Partnerships with private companies play a growing role in Seattle Children’s Research Institute’s quest to cure childhood diseases.

Seattle Children’s Research Institute’s investigators are making discoveries about the underlying mechanisms of childhood diseases and developing better ways to treat and cure them. We are joining forces with more and more companies who have the expertise – and the funding – to translate these discoveries into real-world treatments.

“We can more rapidly develop cures and better treatments for pediatric diseases by working with companies who are already developing treatments for adults with these same diseases,” says Dr. Jim Hendricks, the research institute’s president.

Game changer for lupus
Collaborations Inline 1

The market for pediatric treatments is too small for pharmaceutical and biotechnology companies to spend millions of dollars developing them – there often aren’t enough pediatric patients for them to justify the investment. But companies can extend the patent on an adult drug if they develop a pediatric application of that drug.

“We can work with companies to develop a pediatric application and that can pay off for them by increasing profits on the adult side,” Hendricks says.

We put this model in motion in 2014 by teaming up with Seattle-based biotechnology company Kineta Inc. to launch the Alliance for Children’s Therapeutics (ACT). Kineta developed ShK-186, a drug candidate that targets the cells that go haywire in lupus and other autoimmune diseases. The drug has disarmed these cells in mice with minimal side effects.

Seattle Children’s Dr. Anne Stevens is working with Kineta’s Dr. Ernesto Munoz to study whether ShK-186 can have a similar impact on cells taken from pediatric lupus patients experiencing lupus nephritis, a complication that can cause kidney failure.

“Anne is one of just a few pediatric rheumatologists in the country who study autoimmune diseases, and Kineta knows how to bring new treatments to market. Combining their expertise means we can quickly figure out if the drug works and shorten the timeline to get it to children,” Hendricks says.

Stevens and Munoz hope to launch a clinical study to test ShK-186 in children with lupus nephritis in 2015; if the trial proves successful, ShK-186 could become the first pediatric lupus therapy ever approved by the Food and Drug Administration. ShK-186 is also in pre-clinical testing by Kineta and Seattle Children’s to see if it could be an effective treatment for pediatric multiple sclerosis, inflammatory bowel disease and asthma – conditions that together affect millions of children.

“The clinical trial for lupus nephritis could help us determine what the drug’s side effects might be and what dose to use in kids,” Hendricks says. “Kineta gets a chance to help children afflicted with this disease and to study a drug that could also have huge adult benefit. It’s a win-win.”

Fueling partnerships

The research institute recently created the Office of Science-Industry Partnerships to foster collaboration with pharmaceutical, biotechnology and medical device firms. The cooperation could range from ACT-like partnerships to arrangements where we license a discovery to a company and collect royalties if it pans out.

For example, we are a key partner to Juno Therapeutics Inc., a biotechnology firm that has raised more than $300 million to develop immunotherapies that use genetically modified T cells to destroy cancer cells. Working with Juno helps researchers at Seattle Children’s, Fred Hutch and Memorial Sloan-Kettering Cancer Center to keep working to replace traditional pediatric cancer therapies with immunotherapies that are more precise and carry far fewer side effects.

“NIH funding is drying up and we need new ways to fund and accelerate pediatric research,” says Dr. Elizabeth Aylward, a neuroscientist who heads the new office. “Sometimes that means working with people who want to make money.”

Aylward is taking stock of the research institute’s projects and identifying those that could have value in the marketplace. She is also traveling around the country to meet with companies and look for opportunities to work together.

“These companies are very transparent about what they’re working on and what they’re looking for – and they’re eager to talk to us because our research has real-world applications,” Aylward says. “We think these partnerships are the wave of the future and are key to bringing better treatments to children faster than ever before.”

Companies are … eager to talk to us because our research has real-world applications. We think these partnerships are the wave of the future and are key to bringing better treatments to children faster than ever before.
- Dr. Elizabeth Aylward