How Many Times Do I Have To Ask? WITH ALL THE ADULT STEM CELL SUCCESSES, WHY DO WE NEED TO KILL UNBORN CHILDREN FOR THEIRS?
by Sally RobbinsApril 6, 2009
LifeNews.com Note: Sally Robbins is an author and freelance writer whose writing credits include Forbes, Business Week, Tribune Media Services, Market Watch, Advertising Age, and New York Daily News. She recently completed an historical novel, Rivaling Paris, and is at work on a biography about Henri Bendel.
LifeNews.com Note: Sally Robbins is an author and freelance writer whose writing credits include Forbes, Business Week, Tribune Media Services, Market Watch, Advertising Age, and New York Daily News. She recently completed an historical novel, Rivaling Paris, and is at work on a biography about Henri Bendel.
The adult stem cell marketplace is one of the most dynamic areas in life science today. There are an incredible number of scientific advancements, especially since 2008 as a heated pace of research discoveries and collaborations are underway among commercial companies, institutions and even a multi-institutional, interdisciplinary network initiative led by an executive department of the U.S. federal government.
Also, big pharma are beginning to invest heavily into regenerative medicine, and at least one state in the U.S. is proposing a bill that would create a special research consortium to maximize the benefits of adult stem cell research for its citizens.
Non-controversial adult stem cells hold great hope for successful treatments and potential cures. In recent years these cells have provided therapeutic benefits to human patients for 70+ diseases and conditions. Adult stem cells exist within the body and have untapped potential to rescue and heal tissue and organs that have been injured or diseased. They can be extracted from many types of tissues such as umbilical cord at birth, peripheral, bone marrow, adipose (fat) tissue, menstrual-derived blood, dental pulp, liver, skeletal muscle, placentas, and more.
A service of the National Institutes of Health called ClinicalTrials.gov(http://www.clinicaltrials.gov), a registry of federally and privately supported clinical trials conducted in the U.S. and around the world, showed at the end of March 2009 there were more than 2,300 adult stem cell clinical trials in various stages (completed, active, recruiting volunteers, and notyet recruiting) investigating these cells use as potential breakthrough therapies for a myriad of diseases.
“We estimate there are up to 300 firms worldwide engaged in some aspect of stem cell research and commercialization and the vast majority of stem cell companies are focused on adult stem cells: allogenic (taken from a different individual); autulogous (taken from an individual and transferred back to original donor); or cultured. And it is becoming increasingly clear that these stem cells can address up to 70 different clinical indications. Much of what is motivating these early pioneer patients is an existing deep-seated demand for these therapies,” according to Robin Young, CFA, CEO of RRY Publications LLC, a market analysis firm in Wayne, Pa. His analysis and market forecasts (2009 - 2019) were presented at the 4th Annual Stem Cell Summit on February 17, 2009 in New York. (“Stem Cell Summit Executive Summary”; http://www.ryortho.com; click-on Bookstore)
“All three types of stem cells are now firmly in the marketplace. Over the course of the last four years, approximately 30,000 patients have been treated with allogenic stem cell products in the U.S.; 2,000 – 3,000 with autologous stem cell products; and more than 1,000 with cultured stem cell products,” stated the market report.
Stem cells as commercial products have demonstrable therapeutic value in 15 medical markets, according to Young’s analysis, such as heart muscle repair, nerve regeneration and diabetes treatment. “In 2008 in the U.S., there were 35 million patients available for eventual stem cell therapeutic treatment in the 15 markets analyzed. By 2018, we forecast stem cells will be used therapeutically in as many as 2 million annual procedures for an aggregate market penetration of approximately 6%. These products will be generating, we estimate, well over $8 billion in revenue.”
Also, big pharma are beginning to invest heavily into regenerative medicine, and at least one state in the U.S. is proposing a bill that would create a special research consortium to maximize the benefits of adult stem cell research for its citizens.
Non-controversial adult stem cells hold great hope for successful treatments and potential cures. In recent years these cells have provided therapeutic benefits to human patients for 70+ diseases and conditions. Adult stem cells exist within the body and have untapped potential to rescue and heal tissue and organs that have been injured or diseased. They can be extracted from many types of tissues such as umbilical cord at birth, peripheral, bone marrow, adipose (fat) tissue, menstrual-derived blood, dental pulp, liver, skeletal muscle, placentas, and more.
A service of the National Institutes of Health called ClinicalTrials.gov(http://www.clinicaltrials.gov), a registry of federally and privately supported clinical trials conducted in the U.S. and around the world, showed at the end of March 2009 there were more than 2,300 adult stem cell clinical trials in various stages (completed, active, recruiting volunteers, and notyet recruiting) investigating these cells use as potential breakthrough therapies for a myriad of diseases.
“We estimate there are up to 300 firms worldwide engaged in some aspect of stem cell research and commercialization and the vast majority of stem cell companies are focused on adult stem cells: allogenic (taken from a different individual); autulogous (taken from an individual and transferred back to original donor); or cultured. And it is becoming increasingly clear that these stem cells can address up to 70 different clinical indications. Much of what is motivating these early pioneer patients is an existing deep-seated demand for these therapies,” according to Robin Young, CFA, CEO of RRY Publications LLC, a market analysis firm in Wayne, Pa. His analysis and market forecasts (2009 - 2019) were presented at the 4th Annual Stem Cell Summit on February 17, 2009 in New York. (“Stem Cell Summit Executive Summary”; http://www.ryortho.com; click-on Bookstore)
“All three types of stem cells are now firmly in the marketplace. Over the course of the last four years, approximately 30,000 patients have been treated with allogenic stem cell products in the U.S.; 2,000 – 3,000 with autologous stem cell products; and more than 1,000 with cultured stem cell products,” stated the market report.
