Bone-marrow transplant teenager: 'I feel angry that my community let me down'

There are myriad complex cultural and religious reasons as to why ethnic minority donor rates are so low. We dont fully understand the reasons but this has to change if more lives are to be saved, says Dr Adnan Sharif, a consultant nephrologist at the Queen Elizabeth Hospital in Birmingham and member of the National Black, Asian and minority ethnic Transplant Association (NBTA). Aneesas case is heartbreaking, but unfortunately it is not isolated. There are simply not enough minority ethnic communities donating.

In August 2012, Aneesa the eldest of three siblings who live in Birmingham with their father Manzoor, 46, a purchasing manager for a car company, and mother Resiat, 46, a primary school teacher started suffering from headaches and feeling lethargic. The following month, her GP took a blood test that revealed Aneesas platelet count platelets help blood to clot was critically low, leaving her at risk of excessive bruising and bleeding.

Aneesa was rushed to the citys Queen Elizabeth Hospital, where, two days later, she was diagnosed with aplastic anaemia after further blood tests and a bone marrow biopsy. A potentially fatal disease of the bone marrow, it affects around two people per million and is caused by a deficiency of all three blood cell types red and white blood cells, and platelets. Symptoms include fatigue and a reduced immune system, which can lead to infection and bleeding.

Blood transfusions are the best treatment for serious cases such as Aneesas, and a bone marrow transplant in which a donors healthy stem cells are injected into the patient the only cure. I felt shocked and isolated, recalls Aneesa of her diagnosis. There was no history of the condition in my family and no reason given as to why I had developed it.

She immediately had a 14-hour blood transfusion, and remained in hospital for a month to have further platelet transfusions every three days. Meanwhile, Aneesas brother Eghshaam, 18, and sister Iyla-Rose, six, were tested to see if they could be donors. For bone marrow stem cell transplants to succeed, there needs to be a close match in tissue type between donor and patient.

When it transpired that her siblings tissue types were less than a 50 per cent match, Aneesa was forced to abandon her studies because of her failing health and she was put on the organ donor list.

My doctor warned me there was a shortage of ethnic minority donors, she says. I was surprised. I naively assumed everybody who needed a donor would find one.

By the end of 2012, Aneesa had developed liver and kidney failure a side effect of the anti-inflammatory and immunosuppressive pills she had to take to protect her immune system. I had to have two litres of fluid injected through a drip every day to stop me dehydrating, she says. I grew jealous of friends leading normal lives.

Last January, Aneesas doctors widened their search to include the international bone marrow donor registry, which contains 10 million people. But, unfortunately, the lack of BAME donors is a global problem.

Although the majority of religious leaders have issued statements of support for organ donation, many Muslims still believe that to donate would contravene their religion. There are certain aspects of the Islamic faith such as the emphasis put on the respect of the dead and not defacing the body that suggest you shouldnt donate, explains Dr Sharif. He says that even though bone marrow donation a relatively simple procedure compared with other organ transplants doesnt require the death of the donor, it is viewed with similar suspicion.

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Bone-marrow transplant teenager: 'I feel angry that my community let me down'

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Asterand ready to grow as it moves HQ back to Detroit

Tom Henderson – Stemgent Inc., a Massachusetts-based company that sells products to the stem cell industry, including lines of stem cells, decided to diversify its business two years ago by buying Detroit-based Asterand plc, a tissue bank company that supplies researchers around the world.Stemgent's investors liked the tissue bank business so well they have decided to undiversify. In September …

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Germantown

Company plans for the future of stem cell use

by Samantha Schmieder

Staff Writer

Next Healthcare Inc. of Germantown recently launched a partnership with Arizona Cardinals wide reciever Larry Fitzgerald to promote its newest venture, CelBank Pro to other professional athletes.

Next Healthcares CelBank is the collection of cell samples and storage of their blood, skin or stem cells to be used in the future. Stem cells are unspecialized cells that are able to renew themselves through cell division and can be scientifically manipulated to become another type of cell with a more specialized function. They offer hope to provide new ways to fight disease or injuries, according to the National Institutes of Health.

Essentially we are in the business of banking cells for people, Vin Singh, the founder and CEO of Next Healthcare, said.

While CelBank is geared toward anyone interested in using their own cells later in their life, CelBank Pro is geared toward sports players who are very likely to get injured or just worn down during their career.

Skin cells and stem cells are stored at a healthy time at someones life for later use in regenerative medicine, Singh said.

In 2006 and 2007, Singh, who lives in Boyds, heard about a method in Japan that was able to turn adult skin cells into stem cells. Singh decided to build Next Healthcare around these induced pluripotent stem cells, or iPS cells.

For me that was the real spark. I heard about that and thought, Wow, this is an amazing, revolutionary breakthrough, Singh said. Thats where the idea came from, what can we do with that technology. There has to be something that I can do for consumers to give them an advantage.

