Why a rare blood cancer has become a testing ground for cancer drug innovation

Some of the most ambitious cancer science is happening in a disease few outside of oncology can name, and it’s revealing a future where cancer is no longer a death sentence.

Multiple myeloma (MM) is a blood cancer that affects the body’s plasma cells, causing them to mutate and grow uncontrollably in the bone marrow. These plasma cells produce abnormal antibodies called “M-proteins” that wreak havoc on the immune system, kidneys, and bones.

MM is also relatively rare. While breast cancer saw approximately 370,000 new cases in the U.S. in 2024, multiple myeloma only saw about 31,600, per internal data from Evaluate. Even among blood cancers, MM only makes up 10% of cases.

Yet, despite its rarity, multiple myeloma has become a springboard for some of the most advanced drug innovations in cancer, fostering the development of advanced gene therapies and antibody technologies. While new breakthroughs extend MM patients’ lifespans, treatment for the disease is exposing a new reality of navigating cancer as a chronic, high-cost condition.

MYELOMA’S MANY INNOVATIONS

Despite only making up less than 2% of cancer diagnoses, multiple myeloma has seen an array of breakthrough treatments that have more than doubled its five-year relative survival rate since 1990. These include:

• Stem cell transplants, which use a patient’s own stem cells to rebuild healthy bone marrow after intense chemotherapy targeting cancer in the bone marrow itself

• Proteasome inhibitors, which kill myeloma cells by interfering with the normal break down of proteins

• Immunomodulatory drugs (“IMiDs”), which stimulate the immune system and eliminate proteins essential for the survival of myeloma cells

• Chimeric antigen receptor T cells (“CAR-Ts”), which custom-engineer a patient’s own T cells to target and destroy malignant plasma cells

• Bispecific antibodies (“bispecifics”), which redirect T cells to kill malignant plasma cells by bringing the two cells in close proximity

• Monoclonal antibodies, which precisely target specific proteins on cancer cells or in the tumor environment, and are behind some of the most promising survivability gains

Though not the first cancer to see some of these novel treatments, MM is one of the first diseases to achieve approval for CAR-Ts, bispecifics, and monoclonal antibodies all at the same time. Its unique attributes unlock innovation, driving $24 billion in U.S. drug sales in 2024 alone, per Evaluate internal data.

MM’S UNIQUE ADVANTAGES

Multiple myeloma has four key advantages that make it a “sandbox” for cancer innovation.

1. MM has a high survival rate. Non-small cell lung cancer (NSCLC)—one of the most common cancers—has a five-year survival rate of 32% across all stages; multiple myeloma’s is 62%, with a median patient survival length of 8–10 years. Longer survival creates a bigger window to offer multiple treatments.

• Multiple myeloma comes with its own “accelerometer.” The M-protein biomarker rises and falls quickly, offering a clear sign of the cancer’s progression and response to treatment. Consequently, trials to develop new multiple myeloma medications show efficacy faster, so are less expensive than those for most other cancer drugs.

• MM can be almost completely eradicated from the body. In 2024, MM became the first cancer for which the FDA endorsed minimum residual disease as a surrogate goal or “endpoint” for drug trials, which qualifies many MM drugs for “breakthrough therapy” designation or accelerated approval. Multiple myeloma received approval for seven new drugs between 2020 and 2022, including multiple first-in-class therapies. This surge in innovation, rarely seen in oncology, is likely to continue now that minimal residual disease is accepted as a surrogate endpoint.

• The incentives for developers to create new MM drugs are strong. Unlike other rare diseases whose treatments only incrementally increase survival, MM treatments drastically improve length and quality of life. The latest combination of drugs being tested to treat MM has been modeled to increase survival without disease progression by a full decade among healthier patients. As a result, MM patients are more likely to spend on high-cost drug regimens to keep the disease at bay, further funding research.

Multiple myeloma’s ability to foster innovation drives comparatively lofty drug sales for its rarity. Evaluate’s internal data shows that, in 2024, non-small cell lung cancer (NSCLC) saw about six times more new cases than MM, yet NSCLC drugs only brought in a third more revenue than MM drugs did that year.

Drugmakers are looking to re-create MM’s success in other cancers, but its innovations have proven difficult to reproduce.

REPLICATING MM’S ADVANCEMENTS

Multiple myeloma drugs have proven more effective because they can directly reach the disease’s liquid tumors within the bloodstream. In contrast, other cancers’ solid tumors are embedded in dense, immunosuppressive tissue environments that can be difficult for drugs to access. Furthermore, solid tumors often lack good cell surface targets that differentiate them from surrounding healthy cells.

Many companies have tried to apply MM’s breakthroughs to other cancers and a few are beginning to see success in solid tumors. Gilead’s CAR-T therapy is showing promise in shrinking deadly brain cancer tumors, while bispecifics are beginning to show promise in treating small cell lung cancer.

The trick for scientists now is to take the base-level innovations that have worked in MM and apply them with more specified targeting, delivery, and immune activation to other cancers. Meanwhile, the treatment innovations MM patients enjoy also bring new, unintended consequences.

CANCER’S CHRONIC FUTURE

Multiple myeloma reveals a future where cancer patients not only live longer but also shoulder continual treatment expenses.

A study examining Medicare claims from 2006–2016 found the average lifetime treatment costs for MM were $184,495, and out-of-pocket costs have only increased since then. As a result, one in four patients with MM experience financial hardship due to treatment costs, causing some to skip doses or delay care.

Despite already high treatment costs, myeloma therapies continue to increase in price. Evaluate’s internal data reveals that Medicare coinsurance patients can expect to pay $105,700 out of pocket for cutting-edge CAR-T Carvykti. However, even the price for the most widely prescribed MM drug, Revlimid, has increased 17% in the last four years, notwithstanding cheaper, generic versions of the drug beginning to roll out in 2022.

To make long-term cancer survival a viable reality for more patients, drugmakers will have to iterate on MM’s playbook while U.S. hospital systems and insurance companies make innovations more accessible at scale.

In the meantime, we can glimpse into the future of cancer treatment in multiple myeloma, where breakthroughs are making long-term survival the new normal. Soon, MM’s innovations will spur discoveries that extend the lives of all cancer patients.

https://www.fastcompany.com/91388585/why-a-rare-blood-cancer-has-become-a-testing-ground-for-cancer-drug-innovation-cancer-drug-innovation?partner=rss&utm_source=rss&utm_medium=feed&utm_campaign=rss+fastcompany&utm_content=rss