OSF dispatches field hospital to Sehwan

September 24, 2010

Courtesy: dailytimes.com.pk

Local nongovernmental organisation Omair Sana Foundation (OSF) has dispatched a field hospital for the flood-hit Sehwan city to provide basic medical facilities to the flood victims. The OSF patrons at a press conference at Karachi Press Club apprised that the hospital consisted of a minor operation theatre, an outpatient department (OPD) clinic, pharmacy and basic medical facilities, while it could facilitate about 400,000 people. They said the OSF was working for the prevention and eradication of thalassemia but due to floods, a field hospital was being sent to help the flood victims. The OSF mobile OPD clinics were serving 2,000 patients every day across the country while the OSF was planning to build a model village of 100 houses in Thatta, they added. staff report


Engineered version of HIV is used to cure genetic blood disorder

September 24, 2010

Courtesy: bellinghamherald.com

For the second time, researchers have used the HIV virus in gene therapy to cure a severe genetic disease, this time the blood disorder beta-thalassemia, which causes life-threatening anemia.

French researchers had previously used a “defanged” version of the virus that causes AIDS to cure two boys with the rare disorder adrenoleukodystrophy, which was at the heart of the popular movie “Lorenzo’s Oil.” Beta-thalassemia is a much more common disease, and although the new research involved only one patient, it suggests that this approach could have wide applicability.

“This work represents a major step forward for the gene therapy of hemoglobin disorders,” wrote Dr. Derek A. Persons of St. Jude Children’s Research Hospital in Memphis, Tenn., in an editorial accompanying the report in the journal Nature.

The feat marks at least the sixth disease for which gene therapy has recently been shown to be beneficial.

Beta-thalassemia is caused by defects in the production of the beta-globin chain, a key component of hemoglobin, the molecule that carries oxygen in red blood cells. It affects primarily people of Mediterranean, Middle Eastern, South Asian, Southeast Asian and Chinese descent, with about 60,000 children being diagnosed with it worldwide each year.

Treatment generally involves frequent blood transfusions – typically monthly – but that leads to a buildup of iron that can damage organs. As a result, patients must also undergo chelation therapy to remove as much of the iron as possible. The disease can be cured by bone marrow transplants, but only if a suitable donor is found.

The researchers in the new study used an HIV virus that had been engineered to carry the correct version of the hemoglobin gene. Most previous research on gene therapy has used other retroviruses, such as the mouse leukemia virus, to carry the desired gene into cells. But those so-called vectors had a tendency to insert genes in the wrong locations, leading in some cases to cancer. Attempts to try gene therapy in patients were put on hold until scientists could overcome the problem.

Dr. Philippe Leboulch of the University of Paris and his colleagues used the HIV-based vector – produced by Bluebird Bio of Cambridge, Mass., and Paris – to treat an 18-year-old French boy who had been diagnosed with beta-thalassemia at age 3. Before the treatment, he was receiving three packs of red blood cells monthly and chelation therapy to remove iron, but was still ill. No suitable bone marrow donor was available.

The team removed blood-forming stem cells from the patient’s bone marrow and treated them in a test tube to add the working gene. The boy was given chemotherapy to destroy as many of the remaining stem cells as possible, then the treated cells were injected.

Within a year, the boy was able to stop receiving transfusions, and he has remained stable for 22 months since then.

“He is happy to have a normal life back, and for the first time has a full-time job in a main restaurant in Paris,” Dr. Francoise Bernaudin, the clinical hematologist who has been monitoring his condition, said in a statement. The team has also been able to remove excess iron that has built up over the years by bleeding him regularly, she said.

The researchers are planning to enroll another 10 patients for more studies.


NATURE Publishes Promising Results on Treatment of First Patient in bluebird bio’s Phase 1/2 Beta-Thalassemia Study

