Gene-Based Therapy That Reprograms Immune Cells Offers Hope For Multiple Myeloma Cure


A gene-based therapy that re-engineers immune cells offers hope to patients suffering from a type of blood cancer known as multiple myeloma.

Multiple Myeloma

The United States has more than 30,000 cases of multiple myeloma occurring each year. The condition affects the plasma cells that produce antibodies that fight infection.

Between 60,000 and 70,000 American currently have the disease. Only about half of these patients are expected to live five years after their diagnosis.

Now, a gene-based therapy offers hope to patients suffering from this type of blood cancer. In an early trial conducted in China, researchers found that genetically tuning the immune cells of a person to target cancer appears to give long-lasting protection against the disease.

CAR T-Cell Therapy For Multiple Myeloma

The treatment dubbed CAR T-cell therapy, resulted in 33 out of 35 patients suffering from multiple myeloma to experience significant reduction in their cancer, or even enter full remission.

Researchers hailed the results of the trial as impressive. American Cancer Society deputy chief medical officer Len Lichtenfeld said that the patients involved in the study had their disease return with prior treatment and all reported to have had some form of meaningful response to the therapy.

The new therapy is tailored for individual patients. Doctors gather samples of the patient's T-cells, one of the main cell types of the immune system, and then genetically reprogram these to target and attack the abnormal multiple myeloma cells.

Researchers compared the process to fitting immune cells with a GPS that points them to cancer cells, making these reprogrammed immune cells better at targeting the abnormal cells.

The therapy is promising because the reprogrammed T-cells will roost in the patient's body, where they will multiply and provide long-term protection. The T-cells are supposed to attack the tumor and then continue to grow to become the body's long-term monitoring and treatment system.

Cancer experts described the therapy as a next step forward in immunotherapy for cancer. Immunotherapy offers hope to cancer patients that do not respond to standard chemotherapies.

"I can't say we may get a cure but at least we bring hope of that possibility," said Frank Fan, of Nanjing Legend Biotech, which tested the treatment with doctors at Xi'an Jiaotong University.

Treatment For Lymphoma And Lymphocytic Leukemia

It is not the first time that CAR T-cell therapy has been used for serious illnesses. It has already been used as treatment for lymphoma and lymphocytic leukemia. Researchers decided to try the therapy for multiple myeloma re-engineering the patients' T-cells and then reintroducing them to the body in three infusions that were done in just a period of one week.

The result of the trial has been promising, but 85 percent of the patients experienced a potentially dangerous side effect called cytokine release syndrome (CRS). It is characterized by low blood pressure, fever, breathing difficulties, and impaired organ function. Most of the patients though experienced only transient symptoms.

The findings were presented at the American Society of Clinical Oncology annual meeting, in Chicago on Monday, June 5.

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