Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering

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Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #15 on: July 30, 2014, 02:15:41 PM »
Hi Andy,

Any update on Sloan Kettering Trial schdule. They were suppose to provide an update by July and we are already ending July. I hope everything fine with their Trial.

Regards
Sushil Thakur

Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #16 on: September 01, 2014, 02:06:16 PM »
Hi All,

I am wondering if there is any update on gene Therapy trial. I trust that Sloan Kettering and Blue Bird are agressive in there trial.

Regards
Sushil Thakur

Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #17 on: September 08, 2014, 02:52:21 PM »
Update has been posted by Sloaan Kettering on Government Website.

Estimated Study Completion Date is now extended to July 2015.

http://clinicaltrials.gov/ct2/show/NCT01639690
Regards.

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Offline Sharmin

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Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #18 on: September 09, 2014, 02:12:35 AM »
Thank you for posting Canadian Family.  Hoping that you and your family are doing great!

Sharmin
Sharmin

Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #19 on: September 09, 2014, 03:43:36 PM »
Thanks for asking Sharmin.

Hope you and your family is doing great, I know the extension is something we were not expecting. I rather have concrete results stemming from this study than just preliminary results (as announced by Blue Bird).

Regards.

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Offline Sharmin

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Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #20 on: September 09, 2014, 07:12:45 PM »
I agree Canadian_Family, it is great to see the promising and rather ground breaking results bluebird bio posted - but I am also praying for Sloan Kettering's slow and steady progress to yield safe and great results by next year.   If the myelosuppression method yields comparable results it would be a better choice for most. 

Sharmin
Sharmin

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Offline Pratik

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Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #21 on: September 13, 2014, 06:37:04 PM »
Thanks for the updates. Indeed, it is much preferred to get a concrete information rather than a bit of a hit and miss.

Till then all my fellow thals (including me, definitely), supercharge your bodies and keep chelating your iron and being healthy! :)

Best,

-P
Every child is special.

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Offline Andy Battaglia

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Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #22 on: September 24, 2014, 02:31:44 PM »
I heard from Pat G and he had this to say about the current status of gene therapy trials.

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The thal family has been investing in gene therapy for almost 2 decades. Forces have been joined from around the globe all pushing in their way towards a cure.

Til today 3 Beta thals have been treated. They were treated at Mem Sloan Kettering by dr boulad using the sadelain vector.  The myelosuppressive pre treatment was easily tolerated. Their ages were from 20-40.

The best result has been in patient number 2 reducing transfusions and all that goes with them by 50%.

This is truly wonderful and if this is the end result, it goes without saying that it is a therapy worth using.

Bluebird has treated 2 E thals from Thailand using myeloablation.

Ski used 8mg/kg and will fortify the dosage of bulsufan on the next 3.

We salute all of the people working so hard to bring this therapy home.

Andy

All we are saying is give thals a chance.

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Offline Sharmin

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Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #23 on: September 26, 2014, 06:24:45 PM »
Andy,

Thank you for posting.  I hope that as they learn - outcomes will get better and better. 
Sharmin

Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #24 on: October 06, 2014, 04:08:53 PM »
Thanks for the update Andy. I was away on vacation at Disney World creating magic.....
Regards.

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Offline sofear

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Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #25 on: October 13, 2014, 10:34:56 AM »
1.) Does anybody know how much money has been used for the studies in the last two decades mentioned?
2.) That one patient has no need for transfusions does not mean he/she is fine without them, right? It's just not needed to "survive" anymore? I assume this person ends up on a level that is better for him/her, but to me it sounds like it's only some "better than nothing"-solution. If I look at the things HIV can cause and how today people can live without any serious issues or an AIDS breakout, I feel there is still incredibly much to achieve in Thalassemia research. I'm sure there can be a cure. How fast we get it will also depend on what financial means are there for research or how the general priorities are (probably low compared to other areas).

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Offline Andy Battaglia

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Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #26 on: October 13, 2014, 03:06:12 PM »
Funding for gene therapy trials has been slow until recently, when well financed companies like Bluebird Bio entered the scene.

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That one patient has no need for transfusions does not mean he/she is fine without them, right? It's just not needed to "survive" anymore?
The object of gene therapy regarding thalassemia is to free patients from transfusions, which would be a vast improvement in the lives of transfusion dependent thals. By allowing a patient to create his/her own blood, it eliminates the health problems related to thalassemia and allows the patient to lead a normal life without the need for the blood of others and the life-threatening iron overload that accompanies thal major. Overall, it will mean the patient is fine without transfusion and perhaps eventually, the process can be made strong enough where normal Hb levels are maintained. Since most thal majors on earth still do not see their 30th birthday, a cure is absolutely essential.
Andy

All we are saying is give thals a chance.

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Offline sofear

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Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #27 on: October 13, 2014, 10:11:57 PM »
and perhaps eventually, the process can be made strong enough where normal Hb levels are maintained.

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Even better would be a "one-time, transformative treatment" that could provide a cure for these patients.

I think this is exactly what I would somehow call a "cure". It seems like this is also a goal that is atleast being recognized, even if it is not the main priority right now.

