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HemaQuest Announces Initiation of Phase I Clinical Trial of HQK-1001

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Sharmin:
Andy,

This is so terrific!!!  Is this drug something that is most likely to help thalassemia intermedias only or would it be helpful to thal majors as well?  I ask because during our recent visit the doc told us that my son is definitely a beta zero and unlikely to benefit from drugs such as hydroxy urea. 

If this drug would come in time we can stop worrying about antibodies etc..... and so many people world wide would benefit so much!!!

Thank you for sharing this news Andy,

Sharmin

Andy Battaglia:
Sharmin,

I would not discount the possibility that fetal hemoglobin inducers may have benefit for beta zero majors. In her article from 2005, Dr Perrine talks about the potential for using a combination of these drugs to raise the hemoglobin level even more. (http://asheducationbook.hematologylibrary.org/cgi/reprint/2005/1/38.pdf) I think we will have to wait and see what a longer trial brings in terms of overall improvement in hemoglobin. Dr Vichinsky has spoken to you about the possibility of using some of these drugs. Obviously the best way to avoid blood reactions, including antibody reactions, is to not take blood, so this does have to be examined as a possibility for little A, if it becomes possible. Part of the ongoing investigation with these drugs involves what combination of inducers works well, along with what else can be done to help create more red blood cells and extend their lives. I think antioxidants will play a role in this combination.

One ironic point, is that it has been found in intermedias using Hb inducers, is that they often need iron supplementation to fully take advantage of the potential of these drugs, even though they have high iron stores. The body does not easily use stored iron and prefers to extract it from the gut, so it may be necessary to provide the iron to promote the formation of new red blood cells.

Zaini:
This can be the answer to our prayers.

Thanks for the info Andy.

Zaini.

Dori:
I'm glad for you guys!!!

 :thumbs

Dori:
My apologies, but I am not in the correct shape to understand this perfectly. Do you think it can only help patients with pyruvate kinase deficiency? To respons at my previous post: I don't think so.... (Does I use the correct English grammar?)

Dore

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