At its simplest, gene therapy consists of transplanting normal or working genes to replace missing or defective genes in order to correct genetic disorders. The difficulty is getting the working genes into the body and to the place where they are needed without causing unintended side effects. Viruses are currently used. Dr. Lewis gave the analogy of Federal Express: viruses are delivery systems for genes.
Commercially approved gene therapy
In November 2012, for the first time in the Western world, a gene therapy was approved for sale. The European Commission approved alipogene tiparvovec (Glybera®) to treat adult patients with the rare genetic disorder familial lipoprotein lipase deficiency. It is expected to be approved soon in the US. A variety of other gene therapies are in clinical trials. Read More
One of the first examples of personalized medicine is the drug Herceptin
“’Personalized medicine’ refers to the tailoring of medical treatment to the individual characteristics of each patient,” according to the President’s Council of Advisors on Science and Technology report, Priorities for Personalized Medicine. “It does not literally mean the creation of drugs or medical devices that are unique to a patient, but rather the ability to classify individuals into subpopulations that differ in their susceptibility to a particular disease or their response to a specific treatment. Preventive or therapeutic interventions can then be concentrated on those who will benefit, sparing expense and side effects for those who will not.”
We’re moving from the age of one size fits all medicine to personalized medicine based on the genetic profile of individual patients.
Cancer and personalized medicine
Personalized medicine in cancer has made tremendous strides due to the discovery of biomarkers and their corresponding targeted therapies. Biomarkers are defined by the National Cancer Institute as “A biological molecule found in blood, other body fluids, or tissues that is a sign of a normal or abnormal process, or of a condition or disease. A biomarker may be used to see how well the body responds to a treatment for a disease or condition.” Biomarkers for a variety of tumor types, such as breast, colorectal and lung cancer, have been identified. These predictive biomarkers can identify the patient subpopulations that are most likely to respond to a specific therapy.
I was recently among several Chicago CEOs asked to discuss leadership by CEO IntroNet. The invitation resulted from Siren’s selection by Inc. magazine to their Inc. 5000 list of the fastest growing private companies in the US. I was pleased to share my journey from starting a digital agency to diagnosing my daughter with a rare disease (recognizing that marketing rare disorder therapies required a unique approach) to shifting Siren’s focus to orphan drugs.
In this video I talk about Siren’s evolution. I was then interviewed about my leadership style along with another CEO. Part of the discussion focused on the importance of purpose-driven work. I hope you enjoy the clips.
To keep up with what’s new and innovative in technology, as well as the biopharmaceutical industry, we have a learning culture at Siren Interactive. We are constantly sharing articles and ideas, and brainstorming on how we can apply these to our clients’ business. That’s the mindset that led to the creation of InnovationPharm, a series of events featuring industry experts on leading-edge trends.
On May 9, 2012, we invited a few of our clients and our partner, Google, to take part in an InnovationPharm focusing on search and YouTube. Get a sense of the event from video snippets below. The interactive and casual event was held at Cooper’s Hawk Winery—hence the wine barrels in the background!
I talk about keeping up with new technology and highlight Pinterest.
David Blair, head of industry at Google, discusses the power of Google+ and the future potential for biopharma.
The marketing monologue has turned into a conversation
“We don’t believe in digital marketing. We believe in marketing in a digital world, and there’s a huge difference,” said Clive Sirkin, new chief marketing officer of Kimberly-Clark, in an Ad Age article.
I take this to mean that at Kimberly-Clark, digital marketing isn’t in a silo but incorporated as part of the larger marketing team. That interactive tactics are viewed as an integral part of a holistic marketing campaign. Marketing in a digital world still requires offline tactics—they just need to be fully integrated. I hope Sirkin is including social media in his use of the word digital because it’s essential that social media tactics support the overall company or brand marketing objectives.
Most of our target audiences are living in a digital world—one of multitasking, near constant noise, and information available 24/7. Interactive devices, especially smart phones, keep many of us constantly digitally connected. In just a few years, this access has become an essential part of our daily lives. I thought this Domino Theory blog post had an interesting way to think about the topic: “Marketing in a digital world isn’t about the tools YOU use, it’s about the tools your customers use.”
Most of our medical science comes from people with rare diseases
Rare disease research is on the rise, and that’s good news for the 25 million rare disorder patients in the U.S. In fact, it’s good news for all of us, because the insights that we gain from this research can lead to pharmaceutical innovations that extend beyond the rare sphere, affecting the medical industry as a whole. By focusing on a few rare patients, we have the potential to change the lives of many.
Since the Orphan Drug Act was passed in 1983, the number of rare disorder treatments that are available has been steadily increasing every year. In addition to increasing awareness of the rare disease community, this high level of activity has led to pharmaceutical discoveries and advancements in the treatment of other more common diseases.
A Broad Impact on Health Care
There are numerous examples that illustrate the influence rare disease research has on health care. Alpha-1 antitrypsin deficiency (AATD) is a rare lung disorder that affects less than 100,000 people in the U.S. However, as scientists learned more about AATD, it was discovered that the alpha-1 antitrypsin protein could potentially be used to treat Type 1 diabetes, a disease that affects tens of millions of people worldwide. The FDA has since granted regulatory clearance for the protein to be evaluated in clinical trials.
there will be a pharmaceutical brand that will have a compelling use for this type of technology
I’m not embarrassed to admit it: I love Foursquare. It may be a silly game, but it shows how technology – and society – is evolving and how location-based services will be part of our future.
Forrester Research recently found that only 4% of U.S. online adults have ever used location-based mobile apps like Foursquare, Gowalla and Loopt. The majority of users are males age 19-35 and Forrester recommends that most brand marketers wait for a larger audience before engaging. Others endorse jumping in now. I’m not suggesting that pharma brands should start using Foursquare, however it is important to learn about new marketing tactics so we can adapt them for our unique industry.
What is Foursquare?
Foursquare is a location-based social application. Go to Foursquare.com, create a free profile and download the mobile application to your Smartphone. The next time you are out, open the app to use your phone’s geolocator to show places near you and “check-in” at your location. You can also give permission to your friends on Foursquare to allow them see your location and vice versa. Read More
The challenge with innovation is to not get distracted by what’s shiny and new
Innovation. What a loaded word. And a bit intimidating. But according to Merriam-Webster Dictionary all it means is “the introduction of something new.” It’s in my title (Director of Search & Innovation) and for me it’s about paying attention to what’s new and different in pharma, rare disorders and online. It means being open to explore different ideas and adapt them to the unique situation of our clients.
Oooh, it’s a Shiny Object
The challenge with innovation is to not get distracted by what’s shiny and new at the cost of losing sight of the marketing basics. Social media is particularly appealing because everyone is talking about it and it’s “free.” As I’ve written before, social media is a tactic, NOT a strategy. The same goes for mobile and video. It’s important to stay focused on the tools that support the brand strategy. And it’s important to remember that successful strategies build trusted relationships by offering information, help and support the audience truly needs. Read More