Posts Tagged ‘gene therapy’

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.


February 4, 2009

Sometime in the future….

Fred knew there was trouble when he was shown not to an examining room, but to his Cardiologist’s private office. In a few moments the doctor walked in.

“Lean back, Fred,” the doctor said, pulling out his pocket sized Echocardiogram reader. After a few moments, the doctor read the results as he sat down on the edge of the desk.

“You know what’s going on here so I won’t sugarcoat it, Fred. Your heart has taken you a lot further than anyone ever thought it would, but it can’t take the stress anymore. We have to do something soon.”

Fred sighed. He had a feeling that he wouldn’t like the news that he would receive today. “Am I a candidate for a transplant, Doc?”

The doctor shook his head. “We rarely do transplants anymore. Tomorrow I’m going to take a sample of your heart tissue for gene matching and then give you an injection that will regenerate your heart muscle. We have to sedate you for that, and you’ll be too groggy to drive. So get your girlfriend to come in with you.”

“Hopefully the gene matching will show us a therapy that can work. If it doesn’t, we still have options. We can give you a new Aortic valve without surgery. I can extract more heart tissue and we can use it to build you a new ventricle from scratch. We can replace that damaged artery. And if worse comes to worse, we can try an artificial heart until we figure out what’s next. You’re going to dance at your child’s wedding!”

“Doc, I haven’t even asked Anne to marry me yet!” Fred sputtered.