StemCell Therapy

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Yet today the 59-year-old father of two is planning a dream family trip to South Africa, something that would have been unthinkable a mere nine months ago.

The reason for this remarkable transformation is that last September Gordon, who suffered from severe heart failure after a series of heart attacks, underwent revolutionary stem cell therapy to repair the diseased muscle tissue in his heart.

I couldnt walk up the stairs without having stabbing pains in my heart and burning in my lungs. Sometimes I had to crawl for the last few steps.

I felt so low and helpless, says Gordon, who is married to Joanne, 50, and lives in Thorneholme, East Yorkshire.

Within a week of the operation I could climb the stairs again. Small things like that have made a huge difference to my life.

More than a million people in the UK suffer from heart disease. The general term for heart disease is cardiomyopathy, a condition in which the walls of the heart chambers have become stretched, thickened or stiff.

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This affects the hearts ability to pump blood around the body. Some types of cardiomyopathy are inherited and it can affect children and younger people.

I couldnt walk up the stairs without having stabbing pains

Gordon Foster

In others, lifestyle factors such as smoking, an unhealthy diet or a sedentary lifestyle can be to blame. There is no cure and although it can be treated with drugs such as ACE inhibitors, they often have side effects and arent a permanent solution.

In Gordons case his condition was the result of a series of heart attacks, the first of which struck when he was 30.

I woke up one morning feeling horribly sick so I ran to the bathroom. I was banging my head on the floor to try to get rid of the pain in my chest, he recalls. Everybody thought I was a goner because it was such a massive heart attack.

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10 Step plan to eliminate your risk of heart disease

Joanne and I got married two months later because she was worried I might not live until our wedding date.

However he did survive and he and Joanne went on to have two children, James, now 26, and Rebekah, 24. Then when James was just a year old Gordon had another heart attack and three years later, aged 37, he suffered a third.

He was diagnosed as suffering from heart failure which most commonly occurs following a heart attack when the heart muscle suffers irreparable damage.

Symptoms can include fatigue, shortness of breath and swelling. In severe cases people with heart failure are left unable to perform ordinary, day-to-day activities such as walking upstairs or are left breathless even when resting.

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By 2012 Gordons heart was functioning at just 17 per cent and he had been forced to retire on heart attack and three years later, aged 37, he suffered a third. from heart failure which most commonly occurs following a heart attack when the heart muscle suffers irreparable damage.

Symptoms can include fatigue, shortness of breath and swelling. failure are left unable to perform ordinary, day-to-day activities such as walking upstairs or are left breathless even when resting.

Functioning at just 17 per cent and he had been forced to retire on medical grounds from his job overseeing welding and fabricating sites.

For severely affected patients a heart transplant can be the only option but the chances of failure are high. Around 10 per cent of transplant patients die within a year of the operation and 25 per cent die within five years.

The need for treatment in this field has never been greater, says Professor Anthony Mathur, consultant cardiologist at St Bartholomews Hospital, London.

Now stem cell therapy is offering new hope to desperate patients and their families. The procedure involves extracting stem cells from bone marrow in the spine and injecting them into the heart.

Researchers hope that the stem cells, which are unique because they can grow into any type of body tissue, will grow into healthy heart cells and take over the work of the diseased or damaged ones.

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The procedure takes about 20 minutes and patients can usually go home the following day. Gordon became the first man in the UK to be offered the operation under the Compassionate Treatment Programme funded by the Heart Cells Foundation charity at St Bartholomews Hospital after his doctor put him forward to take part in a trial.

The charity has so far raised more than 6.5million to fund the Stem Cell Research Programme and is campaigning to raise further cash to treat thousands more patients.

Stem cell therapy is still in the development and research stage and the Compassionate Treatment Programme is funded purely by the Heart Cells Foundation charity, says its chairman Jenifer Rosenberg.

To treat one person costs 10,000 so we need the continued support of our donors to save lives.

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Queen Elizabeth II visits a ward during a tour of Great Ormond Street Hospital for sick children, 23rd July 1952

The treatment is currently in the second phase of clinical trials and phase three will start once funding is secured. If this is successful it is hoped that the treatment could eventually be offered on the NHS.

Gordon says he and his family will be forever thankful to the Heart Cells Foundation and his medical team at St Bartholemews for saving his life.

Without them, I believe I wouldnt be here today, he says. Im now able to lead a near-normal life and Im enjoying every moment I spend with my wife and children. I now live every day with hope.

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How a revolutionary stem cell treatment could save your heart - Express.co.uk

Twiga, a 14-year-old female giraffe with advanced arthritis and osteoporosis in her feet, was fitted with custom shoes. (Photo courtesy of Cheyenne Mountain Zoo.)

Two medical breakthroughs have helped heal two giraffes at Cheyenne Mountain Zoo in recent weeks, the zoo announced Wednesday.

Mahali, a 14-year-old male giraffe who suffered from chronic lameness and had not been moving well, is believed to be the first in the world to be injected with stem cells grown from giraffe blood, according to a news release from the zoo.

Stem cell therapy was chosen in the efforts led by Dr. Liza Dadone, the zoo's head veterinarian, because it has proven to repair damaged tissue. Staff at Colorado State University's James L. Voss Veterinary Teaching Hospital in Fort Collins helped with the treatment.

Nearly a month after the procedure, when Mahali was injected with about 100 million stem cells, thermographic images of the giraffe's front legs show "a considerable decline" in inflammation in his front left leg, the leg that had been giving him trouble, the zoo said.

"This is meaningful to us not only because it is the first time a giraffe has been treated with stem cells, but especially because it is bringing Mahali some arthritis relief and could help other giraffes in the near future," Dadone said in a written statement.

Dadone said it's not clear whether the successful results are due only to the stem cell treatment or a combination of treatments.

"Prior to the procedure, he was favoring his left front leg and would lift that foot off the ground almost once per minute," she said in the statement. "During the immobilization, we did multiple treatments that included hoof trims, stem cell therapy and other medications. Since then, Mahali is no longer constantly lifting his left front leg off the ground and has resumed cooperating for hoof care. A few weeks ago, he returned to life with his herd, including yard access. On the thermogram, the marked inflammation up the leg has mostly resolved."

Twiga, a 14-year-old female giraffe with advanced arthritis and osteoporosis in her feet, was fitted with custom shoes with the help of farriers Steve Foxworth and Chris Niclas of the Equine Lameness Prevention Organization.

"We've had Twiga on medicine to help reverse her osteoporosis, but we wanted to do more to protect her feet. So with the help of the farriers, we gave her 'giraffe sneakers' to help give her some extra cushion," Dadone said in a written statement.

The giraffe's behavior was immediately changed - "Twiga instantly shifted her weight off of her right foot, indicating she was comfortable and her pain had considerably lessened" - but she will likely wear the shoes for about six more weeks, the zoo said.

Giraffes' size can make them more susceptible to issues like arthritis and osteoporosis. "Like all animals, these issues are exacerbated as they age," according to the zoo news release.

The zoo has a herd of 17 giraffes, including a newborn in April. The calf, a girl, was the 199th to be born in the 63-year history of the zoo's breeding program.

Giraffes' status was recently changed from "least concern" to "vulnerable" by the International Union for Conservation of Nature because the population in the wild has decreased by 40 percent in the last 30 years, the zoo said.

