Interview by Craig Gustafson
Jon Kaiser, MD, has treated patients with chronic fatigue syndrome, HIV/AIDS, cancer, and other immune system disorders for the past 25 years. To that end, he strives to help patients and health care providers treat complex medical conditions using an integration of natural and standard medical treatments.
He is a clinical instructor in the Department of Medicine at the University of California, San Francisco Medical School and medical director of K-PAX Pharmaceuticals.
Dr Kaiser was the principle investigator of a 2006 study published in the Journal of AIDS1 showing that broad-spectrum micronutrient therapy can strengthen the immune systems of people with immune deficiency disorders. He is currently coordinating the development of a new treatment for chronic fatigue syndrome based on enhancing mitochondrial energy production. The second of 2 clinical trials investigating this treatment is scheduled to complete in mid-2015. He has written 2 books on improving immune system health: Immune Power2 and Healing HIV.3
Alternative Therapies in Health and Medicine (ATHM): It seems that recently, mitochondrial health has become a buzzword in medicine. What are your observations?
Dr Kaiser: During the past 15 years on the basic science side of the fence, there has been an explosion of research findings linking mitochondrial dysfunction to adult-onset chronic disease. I have been doing mitochondrial research since 1999 and, before then, there was some research on mitochondria. But in the past 15 years, there has been an exponential amount of published research linking mitochondrial dysfunction to free-radical stress and the occurrence of adult-onset chronic diseases.
That part is happening, but everybody wants to see potential corrections to this problem developed and tested. The only entities that have the resources to do multimillion-dollar, placebo-controlled trials are pharmaceutical companies. Because they just do not understand mitochondrial disease and how to approach it, they have chosen not to do that.
ATHM: Aren’t pharmaceuticals often mitochondrial poisons? If so, wouldn’t it behoove the pharmaceutical companies, even if they cannot have a monopoly on something that is natural, to support something that improves mitochondrial status so that they might avoid the adverse effects associated with the drugs?
Dr Kaiser: I actually just read a paper on this exact topic. There is evidence linking many current pharmaceuticals to mitochondrial toxicity and mitochondrial damage. Many antibiotics, antidepressants, chemotherapy, and neurologic interventions have been associated with mitochondrial damage. Statins are also part of this group. K-PAX Pharmaceuticals has recently generated data that suggest coadministering a mitochondrial support micronutrient compound with a specific drug can virtually eliminate many of this drug’s side effects.
This is breakthrough stuff. Our studies suggest that this potential exists and that our data show it to be true. Yes, pharmaceutical companies might be interested in a patented mitochondrial support micronutrient compound. Our compound has received 4 separate US patents for use in infectious diseases and in chronic fatigue. It has taken a tremendous amount of persistence and resources, but we have achieved that.
ATHM: A few months ago, I had the privilege and opportunity to interview, James Forsythe, MD, an oncologist. He has been experiencing terrific results in cancer with insulin-potentiated chemosensitivity therapy. In addition to that, he is also supplementing with a nutritional regimen including antioxidants that bookends the chemotherapy delivery. He is finding that in a lot of cases between the nutritional support, the antioxidant support, and the lowered chemotherapy dose, that his patients are not suffering all of the traditional chemotherapy adverse effects.
Dr Kaiser: I think what you have just described is the new dawn on cancer treatment. In 50 years, we are going to look back to delivering toxic chemotherapy and radiation without effective mitochondrial support nutrients as the Dark Ages of medicine. The use of chemotherapy without simultaneously supporting mitochondrial health with micronutrients is going to be seen the way we look at leeches right now. You can quote me on that.
ATHM: How did you become interested in addressing mitochondrial health in your practice?
Dr Kaiser: Fortunately, I was always enamored by nutritional biochemistry and the interior organelle functioning of cells. That is what I did my premed and medical school research on. It is my favorite area of interest. Then take an individual with that interest and drop them on the front lines of the AIDS epidemic in San Francisco 25 years ago, when there were no effective treatments to support the immune systems of AIDS patients. The only tool I had at my disposal was nutritionally supporting the immune system of my patients while we waited for effective drugs to be released. That is chapter 1.
