Dr. Robin A. Ely, Clinical/Science Advisor, Director of the National Gaucher Foundation (NGF) Global Diagnostic and Treatment Initiative, and NGF Co-Founder, recently presented at the WORLDSymposium Conference. Here, she shares her experience at the event and her work on behalf of NGF.

I recently returned from the 21st annual WORLDSymposium conference, held in early February in San Diego. WORLD, which stands for “We’re Organizing Research on Lysosomal Diseases”, is a yearly research conference dedicated to lysosomal diseases that brings together more than 2,000 participants from 50 different countries.

Topics covered at WORLD include numerous disorders, including Gaucher disease. It’s a packed, 4-day conference whose mission is to “provide an interdisciplinary forum to explore and discuss specific areas of interest, research and clinical applicability related to lysosomal diseases”.

During the event, new research is introduced during lectures and poster presentations, and awards are given to individuals whose research stands out.

Perhaps most importantly, WORLD is a place where colleagues, peers in both basic and translational science, and clinicians can meet in person, form new connections, and fortify already existing relationships.

This year, I met with members of the NGF Medical Advisory Board as well as scientists and physicians from numerous countries. I had many fruitful interactions. Throughout the event, I was focused on discussing early diagnosis and when to initiate treatment, which is a major subject of debate amongst Gaucher Specialists.

My presentation at WORLD discussed the survey that Dr. Neal Weinreb and I conducted on the use of cannabis products for the relief of symptoms not resolved by either ERT or SRT. Here were the aims of our survey:

Using an investigator-designed survey tool to confirm that adult patients with type 1 Gaucher disease (GD1) often self-prescribe cannabis products to try to alleviate symptoms such as lingering fatigue, chronic bone and joint pain, loss of energy, anxiety, and depression that persist despite enzyme replacement therapy (ERT) or substrate restriction therapy (SRT). Additionally, to explore whether patient reports of symptom relief and adverse side effects relate to frequency and duration of cannabis use.

We found that 39% of our respondents use cannabis, some with very good benefits, and there was meaningful interest in the topic by a number of individuals. Click here to review the paper and view the results.

Overall, the conference was a success, and I’m glad that I attended.