DAY #2
8:30am: Check-in & Breakfast
9:00 – 10:00am: Panel Discussion: Revolutionizing Clinical Trials with Video – If a Picture is Worth a Thousand Words, a Video is Worth a Million
To make clinical trials for CMT successful, we need new ways to measure how the disease affects people. One idea is to use videos to see how it affects things like walking and step length. Using videos could allow us to use footage from the past or even videos taken by regular people, not just clinic experts. Given the increased use of telemedicine, video may be a useful addition to clinical trial assessments.
Klaus Romero, MD, MS, FCP, CEO, Critical Path Institute
Allison Moore, HNF
Tina Duong MPT, PhD – Stanford Medicine
Michelle Campbell, PhD FDA, Associate Director, Stakeholder Engagement & Clinical Outcomes, Office of Neuroscience
Roundtable Discussions
10:00 – 10:25am CMT Lounges: In-Person Support, Vendor Resources & Socializing!
CMT Social – Meet & Greet w/ Vendor Tables
CMT Adaptive Exercise – Julie Stone (Aurora Room)
10:30 – 10:50am Presentation: What Does it Take? A Fighter Mom’s Journey from Kitchen Table to Injection!
Learn how “fighter mom” Lori Sames raised the necessary funds and curated the qualified experts to tackle a gene therapy for Giant Axonal Neuropathy (GAN), an inherited neuropathy. Topics covered will include lessons learned, accelerating progress, cost-effective strategies, and regulatory considerations. Hear from Lori about the next steps needed to cross the finish line and how this will transform the future of gene therapies in CMT.
Lori Sames, Founder Hannah’s Hope Fund
11:00 – 12:25pm Panel Discussion: What’s Next for Gene Therapies in CMT?
How does the drug development process work and how long does it take for a drug to get from a research lab to your pharmacy? What is gene therapy and why is it important to develop treatments for specific CMT types? What are the pros and cons of gene therapy? What is currently in the gene therapy pipeline for CMT?
You’ll learn the good, the bad and the ugly of gene therapy during this overview of the various approaches. Topics will include targets and delivery options and which subtypes have the best chance of success, Conventional therapeutic approaches vsx gene therapy, can patients participate in both? Other questions answered during this session will include: How does the FDA view gene therapies for CMT? What are the pros & cons, what are the obstacles, and is it right for you? Patients and stakeholders will walk away with the basic knowledge & understanding of what it takes to develop a gene therapy for CMT.
Sharon Hesterlee, PhD, Chief Research Officer, Muscular Dystrophy Association, (Presenter & Moderator)
Sabrina Yum, MD CHOP (Presenter/Panelist)
Brian Lin, MDA (Presenter & Panelist)
Lori Sames, Founder, Hannah’s Hope Fund (Panelist)
Audience Q & A
12:30 – 1:15pm Lunch
1:15 -2:15pm
For Industry & Researchers Only: Surrogate Biomarkers
Matthew Jarpe, PhD, HNF Scientific Advisor
Anna Pfalzer, Phd, COMBINEDBrain
For Patients & Caregivers: Workshop Managing Mental Health & CMT
From general coping mechanisms to nuanced discussions on self-love, romantic relationships, and family dynamics, our agenda is packed with insightful content. Break into small groups, share your experiences, and gain valuable insights from others who understand the CMT journey. Explore mental health strategies tailored for living with CMT, addressing unique challenges and fostering resilience.
Julie Stone, Founder, All Bodies Community
Estela Lugo, Program Development Manager, HNF
2:30 – 4:00pm Presentation: Overview: Upcoming CMT Pipeline & Clinical Trials
Hear from HNF’s Therapeutic Research in Accelerated Discovery (TRIAD) partners about their latest research milestones and what’s next in the pipeline for CMT.
Gideon Shapiro, PhD, Founder/CSO, Miralinc Pharma
Matt Jarpe, PhD
4:00- 4:30pm Philanthropic & Investment Opportunities
4:30pm Closing Remarks
6:00pm HNF Cocktail & Dinner Party
DAY 1
8:00am Registration & Breakfast
9:00 – 9:20am Keynote Address: “From NEW Gene to Phase 3 Clinical Trial – Treatment is Within Reach!”
