Moving Cell Therapies Forward

We’re investing hundreds of millions of dollars to develop, manufacture and test cell therapies that aim to cure diseases at the source.

In 2016, we launched a major initiative to increase our ability to develop, manufacture and test “cell therapies” – an emerging approach that uses living cells to treat and cure disease.

cell_neonTo create cell therapies, researchers remove a small number of cells from a patient’s blood. Then they change the genetic instructions in those cells and infuse them back into the patient. Some of these therapies edit or replace the genes that cause disease. Others insert genes that tell cells to fight disease.

We’re among a small handful of institutions at the forefront of developing, testing and refining cell therapies for children. In our initial studies, these therapies have shown great promise against leukemia and severe immune disorders.

Now our goal is to speed our progress and help our researchers pursue cell therapies for dozens of other cancers and diseases. To achieve this, we plan to add more all-star researchers to our team, and we recently broke ground on a $300 million state-of-the-art research building that will open in 2019.

“In the past few decades, researchers have made tremendous progress on treatments that control diseases or their symptoms,” says Dr. F. Bruder Stapleton, Seattle Children’s chief academic officer. “We hope our investment in cell therapies helps us take the next step, which is to find cures.”

Progress against cancer

Cancer immunotherapy research is a key part of our cell therapy research.

These therapies build on a new generation of CAR T-cell therapies developed by Dr. Michael Jensen to reprogram T cells by inserting genes that tell them to find and kill cancer cells. Our first phase 1 and phase 2 clinical trials of this approach helped 93% of children with acute lymphoblastic leukemia achieve initial remission, with about half of those children relapsing.

Now Jensen and his colleagues are testing versions of the leukemia therapy that could prevent relapse. Seattle Children’s researchers are also designing immunotherapies for more complicated cancers. For example, we are leading one of the nation’s first clinical trials of T-cell therapy for neuroblastoma.

“Our cancer immunotherapy progress gives us confidence that cell therapies can work, that we have the right team to develop them and that a bigger investment could help transform treatment,” says Dr. Jim Hendricks, president of Seattle Children’s Research Institute.

Fixing immune disorders

Our cell therapy research includes a push to apply these therapies to immune disorders.

Rawlings and ScharenbergDrs. David Rawlings and Andy Scharenberg have pioneered ways to edit or replace faulty genes. They recently opened a clinical trial of a genetic treatment that could cure severe combined immunodeficiency without a bone marrow transplant. Rawlings plans to open similar trials within two years for two other rare immune diseases, Wiskott-Aldrich syndrome and X-linked agammaglobulinemia.

Rawlings and Scharenberg are also developing cell therapies for autoimmune diseases. The researchers are using gene editing to create a T cell that functions as a peacekeeper, telling attacker cells to calm down. They have used this approach to stop autoimmune disease in animals and plan to start clinical trials in children within three years.

“The first step is to show that these therapies are safe and effective for diseases that can’t be stopped any other way,” Rawlings says.

A game-changing facility

Cancer and immune disorders are only the beginning. But our current research facilities can’t accommodate all the clinical trials we have in the pipeline.

Our new research building will double our existing research space and give us room to recruit more researchers. And it will include a larger facility for manufacturing cell therapies, boosting our capacity to support clinical trials for many different diseases.

“I hope that, someday, doctors will send us cells from children who are born with an immune disorder or diagnosed with cancer. We’ll edit or reprogram those cells and send them back,” Hendricks says. “This is about more than finding cures for kids in Seattle – it’s about transforming treatment around the world.”

Published in the Academic Annual Report, March 2016