5 Developments in the Pipeline at Seattle Children’s Research Institute

Ranked as one of the top pediatric research centers in the U.S., Seattle Children’s Research Institute has accomplished so much in its 11-year history, and there is much to look forward to in 2018. Here, Dr. Jim Hendricks, president of Seattle Children’s Research Institute shares with On the Pulse what’s in store for the year ahead.  

1. Cancer immunotherapy

Seattle Children’s continued immunotherapy work is going to be very exciting this year.

In 2017, Seattle Children’s opened three new clinical trials offering innovative chimeric antigen receptor (CAR) T-cell immunotherapies for children and young adults with relapsed or refractory acute lymphoblastic leukemia. Currently, there are four Pediatric Leukemia Adoptive Therapy clinical trials underway at Seattle Children’s (PLAT-02, PLAT-03, PLAT-04 and PLAT-05), with others expected to open this year.

While first generation CAR T-cell immunotherapies have proven effective at initially eliminating cancer in patients who are very sick, preventing relapse continues to present a problem in immunotherapy trials across the country.

Seattle Children’s researchers are dedicated to improving the therapy with the goal of long-term remission for all patients — and ultimately, a cure. PLAT-03, PLAT-04 and PLAT-05 each feature an innovative immunotherapy strategy to address why cancer is able to return after the initial CAR T-cell therapy.

As we continue to make progress in treating leukemia, we are confident that the promise of immunotherapy can extend to other cancers types, as well as to other diseases like HIV, lupus and type 1 diabetes.

2. Non-cancer gene therapy

Primary human B cells could offer the next promising cell therapy. Credit: NIAID

Seattle Children’s is also a world leader in developing a type of cell therapy called gene therapy.

Gene therapies fix the genes that cause disease by inserting new, synthetic genes into cells, repairing the genes in those cells, disabling faulty genes or manipulating genes to change a cell’s function.

Just this last year, one of researchers made a discovery that I think could be the biggest home run yet in the research institute’s history.

A seminal paper by Dr. Richard James, published in Molecular Therapy, describes how the research team genetically reprogrammed primary human B cells to act as cell factories capable of delivering sustained, high doses of therapeutic proteins. The gene editing techniques used reprogrammed B plasma cells to secrete a protein at levels that could treat patients with hemophilia B. This research also suggests that engineered B cells could deliver proteins to disarm infectious agents or dampen the overactive immune response seen in autoimmune diseases.

3. Building Cure

When Building Cure opens in 2019, it will more than double the size of our research institute campus to more than one million square feet.

Much of 2018 will be spent preparing for the opening of Building Cure.

Building Cure is Seattle Children’s investment in cures for childhood diseases. When the new building opens in 2019, it will more than double the size of our research institute campus to more than one million square feet. The building will include 11 floors of laboratory space devoted to research.

It will help us accomplish more life-changing research that will make a difference for kids all around the world with cancer, diabetes, sickle cell disease and other childhood diseases. For example, we’ll be able to treat 1,000 cancer patients a year with groundbreaking T-cell immunotherapies, compared to the 150 patients a year we can treat with current resources.

Building Cure will house the “Cure Factory” facility, a state-of-the-art manufacturing center that translates laboratory discoveries into cell therapies for patients using immunotherapy and gene therapy.

Building Cure will also create an improved space for The Science Discovery Lab, a teaching lab and classroom for children and teens. Through Seattle Children’s science education programs, we’re inspiring students to pursue careers in science, technology, engineering and math (STEM) so they’ll become our researchers of the future.

4. Science industry partnerships

By developing collaborative relationships within the scientific industry, we will continue to translate the work of our research investigators and clinicians into innovative products, devices and medical technology to improve the lives of children and their families around the globe.

One such example is our research collaboration and exclusive license agreements with Casebia. This partnership builds on pioneering discoveries made by Seattle Children’s teams to engineer, manufacture and expand T-cell therapies. Through significant funding for research at Seattle Children’s, and access to Casebia’s innovative gene-editing tools, the agreements provide an opportunity to rapidly advance this work.

Specifically, teams will combine efforts to develop gene-edited T cells to treat a broad spectrum of autoimmune diseases. Such therapies would offer considerable improvement over current therapies, which broadly suppress the immune system and make the body vulnerable to infection and other malignancies.

5. Precision medicine

A novel drug that targets a genetic mutation found across different types of cancer is offering precision medicine treatment options to patients like Ashton Leeds.

Precision medicine remains at the forefront of Seattle Children’s research priorities. In 2017, we celebrated the launch of the Brotman Baty Institute for Precision Medicine – a partnership that combines the research strengths and capabilities of Seattle Children’s, UW Medicine and Fred Hutchinson Cancer Research Center to pursue new, personalized approaches to the prevention and treatment of disease.

We’re already witnessing how sequencing technologies can help provide a genetic diagnosis for children with epilepsy. In some cases, this information can help a neurologist better direct which therapies may be most effective in stopping the child’s seizures.

In cancer, we’re matching patients to therapies based on the genetic makeup of their cancer through the High-Risk Leukemia Program. Another trial is studying the use of a novel drug to target a genetic mutation found across different types of cancer.

With precision medicine and genetic sequencing come vast troves of data. Interpreting this data into clinically significant advancements for patients will be important work of the institute.

Efforts will support ongoing research to identify the genetic mutations behind diseases, so we can develop individualized therapies that target the root cause of a patient’s disease. Our goal is to find new strategies with the potential to cure disease, not just treat the symptoms.

Research is essential to fulfilling Seattle Children’s mission to treat, prevent and ultimately eliminate pediatric disease. Learn how you can support research programs at Seattle Children’s or participate in a research study.