12:50 - 1:10 pmSaturday, September 6
LK 130
ALD Connect: Orphan disease patient advocacy meets medical consortium
LK 130
ALD Connect: Orphan disease patient advocacy meets medical consortium
Executive Director, ALD-Connect
Ben is founder and acting Executive Director of ALD Connect, a patient powered research network for the rare neurometabolic disease X-linked adrenoleukodystrophy. Ben himself is a patient, having been... Read more

Description

Ben is founder and acting Executive Director of ALD Connect, a patient powered research network for the rare neurometabolic disease X-linked adrenoleukodystrophy. 

Ben himself is a patient, having been diagnosed in 2011 at age 46 with a late-onset phenotype.

Advocacy efforts:

- We, ALD Connect, have taken a leading role in advocating for the inclusion of X-ALD in the newborn screening blood panel. We have built a public policy dossier on this measure. It contains a family survey, a medical justification, and a cost/benefit analysis comparing lifetime cost of care for late-diagnosed or undiagnosed patients vs. patients diagnosed as newborns. The dossier contains a feasibility analysis of volume screening using commercial off-the-shelf mass spectrometry tools. We also developed FAQs to counter some common objections from physicians working in the Lysosomal Storage Disorder space. 
 
- Newborn X-ALD screening is now implemented in the State of New York, with Minnesota, Massachusetts, Maryland and California all actively assessing legislative proposals to that effect.
 
- ALD Connect is building an eCRF infrastructure in the US and in close collaboration with the European Adrenoleukodystrophy Board (EAB). The data sharing allows Centers to retain data ownership while aggregating datasets on legacy and prospective data for insights into demographics and phenotypes.
 
- Patients will also be invited to consent to, and volunteer self-reported clinical data. They will be profiled based on this data to gauge interest, and subsequently enroll, in targeted clinical trials. We currently are building protocols for two new clinical studies on adult patients with the adrenomyeloneuropathy (AMN) phenotype, using proprietary antioxidant molecules, neurological tests, and new brain imaging techniques developed at Stanford.
 
- Unlike Europe which has a unified patient advocacy collective (the European Leukodystrophy Association - ELA), the US suffers from fragmented patient advocacy groups with limited impact. The United Leukodystrophy Foundation (ULF), StopALD, Myelin Project, ALD foundation are just a few examples. ALD Connect is a Patient Powered Research Network, an umbrella organization between patient advocacy groups and a new medical research consortium. ALD Connect intends to federate many of the current patient forums (ULF's Inspire bulletin board, Facebook group, HealthUnlocked) into a system for aggregating medical profiles, sending out targeted surveys and questionnaires, and enrolling patients into clinical trials. The ALD Connect platform already offers educational resources, including a 10-minute intuitive tutorial produced by Youreka Science, with more on the way for exercise, locomotive aids, post-diagnosis decision tree, etc.
 
- We have intentionally designated two distinct platforms for patients and clinicians, both accessible via a common web portal.  The common portal is home to publicly viewable, educational material produced by physicians, scientists, and advocates for general use of the ALD community.  The clinical trial/clinician network was designed to stand separately from the patient social network to allow the patient community a degree of privacy and autonomy with clear boundaries distinguishing what is intended for research and what is intended for patient self-betterment, leisure, and casual interaction.  In contrast with the prevailing paradigm of “all your information belongs to the network managers”, the patient social network is intended first and foremost as a service to the patient; it will allow social engagement, fellowship, and conversation that will not be data-mined. The research-oriented tools on the site will be clearly demarcated and voluntary. These tools will include self-tracking features and survey-reporting, with the purposes and disclosures explicitly stated. In sum, this standalone structure for the patient social network allows the patient community a clear (and very real) sense of privacy, autonomy, and trust that cannot be achieved within existing clinical trial databases, while still quantifying a patient’s subjective sense of health (e.g. self-reported outcomes), values (e.g. clinical trial design preferences), and priorities (e.g. most troublesome symptoms).
 
- Transparency in terms of funding is a topic we take very seriously, as some of our funding comes from the biotech industry. Our BoD adheres to strict conflict of interest rules, and our industry funding is applied only to building our general infrastructure.
 
- Privacy is paramount, with ALD Connect's active participation in the PCORI Privacy Task Force, using best practices from NIH Research Rules and HIPAA, and implementing our own data security, ethics and regulatory compliance rules.
 

Ben Lenail is a founding officer of ALD Connect, a research consortium focused on the neuro-metabolic disease X-linked adrenoleukodystrophy (X-ALD).

Ben is currently Director of Business Development at Alta Devices, a solar energy startup in Sunnyvale. He was previously a distinguished visiting scholar at Stanford University, and Senior Director of Corporate Development at Sun Microsystems. Ben has a Bachelor’s from Sciences Po (Paris, France) and an MBA from the University of Washington with a concentration in Finance and Operations Management.

Among his significant experience in the nonprofit world, Ben is currently on the Executive Committee of Silicon Valley Social Ventures (SV2). Ben served on the Board of the International School of the Peninsula for seven years, including a year as the head of the Development Committee. He was the Northern California ambassador for Agros International, leading several service trips to Central America and recruiting families to sponsor a farming cooperative in Nicaragua.

Additionally, Ben has served on several committees including the Skyline Committee of the Peninsula Open Space Trust, and the Citizens’ Oversight Committee for the Palo Alto Unified School District.

In his free time, Ben enjoys spending time with his family and his beloved Golden Retriever Hazel, cycling, and walking.

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