Developing Drug-Based Preventative Treatments for Autism Utilizing Digital Toolkits


New research insights into the neurological underpinnings of autism may provide a basis for the development of early-life pharmacological intervention strategies. Image Credit: Flickr user National Human Genome Research Institute

A November 2016 study in Nature Communications has revealed new insights into the brain physiology underlying autism. It was previously known that mutations in the phosphatase and tensin homolog (PTEN) gene, which plays a key regulatory role in the mTOR pathway, can cause a certain form of autism, known as autism syndrome. Building on this information, scientists at the Scripps Research Institute showed that in mouse models, mutating PTEN results in an increase in the number of neurons that project from the prefrontal cortex to the amygdala, creating more synapses, which leads to unusual activity in the amygdala and, consequently, altered social behavior. They also discovered that targeting the mTOR pathway with small molecule drugs shortly after birth could prevent these synapses from being formed.1

The researchers believe that this finding opens the door to explorations into mTOR-targeting therapeutic strategies for preventing autism that can be applied during early development.2 As life science companies look to develop early-life drugs for autism based on the study’s results, utilizing digital software solutions to streamline the research and development process will be imperative to bringing them to market sooner.

Sharing Autism Research Results Across the Organization

Developing a safe, effective and marketable early-life autism therapy will require the input of researchers and business leaders throughout a life science company. There are several ways that modern software can support this specific kind of necessary information sharing:

  • Supporting collaborations between researchers from different disciplines

Autism is a highly complex disorder, so the development of an effective early-life treatment strategy will likely require the input of scientists with expertise in a variety of different fields, including molecular biology, neuroscience and neonatal care. Therefore, researchers from across a life science company will need to play a role in the drug development process, and the company may even need to contract out some of the research to a CRO. Collaboration platforms that makes it easy for many researchers to easily access and weigh in on the interpretation of research results, regardless of their location, will be indispensible. With such a diversity of voices involved in the R&D process, it is more likely that the company will ultimately come up with a safe and effective autism drug on a more time-efficient track.

  • Enabling separate research groups to share results

The mTOR pathway involves dozens of enzymes, so large life science companies may decide to have multiple research groups working on different potential drug targets. Since every part of the pathway is interconnected, different research groups may sometimes need to conduct the same experiments. Experiment duplication wastes valuable lab resources and adds to the time it takes to get candidate drugs through the R&D process, but with modern software, separate research groups can share results with each other in real time, so only one group needs to conduct shared experiments.

  • Ensuring the availability of key information for business leaders

Another potential bottleneck in the drug development and commercialization process is the difficulty in accessing the scientific information necessary to make sound business decisions regarding production and distribution. Given the safety and ethical considerations that go into the use of an early-life autism drug, it is critical that business leaders in the company have all of the facts when giving the go-ahead to move forward with a certain drug candidate or deciding to abandon another. However, when it comes time to make critical business decisions, leaders may find that key background information is locked in disparate silos. It is extremely important to aggregate key results and make them accessible to the decision-makers who need it, without compromising time spent on actual research.

Streamlining the Steps of the Drug Development Process

The need develop effective, affordable treatment methods for autism can’t be understated. As of 2016, one in every sixty-eight children is diagnosed with the disorder, and the economic burden for therapy in the United States alone is in the billions.3 By standardizing key steps in the research, development and commercialization processes, digital software solutions can significantly reduce the time-to-market for new autism drugs. Moreover, decreasing the time it takes to get a new drug through the pipeline cuts down on overhead drug development costs, so the final product is more affordable for the autism patients who need it.

BIOVIA ScienceCloud is an advanced digital software solution that supports research efficiency through information sharing platforms and standardization procedures. Contact us today to get more information about how this digital toolkit can revolutionize drug development at your life science company and to learn more about our other software solutions.

  1. “Hyperconnectivity of prefrontal cortex to amygdala projections in a mouse model of macrocephaly/autism syndrome,” November 15, 2016,
  2. “Scripps Florida scientists discover clues to altered brain wiring in autism,” November 17, 2016,
  3.  “Autism Spectrum Disorder,” September 26, 2016,