Development and Implementation of Gene Therapy Treatment

Many of you may be aware that earlier this week, news broke that the FDA approved the second ever gene therapy treatment for adult aged individuals with non-Hodgkin lymphoma. This cancer accounts for about 4% of all cancers in the USA, and the B-cell category of non-Hodgkin lymphoma is a particularly difficult type of cancer to treat.Yescarta

genetically modifies the patient’s own white blood cells, which are injected back into them, as a means of targeting and killing cancer cells. The treatment can be given at certified hospitals and the treatment is still undergoing observational study to ensure the safety and effectiveness of the therapy. This is particularly due to the potentially serious side effects that can result from using the therapy. The FDA’s approval of this gene therapy treatment shows its continued support and enthusiasm towards turning gene therapy from a theory into an effective, accessible treatment for so many people. In fact, the FDA Commissioner Scott Gottlieb was quoted on the FDA website saying, “This approval demonstrates the continued momentum of this promising new area of medicine and we’re committed to supporting and helping expedite the development of these products…We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms.”

This comes shortly after the FDA approved the first every gene therapy treatment plan for use in the USA in August of this year. The first treatment type approved was Kymriah, a treatment approved for patients under 25 years of age with B cell acute lynphoblastic leukemia (ALL). Understanding the need to follow patients closely, especially since gene therapy is such a new treatment method, Novartis, the producer of Kymriah is planing on a long term follow up study to evaluate lasting risks and health outcomes.

Of note, these are not the only gene therapy treatments available globally. In fact, in 2003, China approved a therapy called Gendicine for head and neck squamous cell carcinoma. This treatment causes tumor cells to upregulate their expression of the p53 oncogene. This gene is implicated in tumor and cancer development, and the increased expression of the gene causes the cell to marked at damaged. These cells then undergo apoptosis (cell death) or are attacked by the body’s natural tumor fighting immune responses. Due to the diverse nature of cell types and cancer types, many of these treatments available on the market or under development are only available to a specific subset of the population.

Although gene therapy research and development has evolved enormously over the last 15 years (at least), this article, ‘Gene Therapy for Cancer Treatment: Past, Present and Future’ posted in Clinical Medicine and Research is a great resource that explains the 3 main categories of gene therapy development and treatment methods. The article is a little dated, since it was published in 2006, but is never the less a great resource! As these sources point out, most of these treatments plans are recommended for patients who have failed to respond to the other available treatment options and have no other choices left, and many are recommended to be used in conjunction with existing treatment methods. Looking into the price of these new gene therapy options shows one limiting factor- cost. A single Kymriah treatment costs $475,000! Ideally, as the development and use of gene therapy methods increases, the price will decrease, and we will replace older, less refined treatments with a new standard of care. The battle with cancer has been a long and winding road, but it looks that a major breakthrough in treatment and patient management is in our future!


V-v-v-vaccines: Part 3: Facts and Myths

In the United States, the CDC recommends that all children follow a vaccine schedule, outlined from birth to 18 years of age, to minimize risk to serious infection and reduce the spread of specific diseases. Childhood vaccines are important because they improve the health of our population’s children, reduce their risk of acquired disabilities or death, and prevent outbreaks of illnesses. Additionally, vaccines are an important element of public health. Expectant mothers can protect their unborn child through their routine vaccines and healthcare practices. Vaccinated members of the community help protect those who cannot be vaccinated due to health conditions, and additional vaccines and resources are available to individuals with compromised immune systems. In face, The National Foundation for Infectious Diseases states, “Approximately 50,000 adults die each year from vaccine-preventable diseases in the US.” So, not only do immunizations help the individual stay healthy, they also elevate the health of the whole population.

Despite this information, there remains some controversy in the United States regarding the effectiveness, safety, and necessity of these vaccines. Much of the hesitancy grew from a fabricated and poorly conducted study released by Dr. Wakefield in 1998. He falsely linked the MMR vaccine to the development of autism, which has led to a concern over the safety of vaccines and a subsequent increase in unimmunizated children. It is important to note that numerous studies have failed to confirm any sort of connection between vaccines and developmental delays or behavioral diagnoses. NY Times explains how Dr.Wakefield’s study was retracted from the publishing journal and he was stripped of his medical license upon recognition of the fallacies included in the claims of the study. Furthermore, concerns that spurred from the public as a result of this dramatic event are clearly addressed in the CDC’s website, citing the CDC’s own research as proof against the theory.

