Presented by the Alzheimer's Drug Discovery Foundation

February 1-2, 2010  ●  Houston, TX - USA



January 31, 2010

5:00 – 7:00 pm

On-Site Pre-Registration

February 1, 2010

7:45 – 8:30 am

Registration & Continental Breakfast

8:30 – 8:40

Overview of Drug Discovery for Neurodegenerative Disease

Howard Fillit, MD, Alzheimer's Drug Discovery Foundation    read bio

8:40 – 9:00

Stem Cells to Accelerate Drug Discovery for Neurodegenerative Disease

Lee Rubin, PhD, Harvard University    read bio

I. Basics of Medicinal Chemistry


Chair: D. Martin Watterson, PhD, Northwestern University   read bio

The goal of this session is to review fundamentals of medicinal chemistry relevant to drug discovery for CNS disorders. Although the focus is on small molecule drugs, due to the extensive state of knowledge, general themes relevant to peptide and protein therapeutics will also be discussed. The attendee should leave the session with a practical, working familiarity of contemporary approaches and key issues that should be considered by interdisciplinary teams as they take hits to lead compounds to candidate small molecule therapeutics.

9:00 – 9:20

Overview: Medicinal Chemistry Rules of Thumb, Myths and Realities in CNS Drug Discovery

D. Martin Watterson, PhD, Northwestern University   read bio

9:20 – 9:30


9:30 – 10:00

Designing Small Molecules with Increased Potential for CNS Penetration - (slides)

Laura Chico, MS, PhD, LKC Pharma Services   read bio

10:00 – 10:10


10:10 – 10:40

Synthetic Chemistry Essentials for Biologists - (slides)

Heather Behanna, PhD, Astellas Research Institute of America  read bio

10:40 – 10:50


10:50 – 11:05


II. In-vitro Testing: Early Phases of Drug Discovery


Chair: Marcie Glicksman, PhD, Harvard NeuroDiscovery Center   read bio

A key component of the development of new therapeutic agents is the identification of molecules that can serve as initial lead structures on which drug discovery programs can be built.  High-throughput screening of large collections of drug-like molecules for modulatory activity in disease-relevant assays is an important means to discovering these lead molecules.  This session will first address strategies for selecting your target, then the development of assays that are suitable for high-throughput screening and for secondary assays to validate the primary screening results.  Most drug development efforts fail due to toxicity and ADME (absorption, distribution, metabolism and excretion) properties. This session will end with a presentation on technology used for in vitro toxicity and ADME testing.


11:05 – 11:10

Session Overview – Marcie Glicksman, PhD, Harvard NeuroDiscovery Center 

11:10 – 11:35

Developing Relevant High-Throughput Assays for the Identification of Potential Drug Candidates

Marcie Glicksman, PhD, Harvard NeuroDiscovery Center  

11:35 – 11:45


11:45 am – 12:10 pm

Role of In Vitro Models in Drug Discovery for Neurodegenerative Disease (slides)

Lisa Minor, PhD, Johnson & Johnson   read bio

12:10 – 12:20


12:20 – 1:20


1:20 – 1:35

In Vitro Toxicity Testing: What, Why & How

Karen L. Steinmetz, PhD, DABT, SRI International    read bio

1:35 – 1:45


III. Pre-Clinical Proof-of-Concept & Development


Chair: Edward Spack, PhD, Fast Forward LLC   read bio

Preclinical development, the phase of drug development between discovery and clinical trial, is often referred to as the “Valley of Death”.  Several factors contribute to this barren landscape.  Many promising leads fail due to problems of formulation, delivery, bioavailability, scalable manufacturing, or safety.   Investigators seeking to navigate past these hazards also encounter challenges in funding, resources, and expertise.  This session offers an overview of the steps involved in optimization of a lead compound and in vivo efficacy and safety studies that determine dosing strategies.  Presentations will include examples of go/no-go stage decision points, funding resources, and outsourcing options to complete the journey of discovery to an Investigational New Drug (IND) application for clinical trial.


1:45 – 1:50

Session Overview – Edward Spack, PhD, Fast Forward LLC

1:50 – 2:15

Requirements for a Lead Compound to Become a Clinical Candidate (slides)

Nancy Wehner, PhD, Nancy Wehner Non-Clinical Consulting Services   read bio

2:15 – 2:25


2:25 – 2:50

Behavioral Testing in Neurodegenerative Disease

Taleen Hanania, PhD, PsychoGenics    read bio

2:50 – 3:00


3:00 – 3:15


3:15 – 3:40

Regulatory Requirements & Strategy

Joy Cavagnaro, PhD, Access BIO   read bio

3:40 – 3:50


3:50 – 4:00

The Basics of Pre-Clinical Development

Edward Spack, PhD, MS Fast Forward    read bio

4:00 – 4:10


4:10 – 4:20

Closing Remarks

Howard Fillit, MD, Executive Director, ADDF    read bio

4:20 – 6:30


February 2, 2010

8:00 – 8:30 am

Continental Breakfast

8:30 – 8:35

Welcome & Opening Remarks

Diana Shineman, PhD, Alzheimer's Drug Discovery Foundation                

IV. Issues in Technology Transfer: Interactions and Intellectual Property


Chair: Kathleen Denis, PhD, Rockefeller University    read bio

This session will focus on the interactions among academic researchers, their technology transfer office and industry partners.  An introduction will discuss the various roles and responsibilities of all of the parties involved and hope to begin to demystify academic – industry relations.  The basics of patents will be presented with an emphasis on what they can and cannot successfully cover, as well as what a researcher needs to do to maximize the chances of a positive outcome.  A variety of agreements used in academic – industry relationships will also be discussed, with an emphasis on the importance of creating a good relationship amongst all parties.  The session will continue with an honest appraisal of the good, the bad and the ugly of new company formation in the biotech industry, and finally close with an industry perspective on dealing with academic institutions.


