HW 6 - Clinical Trials


Brian Mere
BMED 213-70
HW # 6

1. Go to [clinicaltrials.gov](http://clinicaltrials.gov) and search for either gene therapy or protein therapy trials. From the list of ongoing clinical trials, select a disease of interest that is amenable to one of these two biotherapies and describe the mechanism of the disease.  This should include a physiological description of the disease that will be linked to the therapeutic approach of the study.  You might need to find other sources of information to answer this question.  If so, please cite the sources that you use.

Cystic Fibrosis (CF) is a disease that causes "severe damage to the lungs, digestive system and other organs in the body" [1]. It effectively is caused, similar to DMD, by a deficient gene that doesn't allow generated mucus and liquids supposed to be sticky in the body to be not so sticky. This is especially and issue because the inner linings of the lungs require a stick liquid material in order to catch oxygen better.

  1. Describe the therapeutic approach of the study.  Is it gene or protein therapy?  What is the target of the therapy?

The study in question looks at the effective use of gene treatments [2]. The idea is that they want to look at the cystic fibrosis gene pGT-1 to see if they can inject the gene into specifically the nose linings so they can see if it can affect things like mucus, which would be a more common indicator of success of injection of the gene, since mucus samples can be obtained to see if the mucus is stickier or not. It's also not a super invasive process, as it only affects the very accessible nose, and not something more critical like the lungs.

  1. Describe how the study will determine if the gene or protein therapy was effective. For example, what measurements are made for each subject?  Is there a control group that does not receive the therapy?

While no mention of how the study will measure the effectiveness is mentioned, I hypothesize that they would look at things like mucus generated after the injection of these "cationic loposomes" to see if the mucus is more 'sticky', as the disease mainly causes this in relation to the nasal region [1]. It only looks like 9 people were part of the study, so it's highly unlikely that there was a control group for this study [1].

  1. When did the study begin and when will it end?  What is the current status of the study?

The study was first posted on October 18, 1999. It officially ended in May of 2001, with a small update on March 29th, 2011. The study has the status of "completed" [1].

References

[1] “Phase I Pilot Study of Gene Therapy for Cystic Fibrosis Using Cationic Liposome Mediated Gene Transfer,” ClinicalTrials.gov, https://clinicaltrials.gov/study/NCT00004471?cond=Cystic+Fibrosis&intr=gene+therapy&rank=1 (accessed May 20, 2024).

[2] “Cystic fibrosis,” Mayo Clinic, https://www.mayoclinic.org/diseases-conditions/cystic-fibrosis/symptoms-causes/syc-20353700 (accessed May 20, 2024).