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Genetic markers and clinical trials have improved treatment strategies

Professor Patrick Thornton

Consultant Haematologist, Senior Lecturer RCSI, Clinical Director Hermitage Medical Clinic Laboratory, Beaumont Hospital

Chronic lymphocytic leukaemia is the most common leukaemia in the western world, affecting around 1 in 30,000 individuals. Ireland sees around 250 new cases each year.


Chronic lymphocytic leukaemia (CLL) is generally a disease of the over-60s. It is a blood cancer of B lymphocytes, which are white blood cells of the immune system.

“Watch and wait disease”

Many instances often do not require treatment with patients being observed instead. Three quarters of patients eventually require treatment rather than the ‘watch and wait’ approach.

Over the last decade, treatment for CLL has changed due to increasing knowledge of the biology of the disease. Various cancer genetic markers have improved the treatment strategies for CLL, enabling more targeted therapies with fewer side effects. These treatments can be more effective than traditional chemotherapies and less toxic, enabling people who were previously ineligible for treatment to have effective treatment and achieve remission for their leukaemia. These drugs include more targeted antibody therapy and oral medications. These target the cell surface and the cancer cell self-destruct pathways.

Ireland has fallen behind Europe in getting drugs to market

Phase 1,2 and 3 clinical trials must take place before new treatments are licensed worldwide. The USA usually receive drug licensing at a similar time or shortly before Europe. Each European country decides whether patients will receive the drug through a process called reimbursement. Unfortunately, Ireland has fallen behind the rest of Europe. Ireland has some of these licensed but unavailable/not available for their fully licensed indication.

Ireland has fortunately participated in many of the phase 3 trials, which have led to approval of these drugs in Europe. This has enabled Irish patients to benefit from these drugs ahead of their approval and Irish patients have participated in licencing of these drugs world-wide.

These trials reported in the medical press with game changing improvements in survival and remission rates. This is of tremendous value in encouraging the HSE to make these drugs available to all patients. Patients who need them and maintain equal therapeutic opportunity for Irish patients in Europe.

cal press with game changing improvements in survival and remission rates. This is of tremendous value in encouraging the HSE to make these drugs available to all of the patients who need them and maintain equal therapeutic opportunity for Irish patients in Europe.

Personalised medicine and clinical trials

Advances in technology – such as next generation sequencing – have enabled efficient methods to read cancer DNA and define patients into low- and high-risk groups. This will eventually lead to a personalised medicine approach, where patients will be treated with the drugs that are most likely to achieve remission, or even perhaps cure, and not exposed to side effects of treatments that are unlikely to be effective or only effective for a short time.

Through its ongoing participation in CLL trials, Ireland has showcased its ability to perform well in clinical trials and, as a direct result of this, has been co-opted into the current German CLL trial CLL13 (through Cancer Trials Ireland) to fund Ireland’s participation. This trial is testing the combination of all the most modern treatments of CLL, by looking at certain drugs in combination as initial therapy, instead of chemotherapy, there is hope this may even be a cure for CLL.

Communication improves patient pathways

Patients being made aware of the availability of these trials through the media and also support groups is also of extreme importance.The past two years has seen a growing patient focus group called CLL Ireland – the group set up by patients, for patients, to boost awareness of the support network available. It’s an opportunity for patients to learn from and meet others who’ve been living with their leukaemia for many years.

In short, this decade has seen a revolution in knowledge of the disease. Targeted treatments have followed this knowledge and clinical trials have proven their effectiveness. Patients are more aware of their options and appropriately asking about the. Through Ireland’s on-going support of clinical trials, they will keep in step and perhaps find a cure for CLL.

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