Seattle Children’s Aims to Partner With Medical Industry to Develop Pediatric Therapies

While there are approximately 10,000 children in the United States living with lupus, a lifelong disease that causes inflammation throughout the body, there are currently no U.S. Food and Drug Administration (FDA) approved medications to treat these kids, forcing physicians to prescribe medications that are approved for adult patients but may not be appropriate for developing bodies.

It’s a problem that impacts all areas of pediatric medicine. In 2013, the FDA in total approved 27 new drugs; just seven of these were approved for pediatric use. Of the 55,000 clinical trials conducted between 2005 and 2010, only 9% were designed for children.

“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 National Institutes of Health’s budget is devoted to pediatric medicine and care,” said Dr. Jim Hendricks, 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.”

Seattle Children’s is hoping to bridge the gap between promising research and potential treatments and cures for pediatric diseases by partnering with drug manufacturers and biotech companies through its new Office of Science-Industry Partnerships.

A historic problem

While tightening budgets at the National Institutes of Health have made it more difficult to obtain funding for medical research in recent years, the medical industry has always had greater incentive to invest in therapies for adults than those designed for children. This is primarily because there is greater demand and less risk involved with adult patients.

Most pediatric diseases are known as “orphan diseases,” meaning they affect fewer than 200,000 patients in the U.S. Even pediatric cancer is diagnosed in only 12,500 U.S. children each year.

Developing treatments for children can also be more expensive than for adults. Clinical trials with children are more complicated. Both the parent and the child must consent to the study and be willing to participate, and unique ethical considerations must be weighed. Additionally, therapies must be tested at various developmental stages.

With less demand and greater costs, the medical industry faces significant risks when developing treatments for children.

“There’s not enough money to be made in pediatric medicine,” said Dr. Elizabeth Aylward, director of the Office of Science-Industry Partnerships. “It’s not that these companies don’t care about kids, but they must consider the potential return on their investment.”

A new solution

Staff at Seattle Children’s Research Institute have carefully considered the challenges that keep the medical industry from investing in pediatric research. Now, the institute is proposing a solution: The Office of Science-Industry Partnerships. Seattle Children’s Research Institute will fund research to study potential new treatments and will collaborate with drug manufacturers or biotech companies so new treatments can be brought to market.

“We want to develop collaborative relationships with industry partners that will allow us to translate the work of our clinicians and research investigators into real world products,” Aylward said. “This will reduce their risk and speed up the development of pediatric therapies.”

Industry partners have even more reason to partner with Seattle Children’s since the FDA recently began offering new incentives to manufacturers working on treatments for children, including longer drug licensing.

“Let us help bring your drug or device to the market,” Aylward said. “If you have products that won’t be tested in children because there is too much risk and too little potential profit, give us access to them so we can fund the research and find out if they could be applicable to children.”

While other research institutions around the country have technology transfer offices to help researchers with patents and licensing of new technologies, Seattle Children’s intent to collaborate with industry partners and jointly fund both early- and later-stage research is unique.

Collaborating to fight lupus

Seattle Children’s Research Institute is currently partnering with local biotech Kineta to study a potential new treatment for children and teens with lupus nephritis. The drug they are testing is derived from sea anemone venom.

One example of such a partnership is the Alliance for Children’s Therapeutics, a collaboration between Seattle Children’s and local biotech company Kineta 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.

This unique and collaborative funding model combines philanthropic gifts made to Seattle Children’s Research Institute with equity investments made to Kineta.

For their first collaborative project, Dr. Anne Stevens, a researcher at Seattle Children’s Research Institute, is working with researchers from Kineta to conduct pre-clinical laboratory tests to advance Kineta’s lead compound, called ShK-186, as a potential treatment for lupus.

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.