The CDC does a great job of addressing these concerns by explaining that both vaccine-induced immunity and natural immunity are both considered active immunity because they utilize the host’s own immune system to develop antibodies and launch a defensive attack. Despite this information, many parents and patients have questions about the other ingredients contained in the vaccine and how vaccines differ from the real-deal illness. There is a wonderful, plain language parent’s guide on the CDC’s website that not only explains the content of a vaccine (dead versus weakened pathogens and additional content), but it also addresses the patient’s risk of contracting the illness and patient rights regarding immunizations. It is important to note that the diseases we vaccinate against are still very much prevalent in our community. In addition to this, the World Health Organization addresses the importance of global vaccines and the impact it has on disease through their website. They even host an annual World Vaccination Week. For patients and their families to understand the importance of their role in this public health endeavor, the WHO is a great resource. It really puts your individual actions into the context of a collective goal. For instance, did you know that “Polio cases have decreased by over 99% since 1988. Today, only 3 countries (Afghanistan, Nigeria and Pakistan) remain polio-endemic, down from more than 125 in 1988.”

For families that choose to vaccinate your children, do not fret about the doctor’s visit! Most children dislike shots (as do most adults!) However, there are numerous resources on the web about how to care for your children after their appointment or make the process less stressful. For instance, tips such as modeling behavior by keeping calm and being truthful about the appointment can calm your child’s nerves and prevent unpleasant surprises. There are even suggestions on comforting ways to hold your child during the appointment to reduce stress or struggling during the shot. If you are a parent and you feel that you need more information, please browse the information on the CDC’s website that is specifically aimed at educating parents. Additionally, do not forget to address your child’s physician for further information or advice. For parents that are considering not vaccinating their children, please understand your responsibility fully before making the decision. Know the symptoms of diseases we vaccinate against as well as the potential outcomes. Be aware if these diseases are in your community, especially during an outbreak, and understand the seriousness of contracting these conditions. Knowing your role as a caregiver means that you understand the risks and responsibilities of your choices.

I feel that it is important to include a personal note here. Many parents I talk to feel like we vaccinate against many mild illnesses that are not common in our community, without understanding the very serious complications of some of these diseases. Additionally, for more serious diseases, parents often feel like those things “only happen to someone else”- like these diseases don’t happen in their community or even in our country. It is important to understand how false these assumptions are. In my personal experience in the emergency room (here in the urban USA), I have seen bacterial meningitis, viral meningitis, chickenpox (vericella), mumps, and whooping cough (pertussis). As much as I want to say that our medical care techniques have advanced enough to provide each child with a positive outcome and full recovery, it’s simply not true. We have lost patients to these illnesses, sent many to the ICU, and had many leave with life altering complications from their diseases. Caring for your children is an important responsibility. Being educated on diseases and vaccines is an important element of parenting that should not be overlooked.

If you would like to see the other posts in this series, please check out Part 1 and Part 2!

<You can find the first to parts of the series on the blog. Here are the links for Part 1 and Part 2.


Gun Violence

Hello everyone! The past few weeks have been quite an adventure in my personal life due to family members moving, medical school interviews, and the unfortunate febrile illness going through the house. It was never my intent to leave readers hanging without a weekly post, but here we are, unfortunately.

Due to the recent mass shooting attack on the eve of October 1st at the Route 91 Harvest Festival in Las Vegas, Nevada, I would like to bring recognition to gun violence and its associated research, or lack thereof. I have already written a post regarding these issues when I was an author with UT Dallas’ Public Health Initiative. My post, with pertinent links and sources, can be found here. Although the article primarily focuses on the single victim incidents that were relevant at the time of my post (made almost a year ago in November 2016), the information is still pertinent to the mass shooting that occurred recently.

