Category Archives: Cell Medicine

Last week, a patient with blurry vision in his right eye walked into a doctors office and became the first person to receive reprogrammed stem cells from a donor to treat his age-related macular degeneration.

The patient a Japanese man in his 60s is not alone, as four other patients have been approved for the procedure by Japan's health ministry. The first medical case was reported on March 28 by Nature.

In a one-hour operation by surgeon Yasuo Kurimoto, the patient received skin cells from a human donor at Kobe City Medical Center General Hospital. The donors skin cells were reverse engineered into induced pluripotent stem (iPS) cells. These cells are often seen as a game-changer in the world of regenerative medicine as they have the ability to become almost any type of cell in the body.

In this case, the iPS cells were turned into retinal cells, which were then implanted into the retina of the patient, who has age-related macular degeneration. It is hoped the procedure will stop the progression of the disease, which can lead to blindness. The transplantis not being touted as a cure for the condition, merely a prevention methodfrom further damage.

During the procedure, the surgical team injected 50 microliters of liquid containing 250,000 retinal cells into the patients eye, according to the Japan Times. The real test, however, will be the next phase of monitoring.

What sets this transplant apart is also what makes the recovery process precarious. Doctors will need to keep a careful watch on the patient, as iPS cells from a donor are not a genetic match and could cause an immune rejection.

At this point, you might remember a similar case in 2014 with a Japanese woman at the same hospital. She also received retinal cells derived from iPS cells, however hers were taken from her own skin, not a donor's.

"A key challenge in this case is to control rejection," said Riken researcher Masayo Takahashi to the Japan Times. "We need to carefully continue treatment."

In an update, the team said the Japanese woman was doing well and her vision had not declined. They decided to change track and use donor cells for this study because it holds a more viable future for such transplants.

It's hoped, if all goes well here, that researchers can create a bank of donor stem cells. Such a future would cut down on costs and reduce wait times, as cultivating ones own cells can take several months. However, there's stillmuch to be done.

After the procedure, Takahashi told a press conference that the surgery went well. They will continue to monitor the situation and provide further updates in the future.

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Man Receives Reprogrammed" Stem Cells From Donor In Medical ... - IFLScience

April 4, 2017 by Mirabai Vogt-James T cells (red) that were produced using artificial thymic organoids developed by UCLA scientists. Credit: University of California, Los Angeles

UCLA researchers have created a new system to produce human T cells, the white blood cells that fight against disease-causing intruders in the body. The system could be utilized to engineer T cells to find and attack cancer cells, which means it could be an important step toward generating a readily available supply of T cells for treating many different types of cancer.

The preclinical study, published in the journal Nature Methods, was led by senior authors Dr. Gay Crooks, a professor of pathology and laboratory medicine and of pediatrics and co-director of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, and Amelie Montel-Hagen, an associate project scientist in Crooks' lab.

The thymus sits in the front of the heart and plays a central role in the immune system. It uses blood stem cells to make T cells, which help the body fight infections and have the ability to eliminate cancer cells. However, as people age or become ill, the thymus isn't as efficient at making T cells.

T cells generated in the thymus acquire specialized molecules, called receptors, on their surface, and those receptors help T cells seek out and destroy virus-infected cells or cancer cells. Leveraging that process has emerged as a promising area of cancer research: Scientists have found that arming large numbers of T cells with specific cancer-finding receptorsa method known as adoptive T cell immunotherapy has shown remarkable results in clinical trials.

Adoptive T cell immunotherapy typically involves collecting T cells from people who have cancer, engineering them in the lab with a cancer-finding receptor and transfusing the cells back into the patient.

However, adoptive T cell immunotherapy treatments can be time-consuming, and people with cancer might not have enough T cells for the approach to work, according to Dr. Christopher Seet, the study's first author and a clinical instructor who treats cancer patients in the division of hematology-oncology at UCLA.

Since adoptive T cell immunotherapy was first used clinically in 2006, scientists have recognized that it would be more efficient to create a readily available supply of T cells from donated blood cells or from pluripotent stem cells, which can create any cell type in the body. The challenge with that strategy would be that T cells created using this approach would carry receptors that are not matched to each individual patient, which could ultimately cause the patient's body to reject the transplanted cells or could cause the T cells to target healthy tissue in addition to cancer cells.

"We know that the key to creating a consistent and safe supply of cancer-fighting T cells would be to control the process in a way that deactivates all T cell receptors in the transplanted cells, except for the cancer-fighting receptors," Crooks said.

The UCLA team used a new combination of ingredients to create structures called artificial thymic organoids that, like the thymus, have the ability to produce T cells from blood stem cells. The scientists found that mature T cells created in the artificial thymic organoids carried a diverse range of T cell receptors and worked similarly to the T cells that a normal thymus produces.

Next, the team tested whether artificial thymic organoids could produce the specialized T cells with cancer-fighting T cell receptors. When they inserted a gene that delivers a cancer-fighting receptor to the blood stem cells, they found that the thymic organoids produced large numbers of cancer-specific T cells, and that all other T cell receptors were turned off. The results suggest that the cells could potentially be used to fight cancer without the risk of T cells attacking healthy tissue.

Montel-Hagen said the artificial thymic organoid can easily be reproduced by other scientists who study T cell development. The UCLA researchers now are looking into using the system with pluripotent stem cells, which could produce a consistent supply of cancer-fighting T cells for patients in need of immediate life-saving treatment.

