Discussion Forums > Working Towards a Cure
Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
JV:
Sad but true Andy. SK did not communicate well and their preliminary results were disappointing, as we know. All aboard the Bluebird train!
Parin:
So all hopes on Bluebird....
catchR:
This is really sad... All hopes pinned to Bluebird now.
Sharmin:
Although I am very happy about the success bluebird has had, I am very disappointed that SK seems to have lost momentum. My thoughts are with Pat.
Parin:
Latest update from Patg The 'g's for Gene Therapy
My dear family,
Happy Holicays...
Gene therapy is getting closer. Bluebird has now treated beta 0 beta 0 as well as E beta.
We, the Sadelain team are blocked at 3 and this is very concerning as many are convinced that the Sadelain vector is safer and better.
We hoping that the log jam is moved quickly.
I say this because it was just published in 'Current Gene Therapy' of Feb15 that the Bluebird vector is very similar to the vector used that caused a halt in clinical trials because it created clonal dominance (assertional mutagenisis).
The biggest change in the Bluebird vector ia that the cHS4 insulator was removed.
We need to see the Sadelain vector get a chance.
Bluebird used the heaviest chemotherapeutic regiment on their patients. Sadelain (we) used a mild dosage resulting in 20% (est) of the Bluebird dosage.
We treated the toughest patients, they were also older than Bluebird's patients.
Our results were fabulous, significant reduction in transfusions.
Our first patient was treated in the 4th quarter of12, Bluebird in 14.
You cannot compare 20% dosage with the worst patients against 100% against milder forms of the disease.
The whole situation is confusing and conflicting.
We, (Americans) saw the premature deaths of our brothers and sisters over the desferal scandal. Those of you who don't know about it forgive me.
The book, 'The Drug Trial' vividly explains how money, ego, treachery and arrogance can damage and even kill patients.
We don't need this to happen again with gene therapy.
The patient deserves the best drug not the best funded drug.
Please send your prayers and warmest wishes to all involved.
We have been speaking about 'The cure' for over a decade. Don't get discouraged now.
A big salute of admiration to all of our centers across the globe...
Happy Holidays and may the Orphan Dream be yours.
patg
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