Study finds new medication could prevent deadly swelling of the throat

USF Health professor Dr. Richard Lockey helping develop rare blood disorder drug

Asphyxiation is a frightening experience, not just for the sufferer, but also for those who’ve witnessed someone’s inability to breathe.

University of South Florida professor Dr. Richard Lockey is working to prevent some people from ever facing that life-threatening attack.

He’s involved in a study recently published in the New England Journal of Medicine testing a first-of-its kind medication for those with hereditary angioedema and a C1 inhibitor deficiency. The rare blood disorder causes spontaneous swelling in various parts of the body, which could be deadly if it occurs in areas such as the tongue or larynx.

Richard Lockey, MD

Lanadelumab is a monoclonal antibody administered by injection every two weeks, helping patients avoid asphyxiation and costly trips to the emergency room, allowing them to live a full life. Previously, up to 20 percent died before the age of 20. Existing long-term preventive treatments can cause serious side effects for a significant percentage of patients.

Thirty-seven patients from across the country participated in the multisite, double-blind, placebo-controlled clinical trial (Phase 1).  Dr. Lockey says in this early study lanadelumab was almost 100 percent effective, with minimal side effects. The drug works by inhibiting the enzyme kallikrein, blocking a cascade of molecular processes leading to angioedema.

“If this disease is in your family and you inherit the gene, we can give you this monoclonal antibody every two weeks to prevent attacks (of spontaneous swelling) from occurring,” said Dr. Lockey, director of the Division of Allergy and Immunology in the USF Health Morsani College of Medicine Department of Internal Medicine.  “It can enable people to live a normal life.”

Once the study is complete, researchers hope to win FDA approval, stocking pharmacy shelves in the next couple years.

About 10,000 people are diagnosed with hereditary angioedema.

– Story by Tina Meketa, USF Communications