Sigilon Therapeutics approaches the clinic – Vantage

The haemophilia A programme SIG-001, which has orphan drug status, leads the companys pipeline for rare diseases.Mr Vivaldi has form when it comes to haemophilia: he was formerly chief commercial officer atSpark, one of the groups developing a gene therapy for the disorder and a takeover target of Roche.

Haemophilia A, in which patients cannot produce clotting factor VIII, is the subject of intense gene therapy research, by companies including Biomarin, Sangamo and Spark. Clinical progress was initially highly promising, but long-term effectiveness remains to be proven (Two more patients bolster Sangamos haemophilia A gene therapy, July 6, 2019).

Mr Vivaldi says Sigilons technology could have several advantages over gene therapy, even if these projects do make it through the clinic to market. Haemophilia gene therapies tend to be unsuitable for children, liver or kidney disease patients, or those with pre-existing antibodies to viral vectors. This could exclude up to 80% of patients, he says.

Gene therapies cannot be re-dosed if their efficacy starts to wane; Mr Vivaldi says that patients can be re-treated with SIG-001 if necessary or have some spheres removed if the dose is too high.

The most important potential advantage is cost. Gene therapies are hugely expensive and no satisfactory payment model has so far been worked out. SIG-001is off-the-shelf and,according to Mr Vivaldi, easily scalable and more cost-effective.

Ofcourse this is all academic until SIG-001 justifies itself in human trials.He explains that the studies will be essentially the same as those for haemophilia A gene therapies, with endpoints including factor VIII levels, bleeding rates and quality of life measures. Data could appear by the end of next year or beginning of 2021.

Diabetes

The other main area of the groups research is diabetes. The timing of clinical trials here is under wraps for now owing to the collaboration agreement Sigilon has with Lilly. In April 2018 Lilly paid $63m up front and made what Mr Vivaldi calls a small equity investment in Sigilon. Milestones of up to $410m and high single-digit royalties on worldwide sales are also part of the package.

The cells are intended to provide a functional cure for type 1 diabetes a condition with which Mr Vivaldi himself lives.This is the same goal being chased by Semma Therapeutics, which was bought by Vertex this month and whose projects are also still preclinical (Vertex spends nearly $1bn on Semmas unproven diabetes cell tech, September 3, 2019). Two other companies, Viacyte and Sernova, have posted very early human data.

The central problem with implanted cell therapies is that unshielded cells have tended to work, but prompted an immune response, whereas encapsulated ones are non-immunogenic but have limited efficacy. Mr Vivaldi believes that Sigilons platform can overcome this, but proof is still some way away.

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Research report explores the Animal Stem Cell Therapy Market – Zebvo

Animal stem cell therapy is a usage of animals stem cell to treat a disease or disorder. The ability of stem cell is to divide and differentiate into a cell with specialized function useful for repairing body tissues damaged by injury or disease. The animal stem cell therapy process involve three steps which include collection of stem cell sample from animals and preparing the sample to concentrate the stem cells. Finally, the therapy includes transferring the stem cells into the injured site for treatment. Animal stem cell therapy increases the expectancy of life in animals with no side effects. It is available for the treatment of arthritis, degenerative joint disorders, tendon, and ligaments injuries in animals. Stem cell therapy is most often used to treat dogs, cats, and horses. But recent developments made it possible to use animal stem cell therapy in tiger, pig, etc. Present animal stem cell therapy is studied in treatments of the inflammatory bowel, kidney, liver, heart and immune-mediated diseases respectively.

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Animal Stem Cell Therapy Market: Drivers and Restraints

Increasing prevalence of disease in animals with growing population and to increase the animals quality of life, the companies focus shifting towards animal stem cell therapies. Along with increasing government funding for the protection of animals and fast approvals of FDA contributing towards the rapid growth of the animal stem cell therapy. The research in animal stem cells offers great promise for understanding underlying mechanisms of animal development; it gives great opportunities to treat a broad range of diseases and conditions in animals. Animal stem cell therapy is increasingly recognized as critical translational models of human disease for treatment. All these factors act as drivers for the robust growth of the animal stem cell therapy market.

There are little evidence-based preclinical animal studies acts as restraint in the animal stem cell therapy market. The evidence-based clinical trials of animal stem cell therapy provide tremendous opportunities for the efficient advancement of other species.

Animal Stem Cell Therapy Market: Segmentation

Segmentation based on Applications

Segmentation based on End-user

Animal Stem Cell Therapy Market: Market Overview

Studies in the animal stem cell therapy continue at a breathtaking pace due to increasing demand and treatment cost covered in reimbursements. And animal stem cell therapy is more effective than traditional treatment available in the market which is boosting the companies to increase the spending in the R&D for innovative methods. Because of the novelty and complexity of animal stem cell therapy, FDA encourages individuals, universities and drug companies for further innovations. The future expected with double CAGR during the forecasted period.

