Autism Drugs

Cambridge, MA-based Seaside Therapeutics recently announced that it has raised $30 million to pursue mid-stage clinical trial development on new therapies for Fragile X Syndrome and autism.

CEO Randall L. Carpenter explained that the aim of the drug is to not only help symptoms of irritability found in many autistic children, but also to improve other symptoms of social interaction difficulties.

“It may allow individuals to speak, to learn normally,” he explains. “It may enhance their ability to relate to the environment, be more calm and less anxious and potentially more interactive. We’re seeing profound effects in our animal models; how that translates to humans is what we’ll find out in the next year or two.” 

Read more: http://www.fiercebiotech.com/story/seaside-gets-30m-pursue-new-autism-drug/2009-09-17#ixzz0T4pnzFRW

Despite a condition affecting hundreds of thousands and being represented by pressure groups made up of highly motivated parents, very few drug companies have treatments for autism as part of their reasearch goals.

XConomy.com published an interesting article enquiring into the reasons for the lack of good drugs for autism.  The sheer complex range of the symptoms of autism are part of the factor.  However, the article does report on an interesting development in relation to the possible use of oxytocin as an autism treatment.  The problem with oxytocin is that it degrades in the blood stream in a matter of minutes.  One drug company (Anatrope) is trying the route of researching with a molecule very similar to oxytocin – Carbetocin, which lasts up to 4 hours in the blood stream and could be inhumed via a nasal spray.

The development of effective drugs to treat autism moved a step closer this week after possibly the biggest breakthrough in the history of research into the illness.  A massive genetic study comparing the DNA of more than 12,000 individuals has apparently established conclusively a specific genetic link to autism.  Researchers and autism campaigners have already hailed the findings, published in Nature, as comparable to similar research into the genetic causes of cancers which quickly led to a plethora of cancer drug treatments in the 1970’s.  It seems that it is no longer a question of if, but when autism drugs start appearing on the market.

Researchers at the Stanford University School of Medicine and Lucile Packard Children’s Hospital in the US are trying to establish a possible link between the hormone oxytocin and a possible cause of autism.  Oxytocin, ‘the love hormone’ or ‘the cuddle hormone’  has been in the news a lot recently as a result of growing research documenting its role in promoting trust and social bonds between individuals.  With the difficulty to relate socially and emotionally to others being the key characteristic of autistic patients, it is hoped that a lack of oxytocin may be a cause of the illness.  If a link is established, it could pave the way for blood tests for autism leading to earlier and more objective diagnoses and thus treatment.  Oxytocin could also be used to provide the first truly effective pharmaceutical approach to the treatment of autism.  The discovery of a biological basis for autism would lead inevitably to autism drugs that could directly treat its chemical origins.

Researchers at the Massachusetts Institute of Technology have pinpointed two genes related to autistic like symptoms in mice.  The breakthrough brings hope that autism drugs could soon be developed that target the signaling mechanisms between the equivalent two genes in the human DNA.  The two genes involved are the PTEN gene, which encodes the phosphatase and tensin homolog protein, and the serotonin transporter gene.  Mutations in either were found to impair sociability in the mice.