Leukine
Background
A century after Aloysius Alzheimer described a form of progressive dementia, the mechanism of the disease that bears his name has been fairly well established. Alzheimer’s disease results from buildup in the brain of a protein called beta amyloid. Although some beta amyloid is normally present in the brain, too much causes brain cells malfunction and, ultimately, Death. Thus, the person suffering from Alzheimer’s disease slowly loses brain function, and with it, Life itself.
The discovery of beta amyloid led to three exciting suggestions. If we could prevent the buildup of beta amyloid, could we prevent Alzheimer’s disease? If we could stop the buildup of beta amyloid, could we arrest the progression of Alzheimer’s disease? If we could remove excess beta amyloid from the brain, could we reverse, or possibly cure, Alzheimer’s? This leads us to further investigation of beta amyloid.
Beta Amyloid
Beta amyloid begins as amyloid precursor protein (abbreviated APP). Although normally present in the body, the function of APP remains unknown. APP’s genetic code is found on chromosome 21, a chromosome which bears an interesting association with Alzheimer’s disease. An extra copy of chromosome 21 results in Down’s syndrome, and everyone with Down’s syndrome develops Alzheimer’s disease at a young age, usually in their 30’s.
Two enzymes break APP into beta amyloid. Again, it is not clear why beta amyloid sometimes malfunctions, builds up, and begins destroying nerve cells.
The brain circulatory and immune systems remove beta amyloid from the brain via a specialized structure, the glymphatic clearance pathway, discovered in 2012. Since this discovery is relatively recent, exactly how the pathway works remains poorly understood, yet it has led to interest in whether stimulation of the immune system could be an effective treatment for Alzheimer’s disease.
The Immune System
Our immune system contains two interworking components: antibodies and immune cells. Human antibodies can be harvested from healthy donors and given intravenously to patients to need them. This simple (but very expensive) approach was tried first on people with Alzheimer’s disease, but, unfortunately, failed to work. Next, the interest turned to immune cells, called granulocytes and macrophages. These cells can never be transplanted from one person to another, but would it be possible to stimulate a person’s own immune cells, so they increase in number and, possibly, in vigor to remove beta amyloid from the brain of a person with Alzheimer’s disease?
Leukine®
Leukine® was introduced in 1991 as a medication to stimulate regrowth of immune cells following chemotherapy, usually for leukemia. It is produced in the laboratory using recombinant DNA technology, which makes a copy of a protein that is normally present, but in smaller amounts, in the human body. This protein is called granulocyte macrophage colony stimulating factor (GM-CSF), discovered in 1985. Following this discovery, the gene that codes for GM-CSF was cloned into a strain of brewer’s yeast, which then started producing recombinant GM-CSF, patented and sold as Leukine®.
Following the failure of donor antibodies in Alzheimer’s disease, interest turned Leukine® as a possible treatment. Several centers around the country enrolled volunteers in trials, giving some higher-dose Leukine®, some lower-dose Leukine®, and others, nothing at all (placebo). This should answer the question of whether Leukine® works for Alzheimer’s disease.
Questions & Answers
1. Can I enroll myself or someone with Alzheimer’s in one of those clinical trials?
As a disclaimer, this is not a web site for information or enrollment in clinical trials, and we do not keep timely information on clinical trials here. As far as we know, however, all the clinical trials for Leukine® in Alzheimer’s disease are closed.
2. Can I still get Leukine® for someone with Alzheimer’s disease?
Yes.
3. How can I get it?
Alpine Neurology can obtain Leukine® for our patients with Alzheimer’s disease. You may telephone (303)493-1922 to schedule an appointment. When you do so, please indicate that the person for whom you are scheduling has Alzheimer’s and an interest in Leukine®. We shall have the appropriate information ready for your initial visit.
4. Will insurance or Medicare pay for Leukine® for Alzheimer’s?
No. Leukine® is still considered experimental. No insurance company pays for it. Medicare does not pay, either. Your consultation will be paid by insurance, as long as we are a participating provider. (Check that section of this web site.)
5. How much does Leukine® cost?
The cost depends on the dosage, which is based on your height and weight. You should know that we administer the same amount as the higher dose of the protocol of our local university. You are guaranteed to get full-dose Leukine® from us. The cost of the Leukine® protocol usually runs around $9,500. This includes injection supplies and instruction.
6. How often will I have to repeat Leukine®?
We don’t know. Here’s how we intend to find out. We are monitoring the literature for emerging reports from the experiments being done around the country. We also ask the care givers to watch for a response either improvement or a long period where there is no worsening. Once this response wears off, it is time to repeat the Leukine®. We estimate that this will occur over about two years.
7. Are side effects expected?
Since it is normally present to some extent in the body, most patients tolerate Leukine® with few side effects. Sometimes there is temporary fluid retention or mild reaction where the Leukine® was injected. An elevation in the white cell count is listed as a side effect; we call this the desired effect of the treatment.