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Contact Ellie Mulder: 636-0198

Twitter: @lemarie

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'Sneakers,' stem cells help heal Cheyenne Mountain Zoo's giraffes - Colorado Springs Gazette

Type 2 diabetes can wreak havoc on your health. While lifestyle changes can help keep diabetes under control, many patients require oral medications or insulin injections as forms of treatment, too. Watch the video for how diabetes affects your body. Time

Pharmaceuticals maker Roche introduced a new blood glucose monitoring system called Accu-Chek Guide, paired with a savings card that allows patients to get the device for free as well as discounted test strips.(Photo: Roche)

Pharmaceuticals maker Roche overhauled its blood glucose monitoring system and introduced a new discounting offer that it says could save uninsured diabetics by thousands of dollars per year.

The move could help alleviate political pressure as the drug industry faces mounting scrutiny over prices. It also comes amid increasing competition among blood glucose monitoring makers as diabetes rates rise.

The new system pairs a free blood glucose meter with a smartphone app and discounted test strips. With some diabetics paying as much as $2 a strip for other offerings, the new Roche system paired with a free savings card could cut costs to as little as 40 cents per strip in the first 50-count box, then 20 cents per strip in subsequent boxes.

The nation's 29-million diabetics pay widely varying prices for testing products, in part because many of them are covered by insurance. Roches' move is likely to provide the biggest help to the uninsured.The average American diabetic paid $1,922 in out-of-pocket expenses for care in 2013, compared to $738 for someone without the condition,according to the Health Care Cost Institute.

For "the average patient, managing diabetes and acquiring all of the testing and therapy supplies can be very difficult to navigate, really complex and very often very expensive," said Brad Moore, head of Rochediabetes care in North America.

The new system offers a spill-resistant vial, a larger blood application area on upgraded strips and a light on the strip port for improved visibility when testing. The device wirelessly transmits data through Bluetooth technology to a free smartphone app that logs data.

Moore said Roche technicians worked on the new Accu-Chek Guide Systemfor at least three years, including a "very significant investment in capital."

Test strips read by devices to monitor blood glucose data are typically a significant source of profit for the pharmaceutical industry, which is under fire for its contribution to increasing health care costs. President Trump has threatened to battle drug companies over costs, while many Washington lawmakers have decried health care's effect on the average American's budget.

Although industryprices can be more than $2per strip, manufacturing costs don't typically top 15 cents, DiabeticInvestor.com analyst David Kliff told Diabetes Forecast magazine in 2012.

Roche had 8.5% market share in the blood glucose monitoring industry, trailing only Johnson & Johnson at 22.5%, according to an October 2016 report by market-research firm IBISWorld analystJonathan DeCarlo.

But competition is increasing, as big-box retail chains Target and Walmart and other retailers have introduced low-cost, private-label options. Consequently, the blood glucose monitoring industry's profit as a percentage of revenue was projected to fall from 10.1% in 2015 to 9.5% in 2016, IBISWorld'sDeCarlo estimated.

Moore declined to discuss the profitability of Roche's new test strips, which contain a new chemical makeup.

"We knew that access was a problem. We heard that from our patients," Moore said. "So the timing was perfect in that weve developed a new technology platform that the Accu-Chek Guide System is based on."

Meanwhile, drug companies are under pressure to shield patients from increasing costs, though they often blame insurers and other health care intermediaries for saddling patients with additional expenses.

With a free savings card available online, through pharmacies and at health care centers, the new Roche monitoring meter is free, the first box of 50 test strips is $19.99 and all additional boxesare $10.That's cheaper than major competitors atAmazon, Rite Aid, Walgreens, CVS and Walmart with the exception of the ReliOn Prime option at Walmart, according to data collected by USA TODAY.

Most options are more than $40 for a box, with some significantly more expensive. Accu-Chek's previous box of Aviva Plus strips ranged from $44.99 at Amazon to $109.99 at Walgreens.

The average patient tests once a day but some must test eight to 10 times a day. At those rates, savings from typical diabetes tests could range from hundreds to several thousands of dollars per year.

A recent study commissioned by Roche of 500 U.S. adults living with diabetes found that 58% "cut corners" to save money in their daily testing regiment, including by skipping tests.

Contributing: Diana Kruzman

Follow USA TODAY reporter Nathan Bomey on Twitter @NathanBomey.

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An experimental foot-temperature monitoring system might one day be able to detect when diabetic patients are developing foot ulcers, a common complication that can lead to infections and amputations, a small study suggests.

Diabetic foot ulcers typically develop on the bottom of the big toe or the ball of the foot, often when people wear ill-fitting shoes. Patients with diabetes frequently have nerve damage that limits their ability to feel pain, and as a result they dont notice developing ulcers.

For the study, researchers tested a so-called smart mat designed to use variations in temperature at different points on the foot as a predictor of recurring foot ulcers in 129 patients who had this problem before. Skin temperature typically increases as ulcers develop.

When the study team tested for variations of 2.22 degrees Celsius (about 4 degrees Fahrenheit), they found the smart mat correctly identified 97 percent of foot ulcers observed by clinicians. But it also had a false positive rate of 57 percent, meaning clinicians didnt find ulcers identified by the mat.

With a larger temperature variation of 3.20 degrees Celsius (about 5.75 degrees Fahrenheit), the false positive rate dropped to 32 percent, but the proportion of correctly identified foot ulcers also declined to 70 percent, researchers report in Diabetes Care.

If we look at this technology as a risk stratification tool with high feasibility to be used at home on daily basis, it could be hugely beneficial to target those who are truly at risk, said senior study author Dr. Bijan Najafi, a researcher at Baylor College of Medicine in Houston.

I dont think the point is having a system with no false-alarm, Najafi said by email.

The device in the study was developed by Podimetrics Inc. in Somerville, Massachusetts, and its approved for sale in the U.S. for the periodic evaluation of temperature variations in the soles of the feet for signs of inflammation. Podimetrics sponsored the study of the mat for predicting diabetic foot ulcers.

In the current experiment, patients used the mat in much the same way they might use a common bathroom scale. Every day, they stepped on it and waited 20 seconds while it measured temperatures at different points on the soles of the feet, then the device wirelessly transmitted the temperature data to servers managed by Podimetrics. The data were saved and analyzed for variations in foot temperature that might signal developing ulcers.

In total, the trial ran 34 weeks, and 37 participants developed 53 foot ulcers during the study period.

For both of the temperature variation settings tested in the study, the mat correctly identified developing ulcers an average of 37 days before they were detected by a doctor.

That lead time might help patients schedule clinic visits and get treatment for ulcers sooner, when theyre easier to treat and less likely to lead to serious complications, Najafi said.

The study wasnt designed to determine whether the mat actually reduced the development of ulcers or curbed costs to treat these ulcers, the authors note. Researchers only followed patients for 60 days, and its possible the rate of false positives or accurately identified ulcers might look different over a longer period of time.

In addition, the study only included patients with a history of diabetic foot ulcers, and the results might be different for people with diabetes who have never had this problem before, the authors point out.

While the high rate of false positives in the study suggests that the device still needs more testing and refinement, the technology holds a lot of potential to aid patients who currently have a high risk of infection and amputation because their developing ulcers go undetected, said Dr. David Armstrong, director of the Southern Arizona Limb Salvage Alliance at the University of Arizona College of Medicine in Tucson.

