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Early intervention: How Johnson & Johnson researchers are working to help patients at risk of developing multiple myeloma

A wellness visit to the doctor becomes anything but routine when someone learns that their blood and urine tests reveal a diagnosis of high-risk smoldering multiple myeloma (SMM).

Multiple myeloma (MM) is a rare and incurable blood cancer in which plasma cells develop a mutation that causes them to grow uncontrolled and take over the bone marrow. In 2024, it was estimated that more than 35,000 people would be diagnosed with myeloma in the U.S. alone. In 2020, an estimated 176,000 people worldwide were diagnosed with MM.

Approximately 15 percent of people with myeloma have had SMM which is a malignant precursor disease to MM. Patients with SMM have a condition that alters certain proteins in the blood but doesn’t yet cause clinical symptoms. These patients carry a risk of progressing to active multiple myeloma, with a subgroup considered as high risk for progression. Approximately 50 percent of these high-risk patients progress to active MM within two-three years of diagnosis.

“Physicians are telling us that patients are asking for treatments, and unfortunately, they have to tell them that there are no approved therapies for smoldering multiple myeloma,” said Robyn Dennis, M.D., Senior Medical Director, Oncology, Johnson & Johnson Innovative Medicine. “This diagnosis can put a heavy emotional toll on patients and their healthcare providers.”

Typically, physicians take a “watch and wait” approach, closely monitoring patients with SMM at high risk of progressing to full-blown disease. They look for signs of an increase in abnormal white blood cells in bone marrow and high levels of an abnormal antibody called M protein, which are hallmarks of MM. Signs and symptoms of active MM can include anemia, excessive bleeding, decreased ability to fight infection, damage to kidneys and other organs, and bone pain with increased risk of fractures.

Progression to active MM means life-long treatment, involving complex drug regimens that can include chemotherapy and bone marrow transplant, which can severely impact a person’s quality of life.

“The good news is that times are changing,” said Dr. Dennis. “Clinicians are not asking ‘Should we treat?’, but ‘Who should we treat?’”

An increased understanding of MM and advances in treating it are leading researchers like Dr. Dennis at Johnson & Johnson to pioneer early intervention, before the disease is full-blown, delaying progress of damaging symptoms. Recent study results suggest that patients at high-risk of progressing to MM could benefit from early therapeutic intervention.

“Most people diagnosed with SMM are in their mid-sixties,” said Dr. Dennis. “If we can delay progression for patients with high-risk of developing MM, they may live the rest of their lives without requiring active treatment for active myeloma.”

By pioneering early intervention where no approved treatment is available yet, J&J is following the science to address the unmet need of patients with smoldering multiple myeloma. It is one of many paths that the company is taking to ultimately realize their vision of eliminating cancer.

“Individuals who are diagnosed with high-risk SMM typically feel well and have no symptoms,” said Dr. Dennis. “We want them to be able to have an educated discussion with their provider about options that may help delay progression to symptomatic disease.”