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This week, we were reminded of the huge strides in cancer treatment that are being made with the help of biotechnology. Two articles in the New York Times over the past few days explain how new treatments that are targeted to the specific genetic profile of a cancer are making a huge difference in patients’ lives.

From one of the articles:

For the melanoma patients who signed on to try a drug known as PLX4032, the clinical trial was a last resort. Their bodies were riddled with tumors, leaving them almost certainly just months to live.

But a few weeks after taking their first dose, nearly all of them began to recover.

Lee Reyes, 30, of Fresno, Calif., who had begun using a feeding tube because of a growth pressing against his throat, bit into a cinnamon roll.

Nothing, he told his mother, had ever tasted as good.

Rita Quigley, who had been grateful just to find herself breathing each morning since learning she had the virulent skin cancer, went shopping for new clothes with her daughters at a mall in Huntsville, Ala.

Randy Williams, 46, who drove 600 miles from his home in Jonesboro, Ark., to the M.D. Anderson Cancer Center in Houston to get the experimental drug, rolled out of bed. “Something’s working,” he thought, “because nothing’s hurting.”

It was a sweet moment, in autumn 2008, for Dr. Keith Flaherty, the University of Pennsylvania oncologist leading the drug’s first clinical trial. A new kind of cancer therapy, it was tailored to a particular genetic mutation that was driving the disease, and after six years of disappointments his faith in the promise of such a “targeted” approach finally seemed borne out. His collaborators at five other major cancer centers, melanoma clinicians who had tested dozens of potential therapies for their patients with no success, were equally elated…

Read the full article here and find Part 2 here.

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  • Hot or Not: Rate the FDA

    Yesterday, our friends at BIOtechNOW posted the following information about participating in a survey about the FDA.  We encourage you to check it out:

    Posted by georgeatbio on February 1st, 2010

    In an ideal world, everyone would have a strong and positive relationship with the FDA. With that as a goal, PricewaterhouseCoopers has launched its fifth survey –Improving America’s Health V Survey – to gauge the working relationship between the life sciences industry and the FDA. Your feedback is important to make this survey a success.

    To participate, please send an email to PricewaterhouseCoopers.and.BIOCOM@us.pwc.com with “Life Sciences FDA Survey” in the subject line. A survey will then be forwarded to you. The survey should take only 15 to 20 minutes to complete. All responses to the survey will be kept confidential and private.

    Those who participate in the survey will receive a preview copy of the results prior to their public release. The survey will close on Feb. 26. Please help foster improved relations between our industry and the FDA.

    We’ve updated our Social Media section with information about BioCrowd!  Check it out here:  http://iambiotech.org/social-media/

    The core doctrine of our biology is this: DNA, the twin helix in our cells, makes RNA, which in turn produces proteins. Because it’s the abnormal production of proteins that causes human disease, drugs are typically produced to block their further production. But today, modern biotechnology offers the opportunity to go well beyond blocking the production of proteins to actually silencing them. This exciting discovery, called RNA Interference, or “RNAi”, won Andrew Fire and Craig Mello the 2006 Nobel Prize for Medicine. RNAi provides new hope for those who suffer from muscular degeneration, Hepatitis C, Huntington’s Disease, HIV, respiratory infections, and various cancers.

    This year, in developing nations, more than 10 million children under the age of twelve will die due to illnesses that are preventable or treatable. Learn about how biotechnology and new health sciences have the potential to promote global health and fight neglected disease in many parts of the developing world.

    by:  David Welch

    I left the office early Friday to catch the first showing at my local theater of “Extraordinary Measures.”  Although Hollywood altered a few names, places and institutions, the essential story is true. It is  about a dad determined to save his two children from the fatal certainty of the dreaded Pompe disease and a brilliant  medical scientist whose theory on how to develop a life-saving enzyme to treat  the disease leads to the two men launching a successful biotech start-up.  As the producer of many true-to-life  short-form films presenting biotech’s amazing potential and the emotional side of the patients who have been saved by biologics, I was natuurally excited about seeing the new flick on Day One.  It didn’t let me down.  I can only hope the millions of people with little or no awareness of biotechnology will come away with a greater appreciation for the potential of this healing science and the dedicated scientists who do “extraordinary” things every day. I also speak as a patient who receives a monthly biotech treatment for a crippling auto-immune disease that has allowed me to enjoy a normal and good life.