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Animal Stem Cell Therapy Market: Region-wise Overview

Regarding geographies, North America is dominating the global animal stem cell therapy market due to the increased incidence rate and awareness about the therapy. U.S represents the largest market share in the North America due to the increasing demand for the therapy. Europe and Asia-Pacific are showing a significant growth rate during the forecasted period due to the growing adoption of the animal stem cell therapy. The animal stem cell therapy market in underdeveloped countries is slow when compared to the developed countries.

Animal Stem Cell Therapy Market: Key Participants

The key participants in the animal stem cell therapy market are Magellan Stem Cells, ANIMAL CELL THERAPIES, Abbott Animal Hospital, VETSTEM BIOPHARMA, Veterinary Hospital and Clinic Frisco, CO, etc. The companies are entering into the collaboration and partnership to keep up the pace of the innovations.

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‘It feels amazing,’ says Alabama man now first in the state to be free of sickle cell after gene therapy – Face2Face Africa

Sickle cell disease is a group of disorders that affects haemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body.

People with this disorder have atypical haemoglobin molecules called haemoglobin S, which can distort red blood cells into a sickle, or crescent, shape.

And thanks to gene therapy, involving a clinical trial for two years, an Alabama man, Lynndrick Holmes, is free of the disease. However, reports say that the 29-year-old, who is being monitored every 3 months, must be sickle cell-free for 5 years before it can be classified as a cure.

The trial has totally changed my life, said Holmes, who took part in a study at the National Institutes of Health in Bethesda, MD.

It feels amazing. I didnt know how bad it was livingwith sickle cell until I got cured. Once I got cured, I was like, I cantbelieve I was living like that and I was expected to live out the rest of mylife like that.'

Holmes said the disease has been a burden to him his entire life.

A pain crisis happens when people with the disease cant getblood to certain parts of their body. The pain can be severe and can lastanywhere from several hours to several days or longer. Some may requirehospitalization.

According to Julie Kanter, MD, director of the Adult Sickle Cell Clinic at the University of Alabama at Birmingham, the treatment involves taking stem cells from the patients bone marrow and tweaking the gene that causes cells to become misshapen.

The modified gene is then put back in using deactivated HIV. The trial is sponsored by biotech company bluebird bio and has test sites across the country.

Previously, treatment involved using someone with a matched sibling transplant, which only gave a 15% chance of a match.

Were moving toward a more universal cure. We hope it will be curative, but we cant say that yet, Kanter said, adding (theres) . frequent follow-ups from the NIH, and it looks promising.

Sickle cell disease affects about 100,000 Americans. It is agroup of genetic disorders that cause red blood cells to become hard andsticky, taking on the form of a sickle, according to the CDC. When these cellstravel through small blood vessels, they can get stuck, causing pain,infection, and stroke.

Its symptoms include painful swelling of the hands and feet,extreme fatigue, and chronic pain, according to the National Institutes ofHealth (NIH).

In the U.S., most people who have sickle cell disease, which is present at birth, are of African ancestry. About one in 13 African American babies are born with the sickle cell trait, according to the NIH. And about one in every 365 African American babies is born with sickle cell disease.

According to the NIH, the trial has about 50 slots but mosthave been filled already with two of those patients set to undergo the therapyat University of Alabama at Birmingham.

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'It feels amazing,' says Alabama man now first in the state to be free of sickle cell after gene therapy - Face2Face Africa

Michael Schumacher Is ‘Conscious’, Released From Paris Hospital After Experimental Treatment – International Business Times

Ailing Formula 1 world champion Michael Schumacher has been discharged from the Georges Pompidou hospital in Paris after experimental treatment.

Interestingly, a hospital staffer said the racing legend was conscious. This put to rest many speculations about the champion's currenthealth condition.

However, broad details on the legends health are still unknown. According to Michael Schumacher news, the seven-time Formula 1 World Champion was admitted to the Paris clinic under a fake name.

Schumacher, 50, came to the hospital last Monday.

He reportedly underwent stem cell surgery, according to the local newspaper Le Parisien. Media reports also quoted a hospital worker who confirmed that Schumacher was conscious.

Surgeon Professor Philippe Menasche, 69, has been famous as the doctor who performed the world's first embryonic cell transplant on a patient with heart failure conditions.

Schumacher has been to this Paris hospital twice before and was carried in a helicopter.

Secrecy about the health condition

Considerable secrecy exists over the German legends health condition ever since he suffered serious brain injuries from a skiing accident in 2013 at the French Alps.

