Gene-Altering Leukemia Treatment

An article in the New York Times discusses the recent recommendation on approval of a gene-altering Leukemia treatment.  The recommendation came from the FDA panel and it would allow allow the genetic alteration of a patients cells to fight their own cancer.  If the FDA accepts the recommendation, it will be the first gene therapy to reach the market in the United States.  Novartis is the drug company that will likely be first; they target the treatment of Leukemia but are currently working on other types of cancer.  Emily Whitehead was the first child to revive the treatment, as a clinical trial, and it saved her life; Whitehead grew very ill and came close to death but she emerged cancer free.  This is one of the main reasons there has been hesitation with this therapy.  In some cases the patients suffer from life-threatning illness; however, this treatment can be an alternative to the toxic treatments like chemotherapy.  There are still questions about the short-term side affects as well as the chances these cells can cause secondary cancer later in life.  In the current studies the cancer has not reappeared but the study will continue continue until fifteen years from treatment.  This could be a huge breakthrough on curing cancer, this is so important because cancer is hard to fight and the current toxic treatments further break down the immune system making it hared to patients to stay healthy.  This is a huge step in the right direction and will most likely be approved by the FDA.

Match Made In Medicine

When you find yourself up in the middle of the nights studying with a headache most likely your first instinct is to go to the bathroom medicine cabinet in hopes that Tylenol will help ease the pain. Although painkiller such as Tylenol has proven to work many factors such as age, weight and kidney function determine just how well it works. One’s genes also plays a factor in how well painkillers works. Codeine is a commonly used painkiller and once it enters the body enzymes break down the chemical into morphine. This allows the pain signal sent through the central nervous system to become numb. Codeine can cause one to become depended on the drug and can cause withdrawal if the one stops taking the drug. This can be prevented by reducing the dosage that is taken over a course of months so that the withdrawal symptoms are not server. However, an individual can have a gene variation in which they have low enzymes levels that do not allow them to break down enough to ease their pain. Then, there are other individuals that have high enzyme levels that produce too much morphine which can cause one to overdose.

There has been studies that suggest that matching an individual’s genetic makeup with medication can eliminate the side effects and risk of medication that is made to fit the general public. This would be a more efficient method to treat pain both in the comfort in one’s home or in a hospital as the medication is administered by nurses. Patients will receive better care that is tailored to their individual health needs. However, this methods can raise a couple of questions and concerns for both patients and professional in the medical field. For instance, there may be an issue with the cost of having each patient’s genetic makeup mapped out. Then, it becomes an issues of whether the physician or patient is responsible for taking care of the fee to get the gene map. Another cost to consider is the price of developing medication to fit each gene map. 

https://www.ncbi.nlm.nih.gov/pubmedhealth/PMHT0009704/?report=details 

https://www.scientificamerican.com/video/how-gene-testing-could-help-you-find-personalized-painkillers/

Predicting Drugs Side Effects Based on Genetic Makeup

As Prof. Bernhard Palsson states in the article, to sum up his work thus far, "this study is a step forward in demonstrating that patients could be precisely treated based on their genetic makeup". What him and his colleagues aim to accomplish through this model is to be able to predict how individuals will react to the same drug based on their genetic makeup, Palsson specifically did this using red blood cells.

Palsson based this model on the idea that people have different variations of the same gene, which as a result would affect how they metabolize a certain drug.  The model was designed to collect data about the genetic type of the person as well as their specific metabolism; and as a result to help predict how an individual's body will react to a drug.

This particular study involved the kinetic model of red blood cells, as defined in the article, a genetic model is "one that stimulates the chemical reaction of a system and takes into account rates and changing levels of products".

Using this model researchers were able to identify people at risk for anemia induced by ribavirin, and through the model identify the regions in the red blood cells that cause this side effect.

Taking this study the researchers want to build models for larger groups in order to test their ideas further, extending the models into platelet cells and eventually cells of the liver, which would be ideal as this is where most drugs are metabolized.

If this model were to succeed and they research eventually reaches the point where they are able to use a model of liver cells, I think this would be a huge step forward in science / medical field especially when creating new drugs, and in the process of clinical trails.  As a result, researchers, doctors, etc., would be able to better treat each individual based on their own genetics, resulting in a better outcome for the patient.  Eliminating unwanted side effects of a drugs just by knowing how ones body would react to a specific drugs, even when it works for some people, would be amazing, not just for doctors / scientists and people in the field, but also for the people and communities as a whole.

Gene Signatures of Yeast May Help Predict Reactions to Medications

This short article discusses research conducted by the University of British Columbia that explored effects of different drugs on many different strains of yeast. It is not a little known fact that any given drug could affect different people in different ways. Anything from blood pressure medication to an anti-depressant to an illicit drug can be experienced differently and cause a plethora of different side effects. In fact, it's not unusual for someone who is looking for relief from some kind of ailment to have to try several different drugs before they find one that allows them to get the desired effects they need without too many negative side effects. This is especially true of psychiatric drugs- for example, an anti-depressant that helps one person feel better, focus, and have more energy may make another person foggy, more miserable, and unable to get out of bed. This experiment involved taking 6000 different strains of yeast and testing their reactions to 5000 different drugs. The results showed that there were around 50 different ways that each strain might react to a different drug. Each of these reactions represents a different gene signature. Because 50 is a relatively small number compared to 6000 different yeast strains, associate professor Guri Giaever believes that this research not only indicates that we will one day be able to use someone's gene signature to predict how they will react to different drugs, eliminating the need to bounce between medicines before finding one that works for them, but also may provide the first steps to mapping possible reactions. The information gathered in this experiment may also prove useful to future cancer research.

(Secondary article: http://www.nature.com/nrd/journal/v1/n1/full/nrd705.html )