Salman pledges bone marrow to help patients

November 23, 2010

Courtesy: Thaindian.com

Bollywood star Salman Khan’s philanthropic side is well known and now the actor has gone ahead and pledged his bone marrow to help people suffering from life-threatening diseases.

“Donating marrow is a simple act, it’s as simple as donating blood. But this simple act can save the life of someone suffering from blood cancer, thalassemia and other major blood-related diseases,” Salman said in a statement.

“I am pledging my marrow with MDRI (Marrow Donor Registry of India) so that in an emergency, if a patient’s sample matches mine, I can be reached to donate my marrow,” the 44-year-old actor said.

Salman’s charitable foundation Being Human has also joined hands with the Marrow Donor Registry (India) to create awareness of how marrow donors can save lives.

“The more the donors, the more lives we save. So I urge that all of you who are blessed with good health come forward and pledge your marrow so that the less fortunate can benefit from this thoughtful act,” Salman said.

The aim of the initiative is to develop a large pool of voluntary marrow donors for transplants that are life saving for patients suffering from blood cancer, thalassemia, aplastic anemia, congenital immunodeficiency states and other such blood related diseases.


First bone marrow transplant performed in Nashik

November 23, 2010

Courtesy: ibnlive.in.com

The city’s first bone marrow transplant has been performed successfully in the district by a leading haematologist on a woman.Bone marrow transplant is a complex procedure like Kidney and liver transplantation and is a boon for patients suffering from various diseases like Thalassemia, Blood Cancer, Myeloma and Lymphomo, Dr Pritesh Junagade, who is also haemato-oncologist, told reporters last night.”The 21-year-old woman from Sangamner, who underwent the procedure, had a plastic anaemia and she needed regular blood and platelet transfusion to keep her alive. Her brother was tested to see if his bone marrow matched. It did and the marrow was removed from the donor and placed in the patient,” he said adding four weeks later, the patients blood count was normal.Dr Junagade, who was in London for seven years and returned to Nashik, said the transplant was carried out successfully at the cost of Rs 5.5 lakh.


Italian docs bring Thalassaemia cure

November 23, 2010

Courtesy: expressbuzz.com

It’s a ray of hope for Thalassaemia patients, particularly so for those who cannot afford expensive treatment. The city will soon have a “Bone Marrow Transplantation” unit (BMT) set up by a pioneer team of doctors from Instituto Mediterraneo di ematologia (IME), Italy. The department is headed by Prof Guido Lucarelli, a renowned transplant physician for Thalassaemia.

‘Cure Thalassemia India’, will be a not-for-profit organisation providing support to health professionals working for the cause of children affected by Thalassaemia, said Eugenio La Mesa one of the organizers for BMT centre and a social business entrepreneur.

Currently BMT is the only curative option for this fatal inherent blood disorder. Usually a patient requires life-long blood transfusion every three to four weeks, in addition to medicines, failing which 90 percent of the children affected by the disease die in early childhood.

The team from Italy is in Hyderabad and has visited hospitals in the city to finalize the hospital for the BMT unit. Once the finalisation has been done, a specialist team from IME will train the local doctors and nurses in this treatment. The treatment includes transplantation for 100 per cent and 50 per cent HLA (human leukocyte antigen) matched donors, said Dr Pietro Rodani, transplants specialist from Rome.

There are 1400 Thalassaemia patients registered with the AP Thalassaemia Society. If they have a chance of undergoing BMT, chances of their recovery will be high, says Chandarakant Agarwal, who is responsible for discussing the possibility of establishing the BMT centre here and whose daughter is also a thalassemic patient.

“A unit supported by them in Pakistan has completed 35 bone marrow tranplantations for Thalassaemia.
It has been two years since the tranplantation and the children treated under their protocol are completely cured of the disease and are now healthy. All procedures were performed by Pakistan’s team of doctors trained under them,” Chandrakant said.Parents of children suffering from Thalassaemia were present.


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.”

A girl who was brought into the world to save her brother

August 27, 2010

Courtesy: dnaindia.com

This Raksha Bandhan will be the Bhavsar family’s most memorable yet. Their two-year old daughter Richa, who is also the saviour of her elder brother’s life, will tie him a rakhi in what will be his first truly happy festive celebration till date. While most parents will smile on this day, the Bhavsars will shed a silent tear of happiness as their last bundle of joy asks for health and happiness for the brother who owes her his life.

