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Myeloid neoplasias

Lead researcher: Dr. Lurdes Zamora

Why do we need to investigate?

What most of these diseases have in common is a poor prognosis because the drugs currently available are, in many cases, inefficacious. Furthermore, many patients can not undergo a bone marrow transplant, which is, at present, the existing possibility for a cure, on account of their advanced age or physical condition, given the aggressive nature of the treatment. Consequently, many people currently have no effective curative therapy available.

Sometimes it is the case that there is an efficacious drug available, as is the case with chronic myeloid leukaemia, but there is a considerable percentage of patients who do not respond to treatment, or who suffer very significant side effects, and this could be avoided if we could offer alternative medicines. At the moment, however, no tests exist that might help us to differentiate these patients at the outset.

We need research to find alternative, personalised treatments that would produce positive and long-lasting responses from patients at minimum levels of toxicity.

The impact of our work

Dr. Zamora, together with other experts in clinical hematology, leads a research team that focuses its work on two main lines of investigation:

1. To find an alternative treatment for chronic myeloid leukaemia

Chronic myeloid leukaemia accounts for 15-20% of all leukaemias, with around 700 new cases diagnosed in our country every year. At the present time, some 15-20% of patients treated with Imatinib, a first line drug to combat the disease, present adverse effects or fail to respond. In some cases, these patients may have a genetic alteration that influences the absorption of the drug, but in others we do not know why they fail to respond to treatment.

The team's work is directed at finding an answer to this question in order to be able to provide the best treatment for each patient and prolong their life expectancy. Also, working on the project are other renowned clinical hematologists such as Dr. Xicoy, Dr. Boqué and Dr. Fernández, amongst others.

2. To provide a therapeutic opportunity to a group of high-risk patients

Patients with high-risk myelodysplastic syndrome and those with acute myeloid leukaemia with multilineage dysplasia do not, at present, have many therapeutic options available to them. They tend to be patients of advanced age or who have another associated pathology and are not, therefore, suitable candidates for a bone marrow transplant. There is a drug that is administered but half of the patients treated do not respond, and many of those that do, stop responding sooner or later. Dr. Zamorra's team is studying the genetic mutations of these patients to find out whether they are related to their response to this drug or not.

The aim of the study is to find a more suitable treatment for every patient to achieve a better and more prolonged response.

This project is being carried out under the auspices of the Cooperative Acute and Myelodysplastic Leukaemias Study and Treatment Group (CETLAM), in which hematologists from a number of Spanish hospitals are cooperating, under the clinical coordination of Dr. Bargay.

3. The impact of our work

The IJC's myeloid neoplasias research group aims to find patients' own genetic characteristics that will make it possible to provide a more personalised treatment with better response results and fewer side effects.

The ultimate aim would be to prolong, and improve the quality of, patients' lives, avoid all the deaths associated with treatment, reduce side effects to a minimum while, at the same time, ensuring no diminution in the effectiveness of treatment.

Josep Carreras Leukaemia Research Institute Dossier

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For more information, the following web page can be consulted:

Josep Carreras Leukaemia Research Institute - Genetics and Epigenetics in Myeloid Neoplasms

Webpage updated 10/11/2017 15:24:13