Huntington’s Disease: New Therapeutic Approach

Ädoctors are testing a therapy for the incurable nerve disease. First results lead us to hope that you succeed köcould

How eradicated: In the case of Huntington’s disease nerve cells in the brain to perish

The woman seems to be a bit erratic: When you Speak, your head weighs easily back and forth, the eyebrows dance to a track. If you läuft, irritate your arms a bit, move in a strange way. Nothing clearly indicate würde that the 39-Jäover the years and is terminally ill, that in your brain and to important buttons, perish Central.

The patient from the instructional video suffers from Huntington’s disease, Huntington’s Chorea, or frühere "Veitstanz" called, a hereditary disease. To be concerned, you pass with a 50-percent probability to their children. The result of a nerve dying &ndash is; especially in regions of the brain that coordinate movements. Typically the Suffering in the middle of the age breaks out, führt to Pflegebedüsatisfy and schließto the death. So far, there are only drugs, several symptoms for a period of time relieve, however, none of the zerstödevastating invasions of process stops.

The disease arises

In the case of Huntington’s disease, the Messenger RNA f&uuml is due to a Gendefkts;r the Nerveneiweiß Huntingtin is faulty. The Eiweißfactory according to the erroneous Instructions, a defective Nerveneiweiß . This clumped in the brain cells and zerstört

The hopes of Professor Bernhard Landwehrmeyer erf&uuml should;cases, the ächange. "Möpossible k&ouml way;can patients with our treatment, a normal life führen." The neurologist of the Universitäclinic of Ulm, is currently testing, together with British practitioners of the so-called Antisense-oligonucleotide therapy. The process is already in HD ­Patients tested.

Therapy with a view

The expectations were übeen bertroffen, verküthe study’s leader, Professor Sarah Tabrizi from University College London, became friends in April at the annual Congress of the American Academy für neurology in Los Angeles. Although you have so far, only the Verträemergency, the method überprüft, but häclear evidence of their effectiveness would have already shown up to 60 percent ließ the sickness Eiweiß in the brain of HD patients reduce.

The cause of Nerve cell death in the head: a malformed variant of the Eiweißit Huntingtin. The defective Gene (see graph). The error lässt does not resolve, however, the Antisense-oligonucleotide-­The­therapy can prevent the defective Gene is transcribed. That slows down the formation of toxic cell product.

The Problem is that Huntington’s Patients also produce normal Huntingtin.The Nervenädoctors müshot, so find a compromise between a sufficient reduction of the defective Huntingtin and preservation of its intact version. That seems to be möurgent: "The current Verträurgent study has no serious side effects gezeigt", Bernhard Landwehrmeyer says. "We know from animal experiments that the achieved reduction of sick Eiweißit is not enough to improve the mobility."

The effect of the therapy

The RNA-drug binds to the defective area of the Messenger RNA für Huntingtin. As a result, the Eiwei&szlig can;factory not reading the Messenger RNA. It is not a defective Huntingtin. The nerve cell remains intact

Impact not yet in sight

What are the clinical effects of this in humans entspräche, however, not to say, says Professor Josef Priller of the Berlin Charité. "It is an Analog-to-conclusion, the clinical studies yet to prove müssen", the Huntington’s disease specialist and Deputy Director of the clinic für psychiatry. Nevertheless, the results agreed with him äußhopeful. The europäof medicines Agency has the drug künewly the "Prime"-Status: The Behörde will the researchers in the other studies unterstüetching, a möas possible fast admission to ermöaligned.

However, there are still some questions to klären. Such as whether enough of the Antisense drug in the hauptsäon the take the affected structures of the Großbrain. This gewäto ensure that injected the researchers, the drug their subjects into the spinal canal. A procedure every couple of weeks to be repeated müto suggest, as the effect of the Agent is not permanently anhält. "It is safe to früh to wecken&quot concrete expectations;, qualified Landwehrmeyer, &quot but optimistic nonetheless: ;Our data show that the principle works."

What expert is also confident: Against two other rare hereditary disease of Recently approved Antisense drugs. "If we also have a ursäfrail therapy against Huntington’s hätten, wüthe question of genetic testing v&ouml would;completely new stellen", ergälandwehrmeyer nzt. Yet the wool in the affected Fa­mi­lien, only a minority know if the disease breaks out in them in the course of life. Köyou could have the Ner­ven­­die but bes&auml stop;ße the frühe diagnosis a quite different consequence, and the Affected hähad reason to hope.

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