Stem cells as commercial products have demonstrable therapeutic value in 15 medical markets, according to Young’s analysis, such as heart muscle repair, nerve regeneration and diabetes treatment. “In 2008 in the U.S., there were 35 million patients available for eventual stem cell therapeutic treatment in the 15 markets analyzed. By 2018, we forecast stem cells will be used therapeutically in as many as 2 million annual procedures for an aggregate market penetration of approximately 6%. These products will be generating, we estimate, well over $8 billion in revenue.”
Cord Blood Fastest Growing Therapy Group
Dallas Hextell is living proof of the healing power of adult stem cells there. He was diagnosed at eight months with cerebral palsy, which has no known cure. Fortunately, Dallas’ parents had his umbilical cord blood stored when he was born. He was accepted in a clinical trial at Duke University in July 2007 and was among the first in the country to be intravenously infused with his own cord blood stem cells to induce healing in his brain. Soon after his procedure Dallas’ parents began noticing improvements. Today, with the help of therapists, Dallas is doing things no one thought possible. He runs, he laughs, and he’s beginning to talk.
“When babies are born, the umbilical cord is generally discarded. This is partly because pregnant women and many of their doctors are unaware that life science has demonstrated cord blood cells have immense therapeutic value. Increasingly families are educating themselves about cryogenic storage of their child’s cord blood because they understand recent progress has revealed it is a safe and ethical source of stem cells for therapeutic use and the technology is evolving rapidly. These cells can becryogenically stored for more than 30 years. It’s a one-time opportunity,” said David Koos, Chairman and CEO of San Diego-based Bio-Matrix Scientific Group, Inc. (OTC Bulletin Board: BMSN; http://www.BMSN.us).
BMSN, an emerging research and development biotechnology company, opened its commercial cryogenic stem cell banking and processing facility in 2008. As of January 2009 BMSN has entered into contractual agreements with private stem cell preservation companies and other entities for the cryogenic banking and processing of stem cells from cord blood and peripheral blood of those companies’ donors’ specimens. BMSN does not have any contact with donor clients.
The Association of Family Cord Blood Banks estimated that about 750,000 cord blood collections are stored in private/family banks in the U.S., as of June 2008. The average cost of private cord blood banking in the U.S. for an individual is approximately $2,000 for the collection and about $125 per year for storage, according to industry figures. There are often other fees involved.
“Stem cell treatments from umbilical cord blood are one of the fastest growing therapy groups, and proven clinically effective for conditions including cancers, leukemia, and sickle cell anemia. In fact, stem cell transplantation recently has overtaken bone marrow cell transplantation in total number of treatments,” Young reported. “As these therapies become more publicized, more effective, and more wide-reaching, the demand for stem cells will grow from two primary factors: increasing numbers of transplant patients, and increasing numbers of parents banking cord blood as a sort of ‘biological insurance.’” Young also noted integral in the growth of the cord blood banking industry is BioE Inc.’s PrepaCyte-CB product line, a new standard for cord blood processing, (http://www.bioe.com), which is positioned to become the dominant and default cord blood stem cellprocessing technology.
“When babies are born, the umbilical cord is generally discarded. This is partly because pregnant women and many of their doctors are unaware that life science has demonstrated cord blood cells have immense therapeutic value. Increasingly families are educating themselves about cryogenic storage of their child’s cord blood because they understand recent progress has revealed it is a safe and ethical source of stem cells for therapeutic use and the technology is evolving rapidly. These cells can becryogenically stored for more than 30 years. It’s a one-time opportunity,” said David Koos, Chairman and CEO of San Diego-based Bio-Matrix Scientific Group, Inc. (OTC Bulletin Board: BMSN; http://www.BMSN.us).
BMSN, an emerging research and development biotechnology company, opened its commercial cryogenic stem cell banking and processing facility in 2008. As of January 2009 BMSN has entered into contractual agreements with private stem cell preservation companies and other entities for the cryogenic banking and processing of stem cells from cord blood and peripheral blood of those companies’ donors’ specimens. BMSN does not have any contact with donor clients.
The Association of Family Cord Blood Banks estimated that about 750,000 cord blood collections are stored in private/family banks in the U.S., as of June 2008. The average cost of private cord blood banking in the U.S. for an individual is approximately $2,000 for the collection and about $125 per year for storage, according to industry figures. There are often other fees involved.
“Stem cell treatments from umbilical cord blood are one of the fastest growing therapy groups, and proven clinically effective for conditions including cancers, leukemia, and sickle cell anemia. In fact, stem cell transplantation recently has overtaken bone marrow cell transplantation in total number of treatments,” Young reported. “As these therapies become more publicized, more effective, and more wide-reaching, the demand for stem cells will grow from two primary factors: increasing numbers of transplant patients, and increasing numbers of parents banking cord blood as a sort of ‘biological insurance.’” Young also noted integral in the growth of the cord blood banking industry is BioE Inc.’s PrepaCyte-CB product line, a new standard for cord blood processing, (http://www.bioe.com), which is positioned to become the dominant and default cord blood stem cellprocessing technology.
Research Validates New Direction for Storage
An important discovery announced on March 25, 2009 by researchers at the National Institutes of Health (NIH; http://www.nih.gov), which has implications for Leukemia treatment and artificially culturing blood cells, validates a new direction for cryogenic stem cell storage facilities that meet rigid state and federal regulatory requirements. The researchers deciphered a key sequence of events governing whether the stem cells that produce red and white blood cells remain anchored to the bone marrow, or migrate into the circulatory system – a key discovery that will advance understanding of how blood cells and immune cells are generated.
