• Skip to main content

ADPKD Treatment Report

www.ADPKDTreatmentReport.com

admin

Salt, water, and vasopressin in polycystic kidney disease

October 1, 2020

https://www.kidney-international.org/article/S0085-2538(20)30635-9/fulltext

https://doi.org/10.1016/j.kint.2020.06.001

https://pubmed.ncbi.nlm.nih.gov/32998813/

TITLE:
Salt, water, and vasopressin in polycystic kidney disease

DATE:
Thu, 01 Oct 2020 06:00:00 -0400

AUTHORS:
Vicente E Torres

SOURCE:
Kidney international

DESCRIPTION:
Excessive salt intake and vasopressin may promote cyst growth in autosomal dominant polycystic kidney disease and glomerular filtration rate (GFR) decline in chronic kidney disease. Kramers et al. confirmed the effects of salt in a large autosomal dominant polycystic kidney disease cohort. Copeptin mediated 72% and 25% of the salt effects on GFR decline and kidney growth. Kidney growth did not significantly contribute to the copeptin-mediated salt effect on GFR. Differences in kidney growth and…

CONTENT:
Kidney Int. 2020 Oct;98(4):831-834. doi: 10.1016/j.kint.2020.06.001.

ABSTRACT

Excessive salt intake and vasopressin may promote cyst growth in autosomal dominant polycystic kidney disease and glomerular filtration rate (GFR) decline in chronic kidney disease. Kramers et al. confirmed the effects of salt in a large autosomal dominant polycystic kidney disease cohort. Copeptin mediated 72% and 25% of the salt effects on GFR decline and kidney growth. Kidney growth did not significantly contribute to the copeptin-mediated salt effect on GFR. Differences in kidney growth and GFR decline trajectories and factors other than cyst growth contributing to GFR decline may explain the divergent effects.

PMID:32998813 | DOI:10.1016/j.kint.2020.06.001

PUBMED ID:
pubmed:32998813

OTHER ID:
pmid:32998813,doi:10.1016/j.kint.2020.06.001

PUBLICATION DATE:
Thu, 01 Oct 2020 06:00:00 -0400
2020-10-01

RETRIEVAL DATE :
10/01/20 12:09PM

LINK – PUBMED:
https://pubmed.ncbi.nlm.nih.gov/32998813/?utm_source=Other&utm_medium=rss&utm_campaign=pubmed-2&utm_content=1jmC0p0kwOiHcoERYElvPhktie_Gw8TL2aOwN185ZhQUSROsDP&fc=20200821211545&ff=20201001120951&v=2.12.1

LINK – DOI:
https://doi.org/10.1016/j.kint.2020.06.001

LINK – FULL TEXT:

NOTES:

Palladio Completes $20 Million Series B Financing: Proceeds will support development of lixivaptan, a potential treatment for ADPKD

September 25, 2020

https://www.businesswire.com/news/home/20200925005062/en/Palladio-Completes-20-Million-Series-B-Financing

Palladio Completes $20 Million Series B Financing: Proceeds will support development of lixivaptan, a potential treatment for ADPKD

Palladio Completes $20 Million Series B Financing
— Led by Samsara BioCapital
— Advancing Phase 3 ALERT Study

September 25, 2020 06:09 AM Eastern Daylight Time
HORSHAM, Pa.–(BUSINESS WIRE)–Palladio Biosciences, Inc. (Palladio), a privately-held, clinical stage biopharmaceutical company developing medicines for orphan diseases of the kidney, announced today the completion of a $20 million Series B investment. The financing was led by new investor, Samsara BioCapital, with participation from new investor, the Roche Venture Fund and existing investors, Medicxi and Osage University Partners. Proceeds from the financing will fund Palladio’s Phase 3 ALERT Study and advance operations.

“We are very pleased that Samsara and the Roche Venture Fund recognize the opportunity lixivaptan represents to patients suffering from ADPKD, and we are glad to be working with these new partners to further develop this important therapy”

Tweet this
Srinivas Akkaraju M.D, Ph.D, Managing General Partner of Samsara BioCapital, stated, “We are very excited about our investment in Palladio to support the development of lixivaptan, a drug that has the potential to deliver a meaningful therapeutic advancement in the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD). The ALERT Study is an important step in assessing the safety differential of lixivaptan.”