Chapter 2 begins 5 years after the first drugs came out when they were ultimately linked to a very high degree of mitochondrial toxicity. This situation presented an opportunity with the goal of protecting my patients from a long list of severe mitochondrial toxicity side effects from these “life-preserving” drugs—to come up with an antidote to that toxicity.
I had the perfect laboratory with which to test different formulas of mitochondrial support nutrients and see which worked and which did not. Eventually, I came upon what would ultimately become K-PAX MitoNutrients. I convinced Bristol-Myers Squibb, the maker of these early AIDS drugs, to fund a double-blind, placebo-controlled trial in patients with mitochondrial toxicity, and the results were enormously positive in the ability to protect the mitochondria from this toxicity.
Since that breakthrough, I have been working ever since to do additional research on this formula, ultimately with the goal of getting it FDA approved.
ATHM: What types of things did you look at first? Were your initial attempts successful? How did you find the groove?
Dr Kaiser: My research has shown that there is a key triad of mitochondrial support nutrients that are critically important for success. Those 3 are N-acetylcysteine, which is a precursor of glutathione; acetyl-L-carnitine, which is a natural mitochondrial stimulant; and a-lipoic acid, which is a highly potent and necessary antioxidant for the interior of the mitochondria. Those 3 were individually studied and reported in the literature as being highly effective at preserving healthy mitochondrial functioning in animal models, in human beings, and in AIDS patients. There was independent research on the separate pieces. I put those 3 together. I chose what I believed, based on my experience, would be the most effective dosages. I sourced it from high-quality manufacturers and I embedded it in a broad-spectrum multivitamin that contained all the nutrients, vitamins, and minerals that the mitochondria would need to function at a higher level.
Then I was able to test it. In AIDS patients, there were 2 expressions of mitochondrial toxicity that reversed very quickly. Those were symptoms of peripheral neuropathy and weak immune systems with below-normal levels of CD4 cells. Within months, I saw improvement in most of my patients. Then I knew I had metaphorically hit the nail on the head. I now had a bioactive compound that could produce clinical benefits in patients experiencing mitochondrial toxicity.
ATHM: Bristol-Myers Squibb was originally on board and supporting what you were doing. That was quite a while ago. What has happened in the meantime?
Dr Kaiser: When we generated this highly significant data, we tried to enlist Bristol-Myers Squibb to continue investing in the development of this natural antidote to the mitochondrial toxicity. They reviewed the proposal and considered it, but we hit a number of roadblocks. First, their scientists could not believe that a natural compound could do this. The second roadblock was their reluctance to invest in developing a natural compound for marketing and treatment of a disease—that is not their model. It didn’t fit in the way they do things.
Their approach was to abandon the drugs that had mitochondrial toxicity and try to develop newer drugs with less mitochondrial toxicity. They declined to further develop this treatment and that’s when I decided to help start K-PAX Pharmaceuticals to continue the research and development of what I was experiencing in the real world as a breakthrough.
ATHM: You say you were still interested in finding a broad-based pharmaceutical company that would be interested in the development and distribution of this combination or this nutrient therapy. So, how did you have to change tack to reinterest somebody in doing something here?
Dr Kaiser: If you go to the pharmaceutical industry and say, “I have a micronutrient formula that I believe helps improve mitochondrial functioning,” they will say, “So what? What disease does it treat? How can you prove that it is safe and effective as a treatment for an accepted disease?” You have got to have a target—a disease target. Initially, our target was HIV infection because of the drug toxicity and because, initially, there were not a whole lot of successful drugs. By 2010, antiviral drugs no longer had significant mitochondrial toxicity and there were over 30 drugs to treat HIV. It became a very—what we call—crowded field.