    But my bigggest hope is that President Obama and the 535 Members of Congress understand one of the movie’s underlying messages:  if  companies are to continue developing drugs to improve and save the lives of millions of people — including children with unusual or “orphan” diseases — their  enormous monetary investment must be protected with strong patent laws and the inclusion of a sufficient period of data exclusivity rights to prevent generic companies from piggybacking on the biotech company’s safety and efficacy data.

    As you might guess, this uplifting movie has an extraordinarily happy ending. First and most important, the lives of a brother and sister are saved and a loving family is preserved. But I am also struck by how intelligent and trained minds are fairly rewarded and how research jobs and careers are maintained. And yes, I am pleased risk-taking investors get a return on their belief and faith against all statistical odds that just maybe this will be a therapy that will work.

    Two thumbs up for the inspiring story of a biotech executive and a biotech scientist.  ”Extraordinary Measures” is worth the watch for Members of Congress and all of us.

    Each year, 300 million people contract malaria, but only a lucky few are given a drug to combat this deadly disease. See how the drug “Artemisinin” has become one of the most effective in treating the disease, yet remain too expensive for the majority of malaria sufferers. Then learn how scientists are working to increase the yield of the plan required to manufacture Artemisinin, saving millions of lives every year.

    The Myth of Quick Cures

    Thank you to our sister site Eleventh Hour Facts for this post:

    I often hear people say things like “we need more government research in X disease”. It’s times like this I want to bury my face in my hands or bang it on a wall a couple of times, because statements like this display a certain naivete which – while cute – isn’t particularly helpful in getting the job done. It’s just not how things work. Of course the government can have a significant role to play in advancing disease-specific research, but the greatest leaps forward are usually through a public-private partnership. So, I thought… why not provide a relatively short summary of just what goes into researching a cure… especially with the movie Extraordinary Measures coming out in only a few weeks? After all, this film deals with a desperate dad trying to save his children’s lives, and is not an uncommon story within the biotechnology community at all.

    Step one – Fund research: This is likely the step that most people are thinking of when they say “we need more government research”. The National Institutes of Health’s (NIH) 50,000 competitive grants support more than 325,000 researchers at over 3,000 universities medical schools and research institutions across the United States. Without progressive increases in NIH funding, the number of researchers will inevitably decrease as the funding dries up. But that is only the first step.

    Step two – Protect patents: Each biologic drug (for example) takes approximately $1.2 billion and 12-15 years in development before it reaches the approval stage. Without strong patent protection, there is little incentive for private research institutions to spur innovation and breakthrough treatments.

    Step three – Invest in start-ups: Of all the FDA-approved biotech therapies on the market through 2005, about one-third were from companies receiving SBIR funding (aka: small businesses or start-ups). Counterproductive tax policies and limiting SBIR funding to research start-ups like these can literally rip the rug out from under promising therapies.

    Step four – Strengthen the FDA: The FDA regulates products worth nearly $1.5 trillion annually, but total FDA appropriations don’t even cover operating costs. By fully funding the FDA, the agency will be able to manage (in a timely fashion) the growing volume of breakthrough therapies that have to be fully vetted before going to market… otherwise, it will act as an unnecessary bottleneck in getting desperately needed treatments to people.

    Step five – Fair pricing: More than 400 biotech drugs are in clinical trials for 200+ diseases such as Alzheimer’s, heart disease, diabetes, AIDS and many others. Government-imposed pricing mandates can eviscerate the necessary early-stage investment, leaving desperate patients with nowhere to turn.

    Step six – Assure safety: Patients must be helped to understand that there are risks as well as benefits to any given treatment. No treatment is 100% effective, and unrealistic expectations of a treatment’s safety will lead to lawsuits and limit access to needed medications. However, enhancing safety through high-tech post-market surveillance can enhance safety dramatically. It’s really all about having a common sense approach to medicine and accepting a certain level of personal responsibility when undergoing treatment.

    All of these are crucial components to finding effective treatments, so I look forward to seeing how this will be portrayed in Extraordinary Measures. In short, finding cures is much more complicated than many people realize, and the people involved in it are taking heroic risks with their careers and sanity. A simplistic demand for “more government research” into a disease doesn’t accomplish much. Providing a competitive environment for researchers to take the necessary risks does.

    What do you think? Is there anything missing from this list?