Gene Therapy:
The Future of Modern Medicine
Although gene therapy has made progress over the year and will continue to do so, there has been debate surrounding the scientific phenomenon.
Innovators
Learn more about the ones who created the gene therapy products that have helped to ease the pain caused by illnesses and genetic disorders.
SPARK THERAPEUTICS
This gene therapy company has had their product, LUXTURNA, officially approved by the FDA. LUXTURNA is a gene therapy product that is designed to treat those suffering from a mutation on their RPE65 gene which eventually leads to blindness. But now this treatment has the potential to halt this disease from progressing.
​
So how this product works is that within LUXTURNA contains a normal RPE65 gene that will go to the retinal cells. In order to have this treatment, patients will undergo surgery. During the surgery, the LUXTURNA which is a liquid, will be inserted into the eye using a microscopic needle. The LUXTURNA contains a vector that will be transporting the functioning version of the RPE65 gene.
​
A total of 41 patients have gone through this treatment between 2007 and 2015 and have been under observation since then.
NOVARTIS PHARMACEUTICALS CORPORATION
This company has been approved by the FDA for their CAR T-cell therapy treatment which is called KYMRIAH. This treatment has been designed to fight against relapsed B-cell acute lymphoblastic leukemia. What this treatment does is that it modifies the patient's T-cells in order for it to target a specific leukemia protein more effectively and then speed up the killing of those cancer cells. After they have been properly modifies they are then returned to the patient via IV where the T-cells can begin killing the cancer cells.
​
​
Dana Farber and Boston's Children are two locations that have been approved for the use of this treatment. They have had eight gene therapy clinical trials where they have been able to treat 25 patients from a total of 11 countries. This is due to the fact that they have one of the most large scale and long-running pediatric gene therapy programs in the world.
SAN RAFFAELE TELETHON INSTITUTE FOR GENE THERAPY
This European institute was formed back in 1995 as a collaboration between the Telethon Foundation and the IRCCS Ospedale San Raffaele to do research involving gene and cell therapy. Then, that research can become effective treatments against genetic diseases.
​
A milestone was hit when their treatment, Strimvelis, was approved in Europe in May of 2016. This is the first ever ex vivo gene therapy to be approved and the road leading up to this wasn't easy. It took a total of 14 years of research and testing to develop. A reported 18 children have been cured of ADA-SCID, which is a severe form of immunodeficiency.
​
​
​
This is how Strimvelis is made:
-
bone marrow is taken from the patient
-
CD34+ cells (they produce lymphocytes) are removed
-
a functional ADA gene is placed in the CD34 cells with the use of a vector
-
it is returned to the patient via infusion into a vein
TRANSATLANTIC GENE THERAPY CONSORTIUM
This organization was formed after the initial gene therapy trials treating X-linked Severe Combined Immunodeficiency led to leukemia as being a dangerous side effect. Their goal is to make gene therapy safer, help to divide up the expenses from the research, and to conduct trials to treat rare illnesses.
​
Their trial treating X-SCID has begun and is having patients from London, Paris, Boston, Cincinnati and Los Angeles be part of it. This trial has the potential to become the ideal trial to treat rare illnesses that future researchers have to meet the standards of. Not just that, this forces those researching gene therapy to address the problems that lies within this field. Also, another goal they are trying to achieve is to improve the safety of the vectors.
(Boston Children's Hospital is one of the founding members of the organization.)
AMSTERDAM MOLECULAR THERAPEUTICS
They developed the first approved gene therapy product, Glybera. It was approved by the European Commission back in July of 2012. It was designed to treat Lipoprotein Lipase Deficiency (LPLD) which is a genetic disorder that causes the sufferer to not be able to break down fat due to a lack of a certain protein. This disorder leads to a build up of fat in the blood.
​
This is how the drug works:
-
it has alipogene tiparvovec
-
disbands the chylomicron
(made of protein and fat) particles
that are in the blood
-
it transports a functional LPL
gene that restores the issue
caused by the disorder
-
it brings the excessive amount
of fat in the blood to a normal amount
​
Glybera is delivered to the body by
injection into the leg muscle.
​
There were multiple clinical trials that were conducted until this treatment was able to be approved. In the Phase II/III trial which was conducted between February 2009 and April 2011, there was 5 patients who had LPLD. The results reported was less triglyceride (fat in the blood). In the next Phase III trial, there was again 5 patients with the same disorder that were tested. In March of 2012, the results were that after only having one dose of the treatment there was a significantly better chylomicron metabolism. But the approval for this treatment came from a trial that took place in Canada and the Netherlands. This trial had 27 patients. The findings from this one concluded that Glybera was well endured and there were no major flags raised regarding the safety of it. It has been shown that it reduces the amount of triglyceride, and only one dosage of Glybera has resulted in the decrease of the regularity of acute pancreatitis.
​​
​
​
​
​