Explore further: Novel 'barcode' tracking of T cells in immunotherapy patients identifies likely cancer

More information: Christopher S Seet et al. Generation of mature T cells from human hematopoietic stem and progenitor cells in artificial thymic organoids, Nature Methods (2017). DOI: 10.1038/nmeth.4237

A new discovery by researchers at the Fred Hutchinson Cancer Research Center in Seattle makes an important step in identifying which specific T cells within the diverse army of a person's immune system are best suited to ...

Adult stem cells from mice converted to antigen-specific T cells -- the immune cells that fight cancer tumor cells -- show promise in cancer immunotherapy and may lead to a simpler, more efficient way to use the body's immune ...

A type of immunotherapy that has shown promising results against cancer could also be used against HIV, the virus that causes AIDS.

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Researchers have developed an artificial structure that mimics the cell membrane, which can switch on immune cells to attack and destroy a designated target. This method has potential to be used as a future cancer immunotherapy ...

People with leukaemia could be helped by new research that sheds light on how the body produces its blood supply.

In a landmark study, researchers at St. Joseph's Healthcare Hamilton and McMaster University have found that providing clinical (low) doses of penicillin to pregnant mice and their offspring results in long-term behavioural ...

Our ability to breathe oxygen is critical to our survival. This process is mediated by the hemoglobin in our blood, which carries oxygen. Since air contains less oxygen on high mountains, the body is under pressure to make ...

UCLA researchers have created a new system to produce human T cells, the white blood cells that fight against disease-causing intruders in the body. The system could be utilized to engineer T cells to find and attack cancer ...

A new method could push research into developmental brain disorders an important step forward. This is shown by a recent study at the University of Bonn in which the researchers investigated the development of a rare congenital ...

A study led by Cardiff University has revealed why CMV - a virus responsible for 1000 birth defects a year in the UK - is so adept at evading the immune system. The new findings could help in the development of treatments ...

Northwestern Medicine scientists have mapped the complete structure of a voltage-gated sodium channel, proteins in the membrane of cells that play an important role in many diseases. The findings were published in Nature ...

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Artificial thymus can produce cancer-fighting T cells from blood stem cells - Medical Xpress

US Stem Cell Inc (OTCMKTS:USRM) is a stock we pointed to back in early March as a potential runner. Weve seen about 230% on the upside since that time, and the company continues to frame its narrative with new catalysts. According to the latest announcement out of USRM, the company has developed a strategic alliance with Advanced Stem Cell Rx (ASC), a US-based provider of regenerative medicine programs, including the development of autologous stem cell treatment centers throughout the US.

Stem cells are critical to our survival and at the core of our regenerative and healing powers. They will play an immense role in redefining the preferred treatment method for the majority of diseases commonly afflicting mankind. We, at ASC, are proud to form a strong alliance with US Stem Cell Inc., one of the oldest and most respected stem cell research and therapy companies in the world, stated Dr J.S. Landow, Managing Director of ASC.

US Stem Cell Inc (OTCMKTS:USRM) bills itself as a company committed to the development of effective cell technologies to treat a variety of diseases and injuries. By harnessing the bodys own healing potential, we may be able to reverse damaged tissue to normal function.

U.S. Stem Cells discoveries include multiple cell therapies in various stages of development that repair damaged tissues throughout the body due to injury or disease so that patients may return to a normal lifestyle.

USRMis focused on regenerative medicine. While most stem cell companies use one particular cell type to treat a variety of diseases, U.S Stem Cell utilizes various cell types to treat different diseases. It is our belief that the unique qualities within the various cell types make them more advantageous to treat a particular disease.

According to company materials, US Stem Cell, Inc. (formerly Bioheart, Inc.) is an emerging enterprise in the regenerative medicine / cellular therapy industry. We are focused on the discovery, development and commercialization of cell based therapeutics that prevent, treat or cure disease by repairing and replacing damaged or aged tissue, cells and organs and restoring their normal function. We believe that regenerative medicine / cellular therapeutics will play a large role in positively changing the natural history of diseases ultimately, we contend, lessening patient burdens as well as reducing the associated economic impact disease imposes upon modern society.

Find outwhen USRM stock reaches critical levels. Subscribe to OracleDispatch.com Right Now by entering your Email in the box below.

As noted above, USRM shares have firmed up once again in part in response to news that the company has formed a strategic alliance with ASC.

According to the recent release, ASC is commercializing many of the proprietary treatments developed by USRM and is currently implementing turnkey programs into qualified practices across the U.S.

Apparently, ASC has contracted with practices in over 20 states in the US, and the company utilizes treatments which employ over 20 years of US Stem Cells international research findings and among the worlds elite cellular scientists and other luminaries in the stem cell field.

Patient selection is critical, with acceptance for treatment based upon patients meeting specific criteria and undergoing a consultation with a member of the clinical team.

The chart shows a bit more than 280% piled on for shareholders of the company during the past month. The situation may continue to be worth watching. USRM stock has a history of dramatic rallies. Whats more, the company has witnessed a pop in interest, as transaction volume levels have recently pushed nearly 310% beyond what we have been seeing over the larger time frame. Thats a continuing explosion of recent interest.