Unfortunately, mass shootings have occurred multiple times throughout the US and elsewhere across the globe in recent history. The Las Vegas shooting is the most deadly mass shooting attack in US history. In response to the recent mass shooting, there have been calls to continue to address the issues surrounding firearm safety. The NRA has even spoken out about the need for more regulation on some types of firearm equipment, most notably the bump stock, which can make guns perform more like automatic weapons. The gun control debate is still ongoing in light of recent events.

I would like to extend my thoughts and condolences to individuals, families, and friends affected by gun violence, as well as my support and recognition to all of the service men and women in out health care, public safety, and protection services that have lent their expertise and support to all who have been affected. Gun violence is not the solution to our country’s issues.

Famous Names · Uncategorized

Dr.Luc Montagnier

This month’s notable figure in public health does not stand alone in his crowning achievement- the discovery of HIV as the cause of AIDS. As a result, the other members that contributed to the discovery of the link between HIV and AIDS will also be given a nod.

Luc Montagnier was born in France in 1932 where he grew up amidst World War II and the German occupation of the country of France. He was inspired to pursue virology after learning of the discovery of the Tobacco Mosaic Virus and the subsequent research conducted on the virus. His early research in virology involved hand foot and mouth disease, which proved that viral double-stranded RNA can replicate in a similar fashion to DNA. His research then turned to oncogenic viruses, or viruses that cause cancers. He discovered that the genetic material contained within the virus is enough to have a cancerous effect. His research specifically focused on oncogenic viruses that contained RNA instead of DNA. These viruses became known as retroviruses. Dr. Montagnier’s lab began searching for retroviruses in human cancer patients.

In 1982, Dr. Montagnier became involved in HIV research in France due to the growing suspicion that the disease may be caused by a virus. Multiple virologists around the world were working to determine the composition of the HIV infectious agent. In early 1983, his lab discovered Lymphadenopathy Associated Virus (LAV), named for the virus isolated from patients displaying swollen lymph nodes in early stages of AIDS. The growth of the lymph cells in cultures utilized the technique discovered and developed by Dr. Robert Gallo. His lab’s discovery allowed researchers to grow T-cells and study the viruses that affect them, thus making LAV research possible. Dr. Montagnier, aided by the assistance of Françoise Barré-Sinoussi and Jean-Claude Chermann who both had previous research and experience with retroviruses, grew and examined LAV cells in cultures. These gentlemen recorded the RT activity in the culture and conducted valuable research in the growth and development of the samples of the new virus. In September 1983, in the midst of the AIDS outbreak, Dr.Montagnier made a presentation of his team’s scientific findings and data that linked the newly determined LAV to the disease in a causal manner. LAV would later become known at HIV. At the time this announcement was controversial because the viral data collected by Dr.Montagnier classified the virus in a different family from HTLV, the only known retrovirus of the time. However, closely followed research performed by Dr. Gallo and Dr.Jay Levy confirmed and extended the findings that the HIV virus was not part of the HTLV family. The 2008 Nobel Prize in Physiology or Medicine was awarded to Dr. Montagnier and Dr.Barré-Sinoussi for the discovery of HIV and their pioneering work in the field of virology. (There has been a controversial debate surrounding the members of the award as Dr.Gallo was notably absent from the recognition despite the use of his techniques and previous research in the discovery and testing of HIV)

Today, thanks to the research of Dr. Montagnier and other scientists, we now know that HIV stands for human immunodeficiency virus. The virus’s transmission to humans is linked to a type of chimpanzee in Central Africa. This transmission from animals established a new disease among the human population and eventually began a worldwide outbreak of HIV. The HIV virus attacks a component of the immune system, called T cells. Since it affects the immune system’s function, people with HIV are more likely to get infections and as the disease progresses, uncommon opportunistic infections and cancers can result from the body’s inability to adequately protect itself. AIDS is the most severe stage of HIV. At this level, the number of T cells is below 200 cells per cubic millimeter of blood (normal is 500 to 1,600). Unfortunately, there is no cure for HIV or for AIDS, but there is treatment available to prolong life and slow the progression of HIV. Without treatment, people with AIDS typically survive about 3 years, but people who are treated with antiretroviral therapy (ART) while in the HIV stages of the disease can live almost as long as their healthy counterparts.

For any readers interested in the complex (and slightly messy) discovery of HIV, a great dramatization of the events is displayed in the movies “And the Band Played On.