RALLY FOR ALI

Deadly Progression of ALS Reversed in an Amazing Stem Cell First

Last May, 39-year-old Ted Harada was diagnosed with ALS, also known as Lou Gehrig's disease. It's one of the worst diagnoses anyone could get.

He and his doctors expected his health to have severely declined by now. But thanks to an experimental stem cell treatment, he has tossed his cane and is once again playing in the pool with his three kids.

"Every day is a good day for me right now," Harada told me. "I've made some quantum leaps after the surgery and... I'm maintaining the drastic improvements I've made."

The prognosis for ALS is likely death in fewer than three years after gradually losing your ability to move, speak and breathe. Harada got exactly that horrible news after losing strength in his left leg and feeling short of breath after just a few stairs or walking to the mailbox. His hands became too weak to open a Ziploc bag.

Then his neurologist told him about an experiment at Emory University that was recruiting ALS patients to test a stem cell treatment.

The surgeons told Harada that injecting the stem cells into his spine likely would not help him personally, and might even cause harm. But the study would hopefully help scientists find an effective treatment in the future. Harada had nothing to lose and expected nothing - he became study subject number 11 and underwent surgery on March 9.

It's incredibly moving to hear Harada talk about his recovery, which he knows might be temporary.

"I've always been the kind of dad to wrestle on the floor with my kids and tickle them and make them giggle, and that was going away before," Harada told me. "Now when we get in the pool and they want to play Marco Polo, I can do that."

The Emory surgeons injected 1 million neural stem cells into 10 locations in Harada's spine (earlier patients received fewer cells; the dosage was gradually increased as the trial progressed). All of the cells came from a single voluntarily aborted and donated two-month-old fetus. Using technology developed by Neuralstem, scientists multiplied the cells and created enough of them to treat all of the patients in this trial and beyond.

"We took one small part of the spinal cord and isolated one million stem cells which are now going to, we hope, treat millions of people around the world," Dr. Karl Johe, chief scientific officer at Neuralstem told me.

Going into the study, expectations were low. As a safety precaution, the FDA forced the researchers to inject only one-quarter the number of stem cells they originally planned to use. The investigators hoped to show the cells were safe to use, but anticipated little more.

Two of the 12 patients died during the trial, one after a heart attack and another because of progress of his ALS. The rest of the patients' conditions have remain unchanged.

The researchers hope the Food and Drug Administration will allow them to add six more patients to the trial so they can collect more data on the treatment's safety. Neuralstem is also awaiting approval to begin the first phase of a fetal stem cell trial in chronic spinal cord patients.

Unregulated stem cell outlets, such as the one promoted by Republican presidential candidateRick Perry, claim success treating ALS and just about every other disease you can imagine. But they haven't gone through the painstaking methodology required to run an FDA-approved human clinical trial, which demands reams of data with the goal of assuring safety and eventually proving efficacy. Such trials can also help convince insurance agencies to cover the treatments. Otherwise, rogue outlets will continue charge up to 10s of thousands of dollars for treatments.

"We go through the FDA process, which is excruciatingly slow, but we do that because it's what the law says we have to do," Johe said.

Update: The original version of this article said the Emory and Neuralstem researchers are awaiting FDA approval for a phase two clinical trial. They are actually waiting for approval to add six more patients to their phase one trial.