Monday, September 7, 2009

Alzheimer treatment--Deadly Wait for Patient Access to Research Breakthrough

Alzheimer's is a particularly horrible affliction. A very good friend of mine had the first symptoms of early onset Alzheimer's at age 58 by age 63, he did not know who is wife or daughter were.

He had been a remarkable man. A talented story teller with a wealth of details in his head. He moved from classroom teacher to district administration and finally to a post as the director of our entire county. Brilliant wonderful person. Now gone. His last years a haze for him and a horror for his family.

Today a research breakthrough in Alzheimer's was announced. A drug was found that reverses Alzheimer's disease in mice! Not only that but it has the potential to treat MANY other diseases including autoimmune and other immune disorders.

The mechanism of action of this ALREADY FDA APPROVED cancer drug is the key. The drug opens up improperly closed and shut down patches of DNA. The DNA can then make the proteins that can reverse Alzheimer's and potentially many many more diseases.

Wow, it's already approved! Whoopee! Let's try it out right now! That would be the reaction of the naive patient or loved one of a patient who has a catastrophic disease. But that poor naive person does not understand the Byzantine slowness of our medical approval process.

Should some doctor try using a drug like this approved medication in an "off label' way then the doctor risks losing his licence and perhaps monetary damages as well. Doctors whose first loyalty is to 'their career investment' will not risk the time and money they spent getting their career in medicine by trying to help a patient like my good friend and his family.

The FDA with its Byzantine labyrinth of rules and regulations slow the translation of medical research breakthroughs to the bedside of patients for even more, not just a few years, BUT DECADES!

Our Big Pharma companies do all in their power to keep new revolutionary treatments or cheaper medicines under wraps and off the market. Nothing must be allowed to affect the tremendous profit they extract from patient's forced to only use existing meds.

A medicine like the one described in the article below that can manipulate and repair genes by fixing our DNA should be tried on a multitude of illnesses immediately upon discovery. There should be a RAPID RESPONSE, BROAD SPECTRUM clinical trial set up in a matter of a few weeks. The trial should include a few, fully informed, volunteers who have such genetic defect diseases as muscular dystrophy, cystic fibrosis, Tay Sachs, hemophilia, and various autoimmune diseases like multiple sclerosis, Rheumatoid Arthritis, Crohn's, psoriasis, etc.

Those with few or no treatments like Lupus and Tay Sachs should be at the TOP of the list. Who knows which of these diseases could be treated and ended with this drug.

This kind of BROAD SPECTRUM clinical testing could answer the question of who could be helped in a matter of weeks.

But no that will not happen. This new medical breakthrough that has already gone through one decade of clinical trials to get approved for cancer will now have to go through decades more of clinical trials for each of the above mentioned diseases IF someone will fund those trials. Of course each new set of clinical trials costs about a BILLION dollars. Yes that is a BILLION with a 'B." In effect generations of patients must suffer and die while the FDA, the Big Pharma Companies and cowardly physicians watch without pity. Patients with diseases that do not affect enough people to promise huge profits may NEVER gain access as no one will pay the BILLION dollars.

Previous posts on this blog have discussed the remarkable promise of these HDAC inhibitors. These compounds have been known about for over a decade. Researchers have talked openly about their incredible potential. Patients wait desperately for a chance to use them. But still today and for the foreseeable future most potential applications to create revolutionary cures with these drugs is VERBOTEN.

Here is the article about the Alzheimer's breakthrough:

(Note: the highlighted line at the end of the article "Because this drug is already approved for cancer we hope clinical trials for Alzheimer's can start in three or four years" NOT THREE or FOUR weeks or even three or four months but THREE or FOUR YEARS!!! Why? Because of the FDA "slow down the cures" stupid rules. Imagine how many decades it would take to get to clinical trials if the F'ing DelAy had not already approved the drug for cancer!

If you are as angry and outraged about these delays as I am, please consider joining Abigail Alliance for better patient access to developmental drugs.


Columbia University Medical Center

Cancer drug may improve memory in Alzheimer's patients

NEW YORK - A drug now used to treat cancer may also be able to restore memory deficits in patients with Alzheimer's disease, according to a new study conducted by scientists at Columbia University Medical Center, which appeared in the September issue of The Journal of Alzheimer's Disease: Volume 18:1.

The loss of short, day-to-day memories is often the first sign of Alzheimer's - a disease that is expected, by the year 2050, to afflict 120 million people worldwide.

