Thursday, January 26, 2012

Stem Cell Developments

Via the MDA...

Preliminary data reported Jan. 17, 2012, by BrainStorm Cell Therapeutics demonstrate that the biotechnology company's experimental NurOwn stem cell technology has not caused any significant adverse side effects in a phase 1-2 clinical trial in amyotrophic lateral sclerosis (ALS). In addition, clinical follow-up of trial participants indicates that the treatment appears to have improved breathing, swallowing and muscle strength.

"I am very excited about the safety results, as well as these indications of efficacy we are seeing," the trial's principal investigator Dimitrios Karussis said in a BrainStorm press release. “This may represent the biggest hope in this field of degenerative diseases like ALS."

The ethical and safety committee at Hadassah Hebrew University Medical Center (Israel), where the trial is taking place, has granted BrainStorm approval to advance its trial and begin treatment in the next group of participants.

Technology uses participants' own cells

The newly reported data are based on results from the first four trial participants, who were in the early stages of ALS. Investigators expect to test BrainStorm's NurOwn therapy in eight more people with early-stage ALS, and in 12 people with advanced ALS.

Treatment for the first 12 trial participants involves a single round of multiple injections to the biceps and triceps muscles. Those in the later groups, with more advanced ALS, will receive a single injection into the cerebrospinal fluid via lumbar (lower back) puncture.

The experimental treatment in BrainStorm's trial uses mesenchymal stem cells, taken from the bone marrow, which are capable of differentiating (maturing) into a number of different cell types. In this trial, mesenchymal stem cells are taken from trial participants’ bone marrow, cultured into healthy cells capable of delivering neurotrophic factors (molecules that support motor neurons, the nerve cells that die in ALS) and then readministered to the participants from whom they were taken.

BrainStorm, with operations in both New York and Petach Tikyah, Israel, was granted orphan drug status for its experimental therapy in February 2011 by the U.S. Food and Drug Administration (FDA). (Orphan drug status provides economic incentives for companies to develop drugs for rare diseases.)

BrainStorm plans to add US trial sites

BrainStorm is in early-stage planning to add U.S. trial sites to the ongoing NurOwn clinical trial. Prospective sites include Massachusetts General Hospital in Boston and the University of Massachusetts Medical School in Worcester, Mass.

Neurologist Merit Cudkowicz will lead the Massachusetts General Hospital team. Cudkowicz has received MDA research funding and directs the MDA/ALS Center at Massachusetts General. The University of Massachusetts Medical School team will be led by Robert Brown, a neurologist and former MDA research grantee who preceded Cudkowicz as director of the MDA/ALS Center at Massachusetts General.

Several steps are necessary before NurOwn stem cells can be tested in the United States. These include:
  • development of a trial design;
  • identification of treatment production facilities;
  • completion of a formal collaborative agreement between BrainStorm and the participating institutions; and
  • FDA approval of the new trials.  

Learn more about the NurOwn trial

To find out more about the ongoing trial in Israel, view Autologous Cultured Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF) in ALS Patients (or enter NCT01051882 into the search box).

You also may contact Dimitrios Karussis at, or +972-2-6776939; or Adi Vaknin_Dembinsky at, or +972-2-6776939.


  1. BrainStorm! What a great name for a company doing this research.

  2. I heartily agree!