“The findings of NIH researchers have implications for culturing infection-fighting immune cells outside the body, where they could be temporarily held in storage during chemotherapy and other treatments which suppress the immune system. This discovery confirms a new trend for commercial cryogenic storage facilities for research and treatment of diseases, and their important role in public and private stem cell research efforts now and into the future,” said BMSN’s Koos. Describing BMSN as a“conservative player in the stem cell industry,” he pointed out that the company is developing a unique niche within the stem cell industry as for stem cell companies to store blood specimens for research and treatment of diseases.
While the concept of private cord blood banking started in the U.S. about 20 years ago, there has been tremendous growth worldwide in recent years. A useful global educational web site dedicated to informing expectant parents with unbiased and current information about how to choose a bank, and all the issues involved, is Parent’s Guide to Cord Blood (http://www.parentsguidecordblood.org), based in Brookville, MD. The site includes listings and snap-shot histories of each U.S. private and public cord blood bank. There are also descriptions of 150 cord blood banks located worldwide.
According to Young’s forecast: “Worldwide estimates show the market for cord blood processing and banking will grow by 25% over the next five years (approaching $450 million annually for cord blood processing), driven by surging demand in the U.S. and Western Europe (where an estimated one-quarter of all newborns will have banked cord blood) and exploding demand in India, China and Latin America – where processing is just beginning to take off.”
“The findings of NIH researchers have implications for culturing infection-fighting immune cells outside the body, where they could be temporarily held in storage during chemotherapy and other treatments which suppress the immune system. This discovery confirms a new trend for commercial cryogenic storage facilities for research and treatment of diseases, and their important role in public and private stem cell research efforts now and into the future,” said BMSN’s Koos. Describing BMSN as a“conservative player in the stem cell industry,” he pointed out that the company is developing a unique niche within the stem cell industry as for stem cell companies to store blood specimens for research and treatment of diseases.
While the concept of private cord blood banking started in the U.S. about 20 years ago, there has been tremendous growth worldwide in recent years. A useful global educational web site dedicated to informing expectant parents with unbiased and current information about how to choose a bank, and all the issues involved, is Parent’s Guide to Cord Blood (http://www.parentsguidecordblood.org), based in Brookville, MD. The site includes listings and snap-shot histories of each U.S. private and public cord blood bank. There are also descriptions of 150 cord blood banks located worldwide.
According to Young’s forecast: “Worldwide estimates show the market for cord blood processing and banking will grow by 25% over the next five years (approaching $450 million annually for cord blood processing), driven by surging demand in the U.S. and Western Europe (where an estimated one-quarter of all newborns will have banked cord blood) and exploding demand in India, China and Latin America – where processing is just beginning to take off.”
Department of Defense Creates AFIRM
Research is well underway among participating institutions since the U.S.Department of Defense announced in April 2008 the creation of a new $250 million institution focused on research called the Armed Forces Institute of Regenerative Medicine (AFIRM). The virtual organization is a multi-institutional, interdisciplinary network working to develop advanced treatment options over the next five years for severely wounded service men and women. AFIRM is managed and funded through the U.S. Army Medical Research and Materiel Command (USAMRMC), with additional funding from the U.S. Navy, U.S. Air Force, the National Institutes of Health, the Veterans Administration and local public and private matching funding.
AFIRM has been designed to speed the delivery of regenerative medicine therapies to treat the most critically injured service members from around the world, but in particular those coming from theaters of operation in Iraq and Afghanistan. There are five major programs: Limb Repair, Craniofacial Repair, Burn Repair, Scarless Wound Repair and Compartment Syndrome Repair.
AFIRM is made up of two civilian research consortia working with the U.S.Army Institute for Surgical Research (USAISR) in Fort Sam, Houston, Texas. One consortium is lead by Rutgers, The State University of New Jersey, and the Cleveland Clinic and the other is led by Wake Forest University Institute for Regenerative Medicine and The McGowan Institute for Regenerative Medicine at the University of Pittsburgh. Each of thesecivilian consortia is itself a multi-institutional network. There are atotal of 28 civilian institutions involved.
"Our researchers are continuing to pursue promising avenues, such as tissue regeneration of fingertips," said AFIRM Investigator William Wagner, Ph.D., Deputy Director of the McGowan Institute for Regenerative Medicine. "We are also testing in animal models materials that could replace damaged abdominal wall muscles, and we might soon embark on a novel strategy that uses muscle-derived stem cells to heal severely injured tissue."(http://www.mirm.pitt.edu)
Dozens of commercial interests are expressing a willingness to work with the AFIRM consortia as commercialization partners. The medical device industry has taken a keen interest in speeding these important new therapies to market, not just for injured service members, but for civilian patients as well. AFIRM believes this participation ultimately will lead to betterhealthcare options for all Americans.
All of the research now being funded will use adult-derived stem cells taken from the patient or from another consenting adult. AFIRM has stated adult stem cells and progenitor cells are an integral part of normal wound healing and the formation of all new tissues. Many of the strategies being developed by AFIRM seek to improve wound healing and tissue repair by increasing the number or improving the function of adult stem cells. A patient’s own cells, or in some cases, cells from another adult, are used inconjunction with special drugs called bioactive factors, or with advanced biomaterials that serve as scaffold for growth of new tissues.
