December 8, 2021
Dear Friends,
Since 2000, The Robert Packard Center for ALS Research at Johns Hopkins (Packard Center) has been at the forefront of Amyotrophic Lateral Sclerosis (ALS) research. Their unique model employs a rapid -funding process to support novel ideas and high risk, high reward projects. Within the next two decades, experts predict that neurodegenerative diseases (including ALS, Alzheimer’s, and Parkinson’s disease) will overtake cancer as the second leading cause of death after cardiovascular disease. The work done by the Packard Center will be critical in combatting this alarming statistic.
Progress.
Developing treatments for ALS starts with gaining a better understanding of the underlying molecular pathways that drive disease. The Packard Center continues to move forward. One of the greatest challenges in ALS is finding biological pathways and drugs specific to sporadic ALS (accounting for approximately 90% of ALS cases). The Packard Center’s Answer ALS program has been instrumental in advancing this progress, by providing over 1,000 ALS-patient derived stem cell lines to study the disease. Because of that resource, in the past year Packard-funded researchers have begun to unravel the mystery of how dysfunction in the nucleus of ALS patient brain cells begins the deadly series of events leading to ALS.
The Center’s founder and director, Dr. Jeffrey Rothstein (a recognized expert and leader in this field) along with a group of talented collaborators in academia and the pharmaceutical industry, recently published a groundbreaking study in Science Translational Medicine that identified some of the earliest cellular steps in ALS, in which the molecules can’t move between the nucleus and cytoplasm. This triggers a molecular cascade of events that leads to ALS. In addition, they have developed a drug that can repair this process—a ray of hope for future clinical trials.
Understanding these basic cellular mechanisms provides hope for tomorrow’s therapies. Packard’s success is due in large part to our spirit of collaboration, and their sharing of data and resources. They are closer than ever to having a disease-altering therapeutic for patients. Then as now, the hope is in the science.
You make a difference in the fight against ALS and other neurogenerative diseases by supporting the Packard Center. To donate, please visit www.packardcenter.org/give
Thank you for your consideration and for your interest in joining the fight against ALS and other neurodegenerative diseases. For any and all questions (about the science or making a gift), please contact Meg Whiteford (mwhiteford@jhmi.edu or 410-955-8684) and tell her I sent you.
Sincerely,
Steven R. Gerbsman
Blog of Intellectual Capital 