Schumachers outstanding racing career is dotted with unique achievements. He won seven world titles and 91 wins.

After the accident, the German champion was in a coma for nearly six months. Upon leaving the hospital in June 2014 Schumacher was put under home care at his Lake Geneva residence in Switzerland.

Reports said that Schumachers medical bills cost 115,000 (US$1,43, 624) a week and a team of 15 nurses and physicians are looking after him.

Some unconfirmed reports had said the legend was paralyzed after the tragic ski accident.

Earlier this year Schumacher's manager Sabine Kehm opened up about the champion. Calling him warm-hearted on the Formula 1 podcast Beyond the Grid, she said, he did not want this side of him to be public

Kehm said the racing superstars condition need not be a matter of public interest.

On the racing front, the latest news on Formula 1 2019 highlights Ferrari owners optimism about winning championships in the remainder of the Formula 1 season.

The expectation is that the low downforce and high-speed nature of Spa and Monza will support Ferrari. German Formula One driver Michael Schumacher gestures at the end of the Brazil's F-1 GP on November 25, 2012 at the Interlagos racetrack in Sao Paulo, Brazil. Photo: YASUYOSHI CHIBA/AFP/Getty Images

The team has been upbeat about the serial victories for Charles Leclerc.

According to Ferrari boss Mattia Binotto the team had a strong pace at other tracks. That is a sure sign that it could challenge for wins anywhere. Meanwhile, the Singapore Grand Prix will take place on September 22.

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Michael Schumacher Is 'Conscious', Released From Paris Hospital After Experimental Treatment - International Business Times

Fighting for a cure: Tennis benefit to fund ALS research – Auburn Villager

John Jerome had recently run a triathlon when he noticed his balance was getting worse and his speech slowing down.

It was the latest indication that something was wrong.

He had already suffered from knee pain and problems running in 1999, and had knee surgery later that year.

But it wasn't until he saw a neurologist in 2003 that the true scope of his ailment came into focus he was diagnosed with amyotrophic lateral sclerosis, a progressive motor neuron disease that affects the cells that control muscles.

Most people with ALS, or Lou Gehrig's disease, die within three to five years of diagnosis. But Jerome is still alive and active after 16 years, working two mornings a week at Yarbrough Tennis Center.

"Some people go real fast and they die, and some like me, and Stephen Hawking, have lived a long time. But without money, were not going to find a cure," said Jerome. "I dont know what Id be doing if I wasnt here. Im very fortunate they still let me work here."

After being diagnosed, Jerome regularly made the drive to Atlanta to get checkups at the Emory ALS Center, one of the most critical ALS research facilities in the nation.

It was there in 2010 that he enrolled in phase one of an experimental stem cell trial, which involved two surgeries where doctors injected more than a million stem cells directly into his spinal cord.

"I wanted to do more than just sit around and waste away. I wanted to do my part to try to find a cure, so I did the trial," he said. "I did not get better, but I believe I am getting worse at a slower rate. But since I was slow progressing anyway, its hard to say whether Im getting worse even slower now."

Emory conducted a second phase of the trial that Jerome didn't take part in before funding ran out. But it is research like that Jerome hopes will eventually lead to a cure for ALS, which spurred him to take action on his own to help out any way he could.

Last year, he spearheaded the first John Jerome "Acing ALS' Tennis Benefit to raise money for Emory ALS Center, which offers a clinic every month where ALS patients can receive care.

"John did this on his own," said Bret Peterson, the Tennis Center events manager who works with Jerome. "He came to me and we figured this thing out and put it on. Hes the driving force behind that. Theres not somebody else thats pushing him to do it. Its a big credit to him, really."

Jerome will be hosting the second annual Acing ALS Tennis Benefit on Oct. 4 at the Yarbrough Tennis Center. It's an event that he hopes will raise awareness about ALS and money for research that could one day lead to a cure.

"When they did the ice bucket challenge, that raised $100 million or more, and it did bring a lot of awareness to everybody hearing about ALS, but its dying down," he said. "Without people really donating money, funds may dry up and we may not find a cure.

"Really, I just want people to be aware of ALS and really help us find a cure when every dollar counts."

The first benefit raised $16,000, with more than 70 people signing up to play tennis. Jerome is hoping to raise even more this year. Emory ALS Center will receive all of the funds raised from the event.

The tennis benefit will be held from 6 to 9 p.m. on Oct. 4, and players of all ages and levels are welcome to participate. Snacks, drinks and pizza will be provided by the Auburn Community Tennis Association, and prizes will be given in fastest serve competitions.

It meant a great deal to see so many people in the Auburn community come out and support the effort to find a cure for ALS, Jerome said.

