Recently, the National Cancer Institute (NCI) announced it would end its funding of the Pediatric Brain Tumor Consortium (PBTC), a leading clinical trial network at the forefront of developing new therapies and technologies for pediatric brain tumors. While the NCI’s transition plan calls for shifting PBTC’s work to a broader, early-phase network covering all pediatric cancers, the move will almost certainly delay discovery and development of new treatments in an already underserved area of medical need.

Just ask the parents of the thousands of children and adolescents with brain tumors who are diagnosed each year, many of whom have limited time remaining to spend with their children due to the lack of effective therapies.

Cancer remains the number one killer of children and young people by disease in the U.S., and brain tumors account for about one out of four childhood cancers. Today, many pediatric cancers are often treated with the same chemotherapies that have been used for decades and that too often simply don’t work.

As two people who have together spent many decades trying to make a difference for cancer patients – one as an industry executive and the other as the father of a son with a malignant brain tumor and a patient advocate – we have seen first-hand how the lack of new medicines impacts children battling brain tumors and their families. Many of these children never get the chance to grow up.

Defunding the PBTC would not only slow the development of new therapies for pediatric brain tumors, it would also dismantle a collaborative, specialized network of researchers who mentor the next generation of clinical trialists. The loss of this infrastructure would weaken both the innovation and the pipeline of expertise that children with brain tumors urgently depend on. The decision to defund the PBTC will dismantle clinical trial infrastructure and brain tumor research expertise that have taken many years to build and that are not easily transferred to another network with different expertise, different capabilities and, most importantly, competing priorities. This type of systemic issue continues to result in the defunding of rare diseases and creates a disincentive for critically needed new and future researchers to get into this field.

It is no surprise that defunding the PBTC has alarmed scientists, industry, cancer patient advocates, doctors, kids with brain tumors, and their families. One bereaved mother and patient-family advocate for PBTC has said, “I am alarmed and heartbroken by the NCI’s decision… When NCI support ends, so too will a critical lifeline for children diagnosed with brain tumors… For families like mine, the PBTC has represented hope. To see that hope taken away… feels like a devastating step backward.”

The pediatric brain tumor community is a tight-knit group united by a shared commitment to a future in which new innovative, targeted medicines are developed and promptly get into the hands of doctors and patients who need them. Collaboration and steady funding for organizations like PBTC—built to tackle the toughest brain tumors—are essential to advancing new discoveries and novel medicines focused on finding cures.

We ask that policymakers act quickly to restore or replace PBTC funding. We’ve already seen all PBTC trials at member hospitals forced to pause clinical study enrollment, leaving patients and their families with no alternative treatment options left to try. Finally, access must be protected so all children with brain tumors, regardless of what part of the country they live in, can benefit from trials and potential breakthroughs. Kids with brain tumors and their families deserve the resources to accelerate progress.

Last month, I read an article in the New York Times about a regimen generated with the help of AI using a drug that was repurposed to treat a rare and aggressive blood disorder called POEMS syndrome in a patient who had been battling the disease for a year. This was a last-ditch effort – he had already been asked to choose where he wanted to die – at home or in the hospital. According to the article, within a week of the new regimen, the patient showed a positive response, and within months he was in remission.

The article stayed with me, not just because of the promising outcome for this patient, and potentially others, but also because of its similarity to our start at Day One Biopharmaceuticals. We were founded to address the lack of new therapies for children with cancer that have resulted from traditional drug development efforts. Children with cancer and their families wait for years for new, life-changing treatments. As Day One was getting off the ground in 2019, Takeda had decided to cease development of tovorafenib (previously known as TAK-580 at Takeda and MLN2480 at its original developer, Millenium) in adults cancer patients. Fortuitously, champions for pediatric cancer within Takeda convinced their leadership to out-license the drug to a new biotech company interested and qualified to move the program forward. That company was Day One. Four years later, in April 2024, we received FDA approval for children with relapsed or refractory pediatric low-grade glioma (pLGG).

Discovering new applications for sometimes decades-old, mature drugs isn’t new. Indeed, it has been an important area of work for certain patient populations for many years. Look no further than the storied history of thalidomide, now being used for the treatment of multiple myeloma, and propranolol which is being investigated for its use in angiosarcoma.

As we all know, the need for new, targeted therapies for cancers and other rare diseases remains urgent. This is especially true in pediatric oncology, where there has long been an innovation gap.

Our first commercial product has been foundational for Day One’s growth and has enabled us to do more for patients and their treating physicians who urgently need new therapies. We would not be in the position we are today to identify and advance new programs that have best-or first-in-class potential as new medicines had it not been for the long and windy path tovorafenib traveled over the course of many years.

We’re using what we call a Search & Develop (S&D) strategy with the goal of making a real difference in the treatment of the right patients at the right time. This can be applied to the development of previously shelved agents or novel targeted agents. An example is our recent acquisition of DAY301, a potential first-in-class Antibody Drug Conjugate (ADC) that may have broad applicability in adult and pediatric solid tumors. A Phase 1 trial of DAY301 in patients started earlier this year.

Can our S&D strategy get new medicines into the hands of kids and adults with rare, hard to treat cancers faster? Given drug research costs are projected to continue rising and the average cost of bringing a new drug to market is estimated at $1.3B driven by factors including increased trial complexity and challenging research areas, our approach is certainly a practical one.

I am inspired by Dr. Fajgenbaum and his team at the University of Pennsylvania who were behind the innovative approach regimen that saved the patient’s life discussed in the NYT. I look forward to sharing more stories like this in the near future, including drugs that we are developing at Day One that will hopefully change the life trajectories of both pediatric and adult patients.

By Dr. Jeremy Bender, CEO

As we kick off 2025, Day One’s CEO Jeremy Bender took the stage at this year’s J.P. Morgan Healthcare Conference to reflect on the Company’s remarkable journey and to unveil our vision for the future. He highlighted the significant progress we’ve made, shared the exciting opportunities that lie ahead, and emphasized how we are poised to deliver life-changing medicines to patients faster, bolder, and with greater impact.

What began with inspiration driven by the urgent needs of children impacted by cancer, has in just six years, transformed Day One into a commercial-stage company with the FDA approval of our first commercial medicine, OJEMDA™ (tovorafenib). Our top priority in 2025 is to help even more patients impacted by a pediatric low-grade glioma (pLGG) diagnosis by increasing the breadth and depth of prescribers, establishing OJEMDA as the standard of care in the second-line setting, ensuring broader patient access, and working to make this treatment available to patients beyond the U.S. through our global partnership with Ipsen.

Driven by our dedication to patients, we’ve also made significant advancements in our pipeline, including progressing the global Phase 3 FIREFLY-2 trial of OJEMDA in front-line pLGG, which we expect to fully enroll in the first half of 2026. Additionally, our DAY301 program, a clinical-stage Antibody Drug Conjugate (ADC) targeting PTK7 in solid tumors for both adult and pediatric cancers, has just cleared the first cohort in the Phase 1a portion of the Phase 1a/b clinical trial. This ADC is designed to maximize the therapeutic window and has the potential to become a first-in-class option in addressing unmet needs in cancer care.

Finally, we will continue to invest in opportunities focused on targeted therapies that have the potential to have a substantive impact over existing medicines in the oncology space. Our achievements to date are a testament to the team’s deep expertise in oncology as well as our ability to identify and acquire high-potential programs. As we continue this journey, we are focused on ensuring that patients of all ages can look ahead from “Day One” to the future they’ve always envisioned.

To learn more about Day One’s vision for 2025 and beyond, as well as key highlights from JPM 2025, click here.