September 24, 2010
bluebird bio (formerly Genetix Pharmaceuticals Inc.) an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced publication in the journal Nature of its promising Phase 1/2 data highlighting positive results of LentiGlobin™ gene therapy treatment in a young adult with severe beta-thalassemia, a blood disorder that is one of the most frequent inherited diseases.
The patient, who had been transfusion dependent since early childhood, has become transfusion independent for the past 21 months – more than two years after treatment with the LentiGlobin vector. The study also identified a subset of cells with the corrected beta-globin gene that overexpressed a truncated form of a gene called HMGA2. The patient has not experienced any adverse events. The data show that while early on, the HMGA2 clone was a significant portion of the corrected cells, the clone levels had declined at the time the paper was prepared, and further follow up indicates the decline is continuing.
“Although based on the first treated patient, we believe these results are impressive and illustrate for the first time the significant potential for treatment of beta hemoglobinopathies using lentiviral beta-globin gene transfer in the context of autologous stem cell transplant,” said Philippe Leboulch, M.D., senior author of the study and head of the Institute of Emerging Diseases and Innovative Therapies of CEA and INSERM; professor of medicine, University of Paris; and visiting professor, Harvard Medical School. “For beta-thalassemia, we have worked intensely for almost 20 years to design, develop and manufacture LentiGlobin to provide a sustained high level hemoglobin production, resulting in a major clinical benefit. It has been very rewarding to follow this patient as his life has dramatically improved since receiving our treatment.”
“For the first time, a patient with severe beta-thalassemia is living without the need for transfusions over a sustained period of time,” said Marina Cavazzana-Calvo, M.D., first co-author of the study and professor of hematology, University of Paris and chief of Cell and Gene Therapy Department, Necker-Enfants Malades Hospital in Paris. Salima Hacein-Bey-Abina, Ph.D., professor of immunology, University of Paris, added, “These results are not only important due to the tremendous medical need that exists for thalassemia patients around the world, but also represents a significant step forward for the field of autologous stem cell therapy as an emerging therapeutic modality.”
Dr. Françoise Bernaudin, the clinical hematologist who has followed this patient since early childhood, said, “It is wonderful to see that this young man is for now free of transfusions and injections for iron chelation. He is happy to have a normal life back, and for the first time has a full-time job as a cook in a main restaurant in Paris. We are now even able to bleed him regularly to help remove toxic iron that had accumulated over the years because of blood transfusions.”
The paper, titled “Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassemia,” is available in the online publication of Nature at http://www.nature.com.
“We believe the human findings in beta-thalassemia, as well as the recently published data in Science on two patients with childhood cerebral adrenoleukodystrophy (CCALD), highlight the significant opportunity for bluebird bio’s gene therapy platform to help patients with severe genetic disorders,” said Nick Leschly, president and CEO of bluebird bio. “We are committed to building a world-class company in gene therapy led by outstanding people as we move aggressively forward with multiple clinical studies, including our ongoing clinical trials for the development of LentiGlobin for beta-thalassemia and our product for CCALD.”

Rouse Organises Blood Donation Drive At Twin Towers

September 13, 2010

Courtesy: albawaba.com

Dubai Investment Properties, one of the leading real estate developers in the UAE, today announced that Rouse, a leading intellectual property (IP) consultancy, is holding a blood donation drive on September 14, 2010 from 9 am to 3 pm at the Twin Towers in Deira. The third annual blood donation drive, which is a part of the company’s global CSR programme ‘Rouse Cares’, will be held in association with the Dubai Health Authority to support patients suffering from thalassemia and other life-threatening ailments.

“Patients suffering from thalassaemia, cancer, accidents and other life threatening conditions need blood to save their lives. This ever-increasing demand of blood can only be met through voluntary blood donations,” says Mr. Jon Parker, Partner, Rouse. “Through our Rouse Cares programme, we aim to help improve the lives of others through sustainable initiatives and by growing roots in each country where we work. We are happy to organise our annual blood donation drive in association with the Dubai Health Authority where our staff, partners and clients can make a contribution to meet the continued need for blood at hospitals.”

The third annual blood donation drive will be held on the third floor of the Twin Towers. Employees, partners and guests are expected to voluntarily come forward to donate in large numbers. Rouse expects the donations to surpass last year’s number of 100 units. The DHA authorities will provide full medical equipment, information and support at the blood donation campaign.

“We commend Rouse for organising the blood donation campaign during the Eid period and are happy to whole-heartedly support it. Twin Towers will offer them all the facilities and services required to make this blood donation a mega success. We are sure DIP’s staff and other tenants at Twin Towers will also come out in full force to support Rouse’s blood donation campaign,” says Mr. Deepak Chawla, Operations Manager at DIP.

Globally, Rouse supports initiatives to help improve the lives of the local community through its ‘Rouse Cares’ programmer. The support ranges from providing legal support on a pro bono basis, supporting charitable institutions and working directly with community organisations.


Fun and feast for special children

September 7, 2010

Courtesy: dawn.com

Around 500 special children of different educational and rehabilitation centres being run by non-governmental and charitable organisations enjoyed exciting games, competitions and other recreational activities at a carnival held on Monday.

Inside a huge tent pitched in a lawn along Sharea Faisal especially for the young visitors, no one was found too shy or too timid to interact with one another.