“The vision of the future is that before soldiers go off on operations they will have their own stem cells harvested through liposuction and stored for use if they get wounded. That way while a casualty is being stabilized overseas, we will be in the U.S. growing them muscle, skin or bone ready for surgery,” Sgt. Glen Rossman of USAISR told Soldier magazine (March 2009 issue).
Project Director Col. Bob Vandre also commented: “AFIRM’s research is very exciting news and should be very reassuring to soldiers. Some day we will be able to fix a lot of things. We currently have 12 clinical trials ready to roll and that is a sign that AFIRM has arrived and is very much for real.”
AFIRM has been designed to speed the delivery of regenerative medicine therapies to treat the most critically injured service members from around the world, but in particular those coming from theaters of operation in Iraq and Afghanistan. There are five major programs: Limb Repair, Craniofacial Repair, Burn Repair, Scarless Wound Repair and Compartment Syndrome Repair.
AFIRM is made up of two civilian research consortia working with the U.S.Army Institute for Surgical Research (USAISR) in Fort Sam, Houston, Texas. One consortium is lead by Rutgers, The State University of New Jersey, and the Cleveland Clinic and the other is led by Wake Forest University Institute for Regenerative Medicine and The McGowan Institute for Regenerative Medicine at the University of Pittsburgh. Each of thesecivilian consortia is itself a multi-institutional network. There are atotal of 28 civilian institutions involved.
"Our researchers are continuing to pursue promising avenues, such as tissue regeneration of fingertips," said AFIRM Investigator William Wagner, Ph.D., Deputy Director of the McGowan Institute for Regenerative Medicine. "We are also testing in animal models materials that could replace damaged abdominal wall muscles, and we might soon embark on a novel strategy that uses muscle-derived stem cells to heal severely injured tissue."(http://www.mirm.pitt.edu)
Dozens of commercial interests are expressing a willingness to work with the AFIRM consortia as commercialization partners. The medical device industry has taken a keen interest in speeding these important new therapies to market, not just for injured service members, but for civilian patients as well. AFIRM believes this participation ultimately will lead to betterhealthcare options for all Americans.
All of the research now being funded will use adult-derived stem cells taken from the patient or from another consenting adult. AFIRM has stated adult stem cells and progenitor cells are an integral part of normal wound healing and the formation of all new tissues. Many of the strategies being developed by AFIRM seek to improve wound healing and tissue repair by increasing the number or improving the function of adult stem cells. A patient’s own cells, or in some cases, cells from another adult, are used inconjunction with special drugs called bioactive factors, or with advanced biomaterials that serve as scaffold for growth of new tissues.
“The vision of the future is that before soldiers go off on operations they will have their own stem cells harvested through liposuction and stored for use if they get wounded. That way while a casualty is being stabilized overseas, we will be in the U.S. growing them muscle, skin or bone ready for surgery,” Sgt. Glen Rossman of USAISR told Soldier magazine (March 2009 issue).
Project Director Col. Bob Vandre also commented: “AFIRM’s research is very exciting news and should be very reassuring to soldiers. Some day we will be able to fix a lot of things. We currently have 12 clinical trials ready to roll and that is a sign that AFIRM has arrived and is very much for real.”
Big Pharma Enter Arena
In what represents a major shift in the field, Pfizer announced in November 2008 it would spend $100 million over five years to develop therapies from stem cells with the launch of Pfizer Regenerative Medicine(http://www.pfizer-regenerativemedicine.com), a global unit based in Cambridge, MA and Cambridge, the UK.
The U.S. unit is focusing on using stem cells to develop therapies for cardiac disorders and cancer through in-house research and a vast array of alliances and collaborations, and will grow its team to 20 scientists. The U.K. arm will grow to 60 scientists over the next two years with a focus predominately on age-related and degenerative disorders with particular interest in common cellular mechanisms and disorders of the central and peripheral nervous system.
Pfizer Regenerative Medicine is operating as an independent research unit and is basing its stem cell research and drug discovery efforts on a fullset of drug discovery capabilities.
Earlier in 2008, Pfizer invested $3 million with La Jolla, CA-based startup EyeCyte, Inc. (http://www.eyecyte.com) to develop treatments fordiabetes-induced retinal damage, a leading cause of blindness, using patient blood and bone marrow-derived progenitor cells.
In addition, Epistem Plc (http://www.epistem.co.uk), the UK-based biotechnology and research services company, announced in March 2009 that it had signed a research and development collaboration with Novartis to identify new drug targets and therapeutics across a variety of disease areas. Epistem, which is focused on the regulation of adult stem cellslocated in epithelial tissue, is commercializing its expertise in the areas of oncology, gastrointestinal and dermatological diseases.
The U.S. unit is focusing on using stem cells to develop therapies for cardiac disorders and cancer through in-house research and a vast array of alliances and collaborations, and will grow its team to 20 scientists. The U.K. arm will grow to 60 scientists over the next two years with a focus predominately on age-related and degenerative disorders with particular interest in common cellular mechanisms and disorders of the central and peripheral nervous system.
Pfizer Regenerative Medicine is operating as an independent research unit and is basing its stem cell research and drug discovery efforts on a fullset of drug discovery capabilities.
Earlier in 2008, Pfizer invested $3 million with La Jolla, CA-based startup EyeCyte, Inc. (http://www.eyecyte.com) to develop treatments fordiabetes-induced retinal damage, a leading cause of blindness, using patient blood and bone marrow-derived progenitor cells.
In addition, Epistem Plc (http://www.epistem.co.uk), the UK-based biotechnology and research services company, announced in March 2009 that it had signed a research and development collaboration with Novartis to identify new drug targets and therapeutics across a variety of disease areas. Epistem, which is focused on the regulation of adult stem cellslocated in epithelial tissue, is commercializing its expertise in the areas of oncology, gastrointestinal and dermatological diseases.
