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

Lisa Tichenor, Dr. Bill Tap and Mac Tichenor at CTOS 2024 in San Diego

Breaking Barriers: Taking Aim at an ‘Undruggable’ Target

The OSI’s bold initiative brings a groundbreaking drug trial to osteosarcoma patients in record time.

Targeted therapy has transformed cancer research by focusing on specific components of cancer cells to disrupt tumor growth or destroy them entirely. Current targeted therapies often zero in on receptors or pathways such as HER2, EGFR, or BRAF, and are widely used to treat breast cancer, lung cancer, melanoma, and other malignancies. Despite significant progress in other cancers, researchers have struggled to develop targeted therapies for osteosarcoma. That may be about to change.

In early 2024, Spanish company Peptomyc published the results of a Phase 1 clinical trial testing its new treatment, currently known as OMO-103, in allcomers solid tumors. OMO-103 is a drug designed to target MYC, an oncogene that plays a critical role in cancer progression but has long evaded drug developers.

“It was only about five years ago that we, as a scientific community, fully understood how significant MYC is in influencing cancer outcomes,” says Dr. Lee Helman, director of the Osteosarcoma Institute. “It was previously considered to be ‘undruggable.’”

Why Target MYC in Osteosarcoma Treatment?

The MYC gene is like a master switch for cell growth, making it a key player in cancer development. In osteosarcoma, having too many copies of this gene — called MYC amplification — is linked to more aggressive tumors and poorer outcomes.

“There is a pretty strong correlation between tumors that have an amplification of the MYC gene and very poor outcomes,” Dr. Helman says. “We are not just talking osteosarcoma, either. MYC is highly involved in many, many cancers.” However, developing a drug to inhibit MYC has proven difficult due to the protein’s ever-changing shape, which has made it resistant to traditional drug design approaches.

OMO-103 changes that. This innovative therapy disables MYC, stopping it from fueling tumor growth. In its Phase 1 trial, the medication showed promising results in a variety of solid tumors. “This drug represents a major step forward in targeting one of the most elusive drivers of cancer,” says Laura Soucek, PhD, CEO of Peptomyc and one of the researchers behind OMO-103. “For osteosarcoma patients, this is a chance to bring meaningful innovation to a disease that has been underserved for far too long.”

A Closer Look at the Phase 2 Clinical Trial

The Phase 2 clinical trial is being conducted at the Vall d’Hebron Institute of Oncology (VHIO) in Barcelona, Spain. Led by Principal Investigator Claudia M. Valverde Morales, MD, the trial aims to evaluate the safety and anti-tumor activity of OMO-103 in 10 patients with advanced osteosarcoma who have already undergone one or two treatments.

Patients will receive weekly intravenous infusions of OMO-103 and will be monitored for progression-free survival at 16 weeks. The trial opened for enrollment in December 2024 and has a planned recruitment period of 18 months.

A Collaborative Effort Led by the OSI

This groundbreaking trial was made possible by the OSI’s leadership and its collaboration with Peptomyc and VHIO. Recognizing the potential of OMO-103, the OSI moved quickly to seize this unique opportunity.

“This drug represents a major step forward in targeting one of the most elusive drivers of cancer.” — Laura Soucek, PhD

Typically, initiating a Phase 2 trial for a rare disease can take four years or more. Thanks to the OSI’s swift action, the study launched in just 25 months, underscoring the institute’s commitment to accelerating research for osteosarcoma patients. We would like to acknowledge OSI SAB members William Tap, MD; Katherine Janeway, MD, MMSc; Brian Crompton, MD; Lara Davis, MD; and Chand Khanna, DVM, PhD, for their expert contributions to the development of this clinical trial.

The trial is sponsored by the OSI and supported in collaboration with Curing Kids Cancer, The Morgan Adams Foundation, and The Kristen Ann Carr Fund, further underscoring the collective effort behind this initiative.

“Getting this trial off the ground has been a true team effort,” Dr. Helman says. “It is a testament to what can be achieved when researchers, clinicians, and organizations come together with a shared goal: improving outcomes for patients.”

Looking Ahead

While researchers are hopeful about OMO-103’s potential, they also see this trial as an opportunity to gather crucial data on MYC’s role in osteosarcoma.

“Science is about pushing boundaries,” Dr. Soucek says. “This trial is not just about testing a drug — it is about opening new doors for osteosarcoma patients and potentially reshaping how we approach cancer treatment.”

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