While there is a lot of recent interest, one must remain well-grounded. Not everything is easy to love here. For example, USRM has a chunk ($271K) of cash on the books. But that cash level is dwarfed by a mountain of debt (about $3M in total current liabilities).

That said, USRM is pulling in trailing 12-month revenues of $3.1M. In addition, the company is seeing major top line growth, with y/y quarterly revenues growing at 66.6%. This will likely continue to be a very interesting and lively trading story over the near term, and we will look forward to updating it again soon. For continuing coverage on shares of USRM stock, as well as our other hot stock picks, sign up for our free newsletter today and get our next hot stock pick!

See the article here:
US Stem Cell Inc (OTCMKTS:USRM) Stock Has a Powerful New Weapon - The Oracle Dispatch

An expert in stem cell treatment is warning of dodgy operators in New Zealand offering unproven and potentially dangerous treatments.

Auckland University Medical School lecturerBronwen Connor's warning comesafter arecently-released scientific paper documented a case of three women in the United States who were blinded by an experimental treatment for macular degeneration.

She said many people had stem cell therapy in the belief it was scientifically valid, but that was not often the case.

Dr Connor told Nine To Noon the cells used most often for the treatments were known as adipose cells, which were obtained from fat tissue in the body. They were popular because they couldbe obtained from a patientby liposuction, isolated out, then re-injected for supposed therapeutic use.

"Adipose stem cells obviously have a very important job, but predominantly their job is to make bone and cartilage. They also do have some anti-inflammatory properties. But they, to date, have not been shown to have any potential or ability to generate brain cells, for example, or new kidney cells or heart cells."

Websites for clinics offering the treatments listed up to 20 or 30 different types of diseases, disorders or conditions that one source of cells could supposedly treat. That was worrying, she said.

Adipose stem cells might be the right choice to help repair cartilage damage in the knee. "However, it wouldn't be your stem cell choice if you ... had Parkinson's disease and you were going to try and replace some of those lost cells in your brain."

Dr Connor said people needed to be sceptical and check if, for example, there hadbeen any human clinical trials involving the treatment.

"There are always dangers around treatments that we haven't taken out long-term and which there haven't been sufficient rigorous human clinical trials undertaken [on]. This is the purpose of clinical trials, to see what is the safety aspect and the efficacy of this procedure."

She advisedpeople to think about it in terms of medicines and drugs that people were used to taking, like aspirin. "If you inject yourself with a stem cell population and you have a bad side effect, you can't get those cells out. So ... we really need to know what those cells are going to do long-term and what any potential risk is, because they cannot be retrieved."

The Ministry of Health did not regulate stem cell therapy in this country because the cells were not regarded as medicine. She said it was a grey area, because cells were being taken from a patient and re-injected into the same patient with their consent. "But really, moving forward in the next 10 to 20 years, we're going to see more and more of these type of therapies that don't involve a pill or a tablet or our traditional thought of a medicine, and we really need to get regulations around that type of therapy."

She had spoken to people who paid large sums of money for the treatments.

"When I questioned them a little bit more ... did it help, they would sort of sheepishly say 'well, no not really'."

Dr Connor said clinics offering the treatments often emphasised the benefits according to the scientific literature, but it might be benefits in animals - not from human trials. She wanted quality control addressed and standardisedprocedures, along with safety and efficacy.

Dr Connor wanted to see clinical trials of many of the therapies,as they hadpotential. "Our fear is that, as with the three women with the macular degeneration, is that anything that goes wrong will really hinder the field because people will just see it as stem cell therapy."

Excerpt from:
The risks of unproven stem cell treatments - Radio New Zealand

AlphaMed Press and the Cord Blood Association (CBA) are pleased to announce that STEM CELLS Translational Medicine (SCTM) is now the association's official journal.

Durham, NC (PRWEB) March 31, 2017

AlphaMed Press and the Cord Blood Association (CBA) are pleased to announce that STEM CELLS Translational Medicine (SCTM) is now the association's official journal. With this partnership, SCTM will launch a new journal section dedicated to cord blood research.

"We are delighted to initiate this partnership with the Cord Blood Association," said Anthony Atala, M.D., Editor-in-Chief of STEM CELLS Translational Medicine and Director of the Wake Forest Institute for Regenerative Medicine. "With this new journal section, SCTM further expands the scope of new research reports for our readers with the potential to accelerate progress in regenerative medicine."

CBA is an international nonprofit organization that promotes the work of the cord blood community for the purpose of saving lives, improving health, and changing medicine. The association is the first to join together public and private banks toward a common mission to advocate for cord blood use in order to expand its potentials in cell therapies and regenerative medicine, and to also jointly advocate for global regulations that will allow use of cord blood and cord tissues in medical applications.

SCTM is an international peer-reviewed journal, publishing articles focused on advancing the clinical utilization of stem cell molecular and cellular biology. By bridging stem cell research and helping speed translations of emerging lab discoveries into clinical trials, SCTM will help move applications of these critical investigations closer to accepted best practices and ultimately improve outcomes.

SCTM provides a platform for reporting the latest research on umbilical cord blood and tissue based therapies; engineering and manufacturing of cord blood immune cells; cord blood and cord tissue banking; the use of cord blood and cord tissue in regenerative medicine; and more. Under the leadership of section co-editors, Joanne Kurtzberg, MD, and Karen K. Ballen, MD, the Cord Blood section launches in early April with its first paper on the safety and feasibly of performing autologous umbilical cord blood infusions in young children with autism spectrum disorder.

