A Dad's Journey

Father of Autistic Twins Speaks Out

Browsing Posts in cure for autism

Three groups control 99% of the money, and novel studies have a hard time getting funding.

 By

We still don’t know what autism is, despite decades of research and billions of dollars spent. We don’t know what causes it or how to treat it. This lack of progress is partly the result of structural deficiencies in how autism research is funded. Fortunately, lessons from financial markets and the venture-capital industry can help solve these problems and accelerate the pace of discovery—for autism and perhaps other medical conditions.

Consider recent research by Robert Naviaux, a professor at the University of California, San Diego. Earlier this year he announced results from a clinical trial involving 10 boys with autism. Half were given the drug suramin and showed significantly improved language and social behavior. The study lends further support to Dr. Naviaux’s theory that a treatable metabolic condition may underlie autism. This promising lead is welcome news, but it reinforces my view that the scientific understanding of autism is years, possibly decades, behind where it would be if the handful of groups that control virtually all funding for autism research had taken a more-diversified approach.

In finance, markets that are deep—made up of many investors with varying opinions—are more efficient and better at price discovery. Similarly in science, many “investors” funding multiple approaches in parallel should lead to more-rapid advances. Therein lies the problem. In the U.S. just three organizations control 99% of all funding for biomedical research on autism: the federal government (primarily the National Institutes of Health); Autism Speaks (which does commendable work raising awareness); and a large foundation funded by a family. Everyone else collectively makes up less than 1% of funding.

These three organizations almost exclusively support research that aligns with the conventional view of autism as primarily a genetic disorder of brain wiring. The problem is that this “genetics-first” paradigm does not fit the emerging research, including Dr. Naviaux’s, and has failed to produce answers. Research that does not fit neatly within this view—or that dares to contradict it—has little chance of being funded.

PHOTO: ISTOCK/GETTY IMAGES

Case in point: None of the three organizations have supported Dr. Naviaux’s recent research or the clinical trial, even after he successfully reversed autism-like behaviors in multiple mouse models. Thankfully, a grass-roots effort by parents and small nonprofits, including the one I run, was able to supply most of the funding. For the rest, Dr. Naviaux went into debt.

A similar story is what led me to start a nonprofit in 2014. While trying to understand my son’s unexpected improvement in autism symptoms while taking a common antibiotic, I was surprised to discover results from a clinical trial published 15 years before. In that study, 8 of 10 boys with severe autism showed significant improvements while taking the antibiotic vancomycin. I met with the researchers years later to find out why they had not followed up on this novel, intriguing finding. They all said the same thing: They could not get funding because their results did not fit the established paradigm.

Portfolio theory teaches that diversification reduces risk, but there is little diversification in autism research funding. In finance, the risk is of capital loss or increased volatility; in autism the risk is a continued epidemic robbing children of their childhood and the prospect of an independent life. Beyond the personal toll, the economic costs of autism in the U.S. have been estimated at between $300 billion and $500 billion a year. Our lack of answers has a high price tag.

As Dr. Naviaux’s recent success shows, radical ideas have great value in science, but to be proven, they need to get funded. At the NIH, grant proposals are scored by small committees of prior grant recipients, a system that virtually enshrines the status quo. Incrementalism, at best, is the result. Playing it safe almost never produces breakthroughs. I’m sure the people on those committees would like to see progress as much as I would. The problem is not intent, but structure.

What we need is for the “market” that allocates capital to medical research to more closely resemble the risk-taking financial and venture-capital markets. Researchers should be rewarded for stretching beyond conventional views in search of breakthroughs. The obvious need is for more funders with adequate capital and diverse views. This could be fostered by formally combining the power of the venture-capital model with the passion of the medical nonprofit, but that will take time.

Meanwhile, some stopgaps may help. To ensure that the NIH and other government agencies diversify their autism research efforts, I propose a hard cap—say, no more than 25%—on how much of their grants can go to genetics-related studies, thus mandating diversification.

Another approach would be to start something akin to what Israel’s Directorate of Military Intelligence created to counter groupthink: an office of “devil’s advocate,” staffed by analysts whose job is to identify and challenge conventional points of view. At the NIH this group could fund studies that run counter to the prevailing paradigm.

This is the approach that the nonprofit I founded, N of One: Autism Research Foundation, takes by committing its limited funds to small studies that buck the conventional view in the hopes of seeding a breakthrough. In finance, we call it seed venture capital. It’s time we apply the lessons and approaches of a system that works to one that has not.

Mr. Rodakis is founder and president of the nonprofit N of One: Autism Research Foundation, which supported Dr. Naviaux’s suramin study.

 Appeared in the September 29, 2017, print edition.

This article was just published in Scientific American.  It points to the great progress that scientists have made in mapping the genes that lead to autism.

It also points to the fact that the more we know, the more we don’t know.

