Harvard Stem Cell Institute and Evotec collaborate to advance ALS research

September 13, 2013

The Harvard Stem Cell Institute (HSCI) has entered into a strategic partnership with Evotec AG to identify compounds that prevent or slow down the loss of motor neurons, which is characteristic of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.

The collaboration "CureMN" (CureMotorNeuron) will identify therapeutically-valuable compounds by leveraging human motor neuron assays based on ALS patient-derived induced pluripotent stem cells that were developed by HSCI Executive Committee member Lee Rubin, PhD, and HSCI Principal Faculty member Kevin Eggan, PhD.

This agreement marks the third collaboration between Harvard Stem Cell Institute and Evotec scientists—and the fourth with Harvard scientists—and significantly expands a partnership model which combines cutting-edge academic research from Harvard with Evotec's drug platform and expertise. This partnership model efficiently and effectively drives the development of innovative drug candidates.

"This important relationship between HSCI and Evotech in the key areas of ALS, diabetes, and kidney disease epitomizes two key aspects of our strategy: the value of using stem cells as critical systems to discover more effective drugs; and, the importance of partnerships in accelerating the transition of science from academic research to the clinic and the market,” said Brock Reeve, HSCI Executive Director.

"Evotec, HSCI, and Harvard share a commitment to accelerating promising research from the lab to the clinic," said Isaac T. Kohlberg, Harvard University's Senior Associate Provost and Chief Technology Development Officer. "Our continued collaboration is based upon a shared vision to match world-class science with industrial capability to address important unmet medical need, which is at the core of our public mission."

Chief Scientific Officer of Evotec Cord Dohrmann, PhD, commented: "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."

“Phenotypic screens based on patient-derived iPS cells are an exciting approach to tackle diseases where tractable mechanisms have remained elusive. Evotec's proven expertise in high-content screening and deep knowledge in the field of motor neurons is a perfect match for this project. In this latest collaborative effort with Evotec, we look forward to putting our combined dedication and knowledge to work identifying new therapeutics for motor neuron diseases," added Vivian Berlin, PhD, Director of Business Development in Harvard's Office of Technology Development.