Drs. Crystal Mackall and John Maris, co-leaders of the SU2C-St. Baldrick's Pediatric Cancer Dream Team, view three areas of research as the most valuable: discovery, development of new therapeutics and clinical trials. Tremendous progress has been made in each area including:
Discovery: The Dream Team has employed technologic advances to define the cell surface landscape of high-risk childhood cancers. This has led to the discovery of several candidate immunotherapeutic targets that have been prioritized for further development based on their potential to translate into new treatments using the newly created IMPACT tool. The team has created a very large dataset that will be shared with the academic community.
Development of New Therapeutics: The multidisciplinary team has selected several candidate immunotherapeutic targets for new therapy development. Beginning with the generation of highly specific "binders" to the cell surface molecules that have been prioritized, the Dream Team is creating therapeutics in three main classes: 1) antibodies, 2) antibody-drug conjugates and 3) chimeric antigen receptors (CARs) for engineering into the patient's own T cells. Examples include new antibodies and antibody-drug conjugates targeting a variety of pediatric solid tumors and new CARs to treat leukemias, neuroblastoma, osteosarcoma and glioblastomas. These new agents are in various stages of preclinical testing in laboratories across the Dream Team.
Clinical Trials: The new Dream Team clinical trials consortium is currently conducting 17 clinical trials – with two more planned to open this year – that have enrolled more than 380 patients. The most important advances have been realized in relapsed acute lymphoblastic leukemia, with stunning and sustained remissions in the majority of patients treated at four cell therapy Dream Team sites. As anticipated, realizing this type of success in childhood brain tumors and other solid malignancies is much more challenging, but the team has made steady progress and is opening novel clinical trials in an expedited fashion, including plans to open the first consortium-wide antibody-based clinical trial in 2016. This novel collaboration with industry will be the first trial open at all eight US and Canadian institutions involved in this Dream Team, and brings a potentially highly effective drug to childhood cancer in an expedited fashion.
"We are clearly at a crossroads in our quest to conquer childhood cancers. The SU2C-St. Baldrick's Pediatric Dream Team has brought together the two most promising arenas in childhood cancer and we think we are revolutionizing the approach to this group of diseases," says Dr. Mackall. "We are now focused on defining mechanism to sustain our progress beyond the four years of SU2C-St. Baldrick's Foundation funding."
Proof of the Dream Team's hard work and dedication has already been realized through the lifesaving work they have been doing. Many young people's lives have been saved, including 8-year-old Phineas Sandi. At age 4, Phineas was diagnosed with chemo-resistant acute lymphoblastic leukemia. He was treated on an immunotherapy trial that resulted from the Dream Team's work. Only 28 days after the procedure, Phineas was in remission and is still cancer free today.
"We are excited about the new lifesaving therapies coming out of the Dream Team's work, using the immune system to fight cancer. We are proud to support this research to use genomics to improve immunotherapies for children's cancers, and are highly encouraged by the outcomes so far. The St. Baldrick's Foundation looks forward to even more new discoveries in the coming year and sharing more success stories like Phineas'," said Kathleen Ruddy, CEO of the St. Baldrick's Foundation.
About St. Baldrick's Foundation
As the largest private funder of childhood cancer research grants, the St. Baldrick's Foundation believes that kids are special and deserve to be treated that way. St. Baldrick's funds are granted to some of the most brilliant childhood cancer research experts in the world and to innovative explorers who bring with them the promise of a future free from childhood cancers. Kids need treatments as unique as they are – and that starts with funding research just for them. Join us at StBaldricks.org to help support the best cancer treatments for kids.
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SOURCE St. Baldricks Foundation