Venture Capital and Autism

The Politics of Autism includes an extensive discussion of autism services  

Michael Bernick at Forbes:

Can venture capital drive innovation in the autism field, as it has in other fields? Can the dynamism of capital markets be applied to autism’s stubborn challenges of diagnosis, therapies, services and employment?

In the past few years, several autism-focused venture capital funds have been launched, including Moai Capital, Autism Impact Fund, and Neuvation Ventures. Their founders and chief leaders have family members on the autism spectrum, and come to their funds with a strong sense of mission and urgency. The funds are structured as traditional venture capital funds, aiming to maximize investor returns, but with focus on products for improving the lives of individuals with autism.

Anyone in the autism community today—persons with autism, family members, advocates—will be encouraged to see the innovation already underway in the field. Companies in the venture capital portfolios are rapidly advancing the sciences in diagnostic techniques, early intervention, and therapies/therapeutics to address autism co-morbidities.

Currently the autism diagnosis is a behavioral one—behaviors are observed and evaluated and fit into categories. The process is given to wide variation in interpretations and to misuse. Companies in the venture capital portfolios are developing blood tests and brain imaging tests that can far more effectively give meaning to any autism diagnosis.

The main behavioral intervention today is Applied Behavioral Analysis (ABA). ABA is difficult to access, very uneven in quality of services, and effective with only a limited number of children with autism. Portfolio companies, drawing on speech and language technologies as well as big data insights, are developing alternatives to ABA.

Duke and Stem Cells

In The Politics of Autism, I write:

The conventional wisdom is that any kind of treatment is likely to be less effective as the child gets older, so parents of autistic children usually believe that they are working against the clock. They will not be satisfied with the ambiguities surrounding ABA, nor will they want to wait for some future research finding that might slightly increase its effectiveness. They want results now. Because there are no scientifically-validated drugs for the core symptoms of autism, they look outside the boundaries of mainstream medicine and FDA approval. Studies have found that anywhere from 28 to 54 percent of autistic children receive “complementary and alternative medicine” (CAM), and these numbers probably understate CAM usage.

Anna Merlan at Vice:

For several years, parents of autistic children have paid between $10,000 and $15,000 to have their children undergo unproven stem cell and cord blood treatments at Duke University, through what’s called an expanded access program, or EAP. That practice has attracted criticism from observers and ethicists in the stem cell field, who have asked why Duke was charging money for a service when its own clinical trials have not been very promising. In recent months, Duke has sent letters informing parents that this program is no longer available to autistic children—raising new questions about what those parents, who’d been led to believe the treatment might be a panacea for their kids, will do instead.

One of the more urgent questions is whether parents who can’t access the treatment though Duke will instead go to a for-profit partner with ties to the school. That would be Cryo-Cell International, which previously announced that it had entered into a licensing agreement with Duke allowing it to offer the same stem cell infusions in private, for-profit clinics the company has said it plans to begin opening this year. (Duke previously told Motherboard that “the licensing agreement does not grant Cryo-Cell the use of Duke’s EAP for the treatment of patients at Cryo-Cell, but will allow Cryo-Cell to develop its own cell therapy program.”)

"Growth Market"

The Politics of Autism includes an extensive discussion of autism service providers.  Since the book's publication, a big change has consisted of a massive increase in private equity investments.

Dan Feshbach at Barron's:

The good news is investors are, at last, recognizing that autism is a growth market. The bad news is much of that investment is flowing toward services that don’t scale, often overpromise, and frequently underdeliver. In many ways, the complexity of the autism market reflects the disorder itself. It is an immensely complex condition, consisting of countless overlying subtypes and a wide range of obstacles. It’s not autism we are all working to address, but many forms of autism. Even with our growing understanding of the condition, families remain haunted by four questions: What type of autism does my child have? Why can’t my child communicate? How can my child get a job? Who will care for my child after I pass?

New startups and companies are, at last, starting to find the answers. Innovations in diagnostics and big data are on the verge of allowing families to discover the condition earlier, with companies such as BioRosa working to develop a blood test and biomarker for autism. Floreo and other platforms are tapping the power of virtual reality to teach important communication and interpersonal skills, while startups like Daivergent and Mentra are linking individuals with autism to employers and providing them with support in the workplace. Impruvon Health is offering medication management devices and Advasys is creating tools to help identify sexual abuse. The nonprofit I founded has created a global database that now tracks over 500 technology and life sciences companies within the autism and intellectual or developmental disability space.

Meanwhile, autism and intellectual disability impact investors are creating early-stage funds to support these kinds of young companies. The Disability Opportunity Fund, founded 13 years ago, has recently been joined by the Autism Impact Fund, and then by the Difference Fund, Divergent Ventures, K-Ventures, Enable Ventures, Neuvation, Arc Capital Development, and Moai Capital. These initiatives invest in startups that have met key private investor criteria and provide them with funding, mentoring, and a greater understanding of the market.

