Novel System to Get Dying Patients an Experimental Cancer Drug Raises Hopes — and Thorny Questions

Excerpt:

“The drug was still experimental, but clinical trials suggested it could be a lifesaver for patients with a lethal form of blood cancer called multiple myeloma.

“And those patients were clamoring to get it. They overwhelmed drug maker Janssen Pharmaceuticals with requests for the medication.

“Most companies don’t know how to handle such requests. Often, it’s the richest patients, or the best connected, or those who run the most compelling social media campaigns who end up getting the drug. Everyone else is out of luck.”

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New Ways to Treat Solid Tumours

Editor’s note: Recent research has uncovered a potential new way to fight some cancer types. The key is a protein called Eph3A, which is made by the cells of blood cancers and solid tumors. Researchers made a new drug called KB004 to target and kill cells with Eph3A. The drug is currently being tested in a clinical trial with volunteer leukemia patients.

“An international team of scientists has shown that an antibody against the protein EphA3, found in the micro-environment of solid cancers, has anti-tumour effects.

“As EphA3 is present in normal organs only during embryonic development but is expressed in blood cancers and in solid tumours, this antibody-based approach may be a suitable candidate treatment for solid tumours…

“Currently, KaloBios Pharmaceuticals is testing the anti-EphA3 antibody KB004 in a multi-centre Phase I/II clinical trial in Melbourne and the US in patients with EphA3 expressing blood malignancies: AML, MDS and myelofibrosis.”


Cancer Patients Want to Be More Involved in Treatment Decisions

Some cancer patients feel that they do not have enough say in their treatment decisions, a recent survey from the UK reveals. While 70% of respondents felt satisfied with their level of involvement, patients with certain rarer cancer types expressed a wish for more participation in treatment decisions, including those with rectal, ovarian, multiple myeloma, and bladder cancers. The desire for greater involvement was also more common in younger patients and ethnic minorities. While the survey was performed in the UK, similar issues are likely to affect patients in other countries, including the U.S. Several U.S. states have recently introduced legislation to support shared medical decision-making.


Cancer Patients Want to Be More Involved in Treatment Decisions

Some cancer patients feel that they do not have enough say in their treatment decisions, a recent survey from the UK reveals. While 70% of respondents felt satisfied with their level of involvement, patients with certain rarer cancer types expressed a wish for more participation in treatment decisions, including those with rectal, ovarian, multiple myeloma, and bladder cancers. The desire for greater involvement was also more common in younger patients and ethnic minorities. While the survey was performed in the UK, similar issues are likely to affect patients in other countries, including the U.S. Several U.S. states have recently introduced legislation to support shared medical decision-making.


Cancer Patients Want to Be More Involved in Treatment Decisions

Some cancer patients feel that they do not have enough say in their treatment decisions, a recent survey from the UK reveals. While 70% of respondents felt satisfied with their level of involvement, patients with certain rarer cancer types expressed a wish for more participation in treatment decisions, including those with rectal, ovarian, multiple myeloma, and bladder cancers. The desire for greater involvement was also more common in younger patients and ethnic minorities. While the survey was performed in the UK, similar issues are likely to affect patients in other countries, including the U.S. Several U.S. states have recently introduced legislation to support shared medical decision-making.


Targeting the BRAF V600E Mutation in Multiple Myeloma

“In multiple myeloma, there has been little progress in the specific therapeutic targeting of oncogenic mutations. Whole-genome sequencing data have recently revealed that a subset of patients carry an activating mutation (V600E) in the BRAF kinase. To uncover the clinical relevance of this mutation in multiple myeloma, we correlated the mutation status in primary tumor samples from 379 patients with myeloma with disease outcome. We found a significantly higher incidence of extramedullary disease and a shorter overall survival in mutation carriers when compared with controls. Most importantly, we report on a patient with confirmed BRAF V600E mutation and relapsed myeloma with extensive extramedullary disease, refractory to all approved therapeutic options, who has rapidly and durably responded to low doses of the mutation-specific BRAF inhibitor vermurafenib. Collectively, we provide evidence for the development of the BRAF V600E mutation in the context of clonal evolution and describe the prognostic and therapeutic relevance of this targetable mutation.”


Targeting the BRAF V600E Mutation in Multiple Myeloma

“In multiple myeloma, there has been little progress in the specific therapeutic targeting of oncogenic mutations. Whole-genome sequencing data have recently revealed that a subset of patients carry an activating mutation (V600E) in the BRAF kinase. To uncover the clinical relevance of this mutation in multiple myeloma, we correlated the mutation status in primary tumor samples from 379 patients with myeloma with disease outcome. We found a significantly higher incidence of extramedullary disease and a shorter overall survival in mutation carriers when compared with controls. Most importantly, we report on a patient with confirmed BRAF V600E mutation and relapsed myeloma with extensive extramedullary disease, refractory to all approved therapeutic options, who has rapidly and durably responded to low doses of the mutation-specific BRAF inhibitor vermurafenib. Collectively, we provide evidence for the development of the BRAF V600E mutation in the context of clonal evolution and describe the prognostic and therapeutic relevance of this targetable mutation.”


Targeting the BRAF V600E Mutation in Multiple Myeloma

“In multiple myeloma, there has been little progress in the specific therapeutic targeting of oncogenic mutations. Whole-genome sequencing data have recently revealed that a subset of patients carry an activating mutation (V600E) in the BRAF kinase. To uncover the clinical relevance of this mutation in multiple myeloma, we correlated the mutation status in primary tumor samples from 379 patients with myeloma with disease outcome. We found a significantly higher incidence of extramedullary disease and a shorter overall survival in mutation carriers when compared with controls. Most importantly, we report on a patient with confirmed BRAF V600E mutation and relapsed myeloma with extensive extramedullary disease, refractory to all approved therapeutic options, who has rapidly and durably responded to low doses of the mutation-specific BRAF inhibitor vermurafenib. Collectively, we provide evidence for the development of the BRAF V600E mutation in the context of clonal evolution and describe the prognostic and therapeutic relevance of this targetable mutation.”