$200,000 awarded to Dr. Vincent Picher-Martel at Massachusetts General Hospital.
Abnormalities in a protein called TDP-43 are present in approximately 97 per cent of all ALS cases. Normally, TDP-43 is found in the nucleus of a cell (a central compartment where our DNA is located); however, in people living with ALS it is often found in the cytoplasm (the area outside of the nucleus) where it tends to form clumps, or aggregate, and is no longer able to function properly.
Researchers have long sought after a biomarker that could detect this TDP-43 abnormality in ALS patients in real time. With this award, Dr. Vincent Picher-Martel will study a promising new biomarker that may be able to do just that, called stathmin-2. This protein is essential for axonal growth and the maintenance of neurons. Recently, it was shown that the mislocalization and aggregation of TDP-43 can lead to the development of abnormal stathmin-2 within cells.
Here, Dr. Picher-Martel will further explore the potential for stathmin-2 as a biomarker for ALS by studying the levels of abnormal stathmin-2 protein found in the cerebrospinal fluid (CSF), blood and post-mortem tissues of ALS patients and correlating it with the individual’s clinical data.
In addition, using motor neurons derived from patient stem cells, he will investigate the role of stathmin-2 in various types of ALS to determine whether stathmin-2 is abnormal in all forms of ALS and how it influences motor neuron health. Using an antisense oligonucleotide (ASO), Dr. Picher-Martel will also investigate whether normal production of stathmin-2 can be restored in cells and if so, have a positive impact on the health of motor neurons. If successful, this would suggest that in addition to its potential role as a biomarker, stathmin-2 may also represent a promising new treatment target for future ALS therapies.
Dr. Picher-Martel will accompany this work with additional training as a neurologist while completing a two-year fellowship at Massachusetts General Hospital in Boston. The program represents an exceptional opportunity for training in clinical trials, drug development, translational medicine, and allows for collaboration with experts in the field worldwide. After completing the fellowship, Dr. Picher-Martel plans to return to Canada where he will undoubtedly be an asset in the drive towards a better understanding of the disease and achieving better treatment outcomes.