Dr. Rubin Goes the Distance for ALS Research
Dr. Adam D. Rubin has seen the destructive effects of ALS, more commonly known as Lou Gehrig’s disease, both personally and professionally. So it makes sense that the 44-year-old laryngologist would blend his love of running with his desire to find a cure for this devastating disease.
That is why Dr. Rubin planned to run his first New York City Marathon on Nov. 4 to raise funds for ALS research. And while the run was canceled following Hurricane Sandy, Dr. Rubin is continuing to fundraise and will run the Anthem Richmond marathon on Nov. 10 in Virginia.
Donations will go to the Program for Neurology Research & Discovery (PNRD) at the University of Michigan Health System, a laboratory directed by Dr. Eva Feldman with which Dr. Rubin has been affiliated for years.
So far, Dr. Rubin and his teammates (friends who will also be running) have received donations of nearly $9,000. That far surpasses his initial expectations when he started training in August for the grueling 26.2 mile race. Now, the determined doctor has set his eyes on raising $15,000 through CrowdRise, an online fund-raising site.
“It started off as a challenge for me, but I also thought it was a great opportunity to do a fundraiser as well. It’s been a lot of fun,” except for some aching legs and knees, Dr. Rubin said. “When I first started this, I didn’t know what to expect. In a lot of ways, now I’m more passionate about the fundraising than the running.”
This run – along with his work with U-M – are dedicated to his own patients and to the memory of a family friend who recently lost his battle with ALS, Dr. Rubin said.
“We recently lost a wonderful neighbor, Dr. Eric Baron, to the disease process,” Dr. Rubin stated. “He was a brilliant physician, entrepreneur and father. He battled the disease for eight years, making every effort to stay alive as long as possible to watch his infant daughter grow. He remained positive and determined, and truly touched the heart of all the people around him. He remains an inspiration to me and my family.”
Amyotrophic lateral sclerosis, or ALS, is a disease of the nerve cells that control voluntary muscle movement. It causes the death of these motor neurons within the brainstem and spinal cord, Dr. Rubin explained, and is a progressive disease that usually leads to death within five years of diagnosis. As neurons die, patients lose the ability to voluntarily move body parts, and eventually, to breathe. When the brainstem is involved, patients lose the ability to speak and swallow. All the while, patients suffering from this disorder stay completely aware of their bodies deteriorating.
Although there currently is no effective treatment for ALS, Dr. Feldman is leading the first FDA-approved human clinical trial of a stem cell treatment for ALS, which is taking place at Emory University in Atlanta. Contributions to Dr. Rubin’s run will go toward improving the procedure and expanding the clinical trial.
As director of the Lakeshore Professional Voice Center PC in St. Clair Shores, the laryngologist said he sees three to five patients a year who come to him believing they have voice problems only for Dr. Rubin to find they have early indicators of ALS.
A native of Huntington Woods and a graduate of the Harvard Medical School, Dr. Rubin’s own basic science interests include using gene therapy to regenerate nerves of the larynx that have been damaged by injury or disease. He began his collaboration with Dr. Feldman, who is also the director of the A. Alfred Taubman Medical Research Institute at U-M, as a resident, during which time he received the U-M Merle Lawrence Basic Science Research and the John L. Kemink Clinical Research Awards.
Many of Dr. Rubin’s donations have come from colleagues, family and friends whose lives have been touched by ALS in some form. He also has spread the word about his fund-raising efforts through social-media outlets such as Twitter (@VoiceDocintheD) and Facebook.
“You might think of it as a rare disease, but a lot of people have a connection to it,” Dr. Rubin said. “It’s amazing – everyone has been incredibly supportive.”
For more information or to donate to Dr. Rubin’s cause, please go to: http://www.crowdrise.com/adamnycrun/fundraiser/adamrubin
ALS stem cell trial is chronicled in Detroit Free Press two-part series
U-M oversees cutting-edge trial that offers hope in fight against Lou Gehrig's disease
By Robin Erb, Free Press Medical Writer
Sometimes she glares at the painting of Jesus in her dining room.
"I just let it loose," said Mary Kleiss at her Royal Oak home. "I look at that picture and I say, 'You get down here and put on your boxing gloves and let's get this over with.' I am so damned angry."
Her son, Regis, was diagnosed two and a half years ago with Lou Gehrig's disease -- amyotrophic lateral sclerosis, or ALS. It is, he writes, "as if God is torturing me."
The disease kills with stunning efficiency -- deadening its victims' peripheral nerves, withering muscles and, in a final assault, shutting down their ability to breathe. An estimated 30,000 people have it at any given time; 5,000 are diagnosed yearly. Most die within years. There is no cure.
The disease has reduced Regis Kleiss, 28, a formerly thick-bodied shot and discus thrower and captain of the track team at Dondero High School, to a bony echo of himself. Paralyzed except for some minor movement of his head, he will spend his final days on a feeding tube.
