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[Parin]

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 –

[Andy Battaglia]

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.

[Pratik]

Would this require Chemo?

Was there any other drug that produced Hb too?

[Andy Battaglia]

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.

[Sunny2701]

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.

[Andy Battaglia]

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

[nijaguna]

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.

Maybe I am wrong but will the gene therapy be tried on minors as well? Does the gene therapy, if successful, be able to eliminate the genes from the germline thereby preventing future generations from the disease?

[Canadian_Family]

Hi,

Minors do not need a gene therapy considering its risks and cost. Prevention is the only way to eliminate thalassemia. All thal minors should check in with their potential spouses if they have thal minor. Take a calculated risk....

[Andy Battaglia]

Agreed with CF. Because it requires chemo conditioning, it would not be considered for minors.

[nitarora]

Hi Andy,

I am a silent Visitor for this website since long.. Now feeling to get some query addressed

My son is Major one & we are regularly Visting Dr. Vijay Pune-India. Now My query to you is Besides Blue bird I heard from Dr. Vijay that Gene Therapy  had been successfully Tested in France too by Dr. Marina Cavazaana.

Do you  have any Informationfor the same?

BR//
Nitin Arora

[Andy Battaglia]

Professor Cavazzana is the primary investigator of the HGB-205 study for bluebird.

[Parin]

bluebird bio’s LentiGlobin™ Gene Therapy Granted Accelerated Assessment by European Medicines Agency for the Treatment of Transfusion-Dependent β-Thalassemia

http://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bios-lentiglobintm-gene-therapy-granted-accelerated

[Andy Battaglia]

It's very close to a reality.

[Parin]

Yes, any idea about USA?

[Andy Battaglia]

No idea yet about the US, but the reason why Europe was chosen first is explained in a very in depth article at http://www.pmlive.com/pharma_intelligence/Bluebird_Bio_on_the_cusp_of_a_gene_therapy_revolution_1246188