Whats really attractive about this technology is that it is probably going to get smarter, Armstrong, who wasnt involved in the study, said by email. This technology is probably going to personalize a heat signature for every patient and identify a hot spot for each patient.

SOURCE: bit.ly/2rAPZz6 Diabetes Care, April 29, 2017.

WASHINGTON U.S. Senate Majority Leader Mitch McConnell said on Wednesday he does not yet know how Republicans will amass the votes needed to pass legislation now being crafted to dismantle Obamacare, but expressed some optimism on another top priority, overhauling the tax code.

LONDON GW Pharmaceuticals is set to file its cannabis-derived drug with U.S. regulators imminently, following publication of detailed data on its success in fighting severe childhood epilepsy.

Eating a small amount of chocolate every week or so may decrease the risk of a common and serious type of irregular heart rhythm, according to a new study of people in Denmark.

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Foot mat may help predict who will get a common diabetes complication - Reuters

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UT-Austin Researchers Seek to Prevent Diabetes in At-Risk Population Patch.com AUSTIN, TX Researchers at The University of Texas at Austin and The University of Texas Health Science Center at Houston (UTHealth) School of Public Health have received a $2.9 million, five-year award from the National Institute of Diabetes and ...

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Northwest Wyoming now claims one of only two health care professionals in the state who holds a certification in Advanced Diabetes Management. West Park Hospitals Registered Dietitian Nutritionist, Liz Fabrizio, has passed the boards for Advanced Diabetes Management from the American Association of Diabetes Educators. Thats an important resource, considering that the adult diabetes rate has almost doubled in Wyoming in the last 15 years. According to West Park Hospital Public Relations Director Ashley Trudo, Fabrizios certification supports her advanced level of knowledge and ability to manage complex patient needs while assisting patients with therapeutic problem-solving. http://dehayf5mhw1h7.cloudfront.net/wp-content/uploads/sites/761/2017/05/24091827/Trudo-Diabetes-Specialist.mp3 In just 15 years, the adult diabetes rate rose from 4.5 percent in 2001 to 8.4 percent in 2016. Christine Revere, Chronic Disease Prevention Program manager with the Wyoming Department of Health, said the trend is not surprising when considered along with several risk factors linked with type 2 diabetes. 65 percent of Wyoming adults are obese or overweight, 83 percent dont eat enough fruits and vegetables, 25 percent engage in no daily physical activity, and 21 percent smoke cigarettes. Uncontrolled diabetes can result in medical difficulties such as blindness, kidney disease and nerve damage. Diabetes is also an important risk factor for heart disease and stroke.

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Diabetes Specialist Now at West Park Hospital - mybighornbasin

The possible complications posed by diabetesheart disease and damage to eyes, feet, nerves and so forthare fairly familiar to the general public. But in recent years, scientists have been scrutinizing a risk that is both less well known and less understoodthe heightened likelihood of dementia.

Researchers have known for several years about diabetes and the higher risk of vascular dementia, the second most common kind. In ways, it seems only logical: Vascular dementia is caused by damaged blood vessels in the brain, just as diabetes hardens blood vessels elsewhere.

The latest research is focused on Alzheimers disease, the most common neurodegenerative disorder and one for which its harder to figure out the precise relationship with diabetes. On this much, many scientists agree: The rate of Alzheimers disease could be cut by close to half if diabetes could be abolished. The connection between the two is so strong that Suzanne M. de la Monte, one of the top researchers in the field, has said that many cases of Alzheimers could be dubbed Type 3 diabetes.

People who havent necessarily developed diabetes might still develop insulin resistance in the brain, said de la Monte, a professor of neurosurgery, pathology and laboratory medicine at Brown University. Thats why she uses the term Type 3 diabetesone doesnt necessarily cause the other. But in both cases, she said, people show certain markers at the cellular level.

Growing evidence supports the concept that Alzheimers disease is fundamentally a metabolic disease with molecular and biochemical features that correspond with diabetes mellitus and other peripheral insulin resistance disorders, de la Monte wrote in 2014 in the journal Biochemical Pharmacology.But the picture is more complicated than that, she wrote, because Alzheimers can occur as a separate disease process, or arise in association with systemic insulin resistance diseases, including diabetes, obesity, and non-alcoholic fatty liver disease.

A 2015 pilot study published in the Journal of Alzheimers Disease found that doses of nasal insulinbypassing the blood/brain barriersignificantly improved memory in people with early Alzheimers disease and mild cognitive impairment. A larger, five-year clinical trial is now underway.

Inflammatory response appears to play a role, de la Monte said. Both diabetes and Alzheimers are inflammatory diseases. And yet, other forms of brain inflammation are not associated with cognitive problems later on.

Multiple sclerosis, encephalitis, none of these lead to dementia, she said. What is the cause of all this, where is the problem? Because Alzheimers disease was not that common before. We can link a lot of the extra cases to diabetes and obesity. So if we have that, is it just because people are fatter? I dont think its just that. But then if you talk about metabolic syndrome, fatty liver, PCOS, infertilityhow many diseases are linked to the same problem of insulin resistance and an inflammatory process?

People need a lot more help in learning how to stave off Type 2 diabetes and other metabolic diseases through diet and exercise, de la Monte said. And because testing can find the same precursor conditions for both brain insulin-resistance and diabetes, theres reason to think more people should be screened earlier, with these more sensitive tests.

Melissa Schilling, a professor at the New York University Stern School of Business, came to a similar conclusion after conducting a review of the relationship between diabetes and Alzheimers.

Her 2016 paper in the Journal of Alzheimers Disease estimated that 40% of all Alzheimers cases were connected to hyperinsulinemia, or excess levels of insulin relative to glucose in the blood. That would include not just people with diabetes but the 86 million Americans estimated by the CDC to have prediabetes.

If we can raise awareness and get more people tested for hyperinsulinemia it could significantly lessen the incidence of Alzheimers disease and vascular dementia, as well as other diabetes-related health problems. Schilling said in a press release.

Research by Margaret Gatz, a professor of psychology at USC, further refined the relationship between the two. She and fellow researchers in Sweden found that not only was diabetes strongly associated with dementia, but that people who are first diagnosed with Type 2 diabetes in middle age, rather than after age 65, are at much higher risk. And that finding was independent of how long people had diabetes before developing dementiain other words, it was the age at diagnosis, not how long they lived with diabetes, that determined their risk.

At this point, Gatz is particularly interested in the role that stress might play in the equation.

One theory is Ive been intrigued by involves the HPA (hypothalamic-pituitary-adrenal) axis, she said. The HPA axis controls the immune system, digestionand reactions to stress. Its what fires when someone is stressed, Gatz said. After its heavily activated, it might potentiate diabetes and hippocampal damage, inflammation and oxidative damage.

Maybe this whole stress process is basically inflammatory damage, oxidative damage.

She agreed that earlier, more sensitive testing might warn people away from the kind of eating and sedentary habits that can cause prediabetes and diabetes. Stress management might be part of the picture, too, she suggests.

Im heavily a physical exercise proponent. When people ask me, Whats the biggest thing I can do to avoid dementia? my answer is exercise. But also workplaces are more stressful, people describe themselves as not handling stress as well. All of these are risk factors.

