Scientists are developing new ways to treat diseases with cells, not drugs

Nichelle Obar was when she met her second child last year pregnant, she did not expect that her pregnancy or her child would be the story. But when the 40-year-old food and beverage coordinator of Hawaii and her boyfriend Christopher Constantino she went 18 weeks ultrasound, they learned something was wrong. The heart was bigger than it should be, and there was no evidence that the fluid started and build the organ. Both were signs that the fetus is working hard to pump his body rapidly growing blood and his heart began to fail. Obar doctor knew what might be causing. Obar Constantine and two vectors of an alpha thalassemia are called genetic blood disease, which can lead to dangerously low levels of red blood cells. Red blood cells transport hemoglobin, which binds oxygen and transports it from the lungs other cell in order to feed a smaller number of red blood cells through small amount of oxygen to cells throughout the body. Neither the parents are affected by the disease, but depending on how the combination of their genes could be their sons. If Obar was pregnant with her first child, Gabriel was told the couple, if you have the disease, its prognosis was bleak. “The information we got was that most children do not survive, and if they survive to birth, you might not live long,” said Obar. Gabriel was lucky. The DNA she inherited from her mother and father, her cells do not just give it to hurt the mutation. But immediately after the 18 week ultrasound, her second child, a daughter, was officially diagnosed with alpha-thalassemia. “We were pretty devastated,” said Obar. They did not have many possibilities: the daughter would transfusions in utero just need to improve their chances of being born, even if they survive until birth, they need regular blood transfusions for the rest of their lives in a healthy make blood donors and They are based on owning the low oxygen in it. Your genetic counselor who have other suggestions, but it was a long shot. He had learned only a study at the University of California, San Francisco (UCSF), testing a new way to treat potentially bold alpha thalassemia: a stem cell transplant, given to the baby in utero. In utero stem cell transplants have been tried before for the blood disease, but with limited success. stem cells, that in all the different species develop from blood cells obtained from a bone marrow donor, processed in a laboratory and injected directly into the umbilical vein connecting the fetus to the mother’s placenta. Ideally, the healthy donor stem cells begin to divide and then take over the defective blood cells for the fetus. But bone marrow eliminates be risky in pregnant women, past attempts alpha thalassemia stem cells involved used by fathers who were often rejected. This new study put into question the ethical question: Was it worth the risk for the mother to potentially save the fetus? There was also the possibility that the transplant may damage Obar daughter more than it helped. But based on new studies out suggesting that a fetus would be a mother transplanted cells better tolerated than a father, Dr. Tippi MacKenzie, professor of surgery at UCSF and director of the study, believed it was worth a try. Obar had concerns, but when cells worked as they had expected, she could give her daughter a chance at life, hopefully a normal life free of their disease. She and Constantino decided to try it. His daughter would be monitored, the first fetus in the world of stem cells from his mother to get treatment clinical trial. While the bone marrow stem cells along a cornerstone of cancers of the blood treatment such as leukemias and lymphomas say, is the study Mackenzie to treat the cells from a pregnant woman to extract a fetus in one uterus growing from various innovative use of stem cells to treat list of diseases associated with cells instead of drugs. And the promising studies have slowly more of these treatments based on stem cells closest finally tested in humans. With stem cells, such as those found in bone marrow, taking scientists use what the body does naturally: it generates new. Many of the bodies and the adult body tissues, they are either dying including fat cells and blood, with their own stem job whose only damaged cells and tissues regenerate in older and can be harvested for research and growth outside of the body. Some institutions are not equipped with this great reservoir of stem cells, but above the muscle brain and heart. So more than two decades ago, scientists have found another source of these flexible cells in embryos that clinics were donated for research by in- IVF. They learned how to grow these cells in the laboratory in all body cells. The possibility open that conditions such as heart disease, diabetes and psychiatric disorders may also possibly treated by replacing healthy tissues or organs that are to influence the care and treatments available that do not require drugs or surgery damaged. raised win but using cells from human embryos serious ethical issues; completed due to extract necessary embryonic stem cells, which some felt for years, federal law prevents scientists with state funds to do research on these cells was a living man. Since 2006, scientists have found a detour around these place ethical block. A Japanese team of Shinya Yamanaka of Kyoto University conducted has shown that it is possible to take one of every person’s skin cell has had to cancel her story of life as a skin cell, and gives them the clean slate she embryo turns in a substantial embryonic stem cells