ALS Research Briefs

Patients Like Me online lithium study utilized social media

An observational study of data self-reported on the online forum Patients Like Me by people with ALS who elected to take the drug lithium carbonate has shown two things:

  • lithium does not slow the progression of ALS — a finding consistent with the findings of several other clinical trials of the drug; and
  • social media sites may prove to be a valuable tool for speeding research and testing experimental therapies in ALS and other diseases.

Although the first finding comes as no surprise, the second represents a departure from conventional clinical trial procedures.

An analysis of the Patients Like Me data was reported online April 24 in Nature Biotechnology. Members of the research team included Paul Wicks, director of research and development at Patients Like Me, and James Heywood, co-founder and CEO of the site.

The results corroborate the findings of several other conventional clinical trials of lithium in ALS, including one supported by MDA, and trials in the United States and Europe.  These trials also found serious adverse affects and no clinical benefit from lithium.

Participants in the Patients Like Me study elected to take lithium and post their results on the online forum. They obtained prescriptions for lithium from their doctors; received regular blood tests to monitor levels of the drug in their bodies; and posted blood-test results, functional-exam scores, and any signs or symptoms possibly attributable to the drug. All data was viewable to the public as the study progressed.

In evaluating the data, investigators matched each participant with multiple control subjects (people with ALS whose disease course matched the participant’s and who did not take lithium).

“The results from this study demonstrate a new way to think about conducting clinical trials," said Valerie Cwik, MDA executive vice president of research and medical director. "We want to utilize technology whenever possible, but first we need to think about how it might be used, where it could prove useful, and when and why conventional clinical trials still will need to be done." 

The conventional "gold standard" in drug testing is a randomized, double-blinded, placebo-controlled human clinical trial.  

In their analysis of the Patients Like Me data, the investigators suggested that Internet-based studies potentially could help researchers determine the variability in disease course; the “disease burden” (the emotional, financial and other costs of ALS); the effectiveness of drugs already in use by patients; and information useful in designing and implementing real-world human clinical trials.

"The validity of research conducted in a self-reported, online data environment," the study authors wrote, provides "rich opportunity for future work."

Literature review provides a clearer picture of neuroinflammation in ALS

A comprehensive review of 20 years of reports on motor neuron degeneration and neuroinflammation in ALS reinforces the clinical importance of the inflammatory response in the disease, and highlights specific questions that still need to be addressed.

Investigators Thomas Philips and Wim Robberecht, both at University Hospital Leuven in Leuven, Belgium, published their extensive review of the inflammatory reaction in ALS in the March 2011 issue of Lancet Neurology. The investigators reviewed reports published between January 1990 and December 2010.

Neuroinflammation is a common characteristic of ALS, stemming from the immune system’s attempts to combat factors associated with the disease process.

This normally neuroprotective inflammatory response is triggered when the immune system activates glial cells (motor neuron support cells) called microglia and astrocytes, and some subtypes of T cells. The resulting neuroinflammation, typically a temporary condition, is sustained in ALS. The infiltration of cytotoxic (cell-damaging or destroying) T cells over time generates a hazardous and toxic environment, leading to the degeneration and death of motor neurons.

A number of recent studies have greatly added to the body of knowledge about the inflammatory response in ALS, but the investigators noted that many questions remain to be studied, such as:

  • What factors determine whether the inflammatory reaction is protective or destructive?
  • What is the origin of the neuro-inflammatory reaction?
  • Which types of T cells are involved in the inflammatory process?
  • What signals cause activation of microglia and astrocytes?
  • What signals mediate interaction between astrocytes and microglia, and in what ways do the two types of cells interact?

For more about the immune system in ALS, see:

Webinar discusses ceftriaxone clinical trial

A webinar (Web-based seminar) about an ongoing phase 3 clinical trial of ceftriaxone in ALS will be held Thursday, May 5, 2011, at 3 p.m. EST. It will be hosted by neurologist Merit Cudkowicz, the principal investigator for the trial and director of the MDA/ALS Center at Massachusetts General Hospital in Boston.

Cudkowicz will explain the scientific rationale behind the trial, describe the study procedures and answer questions from webinar participants.

Ceftriaxone is an antibiotic that may confer neuroprotective benefits to the motor neurons (nerve cells) that die off in ALS. Researchers currently are recruiting participants for the ceftriaxone trial at more than 50 locations across the United States and Canada.

Those interested in viewing the webinar may click here to register. (Early registration is recommended, as space is limited.)

For more information, contact Sarah Titus, assistant project manager, at (617) 726-1398, or email stitus@partners.org.

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