For researchers, it’s known as “the valley of death.” It’s the often-unfunded middle territory between the early part of research, when new ideas are blossoming in the lab, and the end stage years later, when a company is finally ready to bring a new treatment to the marketplace.
Children’s Cancer Research Fund (CCRF) is stepping into the gap with impact investments to help biotechnology companies translate early discoveries into effective treatments for childhood cancer. CCRF recently announced impact investments in OS Therapies and Oncoheroes Biosciences.
“CCRF has funded research mostly in the earlier stages. And that’s really important — obviously, you can’t get to the later stages without that,” explained CCRF CEO Daniel Gumnit. “But there is this gap between lab research and a drug that can be used with patients. We see an opportunity to support research on the later end of the spectrum.”
Impact investments from nonprofits are a relatively new tool in the quest to cure childhood cancer, in part because there historically haven’t been many biotechnology companies interested in focusing on the smaller pediatric cancer market. But that’s changing.
In 2020, the RACE (Research to Accelerate Cures and Equity) for Children Act went into effect. The law requires pharmaceutical companies to test cancer drugs developed for adults in children if both groups share the same molecular target of the drug. At the same time, advancements in genomics and new technologies are propelling scientific research faster than ever before.
“The childhood cancer ecosystem is ripe for acceleration of progress,” Gumnit said. “Things are changing around us, and we see an opportunity to make an even bigger impact.”
The benefits of an impact investment are three-fold: It helps provide the resources a startup company needs to develop promising new treatments; if successful, it would generate financial returns that CCRF can later invest back into other cancer research; and most importantly, it could bring lifesaving treatments to patients more quickly.
The investment in OS Therapies comes from the Zach Sobiech Osteosarcoma Fund at CCRF. Zach, a Minnesota teen musician who inspired the recent Disney+ film Clouds, and his family established the fund before he died of osteosarcoma in 2013. About 50% of patients with this difficult-to-treat bone cancer face a relapse, which is almost always fatal within months.
But osteosarcoma treatment could be on the cusp of a major breakthrough. Pre-clinical research shows that human epidermal growth factor receptor 2 (HER2), a protein that is overexpressed in some breast and ovarian cancers, also plays a role in certain forms of osteosarcoma. Support from CCRF and the Zach Sobiech Osteosarcoma Fund will support OS Therapies in running a national Phase IIb clinical trial, which is currently under way. In canine studies, the treatment significantly improved disease progression and tripled overall survival rates.
Oncoheroes Biosciences is the first biotech company focused exclusively on developing new treatments for pediatric cancer. The company plans to focus on a variety of childhood cancers, starting with new therapies for rhabdomyosarcoma, a type of cancer that affects the muscles, and medulloblastoma, the most common form of brain tumor in children.
Both companies have impressive teams of scientific experts as well advisors from families who have been touched by childhood cancer. “I’m excited that these companies understand the complexities and nuances of childhood cancers and the needs of that community,” Gumnit said. “I think one of the most frustrating things for families is if their child relapses or the cancer never goes away, they don’t have a lot of options. We are all aiming to change that.”