ALS leaves its victims' minds intact.
"It's a miserable, damned disease," his mother said.
Now, a clinical trial overseen by the University of Michigan may provide hope. It is cutting-edge and audacious work -- the only ALS trial so far in which neural stem cells are injected directly into a patient's spinal cord. So far, 15 patients have undergone the procedure -- two of them twice -- as the FDA monitors its safety.
One patient showed a remarkable improvement for a while, though U-M's Dr. Eva Feldman, who heads the research, cautions not to read too much into that. The other 14 showed no improvement.
The trail is tentative and early. But when the rest of a person's life has been compressed to an expectancy of two to five years, it is hope nonetheless.
The trial has been based in Atlanta since 2010, but U-M has requested approval from the U.S. Food and Drug Administration to expand it and move it to the University of Michigan in Ann Arbor.
The trial involves injecting 500,000-1 million stem cells into the spine. The ancestry of the cells dates to the spinal cord of an aborted fetus in 2000. The cells are different from the embryonic stem cells that were the subject of a controversial ballot proposal in Michigan in 2008, when voters approved lifting the ban on embryonic stem cell research.
Feldman and others theorize that these new cells act as nursemaids to damaged nerve cells, sending out repair signals, and somehow halting the progression of the disease.
The procedure worked in rats. It has been shown to be safe in pigs.
If the FDA approves moving the trial to Ann Arbor, Michigan patients will have access to an experimental treatment that not only might offer insight into a disease that kills an estimated 15 Americans a day, but also push back the battle lines against other neurodegenerative diseases, such as Parkinson's and Alzheimer's or Huntington's.
For patients: We appreciate your interest in our stem cell trial and as this article mentions we are working to bring it to Michigan. However, we are not at this time able to accept patient applications. Please check this site monthly for any updates.
Taubman Emerging Scholar Dr. James Dowling makes breakthrough in congenital myopathy research
Dowling and colleagues find new gene mutation associated with congenital myopathy
About 50 percent of congenital myopathy cases currently do not have a known genetic basis, presenting a clear barrier to understanding disease and developing therapy, says James Dowling, M.D., Ph.D., the paper’s co-senior author and assistant professor of Pediatric Neurology at the University of Michigan’s C.S. Mott Children’s Hospital. Finding a new myopathy gene opens the possibility of providing a genetic explanation for disease in these individuals where no genetic cause is currently known.
In addition, “the identification of a new myopathy gene is an essential first step towards understanding why this disease occurs and how we combat its effects.” says Dowling, a Taubman Emerging Scholar who worked with Margit Burmeister, Ph.D. and her team from the University of Michigan’s Molecular and Behavioral Neuroscience Institute to study the new myopathy gene (CCDC78).
Dowling says the gene, which has not been studied previously, is an important potential regulator of muscle function and, in particular, part of an important muscle structure called the triad.
“Many myopathies and dystrophies have abnormal triad structure/function, so finding a new gene product involved in its regulation will help researchers better understand the triad and its relationship to muscle disease,” Dowling says.
Congenital myopathies are clinically and genetically heterogeneous diseases that typically become evident in childhood with hypotonia and weakness. They are associated with impaired mobility, progressive scoliosis, chronic respiratory failure and often early death.
Currently there are no known treatments or disease modifying therapies for congenital myopathies.
The researchers performed linkage analysis followed by whole exome capture and next generation sequencing in a family with congenital myopathy. They then validated the gene mutation and provided insights into the disease pathomechanisms using the zebrafish model system.
Dowling says the researchers’ next step is to further model the disease using zebrafish, in the hopes that this knowledge can be translated into therapy development.
“The study provides the first descriptions of the zebrafish model, and gives insight into how we will use it,” says Dowling, who also is director of the Pediatric Neuromuscular Disorders Clinic at C.S. Mott Children’s Hospital.
“Once we develop and characterize a model of the disease, we can then use it as a platform for therapy development.”
Dr. Eva Feldman receives prestigious U-M honor
Distinguished Faculty Achievement Award recognizes outstanding service
PNR&D Director Eva Feldman, M.D., Ph.D., is among five 2012 recipients of the University of Michigan's Distinguished Faculty Achievement Awards. This program honors senior faculty who have consistently demonstrated outstanding achievements in the areas of scholarly research and/or creative endeavors, teaching and mentoring of students and junior faculty, service and other activities which have brought distinction to themselves and to U-M.
Six new U-M stem cell lines added to national registry
Six new human embryonic stem cell lines derived at the University of Michigan have just been placed on the U.S. National Institutes of Health’s registry, making the cells available for federally-funded research.
U-M now has a total of eight cell lines on the registry, including five that carry genetic mutations for serious diseases such as the severe bleeding disorder hemophilia B, the fatal brain disorder Huntington’s disease and the heart condition called hypertrophic cardiomyopathy, which causes sudden death in athletes and others.
