Stem Cell Research

Transplanted Neural Stem Cells Treat ALS In Mouse Model

Eleven independent studies show that transplanting neural stem cells into the spinal cord of an ALS mouse model slows disease onset and progression, improves motor function, and significantly prolongs survival.

The transplanted neural stem cells did not benefit ALS mice by replacing deteriorating nerve cells. Instead, neural stem cells help by producing factors that preserve the health and function of the host’s remaining nerve cells. They also reduce inflammation and suppress the number of disease-causing cells in the host’s spinal cord.

Read further in the Sanford Burnham Medical Research Institute announcement.

Note: Neuralstem Inc. (CUR), one of our Sector Companies, is preparing for a phase II clinical trial involving transplanted neural stem cells for ALS.

Stem Cell Sector, Neuralstem's (CUR), Short Term Bet On A Molecular Cure For Depression

Neuralstem's technology enables the isolation and large-scale expansion of human neural stem cells from the developing human brain and spinal cord. In addition, the company has developed and patented a series of small molecule compounds -- low molecular weight organic compounds which can efficiently cross the blood/brain barrier -- they are wagering will stimulate the growth of new neurons in the hippocampus and provide a treatment for depression. If they're right, they and their shareholders win big. If they're wrong they'll be in the market for capital and they'll be there with less leverage than they have now.

The company has also developed a Spinal Cord Delivery Platform and Floating Cannula that has been demonstrated to deliver therapeutic agents to the spinal cord safely and effectively, a technology they have just licensed to Salt Lake City-based Q Therapeutics.

Nueralstem's recent venture into the capital markets was small, notable only for the low price of the offering. In mid-April Nueralstem (CUR) sold at $1.07 the day they announced the retirement of their CFO. In the following four months, which included announcements that additional patients had been dosed in their ALS clinical trial, that the first patient had been treated in their depression trial, the beginning of Zack's coverage of the firm with an 'Outperform' rating, and the receipt of a patent on the "Transplantation of Human Neural Cells for Treatment of

Astrocytes Control Generation Of New Neurons From Neural Stem Cells

New research has shown how astrocytes control the generation of new neurons in the brain. Earlier research in the same laboratory demonstrated that astrocytes reduce brain tissue damage after stroke and that the integration of transplanted neural stem cells can be largely improved by modulating the activity of astrocytes.

“In the brain, astrocytes control how many new neurons are formed from neural stem cells and survive to integrate into the existing neuronal networks, said Milos Pekny, Professor and Laboratory Director, Center for Brain Repair and Rehabilitation, Dept. of Clinical Neuroscience and Rehabilitation, University of Gothenburg. "Astrocytes do this by secreting specific molecules but also by much less understood direct cell-cell interactions with stem cells.”

CIRM Provides $17.8 Million Grant To Develop Combined Stem Cell And Gene Lou Gehrig’s Disease (ALS) Treatment

Stem cells alone have the potential to protect damaged motor neurons in ALS. This potential increases when combined with the additional known effects of  the protein GDNF which promotes the survival of nuerons. Currently there is no therapy that will cure or slow the progress of amyotrophic lateral sclerosis (Lou Gehrig's disease).

In the past, delivering GDNF to the brain or spinal cord has been nearly impossible because it does not cross from the blood to the tissue of the spinal cord.  Cedars believes they have a way to overcome this.

“We’re looking at a novel and exciting way of using stem cells as ‘Trojan horses’ that arrive at the sick motor neurons and delivers the protein exactly where it’s needed,” Clive Svendsen, PhD, director of the Regenerative Medicine Institute,. “Our early study indicates this approach has significant potential and we’re excited to bring this treatment a step closer to helping ALS patients.”

Blood-Brain Barrier Building Blocks Generated From Human Stem Cells

The blood-brain barrier -- the filter that governs what can and cannot come into contact with the mammalian brain -- is a marvel of nature. It effectively separates circulating blood from the fluid that bathes the brain, and it keeps out bacteria, viruses and other agents that could damage it.

However, the barrier can be disrupted by disease, stroke and multiple sclerosis, for example, and also is a big challenge for medicine, as it can be difficult or impossible to get therapeutic molecules through the barrier to treat neurological disorders.

Now researchers have transformed stem cells into endothelial cells with blood-brain barrier qualities in a laboratory dish in an attempt to get the blood-brain barrier to share its secrets.

“The nice thing about deriving endothelial cells from induced pluripotent stem cells is that you can make disease-specific models of brain tissue that incorporate the blood-brain barrier,” explains Sean Palecek, a UW-Madison professor of chemical and biological engineering. “The cells you create will carry the genetic information of the condition you want to study.”

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.