Discussion Forums > Working Towards a Cure
Potential cures
Sharmin:
My understanding is that we need to follow the work of the following to know what is happening in terms of a potential cure - or major lifestyle improvement for thalassemia.
Errant Gene - Thalagen
Genetix Pharmaceuticals - Current trials ongoing in Paris
Hemaquest - medication to induce hg F production
Are there any other leads that we may be interested in. I wish the best to these three companies and hope that their trials are successful and SOON!
Sharmin
mudit:
Is it LenrtiGlobin from Genetix pharma that is currently on trial in Paris?
Also, stemcyte (cord blood stem cell bank) had earlier collaborated with various hospitals to treat many thalassemic patients using unrelated umbilical cord blood transplantation therapy; information is available in their website. Not sure about their current status though.
Narendra:
--- Quote ---Genetix Pharmaceuticals - Current trials ongoing in Paris
--- End quote ---
Update on the above trial mentioned by sharmin....
I just read about the clinical trail of gene therapy on a thal patient and is so far doing good. I think there is a lot of positive direction, that we can see will lead to a cure to thalassemia
From:- http://www.cooleysanemia.com/bodies/body419.php
--- Quote ---July 3, 2008 -In June, CAF National Executive Director Gina Cioffi had the incredible experience of witnessing history in the making when she met Paul Louis Beauchesne, the first person to successfully undergo a gene therapy trial for thalassemia.
Paul Louis is part of an ongoing gene therapy clinical trial in France led by Philippe Leboulch and supported by Genetix. The trial is taking the gene that causes thalassemia and replacing it with a working gene, with the primary goals of proving that the process is safe and that the basic premise behind the idea is sound.
All has gone well for Paul Louis. He has gone for two 60-day periods with no transfusion, which is thus far substantially longer than before transplant. The therapeutic hemoglobin continues to be made and is circulating in his red blood cells with higher hemoglobin levels in his blood than before. He will continue to be monitored to see possible long-term benefit on his disease. The researchers will repeat this process again in the fall with another patient already recruited for the trial; one patient prior to Paul Louis received back-up cells as a cautionary measure and is thus unlikely to derive clinical benefit .
“This was truly an incredible experience,” Gina says. “I felt so inspired when I visited Paul Louis and had a chance to discuss his thoughts and feelings in deciding to enter the trial. It was truly a privilege to meet him and to report back to the community this success and progress. I think it is vital that our patients, their families and our funders know that their investment in maintaining the good health and support for research is paying off. Gene therapy trials are expensive and difficult to get launched and so we are truly counting on the continued good will of our donors to help accomplish a cure through gene therapy.”
For patients who would like to reach Paul Louis, you may email him your thoughts (in French) at smoky77@hotmail.fr . Babelfish.yahoo.com, a free online translation service will aid you in your correspondence
--- End quote ---
I hope Paul comes out with flying colors and paves a way for thousands to follow
kabir_love:
oh.. this is very very great... 60 days without bt. is very good. and i hope they had not hide any side effects from us. as these big company`s alwayz does to make there profit..
i pray for paul..
Sharmin:
This is so so so great! This is the first good news we have received regarding these trials! I am so glad that more than one company is working on gene therapy for thalassemia. I hope that the side effects will be much less than those with conventional thalassemia treatment - and that it will be less toxic and risky than bmt. It is difficult to know right now. What are your thoughts Andy?
I also hope for the best for Paul,
Sharmin
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