Brainstorm Stem-Cell Therapy Continues to Show Treatment Effect in ALS Patients

By: Adam Feuerstein | 01/05/15 - 10:52 AM EST

Stocks in this article: BCLINEW YORK( TheStreet) -- An experimental stem-cell therapy developed by Brainstorm Therapeutics (BCLI) continues to demonstrate early but encouraging activity in patients withamyotrophic lateral sclerosis (ALS), the degenerative and fatal nerve disease which has stymied drug developers for years. Brainstorm sharesare down over 20% to about $6 Monday but that's more of an expected, sell-on-the-news reactionthan a negative referendum on the updated, final results from the NurOwn phase IIa study. Brainstorm's stock price more than doubled in the previous eight trading days, so profit taking on today's data announcement isn't surprising at all. Must Read: Jim Cramers Five Best Stock Picks for the Biotech Sector I last wrote about Brainstorm in October.The company's experimental NurOwn stem-cell therapy starts with the harvesting of bone marrow from each ALS patient. Mesenchymal stem cells are isolated from the bone marrow sample and sent to a lab, where a proprietary Brainstorm process first expands their numbers and then transforms them into specialized cells secreting nerve growth factors. After the function and potency of the NurOwn cells are confirmed, the "drug" is delivered back into the ALS patient via injections into muscle and the spine.

Once injected, the NurOwn stem cells bathe the damaged neurons of ALS patients with secretions of nerve growth factors. Brainstorm has a home run on its hands if NurOwn can be shown to slow or halt the progressive destruction of neurons, and if that disease-modifying effect translates into improved muscle function for ALS patients.

Monday's update comes from a Phase IIa trial in which 14 ALS patientswere followed for the three months without treatment. At month four, each patient wastransplanted with their own personalized NurOwn therapy and then assessed every month for six months. Brainstorm evaluated NurOwn's impact on ALS disease progression using the ALSFRS score, a commonly used assessment of treatment response and muscle function in ALS patients. Lung function, another commonly used measure of efficacy in ALS clinical trials, was also measured.

Twelve ALS patients were evaluable for response. Of these, 11 patientsshowed aslowing of ALS disease progression at six months compared to baseline, measured either by improved ALSFRS or lung function scores, Brainstorm said. Two other patients enrolled in the study died. Administration of the NurOwn therapy was well tolerated by patients, the company said.

The final Phase IIa data announced Monday were a small improvement over interim results from the same study presented last June. Further, detailed data from the study will be presented at a medical meeting later this year. For perspective purposes, it's important to note that this phase IIa study enrolled a relatively small number of ALS patients and was conducted at a single hospital in Israel. This doesn't necessarily discredit the positive results, but conclusions about NurOwn's ultimate benefit as an ALS therapy can't be drawnuntil data from larger studies are gathered.

Brainstorm is conducting another, larger Phase II study in the U.S., enrolling 48 ALS patients who will be randomized 3:1 to receive a single NurOwn treatment in the muscle and spine, or a placebo treatment. The study is being conducted at two hospitals in Massachusetts, UMass Medical Center and Massachusetts General, and the Minnesota-based Mayo Clinic. The study's primary endpoint is the safety and tolerability of NurOwn, but investigators will also assess ALS patients for efficacy using measures of ALS disease activity and muscle function. The first patient was enrolled into the Phase II study last June and Brainstorm expects results to be ready in the first half of 2016.

The company is also in the planning stages for another Phase II study in which ALS patients will be treated with multiple doses of NurOwn.

Must Read: 11 Best Small-Cap Technology Stocks That Could Hit It Big in 2015

Source: http://www.thestreet.com/story/12999535/1/brainstorm-stem-cell-therapy-continues-to-show-treatment-effect-in-als-patients.html

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Bangert: An awful thief called ALS

Jim Gardner of Fowler died Saturday after a year-and-a-half struggle with ALS, also known as Lou Gehrigs disease.(Photo: Dave Bangert/Journal & Courier)

During the height of the Ice Bucket Challenge frenzy, last summer's viral fundraiser that sent more than $100 million the ALS Association's way, I turned to Jim Gardner to make a ruling.

Was the fad legit or a scheme built on misplaced good intentions? Did all those progressively creative stunts with five-gallon buckets of ice water pour out hope or just douse an already bleak prognosis?

In other words, I asked Gardner, a friend in Fowler living with amyotrophic lateral sclerosis: Are you OK with all this?

"Do it," Gardner wrote. "I think it's great. I am still gobsmacked that it is happening."

