Three filmmakers are raising funds for a feature-length documentary about how science and the rare disease community’s needs came together to make Biogen’s Spinraza the first approved treatment for spinal muscular atrophy (SMA).
Their month-long, $25,000 Kickstarter campaign ends in four days, at 2:48 p.m. Sunday, Oct. 22. If they fail to reach their goal, they will return the donations. They’re close, at nearly $20,000. They just need an extra push.
Artemis Joukowsky, Cray Novick and Gareth Burghes are inviting anyone interested to listen to a discussion about the film at 10 a.m. Thursday U.S. Eastern Standard Time on Facebook Live. It will be on two Facebook pages, one titled Disorder: The Rare Disease Film Festival Facebook and another titled “The Cure?.”
The talk will feature Burghes and the founders of the film festival, Daniel DeFabio and Bo Bigelow, both fathers of children with rare diseases. The video will remain up for anyone to watch afterward, Burghes said.
Burghes worked in his father’s lab when he was younger. His father’s work with SMA clinical trials prompted him to direct a 24-minute documentary called “Life & Atrophy.”
It’s about 5-year-old Miles McIntosh, who was born with SMA type 2, and his parents as they sign Miles up for a clinical trial to test Spinraza, which at the time had yet to obtain Food and Drug Administration approval. The agency authorized the drug, which works by replacing a protein missing in SMA, in December of 2016.
Patients with SMA are born without the SMN1 gene. Instead, they have two copies of survival motor neuron 2 (SMN2).Unfortunately there is a problem with a piece of protein in SMN2. Spinraza uses an antisense oligonucleotide, which is a synthetic material that replaces the missing protein in SMN2. This results in an increased amount of the SMN protein.
“Life & Atrophy” was shown at the first Disorder: The Rare Disease Film Festival in Boston this month. This is where Burghes met Joukowsky and Novick, who had already started the Kickstarter campaign for their own feature-length documentary.
Burghes had been working on a film that delved more into the science behind SMA treatments. The three decided to join forces to save about two years of filmmaking.
In an interview with SMA News Today, Burghes said the loose timeline for the film, which has a working title of “The Cure?,” is about two years, depending on the budget. The $25,000 in Kickstarter donations will get the project rolling, but will only go so far, he said. Still, it will help capture on film special moments with SMA families and SMA treatment researchers.
Those moments could be the first “whiff of magic” that Burghes says is possible in all films, the stuff that makes a movie special.
“There’s always a certain magic to cinema,” he said. “With documentary, you can put yourself in the right place at the right time to capture that magic that helps make a film a film.”
This is the first phase of funding if all goes according to plan, Burghes said. “We do want to raise more money and workshop the film so it can be shown far and wide,” he said. “SMA is a very specific disease, but all the human elements and the science elements are very universal.”