The U.S. National Institute of Allergy and Infectious Disease (NIAID) has awarded $225,000 to Karyopharm Therapeutics to advance the development of its SINE nuclear transport compound, KPT-350, to treat neurological, inflammatory and autoimmune diseases.
The grant will be used in preclinical trials of the compound as a treatment for systemic lupus erythematosus (SLE), which will be conducted in partnership with Dr. Jenifer Anolik, MD, PhD, an associate professor of Medicine at the University of Rochester, in New York.
“XPO1 inhibition is known to generate potent, multifaceted inhibition of the inflammatory mediator NF-κB. KPT-350 inhibits XPO1 leading to increases in the levels of several natural, cellular inhibitors of NF-κB, thus blocking its inflammatory activity. This results in an increase in anti-inflammatory and neuroprotective responses leading to reduced autoimmune disease activity,” Sharon Shacham, PhD, MBA, president and chief scientific officer of Karyopharm, said in a recent press release. “With this funding support from NIAID, we are eager to advance the development of KPT-350 with Dr. Anolik to further evaluate its potential activity in chronic and painful inflammatory and autoimmune diseases such as lupus.”
KPT-350 is designed to penetrate the blood brain barrier, or BBB, to a greater degree than other SINE compounds. Preclinical data generated largely by external collaborators has demonstrated the efficacy of orally-administered KPT-350 and related SINE compounds in animal models of several diseases, including SLE.
Lupus, or systemic lupus erythematosus (SLE), is difficult to diagnose and a challenge to treat, with a range of symptoms and no known cause or cure. But treatment options exist, including therapies being studied at Yale School of Medicine in New Haven, Connecticut.
YaleNews’s Ziba Kashef recently interviewed Dr. Lenore Buckley — an adult and pediatric rheumatologist and professor of medicine — whose research interests include lupus and inflammatory arthritis, glucocorticoid osteoporosis, and the transition of teenagers and young adults with rheumatic diseases to adult care. Dr. Buckley is current director of the Yale Medicine, Section of Rheumatology, Transition of Care Programs for young adults with rheumatic diseases, which recently launched an innovative clinical program for patients with lupus.