ALS Reversals

Once it starts, ALS almost always continues to progress, disable and shorten the lives of its victims. Very rarely, a patient with a confirmed diagnosis of ALS gets better on validated ALS outcome measures. The ALS Reversals Program at the Duke ALS Clinic focuses on understanding these ALS reversals in order to replicate them in other patients. That’s why the funds raised by The LVH ALS Foundation support the ALS Reversals Program. We believe ALS reversals will be the key to finding a cure.

Duke ALS Clinic receives $250,000 to support research into ALS therapies, reversals in April 2016

The LVH ALS Foundation has donated $250,000 to start a new research endowment within the Duke ALS Clinic. The LVH ALS Research Endowment will allow the Duke ALS Clinic to investigate therapies associated with “ALS Reversals,” cases where symptoms fade and motor function unexpectedly returns. The Endowment will also be used to investigate other potential causes of these reversals, from unknown mimic syndromes, to an individual patient’s genetics, to environmental exposures.

“The LVH ALS Research Endowment is a seed, full of exciting potential,” said Duke ALS Clinic Director Rick Bedlack, MD, PhD. “It has been sown onto the fertile ground of the Duke Multidisciplinary ALS Clinic, one of the largest and most comprehensive of its kind. Nurtured by our passion, commitment, and unique focus on alternative therapies and ALS reversals, I believe this will grow, eventually leading us to an amazing Duke ALS Center that helps us climb toward a cure for this disease.”

How grants support exciting research through ALS reversals in 2015

Authored by Dr. Richard Bedlack, Duke ALS Clinic

The original generous grant from The LVH ALS Foundation continues to fund novel and exciting research in the Duke ALS Reversals Program. There are 2 arms to this: St.A.R. (Study of ALS Reversals) and R.O.A.R. (Replication of ALS Reversals). Significant progress was made in both arms this year.

St.A.R.

In St.A.R. we solicited medical records from patients around the world that reported experiencing an ALS Reversal; we were able to validate 22 of these. For the first time these records are being entered into a common data base so we can determine what might be different about these patients in terms of demographics, concomitant medications and past medical history.

Dr. BedlackNext, we worked with teams at Mass General Hospital and PatientsLikeMe to operationally define an ALS Reversal based on an analysis of a large database called PRO-ACT. This work shed important light on the natural history of ALS progression that was not previously well understood. Namely, that progression is not a straight line. Many patients with ALS experience periods where the disease stops, or even gets a little better for a while; this finding has major implications for future clinical trials.  Less than 1% of patients experience a 4-point improvement in the ALS Functional Rating Scale that lasts 12 months or more, however; this is one way that we will operationally define ALS Reversals for future study. Our work was selected for a prestigious platform presentation at the 2015 International Symposium on Motor Neuron Disease, which was delivered on December 10, 2015 as shown here:

This work was also selected for an article (How common are ALS plateaus and reversals?) in the highly competitive peer-reviewed journal Neurology and was expedited to publication.

In 2016, based on feedback from peers who attended the talk and/or read the paper, we will continue to refine our working definition of an ALS Reversal. We will also begin to collect blood on ALS Reversals to study their antibody profiles and genetics.

R.O.A.R.

In the R.O.A.R. Program we will conduct small pilot trials, testing things that patients were using when they experienced an ALS Reversal. The first thing we will test is a supplement from soybeans called Lunasin. The mechanism of action, and the patient who had an ALS Reversal on Lunasin, are described in ALSUntangled No. 26: Lunasin.

In 2015, we finished the R.O.A.R. Lunasin protocol and contracted with partners at Mass General Hospital and PatientsLikeMe who will assist us with this study. We presented a poster on the protocol at the International Symposium on Motor Neuron Disease on December 11, 2015. We applied for and were granted an Investigational New Drug (IND) Approval by the FDA for the protocol and are currently awaiting Duke IRB approval. We expect to receive this and start enrolling patients in January 2016.

For additional information about the ALS Reversals Program, please visit our our website at www.alsreversals.org, view a YouTube Video of a recent presentation by Dr. Richard Bedlack at the ALS Hope Foundation’s Annual Research and Awards Luncheon, or the following press coverage from 2015:

  1. February 2015 – ABC 11, Raleigh, NC – New Discovery Made About ALS Could Lead to a Cure
  2. March 2015 – WOKV Radio, Jacksonville, FL – Richard Bedlack Radio Interview
  3. August 2015 – Duke Medicine, Clinical Practice Today Publication, Page 11 – Article – Programs Study Rare ALS Reversals
  4. November 2015 – News and Observer, Raleigh, NC – Article – Tar Heel: Duke Dr. Richard Bedlack collecting new ideas to fight ALS
  5. December 2015 – MND Research Blog – Article – Can the progression of MND pause or reverse?