A Randomized Safety and Efficacy Study of Somavaratan (VRS-317), a Long-Acting rhGH, in Pediatric Growth Hormone Deficiency

Interested by today’s topic of discussion in A&P, I decided to look for an article on the use of growth hormones on pediatric growth hormone deficiency. I found a study by Moore, et al. looked to explore the safety and efficacy of somavaratan in treating pediatric growth hormone deficiency. The study took 68 prepubescent children and randomly assigned them to doses of somavaratan. They were monitored for six months, which included a 30-day dose-finding phase. Somavaratan was found to help reduce the amount of injections of growth hormones the patients needed, up to only one injection per month.

As a pharmacist I think that this study was pretty helpful. Growth hormones can be very important for certain pediatric patients, but daily injections may be something a patient is not willing or able to adhere too. If the addition of somavaratan is continued to be researched and becomes an approved usage, it could do a lot to help patients who suffer from pediatric growth hormone stay adherent and could potentially reduce long term costs for the disorder. What did you think of the study? What do you think of using children in research to begin with?

Link to the article

Moore, Wayne V., Huong Jil Nguyen, Gad B. Kletter, Bradley S. Miller, Douglas Rogers, David Ng, Jerome A. Moore, Eric Humphriss, Jeffrey L. Cleland, and George M. Bright. “A Randomized Safety and Efficacy Study of Somavaratan (VRS-317), a Long-Acting RhGH, in Pediatric Growth Hormone Deficiency.” The Journal of Clinical Endocrinology & Metabolism 101.3 (2016): 1091-097. Web.

BACE1 Physiological Functions May Limit Its Use as Therapeutic Target for Alzheimer’s Disease

Following my last post about potential treatments for Alzheimer’s, I found an article by Barão, et al. about inhibiting another pathway to prevent the disease. A certain cleaving enzyme, known as BACE1, is required for the body to produce the amyloid-beta peptide, which is  necessary for the pathogenesis of Alzheimer’s Disease. The article presents that although inhibition of BACE1 could be used to treat or even cure the disorder, there is still much to be known about how the enzyme, and ones similar to it, work. The article discusses the last three years of discovered information about BACE1.

Although this article was a little heavy and tough to digest, I still found it to be somewhat interesting. It was amazing to see how far research had taken us in the last three years and it makes me hopeful that there may be more and more treatments or preventions for Alzheimer’s as time goes on. As a future pharmacist, preventing a diseases like Alzheimer’s will lead to great health outcomes, not just because the disease can be treated, but because things like loss of memory and motor function will not affect how a patient takes their medications. What do you think about this kind of research? Do you think it matters to a pharmacist?

Link to the article

Barão, Soraia, Diederik Moechars, Stefan F. Lichtenthaler, and Bart De Strooper. “BACE1 Physiological Functions May Limit Its Use as Therapeutic Target for Alzheimer’s Disease.” Trends in Neurosciences39.3 (2016): 158-69. Web.

Pharmacological targeting of CSF1R inhibits microglial proliferation and prevents the progression of Alzheimer’s-like pathology

Olmos-Alonso, Schetters, et al. recently conducted a study to research potential ways to prevent patients from developing Alzheimer’s Disease (AD). Specifically they wanted to see the effect that activation of the colony-stimulating factor 1 receptor (CSF1R) had on progression of the disease, since the CSF1R promotes the creation and activation of microglial cells, which can lead to many neurodegenerative disorders, such as AD. The study provided some early evidence that inhibition of CSF1R could be a potential treatment to prevent the progression of AD.

Although this study only used mice, I think it was interesting to see that they had potentially found a pathway to interrupt in order to prevent, or at least slow, the progression of Alzheimer’s. As some one who has Alzheimer’s in his family history, it at least eases my fears when I see that steps are being made towards treatments for the disorder. I do realize that a lot more work and research needs to be done into this pathway and how exactly a medication could treat it, but I still found this article to be noteworthy. What do you think?

Link to the article

Olmos-Alonso, Adrian, Sjoerd T. T. Schetters, Sarmi Sri, Katharine Askew, Renzo Mancuso, Mariana Vargas-Caballero, Christian Holscher, V. Hugh Perry, and Diego Gomez-Nicola. “Pharmacological Targeting of CSF1R Inhibits Microglial Proliferation and Prevents the Progression of Alzheimer’s-like Pathology.” Brain 139.3 (2016): 891-907. Web.

