In sickle cell disease, some red blood cells form a crescent shape.

NHLBI

In sickle cell disease, some red blood cells form a crescent shape.

NIH's translational center scores first drug acquisition by pharma

Jocelyn is a staff writer for Science magazine.

For the first time, a drug being developed in part by a controversial new National Institutes of Health (NIH) center aimed at speeding drug development has been picked up by a major pharmaceutical company. Baxter International has acquired the biotech company developing Aes-103, a small molecule for treating sickle cell disease.

Baxter’s acquisition of this drug is a victory for NIH’s 3-year-old National Center for Advancing Translational Sciences (NCATS). One of its programs is Therapeutics for Rare and Neglected Diseases (TRND), launched in 2009 with the aim of helping NIH and university scientists develop treatments for disorders that pharma has ignored because the target patient population is too small or poor. The $23-million-a-year program does not award grants, but instead provides support, such as chemists who tweak compounds to work better and regulatory experts who help navigate approval for clinical trials.

“This is the first drug to make it out of that process,” NCATS Director Chris Austin tells ScienceInsider. “So it’s a really nice validation” of the center’s model.

In sickle cell disease, which afflicts 100,000 Americans mostly of African descent, a defect in hemoglobin causes red blood cells to form a sickle shape that blocks small blood vessels. This can result in severe pain and complications such as strokes, and patients often do not survive past middle age. The disorder can be treated with a drug called hydroxyurea, but 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 has reduced pain in patients. AesRx, a biotech company in Newton, Massachusetts, worked with NCATS and NIH’s National Heart, Lung, and Blood Institute to move the drug through phase II trials. NIH also provided more than $5 million for clinical trials, among other funding.

But it is NCAT’s role that prompted NIH to tout the Baxter acquisition today. “This is a wonderful example of why NCATS was created,” said NIH Director Francis Collins in the press release.

The sickle cell project was particularly attractive, says Austin of NCATS, because Aes-103 has unusual chemistry and a novel mechanism that made it too risky for most commercial enterprises. “This project was dead in the water” until NIH picked it up, he says. But with help from TRND experts, “within a year we were in clinical trials.” It could still take years of investment, tweaking, and testing for the drug to reach market.

NCATS has drawn skepticism, with critics arguing that it couldn’t improve on industry’s efforts and would siphon funding from basic research—although rare diseases is one area where many agree the agency could play an important role. TRND has completed four other projects; two focusing on drugs for schistosomiasis and Fragile X syndrome were discontinued, and two others “graduated early” from TRND, Austin says, and are being pursued independently by companies.

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