Hi there,

It's great to see the presentations from the conference in November up on the site. However, without the moderator in my living room to decipher, it's very hard to understand what I am reading and looking at. Was anyone there that can share the latest updates in potential treatment, new findings, etc.? Greatly appreciate it.

Best,
Rachel

As for potential therapies in humans, Dr. Kurt Fischbeck stated the NIH is interested in a trial of IGF1 (insulin like growth factor) which has been shown beneficial in mice. Those afflicted with KD are frequently found to have below normal IGF1 level so even though IGF1 can accelerate the growth of cancers there ought to be potential for a reasonably safe therapeutic benefit.

He also stated that the drug BVS857 (I think which acts to improve insulin sensitivity of muscle and perhaps also act on IGF1) which was recently trialed was withdrawn by the manufacturer and won't be pursued despite initial promising results because some in the trial had a problematic immune response to the drug. However he expressed hope that another safer compound might be found with a similar beneficial action.

There was also a discussion of many other therapeutic targets, several of which have been successfully tested in mouse models of KD but I ought to review the presentations before commenting on them.

I too have been waiting for an opportunity to have a comprehensive discussion about the presentations given at the conference. For many years, research has shown that muscle defects occur before neuron involvement, that there are defective and diminished mitochondria involved and that synaptic junctions appear deformed. There is no evidence to suggest that "curing" the aggregates in the nucleus of an affected motor neuron will alter synapses, restore muscle or increase mitochondria.
I believe that our care and treatment is being hampered by an outdated definition of the disease and an ongoing class struggle between biologists and neurologists.
What if (as research shows), the neuron is not driving the disease, but is just the last disastrous event in a long chain of crashes.
I invite all of you to go over Cynthia Jordans slides again; check the research. If the NIH posted definition is wrong, what does that mean?
Fundamentally, to me it means that more research emphasis needs to be put on finding the true triggering event that starts the cascade of symptoms. It means that we must stop using the term atrophy instead of degeneration. It means that we need to explore ways to enhance our mitochondria and above all it means that while we can applaud the dedication of neurologists to to their field of study, we must ensure our clinicians understand that this is not primarily a lower motor neuron disease.
A specialist in mitochondrial myopathy or metabolic disease of muscle may be a better choice when looking for support than one who is only focussed on the nervous system.

Paul once again I find your comments spot on and very reassuring. My views have been evolving in this direction though with great difficulty as it feels dangerous to stray from the herd mind set. I was so glad to see Cynthia Jordan's presentation as it was the strongest 'official' affirmation that I'm not crazy in thinking differently about KD.

For example, I'm now part of the KDA's board and I've joined with the group of people maintaining the website. My first task was to be a review/update of the page of "Doctors familiar with KD". As I started working on it I became obsessed with the fact that they are all neurologists and we need a page of Functional Medicine practioners specializing in endocrinology/metabolism and nutrition who are familiar with KD. Except as far as I know, those people don't exist. Anyway I was paralyzed with contradictions and someone else went and completed the task. I didn't have the courage to bring up my point of view so the KDA webpage continues to propagate what to me is an outdated idea that people should be turning to neurologists when seeking medical care for KD.

I found the following video fascinating. It's by Doug Wallace a doctor/researcher who has focused on mitochondria (and discovered it's maternal inheritance). It's somewhat fast paced and challenging to follow all the details, but important big ideas leap out such as the fundamental role of mitochondria in many disease processes especially in those affecting the most people, the chronic, progressive diseases of aging.
youtube: Doug Wallace on mitochondria and disease

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