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Category Archives: FDA Stem Cell Trials

clinical trial testing the use of human spinal cord stem …

Posted: October 26, 2015 at 1:41 pm

Neuralstem is seeking to treat the symptoms of ALS via transplantation of its NSI-566 human spinal cord stem cells (HSSCs) directly into the gray matter of the patients spinal cord. In ALS, motor neurons die, leading to paralysis. In preclinical animal work, Neuralstem cells both made synaptic contact with the host motor neurons and expressed neurotrophic growth factors, which are protective of cells. View published papers here: 1, 2, 3.

Neuralstem initiated the first FDA-approved stem cell trial for ALS in January 2010, at Emory University. This Phase I safety trial, to evaluate the safety of the NSI-566 cells and surgical technique, was designed to enroll up to 18 patients. The Principal Investigator is Eva Feldman, MD, PhD, 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 Site Investigator is Jonathan Glass, MD, Professor of Neurology, Emory School of Medicine and Director of the Emory ALS Center. The trial was awarded an Orphan Drug Designation by the FDA in February 2011.

In humans, Neuralstem expects that the transplanted cells will:

In a review of the safety data from the initial nine patients, Neuralstem cells were deemed to be safe, with no adverse reactions reported believed to be related to cells or surgical technique.

Neuralstem concluded final surgeries in the companys NSI-566/ALS Phase II trial, primarily evaluating safety, in July 2014. After a six month patient follow-up period, this phase of the study concluded in the first quarter of 2015. A larger control NSI-566/ALS Phase II trial is expected to commence in 2016.

Nine-month Phase II and combined Phase I/II NSI-566 ALS data was presented at the American Neurological Association Annual Meeting by Dr. Feldman, in September 2015. The data showed that the intraspinal transplantation of the cells was safe and well-tolerated throughout the escalating doses, reaching a maximum tolerated dose of 16 million cells via 20 bilateral injections. Further, there appeared to be no acceleration in disease progression due to the therapeutic intervention.

Researchers calculated a 95% confidence limit around the slopes of decline of ALSFRSr scores, forced vital capacity (FVC) and grip strength of the ProAct historical database subjects, and evaluated if trial subjects fell within or outside those limits. 73% of Phase II patients, and 79% of combined Phase I and II patients, fell above the upper confidence limit of the ALSFRSr score. 50% of Phase I and II combined, and 40% of Phase II patients’ forced vital capacity percent predicted fell above the upper confidence limit, compared to the ProAct database. ALSFRSr scores correlated most strongly with FVC preservation, which was the target of the cervical injections. For grip strength control, researchers used the Ceftriaxone (CEF) study database, since grip strength data was not available in the ProAct database. 67% of Phase I and II combined, and 60% of Phase II patients, all at nine months post-intervention, fell above the 95% upper confidence limit.

The Phase II open-label, dose-escalating trial of NSI-566 evaluated 15 ambulatory patients with ALS, averaging a mean duration of disease of 15.5 months. Participants were divided into five dosing cohorts with three patients in each, who received increasing quantities of cells in the cervical region of the spinal cord via bilateral intraspinal injections ranging from two million to eight million cells. The fifth cohort received an additional eight million cells in the lumbar region. There was no control or placebo group included in the trial. The primary endpoint of the study was the safety of the maximum tolerated dose of stem cell transplantation. Secondary efficacy endpoints included stabilization of ALS Functional Rating Scale-revised (ALSFRSr) scores, and assessment of respiratory functioning, grip strength and muscle strength. Nine of the 15 participants in Phase I and all 15 participants in Phase II were included in the 9-month data. The Phase II trial was approved by the FDA to initiate in April 2013, upon conclusion of the landmark Phase I FDA-approved trial to test the safety of the neural stem cells and transplantation surgery in patients with ALS in February 2013. The National Institutes of Health and ALSA committed to generous grants in funding for the Phase II phase of the study.

The Phase I safety trial enrolled 18 patients. The trial began with 12 late- to mid-stage patients who received a series of injections in the L2-L4 lumbar region. The first six patients were all non-ambulatory with permanent paralysis, and then the trial progressed to six patients who were ambulatory. Neuralstem received approval from the FDA to move into the cervical (upper back) stage of the trial in the fall of 2011. The first of six patients in the cervical cohorts to receive stem cells was treated on November 18, 2011, which marked the first FDA-approved intraspinal surgical transplantation of stem cells into the cervical region. The trial then advanced to the final cervical cohort of three patients. The FDA approved the return of three patients from earlier cohorts to receive cervical transplants, making them the first to receive stem cell transplantation in both the lower and upper parts of their spinal cord. The first of these was treated in June 2012, and received five stem cell injections into the cervical region of the back, for a total of 15 injections, including the ten lower-back injections previously received. The last patient in the Phase I trial was treated in August 2012. The trial was designed as a safety trial to treat 18 patients, and concluded six months after the final surgery.

