It wasn’t long ago that gene therapy, an idea that has been around since at least the early 1970s, was being derided as more hype than hope. Gene therapy basically involves identifying that part of a patient’s genetic makeup that is causing a certain problem, and then going in and replacing that part with something better. It’s a little like a factory that is found to be producing faulty products; find the machine that is the source of the problem and fix it. The hope is that gene therapy could someday represent a one-time treatment solution for many diseases, utilizing the body’s own ability to correct problems, versus a patient having to take drugs for the rest of their lives.
When talking about the complex world of human genes, of course, the process is not that simple, and, as with any new concept, the first steps in the application of gene therapy were unsteady, its potential risks and rewards not at all clear. Initial application experiments in the 1990s met with a mixture of success and failure, leaving a lot of questions unanswered. Then, in 1999, an attempted application of gene therapy resulted in the death of a patient in Pennsylvania due to an unexpectedly vigorous immune response following an injection with an adenoviral vector carrying a corrected gene. The FDA slammed on the brakes, suspending some clinical trials and reevaluating the entire notion of gene therapy.
Nevertheless, gene therapy researchers around the U.S. and the world continued to make progress into the new century, learning how to move genes across the blood-brain barrier, and gradually finding success treating a growing list of diseases, including some cancers. Today, gene therapy is once again seen as a rising prospect, with potential for treating a wide range of conditions. But it remains a new and complex science, involving an imposing array of variables. Various factors tend to make the benefits of gene therapy short-lived, requiring future treatments. And balancing the efficacy of introduced genes with the sometimes unpredictable nature of the human immune system is still a challenge.
But it’s the range of potential applications that make gene therapy compelling, from cancer and other high-profile diseases to the rarest of afflictions, applications being investigated by a range of public and private companies. Take for example the sampling below:
• Cardium Therapeutics (NYSE MKT: CXM) (San Diego, CA) is a health sciences and regenerative medicine company. Cardium’s current medical opportunities portfolio includes the Tissue Repair Company, Cardium Biologics, and the company’s in-house MedPodium Health Sciences healthy lifestyle product platform. The company’s lead commercial product, Excellagen topical gel for wound care management, recently received FDA clearance for marketing and sale in the United States. Cardium is developing Generx, a gene therapy treatment to boost the growth of blood vessels in the hearts of patients suffering from coronary artery disease.
• bluebird bio (Cambridge, MA) is a leader in the development of innovative gene therapies for severe genetic disorders. At the heart of bluebird bio’s product creation efforts is its broadly applicable gene therapy platform for the development of novel treatments for diseases with few or no clinical options. The company’s novel approach uses stem cells harvested from the patient’s bone marrow, into which a healthy version of the disease causing gene is inserted. After being grown in culture, the cells are given back to the patient. The company’s approach represents a shift in the treatment of severe genetic diseases by eliminating the potential complications associated with donor cell transplantation and potentially presenting a one-time transformative therapy.
• Ceregene, Inc. (San Diego, CA) is a private biotechnology company focused on the treatment of major neurodegenerative disorders using the delivery of nervous system growth factors. Clinical programs include CERE-110, an AAV2 based vector expressing nerve growth factor, which is now in Phase 2 studies for the treatment of Alzheimer’s disease, and CERE-120, which completed a Phase 2 clinical trial in Parkinson’s disease and is currently being tested in a new Phase 1/2 study. CERE-135 and CERE-140 are in preclinical development for amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and ocular diseases, respectively.
Hearing the news from Europe, Christopher J. Reinhard, CEO of Cardium Therapeutics, stated: “Today’s news represents an important step forward for our field and the millions of patients who will benefit from new and innovative gene-based therapeutics. Gene therapy offers the opportunity to simplify treatment for serious medical problems for which there are no current medical treatments.”
Currently, in the U.S. and Europe, gene therapy remains largely locked inside research labs, with governments exceptionally guarded in their willingness to release the developing science to the greater public. China, on the other hand, has been much more willing to sanction the use of gene therapy. There’s good reason for this. Much of China’s massive population has very limited access to regularly administered advanced technology options more common in already industrialized societies such as the U.S. and Europe. In China, the costs and logistics involved in giving cancer patients, for example, long stretches of weekly or daily chemo or radiation treatments makes the one-time nature of gene therapy more appealing, although additional treatments may be needed in the future. Moreover, the Chinese have enough people to easily populate clinical trials, offering up statistically significant results more quickly.
That’s why the recent announcement that Europe has just taken a major step toward approving, for the first time, a specific gene therapy medicine, Glybera, is landmark news. The European Medicines Agency has recommended the therapy for a genetic disease which leaves people unable to properly digest fats. Although the disease addressed, lipoprotein lipase deficiency, is a rare one, the decision in Europe represents the opening of a potentially huge door. It’s the first time a gene therapy medicine has been recommended for authorization in the European Union. The recommendation will now be sent to the European Commission for the adoption of a marketing authorization. It’s the European Commission that will be responsible for making the final decision on Glybera.
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