"Man, I got choked up last year. Bret had me say a few words and it really meant a lot. Im even getting choked up talking about it now, and it hasnt even happened," he said. "ALS doesnt get the funding of cancer and stuff like that, but its very important to raise money."

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Living With a Secondary Blood Cancer – Curetoday.com

BY Meeri N. Kim, Ph.D.

During the months that followed, she noticed that she grew short of breath more easily. Going up and down stairs left her winded, and her usual training for the Susan G. Komen 3-Day, a 60-mile fundraising walk an event she had successfully completed five times in the past felt like a herculean task. But as a breast cancer survivor, Strommer felt especially determined to go through with it.

I would be going on my walks, and it was such a struggle to walk any amount of distance, says Strommer, now 62. I would sweat so bad, and Im really not a sweater. I just dont sweat very often, and during that summer, it dripped off my face.

After Strommer completed the fundraiser in August, her right leg developed bothersome swelling that wouldnt go away. Finally, she made a doctors appointment for September, assuring her partner and children that it wouldnt result in anything serious. Two days later, she received a diagnosis of a second cancer, acute myeloid leukemia (AML).

I remember wishing that my doctor would leave the room so I could just cry. It was not what I was expecting, she says. I knew that sometimes when you have breast cancer, you might get another type of cancer, but I was thinking maybe uterine cancer or something in my thyroid. I didnt think it would cause me to get leukemia.

RECURRENCE VERSUS SECOND CANCERA growing number of survivors like Strommer are finding themselves with a second cancer, which can be unrelated or random but becoming increasingly common as more people are living longer and surviving their first cancer. But the two diseases can also be somewhat related, sharing the same risk factors for example, lung and bladder cancer are more common in people who use tobacco or caused by the treatment for one of the cancers.

Its different from disease recurrence, which happens when the cancer comes back. Second cancers are associated with many risk factors, such as inherited genes, cancer cells that remain in the body after treatment, smoking, diet, weight, heavy drinking and environmental toxins.Another risk factor involves treatments, such as certain chemotherapies and radiation, given to treat the first cancer. Strommers doctors told her that having chemotherapy and radiation in 2006 for breast cancer likely led her to develop AML.

Survivors have a small increased risk of developing a second cancer, according to the American Cancer Society. Ongoing research is looking to prevent second cancers by delivering chemotherapy and radiation with more precision and identifying those individuals most at risk. However, its crucial that survivors understand that cancer can recur and a new cancer can arise.

The biggest thing to understand from a patient perspective is that just because one bad thing has happened to you, it doesnt mean that more bad things will not happen, says Dr. Aaron Seth Rosenberg, an assistant professor of hematology and oncology at the University of California, Davis Comprehensive Cancer Center in Sacramento. Its a small but real number of patients who go on to develop second malignancies. So the risk is there, but you cant affect that risk by worrying about it, and all that we can do is monitor our patients closely.

Over the past 30 years, the number of cancer survivors has increased fourfold. Today, roughly 17 million Americans live with a history of cancer, according to a report from the American Cancer Society. By 2030, that number is anticipated to rise to more than 22 million.

These individuals must live with a new normal that may include long-term side effects, financial hardships, possibility of recurrence or a second cancer. Survivors can work with their health care team to create a survivorship care plan a record of a persons cancer and treatment history, as well as any checkups or follow-up tests that may be needed in the future.

The likelihood of facing a second cancer varies depending on factors such as the type of cancer first diagnosed, treatment received, age at diagnosis and lifestyle choices. For instance, children and young adults have a higher risk of second cancers related to treatment with radiation and chemotherapy compared with older adults.

With the increasing number of cancer diagnoses, as well as the improved prognosis for many patients, second cancers are more common today than in the past, says Dr. Lindsay Morton, who holds a doctorate in epidemiology and is senior investigator in the Division of Cancer Epidemiology & Genetics at the National Cancer Institute (NCI). In fact, about one in five cancers diagnosed today is diagnosed in a cancer survivor.

THE RESEARCH SAYSResearchers from Texas conducted a study, which was published in JAMA Oncology, that analyzed data from NCIs Surveillance, Epidemiology and End Results (SEER) Program to see the prevalence of prior cancer among individuals newly diagnosed with cancer. Based on 740,990 cases from 2009 to 2013, 18.4% of all newly diagnosed cancers represented a second or subsequent cancer. A quarter of adults ages 65 and older and 11% ages 20 to 64 had a history of cancer when given their diagnosis.