Pastoral power in the community pharmacy: A Foucauldian analysis of services to promote patient adherence to new medicine use

In a recent article published in Social Science & Medicine, Waring, et al. looked to explore the changing field of pharmacy, something that we’ve discussed a lot in our Profession of Pharmacy classes. Essentially they wanted to look into how pharmacists are evolving from glorified pill counters into hands-on healthcare professionals. The article reported an observed increase in the pharmacist’s monitoring of the patient’s medication experience and an increase in pharmacist’s holding patients accountable for their health outcomes.

Although this study was conducted in England, I think it holds relevance in the United States. The article brought up many things that we have touched on in PoP and I am not at all surprised. As technology changes, it is important for pharmacists to stay hands-on and to provide services that a machine cannot. This also leads to better adherence and better outcomes for patients. But as a wise man once said, “with great power comes great responsibility,” which as why the pharmacist’s role as a healthcare provider grows, so too must their scope of knowledge.

Link to the article

Waring, Justin, Asam Latif, Matthew Boyd, Nick Barber, and Rachel Elliott. “Pastoral Power in the Community Pharmacy: A Foucauldian Analysis of Services to Promote Patient Adherence to New Medicine Use.” Social Science & Medicine 148 (2016): 123-30. Web.

Effects of metformin in adolescents with polycystic ovary syndrome undertaking lifestyle therapy: a pilot randomized double-blind study

In an article published by Ladson, et al. the effects of metformin on adolescents with polycystic ovary syndrome (PCOS) were studied. PCOS is a disorder that causes irregular menstruation, increased levels of testosterone, and very often metabolic issues in females of reproductive age. These metabolic issues can lead to increased rates of obesity and diabetes.

The study was looking to see if metformin is an effective treatment for PCOS. Two groups of adolescent females were gathered and educated about lifestyle interventions they could make in order to help cope with their disorder, such as improving diet and increasing exercise. One group was then given increasing doses of metformin, while the other was given a placebo. At the end of the study, the metformin group showed no major difference from the placebo group other than decreased levels of serum testosterone and an increased number of adverse effects. They recommended that metformin not be used to treat adolescents with the disorder.

I feel that this article is important to pharmacy because it deals with off label usage of a drug. Metformin is well known to be indicated for type 2 diabetes, but it has been shown to be useful for other disorders. I think that it is imperative as pharmacists that we try and research studies like these in order to learn about drugs that may be helpful in treating a disorder it is not originally intended for. Although this article recommended not using metformin on PCOS in adolescents, I have read other articles that have highly suggested that adult females with PCOS be put on metformin therapy. What do you think? Should one study like this stop a drug from being used off label?

Link to the article

Ladson, Gwinnett, William C. Dodson, Stephanie D. Sweet, Anthony E. Archibong, Allen R. Kunselman, Laurence M. Demers, Peter A. Lee, Nancy I. Williams, Ponjola Coney, and Richard S. Legro. “Effects of Metformin in Adolescents with Polycystic Ovary Syndrome Undertaking Lifestyle Therapy: A Pilot Randomized Double-blind Study.” Fertility and Sterility 95.8 (2011): n. pag. Clinical Key. Web. 25 Feb. 2016.

Pharmacist initiation of postexposure doxycycline for Lyme disease prophylaxis

In an article published by Jackson, et al. it is reported that a community pharmacy has begun to successfully develop a program to dispense doxycyline to patients who have come into contact with the tick species known to carry Lyme disease. The pharmacy, which is located in Rhode Island, is independently run and wanted to increase the ability of patients to access this treatment for Lyme disease. Patients were asked to report any symptoms of the disease and to assess the performance of the pharmacy. Overall, the project was deemed a great success and was expanded to more pharmacies. It is also being used as a benchmark for treatment of Lyme disease in areas with high infection rates.

I feel that this article is important to pharmacy for a few reasons. First, an independent pharmacy was able to identify a problem in their community and develop a plan to solve it. Second, they successfully executed their plan and have plans for expansion. Third, they have shown the importance of not only independent pharmacists, but the importance of pharmacists in general. This is a great example of collaborative practice. I’m sure that PA, a state that has a fair amount of Lyme disease diagnoses, could benefit from similar programs. What do you think about this program?

Link to the article

Jackson, Anita N., K. Kelly Orr, Jeffrey P. Bratberg, and Frederic Silverblatt. “Pharmacist Initiation of Postexposure Doxycycline for Lyme Disease Prophylaxis.” Journal of the American Pharmacists Association 54.1 (2014): 69-73. Web.