Doctors Perform Historic First in Regenerative Medicine Trial on Human Heart

Monday, December 2, 2013

Doctors Perform Historic First in Regenerative Medicine Trial on Human Heart
 Regenerative Medicine
A University of Utah doctor has performed the historic first procedure using new regenerative medicine technique called retrograde gene therapy to restore function to a human heart severely damaged by cardiac arrest.




Aman in Utah has become the first patient in the world to undergo retrograde gene therapy at University of Utah Hospital, a novel procedure designed to deliver stem cells to the heart to repair damaged muscle and arteries in the most minimally invasive way possible.

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Amit Patel, M.D., director of Clinical Regenerative Medicine and Tissue Engineering and an associate professor in the Division of Cardiothoracic Surgery at the University of Utah School of Medicine. started investigating cell and gene-based therapies for the treatment of heart disease 12 years ago, but only recently received FDA approval to try the therapy on Ernie Lively, who was the first of several patients anxious to receive the treatment.

More than 6 million people are currently living with heart failure. As the condition progresses, patients’ options are usually limited to a heart transplant or assist devices, such as an artificial heart. Patel wanted to find a way to intervene in the progression of heart failure before a patient advanced to the point of needing a heart transplant or device.

Patel and his team came up with the idea of retrograde heart therapy, a concept that has been discussed for 50 years. The first successful procedure was performed on Lively on November. 7.

“It’s incredible. Imagine having a heart procedure that can potentially regenerate or rejuvenate your heart muscle — and it’s done as an outpatient procedure,” said Patel.


Patel used a minimally invasive technique where he went backwards through a patient’s main cardiac vein, or coronary sinus, and inserted a catheter. He then inflated a balloon in order to block blood flow out of the heart so that a very high dose of gene therapy could be infused directly into the heart.

The unique gene therapy did not involve viruses (a rarity for gene therapy, Patel notes) and is pure human DNA infused into patients. The DNA, called SDF-1, is a naturally occurring substance in the body that becomes a homing signal for a patient’s body to use its own stem cells to go to the site of an injury.

Once the gene therapy was injected, the genes acted as “homing beacons.” When the genes are put into patients with heart failure, they marinate the entire heart and act like a look out, Patel said.

“The genes basically act like a light house with a bright signal. They say, ‘ How can we help the ships that need to get to the port — which is the heart –get there. When the signal, or the light from the SDF-1, which is that gene, shows up, the stem cells from not inside your own heart and from those that circulate from your blood and bone marrow all get attracted to the heart which is injured, and they bring reinforcements to make it stronger and pump more efficiently,” said Patel.

After becoming the first patient in the world to undergo the procedure, Lively returned home and is recovering. Before the technique Patel used was available, Lively’s other option would have been a three-to-five day hospital stay. Instead, he is recuperating while daydreaming about what it will be like to be able to ski and enjoy life fully again. He said he has noticed an immediate difference in his health following the procedure.

“I woke up this morning and told my wife, ‘I haven’t felt this good in years,” said Lively. “I moved to Utah because of the snow, but I haven’t been able to ski. I literally didn’t have the heart to do it. Now, I’m excited about living the rest of my life instead of sitting around.”

Patel said watching Lively recover successfully from the surgery is both rewarding and exciting for what the future holds for the procedure and those who may benefit from it. He is already training other physicians around the U.S. to model what he accomplished first this month. He is overseeing a trial of the procedure in which 72 patients will participate over the next few months.

“This is one of the great moments in biological therapy for the heart,” said Patel. “We are providing options for patients who have no possible solutions. This is one of the safest and most reproducible therapies out there for these very sick patients.”



SOURCE  University of Utah

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