In fact, Gardner, diagnosed in August 2013 with the neurodegenerative condition known as Lou Gehrig's disease, already was laying out plans to do things one better.

He'd dug out a song he'd written, recorded and nearly forgotten in the late '70s, called "Get Wet" complete with references to Jacqueline Bisset in the movie "The Deep," and hailing from a time "when I still thought if I wrote enough beach-oriented songs that I could join The Beach Boys." Friends and family made T-shirts that paraphrased the chorus: "Get ready. Get set. Get wet." And they all made invitations to join with one more round of ice buckets in the name of #TeamJim.

"Now," Gardner said, "is the time I have."

And he was going to make of it what he could.

No surprise there.

Gardner, a friend from a local music scene of a different era, told the most touching story I heard all of 2014. I wrote about it just about a year ago now. Here's the short version.

Before ice buckets started to tip, he sent a note out of the blue under a subject line, "Hello, I Must Be Going." In it, he detailed how he was methodically contacting friends and acquaintances to tell them about his ALS diagnosis a disease the ALS Association estimates affects an estimated 30,000 Americans how it was progressing quickly and how he wanted to say "so long" on his own terms.

His note to me was prompted by a column I'd written a few weeks earlier about a Lafayette art installation called "Before I Die ".

Part of his farewell tour included assembling his sons, Ryland and Delaney, along with some other musicians and rushing into a Lafayette studio to record a love note to his wife, Melinda. ("As close to a bucket list item as I had," he said.) By the time he went in to record "I Married a Blonde," he was able to sing, but his fingers were unable to play the keyboards something he'd done since he started putting bands together in high school.

But the tribute to the marriage was recorded and pressed, as Gardner testified, "before I die "

After I wrote about the "I Married a Blonde" episode, several people wanted to clarify that ALS wasn't necessarily a death sentence. That the disease progresses at different rates for each person. That many people are able to continue with their lives, diminished perhaps, for years.

Gardner didn't seem to kid himself, acknowledging the speed and brutal efficiency of ALS as it took away motor skills.

Last summer, his family started reserving the bar and patio at the Benton County Country Club on the second Friday of the month for Gardner to get out and see friends. The September edition was set aside for the "Get Ready. Get Set. Get Wet" Ice Bucket Challenge.

That night, Gardner had the look of a man who had a million things to say. But secured in a motorized wheelchair, hands unable to move, he rolled his eyes at his impossibly slow delivery mind racing, trapped in a body unwilling.

In labored, almost gasping speech, each syllable a considerable effort, it took him what seemed like a minute to get this much out: "It's frus tra ting to see how ra pid ly my speech is de ter i or a ting "

This was a guy who I had at one point in our lives wasted about an hour like it was nothing, hashing out whether "Born Fighter" belonged among Nick Lowe's greatest songs. (I can't remember the verdict. But with Gardner, the testimony was always pure gold.)

At the head of a large round table in the country club dining room that night, his sister, Suzi Lundergan, took his cues, translating his shorthand into deep, rich stories about people who had been stopping by, old friends who'd sent him gifts, music he used to play and some dark humor about an urn someone wanted to make for him.

And how his family had come to his side when he needed them most.

Two days later, Lundergan's home church in Fort Wayne planned an Ice Bucket Challenge in her brother's name. The Rev. Derek Weber preached that day on his initial reluctance to get in on a silly stunt at Aldersgate United Methodist, questioning as I had whether it would do much good. But then he pivoted, reading from Lamentations, Chapter 3, about hardship, darkness and ultimate hope, and reading a letter Gardner dictated to church members about to get soaked.

The ice buckets too late for his own cure, Gardner figured had became one way he could frame the love, attention and care that came his way in his final year and a half.

That included all these people he didn't know personally, doing stunts to raise money for a research in his name.

Infinitely greater still, that included all the people he did know Melinda, his sons, his sister, the rest who set aside lives to do the most basic things for him, all while making sure he was an integral part of the family, not a patient.

"What has surprised me is that there can be anything apart from misery in this kind of existence," Gardner wrote to the congregation. "The remarkable aspect with my experience with ALS is how these others in my life, even those I don't know personally, have responded."

Jim Gardner died Saturday. He was 59.

The hope and faith he put into those millions of ice buckets last summer is some cold comfort.

But, man, ALS remains an awful thief.

Bangert is a columnist with the Journal & Courier. Contact him at dbangert@jconline.com. Follow on Twitter: @davebangert.

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Source: http://www.jconline.com/story/opinion/columnists/dave-bangert/2015/01/07/bangert-awful-thief-called-als/21418337/

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