Research Partnership Seeks Compounds to Slow Loss of Motor Neurons in ALS

A renowned American research institute is collaborating with a German drug discovery company to identify compounds that can prevent or slow down the loss of motor neurons, which is characteristic of the human disease amyotrophic lateral sclerosis (ALS).

ALS is a debilitating disease that induces muscle weakness and atrophy throughout the body caused by the degeneration of the upper and lower motor neurons. The majority of ALS victims die within three to five years from the onset of the symptoms, and only about 10% survive for 10 years or more. The incidence of the syndrome is approximately two per 100,000 people, and there are about 150,000 diagnosed ALS patients worldwide.

Investigators at the Harvard Stem Cell Institute (Boston, MA, USA) will be working with colleagues at Evotec AG (Hamburg, Germany) in a strategic partnership dedicated to the identification of compounds able to prevent or slow down the loss of motor neurons in ALS. The collaboration, which is being called “CureMN” (CureMotorNeuron), will leverage human motor neuron assays based on ALS patient-derived induced pluripotent stem (iPS) cells that were developed by Harvard Stem Cell Institute researchers Dr. Lee Rubin and Dr. Kevin Eggan.

Evotec is a drug discovery alliance and development partnership company focused on rapidly progressing innovative product approaches with leading pharmaceutical and biotechnology companies. Evotec has established an enviable position by assembling top-class scientific experts and integrating state-of-the-art technologies as well as substantial experience and expertise in key therapeutic areas including neuroscience, pain, metabolic diseases as well as oncology and inflammation.

Dr. Cord Dohrmann, CSO of Evotec, said, “Kevin and Lee have made significant contributions to our understanding of the underlying pathology of motor neuron diseases. Their laboratories have developed a large array of ALS patient-derived motor neuron models that allow screening of diseased human cells in culture – an approach that is sometimes referred to as a “clinical trial in a dish.” Our intention is to systematically screen for new mechanisms, targets, and compounds that have the potential to be developed into new products that will modify and ideally halt the progression of ALS and potentially other motor neuron diseases.”

Related Links:
Harvard Stem Cell Institute
Evotec AG