Researchers at U-M and around the country can now begin using the stem cell lines to study the origins of these diseases and potential treatments. Two of the cell lines are believed to be the first in the world bearing that particular disease gene.
The three U-M stem cell lines now in the registry that do not carry disease genes are also useful for general studies and as comparisons for stem cells with disease genes. In all, there are 163 stem cell lines in the federal registry, most of them without major disease genes.
Each of the lines was derived from a cluster of about 30 cells removed from a donated five-day-old embryo roughly the size of the period at the end of this sentence. The embryos carrying disease genes were created for reproductive purposes, tested and found to be affected with a genetic disorder, deemed not suitable for implantation and would have otherwise been discarded if not donated by the couples who donated them.
Some came from couples having fertility treatment at U-M’s Center for Reproductive Medicine, others from as far away as Portland, OR. Some were never frozen, which may mean that the stem cells will have unique characteristics and utilities.
The full list of U-M-derived stem cell lines accepted to the NIH registry includes:
- UM9-1PGD – Hemophilia B
- UM17-1 PGD – Huntington’s disease
- UM38-2 PGD - Hypertrophic Cardiomyopathy (MYBPC3)
- UM15-4 PGD - Hydroxysteroid Dehydrogenase 4 Deficiency, a rare hormone disorder
- UM11-1PGD - Charcot-Marie-Tooth disease Type 1A
- UM4-6 – no disease gene
- UM14-1 – no disease gene
- UM14-2 – no disease gene
“Our last three years of work have really begun to pay off, paving the way for scientists worldwide to make novel discoveries that will benefit human health in the near future,” says Gary Smith, Ph.D., who derived the lines and also is co-director of the U-M Consortium for Stem Cell Therapies, part of the A. Alfred Taubman Medical Research Institute.
“Each cell line accepted to the registry demonstrates our attention to details of proper oversight, consenting, and following of NIH guidelines,” says Sue O’Shea, Ph.D., professor of Cell and Developmental Biology at the U-M Medical School, and co-director of the Consortium for Stem Cell Therapies.
U-M is one of only three academic institutions to have disease-specific stem cell lines listed in the national registry, says Smith, who is a professor in the Department of Obstetrics and Gynecology at the U-M Medical School. The first line, a genetically normal one, was accepted to the registry in February.
Each line is the culmination of years of preparation and cooperation between U-M and Genesis Genetics, a Michigan-based genetic diagnostic company. This work was made possible by Michigan voters' November 2008 approval of a state constitutional amendment permitting scientists to derive embryonic stem cell lines using surplus embryos from fertility clinics or embryos with genetic abnormalities and not suitable for implantation.
The amendment also made possible an unusual collaboration that has blossomed between the University of Michigan and molecular research scientists at Genesis Genetics, a company that has grown in only eight years to become the leading global provider of pre-implantation genetic diagnosis (PGD) testing. PGD is a testing method used to identify embryos carrying the genetic mutations responsible for serious inherited diseases.
Genesis Genetics performs nearly 7,500 PGD tests annually. Under the arrangement between the company and U-M, patients with embryos that test positive for a genetic disease now have the option of donating those embryos to U-M if they have decided not to use them for reproductive purposes and the embryos would otherwise be discarded.
The agreement was worked out between U-M's Smith and Mark Hughes, M.D., Ph.D., founder and president of Genesis Genetics and a pioneer in the field of pre-implantation genetic diagnosis. "These are very precious cells, and it would be unconscionable not to take advantage of such an opportunity for medical science and the cure of disease," Hughes says.
The hemophilia B line also resulted from a collaboration with the Oregon Health Science University, and is believed to be the first of its kind in the world. Through the partnership with the Reproductive Endocrinology and Infertility division, headed by Philip Patton, M.D., and the work of David Battaglia, Ph.D., the single embryo was frozen at OHSU and shipped to Michigan.
Contributors to the A. Alfred Taubman Medical Research Institute's Consortium for Stem Cell Therapies include the Taubman Institute; the Office of the Executive Vice President for Medical Affairs; the Office of the Medical School Dean; the Comprehensive Cancer Center; the Department of Pediatrics and Communicable Diseases; the Office of the Vice President for Research; the School of Dentistry; the Department of Pathology; the Department of Cell and Developmental Biology; the College of Engineering; the Life Sciences Institute; the Department of Neurology; and U-M's Michigan Institute for Clinical and Health Research.
To see the stem cell lines currently listed in the NIH registry, visit http://grants.nih.gov/stem_cells/registry/current.htm For more information about stem cell research at U-M, visit http://www.umich.edu/stemcell
Couples who might be interested in donating embryos for U-M research, and their physicians, may learn more at http://www.stemcellresearch.umich.edu/donation/index.html
Big House, Big Heart
Annual fundraiser at Michigan Stadium draws 10,000
Even a rainy day cannot dampen the spirits of those truly inspired to make a difference for their favorite charities!