“We are very pleased that Samsara and the Roche Venture Fund recognize the opportunity lixivaptan represents to patients suffering from ADPKD, and we are glad to be working with these new partners to further develop this important therapy,” said Francesco De Rubertis, co-founder and Partner at Medicxi.

Metformin as a Novel Therapy for Autosomal Dominant Polycystic Kidney Disease

September 15, 2020

https://clinicaltrials.gov/ct2/show/NCT02656017?type=Intr

https://clinicaltrials.gov/ct2/show/NCT02656017?type=Intr&cond=ADPKD&phase=01245&lupd_s=09%2F14%2F2020&lupd_d=1&sort=nwst

STUDY TITLE:
Metformin as a Novel Therapy for Autosomal Dominant Polycystic Kidney Disease

STUDY DESCRIPTION:
Condition :   Polycystic Kidney, Autosomal Dominant

Interventions :   Drug: Metformin;   Other: Placebo

Sponsors :   Kyongtae Ty Bae, M.D., Ph.D.;   Tufts Medical Center;   University of Maryland, Baltimore;   University of Southern California;   United States Department of Defense

Active, not recruiting

CLINICALTRIALS.GOV ID:
NCT02656017

FIRST POSTED:
Thu, 14 Jan 2016 12:00:00 EST

LAST UPDATE POSTED:
09/15/20 06:51AM

STUDY LINK:
https://clinicaltrials.gov/ct2/show/NCT02656017?type=Intr&cond=ADPKD&phase=01245&lupd_s=09%2F14%2F2020&lupd_d=1&sort=nwst

NOTES:

A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients

September 14, 2020

https://clinicaltrials.gov/ct2/show/NCT03523728?type=Intr&cond=ADPKD&phase=01245&lupd_s=09%2F13%2F2020&lupd_d=1&sort=nwst

STUDY TITLE:
A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients

STUDY DESCRIPTION:
Condition :   Polycystic Kidney, Autosomal Dominant

Interventions :   Drug: Venglustat GZ402671;   Drug: Placebo

Sponsor :   Genzyme, a Sanofi Company

Recruiting

CLINICALTRIALS.GOV ID:
NCT03523728

FIRST POSTED:
Mon, 14 May 2018 12:00:00 EDT

LAST UPDATE POSTED:
09/14/20 07:12AM

STUDY LINK:
https://clinicaltrials.gov/ct2/show/NCT03523728?type=Intr&cond=ADPKD&phase=01245&lupd_s=09%2F13%2F2020&lupd_d=1&sort=nwst

NOTES:

Reproducing the pathophysiology of polycystic kidney disease from human iPS cells: Understanding the pathogenesis of ADPKD and developing new treatments

August 21, 2020

https://www.sciencedaily.com/releases/2020/08/200821094825.htm

Reproducing the pathophysiology of polycystic kidney disease from human iPS cells
Understanding the pathogenesis of hereditary polycystic kidney disease and developing new treatments
Date:
August 21, 2020
Source:
Kumamoto University
Summary:
A research project has successfully reproduced the pathogenesis of autosomal dominant polycystic kidney disease (ADPKD) from human iPS cells in vitro. Although cysts derived from renal tubules have been previously documented, this is the first derivation of cysts from collecting ducts, which is more closely related to the pathogenesis of the disease. This research is expected to lead to a better understanding of disease states and the development of new treatment methods.
Share:

FULL STORY
Joint research, led by Kumamoto University in Japan, has successfully reproduced the pathogenesis of autosomal dominant polycystic kidney disease (ADPKD) from human iPS cells in vitro. ADPKD is a disease that causes multiple cysts in both kidneys and is the most common hereditary kidney disease. Although cysts derived from renal tubules have been previously documented, this is the first induction of cysts from collecting ducts, which is more closely related to the pathogenesis of the disease. The researchers expect that this will lead to a better understanding of disease states and the development of new treatment methods.

Genetics May Predict Effectiveness of Tolvaptan in Autosomal Dominant Polycystic Kidney Disease

August 12, 2020

https://www.karger.com/Article/FullText/509817

https://pubmed.ncbi.nlm.nih.gov/32784291/

Genetics May Predict Effectiveness of Tolvaptan in Autosomal Dominant Polycystic Kidney Disease

Am J Nephrol
2020 Aug 12;1-7. doi: 10.1159/000509817. Online ahead of print.