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Stem cells used in landmark therapy for failing sight

A Japanese woman with macular degeneration is the first person to be treated with induced pluripotent stem cells, made from her own skin

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Stem cells used in landmark therapy for failing sight

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Japan stem-cell trial stirs envy : Nature News & Comment

JIJI PRESS/AFP/Getty Images

Masayo Takahashi is the first to implant tissue derived from induced pluripotent stem cells into a person.

Its awesome, its amazing, Im thrilled, Ive been waiting for this, says Jeanne Loring, a stem-cell biologist at the Scripps Research Institute in La Jolla, California. She is one of several researchers around the world to welcome the news that a Japanese woman with visual impairment had become the first person to receive a therapy derived from stem cells known as induced pluripotent stem (iPS) cells.

A lot rides on this trial. If the procedure proves safe, it could soften the stance of regulatory bodies in other nations towards human trials of iPS cells, and it could pave the way for treatments for other conditions, such as Parkinsons disease and diabetes. It could also cement Japan, recently plagued by a stem-cell scandal, as a frontrunner in iPS-cell research.

Pioneered in 2006 by Shinya Yamanaka, now director of the Center for iPS Cell Research and Applications at Kyoto University, iPS cells are created by inserting certain genes into the DNA of adult cells to reprogram the cells back to an embryonic-like state. The cells can then be turned into almost any tissue type, much as embryonic stem cells can. But because iPS cells can be derived from a patients own tissue, the hope is that they will dodge some of the controversial aspects and safety concerns of those derived from embryos.

In 2012, Yamanaka received a Nobel prize for his work, and the field has now matured, with teams across the world champing at the bit to test therapies based on iPS cells in people. Loring, for example, uses the cells to create dopamine-producing neurons as a potential therapy for Parkinsons disease, and says that she will start clinical trials as soon as the US Food and Drug Administration (FDA) gives the go-ahead.

Still, tissues made from iPS cells carry their own concerns, and that had stopped any country from approving them for a clinical trial. The bodys immune system could attack them, or they might contain some cells that are still in the pluripotent state and cause cancerous growths although Loring points out that this has not happened with human trials of therapies based on embryonic stem cells, for which the same concerns would apply.

In July 2013, however, Japans regulatory authorities gave the go-ahead for a team led by ophthalmologist Masayo Takahashi at the RIKEN Center for Developmental Biology (CDB) in Kobe to collect cells to be used in a clinical iPS-cell pilot study.

Her team took skin cells from the first patient, a woman in her seventies who had retinal damage owing to a condition known as age-related macular degeneration. The researchers then reprogrammed the skin cells into iPS cells and coaxed the unspecialized cells into becoming retinal tissue. On 8September, Takahashi provided evidence that those cells were genetically stable and safe, a prerequisite for them to be transplanted into the eye. The procedure took place four days later, and RIKEN has reported that the patient experienced no serious side effects.

In this instance, the womans vision is unlikely to improve. However, researchers around the world are watching to see whether the cells stop the retina from deteriorating further and whether any side effects develop. Should the woman experience serious consequences, iPS-cell research could be set back years, much as gene therapy was in 1999 when a patient died in a trial that attempted to use a modified gene to correct a type of liver disease. That wakes me up at night, Loring admits.

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Japan stem-cell trial stirs envy : Nature News & Comment

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Controlling the transition between generations

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Rafal Ciosk and his group at the FMI have identified an important regulator of the transition from germ cell to embryonic cell. LIN-41 prevents the premature onset of embryonic transcription in oocytes poised for embryonic development, thus ensuring a successful passage between generations. This finding also holds promise for efforts to reprogram differentiated human cells into induced pluripotent stem cells.

Fertilization triggers one of life’s most sweeping transitions – the transition from one generation to the next and, most intriguingly for developmental biologists, from unipotent germ cells to pluripotent embryonic cells. During this transition, fully differentiated and totally specialized reproductive cells (oocytes and sperms) morph into embryonic cells with an unlimited potential to give rise, once again, to all the specialized cells in the body. It is as if a seasoned astronaut suddenly gained the potential to become a concert pianist, car mechanic, Formula One pilot, banker, physician or philosopher. A better understanding of this transition is essential for progress in stem cell therapy and regenerative medicine, where scientists are currently trying to coax fully differentiated cells, such as epithelial cells, into a more pluripotent state. Unfortunately, the underlying molecular processes are still largely unknown.

FMI scientists led by Rafal Ciosk have now identified a protein that prevents the premature onset of this transition, thus ensuring a successful passage between generations.

“Interestingly, the majority of ‘molecular roadblocks’ to reprogramming that have been identified so far are epigenetic regulators. Here, we show that – at least in germ cells – LIN-41 may fulfill an analogous role in the cytoplasm,” comments Ciosk.

