A critical juncture in medical research and drug discovery is known as the “valley of death” — a gap between academic innovations and their practical application in the real world. It’s a chasm where many promising breakthroughs languish due to insufficient funding. This predicament is especially pronounced in rare indications like childhood cancer, where early-stage support from the private sector is lacking, and industry interest is scarce.
Amid this stark reality, Ricardo Garcia, a determined parent whose child battled a brain tumor, stood up at the October 2016 CanceRx Conference at MIT with hundreds of leaders and researchers in cancer research and drug development, demanding action. He challenged the gathered scientific community, imploring them to support pediatric cancers, “What are you all doing to help children with cancers? We are desperate to find drugs for our children, but we are helpless! Why can’t all the children’s foundations work together? There is not any biotech focused on pediatric cancers. What would it take to build one?”
The plea did not fall on deaf ears. Dr. Sujuan Ba, President & CEO of the National Foundation for Cancer Research (NFCR) and CEO & Co-Founder of the AIM-HI Accelerator Fund, recognized the urgency and underserved needs. She extended an invitation to Garcia to visit the NFCR office and explore the potential for collaboration. This encounter marked the genesis of a remarkable partnership that sought to revolutionize the field of pediatric oncology.
In 2017, Ricardo Garcia, alongside Dr. Cesare Spadoni, a parent who tragically lost his daughter to pediatric cancer, co-founded Oncoheroes Biosciences. With an initial $200,000 in seed funding from NFCR, Oncoheroes Biosciences was able to receive a generous €600,000 matching grant from the Spanish Government agency. With this funding, the duo established a state-of-the-art discovery lab and launched a pioneering medulloblastoma research program.
The momentum only grew. In 2019, NFCR unveiled the AIM-HI Accelerator Fund, a nonprofit organization focused on accelerating innovative translational research from labs to clinical trials, provided the critical seed investment of $300,000 to Oncoheroes Biosciences which supported Oncoheroes’ clinical development plans for volasertib, after the acquisition of this drug candidate from Boehringer Ingelheim. Since then, over a dozen children’s foundations have joined forces with NFCR to invest in Oncoheroes, an unprecedented collaborative effort among cancer charities.
By October 2020, the FDA granted volasertib Orphan Drug Designation for treating pediatric rhabdomyosarcoma and other rare soft tissue sarcomas — a ray of hope for approximately 450 American families facing this daunting diagnosis each year. By October 2nd, 2023, it was announced that the FDA had allowed Oncoheroes to proceed with clinical trials investigating volasertib as a pediatric oncology drug. Volasertib has shown promise in inhibiting an enzyme called Polo-like-kinase 1 (PLK1). This enzyme plays a significant role in the progression of various types of pediatric cancers.
Oncoheroes has licensed another drug, dovitinib, which targets specific receptors in the body, including fibroblast growth factor receptor, vascular endothelial growth factor receptor, and other receptor tyrosine kinases. This category of drugs shows promise in treating bone cancers in children, such as osteosarcoma and Ewing sarcoma. The development of dovitinib is guided by a special biomarker that helps identify potential individuals who may respond well to the treatment. In a recent development, the FDA has awarded dovitinib the Orphan Drug Designation for its application in treating osteosarcoma, having previously been granted the Rare Pediatric Disease Designation by the FDA in September 2022. “We anticipate that this excellent news will expedite the drug’s clinical development, benefiting not just osteosarcoma patients but also other pediatric solid tumors given the design of dovitinib’s Phase 1/2a clinical trial, which has been recently allowed to proceed by the FDA,” stated by Dr. Cesare Spadoni. These two treatments are supported by rigorous scientific research and hold significant potential for improving patient outcomes and offer promising avenues in the fight against childhood cancer.
Oncoheroes’ extraordinary journey, forged through the visionary partnership with NFCR and AIM-HI, demonstrates the power of public-private collaborations in advancing medical research for rare diseases. By daring to invest in early-stage biotech companies, this alliance has propelled Oncoheroes forward and opened new possibilities for pediatric cancer patients worldwide.
Ricardo Garcia, CEO and Co-Founder of Oncoheroes, wholeheartedly acknowledged the contributions of the NFCR and AIM-HI accelerator, “Dr. Spadoni and I are grateful to NFCR and the AIM-HI Accelerator Fund for believing in our vision and taking a risk with our unproven business model to build the first biotech company dedicated to pediatric cancer. Their investments were critical to our ability to make huge progress and attract other foundation investors such as ImpactAssets, Rally Foundation, Catalytic Impact Foundation, and Dreamers Ventures. These types of joint efforts and public-private collaborations are paving the ways to save children’s lives in the future, we commend and welcome more nonprofit organizations and like-minded investors joining us to develop more effective and innovative drugs for children with cancer.”
In acknowledgment of the profound influence that NFCR and AIM-HI have demonstrated on the growth and advancement of Oncoheroes, Dr. Sujuan Ba has been appointed as a member of the Oncoheroes Board of Directors. Dr. Ba has consistently showcased her prowess in mobilizing resources and support, making her an invaluable champion for Oncoheroes since the company’s inception. Her contributions to Oncoheroes will continue to create a significant impact on the lives of young cancer patients.
Today, more charitable organizations and private foundations have teamed up with the NFCR and the AIM-HI Accelerator Fund to support Oncoheroes Biosciences in its remarkable initiative against childhood cancer. Together, we are committed to a strategy that expedites the discovery and development of superior drugs for children and adolescents battling cancer.
Dr. Sujuan Ba expressed satisfaction with the development of significant scientific advancements and the rate of progress made by Oncoheroes, “We at NFCR and AIM-HI are pleased with the progress made by Oncoheroes. The company was able to achieve the target milestones in a very budget-efficient way. We started our partnership in 2017 with an initial grant to launch their medulloblastoma program and provided additional investment to help Oncoheroes launch their clinical research efforts on volasertib. We believe in Oncoheroes’ focused approach and dedication to defeat pediatric cancer, which has been underserved to date.”
The story of Oncoheroes Biosciences serves as a beacon of hope, illustrating what can be achieved when passion, innovation, and collaboration converge to save young lives. Together, they are rewriting the narrative of pediatric oncology, offering a brighter future for children and adolescents facing cancer.
