A mother of two beautiful children, Lucie Clark was diagnosed with multiple sclerosis while she was slowing losing her eyesight and ability to walk.
Using Stem Cells to Identify Treatment for Dementia
Belgian scientists have used the stem cells to identify treatment strategies for an inherited form of Dementia. They took mutated stem cells from patients which will influence the development of frontotemporal dementia and identified treatment strategies using induced pluripotent stem cell (iPSC) technology. The research findings were published in the journal, Stem Cell Reports.
Frontotemporal Dementia (FTD)
This is an inherited disorder caused by progressive cell degeneration in the frontal or temporal lobe of the brain. The cell damage leads to tissue shrinkage and limited activity in the brain region that controls planning, judgement, speech, emotions, language and certain movements. It takes a toll on the personality and behaviour of the affected individuals while speech might become ungrammatical, halting and vague. They may also experience shakiness, lack of coordination, muscle stiffness, frequent falls, body imbalance etc. Once considered rare, FTD accounts to 10-15% of all dementia cases in the present day.
Help of Stem Cells to Identify FTD Treatment Strategy
Mutations in the gene, Progranulin (GRN) is associated with frontotemporal dementia. Although the mutation is observed in mice model, it does not mimic all the characteristics of the human disorder; thus providing a limited access to study the effects and treatment options. This has created the need for induced pluripotent stem cells to model dementia and study the disorder.
According to the research, the scientists took stem cells from the skin of three patients and reprogrammed them to iPSCs which are capable of turning into cortical neuron cells to model dementia. These immature cells from the patient carry GRN mutation which showed difference in generating cortical neurons when compared to normal cell development.
One of the most defective pathways in FTD is Wnt signaling pathway which is involved in neuronal development. The GRN mutation affects the Wnt pathway and thereby causing defects in cortical neuron formation which leads to frontotemporal dementia. After genetic corrections or treatment with compound that inhibits Wnt pathway, the differentiation defects can be restored- a key strategy for treatment options. The researchers believe that targeting Wnt pathway may create novel therapeutic approaches for frontotemporal dementia.
Catherine Verfaillie, the study author said, "iPSC models can now be used to better understand dementia, and in particular frontotemporal dementia, and might lead to the development of drugs that can curtail or slow down the degeneration of cortical neurons,"
This study provides better understanding of the working of GRN mutated cells and helps to identify the precise molecular targets that could be used in future drug screens. Although stem cells didn’t play a therapeutic role in this current research, they helped to study the cell and molecular biology of dementia in neuronal systems of human, which brought us a step closer in finding potential treatment options.