Accelerating Therapeutic DiscoveryTM

News From The Alpha-1 Project

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Fellowship will advance therapeutic strategies for Alpha-1 Antitrypsin Deficiency

Citing “substantial progress” in the first year of a postdoctoral fellowship at the National Institutes of Health’s (NIH) National Center for Advancing Translational Sciences (NCATS), The Alpha-1 Project has announced it will extend funding for a third year.

The fellowship recipient is Michael Iannotti, PhD, who is working in the lab of James Inglese, PhD, chief of the Assay Development & Screening Technology Laboratory at NCATS.

“We are very pleased with the substantial progress that has been made by Mike under the tutelage of James Inglese and the guidance of the Steering Committee. In just 12 months, Mike has developed assays to measure the secretion and degradation of the misfolded protein that is the primary cause of Alpha-1 Antitrypsin Deficiency,” said Jean-Marc Quach, Executive Director of The Alpha-1 Project, the venture philanthropy arm of the Alpha-1 Foundation.

“This innovative partnership combines the passion and deep knowledge of Alpha-1 Antitrypsin Deficiency of the Alpha-1 Foundation/The Alpha-1 Project and the translational science expertise and resources of NCATS, to benefit patients living with this disease,” said Christopher Austin, MD, Director of NCATS.

Iannotti has been focusing on small molecule screens in cell-based and biochemical assays designed to curb the effects of the abnormal alpha-1 antitrypsin protein. “These assays… function as reporters to monitor the accumulation and/or secretion of alpha-1 antitrypsin from cells after treatment with experimental compounds, in an effort to identify novel clinical candidates to treat Alpha-1,” Iannotti said in an interview at the beginning of the fellowship.

“We’re thrilled that collaboration to cure Alpha-1 is working as we had envisioned,” added Quach.

Besides focusing on potential Alpha-1 treatments, a portion of Iannotti’s work under Inglese is training to lead translational research. “Mike is also receiving guidance from a Steering Committee of world-class lung and liver experts,” says Quach.

The Steering Committee members are Bibek Gooptu, Kings College, United Kingdom; Darrell Kotton, Boston University; Richard Sifers, Baylor College of Medicine; Jeffrey Teckman, Saint Louis University; and Adam Wanner, University of Miami.

Alpha-1 can lead to severe lung disease in adults and liver disease at any age. The disease occurs when the body’s blood supply lacks a protein called alpha-1 antitrypsin, or A1AT. The liver is the main producer of A1AT. The protein’s primary function is to protect the lungs from inflammation caused by infection and inhaled irritants such as tobacco smoke.

People with Alpha-1 have an abnormal type of A1AT, and the liver cannot release this protein at a normal rate. The abnormal A1AT builds up in the liver, which can cause liver disease. In addition, the low levels of A1AT in the blood leave the lungs without needed protection, which can lead to lung disease.

For more information about Alpha-1, visit alpha1.org. For more about NCATS, visit ncats.nih.gov.