Thalassemia Patients and Friends
Discussion Forums => Announcements => Topic started by: 7assan on October 30, 2010, 09:16:33 AM
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Written by Craig Butler
Friday, 29 October 2010 13:21
October 29, 2010 - TAG (Thalassemia Action Group), the patient-run support group of CAF, will be holding an informational meeting on gene therapy in thalassemia at Memorial Sloan-Kettering Cancer Center (MSKCC) in New York City on Sunday, November 14 from 11:30 a.m. to 1:30 p.m.
Entitled "Gene Therapy for Homozygous Beta Thalassemia - Overview and Update," the meeting will feature a presentation by Drs. Farid Boulad and Michel Sadelain of MSKCC, who have extensive knowledge of and experience in this area and are in the process of developing a thalassemia gene therapy trial themselves.
The meeting is open to individuals with thalassemia and family members of individuals with thalassemia and will be held in room M107, 1275 York Avenue in New York City. The entrance to the building is between 67th and 68th Streets; at the entrance, go up the escalator, make a U-turn and take the hallway leading to the cafeteria. Room M107 is the last room on the left of that first part of the hallway.
Farid Boulad
Michel Sadelain
Farid Boulad, MD, is Medical Director, Pediatric Day Hospital at MSKCC with clinical expertise in Allogeneic and Autologous Bone Marrow and Peripheral Blood Stem Cell Transplantation. Michel Sadelain, MD, PhD, is Director, Gene Transfer and Somatic Cell Engineering Facility at MSKCC with clinical expertise in Gene Transfer and Genetic Therapies.
The flyer for this event can be downloaded by clicking here. For more information and to RSVP please contact Rucha Shah at rshah.tag@gmail.comThis e-mail address is being protected from spambots. You need JavaScript enabled to view it or Gargi Pahuja at gargitag@mac.comThis e-mail address is being protected from spambots. You need JavaScript enabled to view it . Please RSVP by November 7, 2010
http://cooleysanemia.net/index.php/tag/34-tag/updates/index.php?view=article&catid=1:latest-news&id=359:tag-schedules-informational-meeting-on-gene-therapy&tmpl=component&print=1&page=
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I wish I could afford to fly down to NYC for this.
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Praying that all goes well and these trials start before the end of the year!
Sharmin
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I have heard some very troubling news about the current status of this research. I cannot say much, but corporate greed has become a real factor in this process and threatens the very work that Sadelain has been involved with over the past twenty years.
I ask all to pray that this situation can be resolved and that this work is allowed to proceed as originally planned without unreasonable financial demands from Sloan Kettering. It would behoove SK to put patients above dollar signs.
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Yeah this is very troubling news , but wht about The results of clinical trials , bcz October is over .....?
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Suffice it to say, the trial is not about to start thanks to the greed. While everyone got excited about the weak results of another trial, I was apparently one of the few who realized what this would mean to funding for other more promising trials. SK is in it for the money and not for the patients. The wonders of a for profit health care system never cease (sarcasm). I wish I could say more, but that is it for now.
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OMG Andy, what does this mean? This is devastating. Does this just mean a delay or does it put a stop to the trials indefinitely?
Sharmin
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hey guys
have u guys cheked the stem cell programe being run by the childrenz hospital in boston?....ive got alot of doctors telling me to give it 6 months and then go for it...can any budy guide me...or say smthin about it if they know more?
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I believe what they're doing at Boston is stem cell transplant, very similar to bone marrow transplants, so a matching donor is required. If a donor can be found, it can offer a cure.
As far as gene therapy, I can't see how this would not slow development down. Unfortunately, there is money to be made, so forces have come into play that previously were not a concern. I have long promoted Dr Sadelain as the one person most likely to succeed with the gene therapy process and apparently others now realize this and would prefer he worked for them. I do not know how this will work out but for patients I see longer delays and higher costs once this is perfected.
I wish more people working on behalf of thals actually had thals first in their minds before money, but that is me being naive yet again. Selflessness is taught by many religions and belief systems but you will find very few people engaged in selfless acts. For myself, I have to find a way to stay committed in spite of organizations and researchers who I do not feel place patients first. This situation has left me very discouraged. When it comes to a cure, should not we allow the best to proceed or should it come down to which group does a better job raising money? We could say this same thing about the current dormant status of starch desferal. It's not right. Patients should always come first but as long as the system is so profit oriented, expect little change.
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yes its partly what they do.......buh for people like me..who dun have a match they coreect the mutated gene ..its more like a combination of stem cel and gene therapy
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Nangial,
I wish you luck, I hope that the treatment is successful and curative.