At Brown, de la Monte is planning to publish a paper that looks at what kinds of testing might best be used as early indicators of future dementia. Were looking at peripheral markers that indicate brain disease, she said. If you look at blood, can you find evidence of inflammation in people who have no inflammatory disease but they have mild cognitive impairment and they also have markers of insulin resistance? We can pick out from that who is at risk. At least to inform them so they can start using lifestyle measures.

Karin Klein is a freelance journalist based in Southern California who specializes in writing about health and medicine, education, environment and food. For 27 years, she covered those topics at the Los Angeles Times as an editor and editorial writer. Karin is a graduate of Wellesley College, where she majored in linguistics, and she studied journalism at UC Berkeley's Graduate School of Journalism. When she's not writing, she's usually found on hiking trails and is the author of an interpretive hiking book, "50 Hikes in Orange County." Follow her on Twitter athttps://twitter.com/kklein100

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You are here: Home News Industry news FDA approves first cancer treatment for any solid tumor with a specific biomarker

The US Food and Drug Administration (FDA) granted accelerated approval to a treatment for patients whose cancers have a specific genetic feature (biomarker). This is the first time the agency has approved a cancer treatment based on a common biomarker rather than the location in the body where the tumour originated.

Keytruda (pembrolizumab) is indicated for the treatment of adult and pediatric patients with unresectable or metastatic solid tumours that have been identified as having a biomarker referred to as microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR). This indication covers patients with solid tumours that have progressed following prior treatment and who have no satisfactory alternative treatment options and patients with colorectal cancer that has progressed following treatment with certain chemotherapy drugs.

This is an important first for the cancer community,

said Richard Pazdur, M.D., acting director of the Office of Hematology and Oncology Products in the FDAs Center for Drug Evaluation and Research and director of the FDAs Oncology Center of Excellence. Until now, the FDA has approved cancer treatments based on where in the body the cancer startedfor example, lung or breast cancers. We have now approved a drug based on a tumours biomarker without regard to the tumors original location.

MSI-H and dMMR tumors contain abnormalities that affect the proper repair of DNA inside the cell. Tumours with these biomarkers are most commonly found in colorectal, endometrial and gastrointestinal cancers, but also less commonly appear in cancers arising in the breast, prostate, bladder, thyroid gland and other places. Approximately 5% of patients with metastatic colorectal cancer have MSI-H or dMMR tumours.

Keytruda works by targeting the cellular pathway known as PD-1/PD-L1 (proteins found on the bodys immune cells and some cancer cells). By blocking this pathway, Keytruda may help the bodys immune system fight the cancer cells. The FDA previously approved Keytruda for the treatment of certain patients with metastatic melanoma, metastatic non-small cell lung cancer, recurrent or metastatic head and neck cancer, refractory classical Hodgkin lymphoma, and urothelial carcinoma.

Keytruda was approved for this new indication using the Accelerated Approval pathway, under which the FDA may approve drugs for serious conditions where there is unmet medical need and a drug is shown to have certain effects that are reasonably likely to predict a clinical benefit to patients. Further study is required to verify and describe anticipated clinical benefits of Keytruda, and the sponsor is currently conducting these studies in additional patients with MSI-H or dMMR tumours.

The safety and efficacy of Keytruda for this indication were studied in patients with MSI-H or dMMR solid tumours enrolled in one of five uncontrolled, single-arm clinical trials. In some trials, patients were required to have MSI-H or dMMR cancers, while in other trials, a subgroup of patients were identified as having MSI-H or dMMR cancers by testing tumour samples after treatment began. A total of 15 cancer types were identified among 149 patients enrolled across these five clinical trials.

The most common cancers were colorectal, endometrial and other gastrointestinal cancers. The review of Keytruda for this indication was based on the percentage of patients who experienced complete or partial shrinkage of their tumors (overall response rate) and for how long (durability of response). Of the 149 patients who received Keytruda in the trials, 39.6% had a complete or partial response. For 78% of those patients, the response lasted for six months or more.

Common side effects of Keytruda include fatigue, itchy skin (pruritus), diarrhea, decreased appetite, rash, fever (pyrexia), cough, difficulty breathing (dyspnea), musculoskeletal pain, constipation and nausea. Keytruda can cause serious conditions known as immune-mediated side effects, including inflammation of healthy organs such as the lungs (pneumonitis), colon (colitis), liver (hepatitis), endocrine glands (endocrinopathies) and kidneys (nephritis). Complications or death related to allogeneic hematopoietic stem cell transplantation after using Keytruda has occurred.

The FDA granted this application Priority Review designation, under which the FDAs goal is to take action on an application within six months where the agency determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing or preventing a serious condition.

The FDA granted accelerated approval of Keytruda to Merck & Co.

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FDA approves first cancer treatment for any solid tumor with a specific biomarker - European Pharmaceutical Review

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doi: 10.1097/01.NT.0000520472.01901.8f

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Two new reports on autologous hematopoietic stem cell transplantation (AHSCT) for multiple sclerosis (MS) indicate that the therapy may benefit some MS patients. But whether AHSCT is viable is a matter of debate among some MS experts, who contend that the regimen could be toxic, leading to infection and death.

Two new reports on autologous hematopoietic stem cell transplantation (AHSCT) for multiple sclerosis (MS) indicate that the therapy may benefit some MS patients. But whether AHSCT is viable is a matter of debate among some MS experts, who contend that the regimen, which uses a combination of cytotoxic drugs to ablate the immune system in an attempt to reset the immunological memory could be toxic, leading to infection and death.

Experts who were not involved with the study said that newer, second generation MS drugs may be safer options, though few studies comparing the method with these drugs have been undertaken.

The first new report, published in the April 28 online edition of Neurology, provided a meta-analysis of 15 studies involving 764 MS patients who underwent AHSCT. The report found that the risk-benefit profile of the therapy makes it best suited for patients who have aggressive, relapsing-remitting MS who have not yet become highly disabled.

The second report, which provided long-term outcomes for 281 MS patients from an observational, retrospective study, found that almost half of the patients remained free from neurological progression five years after AHSCT. The study, published in the April edition of JAMA Neurology, reported that younger age, relapsing form of MS, fewer prior immunotherapies, and lower baseline EDSS [Expanded Disability Status Scale] score were factors associated with better outcomes.

Maria Pia Sormani, PhD, professor of biostatistics at the University of Genoa in Italy, and lead author of the report in Neurology, told Neurology Today that skepticism about the treatment approach is likely due to multiple factors.

MS is not a lethal disease, and this procedure is very invasive and has a non-negligible mortality risk, said Dr. Sormani, who also was a study author on the JAMA Neurology study. The lack of data from a rigorous clinical trial of AHSCT for MS has also been problematic.

To gain a clearer picture of what the current evidence shows, her team's meta-analysis pooled data from 15 studies, mostly open label, from January 1991 to July 2016. The researchers found that treatment-related mortality (TRM) declined during the period covered by the review, likely a result of improvements in transplant techniques, more clinical experience, and better patient selection, Dr. Sormani said. Overall TRM was 2.1 percent, but after 2005 it was 0.3 percent.

The meta-analysis found that the rate of disease progression in patients was 17.1 percent at two years following AHSCT and 23.3 percent at five years. The analysis also found that 83 percent of patients had no evidence of disease activity (NEDA) at two years, and 67 percent had no evidence at five years. Doing the transplant earlier, before the patient develops much disability seems advantageous, Dr. Sormani said.

