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Bluebird Presents At ASH 12/15

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Andy Battaglia:
The summary of Bluebird's presentation at ASH is now available. We have seen most of this information previously, but there is one very important addition to this info. A new study of beta zero patients will be designed and conducted. As I have suggested previously, there are alterations that can be done to preparation and dosing that may alter the outcome in beta zero patients. I hope to attend the Bluebird webinar on Thursday to hopefully get more information.


--- Quote ---Summary of ASH Data from bluebird bio
The results shared at ASH continue to support the potential of LentiGlobin BB305 gene therapy in patients with beta-thalassemia major and severe sickle cell disease. No severe drug-product related adverse events have been observed.
 
Beta-thalassemia
·         People living with beta-thalassemia major can have different genotypes. Differences in outcomes among patients treated with LentiGlobin BB305 gene therapy have been noted among the genotypes.
·         In the Northstar (HGB-204) study, transfusion independence has been achieved in all five of the beta-thalassemia patients with non-beta-0/beta-0 genotype treated at least six months ago. Four other patients with the beta-0/beta-0 genotype have had reduced transfusion needs (33 to 100% decrease in volume), but are not transfusion-free.
·         In the HGB-205 study, two patients with the beta-E/beta-0 genotype have remained transfusion-free for 18 and 21 months since treatment, respectively.
·         Several other patients have been treated in both studies, but it’s too early to know their outcomes.
·        bluebird bio plans to open a new study for patients with the beta-0/beta-0 genotype and plans to move forward with the HGB-207 and HGB-208 studies only in patients with the non-beta-0/beta-0 genotype.
 
Sickle cell disease
·         In the HGB-205 study that also included patients with severe sickle cell disease, the first patient treated with LentiGlobin BB305 is producing more anti-sickling hemoglobin (49% of total hemoglobin), has been transfusion-free for nine months, and has not had a post-treatment hospitalization for a sickle cell disease complication.
·         In the HGB-206 study, three patients have been treated and additional patients are undergoing screening. Early data on two of these patients shows a gradual increase in anti-sickling hemoglobin.
 
Multiple Myeloma
·         Three abstracts describe pre-clinical data supporting development of the chimeric antigen receptor (CAR) T cell therapy bb2121 in multiple myeloma, including insights into the manufacturing platform and engineering of more potent CARs.
--- End quote ---

Canadian_Family:
Encouraging results.

Is it that Northstar HGB204 study, Bluebird treated 4 patients with beta 0/beta0 genotype and found reduced tansfusion needs, which encourages them to go with HGB207 studies.

Thanks for the update.

Andy Battaglia:
I'm wondering more about the success with the sickle cell patient, because the dosing and conditioning are different. There may be something that can be applied to beta zero patients.

Canadian_Family:
Point well taken.

Things seems to be moving in the right direction for both thal and sickle.

Sharmin:
Thank you for sharing Andy.  This is all positive news of progress.  

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