without complicated moral origin. induced pluripotent stem cells (iPS cells), these cells are malleable coaxed in a laboratory dish, with the right cocktail of factors always in the cardiac muscle, the cranial nerves or pancreatic cells insulin pumps. Watching these treatments to patients to seek, there have been false starts. In 2009, the FDA approved the first clinical trial of embryonic stem cells, which involved the nerve transplant from stem cells in paralyzed people made cells, the function of the spinal nerves recover. In initial tests with mice, however, the transplanted cells through clumps began to form tumors that had not yet grown enough warnings about the safety of the therapy, the FDA put the study on hold; resume after the trial, decided finally to stop the search is executed, the company him. To try it now with more years of study and experience, prepare scientists whether stem cells could in heart muscle convert dead tissue after a heart attack to replace, for example, or if it can not produce insulin in the pancreas enough can new cells replace the work done in people with type 1 diabetes researchers hope that produce new neurons from stem cells for a day like diseases of the brain such as Parkinson’s disease, which can replace damaged motor nerves in the brain that lead to uncontrollable tremors. “With stem cells we can now get the root cause of a disease and the search for cures, instead of [treatment] begin patch,” says Dr. Deepak Srivastava, director of Rodden Stem Cell Center at the Gladstone Institute and professor at UCSF. Not only can you stem cells lead to new treatments for diseases in which cells can be replaced in trouble, but they can also create a new critical way to study conditions that remain black boxes, because scientists have simply studied the luxury of having living cells. Now the lab across the country has bred so-called mini-brains incubation, consisting of tens of thousands of brain cells from iPS cells to serve as models for the study of psychiatric disorders of autism schizophrenia. could lead to new treatments that knowledge in an area where the therapies so far not been as successful as doctors had hoped. the whole universe of research on stem cells collection of iPS cells for the new use of blood stem cells that the daughter Obar receives from her mother, says Mackenzie, “is to be an incredibly exciting time in medicine, with all these things explode around us. “Wednesday is feeding day for the work Dr. de Jong 300 mini-brain it takes de Jong, a post-doctoral fellow in the department of molecular therapeutics at Columbia University, a few hours by a few micrometers. each ball of waste brain tissue does not suck generated with a pipette last week, taking care not to disturb the cells themselves, and replace the liquid with a liquid pink-orange color of growth factors, diet, nutrients, glucose and protein -building amino acids. cells barely visible on the bottom of small depressions in the laboratory version of neuropsychiatry of a cube tray are somewhere between a poppy and inert and large Pfefferkorn. Made of iPS cells, they were able to understand the first window that goes wrong when psychiatric disorders beat. The treatment of psychiatric disorders is still lagging behind advances in other diseases, mainly because it was almost impossible to get to examine the living brain. were invoked as a substitute neuroscientists mouse brain or human brain tissue slides for people with mental illness have received the dead, as the primary source of data. Now Dr. Sander could Markx, director of Precision Medicine Initiative at Columbia, which oversees the work on the mini-brains de Jong, who hopes for a pioneering study using stem cells lead to find and test new treatments for psychiatric disorders, for first time. So far, the mini-brains contain the same 20,000 genes that each human cell contains DNA and produce all the important proteins that each brain cell would be. (Why, but all the facilities of a whole brain is missing Markx and de Jong prefer to call them “organoids.”) It combines nutrition generates iPS cells from people of the Amish community, the brain tissue balls. Some are healthy people that others affected by a rare genetic disorder of the brain that the symptoms of autism spectrum, mental retardation and related seizures. Stem cells have been developed to study the brain as organoid normal development of the brain affected genetic aberration. “Now we have the opportunity, processes like brain cells [] grow to study and develop and watch them in the laboratory,” says de Jong. He and his team to investigate how closely replicate the actual organoid disease processes in humans and hope to eventually brain cells mini-screen on which promising drugs that can reverse the effects of reversed mutation. Scientists make progress tissue and organ parts in the regeneration of suffering from easily replaceable disease. It was destroyed people with cancer of the trachea or urethral cancers, such as new can grow out of their cells to reduce the risk of transplant rejection. A first study has been completed for the treatment of macular degeneration, in 2014, already shows promising results in patients. The study took embryonic stem cells from IVF embryos growing in the retinal pigment epithelium obtained, the same cells that begin to decay in people with the disease eventually deprive before their eyes. The cells were then introduced into the eyes of patients to replace their retinas errors. After almost two years, plus some reported improvement in more than half of the small number of people who are