The meta-analysis had the usual limitations of such reviews, she noted. The original studies were not all designed or executed in the same way, patient selection and study methodology were not uniform, and transplant techniques and protocols varied.

Even with advanced immunotherapy, such as natalizumab or alemtuzumab, only 32-39 percent maintained NEDA at two years in the phase II clinical trials, wrote Joachim Burman, MD, PhD, of Uppsala University in Sweden and Robert Fox, MD, of the Cleveland Clinic, in the editorial accompanying the paper. They agreed with the research team that the approach is more likely to benefit those with RRMS, not those with progressive forms of MS.

The report in JAMA Neurology included data on 281 patients from 25 centers who underwent AHSCT between January 1995 and December 2006. Seventy-eight percent of the patients had progressive forms of MS. The median follow-up was 6.6 years, with some patients followed for as long as 16 years

The five-year probability of progression-free survival was 46 percent and overall survival was 96 percent, the research team headed by Paolo A. Muraro, MD, a clinical reader in neuroimmunology and deputy head of the division of brain sciences at Imperial College London.

Factors associated with neurological progression after transplant were older age, progressive (versus relapsing) form of MS, more than two previous disease-modifying therapies, and higher baseline EDSS scores.

An accompanying editorial coauthored by Michael K. Racke, MD, professor of neurology and neuroscience at Ohio State University, noted that while the transplant therapy appears to favor those with RRMS with aggressive breakthrough disease, it Z

Dr. Racke told Neurology Today in an interview that he is currently planning a multicenter randomized controlled trial, which will include 55 RRMS patients in each arm. The study will compare AHSCT using what is considered a medium-intensity myelobation (BEAM) technique to best available drug treatment (whatever treatment a given patent is taking).

Dr. Racke said one question that needs to be further considered is, When is the best time to do a transplant? He said drug therapies need to be given a chance, but earlier might be better than later because once you start getting damage to the central nervous system we can't really fix that.

He said the upcoming trial will likely include cost analyses to compare the cost of long-term drug therapy to the mostly upfront costs of transplant, which is thought to be a once-and-done procedure.

Commenting on the two studies, Timothy L. Vollmer, MD, FAAN, professor of neurology at University of Colorado Health Sciences Center and co-director of the Rocky Mountain MS Clinic at Anschutz Medical Center, expressed skepticism about using AHSCT, particularly in light of effectiveness of the second-generation MS drugs that have come into use, such as natalizumab for JCV negative patients, fingolimod, dimethyl fumarate, and ocrelizumab.

Dr. Vollmer said most studies of AHSCT for MS were done before the newer drugs were available. He is concerned about both the immediate risks (infection, death) and potential long-term consequences of undergoing a toxic regimen to eradicate the immune system, noting that it could cause brain atrophy, already a concern for MS patients.

Mark S. Freedman, MD, professor of neurology at the University of Ottawa, senior scientist at The Ottawa Hospital Research Institute, and director of the Multiple Sclerosis Research Unit at The Ottawa Hospital-General Campus, is more sanguine about the procedure.

In a 2016 report in The Lancet, he and a colleague described outcomes for 24 RRMS patients who underwent transplant after failing drug therapy. Dr. Freedman said he has done about 25 more cases since the study came out. He said no patient has experienced a clinical relapse following transplant, none has evidence of new brain lesions on MRI, and none requires disease-modifying medication.

Dr. Freedman said there is a high level of interest in the procedure among MS patients, but it isn't for everyone. Patients must be carefully selected for the procedure, and undergo an aggressive chemotherapy regimen to eliminate their immune system, he said, noting that those with a high inflammatory component to their disease are ideal. Harvested stem cells undergo a special sorting technique at his center before being infused into the body to make sure that no previous disease-causing lymphocytes are accidentally included.

We're taking away immunologic memory, Dr. Freedman said. The new immune system is learning all over again what it should and shouldn't be doing.

He said that while the procedure is only done in patients who have not fared well with drug therapy, the best timing for this treatment would be as early as possible, when disability is minimal.

Probably doing it within five years from the onset of illness would give the optimal results, he said.

Read more here:
Is Autologous Hematopoietic Stem Cell Transplantation Still Viable for MS? - LWW Journals

Education and Technology:

Microsoft Learning Tools is software that helps improve reading skills by reducing visual crowding, highlighting words, and reading text aloud, so students can engage with words in a whole new way.

Again?

Age-related macular degeneration (AMD). You probably havent heard much about it, but if you live in the United States, its the most likely way youll lose your eyesight as you get older. Basically, the cells in your retina that detect lightaka the maculatend to decline as you age, leading to vision loss and, in some cases, total blindness.

But a recent clinical trial tested an unorthodox method to treat and potentially prevent this from happening: giving patients a man-made virus. The trials findings, published last week in The Lancet, show that administering viruses may help doctors stall vision loss and eventually make age-related blindness a thing of the past.

For the trial, doctors gave the virus to 19 patients with advanced, wet age-related macular degeneration, a type of AMD that causes abnormal blood vessels beneath the macula to leak fluid. White Americans over the age of 80 are particularly vulnerable when it comes to getting the chronic eye disease. All the patients in this new trial were 50 years old or older and had little success with standard treatments. Though the virus was not drastically different from a common cold, doctors intended for it to jump-start the patients immune systems and help their eyes drain the problematic eye fluid.

What they found was substantial fluid reduction in four of the patients, some fluid reduction in two patients, and five who saw no improvement. According to Mic, doctors didnt expect eight of the patients to see any improvement from the outset. But for the five who were expected to see some improvement and didnt, it seems their bodies already had antibodies to ward off the homemade virus, deeming it ineffective. But for those who literally saw progress as a result of the virus, the trial is definitely worth replicating.

Read more:
Curing Blindness May Be As Simple As Getting A Virus - GOOD Magazine

Tucked inside a small laboratory at UConns Technology Incubation Program (TIP) in Farmington, Conn., Nicole Wagner is trying to cure vision impairment and blindness for more than 30 million people worldwide.

Using a protein, grown in the laboratory and implanted behind the retina, this promising new procedure offers hope for patients with age-related macular degeneration (AMD) and other retinal diseases.

These are terrible diseases that truly impact the quality of life for many people, said Wagner, the president and CEO of LambdaVision. To offer patients the possibility of restoring their vision provides them the chance to see a new grandchild, resume a golf game, drive again or read a favorite book. For many people, restored vision would allow them to return to an independent life.

LambdaVision uses a light-activated protein, bacteriorhodopsin, to stimulate the retina of patients suffering from impaired or lost vision due to retinal degenerative diseases. The protein, isolated from high-salt environments, including the Dead Sea, is grown and purified in the laboratory. The protein works by absorbing light and converting it into a signal that is picked up by specialized cells in the retina, relayed to the optic nerve and ultimately interpreted by the brain.

More than 31 million people worldwide suffer from irreversible vision loss caused by macular degeneration and retinitis pigmentosa. The incidence of blindness caused by retinal degenerative diseases is increasing at a rapid rate due to an increase in the global geriatric population, Wagner said.

LambdaVisions implant can restore high-quality vision to those patients who are no longer candidates for traditional treatments and have end-stage retinal degeneration, Wagner said. Current treatments only succeed in slowing the progression of disease.

LambdaVision was founded through support from UConns Technology Commercialization Services in 2009. Dr. Robert R. Birge, distinguished professor of chemistry at UConn, led a research group that included Wagner.