"The Cord Blood Association is excited and honored to partner with Stem Cells Translational Medicine to showcase the newest and highest quality translational and clinical applications of cord blood and cord tissue based therapies," said Dr. Kurtzberg. "The mission and goals of both parties are aligned to maximize sharing of advances in these novel cell and tissue based therapies."

About Cord Blood Association: The Cord Blood Association will be an international nonprofit organization that promotes the banking and use of umbilical cord blood and related tissues for disease treatment and regenerative therapies.

About STEM CELLS Translational Medicine: STEM CELLS Translational Medicine (SCTM), published by AlphaMed Press, is a monthly peer-reviewed publication dedicated to significantly advancing the clinical utilization of stem cell molecular and cellular biology. By bridging stem cell research and clinical trials, SCTM will help move applications of these critical investigations closer to accepted best practices.

About AlphaMed Press: Established in 1983, AlphaMed Press with offices in Durham, NC, San Francisco, CA, and Belfast, Northern Ireland, publishes two other internationally renowned peer-reviewed journals: STEM CELLS (http://www.StemCells.com), celebrating its 35th year, is the world's first journal devoted to this fast paced field of research. The Oncologist (http://www.TheOncologist.com), also a monthly peer-reviewed publication, entering its 22nd year, is devoted to community and hospital-based oncologists and physicians entrusted with cancer patient care. All three journals are premier periodicals with globally recognized editorial boards dedicated to advancing knowledge and education in their focused disciplines.

For the original version on PRWeb visit: http://www.prweb.com/releases/2017/04/prweb14204527.htm

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Cord Blood Association Names STEM CELLS Translational ... - Benzinga

April 03, 2017 06:02 ET | Source: Cellect Biotechnology Ltd.

Patent to provide coverage in both U.S. and EU

Method of treatment patent provides protection in diabetes, inflammatory bowel disease, graft-versus-host disease and transplant rejection

TEL AVIV, Israel, April 03, 2017 (GLOBE NEWSWIRE) -- Cellect Biotechnology Ltd. (Nasdaq:APOP), (TASE:APOP), a developer of stem cell isolation technology, announced today that it has received a formal notice of Intention to Grant for a patent (Application No. 11751949.6-1466) covering a key method of treatment from the European Patent Office. The allowed claims relate to the engineering of regulatory immune cells with enhanced apoptotic activity to be used for immunomodulation in treating or preventing immune-related disorders.

Earlier this year, Cellect received a Notice of Allowance from the U.S. Patent & Trademark Office for the same patent.

The patent is expected to protect Cellects technology and method when used to treat multiple medical conditions with significant unmet needs, such as diabetes, inflammatory bowel disease, graft-versus-host disease and transplant rejection. The patent covers a segment of cell-based therapeutics that is separate from the one Cellect is currently working in, is relevant to the fast-growing class of immune therapies and Cellect believes may create an opportunity to enhance Cellects pipeline.

Shai Yarkoni, Cellects CEO, commented, Now covering both the U.S. and the EU, this patent is a base for the future commercialization of our global business. Cellect has seven families of patents and patent applications to protect its core assets for enabling stem cell regenerative medicine. With this patent, Cellect has the opportunity to diversify its pipeline and open up new commercialization routes for additional market segments.

About Cellect Biotechnology Ltd.

Cellect Biotechnology is traded on both the NASDAQ and Tel Aviv Stock Exchange (NASDAQ: "APOP", "APOPW", TASE: "APOP"). The Company has developed a breakthrough technology for the isolation of stem cells from any given tissue, a technology that aims to improve a variety of stem cells applications.

The Companys technology is expected to provide pharma companies, medical research centers and hospitals with the tools to rapidly isolate stem cells in quantity and quality that will allow stem cells related treatments and procedures. Cellects technology is applicable to a wide variety of stem cells related treatments in regenerative medicine and that current clinical trials are aimed at the cancer treatment of bone marrow transplantations.

Forward Looking Statements This press release contains forward-looking statements about the Companys expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as believe, expect, intend, plan, may, should, could, might, seek, target, will, project, forecast, continue or anticipate or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. For example, forward-looking statements are used in this press release when we discuss our expectation that the allowed patent will be issued in the EU and in the U.S., that, if approved, the patent will protect Cellects technology and method when used to treat multiple medical conditions with significant unmet needs, such as diabetes, inflammatory bowel disease, graft-versus-host disease and transplant rejection, that the patent may create an opportunity to enhance Cellects pipeline, may serve as a base for the potential future commercialization of Cellects global business and may provide an opportunity to diversify Cellects pipeline and potentially open up new commercialization routes for additional market segments and that Cellects technology is expected to provide pharma companies, medical research centers and hospitals with the tools to rapidly isolate stem cells in quantity and quality that will allow stem cells related treatments and procedures. These forward-looking statements and their implications are based on the current expectations of the management of the Company only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; results in the laboratory may not translate to equally good results in real clinical settings; results of preclinical studies may not correlate with the results of human clinical trials; our patents may not be sufficient; our products may harm recipients; changes in legislation; inability to timely develop and introduce new technologies, products and applications, which could cause the actual results or performance of the Company to differ materially from those contemplated in such forward-looking statements. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading Risk Factors in Cellect Biotechnology Ltd.'s Annual Report on Form 20-F for the fiscal year ended December 31, 2016 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SEC's website, http://www.sec.gov and in the Companys period filings with the SEC and the Tel-Aviv Stock Exchange.