Andy,
Thank you for taking a stand for us. I am so devastated I don't have words.
Sharmin
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This means that clinical trials with the first patient Thalassemia not yet started and there is no hope to begin clinical trials at the end of This year .
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I think we won't know until Dr Sadelain speaks on November 14.
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Let's all just pray for everything to turn out in the favor of thalassemia patients - we have all waited far too long for anyone to pull the plug on all of our hopes.
Thanks again Andy, no one has taken a stand for us the way that you have. You continually put yourself in difficult situations to advocate for us. You can count on my support.
Sharmin
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Andy Thank you for your cooperation with Thalassemia I pray to God to succeed Gene Therapy.
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thank u so mch andy tht u r always there wth thalasaemia fighting for people like us.............god has given u immense strength and wisdom.............thanx so mch would like to hear soon about what the doc says finally on gene therapy
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Yeah andy u r our reall hero , and im very thankfull for ur cooperation with us and thank u ones agine ........ :wub
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Thank you all for your support. It really means a lot to me. I don't like having to challenge research but...sometimes things don't add up.
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from Pat:
Dear family members,
Some good news that I wanted to share with you. Continue to send warm wishes and prayers. We need them more than ever... I love you all.
pat g
Errant Gene Therapeutics Awarded $245,000 Under U.S. Qualified Therapeutic Discovery Project Program
Chicago, IL. - November 2, 2010
Errant Gene Therapeutics, LLC (EGT), a biopharmaceutical company headquartered in Chicago , Illinois , announced today that it has been awarded a $245,000 grant under the Qualifying Therapeutic Discovery Project (QTDP) program.
The QTDP, created by U.S. Congress as part of the Patient Protection and Affordable Care Act of 2010, is designed to promote medical research that could improve health and save lives.
The QTDP was intended specifically to provide incentive to smaller companies who are focusing on innovative therapeutic discoveries that show potential to produce new therapies that address areas of unmet medical need, and reduce long-term health care costs. The grant covers up to 50% of the cost of qualifying biomedical research and expenses for the tax years of 2009 and 2010, and is only available to firms with no more than 250 employees. In addition, awards took into consideration research that demonstrates the greatest potential to create and sustain high-quality, high-paying U.S. jobs and to advance U.S. competitiveness in life, biological and medical sciences.
"We are extremely pleased that our Thalagen™ project has been reviewed and assessed positively by the federal government, resulting in a grant at the maximum level available under the QTDP grant program" said Pat Girondi, EGT’s Chairman and Chief Executive Officer. "These funds and other sources continue to help support EGT’s ongoing research and development programs."
EGT said it will use the grant to develop Thalagen, a groundbreaking curative treatment for the deadly genetic blood disorder β-Thalassemia (also called Cooley’s Anemia). The company said it plans to launch the Phase 1 human clinical trials of Thalagen in Q1 2011.
About Errant Gene Therapeutics, LLC
Errant Gene Therapeutics™ (EGT) is a privately held biopharmaceutical company established in 2003. The company's mission is the development of treatments for life-threatening diseases with a special focus on rare diseases, commonly referred to as “orphan diseases.” EGT is developing its portfolio of products to correct the erroneous gene expression associated with many of these diseases. EGT therapies are currently being developed for hemoglobinopathies and selected cancers.
For more information about Errant Gene Therapeutics and its programs, visit www.errantgene.com or call (312) 441-1800 extension 11.
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Waiting to hear more details from Pat on this, but I hope it can help keep Dr Sadelain and Sloan Kettering on board.
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This is amazing new - very encouraging. I hope that more good news will follow. Praying.
Sharmin
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Delightful news for patients with thalassemia all this because everyone is praying and standing with the patients and I thank all who contributed to help patients with thalassemia.
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this is indeed a delightful news giving us new hope and reason to fight..........was so happy just by knowing about this and m waiting for a day when i would really hera the news of someone amongst our children getting cured by it
:hugfriend..............
Thank u for showing us the light...or a ray of hope other than the bone marrow
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Time for an analogy.