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clinical trial testing the use of human spinal cord stem …

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Human embryonic stem cells clinical trials – Wikipedia …

Posted: October 24, 2015 at 3:45 am

Human Embryonic Stem Cell Clinical Trials[edit] Introduction[edit]

The Food and Drug Administration (FDA) approved the first clinical trial in the United States involving human embryonic stem cells on January 23, 2009. Geron Corporation, a biotechnology firm located in Menlo Park, California, originally planned to enroll ten patients suffering from spinal cord injuries to participate in the trial. The company hoped that GRNOPC1, a product derived from human embryonic stem cells, would stimulate nerve growth in patients with debilitating damage to the spinal cord.[1] The trial began in 2010 after being delayed by the FDA because cysts were found on mice injected with these cells, and safety concerns were raised.[2]

In the United States, the FDA must approve all clinical trials involving newly developed pharmaceuticals. Researchers must complete an Investigational New Drug (IND) application in order to earn the FDAs approval. IND applications typically include data from animal and toxicology studies in which the drugs safety is tested, drug manufacturing information explaining how and where the drug will be produced, and a detailed research protocol stating who will be included in the study, how the drug will be administered and how participants will be consented.[3] Testing for new drugs must successfully go through three phases of research before a drug can be marketed to the public. In Phase I trials, the drugs safety is tested on a small group of participants. The drugs effectiveness is tested during Phase II trials with a larger number of participants. Phase III trials, involving 1,000- 3,000 participants, analyze effectiveness, determine side effects and compare the outcomes of the new drug to similar drugs on the market.[4] An additional phase, Phase IV, is included to continually gain information after a drug is on the market. Gerons IND application for the GRNOPC1 clinical trial, nearly 28,000 pages in length, was one of the most extensive applications ever to be submitted to the FDA.[2]

Before Geron could test GRNOPC1 in humans, tests in animals had to occur. At the University of California at Irvine, Dr. Hans Keirstead and Dr. Gabriel Nistor, credited with the technique used to develop oligodendrocytes from human embryonic stem cells, injected the cells into rats with spinal cord injuries. The condition of the rats improved after treatment.[5]

The first patient, identified in an article by the Washington Post as Timothy J. Atchison of Alabama, enrolled in the trial in October 2010. The patient was treated at the Shepherd Center in Atlanta, GA just two weeks after he sustained a spinal cord injury in a car accident. The Shepherd Center and six other spinal centers were recruited by Geron to participate in the clinical trial. The Washington Post reported that Atchison has begun to get some very slight sensation: He can feel relief when he lifts a bowling ball off his lap and discern discomfort when he pulls on hairs on some parts of his legs. He has also strengthened his abdomen. Atchison underwent therapy at the Shepherd Center for three months before returning home to Alabama.[6]

Although Geron initially aimed to enroll ten patients in the trial, only three additional patients were added after Atchison. As specified by Geron, eligible patients had to experience a neurologically complete spinal cord injury within seven to fourteen days prior to enrollment. In addition, patients had to be between the ages of 18 and 65 and could not have a history of malignancy, significant organ damage, be pregnant or nursing, unable to communicate or participate in any other experimental procedures. Participants received one injection of GRNOPC1 containing approximately 2 million cells. Even though the trial has officially ended, Geron will continue to monitor participants for fifteen years.[7]

Although no official results from the trial have been published, preliminary results from the clinical trial were presented at the American Congress of Rehabilitation Medicine (ACRM) conference in October 2011. None of the participants experienced serious adverse events, although nausea and low magnesium were reported. In addition, no changes to the spinal cord or neurological condition were found.[8]

After investing millions of dollars in the research leading up to this trial, Geron Corporation discontinued the study in November 2011 to focus on cancer research. John Scarlett, Gerons chief executive officer, said In the current environment of capital scarcity and uncertain economic conditions, we intend to focus our resources on advancing our two novel and promising oncology drug candidates.[9] The companys stocks fell dramatically to $1.50 per share from $2.28 per share when news of the trials discontinuation became public. A spokesperson for the company said that Geron would save money by ending the trial despite the loss in investors. Because many believed Gerons trial offered hope for advancing knowledge related to stem cells and their potential uses, there was disappointment in the scientific community when the trial was cut short. An article on Bioethics Forum, a publication produced by The Hastings Center, stated, “It is one thing to close a trial to further enrollment for scientific reasons, such as a problem with trial design, or for ethical reasons, such as an unanticipated serious risk of harm to participants. It is quite another matter to close a trial for business reasons, such as to improve profit margins.”[10]

