By Paul M. Peloso, MD, MSc
Many people realize they have gout when they wake in the night with excruciating pain and swelling in the knee or the joint at the base of the big toe, as I first did. Treatment is often focused on just relieving this debilitating acute flare pain.
But even when the discomfort subsides, gout is not gone. The condition is a form of inflammatory arthritis caused by a buildup of uric acid throughout the body, and urate crystals can accumulate not just in joints of the arms and legs but in the major blood vessels, kidneys and tissues of the spine. The condition affects an estimated 9.2 million people in the United States; a fraction of those people have uncontrolled gout that has both ongoing signs and symptoms of joint troubles as well as persistently elevated uric acid levels, even while taking urate-lowering medications.
As a company dedicated to helping people with rare and rheumatic diseases, Horizon Therapeutics is committed to learning more about uncontrolled gout through a broad research program. Our R&D team works with academic researchers, undertakes clinical studies and hunts for new treatment options using innovative experimental and computational methods. Our hope is that these efforts will ultimately improve treatment for uncontrolled gout.
In recent years, our academic partners have made advances in understanding gout as a chronic disease that progresses even when it is not visibly causing arthritis inflammation and pain. As uric acid crystals aggregate into hardened visible lumps under the skin, known as tophi, permanent damage can occur to the joints, bones and surrounding tissue. There is a compelling association of gout with other serious medical conditions such as high blood pressure, diabetes, cardiac disorders and chronic kidney diseases, suggesting that persistently elevated uric acid levels have negative consequences beyond just the joints.
Available therapies can address these disordered metabolic processes, but not all patients see benefits. To better understand why some people do not improve on today’s therapies, Horizon is conducting clinical research to identify factors that might boost the effectiveness of existing treatments.
And we are searching for new treatments for uncontrolled gout with HemoShear Therapeutics, a company that studies human cell models in physiological systems that closely resemble living organs. This capability is valuable in gout because there are very limited disease models in non-human species. With the exception of our closest primate relatives, other mammals do not share the metabolic processes that initiate uric acid buildup. HemoShear also uses state-of-art computational techniques to turn data from these models into predictions of the most promising targets for new therapies.
Ultimately, our goal is to reduce the pain, disability and potential complications of gout, especially uncontrolled gout, for as many people as we can. Though often overlooked, this disease affects millions of people and may lead to potentially serious complications. By expanding our understanding of gout, Horizon plays an integral role in the community of researchers, physicians and patients who are uncovering new aspects of this well-known but incompletely understood disease.
Paul Peloso, MD, MSc, is vice president and therapeutic area head of rheumatology at Horizon Therapeutics. He has been diagnosed with gout.