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January 27, 2011

Progress being made in developing stem cell-based treatments for ALS/MND

Earlier this week, many of the world’s leading scientists engaged in developing stem cell-based treatments for Amyotrophic Lateral Sclerosis (ALS) or Motor Neuron Disease (MND) met for the first time as a group to share their work. Held in New York, the meeting was organized by the International Consortium of Stem Cell Networks in partnership with the ALS Association and the Motor Neuron Disease Association. Researchers, physicians, surgeons and patient advocates from more than a dozen countries participated.

At present, there is no consensus on the etiology (i.e. the cause or origin) of ALS, and there is a recognition that the disease presents in many forms. Furthermore, while it is often thought of as a single disease, there is ongoing debate about whether it is a disease or a syndrome (a range of conditions). To patients, it doesn’t really matter:  today, diagnosis is a death sentence within three to five years, as the body slowly and painfully shuts down. But for science, this distinction is important, because understanding the cause will allow treatments that target the cause, rather than relieve the symptoms, to be developed.

Nevertheless, progress is being made. While differences abound on the cause of the disease there is more consensus to how stem cells can help to find the answer. The discovery of iPS cells has revolutionized the process of scientific discovery in this field. Many of the world’s leading ALS labs have formed an iPS consortium funded by the US National Institutes of Health, ALS Association and others to develop new stem cell lines from patients living with ALS. These lines are being used in two different ways: 

  1. They are being studied to look for genetic mutations that may be attributable to ALS, or for abnormalities in the process of differentiation into astrocytes and motor neurons that may yield some clues as to the cause. 
  2. They are being used to look for drugs that mitigate or reverse the process of neural degeneration that is the primary symptom of ALS. Essentially this is reverse engineering the problem -– looking for a drug that may be a cure, and then looking to understand the so-called “mechanism of action” -– understanding why the drug works. Some early hits have already been identified, but there is still a long way to go and many hurdles to cross before these drugs will be ready for the clinic.

The meeting also highlighted several cell-based therapies entering the clinic that are taking another approach. While animal models of ALS are necessarily crude at this point, given the lack of understanding of the cause of the disease (essentially the models seek to mimic symptoms at this point), early data has suggested that there is some potential for the use of mesenchymal stem cells to prolong life and relieve symptoms. The evidence has been sufficient for authorities in the United States, Italy and Israel to approve Phase I trials. All of the physicians at the meeting were anxious to stress that these trials are to test the safety of the procedure, and there is no expectation at this time of any efficacy in humans. But you have to start somewhere, and for a disease as catastrophic as ALS where there is no cure (or much even by way of symptom relief), patient accrual is occurring. At Emory Hospital in Atlanta, nine patients have been treated so far.

The final session of the workshop looked at the issue of stem cell tourism in the context of ALS. The broader issue has been the subject of many posts on this blog. The ALS community has been particularly targeted, as patients are usually desperate, with only a short time to live, and little hope from the current standard of care. Indeed, scamming ALS patients with bogus stem cell treatments was the subject of a 60 Minutes documentary earlier in 2010. The ALS community is aware of the issue and there are several independent efforts to provide patients with credible information. Both the ALS Association and MND Association provide strong guidance for patients, but they recognized that they were often viewed as part of the “establishment” and were therefore subject to suspicion. Both entities saw the value of other “independent voices”, with two examples being fairly prominent. 

ALS Worldwide –- its founders’ son died of ALS and was the subject of the documentary film Indestructible -- has produced a fairly comprehensive guide to the different clinics around the world offering ALS treatments. While ALS Worldwide does a good job of identifying the truly bogus clinics and the legitimate trials, there remains some debate about clinics identified by ALS Worldwide in various shades of “grey”, as physicians are largely reluctant to endorse anything other than “first world” approved trials. Another group doing an outstanding job is ALSUntangled, run by physician Rick Bedlack from Duke University. ALSUntangled offers advice for ALS patients via Twitter, and Rick himself is available to answer questions online.

By the end of the meeting, a couple of things were very evident. First, there remains no cure for ALS, and the disease itself is still not well understood. Anyone guaranteeing a cure at this point is being completely disingenuous, offering “snake oil”. However, some of the brightest minds in the world are working on this terrible disease, in a spirit of collegiality and collaboration, and progress is clearly being made. A cure is not yet around the corner, but I would challenge anyone who spent two days spent in the room with these scientists and physicians not to believe that, eventually, it will come.

--Drew Lyall, SCN Executive Director



UPDATE (Aug. 15, 2011): The full report of the ALS/MND Stem Cell Workshop is now available for download on the ALS Association website.


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Another summary of this event was included in the Advances in Clinical Neuroscience & Rehabilitation newsletter. See page 2 at http://www.acnr.co.uk/MA11/40_ACNRMA11_confs.pdf

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