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Tuesday, May 30, 2017

Possible Game-Changer for Autistic Kids May Never Happen

‘Game-changer for autism’: 100-year-old drug reverses symptoms, study finds

The drug Suramin could be used to alleviate the symptoms of autism © Veri Sanovri / Global Look Press

A drug discovered more than 100 years ago may hold the key to combating autism symptoms, according to a study.

Researcher Dr Robert Naviaux of the San Diego School of Medicine gave suramin, a drug first developed in 1916, to 10 autistic boys between the ages of five and 14, and noted transformative results.

"After the single dose, it was almost like a roadblock had been released," he said. “If the future studies show that there’s continued health benefits, this could be a game-changer for families with autism.”

The study, which has been published in the Annals of Clinical and Translational Neurology, saw five of the participants receive suramin, while the remainder were given placebos. Included in the group were four non-verbal children – two six year olds and two 14 year olds. 

“The six year old and the 14 year old who received suramin said the first sentences of their lives about one week after the single suramin infusion,” Naviaux told the UC San Diego Health website. “This did not happen in any of the children given the placebo.”

The Centers for Disease Control and Prevention estimates that about 1 in 68 children are affected by autism – which is more than four times more common among boys. The causes of autism, however, are not yet fully understood.

Research has shown that cells harden their membranes in response to attacks from viruses or pollutants. The reaction, known as ‘cellular danger response’ (CDR), is a common defense mechanism that allows cells to wait for danger to pass. Autism is thought to develop during early childhood when cells can become ‘stuck’ in this mode.

Dr Naviaux believes that suramin can ‘un-stick’ the cells by inhibiting the signal they release when they sense danger, which can help normalize the response.

One parent, whose son had not spoken a full sentence in more than a decade, said: “Within an hour after the infusion, he started to make more eye contact with the doctor and nurses in the room. There was a new calmness at times, but also more emotion at other times.” 

“He started to show an interest in playing hide-and-seek with his 16-year-old brother. He started practicing making new sounds around the house. He started seeking out his dad more.”

Suramin was originally developed as a cure for sleeping sickness, a parasitic disease spread by the tsetse fly in sub-Saharan Africa.

First tested on mice in 2013, this is the first time suramin has been administered to children.

For Naviaux, the challenge now is to widen his research to a bigger sample testing size. “This work is new and this type of clinical trial is expensive,” he said. “We did not have enough funding to do a larger study. And even with the funding we were able to raise, we had to go $500,000 in debt to complete the trial.”

Time for a change

The problem here is obvious. 100 year old drugs cannot be patented and therefore can be made very inexpensively. The profits for big pharma will not likely ever exceed the extraordinary costs of trials. This is the the huge elephant in the small room of health care. 

The way the system works is that there are no advances in medication that don't cost exorbitant amounts of money. Cheap and easy solutions to many diseases go by the boards because no-one is willing to fund the trials needed for their acceptance.

Governments, all of which are fighting rapidly increasing costs of drugs and health care, have to make some hard decisions. They either have to find new and cheaper and quicker ways to do clinical trials, or they have to fund those trials for medications that will reduce costs for both drugs and healthcare if those trials are successful.

Governments can benefit from funding such trials as this by lowering the costs of drugs which will make them more affordable for patients meaning more patients who need the drugs will get the drugs and consequently be in better health and require less in terms of medical care and, in many cases, social care. Improvement in quality of life can be immeasurable. 

It seems like a no-brainer to me. But I think the trick would be to create a resource pool funded by most countries in the world, perhaps through the WHO. It would not be onerous if many countries contributed and it would be far more beneficial than some international programs currently running.

Of course, big pharma would be mad as hell, but I'm OK with that!