About 10,000 participants braved thunderstorms on April 15 to run, walk or wheel toward the finish line inside Michigan Stadium. Organizers say the 5K alone had over 7,000 people running in it.
Taubman Institute Director Dr. Eva Feldman's lab won the university's Go Blue! Challenge for best participation in the medical center division! Thanks to all who contributed to ALS research and other good causes.
Braylon Edwards Foundation Celebrity Basketball Game
Thanks to the support of sponsors, players and fans, the 2nd Annual Braylon Edwards Foundation Celebrity Basketball game was a big hit.
Not only was the event fun and exciting, it benefitted U-M researchers racing toward a treatment for ALS.
Amyotrophic lateral sclerosis – also known as Lou Gehrig’s disease – is a terrifying and fatal condition that currently has no cure. It progressively destroys the nerves that control voluntary muscle movement, leaving patients unable to move, speak and ultimately unable to breathe. Scientists are focusing their attention on the disproportionate number of athletes who have the disease – like its most famous sufferer, baseball great Lou Gehrig – and have uncovered evidence that sports-related head injuries may be connected to onset.
Funding from the Braylon Edwards Foundation will help Dr. Feldman to continue this groundbreaking ALS research.
FDA approves next step in stem cell trial directed by Taubman Institute’s Dr. Eva Feldman
The Food and Drug Administration has approved the next phase of a clinical trial of stem cell therapy for ALS patients being conducted by Eva Feldman, M.D., Ph.D., director of the A. Alfred Taubman Medical Research Institute.
In the trial’s first phase, Feldman and her collaborators injected stem cells into the lumbar area of spinal cords of 12 patients with amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease). There have been no adverse side effects related to the procedure.
Going forward, the FDA has approved administering the injections into the cervical region of patients’ spines.
“We have been very encouraged by the early transplantations,” said Eva Feldman, MD, PhD, Principal Investigator of the trial and an unpaid consultant to Neuralstem “Cervical injections are essential, because therapy in this region is key to helping patients with their breathing. The deterioration of these functions most dramatically affect the patients’ quality of life and ultimately their life expectancy.”
Dr. Feldman is the Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System.
The Phase I trial to assess the safety of Neuralstem's spinal cord neural stem cells and intraspinal transplantation method in ALS patients has been underway since January 2010. The procedures are taking place at Emory University.
Data recently presented to the FDA on the first 12 patients showed that eight out of ten living subjects in the trial had lower extremity function scores that remained the same or improved after treatment. Two out of ten showed continued decline of lower extremity function.
The trial will now progress to cervical spine injections. The first three of these will receive unilateral injections in the cervical region of the spine. The next three will receive bilateral injections in the cervical region.
For additional information, see the Neuralstem press release.
Taubman Emerging Scholar Dr. James Dowling Wins $350,000 Grant
Dr. James Dowling, director of the U-M Muscular Dystrophy Clinic, has been awarded a $350,000 grant from the Muscular Dystrophy Association.
The group said it made the award to support Dowling's research into potential therapies for myotubular myopathy, a disease that affects skeletal muscles and also leads to respiratory problems and eye-muscle weakness.
Dowling is the Taubman Institute's Frances and Kenneth Eisenberg Emerging Scholar and an assisistant professor of pediatrics, communicable diseases and neurology.
To read the entire article click here.
Dr. Brian Callaghan Receives Endowed Career Development Professorship
Dr. Brian Callaghan, a U-M neurology professor and ALS researcher, has been named the first Fovette E. Dush Early Career Professor by the University of Michigan Medical School’s Department of neurology.
Callaghan received the five-year appointment at a reception Dec. 7 hosted by Dr. James O. Woolliscroft, dean of the U-M medical school.
The professorship, intended to support the study of neurodegenerative muscular diseases, was established with funds donated by the late Ms. Dush, a Battle Creek native known for her investing acumen and lifelong generosity.
“This professorship will help me jump-start my career here, focusing on neuropathy,” said Callaghan. “We’ll be both looking for new treatments and for how we can do the best for patients with what we already have.”
Callaghan is associate director of the U-M ALS clinic, which treats patients suffering from amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease. His ongoing ALS projects include an analysis of DNA changes that may explain why patients develop ALS and a stem cell project studying neurons and other cells derived from ALS patients. Other current research interests are focused on determining the optimal evaluation of peripheral neuropathy.
“Brian is one of our most promising researcher-physicians,” said Dr. Eva Feldman, director of the ALS clinic, director of the Taubman Institute and the Russell N. DeJong professor of neurology at U-M. “We are delighted that his work has been recognized and that he’s received the great honor of being selected as the first recipient of this professorship.”
