Thalassemia Patients and Friends
Discussion Forums => Working Towards a Cure => Topic started by: nwalsh528 on October 28, 2012, 10:23:15 PM
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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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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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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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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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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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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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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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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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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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Andy, Thanks for the update. Hoping for more info of the results and next steps from SK...
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Thanks Andy for the update! Will await more detailed version of the results...
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Thanks Andy,
I trust in coming week of July we will have more information .. Thanks for update
Regards
Sushil Thakur
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Thanks Andy
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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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Andy,
Thank you for this information. I hope that the results from the treatment continue to improve.
Sharmin
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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
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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
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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
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Thank you for posting Canadian Family. Hoping that you and your family are doing great!
Sharmin
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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).
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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
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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
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I heard from Pat G and he had this to say about the current status of gene therapy trials.
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.
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Andy,
Thank you for posting. I hope that as they learn - outcomes will get better and better.
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Thanks for the update Andy. I was away on vacation at Disney World creating magic.....
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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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Funding for gene therapy trials has been slow until recently, when well financed companies like Bluebird Bio entered the scene.
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.
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and perhaps eventually, the process can be made strong enough where normal Hb levels are maintained.
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.
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?
World's top 15 military spenders in 2013 -> USA (2013) 640.0 billion
http://en.wikipedia.org/wiki/List_of_countries_by_military_expenditures
Microsoft acquired Mojang for a smooth 2.5 BILLION dollars.
http://mojang.com/2014/09/yes-were-being-bought-by-microsoft/
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/
Snapchat Spurned $3 Billion Acquisition Offer from Facebook
http://blogs.wsj.com/digits/2013/11/13/snapchat-spurned-3-billion-acquisition-offer-from-facebook/
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...
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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.
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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?
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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
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Hi Andy,
are we hearing anything from Dr. Sadelain & team on their trails at Mem. Sloan Kettering?? Any data they are coming out with?
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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
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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
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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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Thanks for the update from Pat.
Regards,
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Many thanks for the update.
Hoping for the best!!
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thank you for sharing Parin! My prayers are with SK!
Sharmin
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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
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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
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Sad but true Andy. SK did not communicate well and their preliminary results were disappointing, as we know. All aboard the Bluebird train!
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So all hopes on Bluebird....
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This is really sad... All hopes pinned to Bluebird now.
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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.
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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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Feeling sad to read Pat's update. Have lots of hope and prayers for Bluebird and Gene Therapy for Thal. Andy, what are your thoughts?
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where can we compare two vectors side by side?
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Jay,
I do not believe that is possible. As far as I have been told, Bluebird will not release specific information about it's vector.
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Secrecy is dangerous! we are called subjects no problem! we are called guinea pigs no problem!
Secrecy is dangerous! we can guess and smell that it may not be a human patch no problem we need to know.
Secrecy is dangerous! it may invite other problems no problem we need to know.
Secrecy is dangerous! it denies fair competition.
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Believe me Jay, I tried to get that info but they won't reveal proprietary information. I prefer the transparency that Michel Sadelain has demonstrated over the years, but I also understand Bluebird's stance.
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But Andy then whats the point in being a listed multimillion setup. There is something called patents, copyrights. I expect at-least after getting through national insurance they will reveal detailed information if that's the birds ambition.
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Collaboration between MSK and Weill Cornell for finding cure to Sickle cell.
Scientists to develop stem cell therapies to treat blood disorders
Scientists and transplant clinicians at the Ansary Stem Cell Institute at Weill Cornell Medical College and the Center for Cell Engineering at Memorial Sloan Kettering Cancer Center have been awarded a $15.7 million, four-year research grant from the New York State Stem Cell Science Program (NYSTEM). The scientists will translate their approach to manipulate hematopoietic stem cells to cure acquired and inherited blood disorders.
For many patients with such blood diseases, including sickle cell disease, the only hope for a cure requires transplanting normal blood stem cells. But in many instances suitable blood stem cells cannot be found or there are too few cells for transplantation. The consortium of scientists seeks to expand production of stem cells outside the body using specialized blood vessel cells – known as a vascular niche – to nurture the stem cells.
The scientists will conduct two clinical trials using this platform to expand hematopoietic stem cells. The first trial uses the vascular niche to expand umbilical cord blood stem cells for transplantation in patients with blood cancers who cannot be cured by chemotherapy or donors. The second trial aims to correct the genetic abnormality in blood stem cells from patients with sickle cell anemia and return these healthy, functioning stem cells to patients. If successful, the techniques may provide safer, broadly available stem cell transplants to thousands of patients.
“This innovative approach marries Weill Cornell Medical College’s stem cell expansion capabilities with Memorial Sloan Kettering’s robust cell engineering and gene-transfer techniques,” said Dr. Shahin Rafii, principal investigator, director of the Ansary Stem Cell Institute and a Weill Cornell professor of medicine, genetic medicine and reproductive medicine.
“We are indebted to our NYSTEM partners for their support, because this award offers the opportunity for new curative therapies for patients with blood malignancies and sickle cell disease,” said Dr. Joseph Scandura, co-principal investigator and a hematopoietic stem cell physician-scientist and scientific director of Weill Cornell’s Dr. Richard T. Silver, Myeloproliferative Neoplasm Center. Scandura is also an assistant professor of clinical medicine at Weill Cornell.
