After reading another article regarding the results of a gene therapy clinical trial, it is abundantly clear that this type of research needs to continue. There is a lot we have to learn and in the case of Layla who was unresponsive to other treatments, we should quickly try to make therapies like these open to everyone as soon as possible. Listen to my podcast to learn about the new therapy that helped a baby girl named Layla.

https://soundcloud.com/user-578021309/audio-recording-on-Friday

References:
http://www.nytimes.com/2015/11/06/business/a-novel-cell-therapy-untested-in-humans-saves-baby-with-cancer.html?_r=0

Chapter 8: Final Thoughts

After spending the past few weeks doing research on genetic engineering and gene therapies it is very clear there is a lot more research to be done to test the efficacy as well as the possibilities for treatment of patients. These therapies would be extremely beneficial and if created correctly, are thought to permanently cure those with certain congenital diseases. There is some debate however, about whether or not the therapies would be considered ethical due to their nature. Some research has shown promising treatments that have used stem cells to help regenerate cells while the more accepting therapies use viruses as vectors. 

I have a very great feeling that this technology will become very prominent in the healthcare field and how we go about treatment. Ever since President Obama’s speech on the Precision Medicine Initiative, a lot of focus has been on how we can individualize treatment to our genomes. As we become more familiar with genes and which sequences are responsible for diseases and disorders, we will better be able to determine which method of therapies and engineering are best. Initially these therapies will be very expensive, but it is abundantly clear that overtime there will be a price decrease, mainly due to the fact that a lot of pharmaceutical companies are hopping on board with clinical trials and research. Individualized healthcare I feel is long overdue and gene therapy can potentially be a great part of this. 

 President Obama's Precision Medicine Initiative speech

References: 

https://www.youtube.com/watch?v=MKiw7yAqqsU

Chapter 7: Gene Therapy may be the Cure for Retinal Dystrophy

An article was published on the New York Times entitled, “Eye Treatment Closes In on Being First Gene Therapy Approved in U.S.” by Andrew Pollack. In the article, the journalist discusses how gene therapy could be an opportunity much tangible for those seeking treatment in a rare type of genetic disease that causes blindness. The research that was described seemed really promising claiming that not only did it stop the progression of the blindness but in some cases had restored vision in some patients. From a public’s perspective, this is extremely exciting news. However, this article appears to be missing some information. Looking at the journalist himself, Andrew Pollack has his Bachelor’s and Master’s degrees in civil and environmental engineering so I feel as though he may have not have been able to address the proper questions he should have in terms of this new biological technology. It appears as though the side effects of this treatment were only noted as being minor, however, in every article that I have read thus far about gene therapies the list of side effects has been extremely long.

 

References:

http://www.brainfacts.org/~/media/Brainfacts/Article%20Multimedia/Diseases%20and%20Disorders/Degenerative%20Disorders/Seeing%20with%20Macular%20Degeneration.ashx

http://www.nytimes.com/2015/10/05/science/eye-treatment-closes-in-on-being-first-gene-therapy-approved-in-us.html?_r=0

http://topics.nytimes.com/top/reference/timestopics/people/p/andrew_pollack/index.html

Chapter 6: Some General FAQs about Gene Therapies

Q: If I want a child with no “bad” genes, how much would that cost to find if the embryo has them or not?

A: Approximately $3,500 in the United States and twice that in Britain. This process includes testing the embryos for genetic defects as well as in vitro fertilization.

 

Q: How likely is it I can choose my child’s sex?

A: Apparently very likely. According to John Hopkins University, research has shown that as of 2006 65% of clinics have this ability. Now that it is 2015, it is likely to be higher.

Q: How are genes delivered in gene therapy?

A: Genes are actually injected into viral vectors which then “infect” you cells and implant the correct gene into your cell’s DNA.

Q: Can these viruses make me sick?

A: Not at all. The viruses used thus far in clinical trials are under very strict guidelines to insure purity and integrity of the DNA that is being transported into your cells.

Q: What is the difference between gene therapy and cell therapy?

A: Gene therapy is the act of injecting a virus into the blood stream which then injects the correct form of the gene into your cells. Cell therapy is the act of injecting actual cells into a particular area of the body that have relevant function.

Q: What kind of diseases can gene therapy treat?

A: Gene therapy is thought to be a great candidate for fixing any genetic defect in our DNA. From Cystic Fibrosis to possible Cancer treatment usage, there are a lot of options for what may come of this technology.

Q: Why would we want to use Stem cells for cell therapy?

A: Stem cells are able to differentiate into any cell in the body and have a lot of regenerative qualities that could be sustained throughout the lifetime of a patient. Stems cells also would eliminate the concern of the immune system rejection, where the immune cells basically attack thinking that the foreign material is bad. With the use of Stem cells, it would already be the cells of the patient and the body would adapt perfectly.



References:
http://image.slidesharecdn.com/genetherapy-131009190236-phpapp02/95/gene-therapy-3-638.jpg?cb=1381345440

http://www.cleveland.com/nation/index.ssf/2009/04/genetic_embryo_screening_or_de.html

http://www.asgct.org/general-public/educational-resources/

Chapter 5: Are Designer Babies Just a Fad?