Potato race, bubble catch, pottery, freeze dance, dart shooting, jumping castle, illusion show and pin the donkey made the colourful event even more thrilling for the young participants.

It was one of the rare occasions where street children also had an opportunity to attend the “Ramazan kids carnival”.
“The purpose was to give special children, orphans, street children and thalassaemia patients a chance to have fun and also demonstrate their talent,” said Farhan Ahmad, an official of the HSBC that had organised the event.

Children accompanied by their teachers and representatives of their academic and rehabilitation centres were encouraged to participate in games and competitions. While the enthusiasts won prizes which also included toys, all children attending the event were presented with Eid gifts. It was their involvement in the exciting activities that made the carnival a remarkable event.

A couple of celebrities also joined the special children at the evening.

Zaid Rao, a young thalassaemia patient, told Dawn that he found the evening interesting. “We all have good time here,” he said, adding that the special children also had an opportunity to expose their hidden talents and feel, amidst clapping and appreciation, a change they too deserved.

Farhana Khalid, a teacher accompanying the deaf and dumb students of the Family Educational Services Foundation, appreciated the fact that the special children had been offered a recreational facility despite all odds in society.

Rana Asif Habib of the Initiator Human Development Foundation said that the problem of street children was a serious challenge for the civil society. He highlighted the need for civil society and the government to join hands to ensure provision of rights to children.

The special children showed their keen interest in pottery, face painting and bubble catch booths where organisers were present to guide and support them.

Later, Iftar was also served.

The children had come from different educational and rehabilitation centres, including the Family Educational Services Foundation, Karachi Vocational Training Centre, SOS Children’s Villages of Karachi, Concern for Children, Children Health & Education Foundation, HOPE Home Schools, Kashif Iqbal Thalassemia Care Centre and the Initiator Human Development Foundation.


‘Make thalassaemia test mandatory’

September 7, 2010

Courtesy: expressbuzz.com

All the Primary Health Centres in the state should conduct a separate test for pregnant women to find out whether either one the parents is a Thalassaemia carrier, a genetic condition when a person is unable to produce the required amount of haemoglobin, said Dr Revathy Raj, Consultant Paediatric Hematologist, Voluntary Health Services hospital on Saturday.

She was speaking at a meeting of parents whose children were thalassaemic. Health Minister MRK Pannerselvam was also present. Dr Revathy pointed out that the Thalassaemia Welfare Association, formed at the VHS blood bank, had 159 children registered, of which 80 underwent continuous regular blood transfusion.

A blood test called Hb A2 estimation is essential to be done among pregnant women carriers for early detection of a child having thalassaemia, she said. There was a cure available for the condition, known as stem cell transplantation, which cured the child forever. The procedure costed Rs 8 lakhs, of which Rs 1.5 lakhs was covered under the Kalaignar Insurance Scheme. If both the parents were carriers, then there were 25% chances that the child would have thalassemia, and early tests done could prevent the birth of such newborn babies, Dr Revathy said. She said that if only one of the parents was a carrier, then the baby would be normal and the treatment could be started at the earliest.



Panchkula lad clears thalassemia hurdle to get into IAS

September 7, 2010

Courtesy: timesofindia

He has lost count of blood transfusions done on him during the last 22 years, but he never lost track to achieve what he wanted in life. Sukhsohit Singh, a Panchkula resident, has cracked the much coveted civil services examination with an all India rank of 42, in UPSC’s Civil Services (Main) Examination-2008 second merit list, which was declared by the commission on Friday afternoon.

Sukhsohit claims that he is the first patient of Thallassemia major (a rare genetic blood disorder, requiring frequent and regular blood transfusions after every 20-25 days) to have cleared this exam in India. He is currently pursuing his PhD from Panjab University. For the civil services examination, he had opted for public administration and sociology as optional subjects.

Having achieved success in his third attempt, Sukhsohit believes regular study of 15-18 hours helped him achieve the target. Sukhsohit never allowed the disorder to become a hindrance in his success by keeping himself preoccupied with his mission in life. “Disease is inevitable, but weather it causes ‘disease’ or not is entirely optional, just as pain is inevitable but suffering is altogether optional,” exclaimed a jubilant Sukhsohit.

Cracking the civil service was not so easy as before his exams he used to spend the entire day for blood transfusions at PGI, just threedays before his sociology mains exams.

Sukhsohit said he received a lot from PGI doctors and others at the Thalassemia Children Welfare Association and has now himself started contributing to the cause through his NGO Smiles. He now plans thallassemia welfare and management units in remote and far flung parts of India to make the treatment accessible to large number of patients.


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