Texas Bill Would Create Research Consortium
To further adult stem cell research on the state level, Texas Senator Jane Nelson (R-Lewisville) and Chair of the Senate Health and Human ServicesCommittee, has authored Senate Bill 73 that would create the Adult Stem Cell Research Consortium, which would encourage collaboration between researchers at Texas universities and cord blood banks. This body would oversee funding for adult stem cell research received from both public and private sectors.
If passed, the bill would move Texas beyond the debate about whether it isappropriate or inappropriate to destroy human embryos into the realm of adult stem cell research and treatments without ethical concerns. It isestimated that more than 1.2 million Texans are afflicted with chronic degenerative conditions and can benefit from this research that could leadto the discovery of successful treatments and potential cures.
During March 2009 Texas Medical Association physician leader James T.Willerson, MD, who is President and Medical Director of Cardiology Research, and Codirector of the Cullen Cardiovascular Research Laboratories at Texas Heart Institute in Houston, and other authorities in the state’s adult stem cell field testified before senators at the Texas Senate Health and Human Services Committee meeting in Austin in support of Senate Bill 73.
If passed, the bill would move Texas beyond the debate about whether it isappropriate or inappropriate to destroy human embryos into the realm of adult stem cell research and treatments without ethical concerns. It isestimated that more than 1.2 million Texans are afflicted with chronic degenerative conditions and can benefit from this research that could leadto the discovery of successful treatments and potential cures.
During March 2009 Texas Medical Association physician leader James T.Willerson, MD, who is President and Medical Director of Cardiology Research, and Codirector of the Cullen Cardiovascular Research Laboratories at Texas Heart Institute in Houston, and other authorities in the state’s adult stem cell field testified before senators at the Texas Senate Health and Human Services Committee meeting in Austin in support of Senate Bill 73.
Cord Blood Advancements Since 1st Transplant
In cord blood technology, 2008 marked the 20th anniversary of the first cord blood transplantation in the world, carried out by Eliane Gluckman, MD,Ph.D. The recipient of that pioneering effort, Matthew Farrow, now 26 years old, continues in good health. He was born with a rare blood disorder and was expected to die before the age of 10. When he was five his parents took part in a pioneering experiment in France to use stem cells from his sister’s umbilical cord when she was born.
Since then, cord blood stem cells have been used in more than 14,000 transplants worldwide to treat more than 70 diseases in both adults and children and are now showing great promise for regenerative medicine applications, including treatment for type 1 diabetes, brain injury,cerebral palsy and hearing loss, according to Cord Blood Registry(http://www.cordblood.com), the world’s largest stem cell bank.
“As a result of pioneering work and the tremendous progress over the years, for many families cord blood banking is the best option for treating and curing disease, especially as they understand the importance of umbilical cord donations for advancing the science,” said Koos of Bio-MatrixScientific Group.
Since then, cord blood stem cells have been used in more than 14,000 transplants worldwide to treat more than 70 diseases in both adults and children and are now showing great promise for regenerative medicine applications, including treatment for type 1 diabetes, brain injury,cerebral palsy and hearing loss, according to Cord Blood Registry(http://www.cordblood.com), the world’s largest stem cell bank.
“As a result of pioneering work and the tremendous progress over the years, for many families cord blood banking is the best option for treating and curing disease, especially as they understand the importance of umbilical cord donations for advancing the science,” said Koos of Bio-MatrixScientific Group.
Labels: Stem Cell Research
posted by Al @ 7:12 AM
1 Comments:
At 8/4/09 8:08 PM ,
Hope in Regeneration Hair Cells said...
I have been in contact w/ about 148 doctors/scientist about stem cell therapy for hearing loss and they all have different answers as to when stem cells can be used on humans. Here are some of the responses I have gotten.
A. J. Hudspeth (hudspaj@mail.rockefeller.edu)
My colleagues and I are indeed working on that subject, though I regret to say that we are still involved in the most basic research on the phenomenon in simple experimental preparations. In particular, we perform experiments on zebrafish, small aquarium fish that are easy to study, but which have sensory hair cells in the their ears much like our own. By understanding how hair-cell regeneration occurs in these simple animals, we hope to lay a foundation for the treatment of human hearing problems through the regenerating of hair cells in our own ears.
Albert Edge (albert_edge@meei.harvard.edu)
It's true that we have made a lot of progress and that we know a lot more about these cells than we did even a few years ago.
While it is true that we are making progress that is exciting for us, we are still far from a cure that will replace hair cells. Progress in the laboratory always precedes progress in the clinic, often by many years. Most of what we know takes place in a lab dish and there will be work to do in animal models of deafness to be sure it works.
You are right that the bone marrow stem cells are exciting but we can't yet turn them into the type of cells that we would need for human use. We can make cells that are similar to nerves in the ear and to hair cells in the ear but we have more work to do so that they do everything right. We expect that those steps will take time.
Alejandro Sánchez Alvarado (sanchez@neuro.utah.edu)
Our research has just begun, so it is with an amalgam of regret, buttressed by hope and certainty of future progress that I have to tell you that presently we have little information that could be even considered to serve as the basis for framing a therapeutic application.
Allen Barnett (abarnett@kinexpharma.com)
We have not progressed our program to the point of being near human trials with our lead compound for hearing loss. We have been almost fully engaged with progressing two compounds for cancer but still are hopeful of progressing the hearing loss compoiund. One of our problems is getting adequete funding to move this effort.
