The Great Potential of Stem Cells

Science was good to us in the last century: 100 years ago we learned to put patients to sleep for surgery and then reliably wake them up afterwards; 60 years ago we started producing antibiotics; the last 30 years heralded a better understanding of genetics, cancer and heart disease, as well as some new therapies.

Nevertheless, many of our treatments remain primitive and I suspect that 60 years from now, doctors will view our practices in a similar manner to how we viewed the pre antibiotic age, or a time when the lucky patients had a bottle of whiskey for surgical anesthesia. In caring for chronic diseases, targeting therapies, and regenerating damaged tissues, we still have much to learn. Today's presidential order, lifting the ban on using new embryonic stem cell lines for research may help.

Human Embryonic Stem Cells (aka ESCs) have tremendous potential. Unlike other cells they can potentially be "programmed" to replace any cell in the body." The President's decree will allow NIH funded researchers (usually our nation's best) the opportunity to conduct important research with these promising cells.

After the use of human ESCs was banned in 2001, many prominent researchers could no longer pursue this work if they wanted to receive federal funding from the NIH. Many scientists migrated abroad or to places like California, where Governor Schwarzenegger helped secure $3 billion in state funding to support this research.

Today, after a seven-year delay, more researchers from around the country can again begin pursuing this line of research. Dr. Alan Trouson is the President of the California Institute for Regenerative Medicine. Below follow some of his thoughts on several scientific and therapeutic directions where human Embryonic Stem Cell research might lead ( I have taken liberty to simplify for easier reading):

Spinal injuries
Preliminary studies are planned to study use of ESC-derived cells for spinal cord repair. This approach was based on data from an animal studies that show enhanced regeneration and improved mobility when ESC-derived cells were transplanted shortly after a spinal cord injuries.

Diabetes
In the area of diabetes, a partnership between a biotechnology firm and a number of academic institutions has produced pancreatic beta islet cells from ESCs. This work could potentially restore insulin production in Type I diabetics who currently require lifelong insulin therapy. Currently, this group is investigating ways to overcome the immune responses that initially destroyed the insulin-producing cells. With hope diabetics may no longer rquire daily insulin injections.

Heart attacks
After a heart attack muscle cells in the heart die and cease to function. Patients currently cannot regain this function. Researchers have been ablt to produce all of the cells needed for cardiac repair from ESCs. In animal studies, these ESC-derived cells were able to successfully integrate into functioning rat hearts, with some minimal but encouraging improvements in fuction. Most recently, engineers have begun developing fiber scaffolds that could someday be used to grow functional tissue that might be transplanted as a patch after sever heart attacks.

Blindness
Researchers hope to be able to restore sight by generating ESC-derived specialized eye tissues that could be injected or transplanted as patches. Early animal studies have been promising as mice have regained the ability to see light and dark, with the goal of discrimintating shapes underway.

Cancer
ESC research is also shedding light on how a normal cells function. Researchers hope that by creating dormant cancer cells from ESCs, they will understand more about the cellular process of these cells and how to target cells that are likely to recur after initial treatments.

Understanding genetic disorders
ESCs hold great promise for studying many genetic disorders, such as cystic fibrosis and Huntington's disease. The capacity to recreate these disease conditions in cells in the lab will help researchers look for new drugs. Such drugs might significantly prolong the healthy years of life for an individual with a Huntington's or other genetic diseases.

Drug screening and development
ESCs will also allow scientists to screen drugs for safety and effectiveness. Creating artificial cell lines from ESCs can reducing the need for using animals as test subjects. In many diseases, cells derived from normal ESCs can be used to study diseases and new drugs. In addition to reducing the need for animal testing of new drugs, environmental and drug toxicities tests currently performed on rodents can be replaced with tests of toxicity in ESC-derived cells.

Today marks a renewed national commitment to science, medicine and discovery. Now lets hope the scientists can take our hopes further.

Comments

[VanessaHammerRUSVM]

Dr. Klatsky,

President Obama's support of stem cell research is very exciting. It was nice to meet you at Le Ti. Your career path is very interesting and inspiring. I have discussed pursuing higher education with my peers and have decided that it can be a great option. Specifically there are many great careers for a DVM in public health. I think I will start reading your posts on study breaks. ( = Take care, perhaps we will meet again at Le Ti.

Vanessa Hammer

[smileatfear]

I have a progressive form of MS and am thrilled with the new opportunities President Obama is providing in the form of stem cell research. I want American scientists to have all the same opportunities as scientists from other nations.

[dundunwoot]

This article shows some extensive background into the subject area. I feel like it makes a solid argument about stem cell research and that people do really need to take a stand on the subject matter. I do hope that funding does go into this research and people take it more into consideration about the people who may need this research. Good luck Uncle Joe.

