Options for Orphan Patients

By Marcia Faye

Amyotrophic lateral sclerosis (ALS)—Lou Gehrig’s disease—a progressive neurodegenerative disease that claimed the life of iconic New York Yankees baseball player Gehrig in 1941, may now be part of the standard health care lexicon. But ALS is considered a rare condition, one of more than 6,000 “orphan diseases,” defined by the United States Food and Drug Administration as conditions that affect fewer than 200,000 people across the country. Biology doctoral candidate Adriana Mañas Núñez (M.A.S. BENG ’14) and the six other co-founders of the Illinois Tech startup Great Lakes Neuroscience (GLN) are working to give some of these patients hope by developing a drug for ALS and a second orphan disease, primary progressive multiple sclerosis.


In 2014 Mañas Núñez, along with Sai Prashant Boy Reddy (M.B.A. ’15), Devon Nobles (M.B.A. ’15), Christian Osswald (BME ’10, Ph.D. ’15), Siddhartha Pidhadia (M.B.A. ’15), Rama Sashank (Ph.D. BIOL ’16), and Tiwalade Sobayo (M.A.S. CHE ’09, Ph.D. BME ’16) entered the Neuro Startup Challenge, a competition sponsored by the National Institutes of Health in collaboration with the Center for Advancing Innovation to develop a business plan to commercialize a number of inventions conceived and developed through NIH. The Illinois Tech team selected TP5, a patented synthetic peptide drug that has shown promise in the treatment of Alzheimer’s disease. Led by advisor Raja Krishnan, the team became a finalist in the competition and was inspired to continue its work on TP5 by incorporating GLN in 2015.

“Most of us joined with the thought that this would be a good exercise; none of us were expecting that it would go this far,” says Mañas Núñez, who came to Illinois Tech from Universidad Politécnica de Madrid and has been working on projects focused on cell death with cancer researcher Jialing Xiang, professor of biology. “Now we’re working to bring our treatment to the clinical-trial stage and to help these people who have no options.”

The startup has an exclusive commercialization license agreement with the NIH for the use of TP5 in multiple sclerosis, ALS, and 13 other related motor deficit-associated diseases. Funding is nearly completed for the first preclinical study phase, and Mañas Núñez says that if all goes well, GNL anticipates conducting human clinical trials in 2019.