CHỮA BỊNH CỦA NÃO : 
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"Sau 2 lần bị stroke và tổn thương não , tôi xem xét việc nhờ BS thần chết * Kevorkian , " bịnh nhân bịnh lupus đang-lành-bịnh Katherine Hammons nói (hình 1 , đang an ủi một BN bạn) . Thay vào đó , bà đã tham gia 1 cuộc thí nghiệm trong đó nhà ung thư học Ann Traynor , nay làm ở đh Massachusetts , trị bịnh này bằng TBG (tế bào gốc/stem cell) từ chính tủy xương của bà . 6 năm sau khi chữa , sự tiến triển của bà vượt quá trông đợi : Ngay cả tổn thương - nghĩ rằng vĩnh viễn - đang lành . "Tôi đc tái sinh,"bà nói . "Tôi đã gạch tên BS Kevorkian khỏi danh sách . "
Gần đây hơn Traynor đã trị Margaret Laperle (hình 2 , chuẩn bị cho điện não đồ) ; ng vừa bắt đầu hồi phục từ chứng loạn tinh thần (psychosis) - do bịnh lupus - đã làm ngừng trệ (stall) nghề BS của chính BN này .
"Nó cho thấy rằng ngay cả ng với tàn phế lớn lao (tremendous) vẫn có thể phục hồi chức năng," Traynor nói . Theo báo cáo của bà , 75/100 BN của nghiên cứu này vẫn ko tái phát (remain in remission) trong thời gian từ 2 đến 8 năm sau điều trị .
Trong lúc đó , các KH gia ở Stem-Cells ,Inc. tại Palo Alto , bắc Cali đang dùng chuột , hình 1 , để thử nghiệm các giới hạn của TB não người - đã có kết quả đầy hứng khởi (mind-boggling) . Các TBG của hệ thần kinh từ thai nhi ng khi cấy vào não 1 con chột , chúng ko chỉ tồn tại ; chúng bắt đầu hoạt động ngay bên cạnh TB thần kinh của chính con chuột .
" Những TB này ko chỉ sửa chữa những tổn thương tại chổ , nhưng cũng bảo vệ chống lại các bịnh thoái hóa não đang phát triển (progressive neurodegenerative disease) , " ng đồng sáng lập Irving Weissman nói . Những kết quả này giúp các KH gia tiến gần tới việc trị bịnh Parkinson , các rối loạn di truyền tàn phá não , và có lẽ các tổn thương tủy sống , ông nói .
* Rất nổi tiếng ở Mỹ vì giúp BN nan y ra đi thanh thản theo yêu cầu của họ và gđ . Đã gây nhiều tranh cải giửa gíao hội và gđ của BN .

NGHỊCH LÝ : NGƯỜI MÙ MONG ĐƯỢC SÁNG MẮT nhưng có ba triệu đảng viên ĐCSVN thì có mắt nhưng vẫn MÙ , vì họ mù 2 thứ sau : tam quyền phân lập , nhà nước pháp quyền .
"Graziella Pellegrini , Michele de Luca , và đồng nghiệp tại NH Mắt Italia có thể dùng tế bào gốc trưởng thành từ 1 mắt tốt của BN để giúp chửa mắt hư .
Lấy được từ 1 phần của màng sừng/giác mạc (cornea) có tên limbus , những TB này sẽ đc dỗ ngọt (coax) để tạo 1 màng để có thể ghép vào vùng bị thương - của 1 màng sừng bị sẹo do bỏng hóa chất , xem hình 1 . 
Như cơ thể chữa 1 đầu gối bị tổn thương (scrape) , 1 lượng đầy đũ TBG có thể giúp cho mắt tạo các mô mới và khỏe mạnh . Kỷ thuật ghép mô đã cải thiện đáng kể nếu so với ghép mô từ xác chết vì ko bị nguy cơ từ chối của cơ thể bn . 
Bằng cách đưa vào 1 gene phát sáng từ sứa (jellyfish) , bs Prather ở đh Missouri đã tạo heo phát màu xanh , xem hình 2 - có mắt chứa các tế bào được đánh dấu . Khi bs Young và các đồng nghiệp ở Harvard ghép các tế bào võng mạc (retina) từ chuột phát sáng này vào heo bình thường nhưng bị tổn thương võng mạc , họ có thể theo dỏi các TB xanh này và theo dỏi hoạt động của chúng .
"Điều mà chúng làm là tìm tổn thương võng mạc và chữa nó , " Young nói . "Có vẽ tổn thương này hướng dẫn và bảo các TB cách làm ! .'
Kỷ thuật này cực kỳ tinh vi , đòi hỏi mổ xuyên qua 1 lổ nhỏ ở phía sau của mắt . Nhưng qua các thí nghiệm này , việc chữa trị sẽ giúp cho 1 vài con vật lấy lại thị giác" .

