The mission of MAT: Medicine for Autism Today (MAT) is to facilitate autism research and expeditiously identify effective medical treatment options for children with autism.

The Challenge

Autism affects over one-half million individuals and therefore is more prevalent than Cystic Fibrosis (30,000), Downs Syndrome (250,000), Multiple Sclerosis (350,000) and pediatric rheumatoid arthritis ((50,000). However, the funding for research into cures for these conditions is up to ten (10) times greater than that of autism, which receives less than $20 of research funding per individual. This inequity is due primarily to an emphasis on behavioral diagnostic and treatment tools in lieu of the development of a scientific/medical model for autism treatment.

Moreover, the incidence of autism has exploded over the last decade and now occurs in at least 40 births per 10,000 (compared to 1-2 births per 10,000 only a decade ago. MATs demographic analysis also shows that special needs classifications like autism, ADD and speech and language disorders are increasing three times faster than the general population for children ages 5-19. Developmental disorders do not increase at these rates!!! Thus, the only plausible explanation for such data is a medical disease process triggered by a combination of factors, including the environment.

In addition, only one medical agent is under formal FDA review for the treatment of autism compared to 44 for childhood cancer, 14 for cystic fibrosis, nine for epilepsy and three for rheumatoid arthritis. MAT wants to close these research gaps and provide our children with the future they deserve.

The Hypothesis

The medical board that MAT believes offers the most potential for identifying medical treatment options for this generation of children with autism is the Neuro-Immune Dysfunction Syndromes (NIDS) Medical Advisory Board, a nonprofit medical research group that is currently developing a medical model with which to treat autism.

The NIDS Board has developed a Clinical Hypothesis Statement (available upon request) with over 60 peer reviewed journal references to support its research mission. The fundamental premise of the hypothesis is that these children are suffering from a dysfunctional relationship between the immune and neurological systems and require immune modulation to restore their cognitive potential. In addition, the following medical studies have been published in peer reviewed journals since the development of the Clinical Hypothesis Statement that further support the presence of neuro-immune and/or autoimmune dysfunction in children with autism:

• In the June, 1999 issue of the Journal of Pediatrics, a group of six researchers concluded that the presence of (brain auto antibodies) raises the possibility that autoimmuity plays a role in the pathogenesis of language and social development abnormalities in a subset of children with children (with autism and LAS). (J Pediatrics 1999; 134:607-613)
• In the June, 1999 issue of the Journal of Child Neurology, researchers led by Dr. Anne M. Comi of the Johns Hopkins Hospital in Baltimore, Maryland concluded that autism appears to be more common in families with a history of autoimmune disorders. The study included 61 autistic patients and 46 healthy patients. (J Child Neurology 1999;14:388-394)
• Physicians at Memorial Sloan Kettering in New York reported in the New England Journal of Medicine this year that men with testicular cancer and brain damage had a particular type of antibody in their blood that may have caused the brain damage. They believe the brain damage was not caused by the cancer, but by an overly aggressive attack by the bodys own immune system on a protein produced by tumors. This is the third study to link brain damage to an immune system attack on cancer.

The Plan
To this end, MAT has assisted the NIDS Board with the development of a business plan that can be summarized as follows:

Develop a clinical database (with appropriate control subjects) that evaluates the medical aspects of autism, including any immune system dysregulation that may contribute to the symptoms seen in autism

NOTE: The Doug Flutie, Jr. Foundation for Autism has agreed to support this database development.

• Identify clinical subgroups of children with autism based on the immune system factors catalogued in the medical database to more accurately target the types of agents for review

NOTE: A private foundation has been identified that is interested in assisting with the development of a NIDS medical “fellowship” to facilitate this analysis.

• Conduct animal trials with the agents that offer the most potential to evaluate and maximize patient safety during clinical trials
• Secure the licensing rights to multiple immune system agents, including neuropeptides such as VIP and Peptide T, that have the potential to remediate the disease process in a defined subset of children with autism

NOTE: A NIDS Board member is near completion in securing the rights to Peptide T as an immune modulator.

• Develop a network of research sites (which include NeuroSpect brain imaging capabilities) to facilitate the review of these agents. Sites will likely include UCLA, UMDNJ in New Jersey and a medical facility in Sydney, Australia.
• Present the data and research noted above to the pharmaceutical industry and commission interested parties to facilitate FDA approved clinical trials that comply with all federal safety and efficacy guidelines
  NOTE: Glaxo-Wellcome, Smith Kiln Beech and Roche have each requested additional information as outlined above.

Financial Requirements
In order to accomplish these tasks, we have developed a fund-raising goal of $750,000 as follows:

• $100,000 for clinical database development
• $150,000 for animal trial support
• $250,000 for research network development ((NeuroSPECT)
• $175,000 for clinical trial support
• $ 75,000 in general operations expense (10% of budget)
• MAT and NIDS believes that this business plan will lead to clinical trials with immune modulators by Fall, 2000 and put us on the road to solving the medical aspects of autism.