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Arnold and Arlene Goldstein

Imagining a World without Amyloidosis

The Arlene & Arnold Goldstein Family Foundation has given $1.5 million to support research in The Scripps Research Institute’s Department of Molecular and Experimental Medicine.

The funds have made possible the hiring and support of an investigator designated the Arlene and Arnold Goldstein Assistant Professor of Molecular and Experimental Medicine who is contributing to the efforts to develop the next generation of drugs to treat transthyretin amyloidosis. Transthyretin amyloidosis can lead to a number of medical problems, including decreased heart function.

“We are very enthusiastic to partner with the foundation to develop the next generation of drugs to combat amyloidosis,” said Jeffery Kelly, chair of the Department of Molecular and Experimental Medicine, who is the Lita Annenberg Hazen Professor of Chemistry and a member of the Skaggs Institute for Chemical Biology at Scripps Research. “This category of human maladies represents a large unmet medical need and we appreciate the foundation's commitment to provide us with the resources to develop new therapeutic strategies for these degenerative disorders.”

Amyloidosis includes a number of inherited and acquired conditions caused by the misfolding of proteins in the body. In these diseases, microscopic fibrils made up of hundreds of misfolded proteins cluster together and deposit in organs, interfering with their normal function.

The Kelly lab has contributed to efforts to understand the fundamental mechanisms of these diseases. In addition, the lab’s work has included the development of a compound that holds promise for treating transthyretin amyloidosis, which results from the misfolding of one of 100 mutants of the protein transthyretin. This compound is currently in a Phase II/III clinical trial by FoldRx Pharmaceuticals, a company founded by Kelly and colleagues.

The gift from the Goldstein Family Foundation supports work on the development of a distinct but complementary therapeutic aimed at treating senile systemic amyloidosis, a type of transthyretin amyloidosis that affects a quarter of the population 85 years of age or older. This work is a collaboration between the Kelly lab and the laboratory of R. Luke Wiseman, who holds the newly created Arlene and Arnold Goldstein Assistant Professorship.

“I am honored to accept the Arlene and Arnold Goldstein Assistant Professorship in Molecular and Experimental Medicine,” said Wiseman. “I look forward to developing innovative strategies to treat protein misfolding disorders.” 

Goldstein, who passed away in 2014, grew up in the Bronx as the son of the founder of Atlantic Hardware & Supply Corp. A graduate of NYU’s School of Commerce Accounts and Finance, Goldstein co-founded Samson Management, one of New York City’s largest owner management firms.

“I am a firm believer in ‘philanthropic capitalism,’” Goldstein said when he made the gift. “If one benefits from the fruits of capitalism, one should also give back to the community at large.”

A patient affected by amyloidosis, Goldstein was a participant in the ongoing FoldRx clinical trial. Support of medical research is not new to his foundation, however. In addition to a number of Jewish/Israeli causes, the Arlene and Arnold Goldstein Family Foundation has provided significant support to NYU Medical School, New York-Presbyterian Hospital, Columbia University Medical Center, Memorial Sloan-Kettering, and a number of other medical and research organizations.

“My reason for authorizing grants for medical research is that I feel this is an area that can help large segments of the world population,” Goldstein explained at the time of the gift.

Kelly is optimistic that this will be the case for the foundation's gift. “We are confident that we can discover three new classes of drug candidates,” he said, “and make a significant impact on protein folding disease.”

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“My reason for authorizing grants for medical research is that I feel this is an area that can help large segments of the world population,” said Arnold Goldstein, who has supported The Scripps Research Institute through the Arlene and Arnold Goldstein Family Foundation.