BrainStorm Hopes to Bring ALS Stem Cell Trial to US

by Amy Madsen on Fri, 2011-07-22 15:25

Early-stage planning is under way to add US trial sites to an ongoing Israeli stem cell trial in ALS

Biotechnology company BrainStorm Cell Therapeutics has announced plans to collaborate with two American institutions to test its experimental stem cell technology in people with amyotrophic lateral sclerosis (ALS) in the United States. The institutions are Massachusetts General Hospital in Boston and the University of Massachusetts Medical School in Worcester, Mass.

Although it’s still very early in the process, the company's goal is to extend the scope of its ongoing stem cell trial now being conducted in people with ALS in Jerusalem.

BrainStorm's phase 1-2 clinical trial is testing its NurOwn stem cell technology. The trial is sponsored by the Hadassah Medical Organization in Jerusalem, in collaboration with BrainStorm, which is based in New York and Petach Tikva, Israel.

Initiating steps for future collaboration

The three institutions have completed a "memorandum of understanding" agreeing to initiate the necessary steps to collaborate in testing BrainStorm's ALS stem cell therapy in people in the United States.

Those necessary steps include developing a trial design for the U.S.-based ALS clinical trials, selecting appropriate production facilities in the United States, and entering into a formal collaborative agreement.

Neurologist Merit Cudkowicz will lead the Massachusetts General Hospital team. Cudkowicz has received MDA research funding, is a member of MDA's translational research program advisory committee, and directs the MDA/ALS Center at Massachusetts General.

These are "early days" in the new agreement to work together, Cudkowicz said, involving mostly planning protocol and communications with the FDA.

The University of Massachusetts Medical School team will be led by Professor Robert Brown, a previous MDA grantee who preceded Cudkowicz as director of the Massachusetts General MDA/ALS Center. Brown currently directs the MDA clinic and MDA/ALS Center at the University of Massachusetts.

The new trials will require approval by the U.S. Food and Drug Administration (FDA), which granted NurOwn an orphan drug designation in February 2011. (Orphan drug status provides economic incentives for companies to develop drugs for rare diseases.)

BrainStorm trial will assess safety, then efficacy

In Israel, BrainStorm is testing its experimental therapy in 12 people with advanced ALS, and in 12 who still are in the early stages of the disease.

The technology 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.

About Clinical Trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur. MDA has no ability to influence who is chosen to participate in a clinical trial. To learn more, see Understanding Clinical Trials and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to www.clinicaltrials.gov.

The first trial participants — those with early-stage ALS — will receive a single treatment 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 study is designed to establish the safety of the NurOwn technology first; later, investigators will look for signs of any effects on the disease process.

An unrelated clinical trial of stem cells in ALS is currently being conducted at Emory University in Atlanta. Maryland-based biotherapeutics company Neuralstem has reported that the procedure appears safe in the first 12 participants a phase 1 trial. (See Safety Stem Cell Trial in ALS Set to Advance.)

The types of stem cells being used in these two trials are very different. The BrainStorm stem cells are taken from the bone marrow of trial participants, treated with NurOwn technology, and then returned to the patient. Stem cells being used in the Neuralstem trial are human neural cells derived and cultured from a single fetus. These cells are injected into varying locations in the spinal cord.

To participate in the trial in Israel

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 at clinicaltrials.gov.

You also may contact Dimitrios Karussis at karus@cc.huji.ac.il, or +972-2-6776939; or Adi Vaknin_Dembinsky at adembinsky@yahoo.com, or +972-2-6776939. Please refer to the clinicaltrials.gov identifier NCT01051882.

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