Stories of progress, inspiration, and information in overcoming osteosarcoma.

A panel discussion at the 2025 FDA and OSI workshop.

2025: A Year of Collaboration and Innovation in Osteosarcoma Research

By funding promising research and engaging in exciting collaborations with Break Through Cancer, the FDA, and pharmaceutical companies, OSI is advancing osteosarcoma research.

Improving osteosarcoma treatment requires collaboration, innovation, and the translation of promising science. In 2025, The Osteosarcoma Institute (OSI) advanced all three by helping launch the Break Through Cancer (BTC) osteosarcoma project, engaging with the U.S. Food and Drug Administration (FDA) and the pharmaceutical industry, and funding a new slate of high-potential research projects.

These efforts drive our mission to dramatically increase treatment options and survival rates in osteosarcoma patients. As we close the year, we are proud of what we achieved alongside our partners and donors, and we are energized for what is ahead in 2026.

The Launch of Break Through Cancer (BTC)

Research collaborations often face structural impediments, but BTC is a first-of-its-kind partnership among the world’s top cancer centers backed by a $250 million investment aiming to overcome them.

“The motto of Break Through Cancer is radical collaboration, and that is in fact what we need to solve these tumors like osteosarcoma,” adds OSI Director Lee Helman, MD.

An OSI Strategic Advisory Board member who was also involved with BTC connected our two organizations. Osteosarcoma became BTC’s sixth cancer for study. In 2022, BTC agreed to invest $5 million but needed OSI and our community to fundraise an additional $10 million to enable a $15 million project. After three years, the fundraising goal was met in September, and the project commenced.

“This is a dream come true for patients and those of us who have been working on the problem for a long time,” says OSI President Mac Tichenor. “It is a major achievement for the entire field.”

Collaborating with the FDA

Before approving a new treatment, the FDA evaluates data from clinical trials. In rare diseases, there are a several potential challenges to designing and conducting clinical trials that are feasible and able to generate sufficient data to support drug development in a timely fashion. For example, with osteosarcoma, patient recruitment to clinical trials can take years. That slower pace pushes back study timelines and, in turn, delays when new therapies can reach patients.

“We have been trying to figure out how we can work with the FDA to get drugs to patients sooner,” Dr. Helman says. That effort took a major step forward in October, when the FDA and OSI led a workshop (Advancing Osteosarcoma Drug Development – Connecting Research and Regulatory Pathways for Improved Outcomes) that welcomed more than 300 researchers, pharmaceutical industry leaders, and advocates committed to finding solutions to the challenges that slow the ability to bring new promising therapies to osteosarcoma patients.

“The FDA understands our problem and clearly wants to help us (and pharma) find a way out of the logjam we have been in for so long,” Dr. Helman says. “Pharma also saw that if they have a drug they want to try in a rare tumor like osteosarcoma, the FDA will work with all of us to run development programs for osteosarcoma that are as efficient as possible so that we can move drugs forward much faster. And the FDA made it clear they want to keep working with OSI to make that happen. I am very thrilled with that accomplishment.”

“The FDA understands our problem and clearly wants to help us (and pharma) find a way out of the logjam we have been in for so long." —OSI Director Lee Helman, MD

Biopharma Outreach

“Research is the foundation, and it is essential, but it is not sufficient to actually get new treatments to patients,” Tichenor says. “Even if the research succeeds, we have to have pharma to produce the product and get it to patients.”

For that reason, proactive outreach to biopharma is a strategic priority. Recently, OSI helped one major pharmaceutical company identify and engage with key opinion leaders to advance a new drug candidate and has connected several other companies with OSI experts for research guidance.

“We are happy to provide pharma companies with information and expertise, and that’s starting to happen,” Dr. Helman adds.

$1.5 Million in Osteosarcoma Research Funding Awarded

OSI awarded more than $1.5 million in research funding to four innovative projects in our 2024-2025 grant cycle. This year’s winners are exploring a range of approaches, including:

Developing an immunotherapy designed to reprogram the immune system
Leveraging the body’s naturally occurring natural killer cells to destroy cancer cells
Building a machine-learning model that can predict effective treatments
Exploring a new pathway to osteosarcoma treatment that targets telomeres (the ends of DNA strands that play a key role in cancer cell survival).

At a time when U.S. clinical research funding has been significantly reduced, OSI is proud to help advance these ideas.

Looking Ahead to 2026

We are eager to see progress across several initiatives over the next year, including:

Progress reports from BTC’s Defying Osteosarcoma TeamLab, which is designed to align institutions, data, and funding under a single collaborative framework, bringing together more than 20 researchers from eight institutions.
The MIMIC trial to evaluate a new method of removing lung metastases in dogs. This canine lung surgery trial, supported by OSI and Ethos Discovery, explores a way to make lung surgery safer, faster, and more accessible. Exploring this treatment in dogs can provide insights to help inform osteosarcoma treatment and drug development for human patients.
Continued pharma outreach to help expedite development of new therapies for osteosarcoma.
The launch of a research project exploring opportunities to exploit osteosarcoma’s chaotic genome to kill the cancer. This project marks our first prescriptive, OSI-defined research challenge.

As we enter 2026, your partnership is vital to advancing the most promising osteosarcoma research. Please consider donating today.