Bleach "Cure"

In The Politics of Autism, I discuss autism quackery.  One particularly dangerous "cure" involves bleach.

At KABC-TV, David Ono and Lisa Bartley report:

Eyewitness News teamed up with ABC News to conduct a year-long investigation into a supposed "miracle cure" that's peddled by the so-called "Church of Bleach," which has a chapter in Southern California.

The "miracle cure" is really a form of industrial bleach, but that doesn't stop believers from pushing the potion on the sick, the desperate, and perhaps most alarmingly, the parents of children with autism.
...
Dr. Paul Wang, a pediatrician and the senior vice president of Autism Speaks, a nationally recognized advocacy and support group, said parasites do not cause autism.

"No, parasites do not cause autism," Wang said. "She says that MMS is not a bleach, but it is."

"And frankly, it's a poison. It should not be given to anybody with autism or cancer or diabetes or any other condition that they claim it can treat," he continued.

Michigan Says No to Medical Marijuana for Autism

In The Politics of Autism, I discuss alternative treatments.

Bill Laitner reports at The Detroit Free Press:

Gov. Rick Snyder’s top state regulator on Thursday rejected a state panel’s advice to allow medical marijuana as a treatment for autism.

The decision followed three years of efforts by parents of autistic children, their lawyers and supporters to have Michigan become the first state to specify that marijuana could be used to treat autism.

Mike Zimmer, appointed in December as director of the Michigan Department of Licensing and Regulatory Affairs — LARA — said he was concerned that an approval would apply not just to serious cases of autism but to all cases. And he said that parents applying to use medical pot would need the approval of two medical doctors, yet there was no requirement that either doctor be experienced in treating autism.

In a four-page “Final Determination,” Zimmer said that allowing the use of medical marijuana for autism might do more harm than good to mildly afflicted autistic children. That view followed corroborating testimony in Lansing by Dr. Harry Chugani, chief of pediatric neurology at Children’s Hospital of Michigan and a national authority on autism.

In July, Chugani told the Free Press that “the vast majority of kids with autism do not need pot, and I won’t sign for it.” He said the drug should be reserved for those with “very bad behaviors, aggression, meltdowns.” Chugani could not be reached after the release of Zimmer’s order.

Orac writes:

Three weeks ago, I wrote a post likening the use of “medical marijuana” for autism to a form of quackery that I have written about many times over the years, namely so-called “autism biomed.” As I mentioned, a certain segment of the “autism biomed” movement has enthusiastically embraced medical cannabis, to be added to the other dubious treatments used to “treat” autism, such as chelation therapy, various supplements, hyperbaric oxygen, homeopathy, Miracle Mineral Solution (MMS, a.k.a. a form of bleach), and the like. (I’m talking to you, “Thinking Moms.”) As I detailed in that post, the parallels are unmistakable. In the case of cannabis for autism, there is the same evangelical embrace of cannabis as the next big thing that can help autistic children and the same dismissal of the extreme lack of evidence supporting the use of medical cannabis for autism and the lack of knowledge of the effect of long term cannabis use in very young children on their neurological development. Remember, we’re talking about children as young as three years old here and the use of cannabis over potentially many years.

Cure Du Jour: MSG

Many posts have discussed purported "cures" for autism. The San Francisco Chronicle reports:

Katherine Reid, a Bay Area biochemist with a daughter who was autistic, believes she may have found an antidote to the neurodevelopment disorder - and it's as simple as changing a person's diet.

.... She thinks what it comes down to, at least for some people with autism, is permanently eliminating just a single chemical compound known as monosodium glutamate, or MSG - an ingredient many people associate with Chinese food.

...

While there is no science to back up many of her claims, [emphasis added] Reid said the most convincing evidence to her is the results she saw in her daughter. At age 7, Brooke is completely cured, Reid said. And from all outward appearances that seems to be true.

Dr. Robin Hansen, professor of pediatrics at UC Davis and a developmental behavior pediatrician who recently led a study for the university's Mind Institute, said it's fairly common for parents to seek out alternative treatments for their children with autism. Nearly 7 percent of the children with autism they studied were on gluten- and casein-free diets.

"We don't have a lot of diet research to look at, because these studies are difficult to do," she said, describing the trickiness of monitoring a child's food intake in a double-blind study. "And no one has done an MSG study. But what we do have doesn't show a marked difference even with children with gastrointestinal problems."

Still, she wouldn't dissuade parents from trying as long as they make sure the diet is balanced and to keep in mind that it's a big undertaking.

A single case proves nothing, especially when it does not involve rigorous measures (e.g., ADOS) before and after the intervention.

