Tag: Research

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We are looking for Alport Syndrome patients for a study

I am a primary investigator of the Regulus RG012 study using RNA inhibition to treat Alport Syndrome.

Here are some good resources that I used to brush up my Alport and RNA inhibition skills.

First a video review of Alport

https://www.youtube.com/watch?v=mJ6ULJrdW7I

The best part of the video is the detailed description of collagen IV at 4 minutes. This finally allowed me to understand the multiple mutations that cause Alport.

This video is grand-rounds length description of Alport with a focus on how it affects women by my friend Michelle Rheault.

RG-012 uses a drug that is a oligonucleotide that inhibits the activity of miR-21. miR-21 is a micro RNA which affects the translation of mRNA to protein. In high school biology I learned about messenger RNA, transfer RNA, and ribosomal RNA and that was enough RNA for anyone. But then the Nobel Prize guys had to go and give a prize for the discovery of RNA interference in 2006. Here is a basic explainer about RNA interference.

Here is Regulus’ presentation on RG-012

If you are a physician in Michigan with a patient with Alport Syndrome (needs to be pre-dialysis and per-transplant) or a patient with Alport Syndrome please get in touch, we want to hear from you.

Joel Topf (jtopf@mac.com)

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Blogging as an academic endeavor

From Skeptical Scalpel (twitter)

Since I dropped out of the business of training residents, I have been actively blogging and not cranking out mindless publishable research. Here is an interesting fact. I have no doubt that far more people have read what I have written in my blog for a year and a half than ever read all of my 95 published works combined.

Sing it brother! It takes me about a hundred hours to prepare a de-novo talk for grand rounds. I will then deliver it to an cohort of 50-80 docs with a collective GCS of 10. After the last audience question the lecture video will be filed in the medical library never to be found again. The lecture is not searchable or discoverable and the work is largely lost.

An average post at PBFluids will get more views than the grand rounds in the first few days and if it is linked by RenalWeb other aggregator it will get enough page views to fill one of the great conference halls of the national meetings. After the first week the post continues to be an eternal flame of searchable and discoverable data. To me the relative impact tips way toward blogging as a more significant form of academic communication but to the powers that grant career advancement it is a meaningless toy.

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Getting another abstract (or two) ready for Renal Week 2009

We are racing the deadline for our ASN abstract. We have a great data set on geriatric patients in a structured CKD clinic. We need to define stable renal function. Our first try used the CKD stages. Our cohort is restricted to CKD 3b and 4. So stable was any patient who began and ended the study in the same CKD stage. This has been done in the literature, so there is some precedence but it doesn’t feel right to me.

Think about two participants in our study, both GFRs fall by 3 cc/min over three years, just about what the Baltimore Longitudinal Study on aging predicts. Patient A started with an eGFR of 31 mL and Patient B started with an eGFR of 33. These patients have the same clinical course and outcome but Patient A goes from 31 to 28 mL/min and hence from CKD Stage 3 to 4 while Patient B goes from an eGFR of 33 to 33 so his stage does not change.

I need a definition of stable renal function. You can help by filling this 5 question anonymous survey. We are looking

How would you define stable renal function:

Candidate A: Change in GFR less than 2 cc/min/yr (essentially 3x the average rate determined by the Baltimore Longitudinal Study on Aging).

Candidate B: Change of GFR of less than 20% from baseline to the end of the study

Candidate C: Change of less than 10 mL/min from the baseline visit

Note: baseline visit is the 1st contact with us with a GFR<45 mL/min(CKD stage 3b), we removed any patient who does not have a second eGFR < 45 at least 3 months before the initial measurement.

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Data Capture Form

We are going to study renal and patient survival in our CKD clinic. One of our team members who is charged with creating the data capture form wanted to look at prior form. So here it is.