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54504 Posts in 5759 Topics by 5929 Members
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A message for all  parents who are thals. Keeping your iron load under control is an absolute obligation to your children.
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Author Topic: Bluebird Bio Releases Data on Current Gene Therapy Trials  (Read 58605 times)
Parin
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« Reply #165 on: June 23, 2017, 11:37:39 AM »

http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2282785

bluebird bio Presents New Data from HGB-205 Study of LentiGlobinTM Drug Product in Patients with Transfusion-Dependent β-Thalassemia (TDT) and Severe Sickle Cell Disease (SCD) at European Hematology Association (EHA) Annual Meeting

–Ongoing transfusion independence up to 3.5 years in patients with transfusion-dependent β-thalassemia (TDT); three patients have discontinued iron chelation –
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Andy
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« Reply #166 on: June 24, 2017, 12:47:59 PM »

You beat me to it. In was about to post this the other day when I saw you had posted.

Overall, this report was good news.
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Andy

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Pratik
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« Reply #167 on: June 26, 2017, 08:48:37 AM »

Would this require Chemo?

Was there any other drug that produced Hb too?
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Andy
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« Reply #168 on: June 26, 2017, 06:55:13 PM »

Pratik, chemotherapy is required to prepare the patient for the procedure. Gene therapy is a permanent cure. It is very good news that some of the earlier patients in the trials are no longer chelating. Also, their new product appears to be improved, which is good news as they head to a beta zero only trial.

The drug we've been following that increases Hb is Luspatercept. This is not a cure, but an ongoing treatment, but it will probably be the more easily accessible, as gene therapy will come with a high price tag. Phase 3 trials for Luspatercept are now full and underway. There is a good chance it will hit the market by 2020 if the trials go well. The early trials have gone exceptionally well. Depending on the transfusion needs of the patient, this drug should eliminate the need for transfusion in intermedia patients, including many with HbE beta thal, and also greatly reduce the transfusion and chelation requirements of almost all thals. The drug reduces ineffective erythropoiesis, which lowers iron absorption, while also reducing the amount of iron being taken from transfusions. I anticipate that many patients currently using drugs like hydroxyurea and thalidomide will switch to Luspatercept once it is available. I see the other drugs as a temporary measure until better comes along. Better is really close.
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Andy

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Sunny2701
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« Reply #169 on: April 14, 2018, 03:26:39 PM »

Thats a great insight Andy. Are there any new updates on it since your last post?


Pratik, chemotherapy is required to prepare the patient for the procedure. Gene therapy is a permanent cure. It is very good news that some of the earlier patients in the trials are no longer chelating. Also, their new product appears to be improved, which is good news as they head to a beta zero only trial.

The drug we've been following that increases Hb is Luspatercept. This is not a cure, but an ongoing treatment, but it will probably be the more easily accessible, as gene therapy will come with a high price tag. Phase 3 trials for Luspatercept are now full and underway. There is a good chance it will hit the market by 2020 if the trials go well. The early trials have gone exceptionally well. Depending on the transfusion needs of the patient, this drug should eliminate the need for transfusion in intermedia patients, including many with HbE beta thal, and also greatly reduce the transfusion and chelation requirements of almost all thals. The drug reduces ineffective erythropoiesis, which lowers iron absorption, while also reducing the amount of iron being taken from transfusions. I anticipate that many patients currently using drugs like hydroxyurea and thalidomide will switch to Luspatercept once it is available. I see the other drugs as a temporary measure until better comes along. Better is really close.
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Andy
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« Reply #170 on: April 20, 2018, 04:37:21 PM »

Yes, there is an update and you posted it. Hehe. The big news is that approval will be sought in Europe this year.
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Andy

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