Is ALS breakthrough imminent?


Pioneer Founding member
Boomer Health: Is ALS breakthrough imminent?
By Steve Dorfman

Maybe you’ve already dumped chilled water atop your head — and encouraged friends and loved ones to do likewise by taking the ALS Ice Bucket Challenge.

Or, perhaps inspired by seeing the innumerable videos of people doing the Ice Bucket Challenge, you’ve made a donation to the cause, thereby contributing to the unprecedented tens of millions of dollars that this grassroots initiative has raised.

Regardless, it’s likely you’ve heard more about amyotrophic lateral sclerosis — aka Lou Gehrig’s disease — in the past month than the world had in the 75 years combined since the Yankees Hall of Famer made his legendary “luckiest man on the face of the earth” retirement speech.

Of course, we here at The Post are all too familiar with ALS. Our colleague, former award-winning courts reporter and best-selling author Susan Spencer-Wendel, succumbed at age 47 to the neurodegenerative condition earlier this summer, three years after being diagnosed.
What is ALS?

ALS is a progressive, invariably fatal disease that disables the body’s motor neurons — which are specialized nerve cells in the brain and spine. Experts believe that, in 90 percent of ALS cases, the cause is “sporadic” — meaning there is no identifiable origin. Though the symptoms are initially subtle — twitching, cramping and/or general weakness in the muscles — those stricken with the disorder eventually lose all voluntary motor function, and are effectively paralyzed.

Typical life expectancy for a person diagnosed with ALS: three to five years. Statistically speaking, the disease is rare, with just 5,000 new cases diagnosed annually in the U.S., according to the National Institute of Health.

The only FDA-approved medication for ALS — riluzole (brand name: Rilutek) — may help extend ALS patients’ life expectancy by a few months by prolonging the period until patients need to be put on ventilators. But it doesn’t repair damage to motor neurons.

Promising new treatment

There may be encouraging news in regard to slowing the progression of ALS in those already afflicted.

Earlier this year, the FDA approved a Phase II clinical trial — to be conducted in the fall at Massachusetts General Hospital, the University of Massachusetts Memorial Hospital and the Mayo Clinic — of a stem-cell treatment that was created in Israel.

Developed and initiated in Jerusalem by Hadassah Medical Organization neurologist Dimitrios Karussis, the treatment involves harvesting the patient’s own stem cells, multiplying them via laboratory cultures, and then re-injecting them back into the patient’s spinal fluid.

Karussis first began injecting enhanced “autologous” stem cells into the spinal fluid of ALS patients in 2007.

In all of the cases, the patients tolerated the treatment well and responded positively to it. Over a 12-month period, two-thirds of the subjects showed a marked slowing — or “stabilization” — in the progression of symptoms. In fact, three patients were even said to have experienced a “regression” in symptoms.

Karussis and his colleagues at Hadassah believe that the treatment, while promising, is not permament — and, for maximum efficacy, will need to be repeated every few months.

And that’s where all those ice-bucket-inspired donations can really help make a difference. Harvesting and growing hundreds of millions of enhanced autologous stem cells for ALS patients is expensive (the Israel-based biotech company, BrainStorm, will be growing the ALS stem cells for the U.S. trials).

But the potential payoffs are enormous: Karussis and his counterparts in the U.S. believe that if the ALS stem cell trials — which will last for a year — prove successful, then similar treatments for neurological disorders such as Parkinson’s and multiple sclerosis will also be viable.


Pioneer Founding member
Israeli Company Hopes to Find Real Treatment for ALS

BrainStorm looks beyond the Ice Bucket Challenge to treat Lou Gehrig's Disease with their NurOwn treatment

By: Maya Yarowsky and Anouk Lorie, NoCamels
Published: September 1st, 2014

If you’ve been on the Internet anytime lately, you will have noticed that one disease is dominating the headlines; ALS, also referred to as motor neurone disease. With the Ice Bucket Challenge, which is meant to raise funds for ALS research, people around the world, including Hollywood celebrities, are nominating one another to drop a bucket of ice cold water on their heads. But while the spoof has gone viral, many may have overlooked why the “chilling” challenge began in the first place.

ALS is a severe, rapidly progressing neurodegenerative disease in which patients lose all ability to control their muscles, usually resulting in death from respiratory failure within three to five years of diagnosis. Yet way before Pat Quinn, himself an ALS patient, created the Ice Bucket Challenge a year ago, Israeli medical company BrainStorm was already laying the bricks for the most comprehensive treatment so far of this deadly disease.

Treating patients using their own stem cells

BrainStorm’s NurOwn treatment is about to enter Phase II clinical trials in the United States. Following the successful initial treatment of four advance-stage ALS patients at Hadassah Medical Center in Israel, BrainStorm, which focuses on stem cell treatments for neurodegenerative disorders including Parkinson’s disease and Multiple Sclerosis (MS), is hoping for similar successes in the American trial study.

Based on the research of Profs. Eldad Melamed and Daniel Offen of Tel Aviv University, NurOwn works by extracting stem cells from the ALS patient, adding chemicals to the cells until they are able to autonomously grow new neurons, then re-injecting the stem cells into the muscles and spinal region of the same patient. According to Melamed, “These nerve cells secrete a growth hormone that supports existing nerve cells, slowing down, and even halting degeneration.”

Promising results in Israeli trial

For the four patients treated at Hadassah Medical Center, the NurOwn trial was the first glimmer of hope they had been given in a long time. One of them, Rabbi Refael Shmulevitz, was given two to four years to live before receiving the NurOwn injection. Two years later, Rabbi Shmulevitz is alive and well, with a dramatic improvement in his symptoms soon after he underwent the treatment.

This article was first published on NoCamels - Israeli Innovation News and was re-posted with permission. To continue reading this article on the site, click here.