Reid's claims are not new:  she said the same things at Santa Cruz.com more than a year ago and has a nonprofit.

 

Medications and Autism

The New York Times reports:

With the diagnosis of autism on the rise and drug companies facing major setbacks in developing successful treatments, the University of California, Los Angeles will lead a $9 million effort financed by the National Institute of Mental Health to find effective drugs, officials said Wednesday.

Under a contract with the institute, U.C.L.A. will form a network of researchers at other academic centers that will try to identify promising new and older drug compounds quickly, and conduct early tests to see if they merit additional investment.

The program, part of the “Fast Fail” initiative at the institute, aims to determine within weeks whether a drug works, rather than the years it traditionally takes to evaluate a new drug.

...

Several major drug companies, including GlaxoSmithKline and AstraZeneca, have scaled back their research in the neurosciences because of the high failure rate, Dr. McCracken said.
Developing drugs to treat neurological disorders is difficult, in part because brain science is still evolving. The field is littered with drugs that scientists had hoped would be effective against diseases like Alzheimer’s and schizophrenia but that performed poorly in clinical trials.

Despite the setbacks, scientific advances in understanding the genetic underpinnings of autism have accelerated, leaving the door open for new drug discoveries, said Robert H. Ring, vice president of translational research at Autism Speaks, a patient advocacy group.

Talking Points for the Hearing

Left Brain/Right Brain suggests some talking points for the congressional hearing next week:

1) We need focus on improving the quality of life of Autistics
2) While not all Autistics can self-advocate, if we are going to have autism organizations represented, we need to have Autistic-run organizations represented.
3) Autism is a very broad spectrum, all with challenges of some sort. The government’s response needs to be broad (read-larger than it is now) in order to encompass all the needs of these communities.
4) The vaccine-epidemic hypothesis has been very damaging to the autism communities. Please don’t allow this meeting to be a way around the science in order to keep that idea alive.
5) There are faux therapies in common use for autism. Many are harmless. Some are dangerous and based on incredibly poor science. We need to get accurate information out about these practices.

The Thinking Person's Guide to Autism observes:

We at TPGA are concerned that no autistic-run organizations have been invited to the meeting as of this writing. We are also concerned about the track record of such congressional hearings: they have been used in the past to promote harmful misinformation about autism, and one of the past and current committee members is Rep. Dan Burton, who still firmly believes his grandchild's autism was caused by vaccines.

FDA v. Chelation

The Los Angeles Times reports:

Products called chelators that are sold over the counter as treatments for autism, heart disease and other conditions are dangerous and illegal, the U.S. Food and Drug Administration warned in a crackdown announced Thursday.

The chemicals, which help remove metals from the body, are potent drugs that carry serious risks, including kidney damage, dehydration and even death, said FDA Medical Officer Dr. Charles Lee. "Chelation can be dangerous and can cause serious harm," Lee said.

The FDA sent letters this week warning eight companies they are in violation of federal law by selling the products. The letters also served to caution parents and others who might be tempted to buy the chelators, which are touted on scores of Web sites and blogs as effective therapies for a wide range of chronic medical problems.

See FDA consumer update.

Here is the FDA release:

The U.S. Food and Drug Administration today warned eight companies that their over-the-counter (OTC) chelation products are unapproved drugs and devices and that it is a violation of federal law to make unproven claims about these products. There are no FDA-approved OTC chelation products.

The companies that received the warning letters claim that their products treat a range of diseases by removing toxic metals from the body. Some also claim to treat autism spectrum disorder, cardiovascular diseases, Parkinson’s disease, Alzheimer’s disease, macular degeneration, and other serious conditions. Some companies that received the warning letters also claim their products will detect the presence of heavy metals to justify the need for chelation therapy.

The drug products involved have not been evaluated by the FDA for treatment of these diseases, and violate the Federal Food, Drug, and Cosmetic Act (FFDCA). Despite the claims of the companies that received warning letters, the effectiveness in treating any of the diseases listed is unsubstantiated. Depending on the condition, when relying on unproven OTC chelation products to treat serious conditions, patients may delay seeking effective medical care.

In addition, there are serious safety issues associated with chelation products, which can alter the levels of certain substances in the blood. Even when used under medical supervision, these products can cause serious harm, including dehydration, kidney failure, and death.

“These products are dangerously misleading because they are targeted to patients with serious conditions and limited treatment options,” said Deborah Autor, director of the Office of Compliance in the FDA’s Center for Drug Evaluation and Research. “The FDA must take a firm stand against companies who prey on the vulnerability of patients seeking hope and relief.”

The agency advises consumers to avoid non-prescription products offered for chelation or detoxification. The only FDA-approved chelating agents are available by prescription only and are approved for use in specific indications such as lead poisoning and iron overload. Procedures involving these agents carry significant risks and should be performed only under medical supervision.

