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A message for all  parents who are thals. Keeping your iron load under control is an absolute obligation to your children.
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Author Topic: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering  (Read 46206 times)
nwalsh528
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« on: October 28, 2012, 05:23:15 PM »

I was so encouraged to read this. For those of us who would not attempt a BMT (with or without a perfect sibling match), this (if successful) would truly be a "cure" without risking host v. graft. The patient's OWN stem cells would be used. Hoping the vector being used in these clinical trials will have better results than previous one(s) (as they caused cancer...)
http://www.mskcc.org/blog/launch-stem-cell-therapy-trial-offers-hope-patients-inherited-blood-disorder
Nicole
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Nicoler

My son, adopted from China at age 3 in April 2010, has beta thalassemia major.
Andy Battaglia
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« Reply #1 on: October 29, 2012, 09:33:05 AM »

The very first gene therapy trial did lead to cancer, but since then, the type of virus used for the vector has been changed. This vector was developed by Dr Sadelain's group through Thalagen and was used in the single patient French trial of a few years ago. No cancer has developed in this patient and it is not expected with the new vector. Since the French trial, the vector has been improved and is now at least 10 times as potent. This vector will be used in the trial at SK.
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Andy

All we are saying is give thals a chance.
nwalsh528
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« Reply #2 on: October 29, 2012, 06:39:11 PM »

The very first gene therapy trial did lead to cancer, but since then, the type of virus used for the vector has been changed. This vector was developed by Dr Sadelain's group through Thalagen and was used in the single patient French trial of a few years ago. No cancer has developed in this patient and it is not expected with the new vector. Since the French trial, the vector has been improved and is now at least 10 times as potent. This vector will be used in the trial at SK.
This is great to hear regarding the vector. I realize there is a big difference between a stage 1 and stage 2 clinical trials, but to even be at the point of the stage 1 is amazing and encouraging!
Nicole
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Nicoler

My son, adopted from China at age 3 in April 2010, has beta thalassemia major.
Shaheera
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« Reply #3 on: June 15, 2014, 10:57:18 AM »

Bluebird's gene therapy 2.0 weans beta-thalassemia patients off blood transfusions

June 14, 2014 | By John Carroll

Nick Leschly, CEO of Bluebird Bio
Gene therapy 2.0 at bluebird bio is demonstrating some early signs of success with an upgrade to its experimental therapy for the genetic blood disorder beta-thalassemia demonstrating promising results in a pair of patients, weaning them off the regular blood transfusions needed to stay alive.

Cambridge, MA-based Bluebird ($BLUE) has been testing an improved lentiviral vector needed to get a corrective beta-globin gene in place to repair their ability to produce hemoglobin in a study of LentiGlobin BB305. One of those subjects produced 6.6 g/dL of therapeutic betaAT87Q-globin at 4.5 months as a result of the therapy and another hit 4.2 g/dL of betaAT87Q-globin at two months--enough to prevent the need for further monthly transfusions and symptoms like severe anemia and splenomegaly, at least for now.

Bluebird has been tinkering with its lentiviral tech to come up with a new-and-improved therapy that can be easier to produce and more effective for patients. Their presentation this weekend at a scientific conference in Milan suggests that their work is paying off as hoped for, and bluebird CEO Nick Leschly tells FierceBiotech that the results provide a positive sign that the new tech can be more widely used in its other pipeline projects.

"If you step back," says Leschly, "one of the big questions is how do you do gene therapy in an industrial fashion, one that is scalable and reproducible." And this new product "well exceeded our expectations."

Gene therapy work has been going on in the clinic for decades now, but a string of biotechs like bluebird feel that they've managed to overcome the flaws that destroyed so many earlier efforts. And in small studies like these, they've been presenting key proof-of-concept data for a new wave of treatments pointed to future registration trials. In bluebird's case, stem cells are extracted from patient and then treated with the lentivirus before being reinjected into the patient, with the correcting gene sequence inserted into the chromosome.

