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Here are some suggested organisations that offer expert advice on special needs.

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Here are some suggested organisations that offer expert advice on special needs.

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Autism drug to be tested on children

49 replies

CFSKate · 14/03/2013 10:38

www.dailymail.co.uk/health/article-2293113/New-drug-help-reverse-autism-tested-children-time-successful-clinical-trials-mice.html

"Preliminary results show that the drug called suramin, which is already used to treat sleeping sickness in Africa, corrects autism-like symptoms in mice.

At the molecular level, it normalises faulty brain connections, cell-to-cell signalling, and metabolic effects thought to underlie the disorder.

Professor Robert Naviaux, co-director of the Mitochondrial and Metabolic Disease Centre at the University of California in San Diego, said: 'Our theory suggests that autism happens because cells get stuck in a defensive metabolic mode and fail to talk to each other normally, which can interfere with brain development and function.

'We used a class of drugs that has been around for almost a century to treat other diseases to block the 'danger' signal in a mouse model, allowing cells to return to normal metabolism and restore cell communication.'
Professor Naviaux and his team believe both genetic and environmental causes of autism can be traced to a sustained cell danger response linked to immunity and inflammation.

'When cells are exposed to classical forms of dangers such as a virus, infection or toxic environmental substance, a defence mechanism is activated,' said the professor.

'This results in changes to metabolism and gene expression (activity) and reduces the communication between neighbouring cells. Simply put, when cells stop talking to each other, children stop talking.'"

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CFSKate · 27/05/2017 13:02

Chasingmytail17 - the problem is money not time.
It says near the end here that if money was no problem it would take 5 years. "As a result of going into debt to fund the study, I've had to dramatically shrink my lab and slow our research progress greatly. We are currently working at only about 20% of our capacity if we actually had adequate funding."

QUESTION: What is the rate-limiting factor to progress right now? Where are the bottlenecks?

ANSWER: The rate-limiting factor is money. Lack of funding has slowed our research progress on the CDR and purinergic signaling in autism for the past nine years. We can’t do the next studies without new funding.

We have plans for five additional studies over the next five years to collect all the data the FDA will need to decide about the approval of suramin for autism. With adequate funding, culminating in a multicenter, phase III, registrational trial, these studies can be completed without further delay.

Usually, the multimillion dollar cost of new drug development is covered by the Big Pharma that will benefit from FDA approval. Unfortunately, since suramin is 100 years old, the usual patent laws don’t apply and the next clinical trials will require grass roots support from families and foundations and other approaches to raise the needed funding.

further up it also says "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 SAT-1 trial."

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