But the implications of Ciosk’s findings may go beyond the worm: LIN-41 has recently been shown to promote reprogramming of differentiated human cells into induced pluripotent stem cells. “This is the beauty of the worm as a model organism. We can observe and interfere with fundamental processes in living organisms and thus identify candidates which are highly likely to be relevant in similar processes in humans.”

Explore further: Nuclear transfer to reprogram adult patient cells into stem cells demonstrated

More information: Tocchini C, Keusch JJ, Miller SB, Finger S, Gut H, Stadler MB, Ciosk R “The TRIM-NHL Protein LIN-41 Controls the Onset of Developmental Plasticity in Caenorhabditis elegans” PLoS Genet. 2014 Aug; 10(8):e1004533

The capacity to reprogram adult patient cells into pluripotent, embryonic-like, stem cells by nuclear transfer has been reported as a breakthrough by scientists from the US and the Hebrew University of Jerusalem.

A group of researchers from the Centre for Genomic Regulation in Barcelona have described the role of a protein that is crucial for cell reprogramming. The discovery also details the dynamics of this protein as well as its …

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Japan carries out first iPS stem cell implant surgery (Update)

Japanese researchers Friday conducted the world’s first surgery to implant “iPS” stem cells in a human body in a major boost to regenerative medicine, two institutions involved said.

A female patient in her 70s with age-related macular degeneration (AMD), a common medical condition that can lead to blindness in older people, had a sheet of retina cells that had been created from iPS cells implanted.

“It is the first time in the world that iPS cells have been transplanted into a human body,” a spokeswoman for Riken, one of the research institutions, told AFP.

The research team used induced Pluripotent Stem (iPS) cellswhich have the potential to develop into any cell in the bodythat had originally come from the skin of the patient.

Until the discovery of iPS several years ago, the only way to obtain stem cells was to harvest them from human embryos.

“We feel very much relieved,” ophthalmologist Masayo Takahashi, the leader of the project at Riken, told a news conference after the surgery in Kobe.

“We want to take it as a big step forward. But we must go on and on from here.”

In a statement, the institution said that “no serious adverse phenomena such as excessive bleeding occurred” during the two-hour procedure.

The surgery is still at an experimental stage, but if it is successful, doctors hope it will stop the deterioration in vision that comes with AMD.

The patientone of six expected to take part in the trialwill be monitored over the next four years to determine how well the implants have performed, whether the body has accepted them and if they have become cancerous.

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Technology and Science News – ABC News

Get the latest science news and technology news, read tech reviews and more at ABC News.

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Japan carries out first iPS stem cell implant surgery

Japanese researchers Friday conducted the world's first surgery to implant "iPS" stem cells in a human body in a major boost to regenerative medicine, two institutions involved said. A female patient in her 70s with age-related macular degeneration (AMD), a common medical condition that can lead to blindness in older people, had a sheet of retina cells that had been created from iPS cells …

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Japan carries out first iPS stem cell retina surgery

TOKYO: Japanese researchers on Friday (Sep 12) conducted the world’s first surgery to implant “iPS” stem cells in a human body in a major boost to regenerative medicine, two institutions involved said.

A female patient in her 70s with age-related macular degeneration (AMD), a common medical condition that can lead to blindness in older people, had a sheet of retina cells that had been created from iPS cells implanted. “It is the first time in the world that iPS cells have been transplanted into a human body,” a spokeswoman for Riken, one of the research institutions, told AFP.

The research team used induced Pluripotent Stem (iPS) cells – which have the potential to develop into any cell in the body – that had originally come from the skin of the patient. Until the discovery of iPS several years ago, the only way to obtain stem cells was to harvest them from human embryos.

“We feel very much relieved,” ophthalmologist Masayo Takahashi, the leader of the project at Riken, told a news conference after the surgery in Kobe. “We want to take it as a big step forward. But we must go on and on from here.”

In a statement, the institution said that “no serious adverse phenomena such as excessive bleeding occurred” during the two-hour procedure. The surgery is still at an experimental stage, but if it is successful, doctors hope it will stop the deterioration in vision that comes with AMD.

The patient – one of six expected to take part in the trial – will be monitored over the next four years to determine how well the implants have performed, whether the body has accepted them and if they have become cancerous.

AMD, a condition that is incurable at present, affects mostly middle-aged and older people and can lead to blindness. It afflicts around 700,000 people in Japan alone.

The study was being carried out by researchers from government-backed research institution Riken and the Institute of Biomedical Research and Innovation Hospital.

Stem cell research is a pioneering field that has excited many in the scientific community with the potential they believe it offers. Stem cells are infant cells that can develop into any part of the body. Harvesting from human embryos is controversial because it requires the destruction of the embryo, a process to which religious conservatives, among others, object.

Groundbreaking work done in 2006 by Shinya Yamanaka at Kyoto University, a Nobel Laureate in medicine last year, succeeded in generating stem cells from adult skin tissue.

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