Stem cells in small hearts fight birth de­fects

Step to adult re­pairs

The Washington Times Daily - - METRO - BY LAURAN NEERGAARD

BAL­TI­MORE | The 4-month-old on the op­er­at­ing ta­ble has a shock­ing birth de­fect, nearly half his heart too small or miss­ing. To save him sur­geons will have to reroute how his blood flows, a dras­tic treat­ment that doesn’t al­ways work.

So this time they are go­ing a step fur­ther. In a bold ex­per­i­ment, doc­tors in­jected do­nated stem cells di­rectly into the healthy side of Jo­sue Sali­nas Sal­gado’s lit­tle heart, aim­ing to boost its pump­ing power as it com­pen­sates for what’s miss­ing.

It’s one of the first at­tempts in the U.S. to test if stem cells that seem to help heart at­tack sur­vivors re­pair car­diac mus­cle might help these tiniest heart pa­tients, too.

“We think the young heart is able to be more re­spon­sive,” said Dr. Sun­jay Kaushal, chief of pe­di­atric car­diac surgery at the Univer­sity of Mary­land Med­i­cal Cen­ter, who is lead­ing the study work­ing with Univer­sity of Mi­ami re­searchers.

“We’re not say­ing we’re go­ing to cure it,” Dr. Kaushal said of the birth de­fect, called hy­poplas­tic left heart syn­drome. But “my whole quest is to see if we can make these lit­tle kids do bet­ter.”

Jo­sue’s par­ents knew there was no guar­an­tee the ex­per­i­men­tal in­jec­tions would make a dif­fer­ence. But their son had been hos­pi­tal­ized since birth and needed open-heart surgery any­way for a chance to go home. Teary-eyed, they clasped hands and prayed over Jo­sue’s crib mo­ments be­fore nurses wheeled him to the op­er­at­ing room.

“We are march­ing ahead with God,” said Jo­sue’s fa­ther, Hidel­berto Sali­nas Ramos, speak­ing in Span­ish through a hos­pi­tal in­ter­preter.

Nearly 1,000 ba­bies are born with hy­poplas­tic left heart syn­drome in the U.S. each year. It’s the most com­plex car­diac birth de­fect.

Jo­sue is miss­ing his left ven­tri­cle, the main pump­ing cham­ber that pushes oxy­gen-rich blood to the body. Other key struc­tures on his heart’s left side are too small or mal­formed to work.

Al­ways lethal un­til a few decades ago, this de­fect now is treated with three open-heart surg­eries per­formed between birth and age 3. Doc­tors route blood around the ab­nor­mal left heart and they con­vert the right ven­tri­cle — which nor­mally would shut­tle oxy­gen-poor blood to the lungs — into the main pump­ing cham­ber.

To­day about 65 per­cent sur­vive at least five years, and many reach adult­hood, said Dr. Kristin Burns, a pe­di­atric car­di­ol­o­gist at the Na­tional In­sti­tutes of Health.

But too many chil­dren still die or re­quire a heart trans­plant be­cause the right ven­tri­cle wears out un­der its in­creased work­load.

That’s why doc­tors are con­duct­ing this earlystage study of whether stem cells help that ven­tri­cle work bet­ter.

“This is very dif­fer­ent than a sur­gi­cal ap­proach or giv­ing a medicine just to treat the symp­toms. This is try­ing to treat the un­der­ly­ing prob­lem,” said Dr. Burns, of NIH’s Na­tional Heart, Lung and Blood In­sti­tute.

Even in adults, stem cell re­gen­er­a­tion is highly ex­per­i­men­tal. But small stud­ies in­volv­ing heart at­tack sur­vivors and older adults with heart fail­ure have found what Dr. De­nis Bux­ton, a stem cell spe­cial­ist at NIH’s heart in­sti­tute, calls a mod­est ben­e­fit in how well their hearts pump blood.


Doc­tors in­ject stem cells into the healthy side of a pa­tient’s heart at the Univer­sity of Mary­land Med­i­cal Cen­ter in Bal­ti­more to com­pen­sate for a heart birth de­fect.

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