Ms. Dush graduated from Battle Creek Central High School in 1937; she completed secretarial studies at Argubright Business College and then began a lifelong career as an administrative assistant at the Battle Creek Federal Center. According to family members, she was an active artist, gardener and volunteer as well as a stylish woman who drove only Cadillacs yet lived frugally in a modest family farmhouse until her early 80s, when she moved to assisted living.
Known as a careful investor who read every prospectus from cover to cover, Ms. Dush consulted U-M doctors about health problems in her older years and – impressed with what she heard and saw in Ann Arbor – made plans for a substantial bequest to the medical school. Ms. Dush died in 2010 shortly after her 90th birthday.
Eva Feldman to Lead American Neurological Association
Eva Feldman, M.D., Ph.D., became president of the American Neurological Association on Sept. 27 at the organization's 136th annual conference in San Diego, Calif.
Feldman, director of the A. Alfred Taubman Medical Research Institute and a professor of neurology at the University of Michigan, will serve a two-year term as head of the 1,800-member ANA, which was established in 1874 by a group of physicians aiming to promote training and research in the field of neurology.
"It's a great honor to lead a distinguished group of neurologists who share my mission of furthering the research and education that will enable us to find ground-breaking therapies and cures for diseases of the nervous system," says Feldman, whose faculty appointment at U-M is named for the late Russell N. DeJong, the longtime head of the U-M medical school's neurology department who also served as ANA president in 1965.
Feldman previously has served as a vice president for the ANA, and she is recent past president of the Peripheral Nerve Society. At U-M, she has an active clinical practice, serves as the research director of the ALS Clinic, and directs the Program for Neurology Research and Discovery, a team of 30 scientists.
Feldman has been listed among the Best Doctors in America for 10 consecutive years.
In her own work, Feldman focuses on amyotrophic lateral sclerosis (ALS – also known as Lou Gehrig's disease) and the neurological complications of diabetes. She is a leader in applying stem cell research to human disease; most notably she is the Principal Investigator of the first clinical trial of intraspinal transplantation of stem cells in patients with ALS. This Phase 1 trial is currently under way at Emory University in Atlanta, GA. Feldman's laboratory has begun the work of adapting this therapeutic approach for patients with Alzheimer's disease.
As director of the Taubman Institute, Feldman spearheads the program which provides unrestricted funding to Taubman Scholars -- leading researchers seeking cures for conditions and diseases ranging from diabetes to cancer to obesity. The Institute also supervises the Consortium for Stem Cell Therapies and several other initiatives.
Dr. Eva Feldman of the University of Michigan Begins Work On a Stem Cell Treatment for Alzheimer’s Disease
Alzheimer’s Disease is a devastating neurodegenerative disease characterized by dementia and memory loss. It afflicts more than 5 million people in America alone, exacting a terrible toll not only on patients and their families, but on the health-care system as a whole.
The University of Michigan Program for Neurology Research & Discovery, under the direction of Eva Feldman, M.D., Ph.D., has proposed a stem cell approach to Alzheimer’s Disease, which may for the first time bring relief to those suffering from the terrible affliction. It is based on a stem cell therapy that Dr. Feldman pioneered for the treatment of ALS (also known as Lou Gehrig’s Disease) and that is undergoing human clinical trials at Emory University in Atlanta.
“There are similarities between ALS and Alzheimer’s Disease that make us believe that our stem cell therapy should prove effective for both,” says Dr. Feldman. “Both diseases affect the same kind of neuron, though in different parts of the body.
“If the treatment works for ALS, we think it will also nurture and protect neurons under attack in Alzheimer’s Disease.”
The cells under siege in both diseases are cholinergic neurons. In the case of ALS, they are located in the spinal cord of patients, impairing the connections they make with muscles throughout the body.
Dr. Feldman has shown that injecting a type of stem cell called a neuronal precursor cell into animal models of ALS has decreased the progression of the disease. Neuronal precursor cells can be grown in tissue culture. They continue to divide and, under the appropriate conditions, mature into specific types of neurons.
Dr. Feldman has demonstrated that these cells, when injected into animals with ALS, are able to protect cholinergic motor neurons that otherwise would have been lost to the disease. That is the approach being tested in the human clinical trials at Emory.
In Alzheimer’s Disease, the cholinergic neurons are situated in a portion of the brain known as the basal forebrain. These neurons create a network that connects areas of the frontal lobe, which regulate personality and behavior, with areas of the temporal lobe, which regulate learning and memory. Loss of these neurons is thought to be a major cause of Alzheimer’s Disease.
Dr. Feldman’s work on Alzheimer’s Disease will begin with an animal model of disease, and if successful, progress to human clinical trials. Neuronal precursor cells will be directly injected into the cholinergic centers of the brain in animals with the disease.