Dr. Anthony Atala, MD: New body parts – the shape of things to come?

December 2013

Imagine a world where if the need arose you could order a spare body part to replace a diseased or dysfunctional one, where doctors could cure rather than simply manage chronic, life-threatening diseases. Could this ever become a reality? Dr. Anthony Atala, MD, Director of the Wake Forest Institute for Regenerative Medicine and the W.H. Boyce Professor and Chair of the Department of Urology at Wake Forest Baptist Medical Center, a pioneer in regenerative medicine, believes it can.

Every 30 seconds a patient dies from diseases that could be treated with tissue replacement.

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Dr. Atala is driven by a deep-rooted desire to offer his patients the best possible treatments. “As a surgeon, there is nothing more devastating than being in an operating room and having to replace a piece of tissue or an organ and not having one to replace it with or not having the ideal treatment,” he said. “To create these tissues and organs outside in the laboratory and to have them available would be a really good option for some patients. That’s what has inspired our work.”

Dr. Atala and his multi-disciplinary team of researchers are adopting parallel strategies to find ways to grow the solid organs people need. Using 3-D printing technology the team is working on projects to create biodegradable scaffolds to produce bone, muscle, cartilage and in the longer-term to print a kidney. The team also re-uses discarded organs which after a washing process are repopulated with a patient’s own cells. (Photo: Wake Forest Baptist Medical Center)

Regenerative medicine offers the potential to transform the medical landscape and patients’ lives, offering new treatments for previously incurable conditions. “The ultimate promise of regenerative medicine is not just to help manage disease but to really improve the lives or even provide a cure,” Dr. Atala said.
Unlike established medical practice, regenerative medicine is patient-specific and targets the underlying cause of a disease by repairing, replacing or regenerating damaged cells. While the idea was aired as far back as the 1930s, “it has taken us several decades to get where we are today,” Dr. Atala notes. Just 30 years ago, he explains, it was not possible to grow most human cell types outside the body. “Today we are at a point where we know how to grow human cells, and we know how to expand them outside the body. We are not yet at a stage where we are implanting solid organs but we are implanting flat; tubular; and hollow, non-tubular organs in patients.”

Levels of complexity

Regenerative medicine recognizes four levels of organ complexity. “Flat structures, like skin, are the least complex, made up mostly of one cell type. They are not as complex as a tubular organ, like a blood vessel or a windpipe which has two cell types and architecturally is a little more complex as it remains open. It is really just a tube acting to allow fluid or air to go through it at a steady state within a defined range,” Dr. Atala explained. Hollow, non-tubular organs such as the bladder, offer a third level of organ complexity in terms of cells, shape and function. In 1999, Dr. Atala led a research team that successfully implanted the world’s first laboratory-grown bladder into a patient who today is living a normal, active life. Solid organs such as the kidney, liver and heart are the most complex organ type. With these organs “there are a lot more cells per centimeter and they require massive amounts of blood vessel supply and involve many more cell types,” he explained.
While the first three types of organs - flat; tubular; and hollow, non-tubular organs – have been successfully implanted in patients, using a combination of cells and/or scaffolds made from biodegradable materials, “the goal is to keep increasing the number of organs that we implant and someday to be able to implant solid organs. Every day we are getting closer,” Dr. Atala said.

Growing need for human organs

Regenerative medicine is evolving in response to a real need. The demand for human tissue is growing. “Every 30 seconds a patient dies from diseases that could be treated with tissue replacement,” Dr. Atala observed. Organ transplant waiting lists continue to grow; every 10 minutes someone is added to the transplant list in the US alone. This is a major problem. “Over a period of a decade the actual number of transplants went up by about one percent but the number of patients on the wait list has doubled,” Dr. Atala noted. “We have a major crisis right now because we are living longer and there’s more chance of organs failing. There is really a need to have organs available so we don’t have to wait until someone dies to be able to transplant one.”

