Blood-forming stem cells treat advanced stage of MS

A group in Greece has found that transplants of blood-forming stem cells in the bone marrow can treat some patients with multiple sclerosis. That work, published in the journal Neurology, could one day help the 400,000 Americans and 2.1 million people worldwide have MS (from the National MS Society).

The key here is "one day." Stem cell scientists have long listed MS, along with a variety of other autoimmune diseases such as lupus, as a likely candidate for treatment by blood-forming stem cells. The problem is that the transplants are extremely risky. Case in point, in the Greek study two of the 35 patients died from transplant-related complications.

The idea behind why the transplant could treat autoimmune diseases is simple. In diseases such as MS or lupus, the immune cells that are suppose to fight off infections instead begin attacking the body's own tissues. In the case of MS, they attack the lining of neurons in the spinal cord and brain. Without their protective insulation the neurons can't effectively transmit signals instructing the body to move.

Swap out the defective immune system with a fresh one and the person would be cured, right?

The problem so far has been in getting rid of the existing blood-forming system. In a bone marrow or blood-forming stem cell transplant, first the doctors must destroy a person's existing immune system with strong chemotherapy or radiation. This step is extremely risky. That's why a HealthDay story on this work quotes Aaron Miller, chief medical officer for the National Multiple Sclerosis Society and a professor of neurology at Mount Sinai School of Medicine in New York City as having doubts about this technique's widespread use:

This is a very heroic form of therapy for multiple sclerosis [MS], which is unlikely, in my view, ever to have a major impact on the field," added Miller. "It's a substantially risky therapy -- the mortality rates have been in the 2-3 percent range . . . and it's hugely expensive.

Many groups are working on less toxic ways of clearing the patient's problematic blood-forming system. You can read about some of those attempts in a WebMD story about the MS trial. If they are successful, this approach could become less risky and offer an effective way of treating not just MS but all autoimmune diseases. That would be something to celebrate.

Here's a list of CIRM awards targeting MS,  and our MS disease information page.

- A.A.

Stem cell progress on brain awareness week

This week marks Brain Awareness Week, with events worldwide to bring people up to speed on brain research. I went to the cool search tool on the Dana Foundation web site and found that several CIRM grantees are hosting events this week. That makes sense, given that roughly a quarter of our funding goes to neuronal diseases. (You can see charts of CIRM stem cell research funding allocations here. The charts are slightly out of date — stay tuned for some updates in the next month.)

Brain diseases are seen as a big challenge for stem cell therapies, in part because the brain itself is such a complex web of neurons. Simply replacing a few lost neurons won't necessarily replicate the lost connections. We have a story discussing some of those issues and describint innovative approaches CIRM grantees are taking to developing new cures for brain diseases.

The good news is that some CIRM grantees are learning that stem cells can be coaxed to form the support cells in the brain that nourish neurons. These support cells could be what provide a therapy for diseases such as ALS, MS, stroke and spinal cord injury. Other grantees are using stem cells in the lab to test new drugs for Parkinson's disease.

A group at UC Davis is attempting to use the body's own mesenchymal stem cells to preserve unaffected neurons in people with Huntington's disease. This technique won't bring back lost cells, but saving additional cells from dying off could prevent some of the terrible side effects of the disease.

Another team of CIRM grantees at UC Irvine found that at least in rodents, stem cells were able to repair some memory loss due to Alzheimer's disease. This work is a long way from treating humans, but still provides hope for people who have lost loved ones to this devastating disease. Here's a video we produced about that work:



We've produced several other videos about CIRM's brain related research:

• Fred H. Gage talks about using embryonic stem cells to model neuronal disease
• Spinal Cord Injury: Progress and Promise in Stem Cell Research
• Huntington's Disease: Progress and Promise in Stem Cell Research
• Parkinson's Disease: Progress and Promise in Stem Cell Research
• Spotlight on ALS seminar
• Spotlight on Huntington's disease seminar
• Spotlight on Alzheimer's disease seminar
• Spotlight on Batten disease seminar
• Spotlight on Parkinson's disease seminar

 - A.A.

Mysteries of stem cell migration revealed

CIRM-funded Researchers at the University of California, Irvine published an interesting paper this week that helps explain one mystery — how do transplanted stem cells go to the right place? This is an important issue for diseases such as multiple sclerosis, where transplanted stem cells would have to navigate to the damaged nerves.

In a press release, senior author Thomas Lane (shown in photo) said:

"Previously, we've seen that adult neural stem cells injected into the spinal column knew, amazingly, exactly where to go. We wanted to find what directed them to the right injury spots."

What the team found is that in mice with an induced form of MS, transplanted neural stem cells responded to signals being sent by inflammatory cells at the site of the damage. The neural stem cells responded to those signals by migrating to the right place and maturing into a type of nerve cell called an oligodendracyte, which could help heel the disease.

According to the press release, three weeks after the initial treatment, 90 percent of the cells had grown into fully formed oligodendrocytes.

Proceedings of the National Academy of Sciences, Online Edition, June 1
CIRM Funding: Kevin Carbajal and Christopher Shaumburg (T1-00008)

A.A.

Embryonic stem cells repair nerve damage from mutiple sclerosis in mice

Researchers at the University of California, Irvine have found that neurons derived from  embryonic stem cells were able to repair some damage in a mouse model of multiple sclerosis. In people with MS, the immune system attacks the insulation – called myelin – that covers and protects neurons of the brain and spinal cord. The transplanted cells caused a response in the animals that allowed the myelin coating to be repaired on damaged cells. In humans, repairing the myelin would likely also repair the function of those nerves, bringing back feeling and motor control in people with MS. At this time there are no therapies to repair this damage. Instead, available drugs simply slow the progression of the disease. In this early study, the transplanted neurons survived only two weeks. The authors say more work is needed to understand how the remyelination occurred and how to retain the transplanted cells.

Journal of Neuroimmunology: May 23, 2009 (online)
CIRM funding: Chris Shaumberg (T1-00008), Thomas Lane (RS1-0409)

Related Information: University of California, Irvine