A year ago, ALS was a disease that many people only knew through its association with baseball legend Lou Gehrig. The primary challenge in advancing ALS research was to familiarize Americans with this fatal, incurable brain disease.
It's been immensely exciting, then, that things have changed, and ALS is now at the forefront of our cultural conversations through the wild success of last summer’s Ice Bucket Challenge and vocal support from Oscar winners Eddie Redmayne and Julianne Moore.
Sunday night, Eddie Redmayne won a Best Actor Oscar—on top of his recent Golden Globe and SAG Awards—for his poignant turn, in "The Theory of Everything," as physicist Stephen Hawking, who has lived with an ALS-like motor neuron disease for decades. Redmayne went so far as to dedicate his Oscar to “all those people around the world suffering with ALS.”
Best Actress winner Julianne Moore called attention to "Still Alice’s" writer and co-director Richard Glatzer, who lives with ALS, in her Oscar and Golden Globes acceptance speeches.
But even—especially—as ALS receives much-needed attention, we must demand aggressive work toward understanding the disease, and increased money poured into developing rational academic research into effective therapies.
When we founded Project A.L.S. in 1998, we wanted to change the paradigm for brain disease research—to force top scientists from myriad backgrounds to focus on ALS, work together, share data openly, and interrogate all possible avenues toward understanding this complicated disease. We called in experts from all fields to investigate why the brain cells called motor neurons—which are responsible for sending messages to muscles throughout the body—die in ALS, and how we might rescue them.
In the past seventeen years, we’ve achieved measurable results in understanding what goes wrong in this disease and how we might stop its many causes, ranging from rogue genes, to misfolded proteins, to neuroinflammation.
Thanks to the Ice Bucket Challenge, we have been able to accelerate this discovery in the past months—and Project A.L.S. has already committed 100% of monies donated through this viral phenomenon to our research programs.
This has allowed us to undertake the first cutting-edge study of the role lipids play in ALS. We have redoubled our efforts to understand why eye movements remain unaffected in ALS, and to transfer that resilience onto vulnerable motor neuron populations.
We are also able to advance the groundbreaking “ALS in a Dish” model, which Project A.L.S. pioneered in 2008, and which allows scientists to take skin samples from a person living with ALS, and turn them into motor neurons that show his or her unique genetic information. Now, we are homing in on compounds that rescue these motor neurons—including some drugs that are already approved to treat diabetes, epilepsy, and cancer—and are working to leverage our discoveries with pharmaceutical companies able to develop therapies for ALS.
We are proud of this progress—but until medicines can help those currently living with ALS, we must fight to further increase understanding of the disease, demand more from our researchers, and pursue strategic partnerships with companies able to get the first effective treatments to patients.
Advances in genetics have taught us that about 5% of cases of ALS are inherited; beyond these familial strains, ALS has no corroborated causes. It cannot be treated, and commands a life expectancy of 2 to 5 years. It's an unrelenting disease, and its neurodegenerative relatives offer similarly bleak prognoses. Every public mention of ALS excites us—and by turn, compels us to ask every American who's learned about this terrible disease to support research toward its cure.
ALS is no longer a hidden disease; its ravages are front and center. Now, let’s give the media a new story to tell, by working aggressively to stop ALS and related diseases once and for all.