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.


Cord blood stem cell transplantation cures minor girl

September 16, 2009

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.


Crucial Differences Between Non-Embryonic and Embryonic Stem Cells

July 13, 2009

Courtesy by: thebulletin.us

We hear a lot about “stem cells,” which are front-and-center as a major policy debate in America, one that involves science, medicine, ethics, politics, and much more.

What are the issues? What’s at stake? What are embryonic and non-embryonic stem cells? What are the crucial differences and distinctions we need to make as a society and citizenry?

Stem-cell technologies are some of the newest and fastest developing biotechnologies. Typically, along with genetic engineering and cloning, these technologies constitute the kind of 21st century advances that make this “the century of Biology.”

A stem cell is a type of cell that is nonspecific in its function; in contrast, for instance, to a heart or brain cell, which is functionally specific. There are two major sources of stem cells: embryonic stem cells and non-embryonic stem cells. Embryonic stem cells are obtained from 5- to 12-day old embryos. Although removal of a stem cell from an embryo kills the embryo, the stem cells are valued for their potential to produce any type of cell. That is, they have high plasticity. Conversely, non-embryonic stem cells are found in large quantities in placenta, umbilical cord blood, amniotic fluid, and in essentially all adult organs or tissues, including bone marrow, fat, kidney, liver, pancreases, intestines, breast, lung, etc. Any of these non-embryonic stem cells have ample plasticity and can give rise to nearly any type of cells, including heart, liver, lung, muscle, etc.

Thus, the heart of the stem-cell controversy centers on the aforementioned fact that the extraction of stem cells from 5- to 12-day embryos kills the embryo. But that’s not the only issue: In addition, stem cells derived from an embryonic human may, in turn, reject the person who receives them. This situation is called graft-versus-host-disease (GVHD). The problem can be avoided by producing an embryonic clone of the person needing the stem cells. However, the procedure produces an embryo that is indistinguishable from an embryo from a fertilized egg. This embryonic clone would be destroyed during the stem-cell harvesting required by the therapy. This type of cloning is called “therapeutic cloning,” since the production of a human baby is not the goal. (Reproductive cloning, producing a cloned human baby, has been universally outlawed.)

Another problem is that the embryonic stem cells can unpredictably cause cancer in the treated patient.

On the other hand, newly developed treatments associated with non-embryonic (adult) stem cells are way ahead of any hoped-for treatments associated with embryonic stem cells. Recent non-embryonic stem-cell therapies include treatments for non-healing bone breaks, healing damaged hearts, regenerating damaged muscles, correcting scoliosis, regenerating knee cartilage, treating thalassemia, osteoarthritis, diabetes, lupus, multiple sclerosis, spinal chord and nerve damage. Treatments to heal conditions associated with almost any organ or tissue are in view. These advances cast serious doubt on the need to develop embryonic stem-cell therapies, especially since embryonic technologies are morally objectionable, given that they require the death of the human embryo.

The use of one’s own adult stem cells (autologous stem-cell transplant) is a way to avoid the problems of rejection and of killing human embryos. Also, certain types of adult stem cells (mesenchymal cells) can be harvested from anyone and changed in the lab (transdifferentiated) into a desired cell. In both of these stem-cell applications there are no adverse effects to the donor of the adult stem cells. The non-embryonic stem cells are safely harvested, purified from other cells and/or expanded in culture, and introduced into the patient without rejection. In another process, virtually any adult cell can be harvested from one’s own body and treated to become cells capable of producing the needed cell type (induced pluripotent stem cells or iPS). These cells can also be cultured in the lab, and reintroduced into the patient. All of these sources of adult stem cells avoid the problem of having to use patented embryonic stem-cell lines that would be less available to the public.

And yet, the reputed plasticity of the embryonic stem cells continues to make the prospects of doing research on human embryos attractive to researchers who are uninhibited by the prospect of killing human embryos.

It is worth pointing out that, in terms of medical applications and treatments, two major facts are usually left out of these discussions: First, non-embryonic stem-cell treatments have been used to treat tens of thousands of patients, and with dramatic benefits. However, embryonic stem cells have not had one clinical trial with humans. Also, it has been clearly demonstrated that non-embryonic stem cells do not produce cancerous tumors in humans. Whether iPS cells share this non-tumorigenic quality is not yet clear. However, iPS cells have all of the medical application value hoped for in embryonic stem cells.

It must be noted that in a field as rapidly moving as stem-cell research, this situation will likely not be current for long. However, the current progress of stem-cell research as of spring 2009 speaks volumes regarding the effectiveness of non-embryonic vs. embryonic stem-cell research. The promises of embryonic stem-cell researchers are wildly overstated. The claims that embryonic stem-cell therapies will be available in five to 10 years rings hollow.

Aside from these scientific considerations, there are moral-religious matters of obvious concerns to Christians:

Christians committed to the sanctity of human life should look with favor on technologies that preserve and/or improve human life. Consequently, non-embryonic stem-cell advances should be embraced when they: 1) respect the consent and preserve the dignity of the stem-cell donors, 2) enhance the health of the stem-cell recipient, and 3) protect human life at every stage of development. Embryonic stem-cell harvesting remains problematic because the procedure destroys the smallest and most helpless members of the human family: embryos.

