in sickle cell disease, some red blood cells form a crescent shape.
NHLBI
For the first time, a drug developed in part by a new controversy National Institutes of Health (NIH) Center to accelerate the development of drugs was picked up by a major pharmaceutical company. Baxter International acquired the biotech company developing Aes-103, a small molecule for the treatment of sickle cell disease.
Baxter's acquisition of this drug is a victory for 3 years NIH National Center for the Advancement of Translational Sciences (NCATS). One of its programs is Therapeutics for Rare and Neglected Diseases (TRND), launched in 09 to help the NIH and university scientists to develop treatments for disorders that the pharmaceutical industry has ignored because the target population is too small or poor. The program of $ 23 million per year does not assign subsidies, but rather provides support such as chemists tweak compounds to work better and regulatory experts that help navigate the approval of clinical trials.
"This is the first drug to get him out of this process," said Director NCATS Chris Austin science Insider. "So it's really a nice validation" of the model center.
In sickle cell disease, which affects 100,000 Americans mostly of African origin, a defect in the hemoglobin causes red blood cells to form a sickle shape that blocks small blood vessels. This can cause pain and complications such as severe beatings, and patients often do not survive past middle age. The disease can be treated with a drug called hydroxyurea, not all patients respond and many suffer side effects
Aes-103 binds to hemoglobin and prevents red blood cells from sickling. in clinical trials, it reduced pain in patients. AesRx, a biotechnology company in Newton, Massachusetts, and has worked with NCATS NIH National Heart, Lung, and Blood Institute to move the drug through Phase II trials. NIH has also provided more than $ 5 million for clinical trials, among other funding sources.
But it is the role of NCAT which prompted the NIH to boast the Baxter acquisition today. "This is a wonderful example of why NCATS was created," said the director Francis Collins NIH press release.
The sickle project was particularly attractive, said Austin NCATS because Aes-103 has the unusual chemistry and a new mechanism that made too risky for most commercial enterprises. "This project was dead in the water" until the NIH has picked up, he said. But with the help of experts from TRND, "in a year, we were in clinical trials." It could take years of investment, tweaking, and testing for the drug to reach the market.
NCATS drew skepticism, with critics arguing that he could not improve the industry's efforts and would siphon off funds fundamental research-although rare diseases is an area where many agreed the agency could play an important role. TRND has completed four projects; two focusing on drugs against schistosomiasis and fragile X syndrome were dropped, and two others "graduated early" to TRND, Austin said, and continued independently by companies.
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