Monday, September 28, 2009

Nanotechnology diagnosis of cancer, autoimmune next?

All of us who have suffered through dozens of doctors appointments and many many medical tests that come back inconclusive before we finally get a diagnosis of what is wrong can only hope that the following nanotechnology will soon be applied to diagnosing autoimmune diseases.

Here is the article:

University of Toronto

U of T researchers create microchip that can detect type and severity of cancer

TORONTO, ON – UofT researchers have used nanomaterials to develop a microchip sensitive enough to quickly determine the type and severity of a patient's cancer so that the disease can be detected earlier for more effective treatment.

Their groundbreaking work, reported Sept. 27 in Nature Nanotechnology heralds an era when sophisticated molecular diagnostics will become commonplace.

"This remarkable innovation is an indication that the age of nanomedicine is dawning," says Professor David Naylor, president of the University of Toronto and a professor of medicine. "Thanks to the breadth of expertise here at U of T, cross-disciplinary collaborations of this nature make such landmark advances possible."

The researchers' new device can easily sense the signature biomarkers that indicate the presence of cancer at the cellular level, even though these biomolecules – genes that indicate aggressive or benign forms of the disease and differentiate subtypes of the cancer – are generally present only at low levels in biological samples. Analysis can be completed in 30 minutes, a vast improvement over the existing diagnostic procedures that generally take days.

"Today, it takes a room filled with computers to evaluate a clinically relevant sample of cancer biomarkers and the results aren't quickly available," says Shana Kelley, a professor in the Leslie Dan Faculty of Pharmacy and the Faculty of Medicine, who was a lead investigator on the project and a co-author on the publication.

"Our team was able to measure biomolecules on an electronic chip the size of your fingertip and analyse the sample within half an hour. The instrumentation required for this analysis can be contained within a unit the size of a BlackBerry."

Kelley, along with engineering professor Ted Sargent – a fellow lead investigator and U of T's Canada Research Chair in Nanotechnology – and an interdisciplinary team from Princess Margaret Hospital and Queen's University, found that conventional, flat metal electrical sensors were inadequate to sense cancer's particular biomarkers. Instead, they designed and fabricated a chip and decorated it with nanometre-sized wires and molecular "bait."

"Uniting DNA – the molecule of life – with speedy, miniaturized electronic chips is an example of cross-disciplinary convergence," says Sargent. "By working with outstanding researchers in nanomaterials, pharmaceutical sciences, and electrical engineering, we were able to demonstrate that controlled integration of nanomaterials provides a major advantage in disease detection and analysis."

The speed and accuracy provided by their device is welcome news to cancer researchers.

"We rely on the measurement of biomarkers to detect cancer and to know if treatments are working," says Dr. Tom Hudson, president and scientific director of the Ontario Institute for Cancer Research. "The discovery by Dr. Kelley and her team offers the possibility of a faster, more cost-effective technology that could be used anywhere, speeding up diagnosis and helping to deliver a more targeted treatment to the patient."

The team's microchip platform has been tested on prostate cancer, as described in a paper published in ACS Nano, and head and neck cancer models. It could potentially be used to diagnose and assess other cancers, as well as infectious diseases such as HIV, MRSA and H1N1 flu.

"The system developed by the Kelley/Sargent team is a revolutionary technology that could allow us to track biomarkers that might have significant relevance to cancer, with a combination of speed, sensitivity, and accuracy not available with any current technology," says Dr. Fei-Fei Liu, a radiation oncologist at Princess Margaret Hospital and Head of Applied Molecular Oncology Division, Ontario Cancer Institute. "This type of approach could have a profound impact on the future management for our cancer patients."


The research was funded by the Canada Foundation for Innovation, the Natural Sciences and Engineering Research Council of Canada, the Ontario Genomics Institute, Genome Canada, the Ontario Institute for Cancer Research, the Ontario Ministry of Research and Innovation and the Prostate Cancer Research Foundation of Canada.

Broken bones? Blame it on autoimmune disease. TNF alpha the culprit

After having had three broken arms, a broken collar bone, a broken pelvis, a broken hand bone, and 12 or so broken ribs, I think I am an expert on broken bones. I know how they feel when they break (like a stick snapping inside--but with a bit of dizzying shock sensation mixed with relatively minor pain initially--later the pain at the site of the break can be excruciating when the spot is lightly touched).