There was a company that wanted to buy a race horse. They looked at two competing stables and even though the one stable offered a horse with the best possible breeding and training, this company chose a horse from another stable, even though there was some uncertainty about the horse's breeding and its long term viability as a race horse. They trained the horse and with a proper dose of drugs, this horse was able to win the Derby, as the other horse came in a strong second. However, after the race, the company looked at the horse and realized that this horse could never win another race, because it had never been a strong enough horse in the first place and had no long term viability. The company now looked at the horse that placed second with some degree of envy. This horse was in it for the long haul and would have many victories yet to come. So, realizing their own horse had won the only race it was going to win, the company went to the owner of the other horse and offered some big bucks if they would sell both horse and trainer to their company. The trainer, however, has been very happy working where he was and did not want to work for another and also, since the trainer had raised this horse since birth, he felt it was his baby and did not want someone else to come in and take over and tell him how he should train the horse. There also remained a good deal of distrust by this trainer for the trainer of the other horse, and he was very wary of this other company. But he was under contract to his stable and he would be obliged to do what they ruled. Meanwhile, as a result of the back and forth going on, the horse missed the next race, the Preakness Stakes. Now we have to wonder if this horse can possibly be ready for its next scheduled race at Belmont, as the owner and trainer can't agree on whether they should even race there, as the owner is now starry eyed over promised monies from the other company.
Folks, that is where we are right now with gene therapy. Stay tuned and see where this trainer and his horse end up and how much this delay will cost in the long term.
Story © A. Battaglia 2010
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let's all pray together for gene therapy's success and availabilty in the very very near future!!
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Pat Girondi and Andy Battaglia,
I am praying with all of my heart and soul for gene therapy to cure our children and our friends in the very near future.
When this happens - I will have both of you to thank for your ongoing efforts and your commitment to us.
Andy, thank you for your dedication and your courage to support and to be a voice for us. We don't always understand what is going on - but you do and you have the courage and heart to ask the questions on our behalf.
Pat, you are in the same situation that we are in - but you have pushed forward and made the changes that will help not only your own son - but all of us.
You two are heros for us,
Thank you from the bottom of my heart,
Love Sharmin
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i agree wth sharmin.......................we gonna pray about gene therapy daily in our prayers ................cos god does answer u
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Thank you all for your support. I do myself no good in terms of my relations with thal organizations and some medical people by being so outspoken, but I am not one to be afraid to speak the truth, and when I am, I will know it is time to sit down and shut up.
Pat is a regular guy, very much like myself. Unassuming and not pretentious. I think these are good qualities but when it comes to negotiating with investment companies, this hasn't helped Pat. These people are used to meeting with other "suits" and a man like Pat may not meet their preconceived notions of what the head of a company should be. I think this has been a big factor on which group got financing for gene therapy, but now that company has had second thoughts, because any fool can see that if you want to succeed at perfecting this direction in gene therapy for thalassemia and do it so it is safe and effective, Michael Sadelain is going to have to be involved. (I would also like to make clear that I am not commenting on other directions in gene therapy, like what is being done by Dr Persons). So, right now when we should be seeing patients start on this trial, we are instead delayed yet again because monetary pressures have been put on our good doctor's employers, who have upped the per patient price and made unreasonable demands for money in advance. Meanwhile, another player has come into the picture and this could provide a much lower cost solution, but will the good doctor be allowed to participate by his employer? Hopefully, all will be made clear at this November 14 meeting.
Now, for my opinion on what should be done. Dr Sadelain's group has worked through Sloan Kettering for many years. It only makes sense to continue and do the trials there. However, SK is completely missing the big picture here as they try to latch on to some short term money. What would I do if I was in charge at SK? I feel like saying "duh" because I cannot fathom how this has slipped the grasp of the decision makers at SK.
Gene therapy is going to be huge and not just in terms of curing hemoglobinopathies. Once this process is developed and is viable, we will see it transferred to countless other genetic illnesses. This very fact alone, should be enough to convince SK to not only cooperate with the trials but to offer their services at no charge whatsoever. This short term investment is going to pay dividends thousands of times higher than any initial development. SK, how do you not see this? Do you wish to be left behind? Or do you want to be the leader in the next generation of curative medicine? SK, the ball is in your end of the court. Are you going to play or are you going to take your ball and go home?
I won't even address the moral issues of leaving patients hanging and the inevitable delay towards a cure that this is causing, because it has become quite evident that this is not a consideration by the decision makers. However, the eventual monetary bonanza that gene therapy will be should be plenty to convince the bean counters.
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I am praying that this meeting goes well. If this meeting goes our way, the way that all thalassemia patients and their families need it to go, then I believe that we will all have a much brighter future.
Praying,
Sharmin
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I expect a commitment from Dr Sadelain to continue working with Thalagen to perfect this process. We will know more after tomorrow, but I would be very surprised if this is not what we hear. NIH may become involved as a result.
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Today's the big day... :pray :pray :pray :pray :pray :pray :pray :pray :pray :pray
Please come through for us Dr. Sadelain....
Sharmin
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Yeah 2day is big for thal and I am praying with all of my heart and soul for gene therapy .
I know god will help us in this .
We are with Dr. Sadelain and andy we want from Dr. Sadelain 2 be with us until last momant like andy .