At some point in the not too distant future, it may be possible for kids to hbig dataave a genetic correction to mitigate the affects of autism.  Probably not for another 20 years, but progress is being made.

https://www.scientificamerican.com/article/using-big-data-to-hack-autism/

“If we ever saw a self-correcting defect in two mutations in autism,” Wigler says, “I would stand up and cheer.”

The curious connection between autism and cancer

A surprising number of genes associated with autism also have links to cancer. Does that mean cancer drugs can treat autism?

https://spectrumnews.org/features/deep-dive/the-curious-connection-between-autism-and-cancer/

pathways_spot-2__revision_2

In the movie, All About Bob, one of the core lines is the idea of baby steps.  You need to start somewhere.  In the area of understanding autism we are building on baby steps.  Alone those lines is this piece of research which looks to better understand what’s going wrong with the neuron connectors in autistic kids.  And perhaps, someday soon, there may be a way to mitigate the traffic jam:

Activity between the cells improved when researchers added IGF-1, a growth promoting protein known to enhance connections between neurons.

Read more:  http://www.upi.com/Health_News/2016/07/08/Stem-cell-reprogramming-allows-scientists-to-model-autism-development/6741467977370/ Stem-cell-reprogramming-allows-scientists-to-model-autism-development (1)

Is the title of a documentary describing a young autistic man who defined his life through Disney Animated films.   He had retreated into a world and one day asked for help to get out. life, animated

Life, Animated, which takes its name from a 2014 book by Ron Suskind, begins and ends with Owen as he is today: a young man of 23, living on his own, working, having relationships. Between those happy bookends, though, is a story of heartache, frustration, childhood cruelty and one very significant, secular miracle.

Blocking specific cells during pregnancy can help prevent the disorder developing

  • Mice exposed to high IL-17a levels in the womb exhibited autism symptoms
  • This is a signalling protein that boosts how a body fends off infections 
  • Blocking related cells restored normal structure to the brains of the pups 
  • This was regardless of whether this was achieved by treatment with antibodies or by shutting down the IL-17a gene completely

By VICTORIA WOOLLASTON FOR MAILONLINE

Read more: http://www.dailymail.co.uk/sciencetech/article-3421329/Have-scientists-cure-autism-Blocking-specific-cells-pregnancy-help-prevent-disorder-developing.html#ixzz3yeCk1e4v
Follow us: @MailOnline on Twitter | DailyMail on Facebook

Parents can learn how to give effective therapy to their children with autism, a new study in the Journal of Child Psychology and Psychiatry finds.

Researchers at Stanford University looked at a type of therapy called Pivotal Response Training (PRT), which is one the of the handful of treatments shown to be effective for kids on the autism spectrum, says Kari Berquist, PhD, study co-author and a clinical instructor in psychiatry and behavioral sciences and an autism clinician at Lucile Packard Children’s Hospital Stanford. The therapy focuses on improving kids’ motivation language skills by reinforcing their use of language related to the task at hand. One of the advantages is it can be done anywhere: anytime a child attempts to ask for something by name—a toy, say—they’d be rewarded with the item they requested, which reinforces their use of language.

well, there may be something there, but for the time being, it’s just something to follow.  First of all, you need to get extremely high doses of  sulphoraphane into your child.  And for those who had a positive effect, it went away after a month.  Still, the idea of stressing the body (akin to a high fever) may lead to something…

https://www.yahoo.com/health/broccoli-may-hold-the-secret-to-improving-autism-99994821267.html

is it selfish of me to say I only wish this article had been posted 14 years sooner?

 Autism research is in a race against time. For years, researchers have sought to screen for symptoms of autism spectrum disorder (ASD) at younger ages, and enroll children in behavioral therapy earlier and earlier. A new study published Tuesday pushed the age limits of the research with a small number of extremely young infants, some as young as six months old. By the time the children turned four, having received therapy administered entirely by their parents, almost none of them required any form of autism services.

http://www.newsweek.com/autism-therapy-6-month-old-babies-eliminates-symptoms-limited-study-269155

 

 

Interesting and positive story, because the closer we get to understanding the triggers, the closer we are to mitigating the symptoms of autism.

Shedding new light on brain function in autism, a new study suggests that there is an oversupply of synapses in at least some parts of the brains of children with autism, and that the brain’s ability to thin out the number of synapses is compromised.

The finding provides clues to how autism develops from childhood on, and might help explain some symptoms like oversensitivity to noise or social experiences, as well as why many people with autism also have epileptic seizures. It could also help scientists in the search for treatments, if they can develop safe therapies to fix the system the brain uses to clear extra synapses.  http://www.nytimes.com/2014/08/22/health/brains-of-autistic-children-have-too-many-synapses-study-suggests.html?_r=0

The study, published Thursday in the journal Neuron, involved tissue from brains of children and adolescents who had died from ages 2 to 20. About half of the children had autism; the others did not.