Monday, December 23, 2013

Stem cell agency funded heart therapy approved for Phase 2 clinical trial

To be involved in science you need to be committed and persistent. Good science takes time. Sometimes it takes a lot of time. So when you hear that a project that you have been supporting has been approved to move into a Phase 2 clinical trial that you are funding, that’s pretty exciting.

That’s just what happened with Capricor Therapeutic’s treatment for people who have had heart attacks. It’s been cleared to move into the next stage clinical trial, a trial that the stem cell agency is funding.

In a news release CIRM President, Alan Trounson, Ph.D., said:
“This is really exciting news and marks a major milestone for the use of stem cells to treat heart disease. We are delighted to be involved in funding this work. This is precisely what our Disease Team Awards were designed to do, to give the most promising treatments up to $20 million dollars to develop new treatments for some of the deadliest diseases in America.”
For those of you not familiar with clinical trials they are done in stages. Phase 1 is typically the first time this therapy has been tested in people so this stage is to make sure that it’s safe, that it doesn’t have any unexpected or nasty side effects. Phase 2 involves testing it in a larger group of people to further test for safety and also to see if it has any beneficial effect on the patient. Up till now the research has involved testing in the laboratory and in animal models, where it has obviously shown promise. Phase 2 is an important step on the road to approval because it attempts to see if this therapy works in people.

Capricor’s work involves using stem cells called cardiosphere-derived cells. In the first phase of human testing these cells appeared to reduce the scarring in the heart muscle after they were infused into a patient who had experienced a heart attack. This is important because if you can reduce the scarring you may be able to improve the function of the heart, even after something as traumatic as a heart attack.

An earlier version of this therapy involved harvesting the stem cells from the patients themselves, reprogramming those cells and then re-introducing them to the patient. One of those who underwent the procedure was Fred Lesikar. He says before the procedure the after-affects of a heart attack left him weak, and without any energy. After the therapy he says he has his life back.

The version of the treatment that is now heading into Phase 2 trials does not involve taking cells from each individual heart attack patient, instead it uses donor cells, this means it can become an off-the-shelf-therapy, something that is available when and where the patient needs it rather than having to create it new each time.

The first trial involved a small number of people. This next one will involve around 300 people who have had heart attacks. One group will be treated within 30-90 days of the attack; a second group will be treated within 91 days to a year after the incident.

It’s taken time to reach this point, but if it pays off it will be time and money well spent. It’s estimated that close to 5 million Americans have some form of heart disease, with 400,000 new cases diagnosed each year. Clearly there is no shortage of need for new and more effective treatments. That’s why we fund a lot of other work in heart disease

kevin mccormack 

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