legally blind, at the beginning of the study in their vision. Treatments also begin use of iPSC technology to use adult stem cells act to act as if she avoids embryonic-ethical concerns that cling to the real type. The process is particularly critical to heal the human heart. for adult heart muscle actions are no longer, or shares of them so rare that when the damaged heart tissue, as in a heart attack, it is not regenerate. Instead, it makes scar tissue, the heart’s ability to pump blood by hindering. But Srivastava of the Gladstone Institute found that in a developing fetus, the heart muscle cells actively dividing to form, the heart, and he isolated four genes that are activated during this time and then passed to the birth of cells stopping of the heart to continue to share. reactivate these genes in healthy adult heart cells made his share again. And turning on embryonic genes, even in the heart of the scar tissue and converted these cells into a new muscle. we can “We have found what is the nature of the box of tools for the production of embryo heart,” he says, “and we have grouped the same signals in reprogram adult cells to support more new heart muscle.” Srivastava says the strategy to be useful not only for the new heart muscle but produced to the cultivation of other types of cells. was co-founder hone Tenaya Therapeutics, at the end of 2016 the technology, and the company is now preparing a treatment testing in patients. These biotech companies based on stem cells are popping up across the country to fight for various types of diseases. In Semma Therapeutics, based in Cambridge, Mass., Douglas Melton, co-director of the Harvard Stem Cell Institute, pursuing ways affected a population of pancreatic cells insulin pumps for people with type 1 diabetes, as his two children to create. His recent studies have shown that cell-based iPS cells can recognize the changing amount of sugar and react effectively and dial up and down how much insulin they produce. But with type 1 diabetes, can not solve the whole problem to replace those cells with new stem cells because the immune system appears to attack the pancreatic cells. So he and his colleagues Semma has developed a way to protect cells in the pancreas that produce insulin newly formed from destruction by enclosed in a membrane that can slip past the immune system. Melton hopes that the test delivery system and its insulin-producing cells from stem cells in the next two years. “Insulin was discovered in 1920, and I like the idea that the 100-year mark, we can injection are carried out by insulin,” he says. How to represent any new technology, the chances that stem cells have been overshadowed by the potential for exploitation. A report in the New England Journal of Medicine published in 2017, described a study in which stem cells generated by retinal cells from fat cells of the patient extracts for the treatment of macular degeneration have been transplanted; It ‘was after three people left in the study were closed with severe loss of vision after treatment. A review of the study showed that the volunteers running the company paid for the experimental treatment study, which is unusual for clinical trials. The review also exposed irregularities in the way people are recruited and informed about the study, and raised the question of exactly receive what types of human cells. “Whatever we do our clinical trial, we would use the therapy we need to make sure,” says Srivastava. Obar fears about the first woman pregnant your own stem cells in a study using her baby thalassemia treated in utero were breastfed quickly while watching blood transfusions on hold. “I have observed on a video machine of stem cells and saw white dots swirling in the needle, the stem cells that was going on inside me,” he says. His daughter received five blood transfusions through an injection into the umbilical vein from Obar belly. “I was overwhelmed by the look of it,” he says. “It ‘was pretty cool.” For Obar that the possibility that stem cells could be obtained was for the daughter of a permanent solution to be worth the pioneer of the risks. And the process seems to work. Before the birth, doctors Obar warned her that her daughter may appear blue when the breaths taken before, and that may seem weaker than other babies. But not only that, also to leave a lusty cry of his daughter, survive the pregnancy, but she was born that immediately Obar a calm mind. Now seven months of age, the child, who called Elianna, the food is good and is working to roll. There is still a chance that they may show some developmental delays and cognitive effects of their condition in the future, but Constantino Obar and hope for the best. Mackenzie is Elianna another blood transfusion once a month, just to be safe, and plans to look carefully for a year for signs continue to control the Obar blood cells begin to fill her daughter. Depending on how it behaves Elianna plans to enroll Mackenzie most expectant mothers whose children are affected by the disorder of the blood in the study. The scientific impact of stem cells to history-making Mackenzie can study still unknown, but the impact on the family are right there in the child’s name. “I have a name fighters would mean he is and what he went through,” said Obar. During pregnancy, it did not seem quite right until they hit the nurse with her daughter before helped in utero blood transfusion. The name of the nurse was Elianna, which means they have learned “God answered.” “It ‘s perfect,” said Obar. This seems to September 24, 2018 issue of time.
Photo copyright Cole Wilson for TIME