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Cellect Receives Notice of Intention to Grant from European Patent Office for Patent Protecting Company's Technology ... - GlobeNewswire (press...

NEW HAVEN >> Sickle cell disease is a disease of pain, compelling its patients to frequent emergency departments and forcing many sufferers to be admitted to the hospital.

The Sickle Cell Disease Association of Americas southern Connecticut chapter plans to change that outcome, bringing care and treatment to patients in their homes. The organization also plans to push for better screening for couples at risk for passing on the disease and for more research into treatments for suffering patients.

Well be tracking how we can reduce [hospital] admissions as well as emergency room visits, said James Rawlings, chairman of the board of the local organization. He and others in the organization believe that bringing pain relief to patients in their homes will be far more effective than relying on hospital stays and emergency department visits.

This approach is necessary because sickle cell disease is a stubborn, debilitating illness. The only cure is a bone marrow or stem cell transplant, according to the Centers for Disease Control and Prevention.

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Weve plateaued in sickle cell and we just have not had any new treatment approaches in decades, said Dr. Marcella Nunez-Smith, chairwoman of the Medical Advisory Committee for the local association. She calls the progress of research the leaky pipeline its often just a dry pipeline, frankly. Nunez-Smith is professor of medicine and epidemiology and founding director of the Equity Research and Innovation Center at the Yale School of Medicine.

That lack of research into new treatments has been frustrating to those involved in care and treatment of its patients. Overall I think people would say that innovation in treating the disease has been slow, said Dr. Dowin Boatright, a member of the Medical Advisory Committee. I think it has to do a lot with the fact that a lot of people in this country who have the disease have been African American.

Boatright is a clinical instructor in the medical schools Department of Emergency Medicine and a fellow in the Robert Wood Johnson Foundation Clinical Scholars Program.

Patients also carry a stigma, often being seen as drug seeking, he said. In surveys of emergency department doctors, a majority of them will say that they felt that sickle cell patients are addicted to opioids.

I think race does impact that type of interaction, Boatright said.

Advocates for sickle cell disease research are especially frustrating because the cause of sickle cell disease was discovered back in 1951 by Dr. Linus Pauling, who labeled it a molecular disease inherited from two parents who carry the sickle cell trait in their genes.

The disease is the most common inherited blood disorder in the United States, affecting 70,000 to 80,000 Americans, according to the U.S. National Library of Medicine. It occurs in one in 500 African Americans and in more than one in 1,000 Hispanics. It is also found in people of Mediterranean, Latin American and Caribbean descent.

Nunez-Smith called sickle cell a disease of young adults, because life expectancy is only 45. That is an improvement from the 1970s, however, when a typical life span was only 21.

That was the impact of penicillin, Rawlings said. However, sufferers of sickle cell disease may also be afflicted with diabetes and cardiovascular diseases, making treatment complicated.

The disease is caused by a mutation in the sufferers hemoglobin, which causes red blood cells to form in a crescent, or sickle, shape. The cells are unable to deliver enough oxygen to the bodys tissues, causing debilitating pain, said Nunez-Smith. All treatments are basically designed to increase oxygen delivery, she said. Those include administering the drug hydroxyurea and hydration.

One of the mainstays of therapy, hydroxyurea, was developed as a chemotherapy agent, so its a harsh medication with some harsh side effects, Nunez-Smith said. I see that as one of our great pressing issues in terms of sickle cell, is identification of better, newer agents for therapy.

While hydroxyurea manages symptoms of sickle cell disease, many patients resist it because of the side effects, Nunez-Smith said. Those include gastrointestinal problems, hair loss, infertility and a suppressed immune system, making the patient more likely to contract other diseases. Patients are reticent to start therapy because the side effects are so harsh, she said.

And those patients not on hydroxyurea therapy come into hospitals with pain crises, Nunez-Smith said. They dont have medications to keep them well and then, when they get unwell, interacting with the health care system is hard. Sickle cell disease patients average 5.6 hospital admissions per year, she said.

You should have those days just to do what you want, not to be in the hospital, she said.

One of the programs wed like to pilot is thinking about earlier-stage intervention in terms of pain crisis, Nunez-Smith said. If we can deliver them earlier and at home, we might be able to reduce emergency department use and hospital admission and improve quality of life.

If we have a 10 or 15 percent reduction in admissions, thats millions of dollars that are saved by the state, Rawlings said, because 100 days spent in the hospital can cost $1 million, and many patients are on state assistance.

Because of the difficulties of hospital treatment, patients for understandable reasons try to postpone pain care, Nunez-Smith said. The message that the association would like to get across to patients is, at the first inkling of a pain crisis, call us. A nurse can come to your home, she said.

I think patients would like to have more options for pain management and have more options at home as well, Boatright said. In the hospital were able to give stronger medications with an IV and there is potential for doing that with a visiting nurse but thats not standard practice right now, he said.

Boatright said there is an infusion center at Yale New Haven Hospital, where patients can receive opioid medications, but its often crowded, so patients end up in the emergency department.

Probably for the majority of patients theyre not able to access the infusion center during an acute pain crisis, he said.