8:35  – 8:40

Session Overview – Kathleen Denis, PhD, Rockefeller University

8:40 – 9:10

Working Effectively with Your TTO: Roles and Responsibilities

Kathleen Denis, PhD, Rockefeller University

9:10 – 9:20


9:20 – 9:50

Intellectual Property 101: A Primer For Investigators

Colin G. Sandercock, Perkins Coie    read bio

9:50 – 10:00


10:00 – 10:30

Creating Relationships with Industry: Consulting, Research, MTA's and Patent Licensing

Louis Berneman, EdD, CLP, Texelerate    read bio

10:30 – 10:40


10:40 – 10:55


10:55 – 11:25

Should You Start a Biotechnology Company?

John S. Swartley, PhD, University of Pennsylvania    read bio

11:25 – 11:35


11:35 am – 12:00 pm


What Companies Look for in a Licensing Partner

Susan P. Rohrer, PhD, Merck Research Laboratories   read bio

12:00 – 12:10


12:10 – 1:00


V.  Case Studies


Chair: Todd Sherer, PhD, Michael J. Fox Foundation for Parkinson's Research    read bio

This session will consist of parallel breakouts focused on different aspects of the drug development pipeline.  Within each track, a disease-specific case study will be used to illustrate individual aspects of the drug development process.  Track 1 will focus on Target Validation and Optimization using a case study from Alzheimer’s disease.  Track 2 will cover the pre-clinical to IND enabling process with an example from Parkinson’s disease.  Track 3 will focus on specific aspects relevant to the development of a biologic therapy using a case study from MS.  Each parallel session will include a presentation of the disease-specific case study followed by a panel discussion of the relevant issues.  The goal of this session is to provide meeting attendees with tangible examples from relevant neurodegenerative diseases for addressing critical roadblocks in the drug development pipeline.


1:00 – 1:10

Session Overview - Todd Sherer, PhD, Michael J. Fox Foundation for Parkinson's Research

1:10 – 2:10

Track 1:


Target Validation to Lead Optimization


AD Case Study


Track 2:


Pre-Clinical to IND Enabling


PD Case Study


Track 3:

Rational Design and Development


MS Case Study

Moderator and Presenter:

Frank Longo, MD, PhD

Stanford University    read bio

Moderator and Presenter:

Colleen Niswender, PhD, Vanderbilt University    read bio

Moderator and Presenter:  

Gary Olson, PhD, Provid Pharmaceuticals Inc.    read bio

Panel Members:

Panel Members

Panel Members

Neil Buckholtz, PhD

Marcie Glicksman, PhD

Howard Fillit, MD

Taleen Hanania, PhD

Larry Refolo, PhD

Todd Sherer, PhD

Nancy Wehner, PhD

Edward Spack, PhD

Tim Coetzee, PhD   

VI. Resources and Services For Advancing Drug Discovery


Chair: Lorenzo Refolo, PhD, National Institute on Aging    read bio

This session will focus on descriptions of the resources available through a variety of mechanisms within academia, the National Institutes of Health (NIH), foundations and commercial vendors.  Speakers will focus on resources for assay development, target identification, drug discovery, drug development, pre-clinical toxicology evaluation and other components needed for the translation of pre-clinical drug candidates into potential therapies tested in clinical trials. In particular, it will include specific descriptions of programs available to academic investigators through individual NIH Institutes, including the National Institute on Aging (NIA) and the National Institute of Neurological Disorders and Stroke (NINDS), as well as trans-NIH programs including the NIH Roadmap for Medical Research and the NIH Blueprint for Neuroscience Research.

2:10 – 2:15

Session Overview – Lorenzo Refolo, PhD, National Institute on Aging

2:15 – 2:40

How to Outsource Early Drug Discovery (slides)

Mark Creswell, PhD, IDSC Biotech Network    read bio

2:40 – 2:50


2:50 – 3:15

Resources at the National Institute of Health (slides)

William Matthew, PhD, National Institute on Neurological Disorders and Stroke and Neil Buckholtz, PhD, National Institute on Aging     read bio

3:15 – 3:25


3:25 – 3:50

Types of Academic Drug Discovery Programs

Euan Ramsey, PhD, Centre for Drug Research and Development     read bio

3:50 – 4:00


4:00 – 4:30



Foundation Resources

Venture Philanthropy—Howard Fillit, MD, ADDF    read bio

Other Philanthropic Approaches  — Timothy Coetzee, PhD, Fast Forward    read bio

Funding and other challenges for rare diseases (slides) — Leticia M. Toledo-Sherman, PhD, CHDI Foundation    read bio

4:30 – 4:40


4:40 – 4:50

Closing Remarks

Howard Fillit, MD, Executive Director, ADDF    read bio







www.alzdiscovery.org © 2009 ADDF