Can a nonprofit research institution operate more effectively by taking a page from the commercial biotech industry?
That’s the idea behind Seattle Children’s Therapeutics, a new effort by Seattle Children’s Hospital to reorganize and expand its existing research efforts, running them more like a biotech company and innovation hub and less like an academic institution. The goal is to boost the development of new cell and gene therapies for pediatric cancer and other diseases afflicting children.
Developing pediatric treatments is a challenging, lengthy, expensive process. Academic researchers can discover promising drugs, but it’s difficult to navigate the steps required to bring them to patients, stretching from “the last mouse to the first human” — a journey dubbed the “valley of death” by those in the field.
Seattle Children’s Therapeutics will house, in one place, the resources and expertise to perform the research and development, manufacture the therapeutics, comply with FDA regulatory affairs, manage animal and clinical trials, and analyze research results in human patients.
In addition to supporting Seattle Children’s research, the nonprofit facility will provide resources and space for outside partner institutions and biotech companies working on pediatric medicine.
“The interest in cell therapies right now is tremendous on a global basis,” said Leslie Alexandre, CEO of Life Science Washington, a trade organization. And as a region, “we have staked out an early leadership position in this area. I’m thrilled to see what Seattle Children’s has done.”
“It’s quite remarkable. And it really shows the innovation of thought at Seattle Children’s Hospital with its leadership and its board,” said Dr. Michael Jensen, vice president of Seattle Children’s Therapeutics.
Jensen brings to the effort his personal experience as a founding scientist at Seattle’s Juno Therapeutics. The immunotherapy company spun out of the Fred Hutchinson Cancer Research Center and was acquired by Celgene for $9 billion nearly three years ago. Juno focuses on cancer treatment called chimeric antigen receptor (CAR) T-cell immunotherapy, which genetically reprograms patients’ immune cells to kill cancer.
A startup that is working in partnership with Seattle Children’s today announced that it had raised a $53 million Series A round. The company, called Umoja Biopharma, is developing CAR T-cell immunotherapy treatments for blood-based and organ-based tumors in adult and pediatric patients. Jensen is a co-founder of the company, which is also affiliated with Purdue University.
Seattle Children’s has its own research program that designs, manufactures and tests CAR T-cell immunotherapy in clinical trials. The program is tackling leukemia, lymphoma, brain tumors and solid tumors in children and young adults. The hospital is also home to a facility that produces therapeutic cell products called the Cure Factory. It’s also working to build out a facility called VectorWorks that will manufacture a key reagent used in cell and gene therapy products.
These efforts are being integrated into one physical space through the creation of Seattle Children’s Therapeutics.
“We’re trying to create a seamless organization and build out a lot of biotech enterprise,” Jensen said.
The initiative has 130 employees and an annual budget of $30 million. The hope is that partnerships with other biotech ventures and licensing of intellectual property will help offset some of the costs.
Developing treatments for kids has its own set of challenges. For some ailments, there might be only 200,000 pediatric cases a year in the U.S., making them a less attractive investment for for-profit businesses. Federal grant funding doesn’t have the dollars behind it to pay for early clinical trials, which can cost $5-10 million, Jensen said. Seattle Children’s Therapeutics could provide an alternate path to new therapies.
And the need is clear. Common cancer treatments for kids are still chemotherapy and radiation — therapies developed decades ago for adults that were modified for kids. Jensen would love to see those more primitive approaches relegated to medical history textbooks. Seattle Children’s is already conducting trials for new treatments for all of the major kinds of pediatric cancers.
“Pediatrics historically has been a neglected area for R&D, especially beyond cancer,” Alexandre said.
The new center could logically expand into other diseases. Research on pediatric autoimmune diseases including rheumatoid arthritis and some kinds of diabetes would be a logical fit. Another area would be inherited genetic diseases that could be cured with gene editing and gene repair technologies such as CRISPR.