In truth, embryonic stem-cell use is being trumped by successful and surprising advances in adult and other non-embryonic stem-cell research. These advances protect the dignity of the donor and recipient while recognizing the value of all humans, regardless of their stage of life, from conception through old age. Hence, all frozen human embryos should be given a chance to be born, not given over to researchers to be destroyed for the sake of a research project.


No yardstick set to check quality of imported drugs

July 13, 2009

Courtesy by: nation.com.pk

KARACHI – The Health Ministry has allowed the import of life-saving drugs from China under the FTA and India as well despite the fact that it will greatly diminish the local drugs market worth of Rs1.5 billion, the Nation has learnt.
It has been learnt that the Health Ministry has refused to stop the imports of finished goods that are not produced in Pakistan like anti-cancer vaccines and thalassemia drugs etc, from China and India although there are clear indications that these drugs are not standardised.

For instant, Interferon, an injection for Hepatitis, which is not produced in Pakistan and available at different prices up to Rs 12,000, is being imported from China. But the issue is that no standard has been set to determine its quality that make the lives of millions Pakistanis so vulnerable.

“Though the import of these drugs are allowed, our main concern is that only those drugs should be allowed to Pakistani market that have been registered at least at 2 places in the big countries of the world,” said Qaiser Waheed, ex-chairman Pharmaceutical Association of Pakistan.

He pointed out that Bangladesh had made it mandatory only those drugs should be imported into the country that were registered in 2 countries of Europe or Japan and Australia. However, in Pakistan, the case is entirely different as no system has been put in place to check these drugs coming from China and India, he wondered.

“It is to be noted that the anti-cancer and antibiotics coming from China are not original and have no certified information that make them standardised,” he disclosed.

However, the Health Ministry is of the opinion that China is a friend of Pakistan and there exists FTA between both the countries, therefore, it is not possible for them to stop the import of such life-saving drugs into Pakistan.

It was learnt that the Ministry is habitual to register drugs at the rate of Rs 15,000 and does not carry out any necessary investigation to make it sure whether the drug is worthwhile to use or not. On the other hand, China takes from Rs 0.7 to 0.8 million to register Pakistani drugs and even then they put a clause that the drug has to be tested in the government laboratories for 4 years to make it available for public there.

Interestingly, the Health Ministry is busy in making random registration at cheap rates without considering the fact that human lives are more precious than money, he added.

The Chinese and Indian products are very cheap due to the fact that industrialists there are provided with free land, free power and money to establish industries, but in Pakistan it is a dream to have free land for establishing any sort of industry.

The Pakistani drug market has potential of billions of rupees in which just 25% share is of imports but through the measures of the Health Ministry it has been cumbersome for Pakistan to cope with such issues of future threats by unregistered imported drugs being spread in the Pakistani market.


Businessman a father figure for ‘orphan’ diseases

July 13, 2009

Courtesy by: chicagotribune.com

When Patrick Girondi got into trouble with the law as a teenager, he enlisted into the Air Force to get his life on track. When he had few options as a young adult with no high school diploma, he worked his way up from runner to trader at the Chicago Board of Trade. And when his son Rocco was diagnosed with a rare blood disease at age 2, he left trading for the pharmaceutical industry.

Seventeen years later, Girondi believes a breakthrough is near for thalassemia, a disorder afflicting Rocco and about 100,000 newborns worldwide each year, in which insufficient hemoglobin is produced to carry oxygen throughout the body. Thalassemia patients on average survive to their late 20s, Girondi said.

“I promised God a long time ago, ‘If you help me with Rocco, I’ll do my best to help others,’ ” he said.

Rocco has a severe form of the disease and must have blood transfusions every three to four weeks and pills that work to deplete the excess iron in his system, a result of the transfusions. A year after his diagnosis, he was hospitalized for 40 days to undergo experimental treatments, a $75,000 cost not covered by insurance, Girondi said.

Girondi, who is from the Bridgeport neighborhood, founded the Chicago-based biotech company Errant Gene Therapeutics (EGT) in 2003, and the company has been working to develop products to treat “orphan diseases” like thalassemia. He splits his time between Chicago and southern Italy, where experimental treatments have been more available as the disease affects more children of Mediterranean descent.

An orphan disease is described by the National Institutes of Health as one with fewer than 200,000 cases nationwide. To promote more funding for treatments for them, Girondi created the Orphans Dream Foundation in 2007. About $35 million has gone into the project, he said, with much of the money coming from parents with children suffering from orphan diseases and his fellow traders.

Girondi has high hopes that the answer to thalassemia lies in a controlled virus developed by Michel Sadelain of Memorial Sloan-Kettering Cancer Center in New York City. The first clinical trials of the resulting drug, which would be produced by EGT, may be tested on the first human patients by the end of this year, Girondi said.

Christopher Ballas, the director of gene therapy with EGT and an assistant research professor at Indiana University‘s School of Medicine, said the controlled virus will be used as a carrier to deliver what the patients are lacking. If it works, the technique used for thalassemia could be used to treat other orphan diseases, including sickle cell anemia, Ballas said.

