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Author Topic: Bluebird bio Webinar 12/10/15  (Read 6487 times)
Andy Battaglia
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Will thal rule you or will you rule thal?


« on: December 10, 2015, 02:06:06 PM »

I took part in bluebird bio's webinar today. I wanted to get their point of view of the recent negative press that has been directed at the gene therapy efforts. Personally, I feel that those making these negative reports have an agenda that includes attempting to lower the stock value, so they can buy it at a cheaper price. Following this webinar, I feel even more strongly about that.

Bluebird will begin trials on beta zero patients in a separate trial. They pointed out that it is still early in the trials and the results so far have been very good overall. They do expect to continue to learn which approaches work best with different genotypes. When I asked if they were optimistic about the future of their attempts with beta zero patients, without hesitation, the doctor stated that they are very optimistic. He mentioned that they continue to work on improving the vector used. We also need to understand that these are early trials and that things like the preparation and the dosing are variables that will become less variable once they have done more patients and see what is working best for different sets of patients. He also mentioned that in most of the beta zero patients already treated, there is a slow ongoing increase in the hemoglobin produced by the new gene.

I remain quite optimistic about the future of gene therapy. Today's vector is far superior to the original vector and there is reason to believe that further improvements will be made. And we must not forget that there are other efforts going on to cure thalassemia with gene therapy, and we know very little about the potential of these other efforts.
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Andy

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« Reply #1 on: December 10, 2015, 06:29:18 PM »

Andy,

a big Thank you for attending BlueBird Bio's webinar even with all your busy life and giving us an update. your understanding gives us more hope now.
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« Reply #2 on: December 11, 2015, 09:11:17 AM »

Valuable information. Thanks Andy.

What I liked most about the information is that they don't want to paint everybody from the same brush. Different approaches is required for different genotypes. Second, I hope with the improvement of the vector, most likely less conditioning of the patients with less side effects.

Last, we hope to hear from MSKCC soon. Healthy competition is infact good for everybody.
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« Reply #3 on: December 11, 2015, 10:45:08 AM »

Thank you for sharing this Andy.  We must not lose sight of the fact that immense immense progress has been made and eventually these efforts will see us to a cure.  We must keep this passion and positive attitude going if we want it to happen. 

Thank you again,

Sharmin
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Andy Battaglia
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« Reply #4 on: December 11, 2015, 11:28:23 AM »

I am glad to see a better understanding of this process from our friends in this group than I am seeing on Facebook. People are trying to dismiss gene therapy because the first efforts with beta zero patients have not made them completely transfusion free, even though their Hb levels has risen more than 5 points. I think it's a fantastic first effort, and as Canadian family said, you can't paint everyone with the same brush.

I am in the midst of arranging some phone calls for next week which should shed a little light on where they think this will lead in the future. I think we all need to maintain hope and faith that they will continue to refine this process, so that it will help all beta thal patients. They have already succeeded in the process that will cure well over half the thal majors on earth, so I say, let's have faith that these excellent scientists will continue to improve this process, just as they have done over the past 5 years.
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Andy

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« Reply #5 on: December 12, 2015, 12:17:36 PM »

Well Said Andy.

All great inventions took several shots to perfect them. Read history, tell me one invention which was invented in first try...........
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Andy Battaglia
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« Reply #6 on: December 13, 2015, 02:21:03 PM »

I've actually been quite surprised at how well the first efforts at gene therapy have gone. Many said it had no hope at all. I truly believe that we will eventually see gene therapy understood well enough to cure a hoist of disorders.
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Andy

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Sharmin
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« Reply #7 on: December 14, 2015, 10:37:49 AM »

I agree Andy and Canadian Family, the immense progress we have seen in the last few years is beyond belief.  Even a hemoglobin of 10 would be great for beta zeros.  Heck, my grandma lived to be 90 with a hemoglobin of 10 - and she did not stop walking and working until her last breath.  Of course even higher hemoglobins are likely possible and would be ideal.  We also need to realize how gene therapy is eliminating most of the risks of BMT - bmt is associated with higher mortality risks and gvhd.  I am grateful for the progress thus far and hope that more progress is made soon. 

Thank you again Andy.   

Sharmin
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« Reply #8 on: December 17, 2015, 12:14:57 AM »

Thanks Andy for sharing this information!! we have lot of hope pinned on success of gene therapy and strongly believe that bluebird will make it reality in coming years...


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