Stem Cell Research

Stem Cell Therapies For ALS, or Lou Gehrig’s disease: The Problem And New Clinical Trials

Two of our Stem Cell Sector companies, Neuralstem (CUR) and Brainstorm Cell Therapeutics (BCLI.OB) are in clinical trials with stem cell therapies for Amyotrophic lateral sclerosis (ALS), also known as Lou Gherig's disease. Neuralstem (CUR), market cap $60M, began its ALS trial in 2008 in the United States.

BrainStorm, market cap $57M, is the first company to receive approval from the Israeli Ministry of Health (MOH) for a differentiated stem cell therapy and approval of its Phase I/II clinical trial utilizing its proprietary NurOwn™ treatment for patients with Amyotrophic Lateral Sclerosis (ALS), often referred to as Lou Gehrig's Disease. BrainStorm's trial effectively began in Isreal on June 6,2011.

On July 19, 2011, BrainStorm announced it will partner with UMass Medical School and its chair of neurology, Robert H. Brown Jr., DPhil, MD, in anticipation of applying for FDA approval to begin amyotrophic lateral sclerosis (ALS) human clinical trials in the United States. The trial, which will be conducted at Massachusetts General Hospital, will investigate the use of the company’s NurOwn treatment for ALS.

We've included a video talk by Fred Gage of the Salk Institute on the subject of how ALS works in the body.

New Cedars-Sinai Regenerative Medicine Institute to Emphasize Induced Pluripotent Research

The Cedars Institute will be housed in new state-of-the-art laboratories being constructed especially for stem cell and regenerative medicine research. At the heart of the Institute will be a specialized core facility for the production of Induced pluripotent stem cells.

Clive N. Svendsen, Jamie Thomson associate and joint leader of the widely-respected Stem Cell and Regenerative Medicine Center at the University of Wisconsin, has been named to lead the new unit.  He has quite a resume; one that includes professor of neurology and anatomy at the University of Wisconsin-Madison, and consulting professor at Stanford University. Svendsen’s research focuses on both modeling and treating neurodegenerative disorders such as amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and Parkinson’s disease using a combination of stem cells and growth factors.

Medicine's New Toolbox: MIT's View Of Induced Pluripotency's Future

"The past two years have been nothing short of a revolution," says John Dimos, a senior scientist at iZumi Bio. "These cells didn't really exist two years ago. This is all brand-new technology, and it's opening up the potential for brand-new science." The company John is with, Izumi Bio, plans to take advantage of that potential by developing a bank of iPS cells from patients with various diseases and using the cells to screen candidates for drug development.

As a postdoc at Harvard, John Dimos built a cellular model of ALS (amyotrophic lateral sclerosis - Lou Gherig's Disease), making it possible to study a neurodegenerative disease outside an animal for the first time. He and his colleagues collected skin cells from an 82-year-old woman with ALS, reprogrammed them into iPS cells, and directed the cells to differentiate into motor neurons that were genetically identical to the donor's defective cells. "It was the first paper to show that you can use a stem cell to see disease pathology in a petri dish," says Douglas Melton, codirector of the Harvard Stem Cell Institute. "That means you can now study diseases in petri dishes and not in people. That's huge."

Motor Neurons Produced From Skin Cells Of Elderly Patients With Lou Gherig's Disease

Human Induced Pluripotent Stem Cells (iPS) came into being for the first time in November 2007, opening the metaphorical flood gates on a large and potentially fertile valley of stem cell research and regenerative medicine.  Stem cell pioneer, James Thomson, head of one of the two teams that independently produced human iPS cells, predicted the primary use of the reprogrammed cells would be for laboratory research on human disease.

Now, eight months after the initial proof of concept for human iPS cells, three major steps have been taken toward Thomson's predicted use: First, researchers from the Harvard Stem Cell Institute and Columbia University have turned human skin cells from patients with Amyotrophic lateral sclerosis (ALS - Lou Gherig's disease) into induced pluripotent stem (iPS) cells.  Second, they then directed the differentiation of these created iPS cells into the type of motor neurons (nerve cells) destroyed by (ALS). Third, they did steps one and two using the cells of elderly patients.