What's the holdup to advancing promising autism drug?

May 28--Parents of autistic children who have read my recent article on suramin (j.mp/autismucsd) are asking me when the next clinical trial will begin, and how their children can apply to get into it.

Here's my best answers:

1: When enough money is raised to fund that trial.

Suramin research for autism is funded through private philanthropy. Groups that have supported the research to date include the William Wright Family Foundation; the Christini Fund; the Autism Research Institute; the Lennox Foundation; the Gupta Family and Satya Fund; and the N of One: Autism Research Foundation.

The Jane Botsford Johnson Foundation provided support for the essential pre-clinical studies and equipment used in the suramin trial.

2: Check the website of Robert Naviaux at naviauxlab.ucsd.edu.

Naviaux is the University of California San Diego researcher who identified suramin's potential for autism.

And if you haven't done so already, please closely read the Q and A with Naviaux. It's online at j.mp/autismqanda.

The Q and A also describes how suramin could be useful for those with genetic conditions that produce autism-like symptoms, such as Fragile X and Angelman syndromes.

The study itself is available free at j.mp/autismsuramin.

Good news for parents before I explain further: According to the preliminary evidence, the drug appears to help children as old as 14.

And while small, the trial on 10 boys was double-blinded and placebo-controlled, so neither parents nor clinicians knew who got the real drug. This is the gold standard for clinical studies.

Could children older than 14 benefit? Possibly, but it's too soon to tell. So as tough as it is, waiting years more for a scientifically valid test doesn't mean it's too late for your child to benefit.

Suramin appears to restart a more normal pattern of neurological development, Naviaux says. If that's true, the brains of people with autism are a lot more plastic than originally thought.

And even without suramin, if your child is middle-to-high-functioning on the spectrum, he or she could not only become self-sufficient, but in some cases a major contributor to society. Being on the autism spectrum can be a gift, even if at times it's a well-disguised one.

Here's a story I wrote about a remarkable woman on the spectrum, Temple Grandin, and the advice she gives to parents.

Grandin received help not only from therapy, but from mentors and a mother who challenged her to go beyond her comfort zone.

The key is to help children learn necessary social skills, while encouraging them explore their natural interests in a way that results in a a career.

If a child grows up to be a socially awkward but financially self-sufficient software coder, to use a stereotype, that's a good outcome, Grandin says.

Sound science, however ...

Now to some depressing news. Drug companies, which normally fund clinical testing, aren't paying in this case. That's because suramin's story isn't the normal one.

It's not a new drug, protected by patents that guarantee a drug company marketing exclusivity. It's a century-old drug, introduced in 1916 to treat sleeping sickness.

Naviaux has been working with suramin for years. After he identified the proposed mechanism of action, removing a danger signal that impedes cell-to-cell communication, Naviaux and colleagues tested it in autism model mice. A single dose of suramin reversed their symptoms.

But most drugs that work in animals don't work in people. So even this tiny clinical trial's indication of potential benefit makes it significant. Even more importantly, the treated boys didn't experience any major bad reactions. So there's no show-stopper to prevent further testing.

Naviaux identified suramin's potential by searching databases to find drugs he hypothesized could relieve an underlying cause of autism. It's a lot easier to get a new approval for a drug than to get the original approval, because the risks and benefits of an already approved drug have been documented.

Other researchers have turned to data mining of already approved drugs, of which there are thousands. It makes excellent medical sense: Why go through the risk and expense of inventing a new drug if an existing drug does what you want it to do?

But good medicine isn't always financially feasible. Since suramin's patent has long expired, drugmakers have no incentive to spend millions of dollars to find a new use for a generic drug that their competitors could also sell.

An additional reason for drug companies to stay away is that the early trial could have been a fluke. Suramin might not provide enough benefit, or turn out to have major safety problems when studied in larger numbers of children. It's also possible that even if Suramin provides the benefits seen in the early-stage trial, that they are transient.

Right now, we can't rule out any of these possibilities.

The only sure thing is that testing suramin will cost a lot of money. That puts everyone concerned with finding better treatments for autism in a bind.

Dangers and ethics

Doctors have the authority to prescribe drugs for off-label uses if justified in their medical judgment. But in the case of suramin, that would raise ethical issues.

Suramin has toxicity risks, so it needs to be given by experts. For a dangerous disease like sleeping sickness, the risks are acceptable. For autism, the risk-benefit ratio has yet to be determined.

For that reason, Naviaux implores parents not to risk their children's health on the basis of preliminary evidence that requires further confirmation.

And that's how things will stand, until someone or some coalition of groups raises enough money to take the story of suramin in autism another step forward.

bradley.fikes@sduniontribune.com

(619) 293-1020

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