Her team of researchers will then assess the animals’ behavior and the rate of disease progression and correlate these measures with the survival and connectivity of the injected stem cells.
Dr. Feldman believes that the neuronal precursor cells will protect the cholinergic neurons, allowing them to maintain important connections within the brain and slowing the disease progression.
The neuronal precursor cells that will be used in these studies have already been approved by the FDA for human use. Therefore, positive results in the human clinical trials for ALS will have a direct bearing on their use to treat Alzheimer’s Disease.
D Business Profiles Dr. Eva Feldman
Leading Metro Detroit business magazine explores the groundbreaking science of Dr. Feldman and her laboratory team.
To read the entire article click here.New Partnership to Speed Pace of Cures
The Program for Neurology Research & Discovery is now an affiliated program of the Michigan Institute for Clinical and Health Research (MICHR).
As part of the agreement, the Program will have access to MICHR's clinical research support, including the Michigan Clinical Research Unit, which supports human tests of new treatments and medications, as well as its biorepository, educational programs and more.
“Adding the Program for Neurology Research & Discovery to MICHR's affiliations and alliances is beneficial to each of the constituencies we serve, and I am very pleased that we are bringing together our collective expertise and resources," said Ken Pienta, M.D. and MICHR Director.
“As we continue to look for ways to enable and enhance clinical and health research, partnering with excellent programs like Eva Feldman's Program for Neurology Research & Discovery help accelerate the pace of scientific discovery at U-M."
Dr. Feldman, M.D., Ph.D, the Director of the Program for Neurology Research & Discovery, is equally excited about the new collaboration.
“We are so pleased to be working with MICHR, whose mission and ideals we share,” she said. “It will help provide the resources we need to quicken the pace of turning our research into new treatments for our patients.”
MICHR seeks to enable and enhance the positive impact of clinical and translational research at the University of Michigan, and to make U-M a world leader in translating scientific discoveries into real health gains.
First U.S. stem cells transplanted into spinal cord
from CNN – For the first time in the United States, stem cells have been directly injected into the spinal cord of a patient, researchers announced Thursday, January 21.
Doctors injected stem cells from 8-week-old fetal tissue into the spine of a man in his early 60s who has advanced ALS, or amyotrophic lateral sclerosis. It was part of a clinical trial designed to determine whether it is safe to inject stem cells into the spinal cord and whether the cells themselves are safe.
Eva Feldman Elected President of ANA
Eva Feldman, M.D., Ph.D., director of the Program for Neurology Research & Discover, has been named president-elect of the American Neurological Association, a professional society of academic neurologists and neuroscientists.
The announcement was made at the association's annual meeting Oct. 11-14 in Baltimore. She will become president in two years.
The association educates physicians in the neurosciences and works to better understand and treat nervous system diseases.
At U-M Medical School, Feldman also directs the A. Alfred Taubman Medical Research Institute, the ALS Clinic and the Juvenile Diabetes Research Foundation Center.
The first stem cell trial for ALS treatment wins FDA approval
New therapy that U-M neurologist helped develop will undergo Phase I trial at Emory University
U.S. Food and Drug Administration gave the green light Friday for a clinical trial of a new stem cell treatment for amyotrophic lateral sclerosis (ALS). University Michigan neurologist, Eva Feldman, M.D., Ph.D., will be the overall principal investigator for the first human clinical trial of a stem cell treatment for ALS, a fatal neurodegenerative disease.
The FDA approved an Investigational New Drug application from Neuralstem, Inc., a Rockville, Md.-based biotech company, to test the safety of a treatment in which patients will receive injections of the company’s patented neural stem cells at multiple sites along the spinal cord.
Director of the U-M ALS Clinic and the Director of the Program for Neurology Research & Discovery, Feldman worked with a team of neurologists and with Neuralstem Inc. to develop the protocol for delivering the stem cells into the spinal cord of patients.
The Phase 1 trial to determine the safety of the treatment is expected to take place exclusively at Emory University in Atlanta, Ga., subject to approval by its Internal Review Board.
“We are very excited about this clinical trial,” said Feldman, the DeJong Professor of Neurology at the U-M Medical School. “This is a major stride forward in what still could be a long road to a new and improved treatment for ALS.
“ALS is a terrible disease that ultimately kills by paralysis. In work with animals, these spinal cord stem cells both protected at-risk motor neurons and made connections to the neurons controlling muscles. We don’t want to raise expectations unduly, but we believe these stem cells could produce similar results in patients with ALS,” Feldman said.
ALS, also known as Lou Gehrig’s disease, affects about 30,000 Americans. It progressively destroys the neurons that control voluntary muscles, leaving affected people unable to move or speak. There are no known treatments for the disease that slow its progression.
