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BEHANDLING-God og treffsikker diagnostikk, behandling og rehabilitering

Precision therapy versus standard therapy in Acute Myeloid Leukaemia and Myelodysplastic syndrome in elderly (PALM) – a randomized trial

Alternative title: Presisjonsterapi versus standard behandling ved akutt myelogen leukemi og myelodysplastisk syndrom hos eldre (PALM) - en randomisert studie

Awarded: NOK 12.0 mill.

Acute myeloid leukemia (AML) and severe myelodysplastic syndrome (MDS) in the elderly have poor prognosis because the patients do not tolerate curative treatment with high doses of chemotherapy or stem cell transplantation. Patients therefore receive treatment with minor side effects that can prolong life, but which cannot cure the disease. The health authorities want more personalized cancer treatment. Personalized treatment can be effective and with less side effects than standard treatment and is therefore well suited for the elderly. It is unknown whether personalized treatment is better than standard treatment for AML/MDS in the elderly. In this project we will tailor treatment based on DNA analysis of the cancer cells, therefore patients will receive different treatment depending on the results of the DNA analysis. We will compare with standard treatment where everyone receive the same treatment. We will randomize 60 elderly patients with AML/MDS between standard treatment and personalized treatment to see if personalized treatment is better than standard treatment. We will also make a health economic assessment of the two treatment options, and we will look at molecular mechanisms that may explain why some patients with MDS develop AML and others do not. The results of the study will determine whether elderly with AML/MDS in the future is treated with a personalized strategy or with standard treatment as today. The study could make it easier to provide personalized cancer treatment for AML/MDS within the current Norwegian health system. The study protocol is now written and the study has been approved by SLV, REK and by the privacy protection officer at Ahus. An electronic case-report form has been created, the tumor board has been established and the diagnostic infrastructure is in place. The study is open and has started inclusion of patients.

Acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS) in the elderly are usually not curable. Next generation sequencing of DNA (NGS) can find changes in tumor DNA that can be targeted by new drugs, so called “precision therapy”. Precision therapy is principally different from today’s standard therapy since it will be individualized based on NGS from the tumor, while in standard therapy all patients receive the same treatment. It is unknown how a precision strategy will perform compared with standard therapy in AML and MDS. The objective of this study is to find out if a precision therapy strategy is better or more cost-effective than standard therapy in elderly with AML or MDS. We will do that by randomly allocate patients to standard therapy or NGS based precision therapies following the advice of a Clinical Molecular Board. There are several potential targeted therapies approved in Norway that may have effect in AML and MDS. In addition, we will seek cooperation with pharmaceutical companies to get access to targeted drugs not yet approved in Norway. A possible challenge is that some companies may not be interested in cooperation, the study will then be done with approved drugs and with the drugs from the companies that cooperate. We will also seek additional funding to be able to pay for some drugs within the frame of the study. We will randomize 60 patients in two years of an estimated number of eligible patients of 160. If precision therapy proves to be cost-effective and prolong survival, this study will change the standard treatment of AML and MDS. We will also be able to tell at what price targeted therapy will be cost-effective. If the study does not prolong survival or demands unacceptable resources, it will be an argument against NGS guided therapy in AML and MDS in the elderly.

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BEHANDLING-God og treffsikker diagnostikk, behandling og rehabilitering