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Acceleron "Luspatercept" drug increases RBC count blocking a type of protein

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Andy Battaglia:
There are so many different approaches to treating thalassemia that are under investigation and most show promise, and it is likely that various treatments could be used together to free patients from transfusion.

But there is constantly one thing working against them and that is lack of funding. Most major drug companies do little to no research of their own on orphan diseases like thalassemia, so that source of funding is usually absent. Typically, it is small time researchers with little funding pursuing these ventures, many falling by the wayside from lack of funding and not because there is a lack of science. If somehow they can get enough funding to develop a drug and run trials that show positive results, then some Big Pharma giant will buy the drug with the hopes of making huge profits, as we have seen in the past year with Novartis paying $30 million for a new chelator. Eventually, they stand to make billions if the drug is a success. Something is wrong with this system. I've talked to researchers with fantastic ideas who have trouble raising $30,000 to continue their research. The pharmaceutical business is the most profitable industry, yet the money does not necessarily flow into research. More money is actually spent on advertising. As long as we have a pharmaceutical industry, and much of the medical industry with profit as its only true motive, progress will not be at the pace we would like to see.

CrazyPharm:
The research on this drug is quite promising and exciting. More information regarding phase 2 trial results in Thalassemia patients was released and was quite promising. Keep on the lookout for this drug in the future :D

catchR:
Fresh update on Luspatercept:
http://www.bizjournals.com/boston/blog/bioflash/2015/05/acceleron-gains-and-bluebird-falls-in-blood.html


More on latest study from Acceleron - Luspatervept: http://investor.acceleronpharma.com/releasedetail.cfm?ReleaseID=910306

Parin:
Thank you for sharing the updats..keep us updating.

Parin:
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

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