Neuralstem Phase II Stem Cell Trial Appears Safe

Researchers at Neuralstem Inc. are investigating a potential ALS therapy that involves injecting stem cells into a person’s spinal cord. This week, the results of the phase II study that tested the safety of this type of stem cell transplantation were published in the journal Neurology. Below we give some background on this latest study.

A Father’s Dream Comes True Despite ALS

My name is Denise Hatfield, and I have been married almost 39 years to the love of my life. We share two beautiful sons, and two amazing grandsons. Rick and I are each others rock, and when he was diagnosed with ALS, my world turned upside down. For 39 years he has been by my side. He has always provided for me. I can’t imagine life without him in it. We do everything together and I will fight this battle to the end and then some. Hoping in his lifetime and in others, I can make a difference.

My husband is a mechanic for big rigs. In January of 2015 he started noticing his hands getting weaker at work. By June he was dropping things.

We took him to the doctor thinking it was maybe carpal tunnel, but I noticed the muscle mass loss in him and the difference in his speech. So we went to a neurologist. He was diagnosed with ALS early, but progressed fast. By October he was out of work.

For six years, he had been building a ’57 Chevy from the frame up. It had been his ultimate dream since he was a kid. So after being off work he decided he had to get his car finished. He worked as much as he could, but got weaker by the day. Our two sons helped around the house, when not working to help him finish this project. We finally sent the car out for a paint job. My husband was still driving but getting weaker every day.

Choice of Stem Cells Can Improve Therapeutic Potential

Stem cell therapy could represent an effective and comprehensive approach to treating ALS. Stefania Corti, M.D., Ph.D., Assistant Professor of Neurology at the University of Milan, and her colleagues set out to find ways to improve stem cell therapy. Their work, supported by The ALS Association, was published on June 6, 2016 in the journal Human Molecular Genetics.

Stem cells are cells that are capable of developing into different cell types, including neurons (brain cells) and glia (brain support cells). Not all stem cells are the same and choosing stem cell populations with specific desirable properties could in fact improve the therapeutic potential of stem cell therapy.

“Everyone has a story”

Craig Bower is the son of the late Clarence Bower, who is the father in this story. Craig wrote this piece “in hopes it may inspire others” and dedicates it to his father’s memory.

Stories of strength, survival, success and failure. Stories of “overcoming the odds” after a life-altering (insert horrible disease name here) medical diagnosis.

I have nothing against those stories. In fact, I’m continually amazed and inspired by them. They make me feel good and give me hope, which I think is why I read them in the first place. Most of the stories seem to summarize an amazing person that became a voice and advocate for their disease. They organized, researched, educated, shared—and most importantly, fought—until the very end leaving me (and others) amazed and inspired.

But some stories are amazing and inspiring for other reasons.

Photographer with ALS Captures Nature As He Sees It

Jim Dolan has always found pleasure in “wandering with a camera and capturing nature as I see it.” Despite being diagnosis with ALS in 2015, Jim continues to engage in his passion by photographing the beautiful natural environment around his Ohio home.

In addition to sharing his photographs, Jim is also generously helping the fight against ALS. The proceeds from every photo that Jim sells on his website will go to advancing the research, care services, and policy projects supported by The ALS Association.

Below are a few of Jim’s most recent photographs. To view more of his work, you can visit his website at http://jimadolan.com/.

Milton Safenowitz Postdoctoral Fellows Gather in NYC for ALS Association Workshop

On June 10, 2016, The ALS Association brought together the current and past awardees of its Milton Safenowitz Postdoctoral Fellowship to participate in a day-long research symposium and awards ceremony. The workshop was funded by The Greater New York Chapter of The ALS Association.

About the Award

The award was founded by the Safenowitz family through The Greater New York Chapter of The ALS Association. It is in memory of Mr. Safenowitz, who died of ALS in 1998. These awards are to encourage and facilitate promising young scientists to enter the ALS field. Fellows work with a senior mentor and receive extensive exposure to the ALS research community through meetings and presentations, like this workshop. After completing this fellowship, approximately 90 percent of the awardees stay in ALS research. They go on to establish their own laboratories to continue studying ALS and mentoring more ALS researchers along the way.  

Mitsubishi Tanabe Pharma Submits New Drug Application for Edaravone to Treat ALS in the US

Mitsubishi Tanabe Pharma Corporation (MTPC), which is locally based in Jersey City, N.J. with a head office in Osaka, Japan, announced yesterday that a New Drug Application has been submitted to the U.S. Food and Drug Administration (FDA) for edaravone (MCI-186) for the treatment of amyotrophic lateral sclerosis (ALS).

Those in the ALS community may have questions about what this means for them. Below we provide some background information on edaravone.

What is edaravone?

Edaravone is believed to act as a free radical scavenger, a compound that works by getting rid of toxic waste generated as a normal by-product of cell function. In ALS it is thought that these by-products are not as effectively removed and the compound may be neuroprotective by relieving the effects of this oxidative stress. The increase in oxidative stress is thought to damage motor neurons (cells that die in ALS).

Targeting Neuro-inflammation to Treat ALS

In 2011, The ALS Association, Wisconsin Chapter began supporting the work of Howard Weiner, M.D., Co-director of the Ann Romney Center for Neurologic Diseases, Brigham and Women’s Hospital around the role of inflammation in ALS. In March 2016, The ALS Association awarded a new Translational Research Advancing Therapy for ALS (TREAT ALSTM) Drug Development Contract  to Dr. Weiner and David Rodman, M.D. at miRagen Therapeutics to continue this work targeting inflammation in ALS.

The goal of their project is to accelerate a novel microRNA (miRNA) therapeutic approach to reduce neuro-inflammation, in order to bring it into clinic trials and meet a significant medical need in ALS. Below we provide some background on this exciting work.

“A Seat at the Table”

Before being diagnosed with ALS at age 28, Rachel Doboga loved her job teaching 5th grade English. Now, she advocates for a cure for ALS and write stories on her blog, “How I Live Now: Life With ALS.”  Through her writing, she hopes to increase ALS awareness and create a community for other people with ALS and their loved ones.

My parents and I live very far apart, but we visit as often as possible. Earlier in my disease, our reunions were more normal: catching up, visiting favorite restaurants, hanging out at the bookstore. We did the things we have always done when we got together. Now, one year after being diagnosed and two years into this disease, it’s getting harder to forget I am sick.

Bringing Brain Computer Interfaces Home

Dr. Andrew Geronimo is a talented young investigator using brain computer interface (BCI) technology to improve the lives of people living with ALS by enhancing their ability to communicate. He and his mentor Dr. Zachary Simmons, also at Penn

Geronimo.Andrew
Andrew Geronimo, Ph.D.

State Hershey Medical Center, have received a grant from The ALS Association to develop new opportunities for BCI technology. Together they work to train people living with ALS and their caregivers on how to setup and best use BCI in their homes through a telemedicine program. Through this work, they are continuously optimizing the BCI system.

“The ultimate goal for our research is make a difference now for people living with ALS. By listening to their needs and engineering a BCI to meet those needs,” stated Dr. Geronimo.

What is Brain Computer Interface (BCI)?

BCI is exactly what it sounds like: a direct connection between the brain and the computer. Normally, connections are made between the brain and muscles via motor neurons. Motor neurons in the brain relay a signal to the muscles to move.

In ALS, motor neurons are damaged and those connections break. This results in muscle weakness and eventually paralysis. BCI is revolutionary in that it makes up for that lost connection.