Sanofi, Shire, Vertex and more back first-ever rare disease film festival

Lucas DeFabio stars in one of the films set to be shown at the new rare-disease film festival, co-founded by his father, Daniel DeFabio, and Bo Bigelow.

The newest stop on the film festival circuit is one with entries that are all about rare diseases.

Disorder: The Rare Disease Film Festival is the first of its kind in the U.S., with pharma sponsors including Shire, Sanofi Genzyme, Vertex, Horizon and Alnylam, and will debut in Cambridge, Massachusetts, on Oct. 2. The two-day event will feature 30 different films about two dozen different rare diseases, with run times ranging from 1 minute to 65 minutes.

Walking the purple carpet of the festival will be the directors, families, actors and advocates affected by the challenges of rare diseases. Two of those directors are the founders of the festival, Daniel DeFabio and Bo Bigelow, both fathers of children with rare diseases.

DeFabio’s documentary is “Menkes Disease: Finding Help & Hope,” narrated by actress Mary McDonnell and starring his son Lucas as one of three boys featured who have the condition, which prevents boys from metabolizing copper. Bigelow's film, "Tess is Not Alone," is about his daughter Tess, one of only 10 people in the world with the USP7 genetic mutation.

The two men met at an advocacy conference and in talking realized they were both thinking the same thing. How could they combine the best of the excitement and mainstream reach of traditional film festivals with the more relevant physician and researcher audiences at costlier medical conferences?

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“When we get all the right people in the room seeing these stories—usually the stories are moving and powerful—they spark conversations,” DeFabio said in an interview. “The best-case scenario of what comes out of those conversations is someone researching a disease says, ‘Oh, this sounds similar to my work, maybe I could apply it to this new disease that I never thought about before.’"

So far, the festival is one-third sold out, with a broad cross-section of attendees from pharma, biotech and research industries as well as physicians, geneticists, nurses and patient families and advocates. Alongside the films, Disorder will host researcher, scientist and advocate chats with the audiences. Shire and Sanofi, as leading sponsors, will present opening-day presentations on the first and second days.

“When you do something like this festival, you create an umbrella that really leverages everyone’s interests together. So yes, somebody shows up because they’re concerned with one rare disease, but the crowd that they bring for that purpose sees the messages of all the other films. The cross-pollination and the collaboration that comes out of that I think is a great way to increase the opportunities for all of us,” DeFabio said.

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According to DeFabio, the festival organizers “were amazed” at the response to their call for entries and the number of submissions they received. And while the festival is more somber in tone than typical film confabs, there is still star power involved. One film on the slate, called “The Magic Bracelet,” has a cast that includes well-known actors J.K. Simmons, Hailee Steinfeld, James Van Der Beek and Bailee Madison. McDonnell, as noted above, narrates DeFabio’s film, and actor William H. Macy narrates “This is Michelle," a short about epidermolysis bullosa.