Posts Tagged ‘Clinical Trial’

New gene therapy for Heart Failure shows promise

June 28, 2010

A new option for combating Heart Failure (or Congestive Heart Failure, also known as CHF) is showing promise as the CUPID Study

Heart Failure occurs with the heart muscle begins to wear out and lose its elastic properties. It isn’t a Congenital Heart Defect (CHD) itself, but a CHD can trigger it as a patient becomes older. And since I have CHF, I’m interested in keeping an eye on it!

The normal heart contains a protein that has a long medical name that I can’t say and can barely type. Thankfully, it is also known as SERCA2a. In a heart going through Heart Failure, the levels of  SERCA2a begin to drop. All is OK for a while, but it is like driving a car without ever checking the oil. You can take care of yourself, eat right, exercise, and write a letter to your mother every week, but if your heart isn’t producing SERCA2a then you’ve got a real problem on your hands. Eventually the CHF wins.

You can’t drink this stuff or take it in pill form. Instead, the gene that makes this protein is inserted into a virus. Not a virus that can make you sick, but acts as a transport mechanism for the gene. The virus (which in its commercial form is called MYDICAR) is inserted directly into the heart by catheter. In the study, there were 39 patients in total and the ones receiving the drug received low, medium, or high doses. And while there were no “adverse outcomes” the results were rather strange. Sometimes patients had the best response to lower doses of the enzyme. That really doesn’t make any sense.

It does look promising, if they can figure out why some people respond better to lower doses of the therapy. But this was a Phase 2 clinical trial studying a limited number of patients. We’ve got a few years to wait before this becomes available.

Clinical Trial stopped!

June 2, 2010

Pfizer has decided to stop the recruitment of patients into the EMPHASIS-HF drug trial early. That headline should raise some eyebrows, since it means one of two possible outcomes: Early results are either very good or very, very bad.

Thankfully, the results seem to be very good! According to the press release (a 5 page .pdf file) the study reached its “primary efficacy endpoint” early – in other words, the investigators proved what they were looking for. Taking Eplerenone (also known as Inspra) in conjunction with recommended Heart Failure standards of care, lowers incidence of hospitalization and/or death in patients with mild to moderate heart failure. The adverse events (things that went wrong) included a higher level of potassium in 8% of the patients taking Eplerenone and 4% had renal impairment. Renal impairment means that their kidneys stopped functioning, but that condition can occur because of heart failure also. Apparently the incidence of Renal impairment caused by the new drug is not much different from the number of kidney problems cause just by having heart failure. The Clinical Trial was a double-blind test, which lends it even more credibility, as neither the subject or the investigator knew which drug was being used – the Eplerenone or a harmless placebo. When carried out correctly, a double-blind test prevents either the subject or the investigator from being biased.

So what happens now? Don’t expect to be getting a prescription for Eplerenone, the company does not have a license to sell to human subjects. The study was a small one – only 3100 people, if it had reached full enrollment. All patients, no matter which drug they are on, will be informed of the decision to close the study early. Pfizer has asked for permission to move all consenting patients into a larger study of the drug.

Sometimes medical research comes in small increments – baby steps. But you have to walk before you can run!

I ain’t got no money, honey!

April 14, 2010

Here’s my third article about the SQUID (Superconducting QUantum Interference Device, the first two are HERE and HERE) but for this post, I’ve found a photo of the machine at work. Here it is, with the “snout” pressed against the obviously pregnant patient. There’s just one problem: Read the article that accompanies the photograph, and you’ll learn that the company that built this particular unit is no longer in business.

This is one of the more painful things about living in a bad economy, and few people realize it. The economy slows down, people lose jobs, and money becomes scarce, Medical Research is one of the fields that suffers.

Assume a Hospital/University/Drug company has a surplus that can be used in any manner they choose. Most of the money will be spent, but the account is  replenished every month. What would you do with it?

If you are head of our fictious University, you’ll probably have a lot of different groups with their hands out. Security needs some of the money – we’re obligated to keep our students safe. Housing gets some, Athletics gets a chunk, the Science department needs some… and of course, some of it will go to Research. And since our University is affiliated with a medical school and a hospital, some of that cash will be earmarked for Medical Research.

Then suddenly there is an economic downturn and our surplus isn’t as large as it once was. Everybody has to make do with less. The economic downturn morphs in to a crisis and the surplus is even less. Now something has to give, and everybody has to figure out what they can live without. Some programs and plans get shifted to the back burner, and we’ll pick them up again when things are looking brighter.

And when the medical research has to be cut back, we all suffer. Take for example the Coapsys, which is a neat little idea. The Coapsys is two pads and a string, passed through the heart and then a little bit of tension applied. The idea was that the cord could reshape an enlarged heart and force a leaky Mitral Valve to work better.

Say what? That sounds pretty far-fetched.

The funny thing is, the bloomin’ thing works! A clinical trial showed that no one who received the device had a complication during the time they were in the hospital to receive the device, and the mortality rate was about half that of those who received the traditional valve replacement.

Sounds great! The bad news is, the company that developed the device went out of business during the trials. Another company bought the Coapsys device and all intellectual property associated with it – but has no plans to further develop the device or market it. The article doesn’t say, but it might be a fair guess to assume that the financial crunch has them down, too.  No one is immune.

