Seattle Children's Research Institute and Kineta, a Seattle, Wash.-based privately held biotechnology company, have launched the Alliance for Children's Therapeutics (ACT), a first-of-its-kind pediatric research and funding collaboration designed to speed development of new medications for children and teens with lupus nephritis and other autoimmune diseases such as multiple sclerosis, type 1 diabetes and rheumatoid arthritis.
"Funding for pediatric research lags disproportionately behind research funding for adult diseases. Thirty percent of the U.S. population is under the age of 21, and yet only 6% of the entire NIH's budget is devoted to pediatric medicine and care," said Jim Hendricks, Ph.D., president of Seattle Children's Research Institute. "This gap results in limited development of new therapies for children, who now often have no other choice than to use adult-only tested medications."
There are significant barriers in developing pediatric therapies and the lack of pediatric medication use information poses risks to children and teens. The shortage of appropriate formulations may deny access and expose them to medications that were not designed for their growing bodies. The challenge of recruiting sufficient numbers of pediatric patients for clinical research also delays the development of better therapies for pediatric diseases and conditions.
In 2013, the FDA approved 27 new drugs; just seven new drugs were approved for pediatric use that same year. And of the 55,000 clinical trials conducted between 2005 and 2010, only nine percent were designed for children.
ACT will rely on a collaborative funding model that combines philanthropic gifts made to Seattle Children's Research Institute with equity investments made to Kineta.
"For too long pediatric studies have been the neglected component of clinical trials," said Charles Magness, Ph.D., Kineta president and CEO. "Kineta has a drug candidate that has completed two adult clinical trials and is well positioned for a clinical trial, which could make a real advancement for autoimmune diseases in children."
For their first collaborative project, researchers from Kineta and Seattle Children's Research Institute have begun conducting preclinical laboratory tests to advance Kineta's lead compound, called ShK-186, as a potential treatment for lupus nephritis, an autoimmune disease that causes kidney inflammation. While medications currerntly available for lupus nephritis reduce the disease's resulting inflammation, they also suppress the immune system, a side effect particularly concerning in children. Early preclinical research studies have indicated that ShK-186, which is derived from sea anemone venom, reduces inflammation while leaving the immune system intact.
Initial funding will help move ACT's lupus nephritis research into a clinical setting. It also will support work to adapt ShK-186 to become a viable treatment for other autoimmune diseases.