Talking Trials

Clinical trials expert Merit Cudkowicz discusses why it's so hard to find therapies for ALS

Article Highlights:
  • Two of the biggest challenges in designing effective ALS clinical trials are late diagnosis and disease heterogeneity (variation in disease characteristics from person to person).
  • Developing an understanding of ALS disease heterogeneity must become a priority for researchers. This might be done by analyzing biofluids, imaging data and post-mortem samples from patients.
  • There are many more scientists and clinical investigators working in the ALS field today, generating more ideas about what might explain disease and progression. More therapeutic targets are being identified, but there is still much to do and learn.
by Amy Madsen on April 1, 2013 - 9:03am

Quest Vol. 20, No. 2

Put together the words amyotrophic lateral sclerosis (ALS) and the phrase clinical trials expert, and one name that is sure to follow is Merit Cudkowicz.

Cudkowicz is chief of neurology and director of the Neurology Clinical Trials Unit and the ALS Center at Massachusetts General Hospital (MGH) in Boston. The longtime MDA grantee also directs the MDA/ALS Center at MGH (one of five sites in MDA’s ALS Clinical Research Network).

In 1995, to help advance clinical testing in ALS and other motor neuron diseases, she co-founded the Northeast ALS Consortium (NEALS), a vast clinical trials network that brings together academic investigators and companies, research sites and scientific resources including an ALS biorepository and several trial databases. (A biorepository collects, stores and makes available specimens of biological material, such as urine, blood, tissue, cells, DNA, RNA and protein.)

In a February 2013 discussion, Cudkowicz described the current state of clinical trials in ALS, including the challenges they face, what investigators need to know to make them successful, and which ones to (maybe) get excited about.

To read the feature in its entirety, visit Talking Trials: Clinical trials expert Merit Cudkowicz discusses why it's so hard to find therapies for ALS.

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