Clinical Trial Updates – For Patients

Last update:  March 2018 


Luspatercept is a new drug for β-thalassaemia patients that is currently under investigation. This drug, which is administered by a subcutaneous injection, increases the haemoglobin level and reduces the need for blood transfusions. This is accomplished by promoting the maturation of red blood cell1. The first clinical trials2 in a small number of patients showed promising results and larger trials are currently ongoing to further document the efficacy and safety of this drug.

Another similar drug with the same mechanism of action that is also under investigation is sotatercept.

LentiGlobin is a novel form of gene therapy that is currently under investigation for the treatment of β-thalassaemia patients. Gene therapy aims at curing β-thalassaemia by substituting the affected genes responsible for the disease with functioning ones. This is accomplished by transferring the functioning genes into the patient’s bone marrow3 using a viral vector. LentiGlobin is a new form of a viral vector that has hitherto shown promising results and further studies on this therapy are ongoing. This therapy has also been tested on a small number of patients with severe sickle cell disease.

Ruxolitinib is a drug that regulates the expression of the gene responsible for the synthesis of haemoglobin and is currently used for the treatment of other blood diseases. This drug has been tested on a small number of β-thalassemia patients and seems to accomplish an increase in haemoglobin level with a reduction in the size of the spleen. Thus, this drug may be an alternative to splenectomy4, an operation that is often required in thalassaemia patients. Further clinical trials are, however, required.

INTERCEPT is a new system for the processing of the blood to be transfused. This system seems to enhance the safety of blood transfusions by reducing the risk of transmission of infections and perhaps the risk of reactions to transfusions, as shown by the first clinical trials. Further testing of this system is ongoing.

Amlodipine is a common drug that is widely used for the treatment of hypertension. This drug may prevent the entering of iron into the heart cells. In the first trials in transfusion-dependent thalassaemia patients, the drug, when used in combination with iron chelation therapy, was more effective than iron chelation alone in reducing iron in the heart. Further trials are now ongoing.

Crizanlizumab is a new drug currently under investigation for the prevention of painful crises in patients with sickle cell disease. This drug seems to prevent the occlusion of small blood vessels caused by the irregularly shaped red blood cells of these patients, an event that is responsible for painful crises and several other complications of the disease.

Hepcidin5 mimetic peptides appear promising in the treatment of different haematological and metabolic diseases in which hepcidin levels are abnormally low.

Now undergoing clinical trials:

PTG-300 LJPC-401 M012

1Red blood cell: the cells of the blood that contain haemoglobin, which is responsible for the transportation of oxygen throughout the body.

2Clinical trial: the testing of a new drug or other forms of therapy in a certain number of patients under well-controlled conditions before this therapy is licensed for widespread use.

3Bone marrow: the part of the bones responsible for the production of the blood.

4Spelenctomy: the surgical removal of the spleen. 

Hepcidin is a hormone produced primarily by the liver and is responsible for regulating the absorption of iron and its circulation into the bloodstream. In thalassaemia,  hepcidin production is reduced due to iron overload. Therefore, by providing hepcidin, the iron load will be better controlled, but also total Hb may be improved since hepcidin and iron have a role in the production of red cells. This means that the process of RBC production will be more effective.

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