Thai, French scientists manage breakthrough in curing thalassemia

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Thai, French scientists manage breakthrough in curing thalassemia

By PRATCH RUJIVANAROM 
THE NATION 

 

 

THAI and French physician-scientists yesterday announced a method for correcting thalassemia, a blood disorder, by gene therapy – a world first in medicine.

 

The team of researchers, led by globally renowned gene therapist Professor Dr Philippe Leboulch of the University of Paris and Harvard Medical School, revealed that this new technique to cure thalassemia is expected be available to the general public in France by next year. Patients in Thailand will have to wait a little longer, as gene therapy for treating thalassemia is still in the clinical trial process.

 

The results of the trial on 12 patients by a group of Thai and French scientists were published in the April 19 issue of The New England Journal of Medicine.

 

Beta-thalassemia has been especially frequent in Thailand and Southeast Asia, and approximately 3,000 babies are born each year in Thailand with a severe form of beta-thalassemia that requires life-long, monthly transfusions. Even with this treatment, complications frequently occur, resulting in a decreased life expectancy. Beta-thalassemia imposes an enormous financial burden on affected nations.

 

 

After Leboulch published the proof of principle of the efficacy of the treatment on single beta-thalassemia and sickle cell patients, he collaborated with Dr Suradej Hongeng and Dr Suthat Fucharoen from Mahidol University in major worldwide phase-two trials to evaluate the gene therapy approach, called LentiGlobin, in a large cohort of patients.

 

The therapeutic vector integrates permanently a corrective normal beta-globin gene into the patients’ blood stem cells, after the defective cells have been first eliminated. Twelve patients with severe beta+- or betaE/beta0- thalassemia – the most common in Thailand – are now free of having transfusions after they were injected with an effective dose of cells that had received the vector, Leboulch said.

 

 

He revealed that the result of trials on humans was very promising, as all 12 thalassemia patients had been cured. It is expected that the therapeutic effect will last for the rest of their lives. Nevertheless, he stated that the safety profile of LentiGlobin, which remains under investigation, has been consistent with myeloablative conditioning with the chemotherapy agent busulfan.

 

Leboulch said that European countries would be the first to adopt this new thalassemia treatment, with French people having access by next year.

 

The cost for curing thalassemia with this new technique was not yet finalised, but he insisted that government health policy would play a major role in keeping it affordable for the general public.

 

 

“We are delighted that our patients are doing fantastic, no longer requiring blood transfusions, whereas they were previously transfused monthly since early childhood. They go to school or work, play sports, and now live a normal life,” said Dr Suradej.

 

He pointed out that existing treatments for thalassemia are expensive, have many side effects, and cast a heavy burden on our health system. The cost of constantly giving blood transfusions to one person for 30 years is up to Bt10 million, and it is estimated that 40 per cent of the stock at Thai Red Cross’s blood bank is used for thalassemia patients.

 

The only curative treatment currently available is transplantation of donor blood stem cells from a healthy, compatible donor. However, only about 25 per cent of patients can find a suitable donor.

 

Source: http://www.nationmultimedia.com/detail/national/30344482

 

-- © Copyright The Nation 2018-05-03

 

[candide]

Of course they put Thai first in the title, despite the fact that they participated knly in phase two trial....

[Lupatria]

1 hour ago, candide said:

Of course they put Thai first in the title, despite the fact that they participated knly in phase two trial....

Exactly. But then I thought the genuine scientist may crave for a freshly brewed coffee during their work.  

[geriatrickid]

Wow, here's a conflict of interest that the Nation created because it failed to report accurately. The principal investigator of the trial is Marina Cavazzana. The sponsor of the trial is  the US public company, bluebird bio. (They paid for it). Philippe Leboulch was a scientific founder of bluebird bio and serves as the co-chairman of its Scientific Advisory Board.  You have to mention  his relationship with the sponsor.

 

The Nation also seems to have missed the fact that of the 47 researchers credited, only 2 were Thai and they were basically participants carrying out the trial.  It also overlooked the fact  the drug was made available for trial courtesy of the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) classification and fast tracking.

