Orphan diseases programs

Four IMSA alumni working in the field of orphan disease research came to IMSA to share their findings and stories as panelists in the IMSA Great Minds Program® Alumni Panel on Orphan Diseases. The program was sponsored by IMSA’s Alumni Association (IAA), the student A.X.O.N. Neurology Club, the IMSA Development Office, the IMSA Great Minds Program and the student advocacy group Shine On.

Dr. Jennifer Ellis Ward '93
IMSA Great Minds Program Alumni Panelists with IMSA Principal Dr. Eric McLaren
IMSA President Dr. Max McGee

An orphan disease is a rare disease which has not been "adopted" by the pharmaceutical industry because there are not enough patients to make drug development cost-effective. An orphan disease also can be a disease which is rare in the industrialized world but common and largely ignored in the developing world. These diseases face special challenges in attracting public attention, research and funding. Examples of serious orphan diseases are amyotrophic lateral sclerosis (ALS), cystic fibrosis and Tourette’s syndrome.

To encourage industry to pursue treatments for rare diseases, Congress passed the Orphan Drug Act (ODA) in 1983 to provide financial and regulatory incentives to develop and market therapeutics for diseases that affect less than 200, 000 Americans. Since the passage of the ODA in 1983, the FDA has approved more than 350 therapies for orphan diseases. Despite this progress, there are still an estimated 6, 000 – 7, 000 (or more) orphan diseases in need of effective treatments.

During the presentation, the following alumni panelists shared their perspectives, stories and research findings with students, staff and guests:

Dr. Irfan Qureshi ‘94, an assistant professor in the Department of Neurology and an investigator for Brain Disorders and Neural Regeneration at the Albert Einstein College of Medicine.

Dr. Jennifer Ellis Ward ‘93, a post-doctoral fellow in the Hematology/Oncology Section at the Boston University School of Medicine.

Dr. Michael Wilson ‘94, a neuro-infectious disease fellow at Massachusetts General Hospital and at Boston University’s National Emerging Infectious Diseases Laboratories (NEIDL). NEIDL is the federal government’s newest biosafety level 4 facility, designed to study the most dangerous and deadly infectious agents.

Dr. Meiye Wu ‘94, a staff scientist in the Department of Biotechnology and Bioengineering at Sandia National Laboratory in Livermore, CA.

In addition, David Lockhart ’90, a biostatistician, visited with students throughout the day to share his knowledge. Lockhart is the author of several peer-reviewed articles analyzing data on HIV, AIDS, Alzheimers, cardiac radiology and many other topics.

Dr. Ward, who developed her interest in research as an IMSA student, spoke about her interest in ‘re-purposing old drugs’ for new uses. In addition to being more cost-effective, re-purposing old drugs can also save time in the fight to find cures to orphan diseases.

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