A magazine for friends of the Warren Alpert Medical School of Brown University.

Hope for Rare Diseases

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It is often said that philanthropy is personal.

That was certainly the case for Sandra “Sandi” Nusinoff Lehrman ’69 MD’76 and Stephen A. Lehrman ’73 when they established the Lehrman Family Research Fund. With two grandchildren, Chase (who passed away in 2014) and Courtney (age 8), afflicted with CLN2 Batten disease (a debilitating neurological disorder), the Lehrmans are on a mission to support translational research to discover new treatments. It is a mission they share with Brown.

“When the children were diagnosed in 2013, there were no therapies available,” says Sandi Lehrman, who retired as global director for scientific affairs for antivirals at Merck Research Labs. Ever since then, she and her husband Steve, an engineer, have been committed to learning about the disease, investing in a variety of researchers, and facilitating approaches to treatments and a cure.

As a member of Brown’s Advisory Council on Biology and Medicine, Sandi recalls hearing Eric Morrow, MD, PhD, Mencoff Family Associate Professor of Biology, present his work on autism. “I was interested in the science and thought it would have applicability to a variety of other neurologic diseases with genetic variations, including rare diseases like Batten,” she says.

Morrow is the founding director of Brown’s recently established Center for Translational Neuroscience, where researchers study the development of brain disease. By supporting Dr. Morrow, the Lehrmans have filled in some important gaps in the small community doing research into rare brain diseases. “Before Dr. Morrow,” says Steve, “we didn’t have people working in gene editing and CRISPR as it relates to Batten disease—and now we do.”

A significant challenge faced by the Batten disease research community is the dearth of funding available. The Lehrmans are committed to providing seed money for researchers like Morrow who have a good idea, want to develop it, publish on it, and eventually go after NIH funding and collaborate with drug companies.

“We are looking for the best researchers in the U.S. and in the world,” says Steve, “and we are trying to be a bridge for people like Dr. Morrow because we want him to stay in this field and bring other Brown researchers into it as well.”

Open science is another important aspect of their gift, and they strongly encourage all of the researchers they support to work together and share their research. For example, they expect Morrow’s work and the stem cell lines he developed to be widely used by the CLN2 Batten disease research community.

“Our philanthropy is very much gauged toward Batten disease and our grandchildren, but lots of people can do the same thing by investing in this kind of work,” Sandi says. “Many patients are waiting for the innovative therapies and potential cures that can be enabled by research [at Brown]. A focus on translational research will hopefully narrow the gap, improve health, and save lives.”

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