In the emergency department, patients run into suspicions that theyre opioid addicts. I think its even more problematic in this heated climate that we have for opioid awareness, Boatright said. Last year, 917 people died of opioid overdoses in Connecticut.

Rawlings said the plan for home infusions is ground-breaking. No one else has done it, he said. The hope is that other cities will build on New Havens experience. If they can see the way weve done it other communities across the country can benefit, he said.

In addition to bringing opioid medications to patients in their homes, one of our initiatives is in screening marriage licensing, which isnt routinely done anywhere, said Nunez-Smith. Connecticut no longer requires a blood test to get a marriage license, according to a spokeswoman for the state Department of Public Health, so the only screening is of newborns.

The association wants to distribute information about how sickle cell disease is inherited so couples can make informed decisions about whether to have children. You can be a carrier of sickle cell or you can have sickle cell disease and you can certainly be a silent carrier, Nunez-Smith said. Two people who are unaware they are carriers for sickle cell may have children with the disease.

By and large individuals dont know their status, Nunez-Smith said. They both may be carriers; they dont even know it.

Boatright said many people find out about sickle cell disease either by having sickle cell themselves or knowing someone closely that has sickle cell, but we dont feel theres enough information about sickle cell otherwise.

A third goal is thinking about how we train a broader base of clinicians to provide comprehensive care to patients with sickle cell, Nunez-Smith said.

Its a really diverse, interdisciplinary, interprofessional [effort], she said. We have every kind of patient provider. The help has been available in the community. What we lacked is really the creativity to think about all the resources we already have in place, she said.

She said a fourth goal of the association is raising awareness about the need for more drug discovery and development. If you look across diseases, sickle cell research [receives] a pretty low proportion of funding, she said.

Rawlings compared efforts to find treatments and a cure for sickle cell disease to the early days of the AIDS epidemic, even though sickle cell predates AIDS by 100 years, he said.

This year, an estimated $3 billion in federal dollars will be spent on HIV/AIDS, and $78 million on sickle cell disease, according to the National Institutes of Health.

I think its been an orphan disease in America thats been a disease of minorities The clients are by and large socioeconomically poor and challenged, Rawlings said.

The local sickle cell disease chapter has launched a capital campaign to support its initiatives. Donations can be made at http://www.scdaaofsouthernct.org.

The things that were doing, not only are they innovative but theyre the first time theyre being done in the United States, Rawlings said.

Call Ed Stannard at 203-680-9382.

Read more:
In New Haven, Sickle Cell Disease Association targets home treatment, raising research dollars - New Haven Register

The Wake Forest Institute for Regenerative Medicine and a nonprofit it founded will undertake a five-year, $20 million project to apply advanced manufacturing to regenerative medicine, a process that will speed up the availability of replacement tissues and organs to patients.

Dr. Anthony Atala, the institutes director, talked about the project Friday during a program celebrating its launch at the institutes Biotech Place Atrium at 575 N. Patterson Ave. in downtown Winston-Salem. About 100 people attended the event.

A public-private partnership that involves the U.S. Army Medical Research and Materiel Command awarded the project, the institute said in a statement. The partnership, known as the Medical Technology Enterprise Consortium, awarded $10 million for projects that the Wake Forest institute will perform.

The institute has founded a nonprofit organization to conduct research to advance regenerative medicine manufacturing, the RegenMed Development Organization, which also is the recipient of a $10 million award from the consortium.

Today, we are launching this program of manufacturing to bring regenerative technologies to a broader audience, Atala said to reporters before the program started. We have been working on these projects in regenerative medicine for a number of years.

Now, this is basically a way to accelerate the manufacturing of these technologies to automate the process here. The goal is to stop making these systems by hand one at a time, but to automate the process, so we can scale it up and make the process more affordable so we can get it to patients faster.

The institute, which has 450 scientists collaborating on regenerative medicine research, will focus on two aspects of the project, Atala said. One is to develop standardized bioinks, which is what we use for our printing system. They are biological inks to allow us print tissues, he said.

Researchers with the institute use printers to apply living cells onto biodegradable structures that allow for tissue regeneration inside the body.

For us, at the end of the day this is about one thing how we can best deliver these technologies to our patients, Atala said.

The institute also will focus on developing standardized cell culture media liquids that support cell growth. These products are used in most regenerative medicine projects because of the billions of cells that must be grown for each patient, the institute said in a statement.

We have been spending many years now working on technologies that we re-create tissues and organs in laboratories, and weve been putting these tissues into our patients, Atala said. Now, the next step is how we can we manufacture these tissues on a large scale and provide these tissues to patients all over the country and all over the world.

During the program, retired Army Maj. Gen. Lester Martinez-Lopez told the audience that developing regenerative medicine with advanced manufacturing will help injured U.S. military personnel.

We are going to speed up the production process, said Martinez-Lopez, the president and chairman of the MTEC board. Its about delivering (tissues and organs) to that soldier, sailor, airman and Marine. Thats a big deal.

Doug Edgeton, the president and chief executive of the N.C. Biotechnology Center, said the project will add to the economic base of the Triad and North Carolina. The states life science industry represents $86 billion yearly in economic activity in North Carolina and $2.2 billion in state and local tax revenue, he said.

You all are doing a lot of good things here, Edgeton said to the audience.