The protein is in pre-clinical trials across the country to determine the stability and efficacy of the implant.

LambdaVision has been incredibly fortunate to have the continued support of UConn and the State of Connecticut, and we owe much of our success to the incredible mentors that have helped us to propel the research and development and commercialization of the technology, she said. In the early stages of development, they were the believers.

LambdaVision has won many awards, including most recently: a 2016 UConn SPARK Technology Commercialization Fund Award and the prestigious 2016 MassChallenge CASIS-Boeing Prize for Technology, which allows the company to carry out experiments aboard the International Space Station. Since gravity can interfere with the uniformity of the retinal implant films, the hope is that work done in microgravity will be faster and yield improvements in the homogeneity and stability of the product.

The company also won the $15,000 Wolff New Venture Prize, sponsored by UConns Connecticut Center for Entrepreneurship and Innovation (CCEI) and a National Science Foundation Small Business Innovation Research Grant.

To be on the brink of a new and exciting medical breakthrough is thrilling, Wagner said. Im very eager to see this technology available in the medical community where it can make a difference in peoples lives.

More:
Farmington Startup Sets Sights on Curing Retinal-Disease Blindness - UConn Today

The expo included an awards ceremony, where awards for outstanding high school and elementary school student and outstanding employee/employer were presented, as well as an independence award, and an equal access award.

Attendees to the expo explored how Pennsylvanians who are blind or visually impaired overcome challenges in education, employment, and independence. BBVS offered simulations of varying visual impairments, while other vendors and organizations gave hands-on demonstrations with guide dogs, tools, and services that facilitate living with a visual impairment.

This year's Master of Ceremonies was Carlton Anne Cook Walker, a certified teacher of students with blindness and visual and multiple impairments. She served as an itinerant teacher of blind and low-vision students in South Central Pennsylvania for more than six years. Currently, she serves as Project Manager for the NFB BELL Academy (National Federation of the Blind Braille Enrichment for Literacy and Learning Academy), a nationwide program which provides summer learning experiences for hundreds of blind and low-vision students ages four through twelve.

In Pennsylvania, it is estimated that more than215,000 individuals aged 40 and under experience severe vision problems. Of those, more than 69,000 experience total blindness. For those over the age of 50, more than 1.76 million suffer from a severe visual impairment.

Following is a list of award recipients:

Outstanding Elementary School Student #AccessEqualsSuccess in Education Video Award Nathan Craig. Watch Nathan's video: https://youtu.be/45KFlBXPtvA.

Outstanding Middle School Student #AccessEqualsSuccess in Education Video Award Jaylen Hallowell and Simon Bonenfant. Watch Jaylen's video: https://youtu.be/Sivw-km-UFc. Watch Simon's video: https://www.youtube.com/watch?v=LwZjl1sTeXk.

Outstanding High School Student #AccessEqualsSuccess in Education Video Award Kayla McDonough. Watch Kayla's video: https://m.youtube.com/watch?v=ZEn8v11j1YQ.

Outstanding Individual #AccessEqualsSuccess in Employment AwardRen Wang

Outstanding Employer #AccessEqualsSuccess in Employment Award Bayer HealthCare, LLC

Outstanding Individual #AccessEqualsSuccess in Independence Award Ron Ream

Outstanding Business #AccessEqualsSuccess in Independence Award PSECU

Great Lakes Regional Braille Readers Are Leaders Award Andrew Godwin

For more information, please visit the BBVS website, or contact the BBVS at 717-787-6176 or bbvs@pa.gov.

Media Contact: Lindsay Bracale, 717-787-7530

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/pa-department-of-labor--industry-hosts-blindness-awareness-expo-300462593.html

SOURCE Pennsylvania Department of Labor & Industry

http://www.state.pa.us

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PA Department of Labor & Industry Hosts Blindness Awareness Expo - PR Newswire (press release)

New book exposes Willful Blindness in connection with murder conviction of local rabbis son

Zeke Goldblum and his mother, Evelyn Goldblum. (Photo provided by Orah Miller)

Willful Blindness, which was published last November and is available at amazon.com and other online retailers, is edited by former Post-Gazette writer David Bear and primarily authored by James Ramsey, a former narcotics detective turned private investigator who has spent the last 10 years researching Goldblums case.

Since the incarceration of Goldblum in 1977, several high-profile figures have come out in support of his release, most notably the prosecuting attorney that tried the case, Peter Dixon, and retired U.S. District Judge Donald Ziegler, who presided over the trial. Dixon and Ziegler, as well as renowned forensic pathologist Dr. Cyril Wecht, have all submitted letters or affidavits at various clemency and commutation proceedings throughout the last three decades, claiming that the evidence and extenuating circumstances require that Goldblum be released.

Still, after 40 years, Goldblum, the son of the late Rabbi Moshe Goldblum who served for 24 years as the spiritual leader of Congregation Beth Shalom, remains behind bars, currently confined at the State Correctional Institution Mahanoy in Schuylkill County.

He is 67 years old, walks with a cane and has other health issues.

On February 10, 1976, Goldblum, a 26-year-old law school graduate working with Ernst and Young, was arrested for his purported involvement with a murder. The previous evening, the victim, George Wilhelm, had been stabbed and thrown off the rooftop of the Smithfield/Liberty parking garage downtown. Rather than falling to the street, Wilhelm landed on the roof of the pedestrian bridge one floor below, and, though mortally wounded, he survived long enough to tell a policeman the name of his assailant: Clarence Miller.

That statement is known in the law as a dying declaration. It is typically given significant evidentiary weight, as it is presumed that a person on his deathbed will tell the truth.

Nonetheless, Wilhelms dying declaration was not enough to raise a reasonable doubt in the minds of the jury, and in 1977, Goldblum was convicted of first-degree murder. He was sentenced to life imprisonment, plus 15 to 30 years.

Bear, a longtime Squirrel Hill resident, began researching the case about two years ago.

When I got involved, I was amazed not only at what was discovered, but the whole process the state uses to deal with life sentences, Bear said, noting how rare it is in the state for a lifer to get his sentence commuted. The attitude of state officials, he continued, is that life means life.

There have been a number of actions brought by the family and others to overturn the wrongful conviction, he said, but only one of those applications the one filed in 1998 received a public hearing. In addition to letters of support from Ziegler and Dixon, the medical examiner on the case, Joshua Perper also said that Zeke didnt do it and that he shouldnt be in prison, according to Bear.

The matter of Goldblums continued incarceration goes beyond his particular case, according to Bear, and to the broader issue of geriatric lifers in Pennsylvania who are routinely denied clemency.

I hope this logjam will break, Bear said. They shouldnt just deny them all.

Willful Blindness is a thorough review of the circumstances of Goldblums case, including a look at media reports at the time, alleged cover-up of evidence and purported misconduct among those seeking a conviction for Goldblum.

Bear presented a talk about his findings at an adult education event at Beth Shalom last week, at which Zekes brother David Goldblum from Baltimore was present, as was his sister Orah Miller, who resides in Israel. The family continues to be active in seeking Goldblums release, and The Chronicle featured an in-depth article about those efforts in December 2015.

Maybe this book will help, Bear said. Its all I can do.

Toby Tabachnick can be reached at tobyt@thejewishchronicle.net.