“Here we have a chance to really do something that is of major benefit to a lot of folks who might otherwise be overlooked,” Ballas said.

Girondi hopes the drug will be available to Rocco within two years, but the process of gaining approval for its use is not easy. Still, he already has passed two major hurdles that exist for orphan diseases: funding and attention for diseases that are so rare that research is often focused elsewhere.

His most rewarding moments come from meeting with parents. Tracy VanHoutan was introduced to Girondi through a mutual friend after VanHoutan found out his son had the rare nervous system disorder Batten disease.

Girondi then introduced VanHoutan to the leading researcher on the disease. While funding has stalled on clinical trials for Batten disease, VanHoutan is grateful.

“In a time when I was lost, after we got the diagnosis, Pat was an incredible resource,” VanHoutan said.

Girondi is more modest.

“I’m just the guy in the middle,” he said.


Minority bone-marrow donors are in short supply

July 13, 2009

Courtesy By: tennessean.com

Cam and Rusty Chittaphong update a family Web site to reach out to the local Asian community for their sick daughter.

The Tennessee Chinese News publicized a recent bone marrow drive for the 7-year-old Laotian girl. Fliers and e-mail blasts with Angel Chittaphong’s story circulate at Nashville-area temples, churches, nonprofit organizations and other groups about her rare blood disease, alpha-thalassemia.

Her red blood cells won’t circulate oxygen to her organs, and she gets transfusions with healthy cells to keep her alive. She needs a bone marrow transplant — and it’s likely that a match will be found only in an Asian donor.

“Minorities are the hardest to find matches for bone marrow,” said Tom Burton, executive director of AGAPE, a family service agency for Middle Tennessee. He has organized more than 30 bone marrow drives since 1996 as a volunteer.

Despite growing minority populations in Middle Tennessee, the Chittaphongs are facing an obstacle not exclusive to Asians. Cultural differences, language barriers and a lack of education can hinder minority groups’ awareness about health issues. access to resources and, in the Chittaphongs’ case, ability to find a donor.

African-Americans, American Indians, Asians and Hispanics are among the ethnic groups that have the toughest time finding donors, Burton said.

According to the National Institutes of Health, minorities make up about 20 percent of the bone marrow donor registry, while minorities account for about 40 percent of the transplant waiting list.

“We get the largest response when we go somewhere that is a natural gathering place for them,” said Burton, who helped organize a drive for Angel at Lipscomb University in April. He held a drive at Tennessee State University for a patient who was African-American.

The Sumner Hispanic Alliance hosts an annual Latin Festival at Volunteer State Community College in Gallatin, where the health department is invited to educate Hispanics on different topics, said Cristina Frasier, chairwoman of the Sumner Hispanic Alliance, who owns a translation business for non-English speakers.

The group also visits soccer fields, popular spots for Hispanics. But the people must be comfortable with those offering information.

“There are trust issues,” Frasier said. “It is more effective when someone who speaks their language and looks like them gives the information.”

More education needed

Dr. Haydar Frangoul, director of the pediatric blood and marrow transplant program at Monroe Carell Jr. Children’s Hospital at Vanderbilt, said minorities might not understand what is involved in bone marrow donation because they haven’t been educated. To join the donor registry, a person has to give a swab of his or her mouth. The donation procedure takes about an hour, and recovery is about two days.

“Unfortunately, bone marrow donors have been lumped in with the solid organ donor community,” Frangoul said.

Angel has been on the transplant list since she was born. Three blood and bone marrow drives have been held for her in the past year. Hundreds of people came to each, but the majority were Caucasian donors.

Her disease is a genetic disorder traced to Southeast Asians. Cam Chit taphong lost her first baby at birth because he had the disease, but she did not know it.

With Angel, doctors did two experimental in-utero blood transfusions that worked. Angel had to be delivered early — at 28 weeks — and weighed a little over 2 pounds.

“I knew she was a fighter then,” Cam said, glancing at her daughter. “The doctors were laughing because she was playing with the needle and they had to sedate her to finish the transfusion.”

Last week, Angel was ordering her brother, Jacob, 6, to turn on The Wizard of Oz, her favorite movie.

“I have a Dorothy doll,” Angel said, pointing to her Dorothy and Toto figurines.

Angel is feeling well. She had a blood transfusion Tuesday as part of the daylong regimen that she does every four weeks and will do until she finds a bone marrow match.

“Very few patients have survived beyond birth,” Frangoul said. “This disease is not compatible with life.”

He said the body can eventually reject the transfusions or there can be an unhealthy buildup of iron.

“If she gets a transplant, she will be cured,” said Frangoul, who has been Angel’s doctor since the girl’s birth.

Frangoul said the most recent, reliable number he had on children living with alpha-thalassemia was 11.

Cam Chittaphong is working with leaders of the Asian community to print information in different languages about bone marrow donation for her daughter and others in need.

“We just have to reach out to more of the Asian community and networking groups,” she said. “Our goal is to educate people about being a donor.”


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