I used to blame my many broken bones on having small diameter bones. Later I found out that the particular kind of immune problems I have caused mal absorbtion in the intestines so that bones did not receive enough calcium and phosphorous. Now I read that it is caused by too much systemic inflammation as in too much TNF alpha--the inflammatory immune signaling molecule that has been found to be a problem in many autoimmune diseases--RA, PsA, AS, and psoriasis to mention a few. Remicade, Enbrel, and Humira are medications that absorb or block TNF alpha thus reducing the effects of several autoimmune diseases.

I remember when my father got made at me for breaking my arm AGAIN! He told me it was expensive to get fixed and I must be more careful. I thought I was being careful. Certainly more so than my friends were being. So when I broke my collar bone and could not raise my right arm, I did not tell my parents. They never noticed. I just laid my right arm against the edge of the table at dinner and used that as a pivot to pick up food with my fork on my plate, then bend my head down and ate. I wanted to use my left hand but thought they would notice I was using the wrong hand. After several weeks the collar bone got better. It healed a little crooked but still works. Later as an adult during a physical, my doctor noticed the crooked bone and mentioned it to me. Other than that I got away with not telling my parents.

I have found over the years that most bones heal on their own with no need for doctors if you do not mind a little pain and a bit of a crooked bone here and there (can't turn over my left hand because one of the wrist bones is pretty crooked but arm works well otherwise.)

Here is the article about TNF alpha causing broken bones and slow healing bones.

American Journal of Pathology

Diabetes weakens your bones

Boston, MA and Newark, NJ – Current research suggests that the inflammatory molecule TNF-α may contribute to delayed bone fracture healing in diabetics. The related report by Alblowi et al, "High Levels of TNF-α Contribute to Accelerated Loss of Cartilage in Diabetic Fracture Healing" appears in the October 2009 issue of the American Journal of Pathology.

Diabetes, a condition where the body either does not produce enough, or respond to, insulin, affects at least 171 million people worldwide, a figure that is likely to double by 2030. Long-term complications of diabetes include cardiovascular disease, chronic renal failure, retinal damage that may lead to blindness, nerve damage, and blood vessel damage, which may cause erectile dysfunction and poor wound healing.

Diabetic patients often experience low bone density, which is associated with increased risk of bone fractures and delayed fracture repair. To examine how diabetes affects bone, Dr. Dana Graves and colleagues of the University of Medicine and Dentistry of New Jersey and the Boston University School of Medicine explored bone repair in a mouse model of diabetes. They observed increased levels of inflammatory molecules, including TNF-α during fracture healing. The diabetic animals had rapid loss of cartilage in the healing bones, which was due to increased numbers of osteoclasts, cells that remove bone and cartilage. Factors that stimulate osteoclast formation were regulated by both TNF-α and a downstream mediator, FOXO1. These results suggest that diabetes-mediated increases in TNF-α and FOXO1 may underlie the impaired healing of diabetic fractures.

Alblowi et al suggest that "TNF-α dysregulation plays a prominent role in the recently identified catabolic events associated with diabetic fracture healing." In future studies, Dr. Graves and colleagues plan to "examine the effect of FOXO1 on mineralized tissue to examine how it may regulate factors that control bone resorption and osteoclastogenesis, in addition to effects it may have on osteoblastic cells."


This work was supported by grants from the National Institutes of Health.

Alblowi J, Kayal RA, Siqueira M, McKenzie E, Krothapalli N, McLean J, Conn J, Nikolajczyk B, Einhorn TA, Gerstenfeld L, Graves DT: High Levels of TNF-α Contribute to Accelerated Loss of Cartilage in Diabetic Fracture Healing. Am J Pathol 2009 175: 1574-1585


The American Journal of Pathology, official journal of the American Society for Investigative Pathology, seeks to publish high-quality, original papers on the cellular and molecular biology of disease. The editors accept manuscripts that advance basic and translational knowledge of the pathogenesis, classification, diagnosis, and mechanisms of disease, without preference for a specific analytic method. High priority is given to studies on human disease and relevant experimental models using cellular, molecular, animal, biological, chemical, and immunological approaches in conjunction with morphology.

Drug to help genes make proteins to END genetic diseases like autoimmune

Chemicals to help cells read genes with "nonsense" mutations that normally stop the reading process, will allow these genes to produce missing proteins whose loss causes many, many genetic diseases including autoimmune diseases, allergy, asthma, muscular dystrophy, hemophilia, Tay Sachs, and a host of others.

