When
searching for what types of treatments are available for patients with
rheumatoid arthritis, I came across an article about gene therapy. In
regards to the article that I discussed last class, anti-IL-1 a/B and
anti-TNF-a drugs are known to ameliorate the inflammatory process of RA.
However one of the drawbacks of taking those medications is that individuals
need to take them continuously. So since there are always advancements in
technology, it would only make sense to gene therapy could be a potential
treatment, right?
Well
according to the authors of this article, they felt that this approach would
provide a more lasting effect in patients with RA via transplantation of
genetically modified cells directly into the joint that is inflamed. It is
reported that there have already been studies involving injections of IL-1Ra
(an IL-1 inhibitor) into rabbits, mice, and rats showing that there is
decreased toxicity, which lead to a phase I clinical trial where cells
transfected with IL-Ra retrovirus were administered. These cells were
rheumatoid arthritis synovial fibroblasts (RASF), which are cells that playing
in a role in the initiation and perpetuation of the disease. It is proposed
that once these cells are administered to the affected site, there would be a long-term
safety outcome as the transfected cells would proliferate and inhibit
inflammation.
But
the ultimate question is, is this safe? The authors state that there are some pros
and cons to this approach. In the phase 1 clinical trial, they state that the
transduced cells were found only in the surface of the synovium (soft tissue
around the joint capsule and joint cavity) in the metacarpal phalangeal joints
of patients one week after administration of the cells. This is ideal because
you would not want cells transducing their effects elsewhere in the body, which
is obviously undesirable. Also in the study, there was a 5-year waiting period
before reporting results of the study, which is important to help assess the
efficacy of treatment long term. However, it was found in a patient receiving a
high dosage of the cells led to migration of these cells in and adjacent
control joint rather than localization to the affected, targeted area.
Overall,
gene therapy appears to somewhat promising in treating those with rheumatoid
arthritis due to emergence of studies such as transgene expression in those
with the disease. Being that this article was published in 2005, it makes me
wonder what other types of advancements have been done in the last 8 years and
if gene therapy has been effective in treating humans with RA. Is it possible
to have multiple joints treated at the same time? However, I feel in the end
getting such treatments would be quite costly so only a certain demographic of
people would be able to afford it.
Gene therapy is about as close as you can get to the sun with wax wings. Such a dangerous form of treatment after the Jesse Gelsinger case, in which he died four days after the therapy due to an immune response (you can look him up on Wikipedia it describes it a bit more in detail). Obviously there have been advancements between the time of that incident, but even if the upper echelon are the ones who can afford it, would they be willing to take the high risk? Gene mutations could be the cure to RA, but who knows what other problems they can arise. I guess I just think that tampering with genes is a very scary way to think of treatment and should only be used as in knock-out or transgenic experiments in mice/rats.
ReplyDeleteI'm excited to see where we are able to go with gene therapy! However I would also think that this treatment would be more effective in conditions where the DNA is non-functional to produce something needed. Versus using it to change functional DNA and what it makes. For example gene therapy has been shown promising in SCID which is an immune deficiency that lacks functioning T-lymphocytes. Due to a defective gene that allows the activation of T cells.
ReplyDeleteWith the arthritis, as gene therapy may prove useful and effective, but as Jordan said it could come with a greater cost. Especially if it is a therapy where we would want to limit traveling of cells to other parts of the body where it could do more damage.