Welcome to our new “Throwback Thursday” (TBT) series. CIRM’s Stem Cellar blog has a rich archive of stem cell content that is too valuable to let dust bunnies take over. So we decided to brush off some of our older, juicy stories and see what advancements in stem cell research science have been made since!
This week, we’ll discuss an aggressive neurodegenerative disease called Amyotrophic Lateral Sclerosis or ALS. You’re probably more familiar with its other name, Lou Gehrig’s disease. Gehrig was a famous American Major League baseball player who took the New York Yankees to six world championships. He had a gloriously successful career that was sadly cut short by ALS. Post diagnosis, Gehrig’s physical performance quickly deteriorated, and he had to retire from a sport for which he was considered an American hero. He passed away only a year later, at the young age of 37, after he succumbed to complications caused by ALS.
A year ago, we published an interesting blog on this topic. Let’s turn back the clock and take a look at what happened in ALS research in 2014.
TBT: Disease in a Dish – Using Human Stem Cells to Find ALS Treatments
This blog featured the first of our scintillating “Stem Cells in Your face” video series called “Treating ALS with a Disease in a Dish.” Here is an excerpt:
Our latest video Disease in a Dish: That’s a Mouthful takes a lighthearted approach to help clear up any head scratching over this phrase. Although it’s injected with humor, the video focuses on a dreadful disease: amyotrophic lateral sclerosis (ALS). Also known as Lou Gehrig’s disease, it’s a disorder in which nerve cells that control muscle movement die. There are no effective treatments and it’s always fatal, usually within 3 to 5 years after diagnosis.
To explain disease in a dish, the video summarizes a Science Translation Medicine publication of CIRM-funded research reported by the laboratory of Robert Baloh, M.D., Ph.D., director of Cedars-Sinai’s multidisciplinary ALS Program. In the study, skin cells from patients with an inherited form of ALS were used to create nerve cells in a petri dish that exhibit the same genetic defects found in the neurons of ALS patients. With this disease in a dish, the team identified a possible cause of the disease: the cells overproduce molecules causing a toxic buildup that affects neuron function. The researchers devised a way to block the toxic buildup, which may point to a new therapeutic strategy.
New Stem Cell Discoveries in ALS Make Progress to Finding a Cure
So what’s happened in the field of ALS research in the past year? I’m happy to report that a lot has been accomplished to better understand this disease and to develop potential cures! Here are a few highlights that we felt were worth mentioning:
Ice Bucket Challenge. The ALS Association launched the “world’s largest global social media phenomenon” by encouraging brave individuals to dump ice-cold water on their heads to raise awareness and funds for research into treatments and cures for ALS. This August, the ALS Association re-launched the Ice Bucket Challenge campaign in efforts to raise additional funds and to make this an annual event.
- ALS Gene Mapping. In a story released yesterday, the global biotech company Biogen is partnering with Columbia University Medical Center to map ALS disease genes. An article from Bloomberg Business describes how using Ice Bucket Money to create “a genetic map of the disease may help reveal the secrets of a disorder that’s not well understood, including how much a person’s genes contribute to the likelihood of developing ALS.” Biogen is also launching a clinical trial for a new ALS drug candidate by the end of the year.
- New Drug target for ALS. Our next door neighbors at the Gladstone Institutes here in San Francisco published an exciting new finding in the journal PNAS in June. In collaboration with scientists at the University of Michigan, they discovered a new therapeutic target for ALS. They found that a protein called hUPF1 was able to protect brain cells from ALS-induced death by preventing the accumulation of toxic proteins in these cells. In a Gladstone press release, senior author Steve Finkbeiner said, “This is the first time we’ve been able to link this natural monitoring system to neurodegenerative disease. Leveraging this system could be a strategic therapeutic target for diseases like ALS and frontotemporal dementia.”
- Stem cells, ALS, and clinical trials. Clive Svendsen at Cedars-Sinai is using gene therapy and stem cells to develop a cure for ALS. His team is currently working in mice to determine the safety and effectiveness of the treatment, but they hope to move into clinical trials with humans by the end of the year. For more details, check out our blog Genes + Cells: Stem Cells deliver genes as drugs and hope for ALS.
These are only a few of the exciting and promising stories that have come out in the past year. It’s encouraging and comforting to see, however, that progress towards a cure for ALS is definitely moving forward.