The FDA has noted an increase in “chelation therapy” products marketed on the Internet that claim to cleanse the body of toxic chemicals and heavy metals. Although some of the products are marketed as dietary supplements, they are unapproved drugs because they claim to treat, mitigate, prevent, or diagnose disease. The products come in various dosage forms, including transmucosal sprays, suppositories, capsules, liquid drops, and clay baths.

Some of the companies also sell unapproved screening tests that claim to detect the presence of heavy metals in urine to justify the need for chelation therapy.

"FDA will seek enforcement action against companies that promote therapeutic benefits of products not yet evaluated by the agency for safety and effectiveness.” said Dara A. Corrigan, associate commissioner for Regulatory Affairs.

Under the FFDCA, companies that market products that claim to prevent, diagnose, treat or cure diseases must file an application with the FDA and provide data that demonstrate their products’ safety and effectiveness.

The companies must take prompt action to correct the legal violations cited in the warnings letters or face possible legal action, including seizure and injunction. The FDA issued warning letters to the following companies:

• World Health Products, LLC: Detoxamin Oral, Detoxamin Suppositories, and the Metal Detector test kit
  
• Hormonal Health, LLC and World Health Products, LLC: Kelatox Suppositories, and the METALDETECTOR Instant Toxic Metals Test
  
• Evenbetternow, LLC: Kids Chelat Heavy Metal Chelator, Bio-Chelat Heavy Metal Chelator, Behavior Balance DMG Liquid, AlkaLife Alkaline Drops, NutriBiotic Grapefruit Seed Extract, Natur-Leaf, Kids Clear Detoxifying Clay Baths, EBN Detoxifying Bentonite Clay, and the Heavy Metal Screen Test
  
• Maxam Nutraceutics/Maxam Laboratories: PCA-Rx, PC3x, AFX, AD-Rx, AN-Rx, Anavone, AV-Rx, BioGuard, BSAID, CF-Rx, CreOcell, Dermatotropin, Endotropin, GTF-Rx, IM-Rx, Keto-Plex, Natural Passion, NG-Rx, NX-Rx, OR-Rx, Oxy-Charge, PN-Rx, Ultra-AV, Ultra Pure Yohimbe, and the Heavy Metal Screening Test
  
• Cardio Renew, Inc: CardioRenew and CardioRestore
  
• Artery Health Institute, LLC: Advanced Formula EDTA Oral Chelation
  
• Longevity Plus: Beyond Chelation Improved, EndoKinase, Viral Defense, Wobenzym-N
  
• Dr. Rhonda Henry: Cardio Chelate (H-870)

Lupron

The Palm Beach Sun-Sentinel reports:

A Maryland medical group has started treating autistic children in South Florida with shots of a drug used for chemical castration, a therapy widely panned by mainstream experts.

The group gives children the cancer drug Lupron to stop their bodies from making testosterone, saying the drug helps expel toxic mercury and quells aggressive or sexually explicit behavior by kids with excessive levels of the male hormone.

A Boca Raton mother who just put her 18-year-old son on the drug said it seems to help.

But numerous physicians, researchers and therapists insist there's no proof mercury causes autism, that Lupron removes mercury or that autistic kids have excessive testosterone. What's more, the drug carries a risk of bone damage, stunted growth and heart trouble, and can render children impotent.

These experts contend that Lupron, costing about $5,000 a month but seldom covered by insurance, is one of many treatments that cash in on the desperation of parents trying to cope with an incurable condition for which medicine has few good answers outside of painstaking behavioral therapy.

"Not only is there no scientific backing whatsoever for Lupron treatments, there are several major concerns for the children's health," said neurologist BethAnn McLaughlin, an adviser to the Dan Marino Foundation autism group in Weston and the mother of two developmentally disabled children.

• See a sidebar on controversial treatments.
• See a 2009 Chicago Tribune piece on lupron.

FDA Steps In

The Los Angeles Times reports:

A product promoted to parents of children with autism is not a harmless dietary supplement, as claimed, but a toxic unapproved drug that lacks adequate warnings about potential side effects, including hair loss and abnormalities of the pancreas, the U.S. Food and Drug Administration has warned in a letter to its maker.

The FDA's June 17 letter to Boyd Haley, a retired Kentucky chemist and hero to the autism recovery movement, details five violations of the Federal Food, Drug and Cosmetic Act related to his product, OSR#1. Failing to correct such violations can result in fines, seizure of products and even criminal prosecution.

The Tribune in January reported that the compound, sold as OSR#1, had been developed to treat mining wastewater, and that it had not undergone rigorous testing to ensure it is safe and effective. The report was part of an investigation into unproven autism therapies offered by health providers who say they can reverse the disorder.