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Nuvarande forskningsläge för potentiella läkemedelskandidater vid behandling av kärnsymtom vid autism
Linnaeus University, Faculty of Health and Life Sciences, Department of Chemistry and Biomedical Sciences.
2018 (Swedish)Independent thesis Basic level (degree of Bachelor), 10 credits / 15 HE creditsStudent thesisAlternative title
Current research on potential drug candidates for treatment of autism core symptoms (English)
Abstract [sv]

Autismspektrumtillstånd kännetecknas av kärnsymtomen stereotypa, repetitiva beteenden samt nedsatt förmåga att kunna hantera sociala relationer. Diagnostisering försvåras dock av att inga biomarkörer finns tillgängliga än. Forskning pågår för att få klarhet i patofysiologiska mekanismer, där funna defekta gener ger en bild av ökade svårigheter för nervcellen att reglera signaler efter behov. Behandling som riktar sig mot kärnsymtomen vid autismspektrumtillstånd finns ej, men forskning pågår för att hitta lämpliga läkemedel. Syftet med detta arbete var att sammanställa senaste forskningen inom detta område, via sökning efter dubbelblinda randomiserade kontrollerade studier, för att utvärdera om något läkemedel kommer finnas på marknaden inom en snar framtid. Studier med D-vitamin, bumetanid (diuretikum), suramin (antipurinergt), memantin ”extended release” (mot Alzheimers sjukdom), metylerad vitamin B12 och celecoxib (anti-inflammatoriskt) som adjuvans till risperidon (neuroleptikum) utvärderades för effekten på kärnsymtom vid autismspektrumtillstånd. Bumetanid, som är ett loop-diuretikum, var det läkemedel som kommit längre än övriga. Om även fas 3 ger positiva resultat så finns möjligheten att detta kan släppas på europeiska marknaden om några år för behandling av kärnsymtom vid autismspektrumtillstånd. Övriga substanser var i ett mycket tidigt utvecklingsstadium.

Abstract [en]

Autism spectrum disorder is characterised by the core symptoms stereotypic, repetitive behaviors and impaired ability to handle social relationships. Diagnostics, however, is complicated by the fact that no biomarkers are available yet, i.e. one cannot take a blood test to decide if the individual can be diagnosed with autism spectrum disorder. Research, however, is ongoing in this field hopefully also leading to markers that can be used when evaluating degree of severity during clinical research where potential pharmaceuticals are evaluated in humans.

Research is ongoing to clarify pathophysiological mechanisms. Identified clusters of defective genes suggest increased difficulties of nerve cells to regulate signals as needed. For example, excitation or inhibition of nerve cells can be enhanced.

Treatment aimed at the core symptoms of autism spectrum disorder is not available, but research is ongoing to find suitable drugs. The purpose of this work was to compile the latest research in this area through a search for doubleblinded randomized controlled studies to evaluate whether any drug will be available on the market in the near future.

Studies with vitamin D, bumetanide (diuretic), suramin (antipurinergic), memantine “extended release” formulation (Alzheimer’s disease drug), methylated vitamin B12 and celecoxib (anti-inflammatory) as adjuvant to risperidone (neuroleptic) were evaluated for the effect on core symptoms in autism spectrum disorder. Bumetanide, which is a loop-diuretic, is the drug that has come further than the rest. If the continued development of this drug also gives positive results, there is the possibility that this drug can be released on the European market in a few years, for treatment of core symtoms. The other substances evaluated were at a very early stage of development. Some of these, however, can be of extra interest. For example D‑vitamin which can have some potential effect. Also it gives minimal side effects.

Place, publisher, year, edition, pages
2018. , p. 39
Keywords [en]
autism, autism spectrum disorder, ASD, drug candidates, current research, ongoing research
Keywords [sv]
autism, autismspektrumtillstånd, AST, läkemedelskandidater, aktuell forskning, nuvarande forskningsläge
National Category
Pharmaceutical Sciences
Identifiers
URN: urn:nbn:se:lnu:diva-75550OAI: oai:DiVA.org:lnu-75550DiVA, id: diva2:1216315
Educational program
Bachelor of Science Programme in Pharmacy, 180 credits; Bachelor of Science Programme in Pharmacy, 180 credits
Supervisors
Examiners
Available from: 2018-06-11 Created: 2018-06-11 Last updated: 2018-06-11Bibliographically approved

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CiteExportLink to record
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