In 2013 Geron’s stem cell assets were acquired by biotechnology firm BioTime, helmed by CEO Michael D. West, the founder of Geron and former Chief Scientific Officer of Advanced Cell Technology. BioTime indicated that it plans to re-start the embryonic stem cell-based clinical trial for spinal cord injury.[11]

Two clinical trials involving derivatives of human embryonic stem cells were approved in 2010. Advanced Cell Technology (ACT) located in Marlborough, Massachusetts, leads the trials aimed at improving the vision of patients with Stargardts Macular Dystrophy and Dry Age-Related Macular Degeneration. Originally, twelve patients were estimated to enroll at three hospitals in the U.S.; participating institutions included the Casey Eye Institute in Portland, Oregon, University of Massachusetts Memorial Medical Center in Worchester, Massachusetts, and the New Jersey Medical School in Newark, New Jersey.[12] Patients eyes were injected with retinal pigmented epithelial cells derived from human embryonic stem cells. While no definitive findings from this study have been produced, an article published in Lancet in January 2012 stated that preliminary findings appear to be promising. In this article, outcomes from two patients treated as part of the trial were discussed. During the trial, neither patients vision worsened, and no negative side effects were reported.[13]

Phase I/II clinical trials involving retinal pigment epithelial (RPE) cells, derived from human embryonic stem cells, for the treatment of severe myopia were approved in February 2013.[14]

The FDA approved a phase I clinical trial with ViaCyte beta cells derived from human embryonic stem cell for the treatment of diabetes in August 2014.[15] The cells will be delivered in immunoprotective capsules and pre-clinical results in animal models showed remission of symptoms within a few months.[16] The company reported the successful transplantation of the cells in the first of the 40 patients that will be treated under the trial in late October 2014.[17]

As state funding for human embryonic stem cell research grows, t
here seems to be more support for state sponsored clinical trials. In 2010, California committed fifty million dollars to early-stage clinical trials. Although approved trials must take place in California, scientists are hopeful that this funding will bolster future research in the field.[18]

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First stem cell trial for ALS treatment wins FDA approval …

Posted: August 22, 2015 at 6:44 am

ANN ARBOR, Mich. – The 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 U-M 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 http://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.

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Interview with Bioheart CSO, Kristin Comella: trial update …

Posted: August 8, 2015 at 8:41 pm

I invited the Chief Scientific Officer (CSO) of Bioheart, Kristin Comella, to do an interview after hearing some buzzthat this couldbe a critical time for the company and that it might have been recently visited by the FDA.

Note that Comella not just Bioheart CSO, but also the primary instructor for physician training in stem cell methods offered by the company US Stem Cell Training.

Can you update us on how Bioheart is doing with its clinical trials/INDs?

Comella: Our completed clinical trials of MyoCell to date have been primarily targeted to patients with severe, chronic damage to the heart who are in Class II or Class III heart failure according to the New York Heart Association, or NYHA, heart failure classification system. We have completed various clinical trials for MyoCell including the SEISMIC Trial, a 40patient, randomized, multicenter, controlled, Phase IIa study conducted in Europe and the MYOHEART Trial, a 20patient, multicenter, Phase I doseescalation trial conducted in the United States. We were approved by the FDA, to proceed with a 330patient, multicenter Phase II/III trial of MyoCell in North America and Europe, or the MARVEL Trial. Thus far, 20 patients, including 6 control patients, have been treated. Initial results for the 20 patients were released at the Heart Failure Society of American meeting, showing a significant (35%) improvement in the 6 minute walk for those patients who were treated, and no improvement for those who received a placebo. On the basis of these results, we have applied for and received approval from the FDA to reduce the number of additional patients in the trial to 134, for a total of 154 patients. The SEISMIC, MYOHEART, and MARVEL Trials have been designed to test the safety and efficacy of MyoCell in treating patients with severe, chronic damage to the heart.

