Day One Biopharmaceuticals to Accelerate Promising Pediatric Brain Cancer Therapy Made Possible by PLGA Fund at PBTF

Kids diagnosed with brain tumors and other cancers are often left behind. Last year alone, the FDA approved 11 new oncology drugs to treat adults, but there has never been a new drug developed and approved specifically for pediatric brain cancer.

Thirteen years ago, a small group of Boston-area families whose children were battling brain cancer decided to stop waiting for someone else to save the day and created the PLGA Foundation/A Kids’ Brain Tumor Cure (AKBTC), now the PLGA Fund at the Pediatric Brain Tumor Foundation.

“Current treatments for children with brain cancer are outdated, toxic and too-often ineffective,” says Amy Weinstein, PBTF’s Director of PLGA Research and Advocacy and former AKBTC executive director. “It was clear that new ideas needed to be explored. Providing researchers with seed funding so that they could focus on PLGA tumors was critical if we were going to expand our understanding of what is possible and help these kids fight for their dreams, not for their lives.”

Today, programs like the Dana Farber Cancer Institute’s PLGA Research Program, which launched in 2007 thanks to PLGA Fund families and supporters, have made rapid progress in our understanding of these tumors and developed this research into clinical trials of targeted therapies for pediatric brain tumor patients. Among the most promising of these therapies is TAK580, an oral, once-a-week treatment in the clinical trial stage that shows potential to become a “gold standard” of care for children battling relapsed glioma brain tumors.

The recent launch of Day One Biopharmaceuticals could take TAK580’s development to the next level. After securing more than $60m in Series A funding, Day One has announced that its first priority will be the widespread development and commercialization of DAY101 (formerly TAK580).

“In the US, children with cancer first gain access to cutting-edge investigational treatments an average of 6.5 years later than adults,” explains Dr. Samuel Blackman, co-founder, pediatric oncologist, and Chief Medical Officer of Day One Biopharmaceuticals, in Day One’s launch announcement. “Our goal is to dramatically change the pace of new cancer drug development for children by identifying and developing novel therapies based on pediatric cancer biology, and then following the biology to the benefit of all patients.”

Enabled by advances in the biological understanding of childhood cancers and informed by its network of leading pediatric oncologists, Day One is equipped to identify patients across all ages who are likely to benefit from a new treatment based on their tumor’s biology. Additionally, they are leveraging improvements in pediatric oncology clinical trial infrastructure and modern study designs to execute more efficient clinical trials for children with cancer.

“For years, industry research has left children with brain tumors behind, focusing on adults, who they view as more profitable. Without support from the PLGA Fund at PBTF, the groundbreaking research that led to the discovery of DAY101’s potential for children never would have been found or even funded,” says Dr. Evan Hecker, an Internal Scientific Advisor to the PLGA Fund and Director of API Development at Spero Therapeutics. “A focused company like Day One can rapidly deliver on the scientific discoveries the PLGA Fund at PBTF supported when no one else did. Although these discoveries have resulted in Phase I and II clinical trials developed by academic scientists, they will need industrial horsepower and financing to get these discoveries approved for children. We are thrilled about Day One’s mission to deliver better treatments for pediatric brain cancer patients, faster.”

“Groundbreaking change in successfully treating pediatric brain cancer patients comes when nonprofits provide critical seed funding to help scientists move the field forward and the pharmaceutical industry lends its support to help take the results of that research from the lab to treating children. The partnership between the PLGA Fund at PBTF, Dana-Farber’s scientists, and Day One offers a model of success that can and should be duplicated as PBTF looks towards the future and pursues other initiatives to identify new targets and drug agents to defeat this devastating disease,” says Courtney Davies, President and CEO of the PBTF.

In addition to the PLGA Fund, the development of DAY101 would not have been possible without the support of organizations such as Catching Up With JackTeam Jack FoundationThea’s Star of Hope, and Why Not Me?, as well as Geared Up For KidsPar For KidsStarry Night Knoxville, and Think Fit For Kids fundraising events.

Read Day One Biopharmaceutical’s announcement here. Learn more about the PLGA Fund at PBTF at

About the Pediatric Brain Tumor Foundation
Every day, 13 children and teens are diagnosed with a brain tumor, the deadliest childhood cancer. Every day after, they are in a fight for their life. It is a fight the Pediatric Brain Tumor Foundation is here to help families win. Since 1991, PBTF has led the way in transforming how childhood brain tumors are treated and how children, teens, and their families are cared for. Through partnerships with leading medical institutions, organizations, families, and individuals, we have funded more than $45.7 million in scientific discovery and we connect thousands of families each year with patient and family education, financial relief, and a thriving community of support. A world without childhood brain tumors is possible when we put our heads together to effect real, meaningful change. Join us in our mission to Care. Cure. Thrive. at

About DAY ONE Biopharmaceuticals
Inspired by children with cancer, Day One Biopharmaceuticals creatively and intentionally develops promising new therapies that change the outcome for people of all ages living with cancer. Day One’s first announced program is DAY101, an oral, once-a-week, type II pan-RAF inhibitor that is currently being evaluated in human clinical trials. Day One partners with leading clinical oncologists, families, and scientists to identify, acquire, and develop emerging cancer treatments that can help both children and adults living with cancer. Through Day One and its collaborators, cancer drug development comes of age. For more information, please visit


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