Thursday, June 12, 2014

Who Owns You?

By Walter E. Williams

Darcy Olsen, president of the Arizona-based Goldwater Institute, and Richard Garr, president of Neuralstem, a biotech company, wrote "Right to Try experimental drugs" in USA Today (5/28/2014). They pointed out that "this year, more than 5,000 Americans will lose their battle with ALS, commonly known as Lou Gehrig's disease." Up until recently, there was no medicine on the market that significantly improved the lives of ALS patients. But now there is one in clinical trials that holds considerable promise, but it has not been granted Food and Drug Administration approval. The average amount of time it takes to get a drug through the FDA approval process is 10 years. That's time that terminal patients don't have.

Legislators in Colorado, Louisiana and Missouri recently approved "Right to Try" legislation, and Arizona voters will vote on the measure this November. "Right to Try" is an initiative designed by the Goldwater Institute. It would give terminal patients access to investigational drugs that have completed basic safety testing. Under a doctor's supervision, people would be given the chance to try promising experimental drugs before they're given final FDA approval.

There's no denying that there's risk in taking a drug or medical procedure that hasn't completed clinical trials. The question is: Who has the right to decide how much risk a person will take — he or some faceless Washington bureaucrat? In my opinion, the answer depends upon the answer to the question: Who owns you? If one owns himself, then it is he who decides how much risk he takes. If government owns you, then you don't have the right to unilaterally decide how much risk you'll take.

The FDA's mission is to ensure the safety and effectiveness of pharmaceuticals. In doing so, FDA officials can make two types of errors. They can approve a drug that has unanticipated dangerous side effects, or they can disapprove or delay a drug that is both safe and effective. FDA officials have unequal incentives to avoid these two types of errors. If the FDA official errs on the side of under-caution — approving a dangerous drug — the victims are visible, and he is held directly accountable. If he errs on the side of over-caution — holding up approval of a safe and effective drug — who's to know? The cost and the victims are invisible. Politicians and bureaucrats prefer invisible victims.

Here are a couple of notable examples. Clozapine was approved and used in 1972 in Europe. Clozapine's ability to treat schizophrenics who did not respond to other medicines became well-known by 1979. Yet the drug was not approved in the United States until 1989 because companies believed that the FDA would reject it on the grounds that 1 percent of patients who took the drug contracted a blood disease. As an article in The New England Journal of Medicine stated, "what is remarkable is that clozapine has a beneficial effect in a substantial proportion (30 to 50 percent) of patients who have an inadequate response to other ... drugs." Nearly 250,000 people with schizophrenia suffered needlessly, when relief was at hand.

According to Robert M. Goldberg, writing for the journal Regulation, "Mevacor is a cholesterol-lowering drug that has been linked to reduction in death due to heart attacks. It was available in Europe in 1989 but did not become available in the United States until 1992. Studies confirm what doctors saw to be the case: taking the drug reduces death due to heart disease by about 55 percent. During that three-year period as many as a thousand people a year died from heart disease because of the FDA delay."

There is self-correction when a drug that has unanticipated dangerous side effects has been marketed. The drug is removed. But there's no self-correction when a safe, effective lifesaving drug is not approved or is delayed. Those 5,000 ALS patients who will die of their disease this year are invisible, and FDA officials are unaccountable. "Right to Try" legislation is a step in the right direction to remedy that.

Walter E. Williams is a professor of economics at George Mason University and author of Race & Economics: How Much Can Be Blamed on Discrimination?



  1. Disease Modification of ALS, Parkinson’s Disease and Ischemic Stroke
    About Genervon Biopharmaceuticals LLC
    - Genervon has discovered and developed a new class of “master regulator” bio-drugs that develop, protect and correct the human nervous system.

    - In the past year, Genervon has been conducting three Phase II trials for amyotrophic lateral sclerosis (“ALS”), Parkinson’s disease and ischemic stroke.
    - Genervon recently successfully completed its Phase IIa clinical trial for ALS. A full analysis of the trial’s result is expected to be completed soon. But preliminary data suggests that Genervon’s novel, proprietary, multi-target biological drug candidate, GM604, shows significant promise for treating ALS.

    - Following the completion of six doses of GM604 over two weeks, treated ALS patients were evaluated 10 weeks later without further treatment. In seven out of eight ALS patients treated with GM6 the preliminary clinical measurements of ALS disease progression have slowed or stopped.

    CEOCFO: Is the medical community paying attention?

    Mr. Ko: We receive many emails from doctors asking for our investigational drug because they have patients who are dying without viable treatment options. However, we have to wait for the FDA’s approval before we can give them the drug.

    they don't want cures...think about this...if we cured cancer would that be helpful or harmful to the economy? more chemo milking clinics, reduction in surgeries, less lab work, etc...

    who wins? who loses? the patient is an annuity.

    this system is a joke...if your sick.

