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Collaborative aims to accelerate immunotherapy development for pediatric cancers
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Experts from several health systems have launched CureWorks, a collaborative effort to accelerate the development of immunotherapies for children with cancer.
Participating institutions include Seattle Children’s Hospital, Children’s National Health System, BC Children’s Hospital and Children’s Hospital Los Angeles.
Michael Jensen, MD, executive director of CureWorks and director of Ben Towne Center for Childhood Cancer Research at Seattle Children’s Research Institute, said key components of the collaboration include the expansion of multisite clinical trials for immunotherapies developed by the academic partners, a centralized facility for reprogramming immune cells to recognize and fight cancer, and a network of member hospitals to share resources on the use of chimeric antigen receptor (CAR) T-cell therapy in pediatric populations.
H emOnc Today spoke with Jensen about the need for immunotherapies for pediatric cancer populations, the advantages of collaboration and the goals of the initiative.
Question: How did this collaboration come about?
Answer: In order to revolutionize treatment for pediatric cancer, academic institutions must work together to achieve better treatments and outcomes for children. CureWorks began through recognition that the technologic and therapeutic platform of CAR T cells requires infrastructure for deployment that is very expensive, and that there are a limited number of experts available to lead such an enterprise. At Seattle Children’s, we are building a very large Good Manufacturing Practice (GMP) facility and ramping up CAR T-cell product manufacturing capacity, enough that products can be manufactured in Seattle and shipped to where patients are treated. If we want to have a greater impact for pediatric patients, we have to expand our reach and allow children access to groundbreaking cancer immunotherapies in their own communities. CureWorks will increase the number of immunotherapy trial sites around the globe, and it will quicken the pace of pediatric cancer discovery by diversifying and expanding the amount of data available to researchers through its scientific network.
Q: What do you mean by sharing resources? Are you sharing information or the technology itself?
A: All of the above. Once these trials are open at a CureWorks member hospital site, immune cells will be collected from the patient and sent to Seattle Children’s GMP facility, where the cells will be reprogrammed to express the CAR protein. The newly engineered, cancer-fighting T cells will then be shipped back to the patient’s health care team for infusion. CureWorks will also streamline the clinical trial enrollment and coordination process for members. The collaborative will allow us to take the most promising products out of a phase 1 trial at any member site and facilitate their testing in multisite phase 2 trials.
Q: What about the challenges involved in shipping the cells to other sites?
A: This barrier is rapidly coming down. It’s a two-way process in which the patient gets apheresis at their own hospital and their blood product is shipped to us. After we reprogram the T cells, they are cryopreserved and sent back in next-generation, small ampules. From there, the nurse at the bedside warms the product and it is infused into the patient through a syringe. It is very easy to utilize. What’s a little more complicated is the apheresis at each institution. We are doing training with our partners to get that nailed down.
Q: How many sites are involved, or plan to be involved?
A: The collaborative includes Seattle Children’s Hospital, Children’s Hospital Los Angeles, Children’s National Health System in Washington, D.C., and our first international site in Vancouver at BC Children’s Hospital. We have a number of other major pediatric hospitals in North America we’re talking to, and another potential site in Europe we’re working with, as well..
Q: What are the short-term and long-term goals of the collaboration?
A: Our mission is really focused on developing innovative cellular immunotherapies for pediatric deployment. We see ourselves as being able to take programs through phase 2 in an efficient and rapid manner. We are optimistic that if we achieve positive phase 2 results, we’ll potentially be able to get these products to the market, but understand if an effective product for a rare pediatric cancer does not get picked up, CureWorks may need to provide this to children indefinitely.
Q: Why does industry appear to lack interest in developing these therapies?
A: Industry is most interested in the low-hanging fruit of developing products that would be used by larger adult populations with hematologic malignancies. Even with more common childhood cancers, the number of cases per year in the United States or globally is relatively small compared with incidence of common adult carcinomas.
Q: Do you have any thoughts on how this collaboration might expand or evolve moving forward?
A: The expansion of CureWorks is going to be based on geographic growth and clinical trial diversity growth. Right now, our lead products are CAR T cells. However, within the collaborative, there are investigators who are developing cellular immunotherapies for chronic infections and to re-establish antiviral immunity in patients with immunodeficiency. Another therapeutic space is the engineering of regulatory T cells to treat autoimmune disease. The primary theme of CureWorks will remain for the foreseeable future in cellular immunotherapy because that is where our GMP, regulatory and deployment expertise is.
Q: Do you have any other comments about this collaboration?
A: We believe a unified effort among leading children’s hospitals is the best way to drive the discovery of new therapies for pediatric cancer. Ultimately, this will allow us to give access to this therapy to more patients because we’re able to distribute it across the country and, eventually, globally. Additionally, we also hope to train the next generation of clinical and scientific immunotherapists to populate this promising field in pediatrics. – by Rob Volansky
For more information:
Michael Jensen, MD, can be reached at Ben Towne Center for Childhood Cancer Research, 1100 Olive Way, Suite 100, Seattle, WA 98101; email: michael.jensen@seattlechildrens.org.
Disclosure: Jensen is a founder of Juno Therapeutics, has patents related to Juno-Celgene CAR T products and receives sponsored research agreement funding for laboratory research.