Additional findings show a link between certain types of chemotherapy and blood cancers, most commonly AML, acute lymphocytic leukemia (ALL) and myelodysplastic syndrome (MDS). In particular, various alkylating agents, platinum compounds and topoisomerase 2 inhibitors have been associated with a fivefold increased risk of therapy-related MDS or AML. Alkylating agents such as mechlorethamine and cyclophosphamide affect the cancers ability to multiply and are most effective for leukemia and solid tumors. Platinum-based drugs like cisplatin and carboplatin inhibit DNA repair and/or synthesis in cancer cells. Topoisomerase 2 inhibitors stop cells from repairing DNA and have been used to treat breast, lung, testicular and other types of cancer. For Strommers breast cancer, her doctors administered the AC-T chemotherapy regimen, which consists of Adriamycin (doxorubicin) and cyclophosphamide, followed by Taxol (paclitaxel). Her doctors told her that it likely contributed to her development of AML.

Late last year, Morton and her colleagues reported in JAMA Oncology about a substantial expansion in the number of patients who are at risk of therapy-related leukemia in the modern treatment era. From 2000 to 2014, patients in the SEER registry database treated with chemotherapy for solid tumors (except for colon cancer) experienced an increased risk of therapy-related MDS or AML. The researchers also noted a rise in the proportion of patients treated with a known leukemogenic agent (alkylating agent, platinum compound or topoisomerase 2 inhibitor), from 10% during 2000 to 2001 to 81% in 2012 to 2013.

Morton emphasizes that developing MDS or AML after chemotherapy remains rare: In most of the patient populations we studied, the risk of developing therapy-related leukemia wasless than 1% at 10 years, she says.

The benefits of cancer treatment typically far outweigh the risk of developing a new cancer due to that treatment.

Although chemotherapy is a greater-known risk factor, radiation therapy can also cause leukemia, MDS and solid tumor cancers. The amount of risk depends on the dose of radiation, the specific region of the body that is treated and the age of the patient at the time of therapy.

Radiation therapy-associated leukemia and MDS most often occur within several years of treatment, generally five to nine years later. It usually takes at least 10 years sometimes more than 15 years for radiotherapy-related solid tumors to develop.

Therapy-related malignancies are part of why we emphasize regular follow-up and survivorship in patients. But one of the things I stress upon my patients is that we have to deal with the cancer in front of us, Rosenberg says. If we dont treat you with the best therapies avail- able because theres a risk five to 10 years down the line, were ignoring the short-term dangers of the cancer thats currently in front of us.

EYEING GENETICSAn active area of research in the field of second cancers is finding a way to identify which survivors are most at risk, so they can be monitored more closely for new disease. One promising method is a peripheral blood test for a recently identified disease entity called clonal hematopoiesis of indeterminate potential, or CHIP. It is defined as the presence of preleukemic mutations those that pave the way to developing leukemia in an otherwise healthy person who does not have cancer.

Doctors and scientists are starting to understand that there may be a small subset of patientswho have an increased risk of therapy-related leukemias that may be genetically based, says Dr. Guillermo Garcia-Manero, a professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center in Houston. The tests are currently in clinical trials, but oneday, they could allow us to come up with effective identification and preventive strategies for patients.

Garcia-Manero specializes in therapy-related myeloid neoplasm (t-MN), a term proposed bythe World Health Organization to cover the spectrum of malignant disorders previously described as therapy-related MDS or AML. In 2017, he and colleagues published a study in Lancet Oncology in which they compared the prevalence of CHIP between patients who developed t-MNs and those who did not. More than 70% of the t-MN group had CHIP at the time of their initial cancer diagnosis, before ever having t-MN, compared with 31% of the control group. Until not too long ago, we thought that this process of developing t-MN was totally random. We didnt know why some patients with breast cancer never get this kind of therapy-related leukemia, (whereas) others do, Garcia-Manero says. CHIP has really transformed this field. Now, for a significant percentage of patients, you can predict who has a higher risk of therapy-related disease.

CHIP is rare, especially at a young age, but the prevalence rises to roughly 10% among individuals ages 70 to 80. Liquid biopsies, which are becoming more common at cancer centers, could also be used to identify patients with CHIP and assess their risk of t-MN.

AVOIDING A NEW DIAGNOSISCancer survivors can lower their overall cancer risk by living a healthy lifestyle, which includes getting regular exercise, eating a healthy diet, avoiding tobacco and limiting alcohol use. Individuals should also keep follow-up appointments and let their doctors know if any symptoms or problems arise. Signs of a second cancer include fatigue, loss of appetite, bleeding, feeling like bones ache and vision changes.