These chemicals allow gene to work and to stop the horror of a genetic disease. Up until now we have been stuck with the genes we were dealt at fertilization. With these new chemicals, even those of us with genetic disease have the chance to live normal lives.

In a perfect world, there would be massive interest in these chemicals. Congress would pass vast spending bills to finance their development and testing and within years possible sooner, these chemicals would be in use by suffering and dying patients.

Of course we do not live in a perfect world. I expect these chemicals to be curing my great grand children but not anyone before that. The FDA likes to delay, delay, delay. The NIH only funds a tenth of the good proposals that they get and it is nice to have political pull to assure an NIH grant.

Here is the article about these new wonderful chemicals:

University of California - Los Angeles

UCLA study identifies 2 chemicals that could lead to new drugs for genetic disorders

Discovery could help people with cancer, muscular dystrophy, A-T

UCLA scientists have identified two chemicals that convince cells to ignore premature signals to stop producing important proteins. Published in the Sept. 28 edition of the Journal of Experimental Medicine, the findings could lead to new medications for genetic diseases, such as cancer and muscular dystrophy, that are sparked by missing proteins.

"When DNA changes, such as nonsense mutations, occur in the middle rather than the end of a protein-producing signal, they act like a stop sign that tells the cell to prematurely interrupt protein synthesis," explained Dr. Richard Gatti, professor of pathology and laboratory medicine and human genetics at the David Geffen School of Medicine at UCLA. "These nonsense mutations cause the loss of vital proteins that can lead to deadly genetic disorders."

Gatti's lab specializes in studying ataxia-telangiectasia (A-T), a progressive neurological disease that strikes young children, often killing them by their late teens or early 20s.

For four years, the UCLA Molecular Shared Screening Resources Center of the campus' California NanoSystems Institute has screened 35,000 chemicals, searching for those that ignore premature stop signals.

First author Liutao Du developed the screening technology in Gatti's laboratory.

"Of the dozens of active chemicals we discovered, only two were linked to the appearance and function of ATM, the protein missing from the cells of children with A-T," said Du. "These two chemicals also induced the production of dystrophin, a protein that is missing in the cells of mice with a nonsense mutation in the muscular dystrophy gene."

The UCLA team is optimistic that their discovery will aid pharmaceutical companies in creating drugs that correct genetic disorders caused by nonsense mutations.

This could affect one in five patients with most genetic diseases, including hundreds of thousands of people suffering from incurable diseases.

Because nonsense mutations can lead to cancer, such drugs may also find uses in cancer treatment.


Gatti's lab is funded by the Los Angeles-based Ataxia-Telangiectasia Medical Research Foundation, the National Institutes of Health and the New York-based Ataxia-Telangiectasia Ease Foundation.

The study's coauthors included Robert Damoiseaux, Shareef Nahas, Kun Gao, Hailiang Hu, Julianne Pollard, Jimena Goldstine, Michael Jung, Susan Henning and Carmen Bertoni, all of UCLA.

More evidence for benefits of Worms--Vietnam study

Intestinal worms get another boost as a way to modulate improperly functioning immune systems. Allergies, asthma and autoimmune all are possible candidates for immune dysfunctions that may be treatable with intestinal worms. Here is the UARL of the latest article:

Gut worms may protect against house-dust mite allergy
Mon, 28 Sep 2009 12:35:00 GMT
PA 252/09

A study conducted in Vietnam has added further weight to the view that parasitic gut worms, such as hookworm, could help in the prevention and treatment of asthma and other allergies.

Led by Dr Carsten Flohr, a Clinical Scientist from The University of Nottingham, and Dr Luc Nguyen Tuyen from the Khanh Hoa Provincial Health Service in central Vietnam, the study is the largest double-blind placebo controlled clinical trial to date looking at the potential links between hookworm and other gut worm infections and allergic conditions such as asthma and eczema.

Thanks to improved hygiene practices parasitic worms have been mostly eradicated among human populations living in developed countries. However, experts believe that over millions of years of co-evolution worms have found methods to dampen down host immune responses to prolong their own survival inside humans. This relationship seems to have become so intertwined that without gut worms or other parasites, our immune system can become unbalanced, which in turn could contribute to the development of asthma and other allergies. At the same time, it is important to remember that gut parasites can cause severe disease and are a major cause of iron-deficiency anaemia in developing countries.

