Bone Cancer: Rare Disease Taking a Share of the Biotech Spotlight

Bone cancer is rare, making up just one percent of all cancer cases. Despite advances in treatment for other cancers, the standard of care for primary bone cancers have remained largely unchanged for the past 40 years. In the past five years, however, encouraging research developments and industry investments have signaled a shift that will allow bone cancer to take a larger share of the biotechnology spotlight.

Treating a Rare Cancer

Primary bone cancer starts in a bone, developing on the surface, in the outer layer, or from the center. As a tumor grows, cancer cells multiply and destroy the bone, and if left untreated, the cancer can spread to other parts of the body.

The most common types of bone cancer are osteosarcoma, which occurs most often in children and young adults, in the bones of the leg or arm, chondrosarcoma, which occurs in the pelvis, legs or arms in middle-aged and older adults, and Ewing sarcoma which arises in the pelvis, legs or arms of children and young adults.

Despite being the most common form of bone cancer, osteosarcoma is considered an extremely rare disease, with around 1,000 Americans diagnosed each year, and nearly half of those under the age of 20, according to the American Cancer Society. Osteosarcoma is aggressive. In 25% of patients, osteosarcoma spreads to the lungs, and survival rates are low.

The FDA has approved nine drugs for bone cancers, including Amgen’s XGEVA® and Novartis’ Zometa®, but the majority of these are indicated for secondary bone cancer – cancer that has spread to the bones – or multiple myeloma, a bone marrow cancer. There is a significant unmet medical need for effective treatments for primary bone cancers.

Revolutionizing Research and Treatment

Engineered bone marrow (eBM) is a groundbreaking material that has the potential to advance effective osteosarcoma and primary bone cancer treatments. While previously researchers were limited to studying osteosarcoma in flat, artificial cultures, or mouse models, eBM offers a life-like medium to grow osteosarcoma cells and observe the impact of treatments on these cells, opening doors for new discoveries.

eBM was developed by an interdisciplinary team of researchers from UC Davis and Lawrence Livermore National Laboratory, who published a study describing eBM’s potential in the Proceedings of the National Academy of Sciences.

An Attractive Investment Opportunity

The global bone cancer treatment market was valued at $1.18 billion in 2022 and is expected to grow at a compound annual growth rate (CAGR) of 4.59% from 2023 to 2030. According to Grandview Research, key factors propelling the growth of the bone cancer treatment market include government efforts, the growing incidence of cancer, and a surge in product approvals and launches.

In September 2023, the FDA approved GSK’s (NYSE: GSK) Ojjaara (momelotinib) for the treatment of myelofibrosis, a type of bone marrow cancer. GSK purchased Sierra Oncology in 2022, to acquire the drug, in a deal valued at nearly $2 billion. It’s an attractive space for the pharmaceutical companies that are successful. Incyte’s (Nasdaq: INCY) Jakafi, a treatment for myelofibrosis, generated net revenues of $2.6 billion for the company in FY 2023, and Bristol Myers Squibb’s Inrebic, also for myelofibrosis, generated $19 million in revenue for the company in Q4 2023.

Investing in Bone Cancer Research

The pace may be slow, but investments are being made in bone cancer drug discovery and development by pharmaceutical and biotech companies. In February 2024, Australian company, Telix Pharmaceuticals (ASX: TLX) announced it would acquire QSAM Biosciences (OTC: QSAM) a company developing therapeutic radiopharmaceuticals for primary and metastatic bone cancer, including osteosarcoma therapy. The deal is valued at $33.1 million, with an additional $90 million in contingent payments upon achievement of certain clinical and commercial milestones.

There is a significant unmet need for new research into bone cancer, to allow for the development of diagnostic tools and targeted therapies to improve treatment outcomes.