New research shows promise in blood cancer treatment
Monday, March 24, 2014
Blood cancers pose many challenges for healthcare professionals engaged in clinical research, patient care and treatment. Several new approaches published recently show promise for the future in this field of medicine.
One approach for treating leukemia, discovered by a team in Montreal, disarms a gene that is responsible for tumor progression. By targeting the gene, known as Brg1, in leukemia stem cells, researchers think this may offer new therapeutic opportunities by preventing the disease from coming back.
Several new drugs are also on the horizon. Idelalisib, taken orally, has been shown to target and then block a form of the PI3 kinase enzyme. This enzyme is critical for the activation and survival of cancerous B cells.
Because this drug narrows the targeting of this enzyme, it has significant promise for cancers that form in the B-cell pathway, such as chronic lymphocytic leukemia (CLL), indolent non-Hodgkin lymphoma (iNHL) and mantle cell lymphoma (MCL).
A breakthrough for treating a rare type of blood cancer used oral vemurafenib, a BRAF inhibitor originally approved for treatment of malignant melanoma. Vemurafenib was found to be successful in treating hairy cell leukemia (HCL). While the study in the U.K. was only in one patient, the results were promising. The malignant cells were cleared from the patient's blood and led to a complete clinical recovery in only a number of days.
Three independent studies, at Memorial Sloan Kettering, the NCI and Children's Hospital of Philadelphia have evaluated the use of genetically-modified T cells for the treatment of blood-borne cancers. In a large clinical study at Sloan Kettering, 88 percent of patients with advance leukemia achieved complete remission after treatment with genetically altered versions of their own T cells.
At NCI, two clinical studies were conducted. In the first trial, 15 patients with the most common type of non-Hodgkin lymphoma were treated with their own modified T cells. Six patients achieved complete remission, and six exhibited partial remissions.
In the second study, researchers treated patients with leukemia and lymphoma who did not respond to stem-cell transplant donors. In that study, 3 of 10 patients showed significant disease regression, one with complete remission. Another significant finding that emerged from this study was that no patients experienced graft-versus-host disease (GVHD).
In an early pilot study in Pennsylvania of children and adults with a highly aggressive form of acute lymphoblastic leukemia (ALL), 18 of 24 patients had shown ongoing complete remission approximately three months after treatment.
As with other immunotherapy studies, this approach gave "hunter" bioengineered T cells to patients who had high-risk ALL that recurred after initial treatment or resisted treatment from the start. Three and a half years later, results were published for a total of 59 patients enrolled in the study.
Forty-seven percent of the adult patients responded to therapy with seven patients going into complete remission. Eighty-six percent of pediatric patients were in complete remission, and all five of the first adults in the study have to date achieved complete remissions.
The use of oral treatments of blood cancers and novel approaches such as bioengineering a patient's own T-cells is leading the way to less chemotherapy and a more targeted personal, individualized approach to treating these diseases.
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