Adult human stem cells are quickly becoming an important research tool in neurological diseases. Fibroblast cells, obtained from an arm skin biopsy, are reprogrammed into pluripotent iPSCs, which means they can be induced to develop into different types of cells--liver, heart muscle, or whatever the scientist is researching. Neurons derived from iPSCs can then be used to compare normal and disease-specific cell lines. Academic laboratories and pharmaceutical companies are now very interested in working with these cells as a way to model human disease and potentially screen therapeutic drugs.

Stem cells

At the recent NINDS sponsored "iPSC Consortia Meeting - Accelerating Discovery and Building Resources" held in Washington DC, January 22-23, 2014, the creation of the FTD iPSC Consortium was announced.  FTD joins ALS, Huntington's and Parkinson's diseases as the focus of this project, which aims to create these disease-specific iPScells and make them available to scientists throughout the world at minimal cost. AFTD Scientific Director Dr. Nadine Tatton represented AFTD, a founding member of the iPSC Consortium, at the conference. Drs. Yadong Huang, UCSF-Gladstone Institute and Fen-Biao Gao, University of Massachusetts Medical School updated the group on the tau, progranulin and C9ORF72 lines that they have created from FTD patients' fibroblasts and will be submitting to the consortium repository.

  

At the same meeting Dr. Jeffrey Rothstein presented results from his work (supported by an AFTD Drug Discovery grant) creating and studying C9ORF72 stem cell lines. C9ORF72 is the most common mutation in familial ALS and FTD. Using these cells, Dr. Rothstein and his team have revealed how the mutation leads to death of the brain cell, knowledge which suggests that these iPScells may also hold potential as a new drug screening tool. Click here for an article on the topic. 

Doing My Best for Bill - April, Vienna, Austria - Anders Fehon will run the Vienna Marathon in honor of his dad, Bill. The Fehons make and sell Bill's Best BBQ Sauce.  Ten percent of the profit is donated to AFTD.

Lon Kieffer is an author, speaker, entertainer and self-proclaimed "Defender of Caregivers" (DOC).  His website offers helpful tips and encouraging ideas to those caring for a loved one. 

  

After collecting five helpful tips from professionals in various fields, Lon posted five steps for having a better day.  To read the tips, visit Lon's website.  

  

If you have words of enCOURAGEment that you would like to share in an upcoming issue of the Gateway, please email them to amaher@theaftd.org.