Thinking back to that fateful summer, Strommer realizes that she should have seen a doctor sooner, right after she noticed something off about her health. But after six grueling months of treatment for AML, which involved chemotherapy, a bone marrow transplant on her birthday and multiple near-death experiences two fungal infections and fluid in her heart she made what doctors call a miraculous recovery and was well enough to go home in April 2014. She still takes Nexavar (sorafenib), a kinase inhibitor originally used for kidney cancer and showing promise for some patients with AML depending on the results of her next bone marrow biopsy, her oncologist may take her off it after six years. Strommer realizes that not everyone beats the odds as she did she often encounters those patients at her new job at the local hospitals cancer center. She switched departments after her recovery, driven by a desire to help others going through what she once did. She credits a positive attitude, her seven grand- children and the grace of God for helping her survive the hardest time in her life.

When the doctors said I only had a 1% chance to make it, its hard to understand why God chose me to win the battle, yet others who had someone to live for lost their battle, Strommer says. There were so many times Id sit in my room in the hospital and didnt know whether I would live or die, but I was really at peace with it. All I can say is, Im a firm believer in miracles.

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Scientists Discover New Strategy That May Protect Against Hair-Loss During Cancer Treatments – Good News Network

Scientists have determined a new way to protect the hair follicle from chemotherapy in an effort to prevent hair loss as a result of cancer treatments.

Researchers based at The University of Manchester have discovered a new strategy for how to protect hair follicles from chemotherapy, which could lead to new treatments that prevent chemotherapy-induced hair lossarguably one of the most psychologically distressing side effects of modern cancer therapy.

Published in the journal, EMBO Molecular Medicine, the study from the laboratory of Professor Ralf Paus of the Centre for Dermatology Research describes how damage in the hair follicle caused by taxanes, cancer drugs which can cause permanent hair loss, can be prevented.

To do this, scientists have exploited the properties of a newer class of drugs called CDK4/6 inhibitors, which blocks cell division and are already medically approved as so-called targeted cancer therapies.

RELATED: Scientists Activate Stem Cells to Make Hair Grow

Dr Talveen Purba, lead author on the study explains: Although at first this seems counter-intuitive, we found that CDK4/6 inhibitors can be used temporarily to halt cell division without promoting additional toxic effects in the hair follicle. When we bathed organ-cultured human scalp hair follicles in CDK4/6 inhibitors, the hair follicles were much less susceptible to the damaging effects of taxanes.

Taxanes are very important anti-cancer drugs commonly used to treat, for example, patients with breast or lung carcinoma and particularly cause anxieties among breast cancer patients for the very distressing and sometimes long-lasting hair loss taxanes can induce. Thousands of patients in the US are currently suing pharmaceutical company Sanofi over a lack of warning of the risk of permanent hair loss after treatment with the taxane drug Taxotere.

Dr Purba emphasizes: A pivotal part of our study was to first get to grips with how exactly hair follicles responded to taxane chemotherapy, and we found that the specialized dividing cells at the base of the hair follicle that are critical for producing hair itself, and the stem cells from which they arise, are most vulnerable to taxanes. Therefore, we must protect these cells most from undesired chemotherapy effectsbut so that the cancer does not profit from it.

CHECK OUT: Husband-Wife Duo Has Developed Gene and Cell Therapy Cancer Vaccine Now Being Tested on Patients

The team hopes that their work will support the development of externally applicable medicines that will slow or briefly suspend cell division in the scalp hair follicles of patients undergoing chemotherapy to mitigate against chemotherapy-induced hair damage. This could complement and enhance the efficacy of existing preventive approaches i.e. scalp-cooling devices.

The researchers underscore that more work is desperately needed in this lamentably under-funded field of cancer medicine, where patients have waited for so long to see real breakthroughs in pharmacological hair loss prevention.

Despite the fact that taxanes have been used in the clinic for decades, and have long been known to cause hair loss, were only now scratching the surface of how they damage the human hair follicle, said Purba.

MORE: Broccoli Isnt Just Good For YouScientists Find It Holds Molecule That Could Be the Achilless Heel of Cancer

We also dont really know why some patients show greater hair loss than others even though they get the same drug and drug-dose, and why it is that certain chemotherapy regimens and drug combinations have much worse outcomes than others.

We need time to further develop approaches like this to not only prevent hair loss, but promote hair follicle regeneration in patients who have already lost their hair due to chemotherapy.

Reprinted from the University of Manchester

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Stem Cells May Be Able To Repair The Brain – Anti Aging News

Transplanted brain stems cells have survived without using anti-rejection drugs in mice by exploiting a feature of the immune system, findings may open new paths for stem cell transplants to help repair the brain.

Johns Hopkins Medicine research may have developed a way to transplant certain protective brain cells without the need for lifelong anti-rejection drugs, as published in the journal Brain; the approach selectively circumvents responses from the immune system against foreign cells to allow the transplanted cells to survive and thrive to protect brain tissues long after stopping immunosuppressive drugs.