In addition, we received approval from the FDA to conduct a Phase I safety study on 15 patients of a combined therapy (MyoCell with SDF1) called the REGEN trial. Advancement of the MyoCell and MyoCell SDF1 clinical development programs is contingent, among many factors, upon the Company obtaining access to sufficient funding to execute the necessary clinical trials to achieve proof of efficacy and regulatory authorization to market such products.

Bioheart has spent over $125 million researching cellular therapies for patients and supporting clinical trials. We are committed to bringing more treatments forward and all revenue that is brought into the company is put towards advancing this science. Our FDA phase 3 MARVEL Trial for congestive heart failure patients is budgeted to cost $10 million dollars for 100 patients. Trying to complete double blind placebo controlled trials is very expensive and there is limited funding for companies who are trying to complete these trials.

How did you and Bioheart get interested in stem cell training courses for MDs? Is US Stem Cell Training owned by Bioheart? Is there some common ground between the missions of the two?

Comella: We are currently offering courses for physicians through US Stem Cell Training which is a wholly owned subsidiary of Bioheart. The field of regenerative medicine is expanding very rapidly and physicians may not have adequate exposure to these topics during medical school. Our goal is to provide physicians with the latest research in cellular medicine. The course includes didactic lecture and hands on demonstration with topics including:

The course is constantly updated with new research and data as more studies are published. The goal is to familiarize physicians with the latest research and how this may affect their practice. We also host a monthly webinar series which includes journal clubs and guest lecturers. This field is growing and we want to provide physicians an opportunity to learn more about regenerative medicine. Many patients are asking their physicians questions about regenerative medicine and our hope is to bridge the gap between basic research and clinical practice.

What are your views on the recent FDA draft guidances including on adipose in which the FDA suggested that SVF is a biological drug?

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The FDA Outrageously Claims That Stem Cells Are a "Drug"

Posted: July 10, 2015 at 7:43 am

By Dr. Mercola

Adult stem cells (as opposed to embryonic stem cells, which are at the heart of the stem cell controversy) are a truly exciting part of the future of medicine, with seemingly limitless potential for anti-aging, arthritis and far more…

But the U.S. Food and Drug Administration (FDA) is attempting to regulate stem cell procedures at a Colorado clinic, stating that stem cells are drugs that fall under FDA jurisdiction.

Outrageously, the FDA is making this claim even though the stem cells being used in the procedure come from the patient’s own body, which means they are essentially claiming that they can regulate a part of your body.

What makes stem cells so special is their potential to develop into many different cell types.

When a stem cell divides, it either becomes another type of cell, such as a muscle cell or cartilage, or it remains a stem cell.

Furthermore, these cells act as an internal repair system in many types of tissues, dividing a seemingly indefinite number of times to replenish other cells.

As you age, your stem cells diminish in quality and quantity, so just when you require strong stem cells the most, you’re becoming increasingly deficient. Hence your organs and tissues eventually wear out and need to be restored or replaced.

The procedure in question by the FDA is known as Regenexx-C. It involves taking a sample of your blood and bone marrow, and using a centrifuge machine to separate out the stem cells. The cells are then cultured to create more cells before they are injected back into your body to help with tissue repair. It is the fact that the cells are cultured that the FDA has attacked, saying this makes them “unregulated drugs.”

As a result the clinic now only offers the procedure outside of the United States, whereas within U.S. borders they use a similar procedure that is done in one day and does not involve culturing. This, reportedly, is less effective because fewer stem cells are delivered to the injury site. As for the promise of stem cell treatments in treating osteoarthritis pain, the positive results appear to be significant, including:

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Stem Cells, FDA, and the Edge of Science: Three Expert …

Posted: July 7, 2015 at 12:41 am

Last week I wrote about a pending lawsuit by the FDA against Regenerative Sciences,a medical company thatdevelopeda stem cell treatment for orthopedic injuries. Whileadult stem cell therapies have been fraught with concerns about safetyand efficacy, this case is particularly important not only because it is unprecedented the FDA is attempting toclassify non-embryonicstem cellsas drugs but also because itbrings into focusthe disconnect between the pace of scientific progressand the struggle ofregulatorsto keep up withrapidly evolving technologies.

I spoke withthree people with aunique perspective onwhats at stake a lawyer who specializes in FDA regulation, a lab director of an in-vitro fertilization clinic, and a bioethicist to geta sense of how thecase is viewed intheir industries. One common threadthat emergedfrom these conversations is that the FDA is conflicted, even confused,about how to regulate stem cell-based products.Theprolonged legal battle with Regenerative(it dates back to at least 2008) is duelargely to the fact that the agency recognizes the absurdity of coming down with a one-size-fits-all regulatory approach thatwould apply to every clinicoffering such treatments.