Richa, the Bhavsars’ youngest child, was brought into the world with the hope that she might save her brother from dying. Urvish, the family’s second child and presently seven-years old, was diagnosed with blood cancer at the age of four. Already a thalassemia major, the only way he could be saved was a bone marrow transplant from a sibling.

Little Richa proved the perfect biological match for her brother. Peripheral blood cells were collected from her blood and a stem cell transplant was undertaken on Urvish. The boy underwent a series of chemotherapy sessions and later, a bone marrow transplant in June. The procedure has borne positive results for Urvish. While his blood cells earlier had XY (male) chromosomes, they now have XX (female) chromosomes, which means that the blood flowing in his body is from his own sister Richa!

Dr Sandeep Shah, head of the Bone Marrow Transplant (BMT) department at Gujarat Cancer Research Institute (GCRI), said that this is a medical miracle where a child has been successfully treated for two major illnesses. Urvish’s blood samples show that the transplant has worked. And he will be cured of both thalassemia and cancer.

“However, he is still admitted at the hospital and has currently developed pneumonia. We wish that along with Richa’s bone marrow which cured him, her best wishes now take him towards good health and speedy recovery,” said Shah, adding that by next Raksha Bandhan, the boy is expected to have completely normalised.

Umang Bhavsar, who runs an optical accessories store in Naroda, was shattered when Urvish was diagnosed with blood cancer, after already being a thalassemia patient since he was three months old.The desperate parents, who were told that a sibling with a bone marrow matching Urvish’s would be the only hope, decided to have a third offspring (their first child’s bone marrow didn’t match with her brother’s).

However, their agony was compounded by the fact that Urvish’s younger brother Harsh, now five, didn’t prove a match. “Finally, in sheer desperation, we planned a fourth child, in the hope that we might prove lucky this time, and get a child who will save Urvish’s life,” said Umang. Richa proved a saviour, as her bone marrow matched 100 per cent with her brother’s.

Dr Shah from GCRI said that so far, 118 BMTs have been done in GCRI, of which six were also cord blood transplants. “However, Urvish’s case is the rarest of all as he was cured of both cancer and thalassemia. And success in his case has inspired us to surge ahead with similar others.”


Newborns and their power to save lives

August 22, 2010

Courtesy by: expressbuzz.com

Doctors say blood obtained from a newborn’s umbilical cord can be used to treat many blood diseases.

“Stem cells extracted from this cord blood as well as cord tissues can be preserved for a long time and used to treat many diseases and cure patients,” said Dr Prem Anand Nagaraja, director of Narayana Hrudayalaya Tissue Bank and Stem Cell Research Centre.

Dr Sharat Damodar, consultant haematologist at Narayana Hrudayalaya, said in absence of a matched donor, stem cells could help cure dreaded heamatological diseases such as thalassemia major, Aplastic anemia, Fanconi’s anemia and 14 such conditions.

Four-year-old Mayur, diagnosed with Fanconi’s anemia, has no sibling (to donate bone marrow) and the marrow from both his parents does not match his HLA. He has to depend on regular blood transfusion to keep up his platelet count. But there is hope for him. His mother is eight-month pregnant and antenatal tests have shown his yet-to-be born sibling is free from Fanconi’s anemia. Mayur can get cured from the stem cells obtained from his younger sibling’s umbilical cord. “Cord blood obtained from the wasted placenta and umbilical cord at the time of childbirth is collected using sterile procedures,” said Dr Nagaraja.

The decision to use a child’s cord blood is made three months before the expected delivery date.

Cord blood is collected immediately after delivery and the window of opportunity to collect and store them is very small. The entire process has to be completed within five minutes of the delivery.


Cryo Cell Pakistan

August 10, 2010

Introduction
Thalassemia is the most common inherited single gene disorder in the world. The thalassemias are a diverse group of genetic blood diseases characterized by absent or decreased production of normal hemoglobin, resulting in a microcytic anemia of varying degree.

Your blood count may be a little lower than other people of your age and sex, but this produces no symptoms. You were born with this condition and you will have it all of your lifetime. There is no need for treatment and most people who have inherited this are not sick and probably do not know they have it. A mild form of Thalassemia minor may be mistaken for iron deficiency anemia. Iron medicines are not usually necessary and will not help your anemia. They could even be harmful if taken over a long period of time.

