Thalassemia Patients and Friends
Discussion Forums => Working Towards a Cure => Topic started by: Bostonian_04 on February 23, 2017, 07:25:42 PM
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Read about Bellicum Pharmaceuticals...it seems they have an ongoing clinical trial using haploidentical stem cell transplant (HSCT) where they add their propriety donor cells that helps with immune reconstitution, infection control and has overall better outcomes. Anyone on this forum know more? There seems to have more and more options opening up for the Thal patients, which is very positive and encouraging. More reason to take care of Iron over load and general health for a bright future :)
http://ir.bellicum.com/phoenix.zhtml?c=253830&p=irol-newsArticle&ID=2154113
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Bostonian,
Thank you for sharing, this is wonderful. If this can be done without chemo - I would choose this treatment over gene therapy. I will try to connect with the researchers for more information.
Sharmin
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I believe someone else is also working on something similar. Sharmin, BMT would make a lot of sense if this is available, as BMT is proven over the long term and we still won't know about gene therapy in the long term for some time still.
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I think this has been misunderstood. It doesn't say no GVHD just no mortality associated with GVHD.
My son had a haplo transplant recently and one of the reasons his doctor recommended haplo is lower incidence of GVHD.
They aren't really sure why yet but think there is something like an HLA typing they dont know of or test for yet but is very likely to be similar in relatives even if mismatched.
Haploidentical transplants are always from close relatives. All parents, siblings & children are at least halfmatched greatly increasing the possibility of transplants. Only 50% match is required.
Zahra
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I should also mention another good thing about haploidentical transplants is they can be done using reduced intensity conditioning. This means less side effects and less chance of mortality if graft fails. This is because if donor cells don't engraft patients own bone marrow reconstitutes. So though the transplant failed the patient is safe from mortality. This is important in benign hemoglobinopathies where it is now possible to survive ( though with issues ) without transplant.
Zahra
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It's amazing how far they have come with haplo transplants. In 2006 at Dubai conference, some people laughed at Dr Sodani after he presented his early results of mother to child BMT. I think Sodani's work is creating a revolution in BMT.
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It is possible.
Zahra
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I understand that Dr Lucatelli in Italy is 16/16 using this technique for haplo transplant pants as well.