  2. Cancer pill fights disease and gives lifelong protection
    British scientists have discovered a drug which fights all cancers by boosting the body's defences and prevents the disease returning by increasing natural immunity

    A pill which boosts the body’s natural defences could help fight off all cancers and stop them ever returning, scientists believe.

    ‘Delta-inhibitors’ were already known to help leukaemia patients, but researchers were amazed to find they also work on a whole range of other cancers.

    The drugs, which are taken orally as a pill, were so successful in leukaemia trials that the control group, who were taking placebos, were immediately switched to the medication on ethical grounds.

    Now, scientists at UCL and the Babraham Institute in Cambridge, have discovered that the same ‘delta inhibitors’ are also effective against lung, pancreatic, skin and breast cancers, and probably many more.

    Cancer suppresses the immune system by producing an enzyme called ‘p100delta’ which tells it to power down, making it difficult for the body to fight the disease. The drugs ‘inhibit’ that enzyme, allowing the immune system to attack tumour cells.

    “Our work shows that delta inhibitors can shift the balance from the cancer becoming immune to our body’s defences towards the body becoming immune to the cancer,” said study co-leader Dr Klaus Okkenhaug of the Babraham Institute at Cambridge University.

    “This provides a rationale for using these drugs against both solid and blood cancers, possibly alongside cancer vaccines, cell therapies and other treatments that further promote tumour-specific immune responses.”

    The drugs are already being used in clinical trials and have been granted Breakthrough Therapy status by the Federal Drugs Agency in the US, which means their development has been speeded up.
    They could be available within just a few years if approved by European regulators and the National Institute of Clinical Excellence.

    in a few years many will have died.................

  3. A ‘Vaccine’ for Heart Disease Could Mean No Pills, Lettuce or a Gym

    It’s the latest in gene therapy, and it’s lowered cholesterol and heart attacks in mice. People are next

    Doctors, and especially doctors who do research, don’t like to use the words cure or eradicate. They know how dangerous that can be, since the human body is so unpredictable. But Dr. Kiran Musunuru is showing some uncharacteristic swagger about his latest success in lowering heart attack risk among some lucky mice.

    Taking advantage of advances in genetic engineering, a team lead by Musunuru, who holds positions at Harvard University’s Department of Stem Cell and Regenerative Biology and Brigham and Women’s Hospital, have edited the genomes of mice and successfully protected them from heart disease. The results, published in the journal Circulation Research, hint at an entirely new way of avoiding the leading killer of Americans by possibly cutting heart attack risk by up to 90%. “What has me excited as a cardiologist is that my goal is eradicating disease,” says Musunuru. “There is no bolder way I can put it. I want to eradicate the disease and this offers one potential way to do it.”

    He admits that it may be 10 years or more before the technique is ready for testing in people, but these first results are enough to justify the research that could make that happen.

    why ten years?...b/c he needs a steady job?

    population control?

  4. Embryonic stem cells offer treatment promise for multiple sclerosis
    Scientists in the University of Connecticut's Technology Incubation Program have identified a novel approach to treating multiple sclerosis (MS) using human embryonic stem cells, offering a promising new therapy for more than 2.3 million people suffering from the debilitating disease.

    The researchers demonstrated that the embryonic stem cell therapy significantly reduced MS disease severity in animal models, and offered better treatment results than stem cells derived from human adult bone marrow.

    The study was led by ImStem Biotechnology Inc. of Farmington, Conn., in conjunction with Advanced Cell Technology (ACT) Inc. of Massachusetts. ImStem was founded in 2012 by UConn doctors Xiaofang Wang and Ren-He Xu, along with Yale University doctor Xinghua Pan and investor Michael Men.
    There is no cure for MS, a chronic neuroinflammatory disease in which the body's immune system eats away at the protective sheath called myelin that covers the nerves. Damage to myelin interferes with communication between the brain, spinal cord, and other areas of the body. Current MS treatments only offer pain relief, and slow the progression of the disease by suppressing inflammation.

    "The beauty of this new type of mesenchymal stem cells is their remarkable higher efficacy in the MS model," says Wang, chief technology officer of ImStem.

    The group's findings appear in the current online edition of Stem Cell Reports, the official journal of the International Society for Stem Cell Research. ImStem is currently seeking FDA approval necessary to make this treatment available to patients.

  5. California Stem Cell Agency Approves $14 Million for Landmark hESC Clinical Trial

    The scientists who reviewed the Asterias application said the therapy could have a “highly significant impact” on the spinal cord injury, which afflicts more than 200,000 people nationwide. According to a CIRM review summary, the reviewers said a successful result from the trial would be a “high visibility achievement for the entire field of stem cell-based/regenerative medicine.”

    The Geron clinical trial was the first ever in the United States for a therapy based on human embryonic stem cells, an area of research roiled by controversy. Some persons believe that deriving such cells is tantamount to killing a human being.

