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Multiple MyelomaFebruary 2021Summary reviewed October 2026

73% of Hard-to-Treat Blood Cancer Patients Responded to One-Time Gene Therapy

In patients with multiple myeloma that stopped responding to standard treatments, a one-time gene therapy infusion shrank tumors in nearly 3 out of 4 people. One in three patients had their cancer disappear completely. Most effects were blood count drops and flu-like symptoms that doctors could manage.

What the trial was testing

The KARMMA enrolled 149 patients with multiple myeloma. The study was sponsored by Celgene and tracked outcomes across the full group of patients who matched the trial's eligibility profile.

It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.

What the results showed

73% of patients with hard-to-treat multiple myeloma responded to this one-time gene therapy.

The New England journal of medicine · 2021 · NCT03361748

These findings — that tumors shrank or disappeared in nearly 3 out of 4 people with hard-to-treat blood cancer — were published in the The New England journal of medicine and represent the headline result of the study.

Researchers tracked outcomes across 149 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.

What this means for patients

For patients with multiple myeloma, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.

What you can do now

This treatment is now FDA-approved and available for multiple myeloma that has come back after at least three other treatments. It uses your own immune cells, reprogrammed in a lab to find and destroy cancer. You get one infusion, not ongoing chemo. Ask your cancer team if this option fits your situation.

Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.