The trial will ultimately consist of 18 ALS patients with varying degrees of the disease. The FDA has approved the first stage of the trial, which consists of 12 patients who will receive five-to-ten stem cell injections in the lumbar area of the spinal cord. The patients will be examined at regular intervals post-surgery, with final review of the data to come about 24 months later.
Jonathan Glass, M.D., director of the Emory Neuromuscular Laboratory, is expected to be the site principal investigator for the trial.
Individuals interested in further information on the trial should contact Emory Health Connection, 404-778-7777, or 1-800 75EMORY, or go to www.neurology.emory.edu/als
Institutional review boards at U-M and Emory University must first approve the protocol.
If Phase I results are favorable, the treatment will need to prove effective in Phase II and III trials and win final FDA approval before it can be available for public use.
Funding: Neuralstem, Inc. plans to conduct and fund the Phase I trial of its patented technology.
Patents/conflict disclosures: Dr. Feldman has no financial interest in or financial arrangement with Neuralstem.
Other coverage:
Big Ten Network video
Stem cell research has the potential of transforming what we know about human biology and how we treat a host of human diseases.
The University of Michigan’s Program for Neurology Research & Discovery is at the forefront of this new technology.
You can see the progress this team of scientists is making in stem cell research in a new video, produced by Michigan Television as part of its “Out of the Blue” series.
This revealing look at stem cell research will be shown two more times this week on the Big Ten Network, a cable channel that broadcasts features and sports from the universities that comprise the conference.
The show will air at:
Thursday, August 6 at 9:30 p.m.
Sunday, August 9 at 10 a.m.
You can also watch the video here.
It takes viewers inside the laboratory to examine the research the Program team is conducting to understand the biology of stem cells and put them to use in developing new therapies for disorders, such as ALS, diabetes and Alzheimer’s disease.
It also talks to a patient with ALS, whose courage and love for family lend a sense of urgency to the work being doing in the laboratory.
As this video so dramatically demonstrates, researchers are on the verge of a new tomorrow in medical science.
U-M launches new embryonic stem cell research consortium
High magnification image of human embryonic stem cells differentiated into neurons (red cells) by treating cells with a growth factor. These could be used to study the development of the nervous system, birth defects or to replace cells lost to injury, aging or diseases such as Parkinson’s. Courtesy of Sue O'Shea, PhD
ANN ARBOR, Mich. --- The University of Michigan today announced the formation of a consortium to create new embryonic stem cell lines that will aid the search for disease treatments and cures.
The A. Alfred Taubman Medical Research Institute Consortium for Stem Cell Therapies is the first major embryonic stem cell research program launched in Michigan since the Nov. 4 passage of a state constitutional amendment allowing scientists to create new stem cell lines using surplus embryos from fertility clinics.
The launch of this center, combined with the recent state law change and President Obama's executive order loosening restrictions on federal funding for embryonic stem cell research, is expected to transform embryonic stem cell research at the University of Michigan.
"The Consortium for Stem Cell Therapies will catalyze efforts by world-class scientists at the University of Michigan who are devoting their full talents to the search for new treatments and cures,” said U-M President Mary Sue Coleman. “At the University of Michigan, we believe stem cell research offers one of our best hopes for finding new treatments and cures for a wide variety of diseases.”
The center will be based at the MedicalSchool, and the work is expected to begin this spring. Funding commitments of nearly $2 million have been secured to start the program, and additional fund-raising efforts are underway.
The consortium will develop new embryonic stem cell lines for researchers and clinicians. In addition, collaborations are being negotiated between the U-M and its University Research Corridor partners, Michigan State University and Wayne State University. Collaborations are also in the works with Oakland University, U-M Dearborn and Case Western Reserve University in Ohio, said center co-director Sue O’Shea, professor of cell and developmental biology.
“We’re talking about an initiative that extends beyond the University of Michigan,” O’Shea said. “This center will provide critical resources to other institutions while building partnerships that could grow to become regional in scope.”
In addition to deriving new embryonic stem cell lines, researchers will use recently developed techniques to convert adult skin cells into induced pluripotent stem cells, known as iPS cells. These reprogrammed cells display the most scientifically valuable properties of embryonic stem cells, while enabling researchers to bypass embryos altogether.
A top priority of the consortium is to derive new lines of human embryonic stem cells and iPS cells that carry the genes responsible for inherited diseases. These cell lines will be used to probe the causes and progression of disease, and to test potential therapies. Likely early disease targets include neurological conditions such as amyotrophic lateral sclerosis (Lou Gehrig’s disease), Huntington’s and Alzheimer’s, as well as diabetes.
“There are very few university programs in the United States that are deriving new embryonic stem cell lines, and even fewer focusing on disease-affected lines,” said consortium co-director Gary Smith,associate professor of obstetrics and gynecology who directed the U-M fertility clinic for the past decade.
