Mediaplanet: What innovations are you most excited about emerging in the space of treatment for blood cancer?

Mounzer Agha: The most exciting aspect of the emerging new treatments for blood cancer are the targeted therapies. Unlike the standard chemotherapeutic drugs that do not distinguish between normal and cancer cells in terms of their effects, the new targeted therapies kills the cancer cells specifically, while sparing the normal cells, thus causing far less treatment related side-effects, while exerting very high and specific anti-cancer results.

Steven Grant: The emerging field of precision medicine as it relates to blood cancer is particularly exciting, as genetic aberrations that cause various hematologic malignancies have been discovered and therapies specifically targeting these molecular abnormalities have been developed. The implication is that further understanding of these genetic changes may permit therapies tailored to patients’ cancer-causing gene defects. This concept was initially validated in chronic myelogenous leukemia (CML) decades ago with the identification of the Philadelphia chromosome translocation responsible for the development of the oncoprotein (cancer-promoting protein) that causes this disease, but the concept is only now being extended to other hematologic malignancies. The CML example serves as a prototype for such targeted forms of therapy.

But because cancer is in most cases caused by multiple genetic abnormalities, it is likely that rational combination approaches to therapy will be essential for more durable and long-lasting responses. For this reason, drug combinations have been the focus of my lab’s research. For more than a decade, we have been working to identify compensatory pathways that allow cancer cells to avoid the otherwise lethal consequences of inhibition of a single pathway and have developed combinations of targeted agents designed to shut down these escape pathways.

John Huber: New drugs that have been developed and new treatments using combination therapies have produced positive results.

MP:  Where do see the future of treatment in blood cancer five years from now?

MA: For many types of blood cancer, we will no longer be using cytotoxic chemotherapy, and we will moving toward using several active oral targeted therapy that patients can receive at home with little side-effects, and they can continue working and maintaining as close to a normal of a lifestyle as possible.

SG: I think there will be many more agents available that target the specific genetic aberrations responsible for various blood cancers. There will also be more rational combination strategies that selectively disrupt cancer cell survival pathways. In a more general sense, there will be a considerably broader range of treatment options for patients.

JH: As we learn more about genetic factors and genomics, diagnosis and treatment will become more targeted and personalized.

MP: What advice can you give our readers who may be supporting someone or may themselves be fighting blood cancer?

MA: I advise my patients and their families to focus on themselves and their lifestyles, rather than the disease itself. There are many effective treatments for blood cancers today, and the question is how can we maintain the best quality of life while beating the cancer. I would advise them to review with their physicians, the different treatment choices that would give them the best outcome for their cancer and the best quality of life simultaneously.

SG: Be open to participating in clinical trials because they provide access to the most promising new therapies that are scientifically driven and help make treatment advances possible. Become familiar with the availability of clinical trials in your area, including at major medical centers that collaborate with the National Cancer Institute and the broader academic community and particularly trials that test for genetic aberrations.

JH: Learn as much about your disease as you can from reputable sources. Select members of your health care team who want to have you as an active participant in your care. Be sure you have assembled a strong personal support team to help you. You don’t need to tackle this alone.

MP: What is the most important thing you would stress to readers about advocacy for blood cancer awareness?

MA: I would love to see patients focusing on beating their disease, while offering a helping hand to future patients. They can do that by participating in clinical trials. While each person will have unknown benefit personally when they participate in a clinical trial, they will definitely help scores of future generations of patients. It is really crucial to help support the clinical research trials, which are always designed to answer important questions about current treatments and discover potentially very active new cancer drugs.

SG: Make clear to your legislative representatives that you strongly support government funding for blood cancer research. Support of research is sorely needed—there have been many new and exciting discoveries in blood cancers whose potential is being held back because of government cutbacks in research funding. Opportunities that could save and improve lives must be pursued vigorously.

JH: Although they may be rare, blood cancers are very serious, life threatening diseases. Just because a disease may be rare doesn’t mean it’s not important or serious.

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