work in progress

1. industryanalyzerQiTianindustryanalyzer
1
Autism
Survey of Emerging Approaches
QiTian industryanalyzer
PI Neil Rubens

2. industryanalyzerQiTianindustryanalyzer
Medical Interventions
2
Disclaimer: not intended and should not be construed as medical advice, nor is the information a substitute for professional
expertise or treatment. you should consult with your health care provider or seek other professional medical treatment

3. industryanalyzerQiTianindustryanalyzer
3
Diagnosis
Early Detection Accuracy Coverage Cause Detection
Treatment (Startups)
Digestion Stem Cells Personalization
Clinical Trials
Purpose Intervention Stages
Potential Future Directions
Deeper Analysis of Clinical Trials Research Works Funding Analysis

4. industryanalyzerQiTianindustryanalyzer
Diagnosis
4
Early detection and intervention of ASD
can lead to substantially better prognosis
Barton, M. L., Dumont-Mathieu, T., & Fein, D. (2012). Screening young children for autism spectrum disorders in primary practice.
Journal of autism and developmental disorders, 42(6), 1165-1174.
https://www.ncnp.go.jp/nimh/jidou/aboutus/review.pdf
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5576710/

5. industryanalyzerQiTianindustryanalyzer
5
First physiological tests for autism prove high accuracy in second trials.
2018, Rensselaer Polytechnic Institute
https://news.rpi.edu/content/2018/06/19/success-blood-test-autism-affirmed
BioROSA
Technologies
95% sensitivity and 92% specificity
based on metabolic profiles
"Advanced glycation endproducts, dityrosine and arginine transporter dysfunction in autism
a source of biomarkers for clinical diagnosis."
Anwar, Attia, et al. Molecular autism 9.1 (2018): 3.
"Autism spectrum disorder screening instruments for very young children: a systematic review."
Towle, Patricia O., and Patricia A. Patrick. Autism research and treatment 2016 (2016).
many upcoming methods from academia e.g.:
availableupcoming
30% accuracy
93.5% upcoming: Smith, Alan M., et al. "Amino acid dysregulation metabotypes: Potential
biomarkers for diagnosis and individualized treatment for subtypes of autism
spectrum disorder." Biological psychiatry 85.4 (2019): 345-354.
Early Detection / Accuracy

6. industryanalyzerQiTianindustryanalyzer
6
Focus Early Detection (18 months+)
Approach Metabotype
Accuracy 30% ref
93.5% upcoming ref
Clinical Trial NCT02548442 ref
Large Scale 1,100 children
Insurance approved e.g. Aetna

7. industryanalyzerQiTianindustryanalyzer
7
The underlying genetic cause can be due to many different types of genetic variations and
no one test can detect them all.
Coverage

8. industryanalyzerQiTianindustryanalyzer
8
Detect whether an autoimmune reaction
is causing neurologic or psychiatric symptoms.
Immunogloubin may help to treat autism.
Cause Detection
Clinical Trials NCT02839915
Estimated Primary Completion Date : January 1, 2022

9. industryanalyzerQiTianindustryanalyzer
Treatment
9

10. industryanalyzerQiTianindustryanalyzer
10
FDA “Fast Track” designation
Aid in the development and expedite the review of drugs which show promise in treating a
serious or life-threatening disease and address an unmet medical need.
Many children with Autism exhibit impaired protein
digestion, which may or may not manifest in self-
restricted diets. The inability to digest protein affects the
availability of essential amino acids in the body. CM-AT,
our lead drug candidate, is designed to enhance protein
digestion thereby potentially restoring the pool of
essential amino acids. Essential amino acids play a critical
role in the expression of several genes important to
neurological function and serve as precursors to key
neurotransmitters such as serotonin and dopamine.
Digestion
https://clinicaltrials.gov/ct2/show/NCT02410902

11. industryanalyzerQiTianindustryanalyzer
11
Stem Cells
Stem Cells from Cord Blood
Unique advantages:
•Anti-inflammatory properties: regulate inflammation and migrate to the
site of neurologic injury.
•Bystander effect: promote the body's own repair mechanism
•Safe and accessible: easily collected at the time of birth and stored for
potential future use by the newborn donor or close family member.
Clinical Trial (Phase 2): NCT01638819 Preliminary results are promising: PMC5866927
produce cerebral organoids
that allow to identify potential drug compounds
that are more likely to be clinically efficacious.
Stem Cells from Patients with Brain Disease

12. industryanalyzerQiTianindustryanalyzer
12
precision medicine for subtypes of ASD patients
correct molecular pathways that lead to abnormal cell functioning in ASD
by repurposing targeted pharmacological treatments.
Personalization

13. industryanalyzerQiTianindustryanalyzer
Clinical Trials
13

14. industryanalyzerQiTianindustryanalyzer
14
Our goal is to identify new/upcoming ways to diagnose and cure autism.
To do so we have analyzed the NIH clinical trials for Autism condition (964 trials as of 2019.08.01).
Even though the number of trials is relatively large (964); there are only 17 positive trials (p<0.05).
Note: the primary reason for small number of positive trials is due to the absence of results and statistical analysis of the results.
It is upcoming for some trials.
Stages
TODO: add years for trials started, completed, etc.