PMID: 32784291 DOI: 10.1159/000509817

Abstract
Background: Tolvaptan is the only therapeutic drug for autosomal dominant polycystic kidney disease (ADPKD). The influence of mutations in polycystic kidney disease 1 and 2 genes (PKD1 and PKD2) on the treatment effects of tolvaptan is not well documented in the literature.

Methods: We retrospectively evaluated the relationship between genotype and the efficacy of tolvaptan in 18 patients with ADPKD who had been treated at Toranomon Hospital and undergone genetic testing between April 2016 and February 2020.

Results: The annual change in estimated glomerular filtration rate (ΔeGFR/y) from before to after tolvaptan was from a median of -5.5 to -2.5 mL/min/1.73 m2 in the PKD1 truncating group, -3.3 to -2.4 mL/min/1.73 m2 in the PKD1 non-truncating group, -3.1 to -1.6 mL/min/1.73 m2 in the PKD2 group, and -1.9 to -2.6 mL/min/1.73 m2 in the group with no PKD1/2 mutation. The median degrees of improvement of ΔeGFR/y were 2.5 (45%), 0.4 (10%), 0.6 (28%), and -0.7 (-37%) mL/min/1.73 m2, respectively. Compared with the group of patients with any PKD1/2 mutation, the group with no PKD1/2 mutation showed significantly less improvement in ΔeGFR/y with tolvaptan (0.6 vs. -0.7 mL/min/1.73 m2, respectively; p = 0.01) and significantly less improvement in the annual rate of increase in total kidney volume (TKV) with tolvaptan (-6.7 vs. -1.1%, respectively; p = 0.02).

Conclusion: Patients with ADPKD and no PKD1/2 mutation showed less improvement in ΔeGFR/y and the annual rate of increase in TKV with tolvaptan. Detecting PKD1/2 mutations may be useful for predicting the effectiveness of tolvaptan.

Keywords: Autosomal dominant polycystic kidney disease; Genotype; Polycystic kidney disease 1; Polycystic kidney disease 2; Tolvaptan.

A Trial of Bardoxolone Methyl in Patients With ADPKD – FALCON (FALCON)

August 6, 2020

https://clinicaltrials.gov/ct2/show/NCT03918447

A Trial of Bardoxolone Methyl in Patients With ADPKD – FALCON (FALCON)

Study Type : Interventional (Clinical Trial)
Estimated Enrollment : 300 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 3 Trial of Bardoxolone Methyl in Patients With Autosomal Dominant Polycystic Kidney Disease
Actual Study Start Date : May 29, 2019
Estimated Primary Completion Date : March 2023
Estimated Study Completion Date : March 2023

A novel ADPKD model using kidney organoids derived from disease-specific human iPSCs

August 4, 2020

https://www.sciencedirect.com/science/article/abs/pii/S0006291X20313590?via%3Dihub

https://doi.org/10.1016/j.bbrc.2020.06.141

https://pubmed.ncbi.nlm.nih.gov/32819584/

TITLE:
A novel ADPKD model using kidney organoids derived from disease-specific human iPSCs

DATE:
Fri, 21 Aug 2020 06:00:00 -0400

AUTHORS:
Tatsuya Shimizu,Shin-Ichi Mae,Toshikazu Araoka,Keisuke Okita,Akitsu Hotta,Kunihiro Yamagata,Kenji Osafune

SOURCE:
Biochemical and biophysical research communications

DESCRIPTION:
Autosomal dominant polycystic kidney disease (ADPKD) is a hereditary disorder which manifests progressive renal cyst formation and leads to end-stage kidney disease. Around 85% of cases are caused by PKD1 heterozygous mutations, exhibiting relatively poorer renal outcomes than those with mutations in other causative gene PKD2. Although many disease models have been proposed for ADPKD, the pre-symptomatic pathology of the human disease remains unknown. To unveil the mechanisms of early…

CONTENT:
Biochem Biophys Res Commun. 2020 Sep 3;529(4):1186-1194. doi: 10.1016/j.bbrc.2020.06.141. Epub 2020 Aug 4.

