Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering

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

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

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

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

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

Thanks for the update from Pat.

Regards,

Many thanks for the update.

Hoping for the best!!

thank you for sharing Parin!  My prayers are with SK!

Sharmin

The estimated time for study completion extended to July 2016. SMKCC keeps delaying the results outcome. This was originally planned for July 2014. Follow the link below.

https://clinicaltrials.gov/ct2/show/study/NCT01639690

At this point, the only question I have about SK is have the people at the top invested in Bluebird? Information from SK has dried up and no new patients have been started for at least 18 months. The fact that Pat G can't get any info out of them is troubling. Meanwhile, Dr Sadelain has become a co-founder of Juno Therapeutics.

Went Public on Nov 14, 2014 / NASDAQ:JUNO
Funding Received
$310 Million in 3 Rounds from 4 Investors
Headquarters:
Seattle, WA
Description:
Juno Therapeutics, a clinical-stage company, develops immunotherapies for the treatment of cancer.
Founders:
Michael Jensen, Michel Sadelain, Isabelle Rivière, Stan Riddell, Phil Greenberg, Renier Brentjens
Categories:
Health and Wellness, Health Care, Biotechnology
Website:
http://junotherapeutics.com

Sad but true Andy. SK did not communicate well and their preliminary results were disappointing, as we know. All aboard the Bluebird train!

So all hopes on Bluebird....

This is really sad... All hopes pinned to Bluebird now.

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.

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