Allen Ryan (afryan@ucsd.edu)
I am performing research on hair cell regeneration in my laboratory, but I am afraid that this work is still many years away from application to patients. We study both the use of stem cells for transplantation into the ear, and sensory cell regeneration induced by gene therapy. Unfortunately, there are many hurdles to be overcome before the procedures that we use in the lab are more reliable, and also would be safe to use in humans. The fact that we are able to induce sensory cell regeneration under ideal laboratory circumstances is encouraging, however.
Anand Swaroop (swaroop@med.umich.edu)
I am on leave from Michigan and have joined National Institutes of Health. We and numerous others are spending a lot of effort on stem cell based therapies; however, the clinical trials require a lot of basic research and preparation. It is hard to predict the pace of research progress and put a realistic time frame for using stem cells for human hair cell or photoreceptor regeneration.
Andy Forge (a.forge@ucl.ac.uk)
There is work in a number of places throughout the world attempting to find ways to restore hearing to a deaf ear. These approaches include stem cells; trying to stimulate the cells that remain in a deaf ear to divide to produce new sensory cells; and gene transfer to induce the remaining cells to "turn into" hair cells. Currently none of this research is at a stage where clinical trials might be envisaged. I do not know when that is likely to occur. At present we have ideas of what might be possible, and as research proceeds I am sure that we will discover how to translate the results of that work into clinically applicable therapies. Situations can change very rapidly as new discoveries are made but at present I do not thinkwe are yet at a stage where we can predict when a clincally feasible procedure will become available.
Kesser, Bradley W *HS (BWK2N@hscmail.mcc.virginia.edu)
I think clinical trials are still a long way away (a decade?), but these preliminary results are certianly promising!
Clifford R. Hume (hume@u.washington.edu)
Optimistically, I would expect a clinical trial within 5-10 years in patients with complete deafness (in both ears). These new types of treatments will probably be used soonest in people with complete deafness. There may be some exceptions, but because any new treatment has a potential to cause people to actually lose hearing, the risks don't outweigh the expected benefits. Another area of ongoing research in many laboratories is how to prevent further hearing loss after it starts. There are roles of underlying genetics and the environment, ie noise, drugs, infections etc.
David A Borkholder (dabeee@rit.edu)
he exciting results on gene-based therapy showing regeneration of cochlear hair cells has only been done (to date) in animal models immediately following acute injury. The goal is certainly to have effective gene based therapies that can be used at any time (even decades) post injury, or to address genetic defects present at birth. I was just trying to put the current state of the art results in perspective with respect to likely clinical use.
David Kozlowski (dkozlowski@mcg.edu)
appreciate you taking the time to write. My lab does what is called “basic science”, which means we try to figure out the genes and pathways that are important for regeneration. We use zebrafish as a model because they do regenerate hair cells. Once we have an idea of how regeneration works in zebrafish we then have to test our ideas in mice (a mammal similar to you and me) and then, if all goes well, begin to think about testing in humans. It is a long process, but increased funding for the NIH (National Institutes of Health), specifically the NIDCD (National Institute of Deafness and Communication Disorders), and fewer restrictions on stem cell research would be helpful.
David N Furness (d.n.furness@cns.keele.ac.uk)
Unfortunately, the type of injury you describe will always likely to be very difficult to treat. Our research is at a very early stage – we hope to be able to help people with age-related hearing loss but I think the kind of damage which you have been exposed to would be unlikely to be treated by the means we hope to use. Many other methods are also being tried by researchers in our field, so I hope that there may be some help for you in the future.
David P. Corey (dcorey@hms.harvard.edu)
In general, researchers in this field are excited about the possibilities, but are realistic about how long it will take to bring these studies to clinical practice. A good idea takes years to work out in animals, and then many more years to test for safety and efficacy in humans, before it gets to the clinic. It will take more funding. We have talented and dedicated young scientists who want to work in this area, but there are not many jobs for them because the NIH budget has been cut back so much during the past eight years. Now, we are excited by the new NIH funding in the stimulus bill, even though it will only last two years.
Dongguang Wei (donwei@ucdavis.edu)
Currently we are still focusing on animal tests, trial experiments on human is pending, we will let you know when it is ready.
Donna M. Fekete (dfekete@purdue.edu)
Want to emphasize that my lab works from the point of view of developmental biology, trying to understand the molecular basis of making the inner ear and the sensory hair cells. We use exclusively animal models, so we are not involved in any clinical trials in humans. In fact, I am not aware of any clinical trials planned in the near future in any laboratories world-wide. The work in the field overall, to implant stem cells or to deliver genes that might induce hair cells to regenerate, is still ongoing in animals. Most particularly in mice and guinea pigs. It is a difficult problem and while there is some promise, these things take time to be sure there is a high chance of success for someone who might take on the risk of surgery.
Douglas Cotanche (cotanche@bu.edu)
I'm afraid the estimate of 10-20 years is probably accurate, if not conservative. But we will keep working!
Ebenezer N Yamoah (enyamoah@ucdavis.edu) fo
I do understand your concerns and the need to push some of our findings to the clinical phase. We are making headways but we have to be extremely cautious. I shall keep you inrmed if and when we are ready for trials.