ABSTRACT

Autosomal dominant polycystic kidney disease (ADPKD) is a hereditary disorder which manifests progressive renal cyst formation and leads to end-stage kidney disease. Around 85% of cases are caused by PKD1 heterozygous mutations, exhibiting relatively poorer renal outcomes than those with mutations in other causative gene PKD2. Although many disease models have been proposed for ADPKD, the pre-symptomatic pathology of the human disease remains unknown. To unveil the mechanisms of early cytogenesis, robust and genetically relevant human models are needed. Here, we report a novel ADPKD model using kidney organoids derived from disease-specific human induced pluripotent stem cells (hiPSCs). Importantly, we found that kidney organoids differentiated from gene-edited heterozygous PKD1-mutant as well as ADPKD patient-derived hiPSCs can reproduce renal cysts. Further, we demonstrated the possibility of ADPKD kidney organoids serving as drug screening platforms. This newly developed model will contribute to identifying novel therapeutic targets, extending the field of ADPKD research.

PMID:32819584 | DOI:10.1016/j.bbrc.2020.06.141

PUBMED ID:
pubmed:32819584

OTHER ID:
pmid:32819584,doi:10.1016/j.bbrc.2020.06.141

PUBLICATION DATE:
Fri, 21 Aug 2020 06:00:00 -0400
2020-08-22

RETRIEVAL DATE :
08/21/20 09:26PM

LINK – PUBMED:
https://pubmed.ncbi.nlm.nih.gov/32819584/?utm_source=Other&utm_medium=rss&utm_campaign=pubmed-2&utm_content=1jmC0p0kwOiHcoERYElvPhktie_Gw8TL2aOwN185ZhQUSROsDP&fc=20200821211545&ff=20200821211655&v=2.11.5

LINK – DOI:
https://doi.org/10.1016/j.bbrc.2020.06.141

LINK – FULL TEXT:
https://www.sciencedirect.com/science/article/abs/pii/S0006291X20313590?via%3Dihub

NOTES:

An affordable and commonly used diabetes drug is being touted as a potential treatment for ADPKD

July 30, 2020

https://www.uq.edu.au/news/article/2020/07/diabetes-drug-could-prevent-kidney-failure

An affordable and commonly used diabetes drug is being touted as a potential treatment, for a rare form of kidney disease

Diabetes drug could prevent kidney failure

30 July 2020
An affordable diabetes drug could save generations of Australian families who live with polycystic kidney disease from kidney failure.

FDA has granted Regulus Therapeutics orphan drug designation for RGLS4326, an investigational therapy being studied to treat ADPKD

July 29, 2020

https://www.ajmc.com/view/fda-grants-orphan-drug-status-to-regulus-therapeutics-treatment-for-common-type-of-pkd

DA Grants Orphan Drug Status to Regulus Therapeutics’ Treatment for Common Type of PKD
July 29, 2020
Mary Caffrey

Although the name suggests the disease affects only the kidneys, development of fluid-filled cysts can spread to the liver, the pancreas, and other organs. While a healthy kidney is about the size of a fist, a kidney filled with cysts from polycystic kidney disease (PKD) can grow to be about the size of a football weigh up to 30 pounds

Regulus Therapeutics announced today FDA has granted the company orphan drug designation for RGLS4326, an investigational therapy being studied to treat autosomal dominant polycystic kidney disease (ADPKD), the most common type of a group of genetic disorders that lead to end-stage renal disease, morbidity, and early death.

Efficacy and Safety of Lixivaptan in the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

July 29, 2020

https://clinicaltrials.gov/ct2/show/NCT04064346

Efficacy and Safety of Lixivaptan in the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Study Type : Interventional (Clinical Trial)
Estimated Enrollment : 1200 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Double-blind, randomized, placebo-controlled
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description: Sponsor
Primary Purpose: Treatment
Official Title: A 52-Week, Phase 3, Double-blind, Placebo-controlled, Randomized Study of the Efficacy and Safety of Lixivaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Estimated Study Start Date : July 2021
Estimated Primary Completion Date : July 2024
Estimated Study Completion Date : December 2024

Safety, Pharmacokinetics, Tolerability and Efficacy of Tolvaptan in Children and Adolescents With ADPKD (Autosomal Dominant Polycystic Kidney Disease)

May 15, 2020

https://clinicaltrials.gov/ct2/show/NCT02964273

Safety, Pharmacokinetics, Tolerability and Efficacy of Tolvaptan in Children and Adolescents With ADPKD (Autosomal Dominant Polycystic Kidney Disease)

