Addressing Rare Pediatric Non-verbal Spectrum Disorder

What is a pediatric non-verbal disorder?

Pediatric non-verbal learning disorder signs and symptoms do not match those of any well-described condition and currently does not have a formally accepted diagnosis possibly because difficulties in characterizing this condition remain to this day. Pediatric non-verbal disorder is accompanied with developmental delay, most notably where the toddlers never speak or will start speaking and then regress. Children will often never speak or will start speaking and then regress in this subgroup.

The diagnosis is based on the identification of deficits in social perception, social judgment and social interaction skills, given such difficulties are secondary to impaired visuospatial development and pervade diagnostic strategies based on recognition of significant perception problems, faulty understanding of facial expressions, tone of voice and speaker’s intention.

 

Low scores in specific motor performance tests involving both hands suggest bilateral brain involvement and confirm the presence of motor coordination impairments, with worse performance in the right hemisphere compared to the left. Children with non-verbal disorder are described as clumsy and uncoordinated.

Visuospatial deficits are the major characteristic of children with non-verbal disorder, even in the absence of severe motor problems. Differences between verbal and performance (non-verbal) IQ scores are not a requisite for the diagnosis of non-verbal learning disorder; still this finding has been particularly emphasized in affected children.

What causes non-verbal disorder?

Causes of non-verbal learning disorder have been linked to several complications that range from a specific mutated gene as with Fragile X Syndrome and Dravet Syndrome or autoimmunity, where the body’s immune system is attacking parts of the brain. Trauma, microbial infections and enviromental factors have also been linked to non-verbal learning disorder. Ongoing research is helping to further explain the root cause of why children become non-verbal or minimally verbal.

Who is most likely to get Non-verbal disorder?

Children born into families where there is a genetic history of autism or epileptic spectrum disorders or that have a sibling that has been diagnosed with an autistic or epileptic spectrum disorder have a much higher chance of becoming non-verbal.

Prevalence

Among the >60,000 US children who develop Autism Spectrum Disorders (ASD) every year, 20,000 become non-verbal.

No drugs are currently available to ameliorate this condition.

Current Standard of care

Of the estimated 20,000 who become non- or minimally verbal, they will require assisted living for the rest of their life. The lifetime cost of that care is estimated at $10 million per person.

 

Cognitive intervention is the only form for treatment that has shown to help improve speech capability and social interaction, however, it has not been able to alleviate the lifetime burden of $10 million per person for cost of care.

The Hard facts and emotional truth 

Currently, there are no drugs available to ameliorate this condition. As a result, lifetime costs of assisted living and supplemental healthcare average $10 million per person. This is compounded by an additional $10 million during the lifespan of the person due to loss in productivity. Not measuring the severe emotional strain of never talking to your child. This pediatric non-verbal disorder, where children lose or don’t develop speech and manifest with ASD symptoms is rare and limited to approximately 20,000 children a year in the US and about the same in Europe. 

Method of action 

 

 QBM-001 is given to high-risk genetically identified children during the second year of life to regulate faulty membrane channels that are known to cause migraines and/or seizures.

 This drug acts as an allosteric regulator of these faulty channels in the brain to potentially alleviate the condition and allow toddlers to actively develop language and speech and avoid life-long speech and intellectual disability of being non-verbal

QBM-001 also reduces inflammation in the brain and by so doing can reduce the amount of long-term nerve loss.

The only approved drugs for these children are anti- psychotics.

Current Medications and 2016 Sales 

Name

Condition

2016 Sales

Abilify®

Irritability

$2.0 B + 

Vyvanse®

ADHD

$2.0 B

Risperdal®

Aggression

$3 .0 B

QBM-001 regulates FAULTY ION-CHANNELS to Allow Language Development

8-12 Months – detection of early symptoms

12-15 months – language regression

Brain density in cortex (speech region) declines after 24 months

fMRI shows “patches of disorganization” due to pruning These are seen in the Cortex (verbal region) of the brain of non-verbal children older than 24 months

    Problem

    Faulty membrane channels associated with becoming nonverbal. 

    QBM-001

    QBM-001 modulates membrane channels to prevent hyperexcitation.

    Established use in Pediatrics

    50+ years of use treating chronic pain from the age of 6 months

    Treatment Window

    12-24 months of age

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