Preliminary Data from Phase 1 Trial for Multiple Myeloma Indicates Complete Response in a Patient

Phase 1 Trial

A Phase 1 trial by Gamida Cell is examining a new potential treatment option for patients with multiple myeloma and relapsed/refractory non-Hodgkin’s lymphoma who have not responded to currently approved treatment options.

The trial hopes to recruit 24 patients who are between the ages of 18 and 70. Eligibility for the lymphoma segment requires that patients produce the CD20 factor. Eligibility for the multiple myeloma segment requires that patients have undergone two lines of therapy previously. This trial will be conducted in Minnesota at the Masonic Cancer Center.

It is investigating the effect of NAM-NK on these two rare diseases.


NAM-NK therapy uses nicotinamide (NAM) to increase the body’s number of natural killer cells (NK). NK cells are vital for fighting cancer however there are too few of them and they die too quickly to be completely effective. The NAM-NK version of the cells live longer, multiply more quickly, and are more effective at combating cancerous cells.

The primary aim of this Phase 1 trial is to determine the highest dose of NAM-NK that non-Hodgkin’s lymphoma and multiple myeloma patients can tolerate. Secondarily, researchers will document how many patients respond to the therapy, the duration of the treatment’s effects, and any potential toxicity.


The preliminary results from this trial include safety data from 14 participants and efficacy data from 12.  These included 8 multiple myeloma patients and 6 lymphoma patients. The data were presented at the 2019 Transplantation & Cellular Therapy Meetings which were held in Houston, Texas February 20th through 24th.

Prior to receiving NAM-NK these patients were given a round of chemotherapy. After the administration of NAM-NK, they received interleukin-2 to help the cells expand. Additionally, both before and after treatment, all multiple myeloma patients received Empliciti and lymphoma patients received Rituxan to help facilitate the effect of the therapy.

Three lymphoma patients achieved complete response and one multiple myeloma patient achieved complete response. Additionally, two myeloma patients reached stable disease. Of the patients who achieved complete response, two were given bone marrow transplants, which could potentially cure them of their condition.

NAM-NK proved to be generally well tolerated. While one patient experienced cytokine release syndrome and one patient died from sepsis, there were no reports of tumor lysis syndrome, neurotoxicity, or graft versus host disease.

Looking Forward

These preliminary results are truly exciting and have reinforced researchers’ belief of this therapies potential.

In addition to this ongoing Phase 1 trial, Gamida has announced that they are now collaborating with Editas Medicine to enhance the therapy’s effects through CRISPR.

You can read more about the Phase 1 trial, its preliminary results, and Gamida’s new collaboration here.

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