No control group, open protocol, self-evaluations.
The structure for a new semi-virtual clinical trial for amyotrophic lateral sclerosis (ALS) readily breaks many scientific conventions.
But the collaborative study design is also inexpensive, fast, and inclusive of ALS patients worldwide — attractive features for an industry struggling with high costs, long timelines, and late-stage clinical failures.
Dubbed the Lunasin Virtual Trial, the study represents a novel collaboration between an online patient community, PatientsLikeMe, and The Duke ALS Clinic. The group announced this week that all 50 core participants have been enrolled, in what clinic director Richard Bedlack called the fastest trial enrollment in the history of ALS.
In a phone interview, Paul Wicks, vice president of innovation at PatientsLikeMe, said the aim was to prove or disprove the potential of an amino acid supplement, lunasin, as efficiently as possible and with the interests of ALS patients in mind.
As it stands, just one FDA-approved therapy is currently available, riluzole (brand name Rilutek), which prolongs life 2-3 months without improving symptoms.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
“What we were trying to look at is to see if there was a faster way and a more convenient way for patients to quickly establish whether or not there might be any merit in new treatments like this,” Wicks said.
An ALS researcher and caregiver, Wicks said the semi-virtual study design came partly in response to the struggle patients face getting to a clinic. The other big issue was time.
ALS is defined as a “rapidly progressive, invariably fatal neurological disease” by the National Institute of Neurological Disorders and Stroke. Most patients die from respiratory failure three-to-five years after diagnosis.
By comparison, it takes an average of six-to-11 years to move an investigational drug through clinical trials.
“The idea was rather than have a placebo group, which is clearly very concerning for patients that have a relatively short time left to live, we would make sure everybody got the actual treatment,” Wicks said.
In addition to the 5o enrolled participants with confirmed ALS, the study protocol has been made available online for all interested patients to access and follow from home. They are also encouraged to record and upload their health information through the PatientsLikeMe website.
The combination of open-access protocol and no control arm would likely cause FDA alarm bells to ring. But Wicks said the study’s aim and the nature of the disease allow it to work as a pilot project. He also believes the dire trajectory of ALS cannot be overcome with placebo-induced optimism.
“We’re not looking for a small effect. The field has traditionally looked for something that might slow the disease down by 20 percent or so. That’s almost imperceptible if you actually have the disease,” Wicks said. “Our study is looking for a really big improvement or a reversal of symptoms, which is very unusual.”
Disproving lunasin’s efficacy would also be valuable for patients. According to a 2014 review, many patients are self-dosing with supplements like lunasin due a the lack of other options. The review authors found no evidence to support the marketing claims of a leading provider of lunasin, Reliv. Taking Reliv’s LunaRich X at full dose costs approximately $240 per month.
In 2010, PatientsLikeMe was involved in a study of lithium carbonate as a possible treatment for ALS. The lithium study was also patient-inclusive and relied on self-reported data through the website. After one year, it was determined that lithium had no benefit and the field moved on, setting some precedent for the current trial.
The lunasin trial does include a number of objective measures, such as weight and blood panels. In the absence of a control arm, each study participant will be closely matched with three-to-five historical controls.
To track their progress, participants will make three in-person visits to the Duke ALS clinic and complete virtual check-ins every 30 days through the PatientsLikeMe website.
By pursuing many unconventional study steps, the team has placed itself on the end of two spectrums. It is remarkably cheap, even for an exploratory trial. Wicks said the entire project is funded by a philanthropic donation of several hundred thousand dollars, compared to the hundreds of millions spent by pharma companies attempting to prove the efficacy of neurological drugs.
On the other hand, the data is unlikely to carry much weight on its own. Wicks readily admits that it’s not designed to replace established clinical trials or to give a definitive result. The aim instead is to cast a wide net, investigating dozens of different compounds in parallel.
“Some of the study features will work, others won’t,” Wicks said. “The aim is to continue to improve and refine as we go.”
Photo: oko_swanomurphy, Getty Images