"Because these conditions are initiated by a mutation causing dysfunction in one type of cell, they present a good target for cell therapies, which involve transplanting healthy cells or cells engineered to not have a condition to take over for the diseased, damaged or missing cells," says Piotr Walczak, M.D., Ph.D., associate professor of radiology and radiological science at the Johns Hopkins University School of Medicine.

The researchers are working on developing ways to stop the immune system responses without side effects, and investigated ways to manipulate T cells which are the immune systems infection fighting force that attack foreign invaders, specifically focussing on a series of costimulatory signals that T cells must encounter in order to begin attack.

"These signals are in place to help ensure these immune system cells do not go rogue, attacking the body's own healthy tissues," says Gerald Brandacher, M.D., professor of plastic and reconstructive surgery and scientific director of the Vascularized Composite Allotransplantation Research Laboratory at the Johns Hopkins University School of Medicine and co-author of this study.

To exploit natural tendencies of these costimulatory signals that train the immune system to accept the transplanted cells as being part of self the researchers used CTLA4-lg and anti-CD154 antibodies to keep the T cells from beginning an attack by blocking signals.

Protective glial cells that produce myelin sheath that surrounds neurons that were genetically engineered to glow were injected into mice brains so they could be studied. Glial cells were transplanted into three types of mice: those engineered to not for glial cells that create myelin sheath; normal mice; and those engineered not to be able to mount an immune response.

Antibodies were used to block an immune response, stopping treatment after six days, and specialized cameras were used to detect the glowing cells and pictures of the mice brains to look for the presence or absence of the transplanted cells. Cells transplanted into control mice without antibody treatment began to die off immediately and was no longer detected after 21 days; while those receiving antibody treatment maintained significant levels of transplanted cells for over 203 days even in the absence of treatment.

"The fact that any glow remained showed us that cells had survived transplantation, even long after stopping the treatment," says Shen Li, M.D., lead author of the study. "We interpret this result as a success in selectively blocking the immune system's T cells from killing the transplanted cells."

To see whether the transplanted glial cells survived well enough to create the myelin sheath key structural differences were looked at between mice brains with and without thriving glial cells using MRI imaging; cells in the treated animals were found to be populating the appropriate parts of the brain. Results confirm the transplanted cells were able to thrive and assume normal function to protect neurons.

Although results are preliminary the cells were delivered to thrive in a localized portion of the mouse brain. Researchers hope to combine findings with studies on cell delivery methods to the brain in the future to help repair the brain more globally.

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MS News that Caught My Eye: Stem Cell Transplants, Remyelination Agent, Tecfidera Study, Plasma Exchange and Tysabri-linked PML – Multiple Sclerosis…

Its been a big week for interesting stories, as the annual meeting of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) has just concluded. The conference offered much to engage healthcare professionals and researchers, but the following are some presentations that appealed to me as a multiple sclerosis (MS) patient.

The debate continued over the risks versus the rewards of stem cell transplants. Joachim Burman, MD, PhD, a researcher at Uppsala University in Sweden, made the case as to why autologous hematopoietic stem cell transplantation may be the most effective MS treatment so far, and why it should be made widely available. A single administration can yield prolonged benefits, and most patients achieve no evidence of disease activity status for at least five years. But safety concerns exist, and the lack of control groups tempers the positive results in studies.

Stem cell therapy, or stem cell transplant, is an emerging yet controversial treatment approach for multiple sclerosis (MS). While some data uphold it as one of the most efficacious MS treatments, to date there have been no controlled studies comparing it to conventional medicines and providing more robust evidence regarding its safety and clinical benefit.

Under the topic HSCT and stem cell treatment in MS,a group of researchers discussed the promise and current challenges of stem cell transplant at the 35thCongress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), being held Sept. 1113 in Stockholm.

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As those of us who have multiple sclerosis know, finding a way to repair the damage to the myelin that covers our nerves would be like discovering the Holy Grail. This research, which looked into whether a potential remyelination agent is safe and might provide some repair, gives some hope.

Treatment with a potential remyelinating agent called liothyronine was safe and well-tolerated by people with multiple sclerosis (MS) in a Phase 1b trial. Preliminary results also suggested benefits in cognition, motor function, and fatigue.

The study, A Phase 1b, open-label study to evaluate the safety and tolerability of the putative remyelinating agent, liothyronine, in individuals with multiple sclerosis, was presented today at the 35thCongress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), by Scott D. Newsome, professor of neurology at Johns Hopkins University School of Medicine.