One of the major advantages of regenerative medicine is that by harnessing the body’s innate potential to heal and replacing damaged tissues and organs with new ones grown from a patient’s own cells, organ rejection is all but eliminated. Moreover by focusing on the underlying cause of the disease, the aim is to cure a patient rather than simply manage symptoms or stem a disease’s progression. This promises significantly improved quality of life for patients and enormous financial savings for national healthcare systems.

Without intellectual property protection people will not invest in the technology, so if we want to see these technologies used for patients we need to have intellectual property protection. The technologies depend on it.

A scaffold for a bladder seeded with a patient’s cells. In 1999, Dr. Anthony Atala led a research team that successfully implanted the world’s first laboratory-grown bladder into a patient who today is living a normal, active life. (Photo: Wake Forest Baptist Medical Center)

Strategies for creating new solid organs

Dr. Atala and his multi-disciplinary team of 300 researchers are adopting parallel strategies to find ways to grow the solid organs patients need.

3-D printing organs

Using computed tomography (CT) images and computer aided design (CAD) software, researchers have developed 3-D printers that are designed to engineer new organs. “Our printing machines are very much like an inkjet printer but instead of using ink we are using cells in the cartridge and they are laying the cells down one layer at a time where they are needed to create three-dimensional structures that can lead to functionality,” he explained. The team is working on projects such as bone, muscle, cartilage and a long-term project to print a kidney.

Re-using discarded organs

Researchers are also using discarded organs. These are taken to the laboratory where all the existing cells are washed away using mild detergents leaving the three-dimensional structure of the organ intact. “We would then use the structure as a mold to repopulate it with the patient’s own cells,” Dr. Atala explained. “The idea is to take a small piece of tissue from the patient’s diseased organ, isolate the normal cells and put them back into the organ which would then be put back into the patient.”

The constant search for solutions

For Dr. Atala, innovation is a way of life. “The first step to innovation is just to try because if you don’t try you will never find a solution,” he said. “Anytime we see a barrier we have to find ways to get around it,” he noted, underlining the need to constantly re-examine accepted truths and develop new approaches on the basis of the new knowledge and tools available.
“Our job as scientists,” he notes, “is really to develop the technologies. If we can create technologies that are transformational and will make patients better then healthcare providers will want to use them. Then someone will need to invest in the technology and make sure the intellectual property is there. If all these pieces come together, the technology will be produced and it will be used and distributed for patients and their benefit. But it all starts and ends with having a technology that is transformational for our patients.”
Despite significant breakthroughs, regenerative medicine is still in its infancy. “We still have a lot of challenges. So many things have to happen for so many different organs. When you start expanding the number of organs you can engineer, you expand the indications, there are new uses, new inventions, new methods, new processes. The field is really wide open. It is an area where innovation can really take hold,” he said.

The role of intellectual property

A veteran-user of the patent system – he has applied for or received over 200 patents worldwide - Dr. Atala is a firm believer that IP has a key role in enabling and advancing medical technologies and ensuring they benefit patients. “Intellectual property is so important. The bottom line is that unless there is intellectual property present we don’t have a tool to commercialize these technologies,” to make them widely available and bring their cost down. “Without investment the technology will never be transferred to patients. It takes literally hundreds of millions of dollars to produce and distribute these technologies around the world,” he said. “People need to know that they’re going to get a return on their investment. Without intellectual property protection people will not invest in the technology, so if we want to see these technologies used for patients we need to have intellectual property protection. The technologies depend on it.”
The IP system also enables researchers to “put a stake in the ground” making it possible for the research community to keep pace with the state of technological development. “When you know where the technology is from an innovation standpoint you can build on that and create more innovation. This sharing of information is very useful in advancing towards the future,” he said.
Dr. Atala urged policymakers, to explore ways to bring down the costs associated with obtaining global IP protection. “To get world protection is a very expensive proposition, so you don’t want to eliminate someone from using the IP system because the cost is too high,” he said. Dr. Atala also urged policymakers to streamline regulatory processes to help reduce lengthy timelines and contain costs. “Safety is paramount but you can shorten the timeline by taking some of the bureaucracy out of the system,” he said.