Shoshana Shendelman, PhD
CEO & Founder, Applied Therapeutics
9:30 – 10:45pm: Panel Discussion: CMT Biobank & Biomarkers for CMT Therapy Development
A biomarker may be used to predict which patients will respond to treatment or see how well the body responds to a treatment for a disease. Without them, clinical trials may enroll patients who will have a lower chance to respond, or they rely on clinical evaluations that may not be sensitive enough to capture change (good or bad). In this session, you’ll hear from experts in the field on the importance of identifying and validating biomarkers for use in CMT therapeutic development. What’s on the horizon, and how is a patient-led biorepository advancing the development of evidence-based biomarkers for all CMT subtypes?
Matthew Jarpe, PhD, HNF Scientific Advisor (Presenter/Moderator)
Shoshana Shendelman, PhD, CEO & Founder, Applied Therapeutics
Anna Pfalzer, PhD, COMBINEDBrain (Presenter/Panelist)
Roundtable Discussions & Feedback Sharing
10:45 – 11:00am Break
11:00 – 12:15pm Panel Discussion: Innovating Digital Endpoints with Wearable Technology
The identification and development of digital measures are vital to monitoring CMT disease progression or improvement in real-world settings. By collecting remote data (not in a clinical setting), researchers, industry, and regulatory agencies can better understand the daily impact of CMT on people living with the disease. This panel session will cover the development of methods to identify digital measures of CMT disease from wearables to generate clinically meaningful patient outcomes. Participants will have the option to participate in a digital wearable pilot study sponsored by BioSensics and HNF.
Klaus Romero, MD, MS, FCP, CEO, Critical Path Institute (Moderator)
Kayla Cornett, PhD, University of Sydney (Presenter/Panelist)
Ashkan Vaziri, PhD, Founder and CEO, BioSensics™ (Presenter/Panelist)
Audience participation Q & A
12:15 – 1:00pm Lunch
1:00 – 1:45pm: “Making Sense & Science of CMT Symptoms”
“Is this symptom related to CMT?” This is a frequent question asked by many individuals living with CMT and their loved ones. Most of us are familiar with the most common symptoms of CMT, such as drop foot & hand weakness. However, many less-recognized symptoms can be just as disruptive to our lives but have not yet been studied or linked to CMT. This is why in 2013, HNF started the Global Registry for Inherited Neuropathies (GRIN) and the CMT Natural History Study.
Joy Aldrich, HNF (Presenter & Panelist)
Robert Moore, GRIN Data Manager, HNF
Jason Colquitt, CEO, Across Healthcare
Roundtable Discussions & Feedback Sharing
1:45 – 2:15 The Power of Sponsored Genetic Testing
The yield of sponsored genetic testing. The relative prevalence for different CMT types, and frequency of VUS in this patient population.
Constantine Farmakidis, MD, University of Kansas Medical Center
2:15 – 2:45pm CMT Lounges: In-Person Support, Vendor Resources & Socializing!
CMT Social – Meet & Greet w/ Vendor Tables
CMT Adaptive Exercise – Julie Stone (Aurora room)
2:45 – 3:15pm Presentation: Is CMT Surgery Right for You?
Join us for an enlightening summit session titled “Is Surgery Right for You” with renowned orthopedic surgeon Glenn Pfeffer, MD. As Director of the Foot and Ankle Center at Cedars-Sinai Medical Center in Los Angeles, Dr. Pfeffer brings over 30 years of experience in treating foot and ankle problems in patients with CMT and has performed over 1000 CMT foot surgeries.
Glenn Pfeffer, MD, Cedars-Sinai
3:20 – 4:45pm: Panel Discussion: Identifying Roadblocks to Success for CMT Clinical Trials
This interactive panel will shed light on the unique challenges faced by CMT clinical trials, including the diverse subtypes & symptoms, limited clinical experience, patient access, investment, and complementary outcome measures that are sensitive enough to measure change in a relatively short period of time. You’ll learn about evidence-based, innovative strategies and solutions that may apply to CMT for enhanced efficacy and the future success of CMT clinical trials. The open format will allow for patient engagement interaction to better define how these potential solutions, such as adaptive and decentralized trials, including telemedicine, patients & caregiver needs, real-world evidence (RWE) data capture (patient registries, collaborative networks & wearable & tech devices) may have an impact in supporting the success of these trials.
Shannon Strom, PhD, RAC, Drug Development Expert (Moderator)
Michelle Campbell, PhD FDA, Associate Director, Stakeholder Engagement & Clinical Outcomes, Office of Neuroscience
Tim Estilow, OTR/L, CHOP (Speaker/Panelist)
Dottie Caplan, SVP Patient Advocacy and Engagement, Applied Therapeutics (Panelist)
Q&A
4:45 – 5:00pm: Closing Remarks
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