Stem cell stories that caught our eye: Lou Gehrig’s disease, misbehaving cells, and nanotech for brain cancer

Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun. 

Clinical trial in Lou Gehrig’s disease moves to Phase 2. It is always rewarding when you read about clinical trials in our field advancing. This is particularly true when the advance is for a uniformly fatal disease like amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease. Over the past couple of years hints of good news have come out about the earliest, so called Phase 1, trials using adult nerve stem cells from the company Neuralstem. Those trials, principally conducted at Emory University in Atlanta and the University of Michigan showed the fetal-derived stem cells are safe.

But in that phase, the researchers used a low dose of cells and injected them in the lower back portion of the spine. In the new phase, the dose of cells will escalate to more than 10 times the dose used in Phase 1, and the cells will be injected in the neck section of the spine in most of the patients. It is that portion of the spine where the cells have the best chance of preserving breathing and improving the quality of life and longevity of these patients. The press release from the company announcing that the first patient had been treated was widely picked up online, including here at The Business Journals.

CIRM funds a team at Cedars-Sinai to develop an ALS therapy that genetically modifies stem cells to produce a protein shown to protect nerves from damage. That team will be using the same complex surgical device for delivering the cells precisely to the spine that was developed for the Neuralstem trial. You can read about that work here.

Industrial scale disease-in-a-dish for Lou Gehrig’s. One of the exciting things about the stem cell field is that it often offers multiple routes to potential therapies. My colleagues and I have written frequently about reprogramming skin cells from patients into iPS type stem cells to create cells in a dish that mimic the disease because they have the same genes as the patient. Now, my former colleagues at the Harvard Stem Cell Institute have convinced a company that these disease-in-a-dish cells are worth investing in to look for drugs to treat Lou Gehrig’s disease (or ALS). The company Evotec plans to use cells created by the Harvard team to conduct industrial scale screening for potential targets, things that are different about the ALS cells, and potential therapies that can alter those targets.

Misbehaving cells as cause of disease. In today’s issue of the journal Science a team from New York’s Mount Sinai Medical Center proposes a new theory for the root cause of disease. They suggest that evolution has programmed our genes to tell cells to cooperate with each other. But when those cooperation genes are mutated or switched off, cells become competitive and that leads to disease. They site the classic example of the cancer suppressor gene p53. When it is not working right, cells misbehave and cancer results. To shore up their argument the researchers conducted a genetic screen in stem cells to look for mutations that result in lack of cooperation. They picked up about 100 genes. The paper represents a first step in an interesting new theory of disease. The web site Science Daily wrote about the work here.

Nano tech enabled stem cell delivery of cancer therapy. The field of nanotechnology—think very very small particles—is even newer than stem cell science. But researchers are increasingly finding ways to marry the two. Last week we wrote about nanotubes filled with an agent that allows tracking of stem cells after transplant. This week a team at the University of Chicago presented data at the American Chemical Society (ACS) meeting on using nanoparticles to load a chemotherapy agent into stem cells. Those nerve stem cells were then injected into mice with brain tumors and the cells and their payload were able to eradicate the tumors. The project takes advantage of the fact that stem cells home to inflammation and they view tumors as inflammation. This allows very targeted delivery of potentially toxic therapies. Chemical and Engineering News wrote about the presentation here. [C&EN is published by the chemical society, where I helped to found one of the first consumer science magazines SciQuest, which was bought up by Discover many moons ago, but I still like keeping track of what folks back there are writing.]

CIRM funds a team at City of Hope in Duarte, California that is also using nerve stem cells to deliver brain cancer therapy. You can read about that project here.

Don Gibbons

One thought on “Stem cell stories that caught our eye: Lou Gehrig’s disease, misbehaving cells, and nanotech for brain cancer

  1. Protein injection holds promise for muscular dystrophy treatment
    Dated: 2013-09-18

    ISLAMABAD: Injecting a novel human protein into muscle affected by Duchenne muscular dystrophy significantly increases its size and strength, scientists have discovered.

    The findings could lead to a therapy akin to the use of insulin by diabetics.

    The study was conducted by Dr. Julia von Maltzahn and Dr. Michael Rudnicki, the Ottawa scientist who discovered muscle stem cells in adults.

    “This is an unprecedented and dramatic restoration in muscle strength,” said Dr. Rudnicki, a senior scientist and director for the Regenerative Medicine Program and Sprott Centre for Stem Cell Research at the Ottawa Hospital Research Institute.

    He is also a Canada Research Chair in Molecular Genetics and professor in the Faculty of Medicine at the University of Ottawa.

    “We know from our previous work that this protein, called Wnt7a, promotes the growth and repair of healthy muscle tissue. In this study we show the same types of improvement in a mouse model of Duchenne muscular dystrophy. We found that Wnt7a injections increased muscle strength almost two-fold, to nearly normal levels. We also found that the size of the muscle fibre increased and there was less muscle damage, compared to mice not given Wnt7a,” he explained.

    Duchenne muscular dystrophy is a genetic disorder that affects one of every 3,500 newborn males. In Canada, all types of muscular dystrophy affect more than 50,000 people. The disease often progresses to a state where the muscles are so depleted that the person dies due to an inability to breath. For people with Duchenne muscular dystrophy, this usually happens in their 20s or 30s.

    “This is also exciting because we think it’s a therapeutic approach that could apply to other muscle-wasting diseases,” said Dr. Rudnicki.
    These results were published in the Proceedings of the National Academy of Sciences.

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