Home > Science > Stem Cells – A New Frontier in Autism Research

Stem Cells – A New Frontier in Autism Research

Daniel Lightfoot, Ph.D., Director of the Autism Tissue Program

Ricardo Dolmetsch, Ph.D. has a vision for autism research.  Using pluripotent stem cell (iPSC) technology to create rare stem cells from other “common” cells of the human body cells, Dolmetsch and his lab at Stanford study neurodevelopmental disorders such as autism.

Unlike embryonic stem cells or adult stem cells which are isolated from existing and often difficult to obtain tissues, iPSC’s are “created” from easy to obtain and plentiful sources, such as skin or hair samples.  This is accomplished through a unique process where cells are developmentally regressed to an earlier state.

To appreciate the concept of a stem cell, consider a seed.  As a single cell it holds the potential to grow into an adult plant.  It is a “stem cell” – one that can change or develop into any cell of the plant, from a leaf cell, to a flower cell or into a root cell.  Through iPSC technology, this process is reversed.  Scientists can developmentally regress an adult cell into an earlier cell like a seed.  In short, scientists can turn a piece of a leaf into a seed, which could then grow into any cell of the plant.  Though this does not at all imply that science can create a whole person from a skin sample, it does, however, allow researchers to easily create a variety of cells that can then be used for scientific study.

Once stem cells are created, they can be induced to develop into brain cells.  For the first time, scientists are directly studying living brain cells in the lab.  How these cells grow, interact, communicate, organize into groups and what helps or impairs these cells’ growth is now being more effectively studied.  Additionally, stem cells have the unique ability to replicate without changing, meaning that from a single skin or hair sample many cells can be created.  This allows a near limitless source of resources for scientific inquiry.

Dolmetsch shared this vision at a keynote presentation at IMFAR.  He and his colleagues have now created an entire repository of stem cells from individuals with neurodevelopmental disorders.  By comparing autism brain cells, with Timothy Syndrome and other disorders, the research team is not only learning about the differences among these conditions, but also the commonalities.  Once the brain cell is created, it is possible to experiment with different compounds to determine whether they can restore neuronal function.  Thus, stem cells provide a platform for drug screening.  A deeper understanding of these disorders will also contribute more generally to a fundamental appreciation of how the human brain works.

  1. Johanna Burke
    May 27, 2011 at 2:50 pm

    Please send me information on this. I would love for my son to participate in a stem cell study. He is 8 years old & has severe autism.

  2. rahul
    August 5, 2011 at 11:42 pm

    hi dear I am very much interested in this study can u send me info on

  3. Amy Burger
    September 28, 2011 at 12:30 am

    Please send info, this is remarkable and I want this for my child.

  4. Mubeen Ali
    January 8, 2012 at 10:29 pm


    Please send me information on this study. I would love for my daughter to participate in this study and we live in the day area.

  5. christa
    January 23, 2012 at 1:48 pm

    please send me info… i would like for my son to participate

  6. Jennifer Collins
    January 29, 2012 at 7:44 pm

    HAVE OWN UMBILICAL CORD STEM CELLS. I would love to participate in a study using stem cell treatment. I have 3 year old twin boys, both diagnosed with autism. One is profound and completely non-verbal and the other is moderate and verbal with some advanced skills such as reading by 2 and an amazing memory. We have save the umbilical cord stem cells from their sister. We have been doing a great deal of research and are very interested in stem cell treatments for one or both of my twins. They are still young and their brains are more plastic and we could provide the stem cells. We would love to participate in a stem cell research study! Please contact me!

  7. fe
    March 5, 2012 at 5:52 pm

    please send me info, I have two Melody just turned 9 and Abraham 7

  1. May 20, 2011 at 10:15 pm

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