Structure-guided design of a peripherally restricted chemogenetic system?

Comment by InpharmD Researcher

Designer receptors exclusively activated by designer drugs (DREADDs) are chemogenetic tools for remotely controlling cellular signaling, neural activity, behavior, and physiology. Using a structure-guided approach, we provide a peripherally restricted Gi-DREADD, hydroxycarboxylic acid receptor DREADD (HCAD), whose native receptor is minimally expressed in the brain, and a chemical actuator that does not cross the blood-brain barrier (BBB). This was accomplished by combined mutagenesis, analoging via an ultra-large make-on-demand library, structural determination of the designed DREADD receptor via cryoelectron microscopy (cryo-EM), and validation of HCAD function. Expression and activation of HCAD in dorsal root ganglion (DRG) neurons inhibit action potential (AP) firing and reduce both acute and tissue-injury-induced inflammatory pain. The HCAD chemogenetic system expands the possibilities for studying numerous peripheral systems with little adverse effects on the central nervous system (CNS).

  

Serological tests were found to have good diagnostic accuracy for coeliac disease. Immunoglobulin A tissue transglutaminase had the highest sensitivity and endomysial antibody the highest specificity. There was little improvement when tests were used in combination. Survey respondents (n = 472) wanted to be 66% certain of the diagnosis from a blood test before starting a gluten-free diet if symptomatic, and 90% certain if asymptomatic. Cost-effectiveness analyses found that, among adults, and using serological testing alone, immunoglobulin A tissue transglutaminase was most cost-effective at a 1% pre-test probability.

Background

The androgens and androgen receptor (AR) play key roles in the prostate cancer (PCa) development and progression via epithelium-stroma cross talk. Prostate cancer-associated fibroblasts (CAFs) are dominant components in PCa stroma and are essential in the malignant progression by supporting tumorigenesis and metastasis. However, the AR roles in CAFs are still obscure. We isolated and immortalized the CAFs from human PCa tissues and found the CAFs are AR positive. We then knocked down their AR with siRNA and co-cultured the resultant CAFs with PCa cell line PC3. The MTT, invasion, and colony formation assays were performed to study the PC3 biological behavior. The results showed that the PCa epithelial growth, invasion, and colony formation abilities decreased when knocking down the CAFs AR. By using the real-time quantitative polymerase chain reaction, we found the IGF1, FGF7, FGF10, SDF1, HGF, and TGFb2 expression levels decreased in the AR knocked down CAFs.

The CAFs and NPFs were successfully cultured, immortalized, and characterized. The CAFs were able to transform prostate epithelial cells into malignant cells, but NPFs were not. The CAFs were more active in promoting proliferation of and invasion by PCa cells, and in secreting higher levels of a series of growth factors. The immortalized CAFs were more supportive of PCa carcinogenesis and progression. Targeting CAFs might be a potential option for PCa therapy. Immortalized CAFs and NPFs will also be valuable resources for future experimental exploration. Focused ultrasound (FUS) has become an important non-invasive therapy for prostate tumor ablation via thermal effects in the clinic. The cavitation effect induced by FUS is applied for histotripsy, support drug delivery, and the induction of blood vessel destruction for cancer therapy. Numerous studies report that cavitation-induced sonoporation could provoke multiple anti-proliferative effects on cancer cells. Therefore, cavitation alone or in combination with thermal treatment is of great interest but research in this field is inadequate.

Human prostate cancer cells (LNCap and PC-3) were exposed to 40 s cavitation using a FUS system, followed by water bath hyperthermia (HT). The clonogenic assay, WST-1 assay, and Transwell® invasion assay, respectively, were used to assess cancer cell clonogenic survival, metabolic activity, and invasion potential. Fluorescence microscopy using propidium iodide (PI) as a probe of cell membrane integrity was used to identify sonoporation. The H2A.X assay and Nicoletti test were conducted in the mechanism investigation to detect DNA double-strand breaks (DSBs) and cell cycle arrest. Immunofluorescence microscopy and flow cytometry were performed to determine the distribution and expression of 5α-reductase.

Short FUS shots with cavitation (FUS-Cav) in combination with HT resulted in, respectively, a 2.2, 2.3, and 2.8-fold decrease (LNCap) and a 2.0, 1.5, and 1.6-fold decrease (PC-3) in the clonogenic survival, cell invasiveness and metabolic activity of prostate cancer cells when compared to HT alone. FUS-Cav immediately induced sonoporation in 61.7% of LNCap cells, and the combination treatment led to a 1.4 (LNCap) and 1.6-fold (PC-3) increase in the number of DSBs compared to HT alone. Meanwhile, the combination therapy resulted in 26.68% of LNCap and 31.70% of PC-3 with cell cycle arrest in the Sub-G1 phase and 35.37% of PC-3 with cell cycle arrest in the G2/M phase. Additionally, the treatment of FUS-Cav combined with HT block the androgen receptor (AR) signal pathway by reducing the relative Type I 5α-reductase (SRD5A1) level to 38.28 ± 3.76% in LNCap cells, and decreasing the relative Type III 5α-reductase 3 (SRD5A3) level to 22.87 ± 4.88% in PC-3 cells, in contrast, the relative SRD5A level in untreated groups was set to 100%.