To be sure, stem cell medicine has a terrible reputation.Stem cell therapies thatpurport to treateverything fromshot knees and slipped disks toautism, multiple sclerosis,and Parkinsons diseaseare rightlyregarded by many as modern-day quackery. That is because unscrupulous doctors have seized on the promise of this exciting new science and used it to peddle unproventreatments to desperateand ill-informedpatients.

In 2010, CBS aired a 60 Minutes special titled 21st Century Snake Oil,in whichDoug Sipp, a researcher at the RIKEN Center for Developmental Biology in Japan and a critic of stem cell fraud,who also runs the watchdog blog Stem Cell Treatment Monitor, warned of the dangers of blossoming stem cell medical tourism. That same year, NPR aired a story called Offshore Stem Cell Clinics Sell Hope, Not Science.Andin 2011, the journal Naturereported on Texas governor Rick Perrys crusadetosoften the FDAsposition on stem cellclinics,whose number in the U.S.is estimated to be around a dozen.The International Societyfor Stem Cell Research (ISSCR), a non-profit organization of medical doctors and scholars,alsofunctions as amyth busterfor clinically untestedtreatments. On its website, the group writes that themesenchymal stem cell, which is at the heart of the FDA lawsuit andis used byRegenerative in its procedures,has become a cell of intense interestbut has not yet been shown to have a clear-cut advantage over existing therapies, is not considered a standard of care for any condition and does not have regulatory approval for the routine treatment of any disease.

Against this background, the FDA is grappling with a difficult decision how to curb the proliferation of unproven stem cell treatmentshawked by unconscientious doctors, while recognizingthatthis branch ofmedicine holds alegitimatepromise whose full potential is only beginning to be discovered.

Areta Kupchyk, a partner at the law firm Nixon Peabody who counsels clients on FDA regulation and development of new biotechnologies, including human tissue and stem cells, says cell-basedproductsare tough to regulate because they are individualized and manipulated to different degrees for usesin individual patients.

The FDA has been trying to figure out how to regulate stem cell product development because it is so difficult individual hospitals, individual doctors are performing individual procedures. It has hada very difficult time over the years trying to regulate something thats not a big manufacturing facility. Its very hard to get your arms around something like that as a regulatory agency, Kupchyk told me ina telephone interview.

Shenoted that where minimal manipulation is concerned, the FDA may face a special challenge. One of the arguments in the agencys case for regulating Regenexx, a procedure in which adult stem cells are extracted, cultured in a special serum, and reinjected back into the same patient, is thatthe cells are more thanminimally manipulated.

I can understand why the FDA is focusing on minimal manipulation that is something they have been focusing on for a number of years. But they havent been able to articulate what minimal manipulation means. Theyre regulating on a case-by-case basis and theyre not informing the public well on where they draw the line,Kupchyk said.One good reason theyre not coming down is that this area is rapidly evolving and there are products created out of cells that have never been used or created before. So theyre struggling with new products, they are not sure how they are going to work in the body. This case could be very important in forcing the FDA to define it more clearly.

The number ofscientists and clinicsdeveloping or providing adultstem cell treatmentsis another reason the FDA may beat a disadvantage trying toestablish hard-and-fast new rules, Kupchyk added. If the FDA is successful in this case, its going to raise a lot of questions for everyone out there whos trying to develop new uses for stem cells for patients. There may be a chilling effect, certainly, and its very expensive to test new products [through the formal regulatory framework]. If every single physician or clinic who was doing something similar applied, the FDA would not be able to handle it. Thats another reason theyre not coming down on every single case.

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Stem Cell Research Pros and Cons – About.com Money

Posted: July 3, 2015 at 9:42 am

Spencer Platt/Getty Images News/Getty Images

Debates over the ethics of embryonic stem cell research continue to divide scientists, politicians and religious groups. However, promising developments in other areas of stem cell research might lead to solutions that bypass these ethical issues. These new developments could help win stem cell research more support from those against embryonic stem cell research, since they don’t require the destruction of blastocysts.

The most recent research has shown that there are many options available other than working with embryonic stem cells. Stem cells can be obtained from cord blood or derived by manipulating differentiated cells (i.e. skin cells) to revert them to a pluripotent state. These are alternatives that may help broaden the acceptance of stem cell research.

In November 1998 the first published research paper reported that stem cells could be taken from human embryos. Subsequent research led to the ability to maintain undifferentiated stem cell lines (pluripotent cells) and techniques for differentiating them into cells specific to various tissues and organs.