15. industryanalyzerQiTianindustryanalyzer
15
Overview
Type of Intervention
Behavioral 465
Drug 363
Other 220
Device 69
Dietary Supplement 41
Procedure 29
Biological 22
Diagnostic Test 7
Genetic 6
Combination Product 2
Purpose
Treatment 598
Supportive Care 38
Health Services Research 31
Basic Science 28
Other 25
Diagnostic 19
Prevention 13
Screening 6
Treatment is the dominant purpose; however its definition is rather broad (in many case treating
symptoms rather than underlying causes; e.g. suppressing aggression via sedatives).
Wide variety of distinct interventions (1047) are being evaluated.
Only a few of these interventions are evaluated across different studies. Most Frequent:
Oxytocin 14
Aripiprazole 14
Risperidone 12
Buspirone 7
Intranasal Oxytocin 7
Sulforaphane 5
Omega-3 Fatty Acids 4
Parent Training 4
CM-AT 4
Pivotal Response Training 4
Memantine 4
Early Start Denver Model 4
Parent Education 4
Arbaclofen 4
Memantine Hydrochloride (HCl) 4
aripiprazole 3
intranasal oxytocin 3
Propranolol 3
Fluoxetine 3

16. industryanalyzerQiTianindustryanalyzer
16
In this part of our study the goal is to identifying upcoming interventions (for diagnosis or cure);
hence examining (17) trials with the positive outcomes (p<0.05).
Most of the trials didn’t match criteria; since aim was to treat the symptoms rather than underlying cause.
E.g. NCT00584701: Pharmacogenomics in Autism Treatment is not relevant for us since it is aimed at predicting efficacy of antipsychotic drug (treating symptoms rather than cause).
However some studies have potentially interesting aspects e.g. in this case the use of genomic profiles to predict medication response.
only one positive trial matched our criteria:
Suramin Autism Treatment-1 (SAT1) Trial
NCT02508259
What: correct the symptoms, metabolism, and brain synaptic abnormalities
How: via digestive system
Other Advantages: very recent: July 16, 2019
Follow Up
http://naviauxlab.ucsd.edu
another ongoing study that focuses on diagnosis via blood:
http://naviauxlab.ucsd.edu/study/
Diagnosis / Cure

17. industryanalyzerQiTianindustryanalyzer
17
Stanford
Intranasal Oxytocin Treatment for Social Deficits in Children With Autism
NCT01624194
Drug for social aspects [Stanford University]
neuropeptide oxytocin (OT) may be an effective treatment for the core social deficits in autism.
p.s. at present, no effective drug treatments for the social impairments
There is another similar study that uses oxytocin (also w/ positive results): NCT01308749
Follow Up
A more recent study that uses hormone:
https://med.stanford.edu/news/all-news/2019/05/hormone-reduces-social-impairment-in-kids-with-autism.html

18. industryanalyzerQiTianindustryanalyzer
18
ID Title
NCT00584701 Pharmacogenomics in Autism Treatment
NCT01624194 Intranasal Oxytocin Treatment for Social Deficits in Children With Autism
NCT02508259 University of California, San Diego (UCSD) Suramin Autism Treatment-1 (SAT1) Trial
NCT00935701 Acupressure and Acupuncture as an Intervention With Children With Autism II
NCT00614198 ScanBrit Dietary Intervention in Autism
NCT01308749 A Study of Oxytocin in Children and Adolescents With Autistic Disorder
NCT01579669 Partnering With Autistic Adults to Improve Healthcare
NCT00541346 A Pilot Study of Daytrana TM in Children With Autism Co-Morbid for Attention Deficit Hyperactivity Disorder (ADHD) Symptoms
NCT00576732 A Study of the Effectiveness and Safety of Two Doses of Risperidone in the Treatment of Children and Adolescents With Autistic Disorder
NCT02361762 Gaming for Autism to Mold Executive Skills Project
NCT00178503 Methylphenidate for Attention Deficit Hyperactivity Disorder and Autism in Children
NCT01177969 Cognitive Behavioral Therapy (CBT) for Anxiety Disorders in Autism: Adapting Treatment for Adolescents
NCT02739321 Examining the Efficacy of Mattress Technology in Improving the Sleep Quality of Children With ASD
NCT00380692
Atomoxetine Versus Placebo for Symptoms of Attention-Deficit/Hyperactivity Disorder (ADHD) in Children and Adolescents With Autism Spectrum
Disorder
NCT01050582
A Study to Evaluate the Safety and the Effects of Risperidone Compared With Other Atypical Antipsychotic Drugs on the Growth and Sexual
Maturation in Children
NCT00205699 Metabolic Effects of Antipsychotics in Children
NCT01906866 Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities
Positive Trials

19. industryanalyzerQiTianindustryanalyzer
Potential Further Directions
19

20. industryanalyzerQiTianindustryanalyzer
20
Autism
e.g. PubChem BioAssay
Research Publications large number of works of potential interest

21. industryanalyzerQiTianindustryanalyzer
21
Funding Analysis
follow the money

22. industryanalyzerQiTianindustryanalyzer
22
$78 million per year
supports over 250 investigators studying autism-related research.
Funding Analysis
high spend

23. industryanalyzerQiTianindustryanalyzer
23
Clinical Trials Deeper Analysis