 

The two Thai physicians (Usanarat Anurathapan & Suradej Hongengs) are both at  the pediatrics department at  Bumrungrad International Hospital. I don't know why there is no mention of  Dr. Anurathapan by the Nation.  Dr Suthat Fucharoen is not credited in the clinical trial, so I have no idea why the Nation made a fuss over him.

 

bluebird bio is a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies. The company’s objective is to develop and bring to market the most advanced products based on the transformative potential of gene therapy to provide patients hope for a better life in the face of limited or no long-term safe and effective treatment options. Headquartered in Cambridge, Massachusetts, bluebird bio has operations  Seattle, Washington, Durham, North Carolina &  Zug, Switzerland.

 

LentiGlobin was granted Orphan Drug status by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of β-thalassemia and SCD. The FDA granted Breakthrough Therapy designation to LentiGlobin for the treatment of transfusion-dependent patients with β-thalassemia major and Fast-Track Designation for the treatment of beta-thalassemia major and for the treatment of certain patients with severe SCD. bluebird bio is participating in the EMA’s Adaptive Pathways pilot program, which is part of the EMA’s effort to improve timely access for patients to new medicines. The EMA granted Priority Medicines (PRIME) eligibility to LentiGlobin for the treatment of TDT.

 

[Golden Triangle]

Thailand, the hub of all cures 

[JAG]

Thailand, the hub of all cures 

Yes, but it rather looks genuine, an international effort with some Thai participation rather than a "Thai cure" like the one for ebola.

I hope it is. "Best Beloved" is a carrier for Thalassemia, so there was a risk that
"Little Miss JAG" could have been born with the condition. Anxious times. She wasn't, but the gene could be passed on to future generations, so a "cure" is good news.

Sent from my BG2-U03 using Thailand Forum - Thaivisa mobile app

[apophyss]

I don't see Mr Joke (from Tourist Police)

where is him ?

[Artisi]

On 03/05/2018 at 10:27 AM, geriatrickid said:

Wow, here's a conflict of interest that the Nation created because it failed to report accurately. The principal investigator of the trial is Marina Cavazzana. The sponsor of the trial is  the US public company, bluebird bio. (They paid for it). Philippe Leboulch was a scientific founder of bluebird bio and serves as the co-chairman of its Scientific Advisory Board.  You have to mention  his relationship with the sponsor.

 

The Nation also seems to have missed the fact that of the 47 researchers credited, only 2 were Thai and they were basically participants carrying out the trial.  It also overlooked the fact  the drug was made available for trial courtesy of the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) classification and fast tracking.

 

The two Thai physicians (Usanarat Anurathapan & Suradej Hongengs) are both at  the pediatrics department at  Bumrungrad International Hospital. I don't know why there is no mention of  Dr. Anurathapan by the Nation.  Dr Suthat Fucharoen is not credited in the clinical trial, so I have no idea why the Nation made a fuss over him.

 

bluebird bio is a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies. The company’s objective is to develop and bring to market the most advanced products based on the transformative potential of gene therapy to provide patients hope for a better life in the face of limited or no long-term safe and effective treatment options. Headquartered in Cambridge, Massachusetts, bluebird bio has operations  Seattle, Washington, Durham, North Carolina &  Zug, Switzerland.

 

LentiGlobin was granted Orphan Drug status by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of β-thalassemia and SCD. The FDA granted Breakthrough Therapy designation to LentiGlobin for the treatment of transfusion-dependent patients with β-thalassemia major and Fast-Track Designation for the treatment of beta-thalassemia major and for the treatment of certain patients with severe SCD. bluebird bio is participating in the EMA’s Adaptive Pathways pilot program, which is part of the EMA’s effort to improve timely access for patients to new medicines. The EMA granted Priority Medicines (PRIME) eligibility to LentiGlobin for the treatment of TDT.

 

Any extremely well presented and interesting post.

Unfortunately you over looked the fact that Thailand is the hub of all things - irrespective of any minimal input to the event. 

[Darcula]

What exactly was the Thai role? Supplying guinea pigs?