Multiple myeloma (MM) is a cancer of the plasma cells and is incurable in most cases. Therefore, new therapies are urgently needed.

The B-cell maturation antigen (BCMA) is a protein expressed by both normal and malignant plasma cells.

Because BCMA is only expressed by plasma cells and a small fraction of B-cells, it is a promising target for treating multiple myeloma.

In this Phase I clinical trial, presented at ASH 2015, researchers extracted immune T cells from patients and genetically engineered the cells to express an anti-BCMA chimeric antigen receptor (CAR-BCMA) to recognise and kill the myeloma cells.

Patients received one round of chemotherapy before their own engineered cells were infused back into their bodies at one of four dose levels.

As of November 2015, 11 patients with advanced MM and a median of seven previous failed therapies have participated in the trial.

One month following infusion, the two patients treated at the highest dose level demonstrated the strongest anti-cancer responses.

One patient achieved a stringent complete remission at two months following the CAR-BCMA T cell infusion.

The other patient had undetectable myeloma in the bone marrow plasma cells but has not yet reached complete remission status.

Of the six patients treated on the lowest two dose levels, one patient experienced a short partial remission of two weeks and the other five remained stable, their disease neither improving nor worsening.

Two patients on the second-highest dose level maintained stable disease, and one patient obtained a very good partial response.

Toxicity and side effects were mild for patients who received the lowest dose levels.

Patients who received the highest doses experienced cytokine release syndrome, a severe and potentially fatal side effect of therapy characterised by high fever, muscle pain, and heart and kidney problems.

Engineered CAR-BCMA T cells were detected in the blood of all 10 patients assessed to date.

Toxicities were similar to those observed in leukaemia patients treated with similar therapies.

These findings suggest that CAR-BCMA is a promising option for advanced multiple myeloma patients who have failed several previous therapies.

The findings were presented by Dr James Kochenderfer from the National Cancer Institute in Bethesda, USA.

Watch the video interview for more.

Source: ASH