Study Type : Interventional (Clinical Trial)
Actual Enrollment : 91 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 3b, Two-part, Multicenter, One Year Randomized, Double-blind, Placebo-controlled Trial of the Safety, Pharmacokinetics, Tolerability, and Efficacy of Tolvaptan Followed by a Two Year Open-label Extension in Children and Adolescent Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Study Start Date : September 2016
Actual Primary Completion Date : December 2, 2019
Estimated Study Completion Date : December 2021

XORTX Provides Overview and Update of Key Activities – Including Update on XRx-008 for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

April 30, 2020

https://www.globenewswire.com/news-release/2020/04/30/2025227/0/en/XORTX-Provides-Overview-and-Update-of-Key-Activities.html

XORTX Provides Overview and Update of Key Activities

The following summarizes the advancements made in 2019 to date in XORTX’s three programs:

XRx-008 for Autosomal Dominant Polycystic Kidney Disease (ADPKD):

Growing outreach to key polycystic disease physicians and patient advocacy groups to advance the XRx-008 program toward a phase 3 registration trial;

XRX-008 program further validated by evidence of a new mechanism of injury in polycystic kidney disease, this study showed that uric acid and oxalate crystals may play a key role in the initiation of new kidney cysts and accelerating the growth of those cysts as well as in the generation of kidney stones in individuals with ADPKD;

Advanced Orphan Drug Designation (“ODD”) documents for completion of filing with the FDA for XRx-008 for the treatment of progressive kidney disease due to ADPKD;

Introduced Dr. Anjay Rastogi, UCLA to XORTX’s Clinical Advisory Board; and

Initiated and ongoing discussions with pharmaceutical company partners for this program.

Deregulation of Long Noncoding RNA in the Pathogenesis of Autosomal Dominant Polycystic Kidney Disease

April 1, 2020

https://projectreporter.nih.gov/project_info_description.cfm?aid=9930079&icde=51360866

Deregulation of Long Noncoding RNA in the Pathogenesis of Autosomal Dominant Polycystic Kidney Disease

Preventing the formation or slowing the growth of kidney cysts is crucial to the long-term survival and quality of life of patients with polycystic kidney disease (PKD). The proposed research is broadly relevant to public health because it will be the first study to identify a lncRNA (Hoxb3os) that directly binds to and inhibits mTORC1 activity. Results from this study will provide genetic proof-of-concept for future studies that focus on targeting lncRNAs as a treatment option in PKD.

Study of the Efficacy and Safety of Tesevatinib in Subjects With ADPKD

March 16, 2020

https://clinicaltrials.gov/ct2/show/NCT03203642

Study of the Efficacy and Safety of Tesevatinib in Subjects With ADPKD

Study Type : Interventional (Clinical Trial)
Actual Enrollment : 80 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Double-blind Randomized Parallel Group Study of the Efficacy and Safety of Tesevatinib in Subjects With Autosomal Dominant Polycystic Kidney Disease
Actual Study Start Date : August 9, 2017
Estimated Primary Completion Date : January 31, 2022
Estimated Study Completion Date : January 31, 2022

Implications of early diagnosis of autosomal dominant polycystic kidney disease

March 9, 2020

https://www.nature.com/articles/s41598-020-61303-9

Implications of early diagnosis of autosomal dominant polycystic kidney disease: A post hoc analysis of the TEMPO 3:4 trial

A high-throughput screening platform for Polycystic Kidney Disease (PKD) drug repurposing utilizing murine and human ADPKD cells

March 6, 2020

https://www.nature.com/articles/s41598-020-61082-3

A high-throughput screening platform for Polycystic Kidney Disease (PKD) drug repurposing utilizing murine and human ADPKD cells

Pravastatin and Alkali Therapy in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD-SAT)

February 26, 2020

https://clinicaltrials.gov/ct2/show/NCT04284657

Pravastatin and Alkali Therapy in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD-SAT)

Study Type : Interventional (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Pravastatin and Alkali Therapy in Patients With Autosomal Dominant Polycystic Kidney Disease
Actual Study Start Date : January 30, 2019
Estimated Primary Completion Date : December 31, 2020
Estimated Study Completion Date : December 31, 2020