Collaborative research

Regenerative medicine is a complex field drawing on multiple disciplines. Researchers at the Wake Forest Institute for Regenerative Medicine share a lab and rigorously test tissues at every stage of development. “Patient safety is of paramount importance to us,” Dr. Atala said. “We are dealing with patient’s lives so whatever we do we have to make sure that at the very least we do no harm and then that we create a benefit,” he said.
Beyond Wake Forest Baptist Medical Center, the Institute is involved in numerous research collaborations (more than 100 national and more than 50 international). “The goal is to create an international network to distribute these cells allowing these technologies to be worked on by many different scientists,” Dr. Atala explained. Such collaboration is also enabling the Institute to build an international network of clinical trial sites. “At the end of the day, this will help advance these technologies for everyone.”

The next big thing

“We are constantly looking out for potential breakthroughs,” he noted, explaining that the next big thing in regenerative medicine is a series of little things. “We are looking at so many different areas, there are so many small challenges to overcome, small victories to achieve to make the next big advances. It all points to implanting solid organs into patients. That is really going to be a major thing,” he said.
“You should never say never,” he reflected. “If a salamander can re-grow a damaged limb, why can’t we? The potential is there in biology to initiate these systems. The question is how can we make it happen and a better question is when? One thing is certain. These technologies do have the potential to make patients better. For us it is not really about the cells we use, or the technologies we choose, it is really all about making our patients better.”
 

Regenerative medicine is ground-breaking because:

• it promises to save lives and improve those of patients suffering from debilitating chronic diseases;
• it signals a move from a one-size-fits-all model to a patient-specific model of healthcare;
• it eliminates the risk of organ rejection;
• it focuses on harnessing the body’s innate capacity to heal and the cause of a disease and could potentially cure certain life-threatening chronic conditions;
• it opens up a new world of medical treatments;
• it has the potential to transform the healthcare landscape and promises to significantly reduce the healthcare costs associated with treating an increasingly aging and ailing population.

Woman grows a nose on her spine after experimental stem cell treatment goes awry

• By Sebastian Anthony on July 8, 2014 at 1:17 pm
• 42 Comments

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A female patient in the US has grown a nose on her back following a failed experimental stem cell treatment that was intended to cure her paralysis. The nose-like growth, which was producing a “thick mucus-like material,” has recently been removed as it was pressing painfully on her spine. If you ever needed an example of the potential perils of stem cell therapy, and just how little we actually know about the function of stem cells, this is it. It’s also notable that this stem cell therapy was carried out in a developed country, as part of an approved trial (apparently unwanted growths are more common in developing nations with less stringent medical safeguards).
Eight years ago, olfactory stem cells were taken from the patient’s nose and implanted in her spine. The stem cells were meant to turn into nerve cells that would help repair the woman’s spine, curing her of paralysis. Instead, it seems they decided to do what they were originally meant to do and attempt to build a nose. Over a number of years, the nose-like growth eventually became big enough and nosy enough to cause pain and discomfort to the patient. As reported by New Scientist, “surgeons removed a 3-centimetre-long growth, which was found to be mainly nasal tissue, as well as bits of bone and tiny nerve branches that had not connected with the spinal nerves.” [DOI: 10.3171/2014.5.SPINE13992 - "Autograft-derived spinal cord mass following olfactory mucosal cell transplantation in a spinal cord injury patient"]

Your olfactory system. 1 is the olfactory bulb (the bit of your brain that processes smells); 6 is the olfactory receptors that bind to specific chemicals (odors). [Image credit: Wikipedia]