Dr Flohr’s study was conducted in a rural area of central Vietnam where two out of three children have hookworm and other gut parasite infections and where allergies are extremely rare. More than 1,500 schoolchildren aged 6-17 took part.

The team investigated whether repeated tablet treatments to clear the body of gut worms made it more likely for children to develop allergic conditions. While the treatment did not demonstrate an effect on asthma or eczema, the treated children had a significantly increased risk of having a positive allergy skin test to house-dust mites and cockroach. This suggests that gut worms have the potential to tone down human immune responses and so further research is now needed to identify precisely how gut worm infection can prevent allergic sensitisation.

Dr Carsten Flohr of The University of Nottingham adds: “The next step is to understand exactly how and when gut parasites programme the human immune system in a way that protects against allergic sensitisation, and for such studies, follow-up from birth will be essential.”

As up to 80 per cent of people with asthma also have allergies to house-dust mites and other environmental allergens, additional research in this area could aid the creation of new treatments that work in the same way as gut parasites, by dampening down or rebalancing the immune system so that the body does not respond to allergens and trigger asthma attacks.

Dr Elaine Vickers, Research Relations Manager at Asthma UK, says: “Asthma affects more than five million people in the UK, with a person being admitted to hospital every seven minutes following an asthma attack. The prospects of further studies in this area are therefore very exciting as we could see groundbreaking treatments for asthma and other allergies developed as a result. It’s now vital that we see more funding being invested in this important area of research, so that we can increase our understanding of the link between gut parasites and the development of allergies from birth.”

Co-applicants on the Asthma UK research grant that funded the work were Professors John Britton, David Pritchard, and Hywel Williams. The Nottingham team collaborated with researchers from the Wellcome Trust Major Overseas Programme at the Oxford University Clinical Research Unit Hospital for Tropical Diseases in Ho Chi Minh City, where Dr Flohr was based for his work. Additional funding was received through the Bastow Award from the Special Trustees for Nottingham University Hospitals. Dr Flohr’s salary was provided by University College, University of Oxford, for two years and by the Wellcome Trust UK for a further nine months.

— Ends —

Notes to editors: The University of Nottingham is ranked in the UK's Top 10 and the World's Top 100 universities by the Shanghai Jiao Tong (SJTU) and Times Higher (THE) World University Rankings.

More than 90 per cent of research at The University of Nottingham is of international quality, according to RAE 2008, with almost 60 per cent of all research defined as ‘world-leading’ or ‘internationally excellent’. Research Fortnight analysis of RAE 2008 ranks the University 7th in the UK by research power. In 27 subject areas, the University features in the UK Top Ten, with 14 of those in the Top Five.

The University provides innovative and top quality teaching, undertakes world-changing research, and attracts talented staff and students from 150 nations. Described by The Times as Britain's “only truly global university”, it has invested continuously in award-winning campuses in the United Kingdom, China and Malaysia. Twice since 2003 its research and teaching academics have won Nobel Prizes. The University has won the Queen's Award for Enterprise in both 2006 (International Trade) and 2007 (Innovation — School of Pharmacy), and was named ‘Entrepreneurial University of the Year’ at the Times Higher Education Awards 2008.

Nottingham was designated as a Science City in 2005 in recognition of its rich scientific heritage, industrial base and role as a leading research centre. Nottingham has since embarked on a wide range of business, property, knowledge transfer and educational initiatives ( in order to build on its growing reputation as an international centre of scientific excellence. The University of Nottingham is a partner in Nottingham: the Science City.

Asthma UK is one of the major funders of asthma research in the UK. Asthma UK is the charity dedicated to improving the health and well-being of the 5.4 million people in the UK whose lives are affected by asthma. For up-to-date news on asthma, information and publications, visit the Asthma UK website For independent and confidential advice on asthma, call the Asthma UK Adviceline, which is staffed by asthma nurse specialists. It is open weekdays from 9am to 5pm on +44 (0)800 121 62 44.

Sunday, September 27, 2009

Stelara APPROVED! by FDA for psoriasis. WHOOPEE!

The long awaited alternative to TNF-alpha inhibitors has FINALLY been approved. More than TWO YEARS after ALL clinical trials were successfully completed and after more than eighteen months since Centocor submitted the New Drug Application for approval, the FDA (aka F'ing DelAy) is allowing patients with psoriasis to use Stelara, a marvelous new biologic.

