Huntington, UK, Professor discussed on The Science Hour

The Science Hour


There is no cure for huntington's but the niece this week concerns and experimental drug injected into a patients spinal fluid that might halt its progression that important step hasn't been proven yet but the early stage trials conducted at nine centers in the uk germany and canada seemed to show that patients can safely be given the monthly injections as rotor barca professor of clinical neuroscience at the university of cambridge talked claudia hammond it's a trial ready a feasibility and taller than it is what we call was could safety is always very difficult to judge when you have such few a numbers of patients but in essence what people have tried to do in this trial of which we've been appalled is they have injected into the fluid around the spine and the brian cycled csa drug which interacts with the message between the gene and the protein so a gene cage for a protein in this process by which that happens and what this does this socalled and he sends is it recognizes specifically the protein huntington binds to it and then takes it away so the idea is actually stops the gene from expressing the protein which causes the disease so how much do we know so far about whether that actually happens whether it actually works so what we know from the trial in the press release which came out yesterday which goes so much excitement was we know that this procedure of injecting this into the spinal fluid seems very well tolerated that no major safety concerns or problems with patients running into side effects and the measure of whether it's engaging with what it said to be doing i reducing the level of this protein has been chained to be the case namely ihd's reducing the level of the protein what we don't know is whether that makes any difference to the patient in terms of how quickly the disease progresses in that individual presumably we'd expect it to make a difference i mean jimmy that's what we'll be child next but it is making a difference to that pertain you would expect it to they make a difference to the patient symptoms can you would expect it to make a difference but there are thus bys are to caveats that right.

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