After the program, Winston-Salem Mayor Allen Joines said the institutes project will generate jobs locally.

I can see companies coming in and creating jobs and creating jobs within the institute itself, Joines said. It will be a huge impact.

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Wake Forest Institute for Regenerative Medicine will lead $20 million project - Winston-Salem Journal

Jim Durgeloh, 59, was desperate to avoid surgery. After a career as a construction contractor and hours of leisure time spent on a motorcycle around his Longview, Wash., home, he was facing an operation to replace his left hip.

Thats pretty invasive, he said, nervous about a surgery that would require being cut open and implanted with an artificial hip; Jims brother had died from complications after a similar operation. In the search for an alternative, he and his wife, Janet, happened upon the website for La Jolla-based StemGenex Medical Group, which touts itself as the worlds first and only Stem Cell Center of Excellence.

But what caught the Durgelohs attention were the words of Rita Alexander, its chief administrative officer and a founder.

Alexander wrote that she had suffered debilitating rheumatoid arthritis until a stem cell treatment sent her into remission. Today it remains my passion to advocate for those diagnosed with debilitating illnesses to have access to cutting edge stem cell treatment, she wrote.

Rita was very inspiring, Janet Durgeloh says.

Durgelohs doctor in Washington was skeptical about the therapy offered by StemGenex. He didnt think it was going to work, Durgeloh says. The therapy isnt approved by the Food and Drug Administration, which says such treatments are not based on scientific evidence and can be unsafe. Then there was the cost: about $15,000, not including airfare. That wasnt covered by Durgelohs insurance, which would have paid for his hip replacement.

But on a recent Wednesday morning, the Durgelohs were at the DoubleTree hotel in Del Mar, where their bill was paid by StemGenex. Durgeloh was still wearing a bandage on his midriff, where a StemGenex doctor had performed liposuction to obtain stem cells that subsequently were reinjected into his body, ostensibly to regenerate his damaged bones and tissues. They were preparing to fly home, infused with the hope communicated by the clinic staff, who seemed very optimistic, Durgeloh told me.

A lawsuit in San Diego federal court suggests that StemGenex may have given the Durgelohs nothing but hope. Three StemGenex patients two with diabetes and one with lupus say they were misled by the medical groups marketing pitch to pay $14,900 each in 2015 and 2016 for therapies that have had no effect.

The lawsuit, which seeks class-action status, claims that StemGenex has made its money by targeting the ill and the elderly with false, fabricated and purposefully misleading claims about patient satisfaction. Selena Moorer, a lupus patient from Florida, and her two co-plaintiffs say StemGenex has no reasonable basis for its marketing claim that the Stem Cell Treatments were effective to treat diseases as advertised. The lawsuit names StemGenex, Alexander and Andre Lallande, the groups chief medical officer, as defendants. The company denies the claims made in the lawsuit.

Durgelohs treatment was typical of the procedures offered as stem cell therapy. He says he received injections directly into his hips, his ailing knees and his back, with whatever was left over suffused into his body via an IV drip.

Whats most important to know is that theres no accepted scientific evidence that treatments using cells from adipose fat tissue layers work.

But as we reported last year, many clinics offering the treatments capitalize on the publics impression that stem cells have become some sort of medical miracle. Dr. Mehmet Oz warned his vast television audience about this misconception in February, when he aired a lengthy undercover investigation of stem cell clinics and called for government regulation. StemGenex wasnt mentioned in the piece.

StemGenex, in its reply to the Moorer lawsuit, asserts that the plaintiffs cannot prove that its representations regarding the efficacy of its stem cell treatments are actually false. The plaintiffs, it continues, do not cite to a single scientific study that disproves [StemGenexs] advertised claims.

StemGenex may not have to prove that in a court of law, but thats not the way federal regulation works. At nearly $15,000 a pop, the companies should have to show a treatment works.

The FDA has been grappling with this very point in pondering how to regulate the burgeoning industry. There are more than 500 clinics offering stem cell treatments in the U.S., according to a survey released last year by stem cell scientist Paul Knoepfler of UC Davis and bioethicist Leigh Turner of the University of Minnesota.

Right now, theres no consensus how these clinics should be regulated.

In 2015, UC San Diego researchers described stem cell treatment as medicines Wild West. As Hermes Taylor-Weiner and Joshua Graff Zivin observed, Because FDA guidelines are ambiguous, stem-cell clinics have in effect been operating without regulation.

The proliferation of the clinics has forced the FDA to take a closer look.

The government agency maintains that using stem cells extracted from a patients fat requires licensing as a drug, device or biological product, which means the clinics have to demonstrate the products are safe and effective, possibly via a clinical trial.

The clinics obviously disagree. Steven Brody, chief scientific officer of StemGenex, testified at an FDA hearing in September that if the FDA took a hands-off approach, this would help our patients have access to stem cell therapies.

Earlier this month, the New England Journal of Medicine reported the devastating outcome for three elderly women injected with fat-derived stem cells directly into their eyeballs by a clinic in Florida as a treatment for macular degeneration. The treatment left the patients totally or mostly blind.

Stem cell clinics typically are cagey about what patients should expect. They neither claim their treatments are effective nor explicitly state that theyre unfounded, Taylor-Weiner and Zivin observed. Their language is intentionally imprecise and exploits the vulnerability of patients with debilitating diseases.