If you marry a person who does not have Thalassemia Minor, your children may have Thalassemia Minor. If you marry a person who does have Thalassemia Minor, some of your children may have Thalassemia Major. You must decide if you want to take this risk in planning your family.

Symptoms of Thalassemia Major
An infant with Thalassemia Major appears normal at birth. If a child is well for the first five years of life, a diagnosis of Thalassemia Major is unlikely. The double dose of two Thalassemia genes causes an anemia that is so severe that regular blood transfusions must be given throughout life

A newborn with Thalassemia Major appears normal at birth. As they grow, infants with Thalassemia Major exhibit paleness and fussiness. Weakness and slow growth appear in the first or second years of life. The abdomen may swell due to an enlarged liver and spleen. Changes occur in the appearance of the face and head. The eyes appear slanted and the cheekbones become prominent.

Treatment for Thalassemia Major
Treatment involves blood transfusions that must be given every 4 to 6 weeks to sustain life.
Complications that may arise from regular blood transfusions include an overload of iron build up in vital organs causing diabetes, liver disease and heart failure. The spleen may become so enlarged or overactive that it has to be removed surgically. In the past, many patients died in their teens due to these complications.

Management of thalassemia is not enough. Researchers are investigating two potentially curative treatments:  Stem Cell transplantation and gene therapy. Both methods have shown promise.

In stem cell Therapy, there are two ways to go about it.

  1. Bone Marrow Transplant
  2. Cord Blood Transplant

Some children with thalassemia can be cured with a bone marrow transplant. However, this form of treatment is most successful when a donor who is an exact genetic match is available. Generally, a sibling or other family member is most likely to be an exact match. The procedure can cure about 85 percent of children who have a fully matched family donor. However, only about 30 percent of children with thalassemia have a family member who is a suitable donor.

Recent studies suggest that using umbilical cord blood from a newborn sibling may be as effective as a bone marrow transplant. Like bone marrow, cord blood contains unspecialized cells called stem cells that produce all other blood cell.

The beneficial results of stem cell transplantation from HLA identical family members for patients with severe thalassemia are clear. Class I patients have a very high probability of cure with a very low early and late morbidity and mortality. Delay of transplantation until the patient is in a risk category beyond class I substantially reduces the probability of transplant success and jeopardizes the reversibility of liver and cardiac damage. It is reasonable to suggest that patients with β-thalassemia who have HLA-identical donors should be transplanted as soon as possible.

Umbilical cord blood (UCB) has been shown to be capable of reconstituting the bone marrow of the patient with thalassemia after myeloablated pre-conditioning treatment. The major advantage of UCB over other sources of stem cells is the ability to cross HLA barriers, and there is evidence of less GVHD. The use of related – donor UCB stem cells with HLA mismatches at one to three antigens needs to be considered. It would be worthwhile to do a prospective study to evaluate the role of UCB stem cell transplantation in the treatment of the thalassemias and hemoglobinopathies.

Thalassemia is widely distributed throughout the world and is one of the major public health problems. The use of bone marrow transplantation, the only curative therapy for thalassemia, is limited because less than 30% of the patients have unaffected and HLA-identical siblings as donors. Cord blood stem cells, an alternative source of stem cells for transplantation, have been successfully transplanted into patients with several diseases after myeloablative therapy.

Testing for Thalassemia
If a person has Thalassemia Minor, the cause of the slight anemia is known and no other blood tests or treatments such as iron are needed. More important, since individuals with Thalassemia Minor can pass the Thalassemia gene to their children, most people would like to know if there is a risk that their children could inherit this severe blood disease.

A safe and reliable prenatal test to diagnose Thalassemia Major in a fetus as early as 10-12 weeks after conception has been developed. Couples who are at risk may want to consider this possibility.

Success rate of Stem Cell Transplantation (SCT) for Thalassemia?
In low-risk cases (less than 10 years of age, having regular chelation therapy, non liver enlargement and no transfusion-associated diseases like hepatitis or HIV), SCT provides a 80-90% cure probability, with 5% mortality rate and a 10% chance of rejection (thus leaving the child thalassemic).

Cost of storing Umbilical Cord Blood
In Pakistan, one Company namely Cryo Cell Pakistan, with the help of their affiliate in USA, offering collection, extraction and storing services. The normal fee was about USD. 2,000 but they are offering handsome discount on their price in the introductory period. Further details may be obtained from their website www.Cryocell.com.pk.


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