Yep. Type of thing that would win a Nobel Prize. Royalties back to the university and the prestige gained from this if it gets approved would be monumental. Bio/Chem/Pharma research at CU could become an absolute cash cow.
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Bill McCartney diagnosed with late-onset dementia/Alzheimer's
- Thread starter Jens1893
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Yep. Type of thing that would win a Nobel Prize. Royalties back to the university and the prestige gained from this if it gets approved would be monumental. Bio/Chem/Pharma research at CU could become an absolute cash cow.
With Anschutz continuing to grow and adding a THIRD research hospital (New VA medical complex) combined with the teaching hospital and new medical system being added at UCCS, CU is definitely positioning itself well in that industry.
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Sounds amazing. More deets, please.
What drug? Who invented it? Who sells it? Is it experimental or in production?
What drug? Who invented it? Who sells it? Is it experimental or in production?
Lukeasaurus
Well-Known Member
Can anyone find a link to the actually drug, news report , or study ? I want to read it but a quick Google search revealed a UW Alzheimer's study this year and a few Alzheimer's studies from CU in 2014 and 16 but nothing this year
If they're doing human trials, that would mean that it's in Phase III, which is the last level of testing prior to approval. I think that Alzheimer's is a priority disease, and if I'm right that means that testing and approval is accelerated vis a vis the normal process. Edit- looks like Phase II (larger group of human subjects in clinical trial prior to getting approval to move forward with the full blown Phase III). Of note, since this is a drug already on the market, doctors can probably prescribe for Alzheimer's right now as an "off label" use since approval for this indication is being sought.
It looks like the drug they're investigating is Leukine, which stimulates white blood cells (produced in bone marrow) and is for use following chemo. Genzyme is the pharma company that owns that drug under the brand name Sargramostim.
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Looks like 9 news has the exclusive to the trial being run by CU. Report airs tonight at 7:15.
This would all be cool but more important would be the impact on the individuals and their families.
Alzheimer's is just brutal in what it does to people. Seeing people who have been intelligent, insightful, and social become walking shells of themselves not knowing their own family members and getting lost on the way to the bathroom is a cruel way to end a life. Having our memories of people become their struggles instead of who they really were is hard.
If CU could be a part of changing this would easily justify a huge amount of the investments made in developing a world class research campus.
My dad never got diagnosed with that, at the end, he didn't even know us really. I'm not so sure he didn't have that too.
jizzrag85
Well-Known Member
Wow, this is absolutely incredible if this works. Having a parent that died from ALS, I know the pain of people that see their loved ones die from equally brutal diseases. It's the most heart breaking and demoralizing thing in the world to watch someone you love lose everything about themselves and they can't control it.
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when ? my work blocks twitter stuff so I don't know what's going on... I can only assume Mac is trying a new experimental drug and it's working ... correct ?
It just ran. It was more of a teaser for the upcoming reports. It did show a brief interview with Mac and he looked well. Not a lot of info. Like I said, more of a teaser.
Cannot say what a huge finding this would be. Father in law is in final stages of this horrible disease and any progress or hope with successful trials is so very welcomed.
CB17Cubed
Well-Known Member
This is definitely something that hits close to home, not only have I lost family to Alzheimers/dementia, I know the McCartney family on a personal level. I work with Tom at Fairview and I talk to him on a daily basis. He's told me some stories recently that haven't been promising, but I knew he was being put on this trial drug at CU. I will keep you guys posted if I hear anything from the family, that I feel that I can share.
Story tonight at 9 news at 7:15
http://www.alz.org/research/alzheimers_grants/for_researchers/overview-2016.asp?grants=2016potter
"Dr. Potter and colleagues are already conducting a small, short-term (15 days) Phase I clinical trial to assess the safety of a manufactured version of GM-CSF called Leukine® (sargramostim) in people who have mild to moderate Alzheimer’s disease. They have observed no negative side effects and with the support of the Part the Cloud Challenge award propose a more extensive Phase II clinical trial to test the effects when the drug is administered for 24 weeks.
Dr. Potter’s team plans to enroll 56 people with mild to moderate Alzheimer’s disease and treat them with Leukine or a placebo. The participants will be monitored for side effects and the researchers will determine if Leukine can help slow or prevent disease-related brain changes and cognitive decline. Because Leukine is already FDA-approved to treat other conditions and significant safety data has been collected in humans, it is allowed to be immediately tested in clinical trials for treating Alzheimer’s disease."
