Past, Present and Future of Neurodegenerative Diseases

GTCbio | July 6th, 2017

Neurodegenerative diseases encompass a broad array of conditions that involve progressive functional loss and neuronal cell death. In 1960s, neuroanatomists started examining neurons, glia, myelin, and synapse structure at the light and electron microscopic levels. At that time, neurology was known as a specialty in which one could diagnose, but not treat. In early 1970s, immunohistochemistry of the central nervous system (CNS) was introduced and electroencephalograms showed evidence of seizure or the slowing of neuronal function from a tumor or stroke. The discovery of the role of dopamine lead to tdetailed studies of the distribution of various neurotransmitters, including neurotransmitter precursors and neurotransmitter metabolites. The study of protein aggregates was advanced in the 1980s because of improved immunohistochemistry techniques. In late 1990s, the role of alpha-synuclein protein in Parkinson’s disease was discovered and family studies yielded more specific disease-related proteins, such as presenilin for Alzheimer’s disease. In the first decade of 21st century, much research has been focused on how and why aggregation occurs and what could be done to prevent it. Molecular chaperones play a critically important role in degrading misfolded proteins by refolding them or facilitating removal from the cell. Proteasome and autophagy are other vital cellular processes in the clearance of misfolded proteins. In this time period the importance of the accumulation, aggregation and misfolding of proteins was clearly established.

Advances in the neurosciences have positioned the field to significantly reduce the burden of nervous system disorders. Various aspects of technological advancements in CNS include nanotechnology-based drug delivery to the brain overcoming the problem of the blood brain barrier. Use of biomaterials to promote graft survival and integration as well as localized and sustained delivery of biologics to CNS injury sites is being actively pursued. Recent FDA approved drugs, such as Edaravone (Radicava) for Amyotrophic Lateral Sclerosis (ALS) and Ocrevus (Ocrelizumab) for primary progressive multiple sclerosis (PPMS), hold a great promise for drug development programs for neurodegenerative diseases. However, drug discovery and translation for CNS disorders still pose many unique challenges.

The next frontier of therapeutics for neurodegenerative diseases may comprise of genetic engineering (gene transfer using viral vectors), cell reprogramming technology (induced pluripotent stem cells, iPSCs), neural regeneration peptides (NRPs), antibodies, plant extracts, and probiotics. Ability to sequence the genetic makeup of an individual may lead to personalized treatment approaches for managing neurodegenerative diseases Furthermore, recent research in the field of Parkinson’s and Alzheimer’s diseases has revealed a role of gut bacteria, perhaps resulting in better understanding of the causes of CNS diseases.

The keynote speakers at GTCbio’s 11th Annual CNS Diseases Summit will be discussing the past, present and future or neurodegenerative diseases. The CNS Diseases summit will be held at the Hyatt Regency on September 11-12, 2017 in Boston, MA. The following are the plenary keynote speakers at this summit.

CNS Keynote Speakers

CNS Keynote Speakers


  • Dennis Steindler, Director, Neuroscience and Aging Laboratory, Tuft’s University.

Dr. Steindler’s research focuses on the growth and transplantation of normal and cancerous stem cells and his lab was the first to clone and characterize a number of these cells. Recent studies in his lab focus on testing stem cell biology and regenerative medicine therapeutics in models of brain cancer, Parkinson’s and Alzheimer’s disease. Also, his laboratory uses novel cell culture assays and biomarker screenings to screen for therapeutics including phytocompounds and precision medicine combination therapies. He has served on the editorial boards of the journals with high impact factor and serves on the scientific advisory board of the Michael J. Fox Foundation for Parkinson’s Research.

Dr. Dunlop was trained as a neuropharmacologist and is currently working to uncover the role of proteostasis mechanisms and mitochondrial dysfunction in neurodegenerative diseases, such as Alzheimer’s disease, Parkinson’s disease, ALS, and in the emerging genetics of brain disorders. He is a scientific advisor to the ALS Association, Target-ALS, the frontotemporal dementia biomarkers SAB, the Weston Brain Institute of Canada and the National Institute for Neurological Disorders and Stroke for translational programs in neurological disorders.

  • Rudolph Tanzi, Vice-Chair, Neurology Research, Massachusetts General Hospital.

Dr. Tanzi has published over 500 research papers and has received the highest awards in his field, including the Metropolitan Life Foundation Award, Potamkin Prize, Ronald Reagan Award, Silver Innovator Award, the Smithsonian American Ingenuity Award, the top national award for invention and innovation, and many others. Most recently, he and his team used Alzheimer’s genes and human stem cells to create the first true model of Alzheimer’s disease and it was coined as “Alzheimer’s-in-a-Dish” by New York Times.

We invite you to join us at the 11th Annual CNS Diseases Summit, which includes the following sessions:

Joint Plenary Sessions
I. Opening Keynote Session – Historical Perspective & Current State of the Industry
II. Round Table Discussions: Neuroinflammation, Neuroimaging & Translational Biomarkers
III. Breakfast with Mentors from Academia and Industry 
IV. Ready, Set, Grow – New Company/Technology Pitch Competition
V. Award Ceremony & Closing Keynote Session – Predictions for future

Neurodegenerative Diseases Research & Development
I. Experimental Therapeutics & Approaches in Alzheimer’s Disease & Related Dementias
II. Advances in Parkinson’s Disease Research & Therapeutics
III. Next Frontier in Multiple Sclerosis to Limit or Reverse Neurodegeneration
IV. Therapeutics for Rare & Orphan CNS Disorders

CNS Partnering & Deal-making
I. What’s New in CNS – Targets, Models, Assays, etc.
II. Industry Partnering with Academia & CROs to Advance Research
III. CNS Perspectives from Venture Capital & Public Markets
IV. Technology Transfer & Licensing: Challenges & Opportunities