While there are currently no regulatory approved invasive therapies for ASD, imagine if there were. Would you want your child treated with a one size fits all protocol? Or would it be best to tailor a treatment program specifically to the root cause of his or her disease?

Precision Medicine, also referred to as Personalized Medicine, can be used to customize treatment based on the patient’s genetics, protein biomarkers, immune profile, environment, age, gender and other factors. Prior to designing a treatment protocol, the treating physician could use diagnostic tests including:  molecular diagnostics, proteomics, imaging or cellular analysis, to define which subgroup the patient belongs to.

One of the first demonstrations of the power of personalized medicine was the discovery of different breast cancer genes. At first all patients with breast cancer were given Herceptin®. Many patients responded poorly. Then researchers initiated a screening for the HER-2 gene over- expression¹. This has allowed clinicians to figure out which patients would benefit from Herceptin® therapy. This has transformed what had been a commonly fatal disease for this subset of patients to one with a cure rate of up to 85%.

Although substantially more complicated than screening for the over expression of one gene as in breast cancer, many researchers are studying the personalized medicine approach to diagnose and treat ASD. Biomarkers are starting to be identified and the field is beginning to show its potential².

The search for genetic, protein and other biomarkers could lead to earlier diagnosis, personalized pharmacological therapies, and could allow for earlier intervention and improved patient care. For example, recent reviews of the use of stem cell treatment for ASD³,⁴ have suggested that only some of the patients are positively impacted by the treatment. If, as in the case of Herceptin®, researchers could screen for children with ASD most likely to benefit from stem cell therapy, the level of success would likely increase.