PKD Foundation Receives $9.3 Million Donation – 100 percent of gift is designated to research and finding a cure for PKD

January 20, 2020

Trust of Alfred Busiel donates over 9-million

FOR IMMEDIATE RELEASE:

PKD Foundation Receives $9.3 Million Donation from the Trust of Alfred Busiel, Former Owner of Lady Esther Cosmetics
This Generous Donation is an Important Step in Reaching a $70 Million Campaign Goal over the Next Five Years
KANSAS CITY, Mo. — The PKD Foundation is pleased to announce the generous gift of a donation in the amount of $9.3 million from the trust of Alfred Busiel, former owner of the cosmetic company, Lady Esther.

Based in Kansas City, Mo., the PKD Foundation is the only organization in the United States solely dedicated to funding critical research and finding a cure for polycystic kidney disease (PKD).

Exactly 100 Percent of Busiel’s Donation to Support PKD Research
This significant commitment from Busiel allows the PKD Foundation to support its Future Focus Campaign, a campaign with a five-year goal to raise $70 million to increase the Foundation’s commitment to PKD research by 300 percent.

Exactly 100 percent of Busiel’s gift is designated to research and directly supports the PKD Foundation’s vision to end PKD.

“We are very fortunate to receive this generous donation from Mr. Busiel. This is a critical milestone in helping us achieve our Future Focus Campaign goal. Now, the Foundation only needs to raise approximately $5 million more to reach our total goal,” Andy Betts, President and CEO of the PKD Foundation said. “With generous gifts such as Mr. Busiel’s, we can spend a significant amount of our resources on doing what’s most important – focusing on new research, which is vital to making progressive strides in treatment research and to support finding a cure – our ultimate goal.”

Quest for the Cure: Testing the Old and New to Prevent Progression of Autosomal Dominant Polycystic Kidney Disease

October 31, 2019

https://www.kidneymedicinejournal.org/article/S2590-0595(19)30147-5/fulltext

https://doi.org/10.1016/j.xkme.2019.10.004

https://pubmed.ncbi.nlm.nih.gov/33015606/

TITLE:
Quest for the Cure: Testing the Old and New to Prevent Progression of Autosomal Dominant Polycystic Kidney Disease

DATE:
Mon, 05 Oct 2020 06:00:00 -0400

AUTHORS:
Godela Brosnahan

SOURCE:
Kidney medicine

DESCRIPTION:
No abstract

CONTENT:
Kidney Med. 2019 Oct 31;1(6):329-331. doi: 10.1016/j.xkme.2019.10.004. eCollection 2019 Nov-Dec.

NO ABSTRACT

PMID:33015606 | PMC:PMC7525136 | DOI:10.1016/j.xkme.2019.10.004

PUBMED ID:
pubmed:33015606

OTHER ID:
pmid:33015606,pmc:PMC7525136,doi:10.1016/j.xkme.2019.10.004

PUBLICATION DATE:
Mon, 05 Oct 2020 06:00:00 -0400
2020-10-05

RETRIEVAL DATE :
10/05/20 12:50PM

LINK – PUBMED:
https://pubmed.ncbi.nlm.nih.gov/33015606/?utm_source=Other&utm_medium=rss&utm_campaign=pubmed-2&utm_content=1jmC0p0kwOiHcoERYElvPhktie_Gw8TL2aOwN185ZhQUSROsDP&fc=20200821211545&ff=20201005125047&v=2.12.3

LINK – DOI:
https://doi.org/10.1016/j.xkme.2019.10.004

LINK – FULL TEXT:

NOTES:

Promising new drug being developed for polycystic kidney disease

September 12, 2019

https://www.news-medical.net/news/20190912/Promising-new-drug-being-developed-for-polycystic-kidney-disease.aspx

Promising new drug being developed for polycystic kidney disease

RGLS4326

Discovery and preclinical evaluation of anti-miR-17 oligonucleotide RGLS4326 for the treatment of polycystic kidney disease

September 12, 2019

https://www.nature.com/articles/s41467-019-11918-y

Discovery and preclinical evaluation of anti-miR-17 oligonucleotide RGLS4326 for the treatment of polycystic kidney disease

