is one of the foremost research centers dedicated to advancing life-saving medical research technologies… technologies that are being developed right here in Louisville,” Yarmuth noted.

Translating scientific discoveries into clinical treatments is expensive, and there is often a gap in funding for early stage clinical trials. “We work to fund that gap,” states Dr. Paula Grisanti, Chair of the NFCTR. “We also support patients participating in early stage clinical trials who might otherwise need to fund their participation out of pocket.”

“We are a national non-profit foundation that raises funds to support adult stem cell research… and this appropriation is a great example of how public-nonprofit partnerships can work together to fund important medical research,” Dr. Grisanti adds. “We are very grateful to Congressman Yarmuth for his instrumental assistance in securing these federal funds in support of Dr. Ildstad’s research.”

About the National Foundation to Support Cell Transplant Research (NFCTR)

Formed in 2005, the National Foundation to Support Cell Transplant Research (NFCTR) was established to support peer-reviewed research and early stage clinical trials in the field of adult stem cell transplantation. Adult stem cell research has the potential to revolutionize the treatment of a large number of diseases and conditions affecting people worldwide. The NFCTR funds research in a number of strong academic settings and supports both collaboration and information sharing wherever possible. Through the funding of these collaborations, the NFCTR hopes to create stronger opportunities for advancements in the field. For more information, visit http://www.nfctr.org or call (502) 379-8544.

Dr Syed Abdul Mujeeb (1957 – 2009)

Courtesy by: dawn.com

KARACHI, Sept 7: A senior microbiologist, in charge of the blood bank at the Jinnah Postgraduate Medical Centre, Dr Syed Abdul Mujeeb, who died of a heart attack on Saturday, was buried here on Monday. He was 52.

He leaves four brothers and three sisters. A number of doctors belonging to the JPMC and other healthcare facilities and educational institutions attended his funeral prayers.

He had received the President’s Pride of Performance Award for his outstanding services in medicine last year. He had written numerous research articles, published in international and national journals.

To his admirers, friends and colleagues, Dr Mujeeb was a thorough professional, committed to the wellbeing of patients suffering from blood diseases.

An expert in infectious diseases, Dr Rafiq Khanani expressed sorrow over Dr Mujeeb’s death and said he would always be remembered for his research and services in the field of transfusion medicine. He always stressed the importance of safe blood and blood products and their transfusion.

In June this year, Dr Mujeeb had participated in the Global Consultation on 100 per cent Voluntary Non-remunerated Donation of Blood and Blood Components, organised by the World Health Organisation in Melbourne, Australia.

In a presentation there, he had said that Pakistan with a population of 160 million had in general disorganised, fragmented and hospital-based blood transfusion services, with no national blood transfusion services and programme for voluntary blood donors. If two per cent eligible population donated blood twice a year, all current requirements of blood could be met in the country, he had said.