As with any experimental procedure, there is always a fairly good chance that something will go wrong. In a 2010 clinical trial, 20 paralyzed patients were treated with olfactory stem cells; 11 showed some signs of recovery, four had “minor adverse events,” one developed meningitis, and in one case the paralysis got worse. Jean Peduzzi-Nelson, a stem cell researcher, says most patients undergoing the olfactory stem cell treatment have a “remarkable recovery,” with “less than 1%” growing an unwanted snotty appendage. [Read: Researchers create brain-computer interface that bypasses spinal cord injury paralysis.]What went wrong, then? Basically, at the top of your nasal passages there is the olfactory mucosa. This region contains all of the machinery for picking up odors, and the neurons for sending all of that data off to your brain’s olfactory bulb for processing. Cells from this region can be easily and safely harvested, and with the correct processing they behave just like pluripotent embryonic stem cells that can develop into many other cell types. These olfactory stem cells could develop into cartilage, or mucus glands, or neurons. The researchers obviously wanted the latter, to cure the patient’s spinal nerve damage — but seemingly they got it wrong, and thus she sprouted a second nose. Moving forward, newer olfactory stem cell treatments have an “isolation” stage to prevent this kind of thing from happening. [Read: The first 3D-printed human stem cells.]
It’s important to note that medicine, despite being carried out primarily on humans, is still ultimately a scientific endeavor that requires a large amount of trial and error. In the western world, it’s very, very hard to get a stem cell therapy approved for human trials without lots of animal testing. Even then, the therapies are often only used on people who have “nothing to lose.” Obviously it’s hard to stomach news like this, and I’m sure that stem cell critics will be quick to decry the Frankensteinian abomination created by these scientists. But when you think about the alternative — no advanced medicine and significantly reduced lifespans for billions of people — then really, such experimental treatments are nothing to sneeze at.

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Lab-Grown Vaginas Provide Normal Sex Lives for Women With Rare Condition

The work of scientists trying to manufacture major human organs like the brain and heart in the lab has generated a lot of buzz, even though it will most likely be decades before the lab-grown organs are exact enough to be transplanted into patients.

But scientists are already successfully replicating some of the less intricate parts of the human anatomy. Two such studies led the editors of The Lancet to trumpet in the most recent issue “Tissue engineering’s green shoots of disruptive innovation.”

The journal marked two sets of results: In one study, Swiss doctors used patients’ cells and a structure made of pig collagen to provide healthy sinus structure in five patients who had lost much of their noses to skin cancer. In another, Anthony Atala, a pioneer in regenerative medicine, documented that young women who received custom-fitted vaginal canals made from scaffolded human cells grown in the lab, saw healthy tissue grow with their bodies and enjoyed normal sex lives 5-8 years after their surgeries.

Okay, it’s a little weird to be talking about vaginas here, but that’s kind of the point. While this work in regenerative medicine lacks the unembarrassed awe that greets lab-grown hearts and brains, the patients’ quality of life — their ability to have normal sex lives — depends on it.

The young women Atala treated suffered from a rare condition, Mayer-Rokitansky-Küster-Hauser (MRKH) syndrome, in which the vagina, and sometimes the uterus, is absent. The girls were between 13 and 18 years old at the time of the surgeries performed in Mexico City between 2005 and 2008. Their subsequent sexual satisfaction was self-reported using a standard set of criteria.

Currently, women with MRKH syndrome undergo dilation of existing tissue or grafts of skin or the tissue that lines the abdominal cavity. But graft shrinkage and infections are common.

“This may represent a new option for patients who require vaginal reconstructive surgeries. In addition, this study is one more example of how regenerative medicine strategies can be applied to a variety of tissues and organs,” Atala said in a statement provided for press.

Though Atala has used stem cells in other processes, in this case, doctors took a tiny sample of vulvar tissue from each patient and used it to cultivate smooth muscle cells and vaginal epithelial cells in the lab. (In other words, they did not first turn the cells into induced stem cells.)

While Atala has used 3D printing in some of his treatments, the scaffolds that gave the tissue its shape were hand-sewn from a decellularised segment of pig intestine. 3D printing would be needed to bring costs down if the number of procedures rises.

The structure was surgically attached to the patients’ reproductive organs. The scaffold gradually biodegraded and the cells expanded and formed normal vaginal walls.

Atala, whose lab was the first to implant lab-grown organs into human patients, earned TED fame for a talk in which he showed off a young man who had received a replacement bladder based on an approach similar to the one used in Mexico City. Research for the MRHK treatment began in the early 1990s and had already shown that once cell-seeded scaffolds are implanted in the body, nerves and blood vessels form and the cells expand and form tissue.

As The Lancet observes, this latest work suggests that many quality-of-life medical issues might be helped using the lower-tech, clinic-ready versions of stem cell-inspired therapies.

Images: Wake Forest University

STEM CELL , BÀI 2 



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STEM CELL .PHẦN MỘT