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Out of 618 RRMS patients followed for 10 years, 192 of them were on continuous Tecfidera (dimethyl fumarate) twice daily treatment throughout that time, and many reported that the disease-modifying therapy (DMT) had helped them. Just over half of these patients remained relapse-free, and 64 percent had no signs of confirmed disability progression over that period. In a separate presentation, researchers found that the DMTs Tysabri (natalizumab) and Lemtrada (alemtuzumab) were better at reducing the annualized relapse rate than Tecfidera.

New 10-year data from the Phase 3 ENDORSE trial confirms the long-term benefits of Biogens Tecfiderafor patients with relapsing-remitting multiple sclerosis(RRMS), the most common form of this disease.

Real-world data from another study also showed Tecfidera to be superior to several other disease-modifying therapies for relapsing MS, namely Copaxone (glatiramer acetate), Aubagio(teriflunomide), andinterferon beta.

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Treatment with Tysabri (natalizumab) carries the risk of triggering the often fatal brain infection progressive multifocal leukoencephalopathy (PML). Several treatments have been tried to attack PML, one of which is a type of blood cleansing called PLEX. It was hoped that PLEX would quickly clear Tysabri from the blood, and in doing so, would improve PML survival rates. Unfortunately, this study reports that two years after PML was detected the survival rate of patients treated with PLEX was no different than patients who had not received the treatment.

Use of plasma exchange (PLEX) is not effective for treating progressive multifocal leukoencephalopathy (PML), a dangerous brain infection that has been associated with using the multiple sclerosis (MS) medicine Tysabri (natalizumab), a real-world study contends.

The findings highlight the importance of closely monitoring Tysabri users to detect PML as early as possible.

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Note: Multiple Sclerosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Multiple Sclerosis News Today or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to multiple sclerosis.

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MS News that Caught My Eye: Stem Cell Transplants, Remyelination Agent, Tecfidera Study, Plasma Exchange and Tysabri-linked PML - Multiple Sclerosis...

Tiny ‘envelopes’ show promise for sun-damaged skin repair – Futurity: Research News

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Exosomes harvested from human skin cells are more effective at repairing sun-damaged skin cells in mice than popular retinol or stem cell-based treatments currently in use, according to a new proof-of-concept study.

Additionally, needle-free injections can deliver the nanometer-sized exosomes to the target cells.

Exosomes are tiny sacs (30150 nanometers across) that cells excrete and take up. They can transfer DNA, RNA, or proteins from cell to cell, affecting the function of the recipient cell. In the regenerative medicine field, researchers are testing exosomes as carriers of stem cell-based treatments for diseases ranging from heart disease to respiratory disorders.

Think of an exosome as an envelope with instructions insidelike one cell mailing a letter to another cell and telling it what to do, says corresponding author Ke Cheng, professor of molecular biomedical sciences at North Carolina State University and professor in the universitys joint biomedical engineering department with the University of North Carolina at Chapel Hill. In this case, the envelope contains microRNA, non-coding RNA that instructs the recipient cell to produce more collagen.

To test whether exosomes could be effective for sun-damaged skin repair, the researchers first grew and harvested exosomes from skin cells. They used commercially available human dermal fibroblast cells, expanding them in a suspension culture that allowed the cells to adhere to one another, forming spheroids. The spheroids then excreted exosomes into the media.

These 3D structures generate more procollagenmore potent exosomesthan you get with 2D cell expansion, says Cheng.

In a mouse model, Cheng tested the 3D spheroid-grown exosomes against three other treatments: retinoid cream; 2D-grown exosomes; and bone marrow derived mesenchymal stem cells (MSCs) exosomes, a popular stem cell-based anti-aging treatment currently in use.

The team compared improvements in skin thickness and collagen production after treatment. They found that skin thickness in 3D exosome treated mice was 20% better than in the untreated and 5% better than in the MSC-treated mouse. Additionally, they found 30% more collagen production in skin treated with the 3D exosomes than in the MSC treated skin, which was the second most effective treatment.

I think this study shows the potential for 3D exosomes to be used in anti-aging skin treatments, says Cheng. There are two major benefits to exosome treatments over conventional treatments: one, you can use donor skin cells from anyone to grow and harvest these exosomesthey arent cells, so you dont run the risk of rejection. And two, the treatment can be administered without needlesexosomes are small enough to be able to penetrate the skin via pressure, or jet injection methods.

Our hope is that eventually people may be able to bank skin samples and come back to them, or use donor exosome treatments that they can administer themselves. We believe that this work is an important step toward potentiating future human clinical trials in the prevention and treatment of cutaneous aging.

The work appears in ACS Nano.

The National Institutes of Health and the American Heart Association supported the research, in part.

Source: Tracey Peake for NC State

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Tiny 'envelopes' show promise for sun-damaged skin repair - Futurity: Research News