Drug for multiple myeloma approved: the rate of 'cancer-free' patients doubled

Good news for multiple myeloma patients: The FDA (U.S. Food and Drug Administration) has recently granted accelerated approval to a new drug for the treatment of the disease. The new drug joins a series of innovations that have fundamentally changed the treatment of myeloma and the prognosis for patients in recent years.

The drug, iberdomide, developed by Bristol Myers Squibb, will be marketed under the name Zanvexus and is intended for adults who have received at least one line of therapy.

"The new treatment targets a disease where vital cells of the immune system themselves become the source of damage. Multiple myeloma is a malignancy of plasma cells that develops in the bone marrow," explains Dr. Julia Vaksman, Director of the Myeloma and Amyloidosis Service in the Hematology Department at the Davidoff Center in Beilinson.

According to Dr. Vaksman, plasma cells belong to the immune system, and in multiple myeloma, they divide uncontrollably and fill the bone marrow, which is the blood cell production factory. The disease can cause kidney failure, high calcium levels, anemia, bone lesions, and fractures. She notes that myeloma treatment has undergone a revolution in recent years. In the past, it was based on steroids and chemotherapy, and life expectancy was short. Today, treatment is mainly based on a variety of biological therapies, which have led to a significant improvement in survival and quality of life.

Breaking down target proteins

Despite the improvement, in most cases, it is still not possible to cure the disease completely. It may return after a period of remission, and sometimes stops responding to drugs that helped initially. According to the American Cancer Society, about 36,000 new cases of multiple myeloma are expected to be detected in the United States in 2026, and about 10,850 people are expected to die from it. In Israel, about 650 new myeloma patients are diagnosed annually.

The new drug is not given alone, but in combination with daratumumab (under the trade name Darzalex by Janssen, a Johnson & Johnson company) and the steroid dexamethasone. Another advantage is patient convenience: the new drug is given in a pill that can be taken at home.

Drugs in this family utilize the cell's natural protein degradation system. Iberdomide binds to a protein called cereblon, which is part of a cellular system that marks certain proteins for degradation. The link between the drug and the protein changes which proteins the system recognizes and marks, including proteins that myeloma cells need to survive and multiply. This is the first time a drug from the CELMoD (Cereblon-Modulating Protein Degraders) family has been approved.

Study with the participation of 13 patients from Israel

The approval was based on a phase 3 study in which 939 patients with relapsed or refractory myeloma participated. 13 patients from Israel also participated in the study: six from Sheba, three from Hadassah, two from Rambam, one from Sourasky (Ichilov), and one from HaEmek Medical Center.

The data on which the approval was based included 420 patients. Of the 207 patients who received the new combination, 41% achieved a complete response, and no disease cells were found in a highly sensitive bone marrow test, compared to 21% in the control group. The difference, measured after a median follow-up of 16 months, was statistically significant.

The test is designed to detect minimal residual disease (MRD). While the absence of MRD is considered a deep response, it does not prove that all cancer cells have disappeared or that the patient is cured. The study is still ongoing, and mature results showing whether the treatment delays disease progression or extends life have not yet been published.

Risks and side effects

Dr. Vaksman emphasizes that alongside the convenience of treatment, risks must be taken into account. "We must be aware of the toxicities associated with this treatment, such as infections and a decrease in blood count, which is an important side effect. The treatment increases the risk of blood clots, so preventive measures must be taken."

Indeed, safety data shows that 90.2% of patients reported a decrease in neutrophils, and 40% experienced serious infections. The drug carries a serious warning against harm to the fetus and blood clots. It is forbidden for use during pregnancy and will be distributed in the United States as part of a limited program requiring monitoring. The timeline for the drug's arrival in Israel is not yet clear.