XORTX Highlights New Research on Polycystic Kidney Disease Awareness Day

September 4, 2019

https://www.globenewswire.com/news-release/2019/09/04/1910654/0/en/XORTX-Highlights-New-Research-on-Polycystic-Kidney-Disease-Awareness-Day.html

XORTX Highlights New Research on Polycystic Kidney Disease Awareness Day

KC charity will invest $5M to fund research into a cure for PKD

June 25, 2019

https://www.bizjournals.com/kansascity/news/2019/06/25/polycystic-kidney-disease-foundation-research-fund.html

KC charity will invest $5M to fund research into a cure for PKD

New drug compound could tackle major life-limiting kidney disease (ADPKD)

June 18, 2019

https://www.sciencedaily.com/releases/2019/06/190618103723.htm

New drug compound could tackle major life-limiting kidney disease
Date:
June 18, 2019
Source:
University of Sheffield
Summary:
Scientists are developing a new class of drugs to treat a common genetic kidney disease which is a major cause of kidney failure.
Share:

FULL STORY
Scientists from the University of Sheffield are part of an international collaboration to develop a new class of drugs to treat a common genetic kidney disease which is a major cause of kidney failure.

Working with Glasgow-based biotech company Mironid and colleagues in the US, the Sheffield researchers have carried out successful laboratory tests of a compound developed by Mironid to treat autosomal dominant polycystic kidney disease (ADPKD), a hereditary, progressive disease which affects over 60,000 people in the UK and around 12 million people worldwide.

Caffeine Accelerates Cystic Kidney Disease in a Pkd1-Deficient Mouse Model

April 13, 2019

https://www.cellphysiolbiochem.com/Articles/000072/

https://www.ncbi.nlm.nih.gov/pubmed/30977988?dopt=Abstract

Caffeine Accelerates Cystic Kidney Disease in a Pkd1-Deficient Mouse Model.

Cell Physiol Biochem. 2019;52(5):1061-1074

Authors: Meca R, Balbo BE, Ormanji MS, Fonseca JM, Iannuzzi LR, Santana Costa E, Onuchic LF, Heilberg IP

Abstract

BACKGROUND/AIMS: Autosomal dominant polycystic kidney disease (ADPKD) is characterized by progressive cyst formation and growth, leading to end-stage renal disease. A higher kidney volume is predictive of a more accelerated decline in renal function. This study aimed to examine the effects of caffeine, a phosphodiesterase inhibitor, on the progression of cystic kidney disease in a mouse model orthologous to human disease (Pkd1cond/cond:Nestincre).

METHODS: Caffeine was administered to male cystic (CyCaf) and noncystic (NCCaf) mice (Pkd1cond/cond) from conception and at the postweaning period through 12 weeks of life (3 mg/d), while control animals consumed water (CyCtrl and NCCtrl). Renal ultrasonography was performed at 10 weeks of life to calculate total kidney volume and cystic index. At the end of the protocol, blood and urine samples were collected for biochemical analysis, and animals were euthanized. Kidneys were harvested to obtain renal tissue for determinations of adenosine 3´5´-cyclic monophosphate (cAMP) by an enzymatic immunoassay kit and phosphorylated extracellular signal-regulated kinase (p-ERK) by Western blotting. Renal fibrosis (picrosirius staining), renal cell proliferation (ki-67 immunohistochemistry) and apoptotic rates (TUNEL analysis) were also determined.

RESULTS: At 12 weeks, CyCaf mice exhibited higher serum urea nitrogen, renal cystic index, total kidney volume, kidney cell proliferation, apoptosis and fibrosis compared with CyCtrl mice. Serum cystatin C was significantly higher in CyCaf than in NCCaf and NCCtrl mice. CyCaf mice had higher total kidney weight than all other groups but not higher heart and liver weight. The levels of cAMP and p-ERK did not differ among the groups.

CONCLUSION: Caffeine consumption from conception through 12 weeks led to increased cystic index and total kidney volume and worsened renal function in Pkd1-deficient cystic mice, suggesting that high consumption of caffeine may contribute to a faster progression of renal disease in ADPKD.

PMID: 30977988 [PubMed – in process]

PubMed:30977988

ADPKD: Polycystic Kidney Disease – Reddit

January 1, 2016

https://www.reddit.com/r/ADPKD/

ADPKD: Polycystic Kidney Disease – Reddit

Powered by Urgent Research

Copyright © 2025 Urgent Research