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FDA grants fast track status to luspatercept for beta-thalassemia

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Bostonian_04:
There is another option opening up for Thal patients.....don't know much details but heard that it increases HgB production in thal patients and reduces transfusion frequency and iron overload.


http://www.healio.com/hematology-oncology/hematology/news/online/%7B5eda39b5-84f2-44fd-86bd-221c11fedfcc%7D/fda-grants-fast-track-status-to-luspatercept-for-beta-thalassemia

Manal:
I heard about this too from a friend but have no details :dunno

Andy Battaglia:
We have a previous thread about this drug at http://www.thalassemiapatientsandfriends.com/index.php/topic,5303.0/highlight,luspatercept.html

Of all the current approaches in development for thal, this is the one that holds the most hope for the most patients. The trial results so far have been amazing. Dr Piga is on board and I can't see him behind anything that doesn't have great promise. As glamorous as gene therapy is, the cost will be prohibitive for many people, whereas a mass produced drug will have a more affordable cost. And, if this works as well as it has so far, boosting Hb while chelating iron, we can expect that chelation drugs will eventually not be needed.

Sharmin:
Bostonian,

Thank you for sharing!  What great news!  This drug definitely has the potential to keep patients healthy while they wait for gene therapy to become main stream! 

I understand that this drug is given sub Q every 3 weeks - I wonder if it can be given at home like desferal or if it is a quick injection every 3 weeks.  We plan to visit Oakland this summer - hopefully more information will be available about this drug.

Andy Battaglia:
Sharmin,

I believe they are still recruiting for the trials. It might be worth inquiring about it.

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