The idea of Designer Babies is more prominent now than ever. Personally, I find this field extremely interesting and so I did some research from the perspective of a parent that would be in interested in such technology. Just searching the term “Designer Babies” on the internet, the first link that comes up is a link to “The Fertility Institutes”. When you click on the link, the first thing that pops up on the screen is a representative live chat where they can instantly reply to any questions you have. It appears as though the first factor someone that is having a Designer Baby would be interested in is gender, not the possibility of removing a genetic disease.

Screenshot of the web page http://www.fertility-docs.com

Honestly, I found this sign alarming. The term Designer Babies has been sensationalized, the idea of gender selection specifically. On the front page of the website, the institute shares a link to an article about how Kanye West and Kim Kardashian have already taken part in gender selection for their second child who is on the way. There is also a link to a video of a “Good Morning America” clip where Dr. Steinberg, the found of The Fertility Institutes has a segment on the show called “Family Balancing”. The institute has also been recognized on the networks ABC, NBC, CNN, Fox, and Reuters as among the worldwide leaders in gender selection.

Interestingly enough, there is little to no mention of the cost of the technology and nearly no mention of the mutational risks that could happen due to the manual moving of the sex chromosomes by scientists. From my perspective it is disturbing that looking at just this one website page people would be willing to be a part of this technology so blindly and by looking at one simple pamphlet.

References:

http://www.fertility-docs.com/programs-and-services/gender-selection/select-the-gender-of-your-baby-using-pgd.php?gclid=CjwKEAjw-vewBRDH1-b52Lig1hkSJACTPfVFeXPcoR-sCuMcg5PIuUSHR0y1YrMGyOdlxMWaQNzQyhoCb-_w_wcB

Chapter 4: The Possiblities for Gene Therapy in Patients with Haemophilia

A review written by The Cochrane Collaboration entitled, “Gene Therapy for Hemophilia” has some telling information in the pros and cons of what gene therapy has to offer the medical community as well as how a clinical trial should be set up. This review discusses how promising gene therapy is for people who are greatly affected by genetic diseases and how this could be used for things other than just hemophilia in the future. Hemophilia is a X-linked recessive gene where clotting factors are unable to function and excessive bleeding occurs in patients.

Something I found interesting was that the clinical trial that would be done for haemophilia would have a lot of cons. Unfortunately, because the gene therapy treatment is not being mass produced it is extremely expensive. In the case of those affected by haemophilia, the therapy would have to be administered every two to three days because of the short half-live of clotting factors in the blood. The price for treatment was not listed, but it can be assumed that at that rate, it would be extremely hard to keep purchasing injections for therapy. There were some interesting points brought up about the side effects of this therapy such as the fact that some patients’ might not adapt well because the treatment introduces new genes into a cell’s DNA which can cause some insert mutations and could leave the patient worse off perhaps giving them another genetic defect.

In contrast, there are an immense amount of opportunities that gene therapy could offer for those affected by genetic disorders. It is believed that if this therapy is successful many patients would have a lifelong cure and mortality rates would greatly decrease. Not only the patients would benefit but the researchers and medical physicians would as well. By gaining knowledge about how gene therapy is successful could help broaden the types of diseases that are treated.
 

References:

Sharma A, Easow Mathew M, Sriganesh V, Neely JA, Kalipatnapu S. Gene therapy for haemophilia. Cochrane Database of Systematic Reviews 2014, Issue 11. Art. No.: CD010822.
DOI: 10.1002/14651858.CD010822.pub2.

Chapter 3: Biomedical and Pharmaceutical Companies are Hopping Onboard

                After reading the article written by the Boston Globe’s Robert Langreth titled, “Money flows again to gene-therapy drugs investors once shunned”, it is abundantly clear that gene-therapies are in our near future for treatment. Although there are still a lot of research that is being done to make sure that the therapies are safe, there are a lot of biomedical and pharmaceutical companies that are hopping on board in terms of funding.

                This treatment approach has always appeared to have a lot of promising points even after some patients suffered fatal side effects in the 1990’s and the early 2000’s. Thanks for financing that enabled more research to be done in the field there have been some extremely promising clinical trials that show new delivery methods that are much safer for patients. Most recent clinical trial successes have been for patients affected by hemophilia, eye disease, and cancer. The funding that is being done by biomedical and pharmaceutical companies are appearing so promising that there are actual companies that are opening stock specifically for gene therapy technology such as the therapeutic company, Regenxbio. 

 

                From a consumer perspective, with all of these companies that are being involved it really begs the question of how much will this therapy cost and how will insurance companies be a part of it, if at all? The article from the Boston Globe does not touch upon how it will be available to patients and I feel as though that is a huge point that should have been mentioned. 

References: 

 http://www.regenxbio.com/

 https://www.bostonglobe.com/business/2015/05/20/money-flows-aain-gene-therapy-drugs-investors-once-shunned/8SfciTEqF032uDRS4fhpxN/story.html