"People often joke that they must have Alzheimer's because they can't remember where they put their keys, but for a person with the disease, this type of short-term memory loss is extremely debilitating," says the study's lead author, Ottavio Arancio, Ph.D., associate professor of pathology and cell biology in the Taub Institute for Research on Alzheimer's Disease and the Aging Brain at Columbia University Medical Center.

Dr. Arancio says that the cancer drug targets a previously unknown defect in the brains of mice with Alzheimer's.

The reason why the drug improves memory lies in the way the brain records new memories. To create new memories, the neurons in the brain must manufacture new proteins. The first step is to open up and read the DNA, which contains instructions for making the proteins.

To read the DNA, the neuron attaches a chemical reactive group to the spool around which DNA is tightly wound. "These groups, called acetyls, unwind the DNA to make it more accessible," says co-author Yitshak Francis, Ph.D., a postdoctoral research scientist at Columbia. "It's like unwinding knitting wool from its spool."

This unwrapping step, the researchers found, is impaired in mice with a form of Alzheimer's disease. The mice with Alzheimer's attached about half as many acetyls to DNA as normal mice and had poorer memory.

The researchers then discovered that they could improve memory in the Alzheimer's-afflicted mice with a cancer drug from a family of compounds, called HDAC inhibitors, which increase the DNA's spool acetylation and gene transcription. The drug improved memory performance to the level found in normal mice.

"Because this type of drug has already been approved for some cancer patients," says co-author Mauro Fà, Ph.D., associate research scientist in Columbia's Taub Institute, "we hope that clinical trials for Alzheimer's disease can start in about three to four years."

"For making memories, you need transcription and protein synthesis at the cellular level. If you don't have that, you don't have memory," said Dr. Francis.


This work was supported in part by Alzheimer Disease Research Zenith Award ZEN-07-58977, National Institutes of Health Grant R01 NS049442 (to O.A.) and by United Kingdom Alzheimer's Research Trust Pilot Grant, The International Sephardic Educational Foundation (ISEF) Scholarship, The Lewis Family Trust Scholarship, The Sidney & Elizabeth Corob Charitable Trust Scholarship, the Charlotte and Yule Bogue Research Fellowships (to Y.I.F).

Authors of The Journal of Alzheimer's Disease study include: Yitshak I Francis, Mauro Fà, Haider Ashraf, Hong Zhang, Agnieszka Staniszewski, David S. Latchman and Ottavio Arancio.

The Journal of Alzheimer's Disease ( is an international multidisciplinary journal to facilitate progress in understanding the etiology, pathogenesis, epidemiology, genetics, behavior, treatment and psychology of Alzheimer's disease. The journal publishes research reports, reviews, short communications, book reviews, and letters-to-the-editor. Groundbreaking research that has appeared in the journal includes novel therapeutic targets, mechanisms of disease and clinical trial outcomes. The Journal of Alzheimer's Disease has an Impact Factor of 5.101 according to Thomson Reuters' 2008 Journal Citation Reports. The Journal is published by IOS Press (

The Taub Institute for Research on Alzheimer's Disease and the Aging Brain at Columbia University Medical Center is a multidisciplinary group that has forged links between researchers and clinicians to uncover the causes of Alzheimer's, Parkinson's and other age-related brain diseases and discover ways to prevent and cure these diseases. It has partnered with the Gertrude H. Sergievsky Center at Columbia University Medical Center which was established by an endowment in 1977 to focus on diseases of the nervous system. The Center integrates traditional epidemiology with genetic analysis and clinical investigation to explore all phases of diseases of the nervous system. For more information about these centers visit:

Columbia University Medical Center provides international leadership in basic, pre-clinical and clinical research, in medical and health sciences education, and in patient care. The medical center trains future leaders and includes the dedicated work of many physicians, scientists, public health professionals, dentists, and nurses at the College of Physicians and Surgeons, the Mailman School of Public Health, the College of Dental Medicine, the School of Nursing, the biomedical departments of the Graduate School of Arts and Sciences, and allied research centers and institutions. Established in 1767, Columbia's College of Physicians and Surgeons was the first institution in the country to grant the M.D. degree and is among the most selective medical schools in the country. Columbia University Medical Center is home to the largest medical research enterprise in New York City and state and one of the largest in the United States. For more information, please visit

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