Excerpt from:
New book exposes 'Willful Blindness' in connection with murder conviction of local rabbi's son - thejewishchronicle.net

Using nanoparticles to carry genes rather than viral vectors could be safer, simpler and allow for larger genes to be transported

24 May 2017 by Selina Powell

Researchers have used gene-carrying nanoparticles to prevent sight loss in mice with a human form of leber congenital amaurosis (LCA), in a study published in Molecular Therapy Nucleic Acids.

The inherited cause of blindness affects two to three babies in every 100,000 newborns, according to the National Institutes of Health.

Although the research focused on leber congenital amaurosis 2, or LCA2, scientists believe the study holds promise for other forms of LCA and inherited diseases that lead to severe vision loss or blindness.

Study author Dr Zheng-Rong Lu, of Case Western Reserve University, told OT that using the nanoparticles for gene delivery had advantages over traditional viral forms of delivery.

The nanoparticles were easy to produce, safe and had unlimited cargo capacity, Dr Lu explained.

Right now, genetic visual disorders are a major cause of retinal degeneration and severe vision loss, yet there are no approved therapies to treat these diseases, Dr Lu shared.

With gene therapy, we can cure the disease by delivering a healthy copy of the mutated gene directly to the cells that need to use it. However, this promise cannot be realized without a safe and effective gene delivery system to carry the gene into the target cells, he emphasised.

Gene replacement therapy using the delivery system in mice with LCA2 resulted in improved vision for more than 120 days.

Dr Lu highlighted that while the gene delivery system had potential, further improvements were needed. Future work would focus on prolonging gene expression and improving tissue specificity.

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Fine-tuning gene delivery to combat childhood blindness - AOP

ANDOVER, Kan. (KAKE) -

The same old routine at the middle school track meet: waiting your turn, watching dozens of events. Then finally, you get the call. But that's a call Rich Yamamoto almost didn't get.

"My coach passed me up and goes, 'Hey, why weren't you at practice yesterday?'I was like, 'Because you didn't tell me I had to run,'and so he goes, 'Yeah,we have a spot for you at the meet if you want to join.' I'm like, 'OK.'"

It wasn't a normal race for Rich. At this meet, the 8th gradergot to run with his dad.

"It's really neat," Rich's mother Jennifer said. "It's nice that they have something they can spend time together with and have bonding over."

Before the race, Rich sets a goal. The race starts. Rich's dad, Richard, is his guide. The reason for that: Rich is blind.

Richard leads his son while letting him know his pace. Four laps around the track for one mile and Rich brings it home.

In a race he almost wasn't going to run, Rich set a personal record, leaving behind a legacy at a routine middle school track meet not soon to be forgotten.

Rich says he plans to run track as a freshman next year.

The Yamamotos are always looking for dedicated runners to work with Rich. If you or someone you know would like to help, email the Yamamotos.

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Blindness doesn't stop Andover middle school runner - KAKE

Forbes

Puma Biotechnology FDA Live Blog Forbes This is a live blog of the meeting of the Food and Drug Administration's meeting regarding neratinib, a breast cancer drug being developed by Puma Biotechnology. The basic questions to be addressed, per my story from Monday. Puma's not applying to sell ... Why Puma Biotechnology Inc Jumped Higher TodayMotley Fool Puma Biotechnology Receives FDA Advisory Committee Support for NeratinibBusiness Wire (press release) Puma Biotechnology (PBYI) PT Raised to $86 at BofA/Merrill LynchStreetInsider.com ExpertGazette -Equities.com -Finance News Daily -Zacks all 71 news articles »

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Puma Biotechnology FDA Live Blog - Forbes

Amicus Therapeutics (NASDAQ:FOLD) is a global biotherapeutics company focused on rare genetic devastating diseases. The company has advanced its precision medicine, Galafold (migalastat), in treating patients in Europe with Fabry disease (alpha galactosidase A deficiency), a rare X-linked genetic lysosomal disorder in which sphingolipids are not metabolized properly. Galafold, an orally administered drug, is the first medicine approved (EMA but not yet FDA) for treatment of Fabry disease. Other goals for 2017 include submitting a J-NDA (Japan) for migalastat, establishing a clinical plan for ATB200/AT2221 in Pompe disease, completion of phase 3 clinical trial in epidermolysis bullosa.

FOLD announced its regulatory plan with FDA for U.S. treatment with Galafold in advancing it's Fabry disease program including two phase 3 trials in late 2016. As previously mentioned, the EMA approved use of Galafold for treatment of Fabry disease. The company published data from its pivotal trial in the New England Journal of Medicine. A statistically significant benefit was conferred by Galafold in 50 patients with treatable mutant galactosidase alleles. However, a closer look at the trial endpoints reveals that the study failed to reach its primary endpoints including greater than 50% reduction in GL-3 inclusions per kidney, and if all 67 patients were included in the study (including mutant alleles that are not expected to benefit by Galafold). More studies are needed for FDA going forward. It could be well worth the company's investment, as the market for Fabry's disease is estimated to be in excess of $1.2 billion by 2024.

December 2016, FOLD announced positive early phase 1/2 data for Pompe disease, a rare genetic disorder leading to the buildup of glycogen in the body, particularly the muscles, which become impaired in function. The study showed a positive safety profile with no serious adverse events and generally showed musculatoprotection as shown by biomarkers of muscle damage. The study is divided into three cohorts: non-ambulatory ERT-switch, ERT-switch, and ERT-naive. Creatine kinase, alanine aminotransferase, and aspartate aminotransferase levels showed a trend towards improvement in half the patients and were stable in all. ATB200/AT2221 has a unique mechanism of treatment that uses ATB200, a recombinant functional alpha-glucosidase enzyme carrying mannose-6 phosphate moieties designed to increase uptake. AT2221 is a pharmacological chaperone co-treated to stabilize the compound. The company believes that its Pompe program (with market of $1.2 billion) will be a driver in its growth with 12% CAGR as a world's leading rare disease company. Key study readout dates include Q2 and Q3 2017.

SD-101 is currently in phase 3 studies as a topical for Epidermolysis Bullosa, and FOLD believes it will be the first-to-market therapy for the rare indication. The inherited disease is characterized by blistering of keratinized outer skin, wet skin (such as mouth), and internal organs. Serious complications include infection, pain, and even death. The company was granted FDA Breakthrough Therapy Designation in 2013 based on results from its phase 2a study, having demonstrated wound closure in all disease types. Strong Bio has previously written about the impacts of FDA Breakthrough Therapy Designation on biotechnology stocks, and if you are an investor and have not perused the article, now might be a good time, as it is a part of the investment plan for FOLD. Phase 3 top-line data for the 160 patient study in which 95% of the patients elected to continue the open-label extension is due Q3 2017. This statement could be the company's way of saying they find it likely it will have clinical benefit for patients. Since it has been observed that FDA breakthrough therapy status stocks get volatile late in stage 3, any sharp drops may indicate stock manipulation that interested investors might jump on with a small position. Strong Bio regards FOLD as a nice investment prospect for any unexplainable late Q2 early Q3 pullbacks. With significant market potential of $1 billion, severe symptoms, and 30,000 sufferers in the U.S. alone, its worth watching for that pullback. One competitor, RegeneRx (OTCQB:RGRX) has initiated phase 3 trials for RGX-137 (active ingredient thymosin beta 4 wound healing gel) in the condition as well.

