Discussion Forums > Working Towards a Cure

Phase 3 Luspatercept Trial

<< < (10/12) > >>

Andy Battaglia:
The goal is raising total Hb, so that's what gets reported. They may be following other values, as well, but in the context of trials, the goal is specific. I am curious if this would raise MCV levels. I imagine it would.

sofear:

--- Quote from: Andy on January 15, 2017, 12:24:22 AM ---I am curious if this would raise MCV levels. I imagine it would.
--- End quote ---

I feel like that probably won't be the case. I just had a look at some older power points of Luspatercept (like this one) and it seems that the increase in hemoglobin always happens with quite a large increase in the RBC count. Why is no one talking about the MCV that always seems to be low in thalassemia patients? We have multiple options to increase the RBC count, that is nothing new.

sofear:
I'm not sure whether it has yet been posted, but I recently stumbled upon a nice animation of the working mechanism of Luspatercept:

https://www.youtube.com/watch?v=oPfig2aOIYw

(I'm not sure how accurate it is, though, but it's pretty cool to watch.)

Manal:
I met Dr. Piga last November in Jordan ( pan middle east conference ) and he said that he expects Laucpatercept to be available in the market in 3 years and he added that in few months they will start the pediatric clinical trails.

Manal

sofear:
After I saw the animation of the mechanism of action of Luspatercept, I tried to understand it a bit better and went through a lot of videos and articles about basic biological topics but also thalassemia-specific ones.

Although I'm not able to judge the quality of the content, I later found a site with articles about thalassemias of which some were really interesting to read:

http://cursoenarm.net/UPTODATE/contents/search.htm?search=thalassemia&menu=0&submitType=Search

One part of an article (http://cursoenarm.net/UPTODATE/contents/mobipreview.htm?29/32/30208) had the following passage:


--- Quote ---Normal hemoglobin biosynthesis requires an intact structural gene and the structural and spatial integrity of silencers, enhancers, promoters, and LCR sequences. Despite considerable effort, we do not fully understand how the sequences and factors regulating globin gene expression interact with one another to ensure high levels of expression of the proper globins at the proper developmental stage and during the proper steps of erythroid differentiation.
--- End quote ---

This information has been last updated in 2011, so I'm not sure how up-to-date it still is, but the question that came to my mind was:

How are the processes of red blood cell differentiation and maturation, which Luspatercept interacts with, actually linked to the expression of the globin genes?

Navigation

[0] Message Index

[#] Next page

[*] Previous page