Seattle Children's Research Institute and biotechnology company Kineta, Inc. today 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 like 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 six percent of the entire National Institutes of Health's budget is devoted to pediatric medicine and care," said Jim Hendricks, PhD, 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 U.S. Food and Drug Administration (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.
Translating Research into Better Medical Treatments for Children and Teens
Bringing together one of the U.S.'s top five pediatric research institutions with a leading biotechnology company, ACT aims to speed development of medicines for children and teens with autoimmune diseases.
Funding is needed to propel this pediatric research forward. ACT will rely on a unique and 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, PhD, Kineta president and chief executive officer. "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 already begun conducting pre-clinical 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 available today for lupus nephritis reduce the disease's resulting inflammation, they also suppress the immune system, a side effect that is particularly concerning in children. Early pre-clinical research studies have indicated that ShK-186, which is derived from sea anemone venom, reduces inflammation while leaving the immune system intact.
"There is a huge need for medications designed for children and teens," said 18-year-old Seattle Children's Hospital patient Kathia Vega Flores, who was diagnosed with lupus when she was 11. "A diagnosis of lupus can present daily challenges not only for the person with the disease, but the whole family. I am so encouraged to see ongoing research and possible new treatments for lupus and other autoimmune diseases that will help so many children in need of better medications and care options."
In addition to the previously mentioned autoimmune diseases, which afflict close to a million children and teens in the U.S., ShK-186 has shown early indications of being a potential therapy for allergic asthma. It is estimated that nearly 10 million children in the U.S. are impacted by asthma.
Initial funding will help move ACT's lupus nephritis research into a clinical setting. It will also support work to adapt ShK-186 to become a viable treatment for other autoimmune diseases.