It’s the height of summer, temperatures are soaring toward searing triple digits, depending on what part of the world you live in, and seeing as I live in the South it’s disgustingly humid to boot. I don’t know about you, but I could go for a cool bucket of ice water right about now (or a cool anything for that matter). If that sounds faintly familiar, think back to 2014 and you might remember your social media feeds blowing up with celebrities and friends alike participating in the Ice Bucket Challenge. The challenge saw over 117 million people dump a bucket of ice water over their heads to raise awareness for amyotrophic lateral sclerosis (also known as Lou Gehrig’s disease), while also raising money for research. The cynical sneered, and articles maligned the “slacktivism” as a mere publicity stunt, but then something funny happened. In a matter of six weeks, the Ice Bucket Challenge became a global phenomenon, splashing across heads and headlines and raising a staggering $115 million for the ALSA. About $77 million of that will go directly into research, with another $23 million will go into patient and community services, and $10 million will go to public and professional education.
If you’re counting, that leaves $4 million for fundraising and processing fees levied by credit card companies. By my math, that’s 4% going to overhead, which is exceptionally low. Admittedly, the ALS Association as a whole spends much more on overhead at 24%, lower than the American Cancer Society’s 40%, but I say good on them to pour this unprecedented influx into research and outreach. During the first year alone, $21.7 million were committed to support “six programs and initiatives to expedite the search for treatments.”
One of these programs, Project MinE, received $1 million in funding to sequence the DNA profiles of ALS patients in the hopes of identifying a genetic cause for the disease, as we currently know little about why it develops. Roughly 10% of patients with ALS also have had the disease in their family, suggesting genetics is a factor. Last month, Project MinE announced in Nature Genetics that their researchers had conducted a study and discovered that 3% of the patients with inherited ALS had a mutated form of gene NEK1. As Quartz explains, NEK1 “helps maintain the structure of neurons and plays a part in their energy flow and DNA repair. If it stops working, it could contribute to the neuronal degradation that causes ALS.” After the discovery, they noticed that non-inherited ALS patients had the mutation, too. Even this low percentage makes NEK1 one of the most common known contributors to the disease. Three additional genes linked to ALS were also discovered separately by Project MinE and published in Nature Genetics. Now that these genes have been identified, researchers can work to understand why the genes malfunctions, and develop treatments to stop or repair them.
In August 2015, Johns Hopkins researchers discovered that a faulty protein, TDP-43, when replicated and reintroduced to mice cells, caused them to return to normal. This is also a promising area for therapies to target. A researcher at Johns Hopkins said the Ice Bucket Challenge funding allowed his team to pursue “high risk, high reward” experiments, critical to making major breakthroughs. However, this also means other attempts may not be as successful as the Ice Bucket Challenge. So what are the takeaways for the scientific and pharmaceutical communities?
The Ice Bucket Challenge highlights crowdsourcing in two ways, most obviously with the numerous individual donations which allowed the research to take place. However, the research was also crowdfunded, and researchers from multiple countries shared common patient datasets. Industry and governmental institutions need to partner with NGOs to make data more commonly available between them to allow for greater analysis.
Social media potential
The Ice Bucket Challenge was a singular event which will not often be repeated. However, pharma should look for opportunities to create or support viral events. It is critical that any such efforts are original and organic, though, or the campaigns will fall flat or scream corporate “me, too.”
The global market for ALS was estimated to be $112 million in 2011, but the relative rarity of the disease has meant a lack of therapies. Business intelligence firm GlobalData describes the therapeutics market for ALS as “woefully inadequate.” Currently, off-label drugs are available to relieve symptoms, while only one treatment is available to directly attack the disease. The breakthroughs outlined above may benefit key players in the pharma industry that have put significant investments into ALS research, including some heavyweights. Several have drugs in clinical trials while another has a robust program dedicated to immune modulation, all of which would add to the arsenal available to treat ALS and its symptoms.
While the Ice Bucket Challenge was a wild success, replicating it may be a challenge. ALS is a niche disease requiring specialized research and treatments, but tackling something along the lines of lung cancer or heart disease with an incidence rate over 20 orders of magnitude higher would require immensely more funding and research to make an impact. At the same time, a very small investment of $1 million dollars in targeted research has already seen a huge return. The ALSA funded several projects, knowing some or all might not succeed, and public and private R&D funding should encourage this kind of wide-ranging, ambitious research. If resources are allocated smartly, it just might make a splash as big as the Ice Bucket Challenge.