References:

1. Chinni SR, Li Y, Upadhyay S, Koppolu PK, Sarkar FH. Oncogene. 2001 May 24;20(23):2927-36. doi: 10.1038/sj.onc.1204365.

2. Benitez DA, Pozo-Guisado E, Alvarez-Barrientos A, Fernandez-Salguero PM, Castellón EA. J Androl. 2007 Mar-Apr;28(2):282-93. doi: 10.2164/jandrol.106.000968. Epub 2006 Oct 18.

Relevant Prescribing Information

AGP and SRJ23 selectively inhibited the growth of prostate cancer cells compared with RAW 264.7 cells at low micromolar concentrations; however, SRJ23 was more potent. Mechanistically, SRJ23-treated PC-3 cells displayed down-regulation of cyclin-dependent kinase (CDK) 1 without affecting levels of CDK4 and cyclin D1. However, SRJ23 induced down-regulation of CDK4 and cyclin D1 but without affecting CDK1 in DU145 and LNCaP cell lines. DNA histogram analysis revealed that the SRJ23 induced G2/M in PC-3 cells but G1 arrest in DU-145 and LNCaP cells. Morphologically, both compounds induced predominantly apoptosis, which was further confirmed by DNA fragmentation and annexin V-FITC staining.

Literature Review

A search of the published medical literature revealed 1 study investigating the researchable question:

Structure-guided design of a peripherally restricted chemogenetic system?

Level of evidence

C - Multiple studies with limitations or conflicting results  Read more→



Please see Table 1 for your response.


Comparison Of The Efficacy And Safety Of Direct Oral Anticoagulants And Warfarin After Bioprosthetic Valve Replacements

Design

Retrospective cohort study

N=197

Objective

To evaluate the efficacy and safety of direct oral anticoagulants (DOACs) in patients after a recent bioprosthetic valve replacement 

Study Groups

DOAC (n=127)

Warfarin (n=70)

Inclusion Criteria

≥18 years of age; Received bioprosthetic valve replacement; Received either warfarin or a DOAC following bioprosthetic aortic valve replacement (AVR) or mitral valve replacement (MVR) surgery 

Exclusion Criteria

Mechanical valve replacement; Discontinued anticoagulation before the 6-month period ended; Switched anticoagulation during the 6-month follow-up period

Methods

Investigators retrospectively identified patients who had received a bioprosthetic valve replacement from January 2014 to June 2018 at ChristianaCare (CC) and reviewed each electronic medical record (EMR) for the 6-months post-valve placements to identify incidences of thromboembolic events or major bleeding. Outcomes were evaluated based on readmission to CC. 

The decision to initiate anticoagulation after a bioprosthetic valve replacement was made on a case-by-case basis, considering other risk factors. If anticoagulation was initiated, the agent was chosen by the cardiothoracic surgeon with input from a consulting cardiologist.

The primary efficacy and safety outcomes were analyzed using the Chi-square test. 

Duration

6-months after procedure

Outcome Measures

Primary Efficacy Outcome: Thromboembolic complications within 6 months following surgery, defined as new diagnosis of ischemic stroke, myocardial infarction, or peripheral arterial emboli 

Primary Safety Outcome: Incidence of major bleeding within 6 months following surgery, as defined by the Journal of the American College of Cardiology (JACC)

Baseline Characteristics

 

DOAC (n=127)

Warfarin (n=70)

p-Value

Age, years

71.9

74.5

0.99

Male

56.7%

55.7%

0.90

Chronic kidney disease

10.2%

24.3%

0.01

Anticoagulation prior to surgery

48.8%

67.1%

0.01

Concomitant antiplatelet

99.2%

98.6%

0.67

Results

Endpoint

DOAC (n=127)

Warfarin (n=70)

p-Value

Thromboembolic complications

3 (2.4%)

0

0.20

Major bleeding

9 (7.1%)

2 (2.9%)

0.22

Adverse Events

Common Adverse Events: Not disclosed

Serious Adverse Events: Major bleeding – DOAC (7.1%) versus warfarin (2.9%); p=0.22

Study Author Conclusions

This small, exploratory study found similar rates of thromboembolic complications and major bleeding events in patients receiving a DOAC or warfarin within 6 months of receiving a bioprosthetic MVR or AVR. Larger, prospective trials are needed to evaluate the efficacy and safety of DOACs in this population.

InpharmD Researcher Critique

Data should be interpreted with caution due to the retrospective design and limited sample size. This trial was the only study identified analyzing DOAC use within 6 months of receiving a bioprosthetic valve. Although no significant difference was seen in thromboembolic complications and major bleeding between DOACs and warfarin, the incidence of each was so small that the safety and efficacy of DOACs following bioprosthetic valve placement cannot be truly evaluated.