[HAP]

Why can say where this will lead--but at least we are pushing in the right direction. As Jon Stewart says: When they stand up, we will stand down.

[ProudMormon]

Good news! Obama wants scientists to decide what research to fund, and scientists have responded!

The NIH just published a list of high priority stem cell research topics open for immediate funding.

http://grants.nih.gov/grants/funding/challenge_award/High_Priority_Topics.pdf#topic_14

1. Induced Pluripotent Stem (iPS) Cells for Aging and Neurodegeneration Research.

2. Delineate Factors That Control The Differentiation Of Pluripotent Stem Cells In The Skin And Musculoskeletal System Into Different Lineages.

3. Precise Reprogramming of Cells from Oral and Craniofacial Tissues: Recent advances in reprogramming of somatic cells into induced pluripotent stem cells (iPS) cells constitute an important breakthrough...

4. Induced pluripotent stem cells--cellular and humanized mouse models of disease.

5. Development of stem cell treatment for degenerative diseases of the eye.

6. Develop molecular signatures for heart, vascular, lung, and blood diseases by profiling reprogrammed induced pluripotent stem cells (iPS) derived from affected individuals of defined genotype.

7. Developing iPS cells for mental disorders.

8. Reverse engineering human neurological disease. ... There will be an emphasis on appropriate validation of iPS cells and their derivatives, evaluating the hetero/homogeneity of any cell populations to be screened and use of cellular assays relevant to normal development, organ function and disease.

I'm glad we can stop politicizing this issue and trying to force scientists to ignore iPS cells in favor of low-priority hESC research for political reasons.

[Dr. Peter Klatsky - Huffpost Blogger]

True. iPS are very promising.
I hope that technology works out as well. It will be challenging getting past the oncogenes that need to be transfected to induce pluripotency, but I hope those researchers are able to work that out. (My own research is tangentially related, understanding the processes and temporal sequences related to pluripotent cell differentiation.)

[ProudMormon]

Yes, very exciting. As you surely know, there are ways to reversibly introduce functional proteins into cells, such as with Tat fusion proteins. Such reversible gene induction has been used in other cell systems, and even to produce experimental vaccines, but I've never personally seen or heard of anyone using that technique in iPS cells.

The future is bright.

[smileatfear]

Please provide on case where scientists have been "forced" to ignore iPS research for political reasons. If you have no evidence, please STOP creating strawmen.

[BruceA]

Dr. Klatsky,
Thank you for your article, my 25 year old son has suffered from Conjestive Heart Failure since the age of 14 thanks to a virus. He is now undergoing Pre transplant work up at Stanford University. A young person should never have to endure so much at such a young age. Articles like yours give hope to people and their families suffering the effects from diseases that they neither asked for or deserved.
Why this nation insists on taking the opinions of pundits and politicians over a Doctor is something I just can't understand.
Hopefully we'll hear from more Doctors like you.
Thank you

[Dr. Peter Klatsky - Huffpost Blogger]

Thank you BruceA.
Your son sounds very courageous. He is at a great institution (with great researchers studying stem cells as well as heart disease). I wish you all the best as you proceed with his treatments.
I will keep your son in my own prayers as well.
Peter

[Dangersea27]

Uncle Joe,
I hope the research that is now underway will someday help you.

[UNCLEJOE]

I have suffered from minor spine injury that caused me to lose my balance to the extent that even crutches barely enable me to walk without falling, These seven past years that Bush banned federal funding for research with ESC has delayed the scientific progress that very well could have cured my balance problem by now and I could be leading a normal active life instead hobbling along like a cripple.

How many moe millions could have benefited by new ESC advances?

[Dr. Peter Klatsky - Huffpost Blogger]

Joe,
Good luck with your treatments. The path from the bench (Research Lab) to the bedside (Meds that can help restore your balance) is long and not always direct. But I will hope that people smarter than me can make progress on treating neurological diseases and help you and others with similar ailments.
Good luck and keep your spirits up (I know it must be difficult).
SIncerely,
Peter

[UNCLEJOE]

I have suffered from minor spine injury that caused me to lose my balance to the extent that even crutches barely enable me to walk without falling, These seven past years that Bush banned federal funding for research with ESC has delayed the scientific progress that very well could have cured my balance problem by now and I could be leading a normal active life instead hobbling along like a cripple.

How many moe millions could have benefited by new ESC advances?

Thank you Dr. Klatsky for your encouraging article.

[Dangersea27]

Uncle Joe,
I feel your pain. Not very "Christian" to deny a suffering person releif.