Rare blood cancers: Progress continues

05/25/2022 – Pharmaceutical research does not forget rare and very rare diseases. More and more of those affected can benefit from medical progress in this way – for example in Waldenstrom’s disease, a very rare cancer of the blood.

The best that can be said about this disease is: Waldenstrom’s disease is very rare. Around 7 people are affected for every 1 million men; in women there are 4. The disease, discovered in 1944, belongs to the group of non-Hodgkin’s lymphomas. Due to gene mutations, the B cells in the blood change so that degenerated cells can multiply. The bone marrow is also affected. Those affected initially notice little that they are ill, because Waldenstrom’s disease is one of the so-called indolent, i.e. painless, but progressive diseases. In the later course, the disease manifests itself as general weakness (fatigue), loss of appetite, neuropathy or symptoms such as fever, night sweats and weight loss. It is incurable and is usually diagnosed incidentally – usually between the ages of 63 and 75. The average lifespan after diagnosis is around 7 years.

Waldenstrom’s disease: treatment options are important

Doctors now have a wide range of therapies available to treat this very rare disease. The treatment options depend on the age and state of health of the patient – and whether the disease is initially treated or has already relapsed, i.e. whether it has recurred after treatment has been completed.

It is good when doctors have therapy options available whose effectiveness and safety are constantly being further developed. In Waldenstrom’s disease, in addition to monoclonal antibodies and immune-modulating agents, these are targeted therapies such as BTKi. They are a very effective therapy option for Waldenstrom’s disease. In some patients, however, the treatment has to be stopped due to adverse drug effects (e.g. atrial fibrillation).

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A new generation of targeted therapies

This is where the latest generation of BTKi comes in: It has a more specific effect, i.e. more precisely, which is apparently the reason for a more favorable side effect profile. So-called off-target effects are avoided – in this case the inhibition of signal cascades that should not be inhibited at all. During the G-BA hearing, Managing Director Martin Völkl from the research company BeiGene reported on the experiences from the approval study: “The patient quickly feels that he is doing better and that his disease is under control again. The response lasts for a long time, and this with an improved side effect profile compared to previous therapy options.” Nevertheless, this therapy also has side effects; the most common are neutropenia (decrease in white blood cell count), upper respiratory tract infection, and diarrhea.

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