The debates over the ethics of stem cell research began almost immediately in 1999, despite reports that stem cells cannot grow into complete organisms.

In 2000 2001, governments worldwide were beginning to draft proposals and guidelines in an effort to control stem cell research and the handling of embryonic tissues, and reach universal policies to prevent brain-drains (emigration of top scientists) between countries.

The CIHR (Canadian Institute of Health Sciences) drafted a list of recommendations for stem cell research in 2001. The Clinton administration drafted guidelines for stem cell research in 2000, but Clinton left office prior to them being released. The Bush government has had to deal with the issue throughout his administration. Australia, Germany, UK and other countries have also formulated policies.

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Stem Cells and the FDA

Posted: July 1, 2015 at 11:53 pm

The Manhattan Regenerative Medicine Medical Group is involved with the Investigational use of Autologous Adipose Derived Stem Cells (ADSCs) for clinical research and deployment.

The Manhattan Regenerative Medicine Medical Group is offering patient-funded research to treat individuals with autologous (their own) fat-derived stem cells and are not involved in the use ormanufacturing of any investigational drugs or devices.

The Manhattan Regenerative Medicine Medical Group is not offering stem cell therapy as a cure for any condition, disease, or injury.

No statements or treatments on this website have been evaluated or approved by the FDA.

This website contains no medical advice.

All statements and opinions on this website are provided for educational and informational purposes only.

We do not diagnose or treat via this website or via telephone.

The Manhattan Regenerative Medicine Medical Group does not claim that any applications, or potential applications, using autologous adult stem cell treatments are approved by the FDA, or are even effective.

We do not claim that these treatments work for any listed nor unlisted condition, intended or implied.

It is important for potential patients to do their own research based on the options that we present so they can make informed decisions.

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FDA Grants IND Approval for Phase IIa Clinical Trial Using …

Posted: June 11, 2015 at 4:46 pm

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SAN DIEGO and Epalinges, Switzerland June 9, 2015 Stemedica Cell Technologies, Inc. received the FDAs investigational new drug (IND) approval for a United States-based, Phase IIa clinical study using its allogeneic stem-cell therapy to treat subjects with mild to moderate dementia due to Alzheimers disease (AD), the sixth leading cause of death in the United States. The study is sponsored by Stemedica International, S.A. and will start at the University of California, San Diego (UCSD) under Principle Investigator Douglas Galasko, M.D. and expand to other sites. Stemedica International will provide management and financial support for this clinical trial. The clinical trial is titled “A Phase IIa Multicenter, Randomized, Single-blind, Placebo-controlled, Crossover Study to Assess the Safety, Tolerability, and Preliminary Efficacy of a Single Intravenous Dose of Allogeneic Human Mesenchymal Stem Cells in Subjects with Mild to Moderate Dementia Due to Alzheimer’s Disease.”

This study was approved based on the excellent safety profile of Stemedicas cGMP-manufactured, hypoxically-grown stem cells and on solid pre-clinical data obtained by Stemedica International in cooperation with the cole Polytechnique Fdrale de Lausanne of Switzerland and with a grant from the Swiss government, said Lev Verkh, Ph.D. Stemedicas Chief Regulatory & Clinical Development Officer. He continued We are very proud of Stemedicas clinical program under U.S. INDs for several indications including ischemic stroke, acute myocardial infarction, chronic heart failure, cutaneous photoaging and Alzheimers disease. At the studys conclusion we will understand if our approach is efficacious versus placebo in subjects with Alzheimers-related dementia, as evidenced by neurologic, functional, and psychiatric endpoints.

Stemedicas bone marrow-derived, allogeneic itMSCs are unique because they are grown under hypoxic conditions that more closely resemble the environment in which they live in the body. Compared to other MSCs, itMSCs secrete higher levels of growth factors usually associated with angiogenesis and healing. Stemedica Internationals AD stem cell therapies feature itMSCs, neural stem cells (NSCs) and stem cell factors, which are described in Stemedica Internationals U.S. Patent application #20140286910.

Promising results were achieved during a three-year, intensive, pre-clinical research project supported by a grant from the Swiss Commission for Technology and Innovation (CTI). The research was conducted at the Laboratoire dOptique Biomedicale headed by Professor Theo Lasser at cole Polytechnique Fdrale de Lausanne (EPFL) in Switzerland.