It works on a completely different cytokine (immune signal) pathways. It blocks different immune signaling molecues--interleukin 12 and interleukin 23. Down regulating these two pathways is supposed to increase the number of T-regulatory cells in the body. T-regs turn down inflammation the main underlying problem in autoimmune disease (and many other conditions as well i.e. cardiovascular disease and even some cancers).

Of course Canada Health approved Ustekinumab (Stelara )a year ago as did the European Union.

In July of 2008 a panel of independent experts convened by FDA to assess Stellara recommended UNANIMOUSLY for approval to the F'ing DelAY. Now just a short (not!) fourteen months later the incompetents at the FDA finally will allow US citizens the right to use the medication.

Sorry to be bitter.

The STUPID and unnecessary delays of this needed NOVEL, FIRST OF ITS KIND medication in our country just demonstrates how very dysfunctional the FDA is.

Good news is we can use it now. One shot is supposed to last a whole month. Now the only problem is paying for that shot. I have read that in Canada--Centocor charges as much as $10,000! Gee, no problem--only $120.000 a year. Let's hope that Centocor will reduce the price in the US. I know I know Big Pharma reducing prices to consumers--like pigs flying I will only believe it when I see it. Perhaps health care reform will force down prices. Hope so.

Read more at this URL:

Here is the article:

FDA Approves Ustekinumab for Moderate to Severe Psoriasis
ROCKVILLE, Md -- September 25, 2009 -- The US Food and Drug Administration (FDA) has approved ustekinumab (Stelara), a biologic product for adults who have a moderate to severe form of psoriasis.

"This approval provides an alternative treatment for people with plaque psoriasis, which can cause significant physical discomfort from pain and itching and result in poor self-image for people who are self-conscious about their appearance," said Julie Beitz, MD, Office of Drug Evaluation in the FDA's Center for Drug Evaluation and Research, Rockville, Maryland.

Three studies of 2,266 patients evaluated the biologic's safety and effectiveness.

The FDA approval was primarily based on 2 phase 3 studies in which a significantly higher proportion of patients receiving either ustekinumab 45 mg or 90 mg achieved at least a 75% reduction in psoriasis as measured by the Psoriasis Area and Severity Index (PASI 75) at week 12 compared with patients receiving placebo.

With every-12-week ustekinumab maintenance therapy, the majority of patients achieving a PASI 75 improvement maintained substantial skin clearance for 1 year.

Since ustekinumab reduces the immune system's ability to fight infections, the product poses a risk of infection. Serious infections have been reported in patients receiving the product and some of them have lead to hospitalisation. These infections were caused by viruses, fungi, or bacteria that have spread throughout the body. There may also be an increased risk of developing cancer.

The FDA is requiring a risk evaluation and mitigation strategy (REMS) for ustekinumab that includes a communication plan targeted to healthcare providers and a medication guide for patients.

SOURCE: US Food and Drug Administration.

Wednesday, September 16, 2009

Color Blindness Cured points to technique for Autoimmune Cure

In Florida researchers have used a virus to ferry in a gene to allow red/green color blind monkeys to see those the entire normal monkey and human color spectrum.

This same technology should allow the transfer of genes into humans. The same virus, adeno associated virus, has been used for at least a decade for gene transfers including very large genes which could cure (yes, cure) a host of genetic diseases--muscular dystrophy, sickle cell anemia, hemophilia, and all autoimmune disease.

My interest is autoimmune disease due to my son. There are a number of gene candidates that could be transferred into the cells in our bone marrow that make our blood and immune cells. The genes for IL-10. Variants of the FOXP3 gene found in healthy folks without autoimmune and allergy/asthma problems are another candidate. Other genes are possible as well.

What we need is a facility, free from all possibility of law suits, free of all FDA stupid bureaucratic clinical trial rules, free of competition between different companies and researchers.

This place should be a WIKI place where all drug companies and researchers share their ideas as a group. Where they all also share in any profits that come as a result of any group's discovery. Where ALL researchers eat together and meet together to share ideas of what is working.

It should be a place where patients can come and have the ability to try anything that might help them. No rules EXCEPT complete transparency and complete openness. NO secrets allowed. Nothing held back. Every patient encouraged to go online daily and tell exactly how they feel about their treatments and doctors without any censorship or fear of retaliation (Possibly only published after the patient is cures, quits treatment or dies).