Success for this approach on beta-thalassemia could be defined as fewer transfusions, says Leschly. "If we can get near transfusion independent, that would be spectacular." Even better would be a "one-time, transformative treatment" that could provide a cure for these patients. The biotech is also looking for, and seeing, a much faster rate of response.

Put in context, adds Dave Davidson, the chief medical officer in bluebird and a veteran of Genzyme, the earlier version of this therapy took a year to generate enough hemoglobin in one of the patients. Now investigators are counting the initial response in a matter of days.

For the two subjects treated in bluebird's study using the earlier version lentiviral HPV569 product, one still no longer requires blood transfusions after 72 months. The two are producing 2.7 g/dL and 0.4 g/dL of therapeutic betaA-T87Q-globin post-transplant, respectively.

In this particular early-stage study bluebird has now recruited four of the 7 patients it needs, while investigators are also at work scouting for 15 patients needed for a sister study.

Bluebird, a 2012 Fierce 15 company, was one of a group of star biotechs to put together successful IPOs last year. Initially backed by Third Rock and Genzyme, the team reorganized and renamed Genetix back in 2010 and went on to strike a CAR-T development deal with Celgene ($CELG). The lead program at bluebird is focused on childhood cerebral ALD while investigators are also pursuing another program for LentiGlobin in sickle cell disease as well.

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Faheem Sultan - Father of Shaheera
Parin
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« Reply #4 on: June 16, 2014, 01:14:18 AM »

Wow, That's a great and promising news.

Any timelines when it will be open for all thalassemia patient?

Regards,
Ashutosh
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Andy Battaglia
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« Reply #5 on: June 20, 2014, 07:01:20 PM »

It is far too early to give any estimates on when this will be available outside of trials. Years of monitoring will be needed to see if any long term side effects develop and if the gene will continue to produce beta globin as years go by. Trials will remain the only way to take advantage of this for the time being.
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Andy

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catchR
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« Reply #6 on: July 09, 2014, 02:00:56 PM »

Hello Andy,
Its July'14 now and do we have any feelers / tentative dates on when Dr. Sadelain and team would publish their work on thalgen trails??

Also, do we know anything from Alicia Somma (part of trails) on how well things have worked out?

fingers crossed....hoping for the very best!!
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Andy Battaglia
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« Reply #7 on: July 10, 2014, 01:42:56 PM »

I just got off the phone with Pat Girondi. They have a meeting come up soon and we should be able to get more information after that. He did tell me that the first two patients who started at SK have a greatly reduced transfusion regimen now, and a third patient in his 40's has had some gene expression but less than is needed. It may be that a higher dose is needed in older patients. Keep in mind that these are all beta thal majors and not HbE beta thals, as the early patients that Bluebird has treated are.
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Andy

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« Reply #8 on: July 10, 2014, 03:14:01 PM »

Hi Andy,

Thanks for the update. I think its about time for SK to announce the results. Looking forward to more detailed version of results.
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« Reply #9 on: July 10, 2014, 07:32:22 PM »

Andy, Thanks for the update. Hoping for more info of the results and next steps from SK...
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catchR
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« Reply #10 on: July 11, 2014, 03:58:21 AM »

Thanks Andy for the update! Will await more detailed version of the results...
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Sushil
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« Reply #11 on: July 11, 2014, 09:24:36 AM »

Thanks Andy,

I trust in coming week of July we will have more information .. Thanks for update

Regards
Sushil Thakur
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JV
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« Reply #12 on: July 11, 2014, 12:03:05 PM »

Thanks Andy
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Pratik
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« Reply #13 on: July 18, 2014, 04:30:51 AM »

I am really hoping for the best!

Yes, it's about time Sloan Kettering releases the results as I think it was supposed to be done sometime in July this year?

Andy, would be eagerly waiting for your response!

-P.
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Sharmin
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Little A


« Reply #14 on: July 20, 2014, 05:30:02 PM »

Andy,

Thank you for this information.  I hope that the results from the treatment continue to improve.

Sharmin
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Sharmin
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