“Our special niche will be creating, studying and understanding normal and abnormal development of disease-affected lines,” Smith said. “And these lines will be invaluable in the clinic, as well as the laboratory. We’ll use the latest derivation techniques to ensure that these lines qualify to be used, some day, on patients participating in clinical trials.”
Smith said the consortium will leverage one U-M’s core strengths: interdisciplinary collaborative research. The new center will build on existing partnerships between scientists and clinicians at the MedicalSchool, the Life Sciences Institute, the College of Engineering, the School of Dentistry, the School of Public Health, the College of Literature, Science and the Arts, and the Gerald R. Ford School of Public Policy.
“The work that we’ve done with embryonic stem cell lines to date is important, but it’s really the tip of the iceberg compared to what the future holds,” he said.
Embryonic stem cells are the body’s master cells; they replicate endlessly and form the more than 200 cell types of the human body. Scientists hope these remarkably versatile cells – and the iPS cells that mimic them -- can someday replace faulty cells or diseased tissues in failing organs. This fledgling field is known as regenerative medicine, and the new consortium positions the University of Michigan to play a leadership role, said Doug Engel, chair of the cell and developmental biology department and chair of the new consortium’s scientific advisory board.
“In addition to enabling important new science and clinical work, this consortium puts us in an incredibly strong position to pursue any new federal funds that become available for embryonic stem cell research, and to recruit the brightest young scientists in the field, Engel said. “This initiative will help move the University of Michigan to the forefront of every aspect of stem cell biology.”
The U-M consortium will be among the first groups in the country to derive new embryonic stem cell lines that are linked to a data base containing genetic and medical-history information about the embryo donors and their families. The data base will enable researchers to examine how the disease genes in a given cell line have manifested themselves in previous generations.
“In my Program for Neurology for Research & Discovery, our scientists study a spectrum of diseases --- ALS, Alzheimer’s, diabetic complications, childhood muscle diseases, to name a few,” said Dr. Eva Feldman, director of the U-M’s A. Alfred Taubman Medical Research Institute.
“We will now be able to obtain stem cell lines to better understand the cause and develop new therapies for these diseases," Feldman said. “Can you imagine what a powerful tool stem cells will be?”
Human embryonic stem cell work has been underway for several years at the University of Michigan. O’Shea currently directs the MichiganCenter for Human Embryonic Stem Cell Research, which stores and studies cell lines approved by the Bush administration, and trains researchers to use them.
The new consortium will supersede that center, which formed in 2002. While taking on the additional tasks of deriving embryonic and iPS stem cell lines, the new consortium will retain many of the core services provided by its predecessor. The consortium will be a cell-line repository, and staff members will train other scientists to work with the lines.
At U-M, human embryonic stem cell work is also underway at the Center for Stem Cell Biology, launched in 2005 and located at the Life Sciences Institute. Researchers at the LSI-based center work with both adult and embryonic stem cells.
Sean Morrison directs the Center for Stem Cell Biology and will serve on the new consortium’s scientific advisory board. He said the two entities are complementary; both contribute to the larger goal of bolstering U-M’s stem-cell research program.
“We’ve been laying the groundwork for this consortium for years, and everything is finally coming together exactly the way we had hoped,” Morrison said.
“By improving the facilities and resources available for embryonic stem cell research at the University of Michigan, this new consortium will enhance our ability to recruit new faculty to the Center for Stem Cell Biology. So the new consortium is one important element of a larger university plan.”
To create new embryonic stem cell lines, the A. Alfred Taubman Consortium for Embryonic Stem Cell Therapies will use surplus embryos remaining following infertility treatment. Several hundred early stage embryos – which would otherwise have been discarded – have been donated and consented for use in research.
“Embryonic stem cell research is the most important advancement in medical science since the advent of antibiotics a half century ago,” said A. Alfred Taubman, philanthropist, retail pioneer and U-M alumnus. “The creation of this consortium positions the state of Michigan at the forefront of this promising scientific and medical frontier. In doing so, we will make this a healthier world for generations to come.”
So far, funding for the project has been pledged by the A. Alfred Taubman Medical Research Institute, Executive Vice President for Medical Affairs Dr. Robert Kelch, Medical School Dean Dr. James Woolliscroft, the Life Sciences Institute, the College of Engineering, the Department of Cell and Developmental Biology, the Comprehensive Cancer Center, the Department of Pediatrics and Communicable Diseases, the Cardiovascular Center, the Department of Neurology, the Department of Pathology, and the Clinical and Translational Science Award consortium.
“Important collaborations like this consortium are critical to meeting medicine’s greatest challenges,” Woolliscroft said. “By partnering with our colleagues in the region, we can accelerate the translation of scientific discoveries into societal benefit.”
The consortium will fully conform to the provisions of the state constitutional amendment approved by Michigan in 2008. Additionally, the U-M will strictly adhere to the guidelines for the conduct of human embryonic stem cell research issued by the International Society for Stem Cell Research.
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