A Patient Giving Back
Philanthropists Arnold and Arlene Goldstein
"We are very enthusiastic to partner with Arlene and Arnold Goldstein to develop the next generation of drugs to combat amyloidosis," said Jeffery Kelly, chair of the Department of Molecular and Experimental Medicine and Lita Annenberg Hazen Professor of Chemistry. "This category of human maladies represents a large unmet medical need and we appreciate the Goldsteins' commitment to provide us with the resources to develop new therapeutic strategies for these degenerative disorders."
Arnold Goldstein came to understand exactly how unmet that medical need was when he was diagnosed with amyloidosis - a category of disease that 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.
Kelly's lab has contributed to efforts to understand the fundamental mechanisms of these diseases. The lab's work has included the development of a compound that holds great promise for treating transthyretin amyloidosis, which results from the misfolding of one of 100 mutants of the protein transthyretin. The compound is currently in a Phase II/III clinical trial by FoldRX Pharmaceuticals - a company founded by Kelly and colleagues - which is how Goldstein first met Kelly.
"My cardiologist recommended that I enroll in the test program run by FoldRX. I've been in the program for more than six months and there have been marked improvements. I met Professor Kelly on several occasions. He was good enough to visit me at my office and told me that he is doing further studies regarding amyloidosis," explains Goldstein.
Goldstein, co-founder of Samson Management, one of New York City's largest owner management firms, and his wife decided to make a gift to further the Kelly lab's work. In their honor, Scripps Research has established the Arlene and Arnold Goldstein Assistant Professorship of Molecular and Experimental Medicine with the goal of contributing to the efforts to develop the next generation of drugs to treat transthyretin amyloidosis. This work will be a collaboration between the Kelly lab and the laboratory of R. Luke Wiseman, who will join Scripps Research this summer and will hold the newly created 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."
Kelly seconds Wiseman's enthusiasm, adding, "We are confident that we can discover three new classes of drug candidates and make a significant impact on protein folding disease."
Goldstein's story - a patient benefiting from the fruits of medical science and then giving back - is what has brought modern medicine this far. You can join Arlene and Arnold in helping to carry it into the future.