Indeed, a disclaimer on the StemGenex home page states, Stem cell therapy is not FDA approved, and, StemGenex Medical Group and affiliates do not claim that treatment using autologous stem cells are a cure for any condition, disease, or injury.

Thats a striking admission for a treatment costing nearly $15,000 out-of-pocket and might help explain why health insurers shun the treatments.

The emotional video testimonials from patients posted on the StemGenex website carry disclaimers that the results experienced by those patients may not be typical or expected. You should not expect to experience these results.

When I asked Jamie Schubert, a StemGenex spokeswoman, to point me to a scientific study or any other evidence that its treatments work, she replied that anecdotal feedback from patients indicates that their symptoms have improved and their quality of life has increased.

There are other red flags. One of the medical groups physicians, plastic surgeon Scott Sessions, was placed on three years probation by the California Medical Board in February. He was accused of negligence related to cosmetic surgery and other procedures he performed on two patients at an unrelated facility in 2011 and 2013.

Schubert told me Wednesday that Dr. Sessions has informed us that he is in compliance with all requirements of the probationary terms of the medical board. But the very next day, his name, photograph and bio had disappeared from the StemGenex website. Sessions didnt respond to a request for comment.

The same thing happened with the logo of the American Board of Surgery, which had been prominently displayed on the StemGenex site, implying the company had the certification boards seal of approval. After I mentioned to Schubert that a board official told me that display was a complete misuse of our logo, it vanished. Schubert called it an error.

Peoples health needs are not suitable for unregulated Wild West experimentation, and anecdotal feedback isnt proof that cutting edge treatments are safe and effective. The course couldnt be clearer for the FDA and state medical regulators across the country: If these stem cell clinics are endangering their customers health and draining their pocketbooks for quack remedies, shut them down.

Keep up to date with Michael Hiltzik. Follow @hiltzikm on Twitter, see his Facebook page, or email michael.hiltzik@latimes.com.

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The stem cell therapies offered by this La Jolla clinic aren't FDA approved, may not work and cost $15000 - Los Angeles Times

Durham, NC (PRWEB) March 31, 2017

AlphaMed Press and the Cord Blood Association (CBA) are pleased to announce that STEM CELLS Translational Medicine (SCTM) is now the associations official journal. With this partnership, SCTM will launch a new journal section dedicated to cord blood research.

We are delighted to initiate this partnership with the Cord Blood Association, said Anthony Atala, M.D., Editor-in-Chief of STEM CELLS Translational Medicine and Director of the Wake Forest Institute for Regenerative Medicine. With this new journal section, SCTM further expands the scope of new research reports for our readers with the potential to accelerate progress in regenerative medicine.

CBA is an international nonprofit organization that promotes the work of the cord blood community for the purpose of saving lives, improving health, and changing medicine. The association is the first to join together public and private banks toward a common mission to advocate for cord blood use in order to expand its potentials in cell therapies and regenerative medicine, and to also jointly advocate for global regulations that will allow use of cord blood and cord tissues in medical applications.

SCTM is an international peer-reviewed journal, publishing articles focused on advancing the clinical utilization of stem cell molecular and cellular biology. By bridging stem cell research and helping speed translations of emerging lab discoveries into clinical trials, SCTM will help move applications of these critical investigations closer to accepted best practices and ultimately improve outcomes.

SCTM provides a platform for reporting the latest research on umbilical cord blood and tissue based therapies; engineering and manufacturing of cord blood immune cells; cord blood and cord tissue banking; the use of cord blood and cord tissue in regenerative medicine; and more. Under the leadership of section co-editors, Joanne Kurtzberg, MD, and Karen K. Ballen, MD, the Cord Blood section launches in early April with its first paper on the safety and feasibly of performing autologous umbilical cord blood infusions in young children with autism spectrum disorder.

The Cord Blood Association is excited and honored to partner with Stem Cells Translational Medicine to showcase the newest and highest quality translational and clinical applications of cord blood and cord tissue based therapies, said Dr. Kurtzberg. The mission and goals of both parties are aligned to maximize sharing of advances in these novel cell and tissue based therapies.

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About Cord Blood Association: The Cord Blood Association will be an international nonprofit organization that promotes the banking and use of umbilical cord blood and related tissues for disease treatment and regenerative therapies.

About STEM CELLS Translational Medicine: STEM CELLS Translational Medicine (SCTM), published by AlphaMed Press, is a monthly peer-reviewed publication dedicated to significantly advancing the clinical utilization of stem cell molecular and cellular biology. By bridging stem cell research and clinical trials, SCTM will help move applications of these critical investigations closer to accepted best practices.

About AlphaMed Press: Established in 1983, AlphaMed Press with offices in Durham, NC, San Francisco, CA, and Belfast, Northern Ireland, publishes two other internationally renowned peer-reviewed journals: STEM CELLS (http://www.StemCells.com), celebrating its 35th year, is the world's first journal devoted to this fast paced field of research. The Oncologist (http://www.TheOncologist.com), also a monthly peer-reviewed publication, entering its 22nd year, is devoted to community and hospital-based oncologists and physicians entrusted with cancer patient care. All three journals are premier periodicals with globally recognized editorial boards dedicated to advancing knowledge and education in their focused disciplines.

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Cord Blood Association Names STEM CELLS Translational ... - PR Web (press release)