Thankfully the Coapsys survives, and the trials look good. And anything that shows positive results with little to no side effects, AND with a lesser chance of passing away… that device will be on the market one day.

But right now, the people who need it have to wait.

Ask Questions!

April 6, 2010

Here’s a scary report, courtesy of Kevin, MD: Patients don’t ask questions of their doctors.

While there are a precious few patients who are totally involved in their health care, the vast majority just take their doctor’s advice at face value. A 2008 study found that when 181 people were prescribed a new medication, they asked a total of 199 questions (or made a comment) about the new drug. That’s an average of 1.09 questions/comments per patient!

What’s worse, the same study showed that the doctors didn’t talk, either. The average office visit was 15.9 minutes, and the patient and doctor spent an average of 49 seconds discussing the medication. The length of discussion ranged from a high of 351 seconds (5.85 minutes) to an amazing 1.9 seconds! (What can you say in 1.9 seconds?!?!)

As noted before, patients who are more involved in their own health care ask more questions. That’s you. Having a Congenital Heart Defect means that you are, for all intents and purposes, a patient pool of ONE. Others may have the same defect that you do, but no CHD ever treats its owner like everyone else.

As I’ve written before, I have a hernia. It’s usually well-behaved, but occasionally it will get pretty angry with me. A hernia repair is a fairly simple operation these days, and usually doesn’t even require an overnight stay… except for me. My Cardiologist does not want to authorize the operation, instead asking me to just fight through the bad times by prescribing couch time and TV. “I could spend a day explaining your anatomy to the surgical team,” he has said. “And they still wouldn’t understand it.” It’s not that he can’t, my doc has a couple of teaching awards to his credit. I’m complicated.

So if you don’t know what’s going on with your body, it is time to learn. And ask questions – what is this medicine supposed to do? What are some of the side effects? What do you think would happen if I decided not to take this drug? Are there any other options available? All of these are legitimate questions – and if your doctor gives you an answer in 1.9 seconds, ask another question. You can control how long he talks to you. Conversely, you can find a doctor who will spend the time needed to help you make a good decision.

It’s your body, and the medical decisions you make affect you, and rarely anyone else.

Change to the Blogroll

February 16, 2010

I’ve made a change in the blogroll, the list of websites and online resources that appear on the right side of the home page. Clicking the link labeled RESEARCH: Congenital Heart Defect Clinical Trials now takes you to a page maintained by Medpedia.

This change was made for two reasons: Medpedia offers summaries of Clinical Trials in either “Plain English” (easy to read) or “Clinical” (More technically correct, but uses a lot of medical terms that you might not be familiar with). Medpedia also offers you a lot of search options: You can search for a clinical trial based on location, actively recruiting participants (or not), Clinical Trial Phase, age of the participants, and other factors. If there is a clinical trial studying retired 70-year-old left-handed tugboat captains, you can find it!

I’ve preset the words Congenital and Heart as the search terms, but feel free to change them if you wish.

A Cure for the Funky Heart?!?!

December 10, 2009

I’ve got Google Alerts searching for the appearance of certain Heart Defect words and phrases across the internet, and they deliver new information to my computer every night. The information is new to me, but not always new.

So imagine my surprise when I read this report from a 1981 edition of the Scandinavian Cardiovascular Journal. Obviously, the internet didn’t exist in 1981 (or exist in the form that we know today) so this couldn’t have been put on line then; apparently another organization recently uploaded it – and Google Alerts “hit” on the phrase Tricuspid Atresia.

In eight patients from 1976 until 1980, tricuspid atresia (TA) was corrected with valved xenograft conduits…


This is unreal – in this trial, eight patients with Tricuspid Atresia were given a conduit that ran from the Right Atrium to the Pulmonary Artery, or a conduit that connected the Right Atrium to the Right Ventricle and “jumped” the missing Tricuspid Valve. This is the first time I have heard of this… but it sounds as if it might work.

All patients suffered from transient right-heart failure postoperatively and eventually developed normalized cardiac function throughout the first two months after operation.

Holy cow, it did work! All eight went through a passing phase of right side heart failure that quickly stabilized, and in two months their hearts began to function normally!

X-ray examination showed normalization of the heart size in the majority of the patients, and in those with conduits between the right atrium and the right ventricle a considerable enlargement of the right ventricular chamber together with normalization of right ventricular contractility had developed. Arterial oxygen saturation, haemoglobin and haematocrit values had normalized in all patients.

“Normalization” is a word I like to read – especially when pertaining to Heart Defect, and for damn sure when it’s MY defect! There was one adverse outcome – one patient died of “intractable right ventricular failure, septicaemia and intravascular coagulation” – in layman’s terms,  there was blood poisoning, runaway blood clotting and the Right Ventricle failed for an undetermined reason.

Two patients with valved conduits between the right atrium and the right ventricle showed a normal unrestricted level of activity without medication, while patients with valved conduits between the right atrium and the main pulmonary artery were digitalized with an almost normal level of activity. Early repair with valved conduits of patients with TA is advocated.

But despite the success of this very limited trial, I can not find any information about followup trials. Today’s Tricuspid Atresia patients do not have this treatment option. Which begs the question:

What happened?

The obvious answer to why this isn’t being used today is “Something went wrong.” – but what? I’d certainly like to know. With 2009 technology, this trial could very well be worth repeating.