U-M creates new stem cell lines for CMT

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U-M creates new stem cell lines

Embryonic tools will boost research into inherited diseases

Kim Kozlowski / The Detroit News

http://www.detnews.com/article/20110404/LIFESTYLE03/104040337/U-M-creates-new-st\

em-cell-lines

Ann Arbor— A collaboration between the University of Michigan and a renowned

Detroit doctor has led to new embryonic stem cell lines that will allow

researchers to study how certain diseases form and progress, officials will

announce today.

Unlike Michigan's first stem cell line created in October, the two new stem cell

lines include genes for inherited disorders.

These are a milestone because they will give researchers tools they have never

had before to study and possibly find ways to delay, better treat or even cure

diseases.

One of U-M's new stem lines carries the genetic mutation that causes hemophilia

B, a hereditary condition in which the blood does not clot properly. The other

carries genes for Charcot-Marie-Tooth disease, another inherited disorder

leading to degeneration of muscles in the foot, lower leg and hand.

The development puts U-M in the forefront of research that some regard as

unethical, others as life-saving.

Only a few other U.S. universities have created disease-specific stem cell

lines. Michigan could also become a world leader, some say, since the Ann

Arbor-Detroit collaboration has the potential to lead to many more

disease-affected stem cell lines.

" These cells aren't necessarily going to drive us toward discoveries of stem

cell replacement therapies, but will give us information we've never had before

with regards to how the diseases form and progress, " said Dr. ,

co-director of the U-M Consortium for Stem Cell Therapies.

" By knowing that, we — and others — can work toward cures and treatments in ways

we haven't been able to before. Once you have a new tool in science, it really

opens up an avenue for many, many different discoveries. It's not just discovery

by our laboratories at the University of Michigan, but discoveries from people

across the world. "

Scientists were able to create the new stem cell lines because of the 2008

voter-approved Michigan constitutional amendment, and embryo donations

facilitated by Dr. Mark , president of Genesis Genetics in Detroit.

is a pioneer in pre-implantation genetic diagnosis, a test that helps

couples have a healthy baby if they have had a child with an inherited genetic

disorder.

The test requires all couples to go through in vitro fertilization — a

reproductive technology normally used by infertile couples wanting to have

children by creating embryos in a petri dish. Before the embryos are implanted

in the mother, tests one cell in each to see if they're at risk of

carrying genetic mutations responsible for inherited diseases.

Embryos that test positive for genetic abnormalities typically have been

discarded as medical waste since couples do not want to use them. But now

is asking couples if they want to donate them to science.

" We are interested in curing these dastardly diseases that affect mostly

children and are generally incurable, " said , who also works with Stanford

University in a similar arrangement. " Current health care is barely capable of

even effectively treating most of these conditions.

" At the moment, the best one can do is use medical technology to assist

high-risk couples in avoiding in future children the disease that afflicts their

family. 'Cure' is the mantra. And for many of these pediatric conditions, stem

cell science offers the best hope at present to achieving that goal. "

Some are disturbed by the technology that will help U-M further its research.

" It sounds like a step toward eliminating imperfect children, " said Dave

Maluchnik, spokesman for the Michigan Catholic Conference. " It's frankly quite

frightening. "

Added Ed Rivet, legislative director for Right to Life of Michigan: " If you look

at the history of ethical research, we don't destroy an individual with a

disease in order to find cure for the disease.

" If it's human enough to know that it has the disease, it must be the same human

beings that are born with the disease. "

But those who are suffering from diseases or spinal cord injuries were thrilled

to hear of the new stem cell lines, and impressed by the swift progress of U-M's

research.

" It's encouraging and makes me hopeful, " said , a 22-year-old

Livonia woman who was paralyzed from the neck down at age 14 and now relies on a

wheelchair and ventilator. " This is a first stepping stone. "

Supporters of the research say U-M's collaboration with Genesis Genetics has

enormous potential, since it tests for nearly 200 inherited genetic diseases

with fertility clinics around the globe.

Ten days ago, the university received a Michigan couple's donated embryos

carrying genes responsible for myotonic dystrophy, a disorder characterized by

progressive muscle wasting.

The university is in the process of gaining consent with 20 couples from across

the U.S. who want to donate their embryos with other genetic abnormalities.

This could put Michigan in the forefront of stem cell research, said Bernard

Siegel, executive director of the Florida-based Genetics Policy Institute, which

coordinates the World Stem Cell Summit, held last year in Detroit.

It also buttresses Michigan voters' passage of Proposal 2, a constitutional

amendment that allowed for embryonic stem cell research in Michigan and has been

fought unsuccessfully.

" The passage of Proposal 2 wasn't just a political statement, " Siegel said.

" This has been followed up with real, tangible research and real results that

have the potential to impact human health. It portends very well for the future

of stem cell research in Michigan. "

U-M will soon be submitting these disease-specific lines to the National

Institutes of Health to be placed on the Human Embryonic Stem Cell Registry.

Researchers across the country will be able to use the lines for federally

funded research. Of the 91 lines currently on the registry, three are

disease-specific stem cell lines submitted by Harvard and Stanford universities.

" This is an important day for Michigan, " A. Alfred Taubman, founder of a U-M

medical research institute in his name, said last week from his home in Palm

Beach, Fla.

" This puts us in the very forefront of science. We're (among) the very first to

create embryonic stem cell lines that contain genetic defects that lead to

specific inherited diseases. For the patients suffering from these diseases and

the doctors trying to treat them, this is a major step forward. It can help us

understand what causes these diseases and how they progress. But more

importantly, these stem cell lines should pave the way for new treatments and

cures. "

From The Detroit News:

http://detnews.com/article/20110404/LIFESTYLE03/104040337/U-M-creates-new-stem-c\

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