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Embryonic Stem Cell Research for Amyotrophic Lateral Sclerosis (ALS)

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ALS is often called Lou Gehrig's disease, but the face of ALS today is Stephen Hawking. He's the wheelchair-bound British astrophysicist who has lived with the crippling disease for almost 50 years.

He got married, had children, and rose to international fame as a professor at Cambridge after his diagnosis. His web site, http://hawking.org.uk, chronicles the life of this remarkable man. My favorite part is the photo: he can still smile.

The life expectancy for most ALS patients is less than 5 years. What makes Hawking so different? Age at diagnosis, support from family and friends, personal drive, and Hawking's rich intellectual life are all possible factors.

Researchers are trying to identify drugs to slow or even stop the progression of ALS. A group at Harvard Medical School has identified a gene that shows loss-of-function mutations in ALS patients. This opens the possibility of testing drugs on cell cultures that contain the target gene.

Enter the team that cloned Dolly the sheep. This group in Edinburgh, UK, discovered that an extract of embryonic stem cells activates genes for cell division in adult cells. They hope to develop the technology to take a few cells from a patient and efficiently produce an unlimited supply of genetically identical cells for research.

Stephen Hawking has spent the last 50 years studying the mysteries of physics and astronomy. There is hope that within our lifetime medical research will give all ALS patients the prospect of 50 years before the disease becomes their biggest problem.


Wilmut I, Sullivan G, Taylor J. A decade of progress since the birth of Dolly. Reprod Fertil Dev. 2009;21(1):95-100.
Kishikawa H, Wu D, Hu GF. Targeting angiogenin in therapy of amyotropic lateral sclerosis. Expert Opin Ther Targets. 2008 Oct;12(10):1229-42.

Add a Comment5 Comments

EmpowHER Guest

I will die soon because I don't have what Hawking has --

money, caregivers, family and social support, assisted technology

October 11, 2010 - 3:08pm

I am amazed at how many people have told me they know of someone who has (or had) ALS. When I was in college and first heard about Stephen Hawking, I thought ALS was a very rare disease. Maybe too many pALS (people with ALS) are hidden away because of the negative feelings about disability in our society. Thank you, Anonymous, for your comment. Your life is still valuable to all of us.

June 18, 2009 - 12:52pm
EmpowHER Guest

Let me state right up front that I have ALS!

It's not 100% about how deeply you want to live, obviously, or there would be many more survivors. That being said, you can find a certain amount of people with ALS that live longer because of their attitude.

Stephen Hawkings made use of every technology available. He breathes through a trach and receives nourishment through a feeding tube. If everyone with ALS had these procedures there would be a lot more of us around. Most pALS (people with ALS) still decide not to have a trach installed. Why? Don't want to live disabled, don't want to burden their familes......

I think a big part of the dialogue is our collective negative feelings about being disabled. Stephen Hawkings didn't think like that (he's brilliant) and that's why he's alive today.

Tessa, pALS

June 11, 2009 - 4:47am
EmpowHER Guest

The explanations for Hawking's unusually long survival provided here are completely speculative. There is no reason to believe any of them.
The underlying mechanism of ALS is still not understood, beyond the fundamental functional issue - muscle mass deteriorates in sync with loss of nerological function. Suggestions as to why one patient falls within the norm of survival probabilities and another is an outlier are not supported by any clinical work thus far, and runs the risk of blaming the victim.

June 10, 2009 - 7:37pm
(reply to Anonymous)

I understand. Stephen Hawking had a strong interest in theoretical physics before his diagnosis, so he was able to pursue the career he wanted even with his disability. At the other end of the spectrum, Lou Gehrig had no chance to continue his career as a professional athlete. I think ALS should receive high priority for medical research because it devastates so many lives.

June 11, 2009 - 10:09am
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