Thursday, September 16, 2010

Autism drug has some promise

This is a very poorly-conducted study: Small sample, naive or absent taxonomy, no control groups and subjective assessment. Similar results could perhaps be obtained from a simple sedative or a placebo. The results seem however to warrant the setting up of a proper trial

A drug for autism has been hailed as the first treatment that could work against the condition. In a clinical trial, researchers found that it eased many of the distressing symptoms, helping sufferers improve their social skills and reduce tantrums. They were also able to make eye contact more frequently and became less irritable.

It is the first time the drug has been successfully tested on patients. And although it is many years away from being available here, the scientist believe their work could pave the way for treatments.

Researcher Dr Craig Erickson, from the Indiana University School of Medicine, who helped run the trial, explained: ‘We observed marked improvement in the majority of patients treated in the study, including reductions in agitation and tantrums. 'This work will potentially open up a door to treating disorders that has, until recently, been firmly shut.’

About one in 100 in the UK are diagnosed with an autism disorder. The severity ranges dramatically, but all sufferers experience problems with communication, imagination and their social relationships.

Although doctors prescribe anti-depressants and anti-psychotics for particular symptoms, there are no specialist autism drugs.

The new drug, Arbaclofen, is intended to rebalance the brain chemistry of those with autism. It was tested on 25 children with the condition aged between six and 17, over eight weeks. They suffered few side effects and by the end of the trial were calmer and more sociable. ‘We observed marked improvement in the majority of patients treated in the study, including reductions in agitation and tantrums'

They made eye contact more easily and were less anxious than at the start.

One teenager who took part in the trial was agitated at the start and was unable to stay in the room with the researchers for more than a few minutes. However, by the end, the patient was writing notes to the scientists and seemed less anxious and less aggressive.

Previous studies have shown that people living with the condition produce too much of the brain chemical glutamate which excites brain cells. Some may also make too little of another neurotransmitter called gamme amino butyric acid, New Scientist magazine reports today.

Dr Randall Carpenter, of Seaside Therapeutics in Cambridge, Massachusetts, which has developed the drug, said: ‘We are trying to normalise signalling functions within the brain. ‘Too much activation with glutamate makes people with autism very sensitive to loud noises and other sudden changes in the environment, increasing anxiety and fear.’

Arbaclofen ‘may stop them being oversensitive’, Dr Carpenter added. The results of the trial were so promising, that a larger-scale test is planned. The results have not been published in a medical journal.

Autism charities welcomed the trial, but stressed that the number of people taking part was small. The results could also be biased because the drug was not compared to a placebo, a harmless ‘dummy’ drug. And the assessment of the children was subjective, meaning that they could be misinterpreted.

Amanda Batten, of the National Autistic Society, said: ‘As the nature of autism is so complex, many interventions have been tried and tested over the years, but what works for one person won’t necessarily work for another. ‘Further rigorous research is required into potential interventions, such as Arbaclofen, to properly understand and assess the impact that they could have on people’s lives.’


Melanoma breakthrough?

A double-blind trial would obviously be ethically impossible here, given the rapid progression of the tumours, but such a trial on an animal model would seem desirable. The two patients who got better could have been spontaneous remissions, which are not at all unknown with cancer. About a third of my skin cancers (BCCs and SCCs) vanish without treatment -- JR

IN WHAT is arguably the most significant use of genetic knowledge to tackle disease, scientists in California have created a drug that prevents the effects of a gene mutation linked to malignant melanoma.

The London Daily Telegraph reports that researchers have built on advances made by the human genome project to develop a drug that specifically targets the deadly cancer.

In a small clinical trial, melanoma tumors shrank by nearly a third in 24 of 32 patients with a mutation known as B-RAF - and in two other people, the tumors disappeared completely - according to the research, published in the journal Nature.

Plexxikon, the company behind the the drug - preliminarily called PLX4032 - has also begun working on a test to determine whether individual melanoma sufferers have the mutation and thus would respond to the treatment.

While the drug's long-term effects are unknown, and it does cause unwanted side effects, its initial results gave hope to scientists and cancer patients alike that specific chemicals can be used to target specific diseases.

Sir Mark Walport, director of UK cancer charity the Wellcome Trust, said that the breakthrough was a "penicillin moment" for cancer researchers. However, he added: "We have got to balance the hype and the hope. Cancer is complicated."


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