So K-PAX Pharmaceuticals shifted tack to address chronic fatigue syndrome because in chronic fatigue syndrome, we have a disease with no approved treatment. It is called an unmet medical need. In fact, there are more patients with chronic fatigue syndrome in the United States than with HIV infection, so it is a very large market.
The next thing you need for a pharmaceutical company to become interested is a patent or patent potential, which we now have. And finally, you need to show data that suggest your treatment is safe and effective. We now have all those pieces together. That’s why we are seeking to find a pharmaceutical industry partner.
ATHM: Is chronic fatigue syndrome associated with mitochondrial dysfunction?
Dr Kaiser: During the past 5 years or so, there has been a growing body of evidence that patients with chronic fatigue syndrome have mitochondrial dysfunction. It perfectly explains the existence of severe symptoms in multiple different systems of the body without any infection causing it. We have never been able to link chronic fatigue syndrome to a specific infection. You have an adult-onset chronic disease with fatigue as the number 1 symptom plus evidence of mitochondrial dysfunction in many systems of the body: the nervous system, the musculoskeletal system, and the endocrine system. This fits the definition of a mitochondrial dysfunction disease.
That is why we were drawn to it with our nutrient compound, but I had to innovate further because the nutrients by themselves do not perfectly address chronic fatigue syndrome. They do not produce a rapid response by themselves in the majority of people with chronic fatigue syndrome. They might help a little, but not enough for the FDA to consider this an effective treatment worthy of approval. There still was a missing piece.
So I then went to the medical literature and found significant evidence for the potential use of low-dose stimulants in people with chronic fatigue syndrome, but these were often limited by persistent side effects. Many clinicians hesitate to prescribe stimulant drugs to sick patients with chronic fatigue syndrome. It’s obvious why—like whipping a sick horse, eventually, they crash.
ATHM: It would make sense that a stimulant would be overdriving their adrenal system.
Dr Kaiser: It overdrives many of their systems, but really what it is overdriving are their mitochondria, which are sick in the first place. The reason the adrenals cannot keep up with the production of cortisol, DHEA, and adrenalin is because their mitochondria cannot make enough energy. The reason they get brain fog is because their neurons cannot make enough energy. The reason that they have trouble standing up for a long time is because the mitochondria of their muscles cannot tolerate it. Our hypothesis was to determine whether a low-dose stimulant balanced with potent mitochondrial nutrient support could take away those long-term side effects and allow the patients to benefit from a little bit of a stimulant. This is what we have shown in our data: that the side effects in the treatment group using low-dose stimulant drugs plus mitochondrial support were no different from placebo.
ATHM: Has improvement in mitochondrial status taken up its own momentum? Are people getting above the threshold where they are able to wean off the drug?
Dr Kaiser: Our long-term experience has shown that, first of all, the usual time frame for improvement when you combine these 2 pieces, the stimulant and the MitoNutrients, is within 4 to 6 weeks. You get two-thirds of the improvement in 4 to 6 weeks; it is rapid. Then over additional time, the most common experience is gradual, continual improvement. In the patients I have given this treatment to who have been on it for upwards of 3 years, there is the ability to begin to wean off the stimulant component of the treatment and be maintained on just the nutrients.
The time frame is very individual. Some patients will need to stay on this treatment to function. Other patients will be able to go off the stimulant in 6 months. For other patients, it may take them 3 years to wean down off the stimulant. It is very individualized, but the usual experience is that, yes, they continue to get better and can wean off of the stimulants.
- Kaiser JD, Campa AM, Ondercin JP, Leoung GS, Pless RF, Baum MK. Micronutrient supplementation increases CD4 count in HIV-infected individuals on highly active antiretroviral therapy: a prospective, double-blinded, placebo-controlled trial. J Acquir Immune Defic Syndr. 2006;42(5):523-528.
- Kaiser J. Immune Power: A Comprehensive Healing Program for HIV. New York, NY: St Martin’s Press; 1995.
- Kaiser J. Healing HIV: How to Rebuild Your Immune System. Mill Valley, CA: HealthFirst Press; 1998.