By allowing a patient to create his/her own blood, it eliminates the health problems related to thalassemia

Correct me if I'm wrong, but if they only reach Hb levels of "transfusion independency", this would also only free them of the serious health problems that majors have in comparison to lighter forms of thalassemia. And if we talk about a "cure" and majors could be treated without serious complications, why should we just ignore the problems of minors and everyone in between and act as if diet, exercise and other things could help us to do everything in a way a "healthy" person can do them? For me, having to build my life around thalassemia is not what I would call a "normal life". I mean, by today's standards, people with a huge variety of diseases are able to live what we call a "normal life".

Since this is only my personal feeling, I'd be curious how many minors/intermedias would do what therapy/take what medication at what "level of risk" to get rid of the problems they are experiencing.

If I go to the doctor and tell him that I'm having depressions, it's taken seriously, I'm being sent to an expert and have a large range of medication even. But I'm sure sufficient Vitamin D, a proper diet, exercise, fresh air, social interaction and other things could easily help people to fix this, while it can't simply "fix" thalassemia - only make us feel better. In contrast, if I tell a doctor that the fatigue my thalassemia causes is a trigger for my depressions and the whole thing starts on a physical level, I'm probably not taken as seriously. I guess it's just better business for all the companies, because the potential "customer base" "in need" of their medication against a wide range of mental problems that are perfectly normal and happen to everyone once in a while and are something that is not caused from a "physical suffering" is large enough to earn a ton of money.

On the general topic of gene therapy:

There is something that shocked me a bit. Gene therapy seems to be extremely important to find a cure for a range of diseases.

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between 2013 and April 2014, US companies invested over $600 million in gene therapy
http://en.wikipedia.org/wiki/Gene_therapy

Sound like much for such an important area?

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World's top 15 military spenders in 2013 -> USA (2013) 640.0 billion
http://en.wikipedia.org/wiki/List_of_countries_by_military_expenditures

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Microsoft acquired Mojang for a smooth 2.5 BILLION dollars.
http://mojang.com/2014/09/yes-were-being-bought-by-microsoft/

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Facebook is acquiring WhatsApp, a company with at most $300M revenues, and 55 employees, for $19billion.
http://www.forbes.com/sites/adamhartung/2014/02/24/zuckerbergs-3-smart-leadership-lessons-from-facebook-buying-whatsapp/

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Snapchat Spurned $3 Billion Acquisition Offer from Facebook
http://blogs.wsj.com/digits/2013/11/13/snapchat-spurned-3-billion-acquisition-offer-from-facebook/

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Snapchat Inc. is near a round of funding at a valuation of about $10 billion from investors including venture capital firm Kleiner Perkins Caufield & Byers, according to a person with knowledge of the matter.
http://www.bloomberg.com/news/2014-08-26/snapchat-said-to-near-funding-with-kleiner-at-10b-value.html

A US smartphone app that is valued as high as the amount of money spent on gene therapy research in the USA in probably decades? Wow, just wow...




« Last Edit: October 13, 2014, 10:31:49 PM by sofear »

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Offline Andy Battaglia

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Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #28 on: October 14, 2014, 12:13:39 AM »
sofear,

Your post shows exactly why thal minors get so frustrated with doctors. Thal minor is not taken seriously by most doctors, even though it can have real effects on quality of life and overall health in general. But thal minor will never be compared to major because major is so much more a severe condition, so the attention once gene therapy is perfected will be towards majors. Because a heavy dose of chemotherapy is required, it is unlikely that this would ever be made available to those with the minor form. It can be justified to save one's life, but it's tough to justify chemo for gene therapy for people with minor conditions. And yes, gene therapy is being investigated for all sorts of genetic disorders and it is potentially a huge financial addition to the medical industry, as well as bringing hope to millions born with genetic disorders. I feel it will change the approach to many conditions, completely eliminating ongoing treatment for many.

I do feel that there is an aspect to the discussion of thal minor that no one seems to want to talk about, but I do feel it has an impact. If it was acknowledged that hundreds of millions of people do have real symptoms from thal minor, it would overwhelm both the organizations and the medical systems. It's a sad state of affairs when the medical system generally won't even give people the basic nutritional counseling that could help them be healthier thal minors. It seems the very least that could be done.
Andy

All we are saying is give thals a chance.

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Offline sofear

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Re: Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
« Reply #29 on: October 14, 2014, 12:39:07 AM »
I've only recently started to read into gene therapy. Is the chemotherapy used to decrease the risk of/avoid cancer, because the viral vectors could start to insert their gene randomly? This would of course be way too serious to use for anything other than severe forms of thalassemia. I guess gene therapy itself will have to evolve much more in order to be useful for genetic defects with less severe symptoms. Though I think if one specific area of gene therapy moves on, gene therapy as a whole could profit from that. This is also what has been mentioned by one of the companies researching gene therapy for thalassemia patients. (Can't find the quote in here anymore)

Edit: Hm, should additional treatments like a chemotherapy not be unnecessary in a "perfectly" working gene therapy? And should this not be the goal to aim for in gene therapy in general?
« Last Edit: October 14, 2014, 12:57:01 AM by sofear »

 

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