Hello APBD Patients, Family Members, and Foundation Supporters:

In mid-October, one of our esteemed researchers, Dr. Orhan Akman, joined me at the NORD Breakthrough Summit in Washington, D. C. I want to share with you some important highlights.
Dr. Akman presented a poster board abstract on guaiacol, a compound that appears to be on track for an Israeli clinical trial in 2018. 

I was honored to be a presenter at the NORD members pre-conference meeting.  The program was entitled Rare Leader Panel: Engaging Medical Professionals. 

Dr. Akman and I spoke with regulatory experts and representatives of small pharmaceutical companies to get a realistic vision of the drug development process and what’s involved with bringing a clinical trial to the US.  We are facing a challenge shared by many small patient advocacy groups: Starting an extremely expensive drug development process takes precious dollars away from research. Absent infusions of funds, difficult choices will have to be made. 
We will explore every opportunity that comes to our attention. Dr. Akman spoke with people from NIH regarding potential grant funding. More conversations about funding sources will take place with the FDA, small pharma and not-for-profits. But it is really up to us
When Dr. Akman returned to his lab, I was joined by Lori Weiss, one of our community supportersLori and I attended a special meeting for the 20 rare disease organizations that are receiving grants and guidance for the FDA-supported NORD program establishing natural history study registries. 
The power and the promise of natural history registries cannot be overstated. When it comes to a clinical trial for a disease which has variability in its evolving symptoms, it is vital that clinical end points be identified in order to know if a particular approach or compound is actually effective. APBD patient community participation in the FDA-NORD natural history study (FAN) could give our researchers a critically needed tool. 
Developing a natural history study of APBD patients could be that the most important goal for our foundation and community. 

Now is the time to donate your work, wisdom, and wealth.  WHAT CAN YOU DO?
1. Jojo and Chloe are running the New York City Marathon to raise funds for the APBD Research Foundation. PLEASE donate any amount to show your support. Additionally, our Executive Director Sharon Steinberg is ready to discuss with you how we might bring a fundraiser to your community.
2. If you’re a patient, make sure that you enroll in both the Columbia APBD registry (CAP) and the FDA and NORD (FAN) natural history study registry They accomplish different things. 

You’ll find links to the two registries and the 2017 NYC Marathon Fundraiser on our foundation’s homepage: www.apbdrf.org  

We’re open to your ideas! Just call or email.

All the best, 
Jeff Levenson