Three NIH Awards for CReM

$9 million will advance stem cell research

Darrell Kotton, director of the Center for Regenerative Medicine (CReM). Photo by Vernon Doucette

The Center for Regenerative Medicine (CReM) at Boston University School of Medicine (MED) and Boston Medical Center has received three awards from the National Institutes of Health (NIH) to advance its induced pluripotent stem cell (iPSC) research and education.

Created from a patient’s skin or blood cells, iPSCs have the potential to form any type of cell, providing a hypothetically inexhaustible source of cells for biomedical research and making possible personalized medicine.

Two of the NIH awards, totaling $5 million, will support a new National Heart, Lung, and Blood Institute initiative called the Progenitor Cell Translational Consortium, which will bring together institutions and researchers from across the country to develop stem cell–based treatments for heart, lung, and blood diseases. The NIH gave out seven such awards to institutions around the country; only CReM received more than one of the awards. CReM researchers will focus on how gene-editing and cell-based therapies can be used for treatment of lung diseases.

“We are honored to receive these awards, which will allow us to gain greater understanding about how we treat the most medically complex cases and improve our patients’ lives,” says Darrell Kotton, director of CReM and the David C. Seldin, MD, PhD, Professor of Medicine. Kotton will serve as co-principal investigator on the two grants along with investigators from Cincinnati Children’s Hospital, the University of Pennsylvania, and the University of California at San Francisco.

CReM and the BU Clinical & Translational Science Institute (CTSI) also received a $4.1 million NIH National Center for Advancing Translational Sciences award to form a national Clinical and Translational Science Awards network for iPSC banking, sharing, and training. This group will be led by Kotton and Andrew Wilson, a MED assistant professor of medicine, and will include researchers from the University of Pennsylvania, Harvard University, and the University of Chicago.

“The discovery of iPSCs provides an unprecedented opportunity for any scientist to derive an inexhaustible supply of patient-derived primary cells,” says David Center, the MED Gordon and Ruth Snider Professor of Pulmonary Medicine and CTSI director. “These cells, which contain each patient’s own genetic background, can now be applied for in vitro human disease modeling, drug screening of personalized therapeutics, drug screening for unknown side effects, and the development of future regenerative cell-based therapies. Coupled with another $2.1 million CTSI award for training individuals in iPSC technology, we have the opportunity to make an impact on patient’s lives for many years to come.”

The group aims to make patient-derived iPSCs, along with the tools and expertise for their genetic manipulation, available to researchers across the country, with the goal of understanding complex diseases and developing potential therapies.

To achieve this goal, researchers propose sharing more than 1,000 iPSC lines—already derived by the teams taking part in the consortium—nationwide to both basic and clinical researchers; developing and supporting a formalized education and training program for other researchers; and supporting the maintenance and sharing of gene-editing tools and gene-edited iPSC lines that will enable scientists to manipulate the human genome at will.

Each team will be led by researchers who, like Kotton and his colleagues at CReM, have championed what is known as open-source biology. The approach encourages researchers to freely share iPSC lines and their reprogramming reagents with others in the field.

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