    Geron submitted nearly 28,000 pages of material to the FDA in its years-long bid to start the trial, which began in 2010. However, in November 2011, the company stunned the stem cell world by giving up on the trial, citing business reasons.

    The action also shocked the stem cell agency, which less than four months earlier had signed an agreement loaning the company $25 million. The agency's governing board gave the go-ahead on the loan during a process that involved major departures from its normal procedures. Geron repaid the loan with interest.

    it took ten years to get this trial going and months to shut it down...why? the money excuse was nonsense. There were patient families willing to move it to Mexico.

  6. The great stem cell dilemma

    Finally, I visit someone who's even more familiar with how game-changing industries are born: Andy Grove, one of the godfathers of the semiconductor industry. He has invested in four biotech funds, is involved in a clinical trial of a drug for Parkinson's disease, has pledged as much as $40 million to the Michael J. Fox Foundation, and has been thinking a lot about the promise of stem cells lately. And yet, at his own foundation's office in Los Altos, I find him to be surprisingly full of doom and gloom. He rejects any comparison of stem cells to transistors. "The sun shone on that industry. The government had a stake, the consumer had a stake, and the telecommunications industry had a stake," he says. "This industry is just as important, but after that the similarities are gone."

    For close to two hours, Grove argues passionately about how the FDA is enabling predatory offshore industries by impeding progress and the many reasons financiers want no part of stem cells. "VCs aren't interested because it's a shitty business," he says. Big Pharma? Forget it. CIRM? "There are gleaming fucking buildings everywhere. That wasn't necessary." When I press him to be constructive, he wearily offers one possible solution. Rather than courting billionaires to put their names on buildings, we need a system of targeted philanthropy in which the 99% can sponsor the individual stem cell lines that matter to them.

    Even suffering from Parkinson's at 76 years old, Grove has plenty of fire in him. On this issue he seems particularly zealous, and clearly frustrated. He argues until he's exhausted, and politely asks if we may conclude. On parting, he looks me in the eye, shakes my hand, and offers one final thought. "What you're trying to do is extremely difficult, but also extremely worthy," he says. "I don't envy you. But you'll be a better man for it when it's done."
    It was clear during our talk that Grove wants an economic model for stem cell research and development to emerge, even if he's not willing to bet money on its happening. And that puts him in good company. According to a recent Gallup poll, 62% of Americans now consider embryonic stem cell research to be morally acceptable, and that attitude is pretty consistent across all age groups. It's a healthy sign that public sentiment is strengthening in the way it often does with scientific breakthroughs. First we fear the different or unknown. Then we realize how much it may help us or the ones we love. It's hard to imagine that there was once moral outrage over in vitro fertilization now that it's become fodder for reality television.

    Keirstead also has progressive ideas about how to lower development costs. He wants to offshore clinical trials in the same way corporate America has outsourced business processes. Many stem cell scientists worry about the U.S. losing a stem cell arms race to China. But Keirstead wants to drag the industry there on his terms. He recently founded China Stem Cell in Shanghai, and expects to start conducting trials there before long. "If you look at the old companies, with all their ups and downs and tiring out investors, they just have so much internal baggage," he says. "Running trials in China would be cheaper. I'm not advocating that people skirt FDA quality. Take the standards with you. You can do 100 or 1,000 patients there and be 20% of the cost of an American trial."

    There's a theory in evolutionary biology called punctuated equilibrium. It proposes that things don't happen gradually. We go through vast periods of stasis in which the status quo rules, and then, once in a great while, profound disruption occurs.

  7. Findings point toward one of first therapies for Lou Gehrig’s disease

    “I’m very optimistic,” said Beckman, who received the 2012 Discovery Award from the OHSU Medical Research Foundation as the leading medical researcher in Oregon.

    ALS was first identified as a progressive and fatal neurodegenerative disease in the late 1800s and gained international recognition in 1939 when it was diagnosed in American baseball legend Lou Gehrig. It’s known to be caused by motor neurons in the spinal cord deteriorating and dying, and has been traced to mutations in copper, zinc superoxide dismutase, or SOD1. Ordinarily, superoxide dismutase is an antioxidant whose proper function is essential to life.

    When SOD1 is lacking its metal co-factors, it “unfolds” and becomes toxic, leading to the death of motor neurons. The metals copper and zinc are important in stabilizing this protein, and can help it remain folded more than 200 years.

    “The damage from ALS is happening primarily in the spinal cord and that’s also one of the most difficult places in the body to absorb copper,” Beckman said. “Copper itself is necessary but can be toxic, so its levels are tightly controlled in the body. The therapy we’re working toward delivers copper selectively into the cells in the spinal cord that actually need it. Otherwise, the compound keeps copper inert.”

    “This is a safe way to deliver a micronutrient like copper exactly where it is needed,” Beckman said.’s-disease