New Treatment helps CML Patients

New treatment recommendations will help standardise diagnosis and management of Chronic Myelogenous leukaemia  (CML) patients

The South African Medical Journal (SAMJ) will be publishing new Clinical Practice Recommendations for the treatment of Chronic Myelogenous Leukaemia (CML) in November 2011. Designed to help standardise the diagnosis and management of CML, the recommendations have been welcomed by experts as a way of ensuring healthcare providers and medical aid case managers are up-to-date regarding the latest generation of targeted therapies.

“Scientific advancements have significantly increased the survival rates of CML patients in the last five to 10 years and we need to make sure we keep everyone abreast of these exciting developments,” explains Professor Vernon Louw, Head of the Clinical Haematology Unit in the Department of Internal Medicine at the University of the Free State and lead author of the new recommendations.

A particularly aggressive form of blood cancer, CML represents nearly 15% of all leukaemias, affecting around 2 out of 100 000 people each year across the world[1], It is no longer the death sentence it once was, and most patients achieve excellent responses with dramatic improvements in survival. “Exciting new therapies offer patients a range of treatments, enabling many with the condition to live long, healthy lives,” Professor Louw enthuses.

Foremost among the therapies offering patients renewed hope are targeted molecular drugs and stem cell transplants. “Contemporary treatments, such as targeted therapies and stem cell transplantation, have dramatically improved patient survival rates,” confirms Professor Louw. “In the case of CML, for example, 85% of patients now survive more than eight years on treatment.”

New therapies have changed everything

“Targeted therapies, which focus on the cancer cells at a molecular level, have changed everything,” Professor Louw maintains. “These breakthrough drugs can kill off cancerous cells in a matter of months, compared to the years taken by traditional chemotherapy. The average time for a newly diagnosed patient to get to a haematological remission is now just one month.”

Not only do these new therapies work much faster than traditional chemotherapy, their side effects are far less severe. Instead of indiscriminately destroying all rapidly dividing cells, good and bad, they mainly disrupt the functioning of cancerous cells. “In fact, one of the challenges is that patients tend to feel so much better that they stop taking the therapy , as they think that they are cured which leads to resistance developing,” observes Professor Louw.

Second generation targeted therapies have been developed that are even swifter and more precise in attacking cancer cells. “Two second generation TKIs (Tyrosine-Kinase Inhibitors) have been tested in Phase III clinical trials,” reports Professor Louw.”

Helping healthcare providers and medical aids

The imminent availability of these second generation targeted therapies prompted Professor Louw and his colleagues to draft the updated recommendations. “The only downside to these rapid therapeutic developments is that it can prove difficult for healthcare providers and medical aid schemes to keep up,” he explains. “Our hope is that these new recommendations will help prevent the misdiagnosis and mismanagement of CML patients.”

Two areas Professor Louw highlights in particular are patient monitoring and the management of side effects. “Monitoring is critical because disease outcome is directly related to making the right dose adjustments according to response levels that need to be reached within very specific time periods,” he explains, “and healthcare providers need to act quickly, otherwise the patient can become very resistant to treatment.”

It’s a similar situation regarding potential side effects. “There are a number of these and most are easy to manage, but, if it doesn’t happen correctly, patients stop their treatment or their doctors take them off the medication and all of the benefits are lost,” explains Professor Louw.

The professor and his colleagues also hope that medical aid case managers will adopt the new recommendations and work them into their protocols. “There appears to be a lack of understanding of the efficacies of these new targeted therapies,” he notes, “and we hope the new recommendations will help medical aids establish where they fit into the treatment paradigm.”

Helping patients to access new therapies

Urging all interested parties to get hold of the new recommendations and follow them, Professor Louw concludes with a reminder that, as a result of the rapid developments in targeted therapies, there are numerous clinical trials running currently that could help patients.

“Just about every unit in the country is running some form of clinical trial and this can be a great way of helping your patients to solve funding issues or to get access to new therapies that might otherwise only be available in other countries at the moment,” he advises. “A great place to start is the clinicaltrials.gov website, which has a database of all registered trials worldwide.”