"Dr. Potter and colleagues are already conducting a small, short-term (15 days) Phase I clinical trial to assess the safety of a manufactured version of GM-CSF called Leukine® (sargramostim) in people who have mild to moderate Alzheimer’s disease. They have observed no negative side effects and with the support of the Part the Cloud Challenge award propose a more extensive Phase II clinical trial to test the effects when the drug is administered for 24 weeks.
Dr. Potter’s team plans to enroll 56 people with mild to moderate Alzheimer’s disease and treat them with Leukine or a placebo. The participants will be monitored for side effects and the researchers will determine if Leukine can help slow or prevent disease-related brain changes and cognitive decline. Because Leukine is already FDA-approved to treat other conditions and significant safety data has been collected in humans, it is allowed to be immediately tested in clinical trials for treating Alzheimer’s disease."
I know its part of the the trial process and is necessary, but The whole placebo portion saddens me...People getting zero treatment, when they think they are...
It's called a control group. It's pretty much necessary with any drug tests. People may show improvements without taking any medication because the body and mind sometimes have amazing capacity to heal. Or, the metrics being tracked (memory is a weird one to measure) can show improvements that may not be real. It's vital to have a control group to form any reasonable conclusions.
And they understand they may be getting the placebo.
Yep. For most things, it's likely to get statistically significant improvements from a placebo. So the measure of whether a drug is pharmacologically active is whether the improvement versus placebo is statistically significant.
I'm not disputing the validity of control groups and placebos and I also completely understand the need for it. I just hate it, necessary evil or not. its as simple as that
I heard about this last summer at a seminar - my Mom is in the middle stages here. I submitted her app to be part of the study and never heard back....
The next phase of study will last 2-3 years. Here's my deal: My Mom doesn't have 2-3 years. Leukine is already FDA approved for cancer. She is dying of this damn disease. Why not let her try it?
The next phase of study will last 2-3 years. Here's my deal: My Mom doesn't have 2-3 years. Leukine is already FDA approved for cancer. She is dying of this damn disease. Why not let her try it?
Looks like folks with Rheumatoid arthritis have a lot of this GM-CSF in their blood and they have less debilitating results when diagnosed with Alzheimer's. Fascinating.
Background
Most cases of arthritis are caused by normal wear and tear on joints. But a small percentage of cases are caused by abnormal activation of the immune system, and these cases are known as rheumatoid arthritis. There is some evidence to suggest that people with rheumatoid arthritis (RA) may not develop Alzheimer’s at the same rates as individuals without RA. Studies have shown that this inverse correlation cannot be explained by the fact that people with RA use non-steroidal anti-inflammatory drugs (NSAIDS).
Huntington Potter, Ph.D., and colleagues have found that people with RA have unusually high levels of GM-CSF (granulocyte macrophage-colony stimulating factor) in their blood. GM-CSF is a hormone-like protein naturally found in the body that is important for the production and function of immune cells. In mice that have an Alzheimer’s-like condition, treatment with GM-CSF inhibits the development of disease-related brain changes and memory problems. It is thought that GM-CSF may help modulate levels of inflammation in the brain or prevent the accumulation of beta-amyloid – both of these factors are associated with Alzheimer’s disease.
http://www.alz.org/research/alzheimers_grants/for_researchers/overview-2016.asp?grants=2016potter
There is also some sort of link with Down's syndrome. Essentially CU noticed that nearly 100% of Down Syndrome cases get Early onset Alzheimers, and are working to understand the genetics involved with that. I remember Dr. Potter discussing this at the seminar I went to.
People with Down syndrome are born with an extra copy of chromosome 21, which carries the APP gene. This gene produces a specific protein called amyloid precursor protein (APP). Too much APP protein leads to a buildup of protein clumps called beta-amyloid plaques in the brain. By age 40, almost all people with Down syndrome have these plaques, along with other protein deposits, called tau tangles, which cause problems with how brain cells function and increase the risk of developing Alzheimer’s dementia.
People with Down syndrome are born with an extra copy of chromosome 21, which carries the APP gene. This gene produces a specific protein called amyloid precursor protein (APP). Too much APP protein leads to a buildup of protein clumps called beta-amyloid plaques in the brain. By age 40, almost all people with Down syndrome have these plaques, along with other protein deposits, called tau tangles, which cause problems with how brain cells function and increase the risk of developing Alzheimer’s dementia.
Hope they will man, keep pushing it.