8. Do I have to travel to Colorado to receive Leukine®?
Inquire at AlpineNeuro@gmail.com
A century after Aloysius Alzheimer described a form of progressive dementia, the mechanism of the disease that bears his name has been fairly well established. Alzheimer’s disease results from buildup in the brain of a protein called beta amyloid. Although some beta amyloid is normally present in the brain, too much causes brain cells malfunction and, ultimately, Death. Thus, the person suffering from Alzheimer’s disease slowly loses brain function, and with it, Life itself.
The discovery of beta amyloid led to three exciting suggestions. If we could prevent the buildup of beta amyloid, could we prevent Alzheimer’s disease? If we could stop the buildup of beta amyloid, could we arrest the progression of Alzheimer’s disease? If we could remove excess beta amyloid from the brain, could we reverse, or possibly cure, Alzheimer’s? This leads us to further investigation of beta amyloid.
Beta Amyloid
Beta amyloid begins as amyloid precursor protein (abbreviated APP). Although normally present in the body, the function of APP remains unknown. APP’s genetic code is found on chromosome 21, a chromosome which bears an interesting association with Alzheimer’s disease. An extra copy of chromosome 21 results in Down’s syndrome, and everyone with Down’s syndrome develops Alzheimer’s disease at a young age, usually in their 30’s.
Two enzymes break APP into beta amyloid. Again, it is not clear why beta amyloid sometimes malfunctions, builds up, and begins destroying nerve cells.
The brain circulatory and immune systems remove beta amyloid from the brain via a specialized structure, the glymphatic clearance pathway, discovered in 2012. Since this discovery is relatively recent, exactly how the pathway works remains poorly understood, yet it has led to interest in whether stimulation of the immune system could be an effective treatment for Alzheimer’s disease.
The Immune System
Our immune system contains two interworking components: antibodies and immune cells. Human antibodies can be harvested from healthy donors and given intravenously to patients to need them. This simple (but very expensive) approach was tried first on people with Alzheimer’s disease, but, unfortunately, failed to work. Next, the interest turned to immune cells, called granulocytes and macrophages. These cells can never be transplanted from one person to another, but would it be possible to stimulate a person’s own immune cells, so they increase in number and, possibly, in vigor to remove beta amyloid from the brain of a person with Alzheimer’s disease?
Leukine®
Leukine® was introduced in 1991 as a medication to stimulate regrowth of immune cells following chemotherapy, usually for leukemia. It is produced in the laboratory using recombinant DNA technology, which makes a copy of a protein that is normally present, but in smaller amounts, in the human body. This protein is called granulocyte macrophage colony stimulating factor (GM-CSF), discovered in 1985. Following this discovery, the gene that codes for GM-CSF was cloned into a strain of brewer’s yeast, which then started producing recombinant GM-CSF, patented and sold as Leukine®.
Following the failure of donor antibodies in Alzheimer’s disease, interest turned Leukine® as a possible treatment. Several centers around the country enrolled volunteers in trials, giving some higher-dose Leukine®, some lower-dose Leukine®, and others, nothing at all (placebo). This should answer the question of whether Leukine® works for Alzheimer’s disease.
Questions & Answers
1. Can I enroll myself or someone with Alzheimer’s in one of those clinical trials?
As a disclaimer, this is not a web site for information or enrollment in clinical trials, and we do not keep timely information on clinical trials here. As far as we know, however, all the clinical trials for Leukine® in Alzheimer’s disease are closed.
2. Can I still get Leukine® for someone with Alzheimer’s disease?
Yes.
3. How can I get it?
Alpine Neurology can obtain Leukine® for our patients with Alzheimer’s disease. You may telephone (303)493-1922 to schedule an appointment. When you do so, please indicate that the person for whom you are scheduling has Alzheimer’s and an interest in Leukine®. We shall have the appropriate information ready for your initial visit.
4. Will insurance or Medicare pay for Leukine® for Alzheimer’s?
No. Leukine® is still considered experimental. No insurance company pays for it. Medicare does not pay, either. Your consultation will be paid by insurance, as long as we are a participating provider. (Check that section of this web site.)
5. How much does Leukine® cost?
The cost depends on the dosage, which is based on your height and weight. You should know that we administer the same amount as the higher dose of the protocol of our local university. You are guaranteed to get full-dose Leukine® from us. The cost of the Leukine® protocol usually runs around $9,500. This includes injection supplies and instruction.
6. How often will I have to repeat Leukine®?
We don’t know. Here’s how we intend to find out. We are monitoring the literature for emerging reports from the experiments being done around the country. We also ask the care givers to watch for a response either improvement or a long period where there is no worsening. Once this response wears off, it is time to repeat the Leukine®. We estimate that this will occur over about two years.
7. Are side effects expected?
Since it is normally present to some extent in the body, most patients tolerate Leukine® with few side effects. Sometimes there is temporary fluid retention or mild reaction where the Leukine® was injected. An elevation in the white cell count is listed as a side effect; we call this the desired effect of the treatment.
8. Do I have to travel to Colorado to receive Leukine®?
Inquire at AlpineNeuro@gmail.com
