My name is Rick Bedlack. I am a neurologist at Duke University in Durham North Carolina, and I started the Duke ALS Clinic 16 years ago. I am working to empower people with this disease to live longer and better lives and to have a greater role in research. I currently run the ALSUntangled Program (www.alsuntangled.org) and The Northeast ALS Consortium (NEALS) ALS Clinical Research Learning Institute. This is the story of a new program I recently started called ALS Reversals.
ALS is a degenerative disease of motor neurons, typically characterized by progressive muscle weakness, increasing disability and shortened survivals. It is widely recognized that ALS progression can be variable. It can be variable between patients, with some folks progressing much more slowly than others. It can also be variable within a given patient, with periods where the disease seems to speed up or slow down for a while. Less appreciated is the fact that ALS progression can stop (plateau) or even reverse with significant recovery of lost motor functions.
I encountered my first ALS reversal in July 2011, more than a decade into my career in ALS, while leading an ALSUntangled review of Dean Kraft, an energy healer. On Kraft’s website was a video about a person named Nelda Buss, who was reported to have been diagnosed with ALS at a top hospital and progressed to where she had lost nearly all movement and was near death. After two years of Kraft’s treatments, she was said to have recovered most or all of her motor function. I was skeptical. Nonetheless, I followed the ALSUntangled standard operating protocol and investigated. Eventually, I found Nelda Buss through social networking. I got her medical records and saw that her ALS diagnosis was sound, and had been confirmed by a trusted colleague at an excellent ALS center. She really did progress to having no strength at all in her arms and legs. Two years later she had recovered to where she was able to walk and use her arms normally. You can read more about this ALSUntangled review here.
Since then, I have found 22 additional “ALS reversals.” Some of these were in old papers, some I found while conducting newer ALSUntangled reviews, one was in a clinical trial and two were my own patients. To me, these cases seemed important enough to focus further research on. I even found a precedent for such work: a study of HIV elite controllers [people who are infected with the HIV virus but never get sick] showed that many of them have the same genetic abnormality, which explains their resistance and helped researchers create a new drug that works for everyone with HIV.
I hypothesized that there were 3 possible explanations for ALS reversals:
- They did not have ALS in the first place, but rather an ALS-mimic that was so obscure that it fooled even experts;
- A genetic factor that made them “resistant” to the disease (like the HIV elite controllers);
- Positive effects from a new treatment these people started or from removing some toxic environmental trigger.
I designed two new programs called Replication of ALS Reversals (ROAR) and Study of ALS Reversals (StAR) to help me work through these hypotheses and was fortunate to receive start up funding for these from the LVH ALS Foundation.
In my ROAR program, I am testing the exact same treatments that folks with reversals tried in several more patients. In addition to energy healing, some of the other treatments associated with ALS reversals include Lunasin, Protandim, Aimspro, and bone marrow-derived stem cells through Brainstorm Cell, and embryonic stem cells through Neuraltus. There will be several novel patient-centric features of the trials in this ROAR Program, including:
- Broad inclusion criteria;
- Use of historical controls (no placebos);
- Few in-person study visits (most visits done virtually, on home computers);
- Results available in real time.
The first ROAR trial of Lunasin is underway; all 50 available spots have already been promised but we have published the protocol online, so that folks who want to try Lunasin on their own can use the exact same products and dosages as we are, and even follow their outcome measures and post them on PatientsLikeMe.
In my StAR program, I will be looking for obscure ALS mimics in people with reversals, and will be comparing their genes to people whose ALS does not reverse. The first thing this program needed to do was come up with ways to objectively define ALS reversals, and to find more of them. Last year, I worked with colleagues at Massachusetts General Hospital (MGH) and PatientsLikeMe to look for people with reversals in a large database called PRO-ACT. Our study showed that ALS plateaus and small brief reversals are not uncommon. On the other hand, large reversals lasting a long time are rare; less than 1% of more than 1000 eligible PRO-ACT participants had an ALS Functional Rating Scale-Revised (ALSFRS-R) improvement of at least four points lasting at least a year. This study taught us a lot about the natural history ALS, including what might be meaningfully different and thus worth further investigation.
Unfortunately, all of the participants in PRO-ACT were “de-identified,” so we do not know who they are and thus have no way to contact or further study any of them. I am currently working with a company called Origent Data Sciences, Inc. to define something called “ALS resistance.” This definition would encompass patients who have very long plateaus (like Stephen Hawking), or large, sustained periods of reversal. We think we might have found a way to identify “ALS resistant” patients in real time, which would allow us to find more of them and invite them into the StAR Program. Stay tuned for more on this exciting work later this year.
If you have been diagnosed with ALS, have experienced a marked improvement in motor function, and are willing to participate in our StAR Program, please contact us through our website at www.alsreversals.org.