Elisabeth Glowatzki (eglowat2@jhmi.edu)
In the inner ear, hair cells pick up the sound signal. Hair cells are connected to spiral ganglion neurons and these nerve cells send information about sound to the brain. Our research focuses on these nerve cells and whether they can be substituted by stem cells when lost. Often, after a sound trauma, not only hair cells but also these neurons are lost, so both cell types may have to be regenerated in the damaged ear. The question is whether stem cells can acquire properties that these neurons have and whether they can make functional connections with hair cells. Our collaborators, experts on stem cell research are Albert Edge and others at Harvard Medical School in Boston. His research group developed a culture system, in which the reconnection of hair cells with nerve cells, or hair cells with stem cell that should become nerve cells can be investigated. We can record the electrical activity in all these cell types and we are trying to test whether in a cultured system, functional connections between hair cells and nerve cells can be regenerated.
Many research group work on this topic. However, it is hard to solve and may take many years. It would be giving false hope if I said that our research field is close to a solution. However, I see no other way than working little by little towards the goal of ultimately restoring hair cells and nerve cells in inner ears.
Elizabeth Oesterle (oesterle@u.washington.edu)
While we are making good steady progress, we are a long way from human clinical trials and funding this research is critical to moving it forward rapidly and its eventual success.
Ed Rubel (rubel@u.washington.edu)
Let me stress that we need a pluralistic approach to the problem of hair cell regeneration research. What has ben done to date is to show that it is possible. That is more than we knew 22 years ago when Doug Cotanche and his colleagues, and my colleagues and I discovered this ability in birds.
It doesn't really matter what approach I am using right now or what I think is "the right" approach. Targeted science does not progress by consensus. It is most often successful by many approaches adding pieces to the puzzle, and then allowing them to come together. It is by hard work from a lot of people and organizations.
As scientists, we do not work in a vacuum. We talk to each other often and read each other's work. We try to replicate it and we build on it. In my opinion, NOBODY can predict at this time WHEN there will be a treatment that restores hearing through hair cell regeneration. It will happen, but whether it is 8 years of 50 will depend on money, attracting dedicated and talented people and luck. What is needed now is sufficient support to sustain groups of scientists using many approaches for significant amounts of time.
Gabriel Corfas (Gabriel.Corfas@childrens.harvard.edu)
As you most probably understand, studies on the use of stem cells as therapeutic tools are very much in their infancy and a lot more research will be required before anything gets into the clinic.
Hernan Lopez-Schier (hernan.lopez@crg.es)
As you probably know, hearing loss due to hair-cell damage is a complex situation. Right now there is no “correct” or “wrong” answer as to when (or even if at all!) hair-cell regeneration will make the jump from the lab to the clinic.
Huda Zoghbi (hzoghbi@bcm.tmc.edu)
Genvec is doing teh gene therapy we are trying to find targets of the gene to see if there could be new ways to modify its function, nothing clinically relevant yet, it will take a while.
Jim Pickles (j.pickles@uq.edu.au)
I don't think that stem cells will provide a cure in the near future - we are talking about a couple of decades for a practical cure in human beings, whatever is found in animal
Joseph Santos_Sacchi (joseph.santos-sacchi@yale.edu)
We are planning to see if we can induce regeneration of hair cells in chicks and mice. A long way from people, though.
Kumar Algaramam (kumar.alagramam@case.edu)
We have a lot to understand about the sensory cells ('microphones') in the cochlea and the neural circuitry that connects these microphones to the brain. Although several papers are putting out promising results, attempts in my lab to seed affected cochlea - in mouse model with hearing loss - with stem cells shows that not all stem cells introduced into the cochlea end-up in the target area and many fail to integrate and repair affected neuroepithelia. Clearly more work is required in the basic science direction and that is what I am doing now.
Marcelo N. Rivolta (m.n.rivolta@sheffield.ac.uk)
We have more robust protocols to push stem cell differentiation into auditory lineages (primarily with fetal and embryonic stem cells) and have developed methods also to drive bone marrow stem cells. We are now testing them in deaf animals, but still waiting for the conclusions of these experiments. there are still a few hurdles that need to be overcome before we can take this into the clinic. We have to be as sure as possible that the therapy is safe before we can deliver them into patients. So, I am afraid that any human trials are still a few years into the future.
Mariola Sliwinska (marsliw@imp.lodz.pl)
I do not plan human clinical trials on hair cells regeneration.
Matthew Fero (mfero@fhcrc.org)
Unfortunayely,there are still a number if technical challenges to overcome before it would be suitable for human studies.
Matthew Holley (M.C.Holley@sheffield.ac.uk)
Hearing can be restored in an animal it takes some 15 years at least to get a treatment to the clinic.Sensory hair cells and we are a very long way from any therapy that can replace them. The cochlear implant is the only 'cure'
Michelle De Silva (mgds@unimelb.edu.au)
do very little work on inner ear hair cell regeneration these days. I believe most work is done in the States at the moment. From what I have done I think human trials are some years off. Maybe take a look at some of the work in Ann Arbor or Harvard.
Neil Segil (nsegil@hei.org)
Basic research cannot have a time frame because it is about new discoveries that have not yet been made. We can judge progress, but we cannot judge when the crucial information needed to allow regeneration will be discovered, because we do not know what it is.
Nicolas Daudet (n.daudet@ucl.ac.uk)
Unfortunately, I think it will be "some time" before hair cell regeneration can be used as a therapy for human forms of hearing loss. Sox genes are important for hair cell formation, but we do not know yet if these particular factors could be manipulated to promote regeneration in humans. One group has succeeded in inducing the formation of new hair cells following damage in the inner ear of guinea pigs, using a gene therapy relying on a gene named "Atonal 1" (Atoh1, also known in mice as Math1). However, this finding has not been replicated by other groups so far, and I am not aware of any experimental human trials in progress for Atoh1 gene therapy. The field is moving fast, but before anything can be applied to humans, one needs to demonstrate a very efficient therapy in small animals, which has not been achieved so far. So how long will it take? No one knows. Even if an efficient treatment was found for small mammals, it would take a couple of years before human trials can be envisioned.
