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Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering

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Sharmin:
sofear,

The chemotherapy is used to prepare the body to receive the corrected cells.  It suppresses the recipient's immune system and depletes enough of the patient's marrow such that the corrected cells can populate the marrow.

Gene therapy has been a very very long and tedious process hopefully thal majors will be able to benefit from it in the near future.   My husband and I are thal minors - yes, I suffer some fatigue and would love to have higher hemoglobin - but the symptoms I have do not warrant the risk of chemotherapy.   Our son however is a thal major and my prayers daily are for him and all other thal major patients to be cured via gene therapy very soon.  

Perhaps if gene correction becomes safe and routine one day, and does not require chemo preparation then thal minors may be considered for a cure.  

Sharmin

catchR:
Hi Andy,
are we hearing anything from Dr. Sadelain & team on their trails at Mem. Sloan Kettering?? Any data they are coming out with?

Parin:
Updates from Pat Girondi

Dearest family,

Thanks to the prayers and sacrifice of all of you, the help of many as Shobha Tuli, Angela Iacono, Ron Capano, Gina Cioffi... The plane is circling for a landing.

There is so much reason for excitement, Bluebird, Sloan kettering-EGT, Acceleron...

In the midst of decisions that are critical, I again ask for your prayers.

I am always with you and more excited than ever that freedom is upon us. Bluebird marches into Beta (they have to date data on E Thal). Sloan Kettering-EGT collaboration begins 4th patient (Beta).

This is a link for Acceleron which is in clinical trials with another magical solution(thanks for alerting me Ronaldo).

http://www.acceleronpharma.com/products/sotatercept/

yours always, patgirondi

Parin:
Latest mail from  Pat Girondi

My dear family,

Thanks for your many communications, wishes, prayers, questions and comments.

Things are moving. The Sadelain, Sloan Kettering, EGT vector is progressing for Beta thalassemia (sickle cell disease is next).

Bluebird has cured 2 E thalassemics and the CEO promised that they will soon treat Beta thalassemic patients.

I and many others have faith in the Sadelain vector which has reduced transfusions by over 50% in a patient using only a mild preparation of myloe-suppression.

Myloe-suppression is a light dosage of chemotherapy.

Myloeblation as Bluebird uses is harsh and not easily tolerated.

15 will be a wonderful year for us all.

As I promised you many years ago, the cure is close and  I promise to do all I can to make it available for all.

We are close.

My beat,

Patg

Pratik:
Wow,

My eyes got wet reading it. Just goes to show how much Dr. Girondi is committed into this too, from his writing.

Yes, 2015 is going to be an awesome year!

Best,

-P

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