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Emergence of Gene Therapy

By Max Deng

The concept of gene therapy is a simple one on paper. A mutated gene causes a disease and thus a new accurate copy must be inserted to correct the problem. Consider how many genetic diseases there are and how many of them can only be treated, not cured. Gene therapy offers these patients a real chance to lead normal lives.

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In an even broader context, cancer occurs as the result of genetic damage. Gene therapy, then, could be a new and powerful method to combat one of the most challenging medical problems that doctors face today. In practice, however, gene therapy has proven to be much more difficult. The basic outline of the process is that the therapeutic gene is inserted into a vector, or carrier. The vector is usually a type of virus that then infiltrates cells and adds the therapeutic gene into the cell's DNA. Then the cell's genetic machinery treats the new therapeutic DNA the same as regular DNA and transcribes it. This idea of gene therapy was first conceptualized in 1972 [1] with the first clinical drug trial in 1989 [2], but a series of unintended side effects hampered the process. Even the first gene therapy success in 2002 was later linked to cases of leukemia like diseases that occurred as a result of the inserted gene interrupting tumor suppressor genes or by activating proto-oncogenes. As a result, interest in gene therapy began to wane [2].

However, recently, the tide has shifted. Due to improved vectors and techniques, 2,000 clinical trials have been initiated in the last five years and no disastrous side effects have been observed [2]. Despite this newfound success, the FDA has not yet approved any gene therapy treatments. But outside of the US, the story is slightly different. China approved Gendicine, the world's first gene therapy drug aimed at treating head and neck squamous cell carcinoma, in 2004 [3]. China's early approval of gene therapy can be attributed to a lack of discouragement from previous failures that occurred in the western world. At the beginning of November 2012, Europe overcame past difficulties and approved Glybera.

Glybera represents the other major target for gene therapy: inherited genetic diseases. The Amsterdam based company uniQure developed Glybera in order to treat victims of Lipoprotein Lipase Deficiency (LPLD), a relatively rare disease. LPLD is caused by disruptive mutations in the lipoprotein lipase gene, which is critical for breaking down long-chain fatty acids. In LPL patients, lipoprotein lipase cannot cleave the triglyceride bundles in the blood stream filling the blood with undigested fats. This not only leads to diabetes and atherosclerosis, as might be expected, but can also cause chronic and severe pancreatitis. Glybera uses adeno-associated virus (AAV) as a vector which has the advantage of not integrating its own DNA into the host genome, which can cause a whole host of problems, and is overall not pathogenic to humans. As a result, less modification is required and the chance of an immune response is smaller. Due to improvements in technique, uniQure was also able to add regulatory elements and proteins that direct the virus to the needed areas. To ensure that an immune response was not triggered, a course of immunosuppressive drugs followed each injection of Glybera. AAV has proven to be a very effective vector and treatments for hemophilia and retinal degeneration using AAV are already in progress. Furthermore, given the similarity between European and American drug regulation, Glybera shows promise for American development of gene therapy as well [4].

Another possible avenue of gene therapy is treatment of HIV. While at first this seems like a strange concept, as HIV is not classically thought of as a genetic disease, the overall process actually makes a surprising amount of sense. In fact, the first person in the world who was ever cured of HIV, owes his health to gene therapy. In 2007, Timothy Brown, a HIV patient, was diagnosed with acute myeloid leukemia and thus required a bone marrow transplant. Brown's German doctors went the extra mile and chose a compatible donor who had two copies of a mutation in the CCR5 gene. CCR5 is a critical co-receptor for the binding of HIV to a patient's cells and as of 2010, Mr. Brown's system was completely clear of the virus, even without any further treatment [2].

Gene therapy is a very powerful technique with the potential to help countless people in the future. Despite a rough start, improvements in technology and techniques have allowed gene therapy to return to the spotlight and once again begin developing into a viable treatment. By the time the next generation of doctors is trained, gene therapy drugs may even become a key part of medicine.

[1]: Basic Information - Wikipedia
[2]: History of Gene Therapy
[3]: First Approved Gene Therapy
[4]: Impact of Glybera



The Affordable Care Act and its Effects on the Future of Primary Care

By Tiffany McAllister

Primary care over the decade has been changing dramatically. This can be due to the amount and diversity of patients that are now present as well as the growth and development of the medical field. The growth of patients has increased tremendously and our current health care system does not have the money or the resources to accommodate everyone. [1]

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Along with this, technology is also developing at a rapid rate and physicians are constantly struggling to keep up to date with the most current form of treatment, which can inhibit the ability to provide the best type of care for the patient. The government has proposed a resolution to these issues in the form of The Affordable Care Act (ACA). The ACA is currently in place and is bringing hope to the future of primary care. ACA is a policy whose focus is to not merely treat the patient for their disease, but to promote overall health of an individual and the community. This act strongly promotes community health and health living, as well as, primary care services.[1]

One of the main ideas of this act is to allow for and create more teamwork between all contributors in a primary care setting. Many primary care facilities tend to have only physicians and nurses. The ACA will encourage everyone from Registered nurses, physician assistants, Dieticians, front desk staff, behavioral therapists, etc.. to work alongside each other in the primary care office. One example of this is through the regulation of converting from insurance company paperwork to electronic paper work. This ensures that each individual team coordinates as necessary to relay the information onto the electronic form. This would require communication among all staff in order to put the most accurate information into the system. Creating a sense of coordinated care will allow time to create prevention methods as well as admitting more patients so they do not have to be admitted into the emergency room (effective January 1, 2012). [2]

Due to the many advantages offered by the ACA, this new way of primary care is expected to work and gain attention within the medical field. Physicians, which include primary care physicians as well as general surgeons who practice in rural communities, will have more financial security, as they will be provided with a 10% Medicare bonus payment (effective January 1, 2011) [2]. Also, small private practices will be able to group together with other small private practices in order to purchase competitive health insurance for their employees (effective January 1, 2011).

Other benefits include allowing physicians the comfort of relying more on electronic medical records for their patients' verses having insurance companies create and distribute the medical records. This will allow for less waste, but more importantly, it will allow for more secure and confidential patient health information. This reduces costs by adopting uniform billing (effective October 1, 2012). Incentives, such as tax relief and loan repayments for primary care physicians in underserved areas will create incentives to expand the number of primary care physicians. Also, there will be a pay raise for all health care professionals in rural areas.

In addition, the ACA will have an effect on the role of the physician. Physicians would be expected to see patients for a longer time, answering their questions and trying to understand each person's complex situation. They would also have fewer patients to see and follow up care would be a major priority. This would provide more time to respond to phone calls or emails written from patients who may have went to the emergency room if they did not get a response from the primary care physician. The benefits of this would be economical. The savings from patients visiting the emergency room would then go primary care services.

In order to resolve the issues that became present due to the ever-changing field of primary care, the government provided the people with the ACA. The ACA was not only created to promote over health and stability for the people, but it also was enabled to create more teamwork and efficiency between physicians and nurses through the use of communication and coordination amongst all. Incentives, such as Medicare bonus pay and tax relief would allow these actions to become established and gain popularity. The anticipated outcome of the ACA not only benefits physicians and their counterparts, but has the most positive effect on the treatment and care of its patients.


[1]: The Developing Vision of Primary Care

[2]: National Physicians Alliance


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