Stemedica Internationals pre-clinical research was led by Chief Scientist Tristan Bolmont, Ph.D. To evaluate the impact of an intravenous delivery of human mesenchymal stem cells on amyloid pathology, the well-established APPPS1 transgenic mouse model of Alzheimers disease was used. Intravenous delivery of itMSC safely reduced cerebral Abeta pathology in APPPS1 animals analyzed one week after the last injection. Both aged and young APPPS1 mice exhibited significantly decreased Abeta amyloidosis following the itMSC treatments. Concomitantly, microglial activation was diminished in aged and young itMSC-treated APPPS1 mice. No increase of vascular amyloid or manifestation of microhemorrhages was observed following the repeated intravenous itMSC delivery. Biodistribution analysis revealed that intravenously delivered itMSC migrate to the brain and could be detected in this organ with the highest value at one hour post-delivery, decreasing after one day and subsequently dropping below detection level at one week after the injection.

According to Alzheimers Disease International, nearly 44 million people have Alzheimers or a related dementia. Alzheimers and dementia are most common in Western Europe followed closely by North America. The global cost of Alzheimers and dementia is estimated to be $605 billion USD, which is close to 1% of the worlds gross domestic product.

We are very excited to take this next step in developing a treatment for this devastating disease, says Nikolai Tankovich, M.D., Ph.D., President and Chief Medical Officer of Stemedica Cell Technologies and Executive Chairman for Stemedica International Our upcoming Phase IIa clinical trial will enable us to make progress towards determining if our stem cell treatment may be able to halt or slow down the progression of Alzheimer’s disease and other forms of dementia.

About Stemedica Cell Technologies, Inc. Stemedica Cell Technologies, Inc. is a global biopharmaceutical company that manufactures best-in-class allogeneic adult stem cells and stem cell factors. The company is a government licensed manufacturer of cGMP, clinical-grade stem cells currently used in US-based clinical trials for acute myocardial infarction, chronic heart failure, cutaneous photoaging, ischemic stroke and Alzheimers disease. Stemedicas products are also used on a worldwide basis by research institutions and hospitals for pre-clinical and clinical (human) trials. Stemedica is currently developing additional clinical trials for other medical indications using adult, allogeneic stems cell under the auspices of the FDA and other international regulatory institutions. The company is headquartered in San Diego, California and can be found online at http://www.stemedica.com.

About Stemedica International S.A. Founded in Epalinges, Switzerland, in 2008, Stemedica International S.A. is a global biotechnology company that develops therapeutic applications for the treatment and prevention of Alzheimers disease and vascular dementia. The company is a subsidiary of Stemedica Cell Technologies, Inc., a global biotechnology company that manufactures adult allogeneic stem cells. Stemedica International has an exclusive license to manufacture and distribute the parent companys allogeneic, ischemia-tolerant mesenchymal stem cell (itMSC) and ischemia-tolerant neural stem cell (itNSC) lines and stem cell factors for Alzheimers disease and vascular dementia indications. The company also has Swissmedic licenses to import, export and distribute Stemedica Cell Technologies cell lines worldwide for human use in approved clinical trials. Manufactured in compliance with cGMP standards, the stem cell lines have a unique, proprietary technology based on the expansion of cells in constant hypoxia, which provides critical benefits in terms of safety, efficacy and scalability. For more information, visit http://www.stemedica-intl.com

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National MS Society FAIL! Repeatedly Refuses to Fund Only …

Posted: April 9, 2015 at 4:40 am

The National Multiple Sclerosis Society (NMSS), by far the largest MS nonprofit organization in the US, has three times rejected grant applications from the Tisch Multiple Sclerosis Research Center of New York, which were submitted in an effort to procure funding for what is now the only FDA approved regenerative stem cell trial being conducted on MS patients in the nation. The trial in question uses a very sophisticated approach to this experimental therapy, arrived at after over a decade of development in the laboratory, in an attempt to repair nervous system tissues damaged by multiple sclerosis (click here). The failure of the NMSS to help fund this trial is, at the very least, extremely disappointing, and should be of great concern to all those who support the organization.

The goal of nervous system repair and regeneration has long been a Holy Grail of MS research, and investigations into using stem cells to achieve this end hold terrific potential. MS patients around the world are eager to see stem cell research accelerated, as dissatisfaction with current treatment paradigms runs rampant in many segments of the MS population. Patients with the progressive subtypes of the disease are especially desperate for innovative new treatment approaches, as no existing therapy has been shown to put a dent in these especially insidious forms of multiple sclerosis.