Olivia Bermingham-McDonogh (oliviab@u.washington.edu)
It is often difficult for scientists to convey how much progress we have made because we focus on the ultimate goal which is curing deafness and we are not there yet. Indeed in the past 10 years we have dramatically increased our understanding of how the cochlea (the auditory part of the mammalian inner ear) develops and what factors are necessary during development to make hair cells and the other important "support cells" of the inner ear that are also necessary for hearing. I think if the field is funded appropriately progress in the next 10 years will be startling.
Peter Steyger (steygerp@ohsu.edu)
The likelihood of this being feasible in the next 20 years clinically is at best 33%.
Petros Vlastarakos (pevlast@hotmail.com)
I am afraid that even though hair cell regeneration might facilitate hearing restoration in the future, this future seems far from imminent at the moment. Although our effort is just begginning, to the best of my knowledge, no experiments in humans are planned at this moment, even in centers quite advanced.
Richard Salvi (salvi@buffalo.edu)
Progress is being made in both scientific areas, but I think it will be more than 10 years before any of this work makes into human clinical trials.
Rob Patuzzi (rpatuzzi@cyllene.uwa.edu.au)
I do not believe it will provide a "cure" for hearing loss, at least not in the next 30 years.
Robert Frisina(Robert_Frisina@URMC.Rochester.edu)
Unfortunately, there are no clinical trials occurring yet to restore hearing. There is still a lot of lab animal work to be done, by researchers like me.
Robert K. Jackler, MD (rjackler@ohns.stanford.edu)
At Stanford, we are working very hard on creating regenerative means of curing deafness. While we are making important strides, it will undoubtedly take a number of years before we have therapies ready for clinical trials.
Ruth Taylor (sjjgrrt@ucl.ac.uk)
We are not at the stage of clinical trials.
Sam Gubbels (GUBBELS@surgery.wisc.edu)
My current studies regarding hair cell regeneration are certainly a ways off from human clinical trials.
Shelley Batts (shelleba@umich.edu)
Human clinincal trials are out of my realm of research, this work is very preliminary and currently only in rodent models or the petri dish. I couldn't even begin to speculate about human work, since it is all in its beginning stages and the road to human research is a long one frought with many difficulties from the FDA and many other hurdles.
Stefan Heller (hellers@stanford.edu)
There are many reasons for not having a treatment. There is a huge jump from doing experiments in a lab animal to doing them in humans. We jump up and down when 20% of the animals have some sort of survival of cell grafts and we get > 80% survival. With humans, this is not doable. Efficacy and safety are having highest priority and we are neither efficient nor are the transplants save. This is where the focus lies. In addition, I wish we were able to make functional hair cells from ES cells in animal models - this is a goal for the next 5 years, we are definitely not at this point yet (don't know where you got the information about researchers making functional hair cells from embryonic stem cells, seems that someone mixed up a couple of stories). My lab has made cells that look like hair cells from ES cells, but we have not been able to show that these cells are functional - another project we are working on. We need do one step at t a time - I wish we could go faster, but for this we need much more funds and many more people. Each of the "mini" projects that I mentioned is work for 2-3 PhD postdocs for 3-5 years and it requires ample funds (millions) - so I spend most of my time fundraising. Lifting the ES cell ban helps, but it does not provide a cure. Hope that this helps - I am off to Europe in a couple of minutes, just boarding a plane
spencernj80@gmail.com on behalf of Nathan Spencer (nspencer@bu.edu)
I think stem cells can be a very promising avenue to restoring hearing. We do not have a project that is headed towards an immediate clinical trial. Luckily though, there are many very smart people working in the field. Who knows what someone will come up with next? I believe that clinical trials will happen, but that it is difficult to say when. People are working on many avenues though-- through hair cell regeneration, stem cells, gene therapy, and improving devices that we have like cochlear implants and hearing aids.
Steven Green (steven-green@uiowa.edu)
About five years ago, I predicted 20-25 years. I would guess that 15-20 years from now is probably about as reasonable a guess as any.
Thomas Van De Water (TVanDeWater@med.miami.edu)
The application of stem cell for the replacement of lost or nonfunctioning auditory hair cells is in its infancy and will take many more years before there will be even any thoughts of clinical trials.
Tracy Dodd (Tracy.Dodd@STJUDE.ORG)
This research is at the very first stages. It is aimed at understanding whether genetic manipulations of several important genes in the sensory cells of the inner ear would help restore lost hearing damaged by various insults including noise exposure. However, it is far from clinical trial use in humans, at least 5-10 years away. There is some testing of these ideas in animal models first and the results so far are promising. Only after successful studies in animal models will human trials be initiated.
Vincent Lin (Otolaryngology) (Vincent.Lin@sunnybrook.ca)
We have had some luck in regenerating hair cells in some mammals but the major stumbling block at this point is to develop treatments to make this regenerative response much more robust just like in birds. Unfortunately, I don’t think we’re looking at any clinical trials for at least a few more years. Certainly I hope within my career we will have medical treatments available.
Wei-Qiang Gao (gao.wei-qiang@gene.com)
It may still take a while before tested on humans.
Yehoash Raphael (yoash@umich.edu)
My studies are at a pre-clinical stage and I cannot predict how many more years will be needed before we can plan clinical applications.
Zheng-Yi Chen (Zheng-Yi_Chen@meei.harvard.edu)
As you can see we are still very far from any clinical work.
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