Meeting on gene therapy November 14 from 11:30 a.m. to 1:30 p.m

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=

I wish I could afford to fly down to NYC for this.

Praying that all goes well and these trials start before the end of the year!

Sharmin

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.

Yeah this is very troubling news , but wht about The results of clinical trials , bcz October is over .....?

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.

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

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?

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.

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

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

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 .

I think we won't know until Dr Sadelain speaks on November 14.

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