Sickle cell disease is caused by a mutation in the oxygen-carrying protein hemoglobin that distorts the size and shape of red blood cells, causing them to clump together and stick to blood vessel walls, cutting off blood and oxygen supply to vital organs. There are no FDA-approved techniques to expand blood-forming stem cells to cure this disease, and any patients who do not receive transplants of normal blood stem cells risks life-threatening complications.
“The expansion of blood-forming stem cells is a critical advance for the successful implementation of a number of genetic therapies based on gene addition or gene correction,” said Dr. Michel Sadelain, principal investigator at Memorial Sloan Kettering (MSK). “Our combined expertise in stem cell expansion and globin gene therapy for thalassemia is a strong foundation for developing a potentially curative therapy for sickle cell disease,” he said, referring to a blood disorder in which the body makes an abnormal hemoglobin.
Consortium scientists expect the vascular niche platform will generate large numbers of patients’ own blood-forming stem cells, enabling a genetic modification of their stem cells and avoiding the risks of transplanting cells from another person. These two trials will require manufacturing clinical grade human blood vessel, or endothelial cells, for intravenous infusion for clinical trials.
The NYSTEM grant supports a collaborative effort led by Rafii and Scandura at the Ansary Stem Cell Institute at Weill Cornell and Sadelain and Dr. Isabelle Rivière at MSK. Their team brings significant expertise with stem-cell engineering and clinical translation of cell therapies. Phase I clinical trials are expected to start within the next two to three years.
Direct Link: http://news.cornell.edu/stories/2015/05/scientists-develop-stem-cell-therapies-treat-blood-disorders (http://news.cornell.edu/stories/2015/05/scientists-develop-stem-cell-therapies-treat-blood-disorders)
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Thank you for sharing this catchR.
This is very interesting after seeing SK nearly disappear for a while.
Yet this approach seems differently - and the focus seems to be SSA.
I wish this group the best,
I am happy to see our sickle cell brothers & sisters cured as well as our thals.
Sharmin
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Speaking of Dr. Sadelain, I had the chance to talk to him personally in the international conference in abu dhabi in 2013. He is such a wonderful, humble and friendly doctor. At that time, they were testing wheather they will succeed with using immunosuppressive medication rather than the harsh chemotherapy.... He was so excited and gave me a lot of time to explain but unfortunatlly told me intermediates are not on his list at least for the time being....and I can understand why....
I really hope this study will continue and would hear any results in the near future
Manal
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Information verified again on September 16, 2015. Good news is they have not extended the estimated study completion date of July 2016. Next round of verification will be in March 2016. Keep your fingers crossed till next announcement.
https://clinicaltrials.gov/ct2/show/NCT01639690
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Thank you for sharing Canadian Family. I am praying for this because of the reduced intensity preparation required as compared to blue bird.
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Thank you Canadian Family for sharing with us.
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Thank you for sharing Canadian Family. I am praying for this because of the reduced intensity preparation required as compared to blue bird.
Hi Sharmin,
I agree with your assessment. This approach uses low intensity preparation which is much safer, I am more inclined towards this approach compared to Blue Bird's. What I am still not sure is the involvement of Dr. Michel Sadelain in these trails. Last I heard from Andy was that he has its own Bio venture and there were some tension between MSKCC and him. We also have no update from Pat Girondi for a while now. I hope silence brings good news in future for us.
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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.
hello sir
its now oct. 15 ,
how much the team have proceeded, and what we can expect of the best?
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I don't know if Sadelain is still involved with gene therapy at SK. Sadelain became part of a group that started a new company directing research towards gene therapy to cure cancer. This company then had a huge influx of funds from a large pharma company, which I believed created a large windfall for all involved.
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Andy,
Thanks for the update.
MSKCC updated the website yesterday. The major change is "This study is ongoing, but not recruiting participants.". Study completion date remains as July 2016.
https://clinicaltrials.gov/ct2/show/NCT01639690
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SK presented at the meeting this week. I understand that they said that their process appears to be working and that they think the lesser preparation regimen was sufficient.
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Thanks for the update.
So relieved to hear that lesser preparation regimen is still on horizon.
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Thanks Andy for sharing the updates
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Thank you for sharing Andy. As parents we champion the lower intensity to protect our children from the affects of the preparation.
Wishing this group success,
Sharmin
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Thanks Andy for the update! good to hear that progress was being made at SKCC.
Andy, was there any other information that was shared in the meeting. how well were the patients coping, hb raise, etc.
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Andy,
Thanks for sharing the update. multiple options for Gene therapy will always be useful for the patients. Hoping for more good news in ASH conference in December.
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Primary results delayed by another year. Now July 2017......
https://clinicaltrials.gov/ct2/show/NCT01639690
Andy had already briefed us the situation in Sloan Kettering and politics going on. Please make your own conclusion.
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They may just want to study it for another year. My understanding is that the rise in Hb has been steadily going up, but at a slow pace. I'm sure they want to see that this continues, even if slow, before making any conclusions.
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Fair enough.
Thanks Andy
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Another year delay. Estimated completion time has no been extended till July 2018. The study started in 2012. Not sure if they are on right track.....
https://clinicaltrials.gov/ct2/show/NCT01639690
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I spoke with Pat yesterday. He is still in court fighting for these trials to resume. This is the best vector, but trials have been stalled.