The level of desperation felt by many members of the MS community has fueled a rapidly growing medical tourism industry largely comprisedof unregulated offshore clinics offering unproven stem cell therapies at prices that can easily amount to many tens of thousands of dollars. The lure of copious bundles of cash to be made by offering such therapies has attracted at least one despicable con artist into the fray, as I wrote about in my last Wheelchair Kamikaze entry (click here). I fear there may be many more lurking in the weeds.

Given the current “Wild West” atmosphere surrounding the commercial MS stem cell industry, it is vitally important that precious research dollars be expeditiously directed to legitimate, scientifically valid stem cell studies being conducted by the best of the best, scientists with proven track records of academic and scientific achievement in the fields of MS and neurodegenerative diseases. Just last week, the Canadian MS society announced that it was providing $4.2 million in funding for a 40 patient trial to be conducted by some of Canadas most respected MS neurologists (click here). While the American NMSS provides fundsfor preliminary research inquiries into the use of stem cells to treat MS, most of them using animal or test tube models of the disease, the organization has inexplicably chosen to reject grant proposals submitted by the Tisch Center, despite the fact that the Centers trial received a hard won FDA approval the lack of which was cited by the NMSS as reason to reject the first grant proposal submitted by the Tisch Center and is using a more sophisticated approach than any other regenerative MS stem cell trial to date.

Most previous attempts at using stem cells to repair MS damage have involved intravenously infusing a basic type of stem cell (called mesenchymal stem cells) back into the patient from which they were taken. The Tisch Center is taking this approach several steps further, using proprietary methods to transform raw mesenchymal stem cells into a type of stem cell known as neural progenitors, which are specific to the central nervous system. The cells are then injected directly into the spinal fluid of trial subjects, where, in theory, they should be more effective at effecting repairs and combating the disease. The first phase of this trial is now underway, and many of the trial’s human subjects (including Richard Cohen, noted journalist and author click here) have started receiving their stem cell injections.

I am a patient at the International Multiple Sclerosis Management Practice (click here), which is the clinic closely associated with the Tisch Research Center. I am therefore very well acquainted with the principal investigators running in this trial. Ive been aware of their arduous efforts in the area of stem cell research almost since the day I became a patient at the clinic, in the summer of 2004, under the care of Dr. Saud Sadiq. The Tisch Multiple Sclerosis Research Center (click here) which is funded entirely by a private, nonprofit foundation that is unaffiliated with any hospital, academic institution, or government agency worked extremely hard at getting the FDA approval before embarking on their trial, submitting applications to the regulatory agency several times before finally getting the coveted approval in August 2013.

Unfortunately, receiving FDA approval and procuring the funding needed to proceed with the trial were two separate battles. In todays tough economic environment research monies are difficult to come by. After receiving their FDA approval, the Tisch Center submitted two subsequent grant proposals for vitally needed research funds to the National Multiple Sclerosis Society, both of which were rejected without any viable explanation. Subsequently, the Tisch Center mounted extraordinary efforts to raise the necessary capital, culminating in a crowd funding effort through the Internet site Indiegogo (click here). The final $300,000 needed to start the trial was raised through this online effort, in large part from donations by patients themselves and those who love them.

With the first phase of the trial now underway, the researchers and administrators at the Tisch Center have their hopeful eyes fixed on phase 2, which is expected to expand the treatment to a greater number of trial subjects using lessons learned during the first phase of the study. This projected expansion will naturally require additional state-of-the-art equipment to be purchased and world-class researchers to be hired, and private fundraising efforts are already underway to procure the financial resources needed to facilitate these necessities, which are expected to require more than $1 million of investment.

Im fully aware that the NMSS does some extraodinarily good work on behalf of the MS community. The organization not only funds research but also provides education and support to tens of thousands of MS patients in the United States, and has innovated many programs designed to help the MS community in a wide variety of ways. I consider several employees of the National Multiple Sclerosis Society friends, and the NMSS workers with whom I am acquainted are wonderful, bighearted, talented people fully dedicated to the cause.

As a patient with progressive MS who is watching himself slowly circle the drain, Im outraged at the NMSSs actions (or lack thereof) in regards to the Tisch Centers ongoing stem cell trial, and as a human being who knows firsthand the dedication of some of the national organizations staff I find myself confused and deeply saddened by the Societys behavior in this regard. If there are politics at work, well, get over it, and if there are some other tangible objections the NMSS has to the Tisch Centers research, by all means, state them loud and clear so they can be properly addressed. Time, and brain, is wasting

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National MS Society FAIL! Repeatedly Refuses to Fund Only …

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