I am convinced that if we, the sick, and their relatives were allowed to create a place of total freedom for research cures for most of the great genetic disease would come in months. Of course, my dream has no chance. Too many people with power at too many corporations make too much money off of people suffering under the current system.

If we cannot even get Medicare for all in the US due to polarization created by big money buying radio and TV con men as spokesman to rile up the mob, we have no chance of a place for quick cures using WIKI type organization. Too bad.

Enough editorialising.

Here is the color blind cure article:

Scientists cure color blindness in monkeys

GAINESVILLE, Fla. — Researchers from the University of Washington and the University of Florida used gene therapy to cure two squirrel monkeys of color blindness — the most common genetic disorder in people.

Writing online Wednesday in the journal Nature, scientists cast a rosy light on the potential for gene therapy to treat adult vision disorders involving cone cells — the most important cells for vision in people.

"We've added red sensitivity to cone cells in animals that are born with a condition that is exactly like human color blindness," said William W. Hauswirth, Ph.D., a professor of ophthalmic molecular genetics at the UF College of Medicine and a member of the UF Genetics Institute and the Powell Gene Therapy Center. "Although color blindness is only moderately life-altering, we've shown we can cure a cone disease in a primate, and that it can be done very safely. That's extremely encouraging for the development of therapies for human cone diseases that really are blinding."

The finding is also likely to intrigue millions of people around the world who are colorblind, including about 3.5 million people in the United States, more than 13 million in India and more than 16 million in China. The problem mostly affects men, leaving about 8 percent of Caucasian men in the United States incapable of discerning red and green hues that are important for everyday things like recognizing traffic lights.

"People who are colorblind feel that they are missing out," said Jay Neitz, Ph.D., a professor of ophthalmology at the University of Washington. "If we could find a way to do this with complete safety in human eyes, as we did with monkeys, I think there would be a lot of people who would want it. Beyond that, we hope this technology will be useful in correcting lots of different vision disorders."

The discovery comes about 10 years after Neitz and his wife Maureen Neitz, Ph.D., a professor of ophthalmology at the University of Washington and senior author of the study, began training two squirrel monkeys named Dalton and Sam.

In addition to teaching the animals, the Neitz research group worked with the makers of a standard vision-testing technique called the Cambridge Colour Test to perfect a way the monkeys could "tell" them which colors they were seeing.

The tests are similar to ones given to elementary children the world over, in which students are asked to identify a specific pattern of colored dots among a field of dots that vary in size, color and intensity. The researchers devised a computer touch screen the monkeys could use to trace the color patterns. When the animals chose correctly, they received a reward of grape juice.

The rest of the article can be found at the URL at the beginning of this post.

Tuesday, September 8, 2009

Researchers Reply to previous "Deadly Wait" post

A researcher form Columbia responded to the previous blog saying:

Let me please first clarify that there is currently no drug that is FDA approved for one disease that is helpful for AD.

The drug that was used in this study, TSA, is not currently approved by the FDA, and is just from the same class of HDAC inhibitors of the drug that was investigated in this study. (A currently HDACi that is FDA approved for cutaneous tcell lymphoma, Vorinostat (SAHA), marketed under the name Zolinska, was not used in this study).

He also wrote:

The drug tested in this study is not FDA approved and happens to be from a class of HDAC inhibitors (of which a current cancer drug belongs).

While I very much appreciate his response, I still feel a great amount of rage over delays to cures for my son and for my good friend who had Alzheimer's.

A Wiki approach to finding cures where EVERYONE contributes to the cures and EVERYONE who contributes gets credit and a piece of the profit could give us cures to the easy diseases like autoimmune, allergy, asthma, and Alzheimer's, hemophilia, and sickle cell anemia in months. That is right MONTHS instead of decades under our current system.

The individualized, private, "hide my discoveries, so I can get a bigger profit," drug discovery process in use today will take decades, if not centuries to get to those cures.

Society at large is at increasing risk for each day of delay in implementing a WIKI type drug discovery process. I am talking drug resistant bacteria (including a now completely resistant strain of tuberculosis--NOTHING kills it!). Until we get serious about finding cures for diseases as fast as possible, we are all at risk and that risk increases daily.

Perhaps when enough of us our dying of resistant bacterial infection, we will adopt the WIKI approach and find cures fast. That is the only hope for those of us either personally or with a loved one afflicted with catastrophic disease.

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:

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