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Hereditary diseases: what will happen to the European gene therapy

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Hereditary diseases: what will happen to the European gene therapy

01:44
Today
Telegraph

likar.info

Photo: likar.info

Science news:Gene therapy is a promising treatment for serious diseases. In 1989 passed the first clinical trials: but what is the situation with gene therapy in Europe now?

First of all let’s understand what is a gene therapy. A method of treatment of hereditary genetic diseases by introducing healthy genes the patient is missing or corrupted. In recent years, scientists have learned finally to effectively deliver a foreign gene to target cells, and provide him there for a long period of operation.

THE GENE THERAPY

The EU is currently approved for marketing two drugs for gene therapy (before it was only used in China). One of them is the drug Glybera, developed by Dutch biotech company UniQureм for the treatment of lipoprotein lipase deficiency (this is a very rare genetic disease) .


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In this disease due to a mutant gene of the enzyme lipoprotein lipase is not produced by the body, which can lead to blockage of the blood vessels.

Sales of Glybera in Europe began in 2015. For the full course of treatment need to pay about 1.1 million euros (one vial costs 53 thousand euros). During this time, the Old Continent only one person treated with this drug, although the number of potential patients reached 200 people. In General, it is not in demand.

This state of Affairs is not satisfied with the company UniQureм, and it intends shortly to cease sales of Glybera in Europe.

The second drug is for gene therapy, which is now sold in the EU – Strimvelis developed by GlaxoSmithKline for the treatment of severe combined immunodeficiency (ADA-SCID), which is most common in children. This genetic disease is characterized by impaired production of body adenozindezaminazy, which it accumulates toxic substance deoxyadenosine destroying cells-lymphocytes, which reduces the immune system.


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Every year in Europe recorded 15 new cases of the disease ADA-SCID. This, of course, very little, but because treatment is inaccessible because of their great value. E. to provide effective care for these patients is almost impossible.

The European medicines Agency has allowed the use of Strimvelis in 2016. For the full course of treatment need to pay about 700 million euros. Currently in Europe two people have undergone gene therapy with this drug. At the end of the current year two more people would be exposed to treatment.

If Glybera everything is clear, the future Strimvelis in Europe are as yet uncertain. The fact that the company GlaxoSmithKline no longer wants to produce drugs to treat rare diseases. She plans to sell all the patents on them. So it is possible that Strimvelis also eventually stop selling on the Old Continent.


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“I am surprised that only three people in Europe have undergone gene therapy. This suggests the existence of certain problems, the pharmaceutical companies and the European health systems funded by the state. Such expensive treatment, many patients state health care to pay for just can’t,” said Hilary Thomas, a medical consultant to audit firm KPMG.

Thus, in Europe, gene therapy is because of its high cost, not demand. Pharmaceutical companies even want to abandon the sales of the relevant drugs on the European market.

However, not all that bad. Some experts say, the main thing that is already beginning to use this method of treatment of genetic diseases. Now it is time for companies and laboratories that will be able to develop more affordable medicines.

WHAT DOES THE FUTURE OF GENE THERAPY IN EUROPE?

In the 1990-ies there was a lot of experimentation in the delivery of healthy genes to damaged cells. Scientists stimulated the idea of using for this purpose the engineering of viruses. But then because of several deaths and cancer the experiments stopped. Years passed before scientists returned to these studies. At the moment it is already known how to make viral delivery of effective and safe.

The future of gene therapy in Europe, in our view, depends on two factors.


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As this method of treatment of rare genetic diseases and cancer will manifest itself in the United States. In this country, while this method of therapy has not one approved. But soon there should be approved several new medical preparations intended for gene therapy.

For example, next month the us government can allow the use of genetically engineered cell therapy against leukemia (this is a variant of standard gene therapy), which was developed by Novartis. In early 2018 in the United States can approve a gene therapy company Spark Therapeutics (it is supposed to help people with a rare genetic form of blindness).


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Whether a pharmaceutical company developing gene therapy, to negotiate with insurers. This method of treatment is very expensive. The EU cannot afford to frequently use gene therapy because of its high cost. In the US, pharmaceutical companies and insurance companies have long been actively negotiated for the cost of such treatment when it is approved. While discussing several models of payment such treatment in the American market, but the final decision is still pending.

Now scientists are conducting clinical trials of gene therapy, approximately 40-50 different diseases. The vast majority of them – is a rare genetic disease. However, there is hope that soon with the help of gene therapy will be possible to treat, e.g., Alzheimer’s, diabetes, heart failure and cancer. Geneticist George Church of Harvard University is also confident that using this method it will be possible to fight the effects of aging of the human body.

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