Cash burn was $55 million in first quarter 2017. Cash on hand at end Q1 was $280 million. The current runway is expected to last through the second half of 2018. Seven analysts average about $12 per share for FOLD, which is currently trading at about $8, which may be a slight pullback from fair value. Strong Bio will look for dips in price below $6 for no a brainer initial position. If all three drugs get FDA approval, this stock could be off to the races. Strong Bio has learned a lesson from Amicus. Rare diseases may have surprisingly large markets!

Risk factors for FOLD could include dilution (which may be yet another entry opportunity). Risks also include FDA approval and/or regulatory delays for all three major indications. Clinical trial design will be key, because FDA wants a clear metric upon which to agree with FOLD to approve these rare disease therapies. Large scale GMP-compliant manufacturing for U.S. application will also be a significant but manageable obstacle. Because the company has a market cap over $1.1 billion dollars, SD-101 key readout will be very important in terms of valuation for FOLD stock. If approved by FDA, the stock could easily triple in value over the next year. With ATB200/AT2221 being regarded as a key driver in value by the company, 2017 is going to be a pivotal year for FOLD. With three candidates all on the verge of pivotal data, this is a must-watch!

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Amicus Therapeutics: A Rare Find In Biotechnology - Seeking Alpha

Wednesday was a good day for stocks, and the Dow Jones Industrials and S&P 500 both climbed through milestone levels. Most market participants attributed the positive sentiment to the Federal Reserve, which released the minutes of its latest monetary policy meeting during the afternoon. The central bank revealed plans to clamp down on the size of its balance sheet, which it initially expanded in the aftermath of the financial crisis to provide liquidity to the bond market and additional stimulus to the U.S. economy. Investors were pleased that the Fed believes that it's no longer necessary to extend that level of monetary accommodation to the economy. In addition, some individual companies had extremely good news, and Abercrombie & Fitch (NYSE:ANF), Triumph Group (NYSE:TGI), and Puma Biotechnology (NASDAQ:PBYI) were among the best performers on the day. Below, we'll look more closely at these stocks to tell you why they did so well.

Shares of Abercrombie & Fitch climbed 6% in the wake of reports that the teen retailer might receive an acquisition bid from a consortium of investors. According to The Wall Street Journal(subscription required), industry peer American Eagle Outfitters (NYSE:AEO) and private equity company Cerberus Capital Management are looking at putting together a potential buyout offer for Abercrombie & Fitch, following speculation that other players in the industry might also be interested in consolidation. A&F has been dealing with takeover speculation for a while, and it has typically noted that any discussions wouldn't necessary translate into actual offers. Yet with Abercrombie set to release its first-quarter financial results Thursday, investors will want to see signs that the company can take care of its challenges on its own -- or else they'll start clamoring more loudly for a buyout to take place.

Image source: Getty Images.

Triumph Group stock soared over 30% after the company announced its fiscal fourth-quarter financial results and resolved a dispute with aircraft manufacturer Bombardier. The aerospace components and systems specialist said that sales fell 13% from year-ago levels, and it posted a GAAP loss of $126.8 million. With challenges in its aerospace structures business, Triumph has focused on amending contracts and addressing operational and financial challenges, and the company's transformation plan has led to improving free cash flow and cost savings. Investors were also happy that Triumph reached a settlement of all of its disputes with Bombardier. Triumph said that the agreement "resets the commercial relationship between [Triumph] and Bombardier and allows each of them to better achieve their business objectives going forward."

Finally, shares of Puma Biotechnology jumped 30%. The biopharmaceutical company earned a hoped-for approval from the U.S. Food and Drug Administration advisory panel looking at its neratinib candidate treatment for breast cancer. The panel voted 12 to 4 in favor of recommending the drug to the FDA, and although panelists expressed some thoughts about potentially limiting the size of the group of women eligible to use the drug, investors nevertheless took the news as a positive. The FDA still needs to make its own decision about Puma's drug, and it isn't bound by the opinion of the advisory panel. Nevertheless, today's recommendation moves Puma one step further to getting a big win under its belt, and shareholders recognized that fact with the second big move in the stock this week.

Dan Caplinger has no position in any stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy.

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Why Abercrombie & Fitch, Triumph Group, and Puma Biotechnology Jumped Today - Motley Fool

While 2017 has been a pretty good year overall for biotechs - the two largest ETFs in the space, the iShares Nasdaq Biotechnology ETF (NASDAQ:IBB) and the SPDR S&P Biotech ETF (NYSEARCA:XBI), are up 10% and 17%, respectively - it's tough to forget that both of these funds are still about 25% off of their 2015 highs. IBB, which is heavily influenced by the largest biotech names, has been impacted by 50% drops in Gilead (NASDAQ:GILD) and Biogen (NASDAQ:BIIB). XBI has a much more diversified all-cap mix but has experienced similar results.

But not all news coming out of the sector is bad. In fact, one biotech ETF has been downright ripping it since its launch at the end of 2014. The BioShares Biotechnology Products ETF (NASDAQ:BBP), which invests in companies that have at least one primary product that's received FDA approval, is up roughly 42% since its inception at the end of 2014 compared to a loss of 4% for IBB during the same time frame.

IBB Total Return Price data by YCharts

As fund advisor Virtus says on its website, companies that the fund invests in are "typically more established companies with much clinical trial failure risk behind them. They have already successfully completed multiple human clinical trials and have received FDA approval to sell and market a drug." That sounds a lot like IBB so what's the big differentiator between the two funds? It's BBP's focus on small- and micro-cap biotechs. Nearly 60% of fund assets are dedicated to this space whereas IBB has nearly 40% of the portfolio alone invested in the big five of Regeneron (NASDAQ:REGN), Biogen , Celgene (NASDAQ:CELG), Amgen (NASDAQ:AMGN) and Gilead .

So what did BBP have going for it that IBB didn't over the past year or so? I think it's a combination of portfolio construction and M&A.

BBP has a portfolio that has performed almost as well as can be expected, especially in 2017. Take a look at this chart with the year-to-date performance of the fund's biggest components.

That's exactly what you want to see out of your ETFs - the largest holdings performing the best. Part of that is due to the fact that the fund is equal-weighted and rebalanced semiannually (the last rebalance was done on December 15th). Still, that's a lot of companies whose stocks have risen by 20% or more.

Among the top 10 holdings, all have posted double-digit gains with seven components delivering 25%+ gains.

The other advantage the fund has is that a number of its holdings are in the sweet spot of being developed enough to generate meaningful revenue from their approved product line yet being small enough that they can be potential takeover targets. We've seen that within the fund multiple times recently. Relypsa (RYLP) has a top holding when it got bought out by Galenica (OTC:GNHAY). Not an M&A deal, but the fund's stake in Progenics (NASDAQ:PGNX) spiked when its partnership with Valeant (NYSE:VRX) was announced. Current holdings such as Exelixis (NASDAQ:EXEL) and Acadia (NASDAQ:ACAD) have been rumored as potential takeover targets for a while so further action could be in store for the fund in the near future as well.

Conclusion

Equal weighting the mature biotech players has been a strategy that's paid off for investors in the last year. The relatively limited exposure to the likes of Celgene and Gilead, which are still nearly 50% off of recent highs, has made the fund more attractive than its more well-known counterpart.

This fund will underperform when the mega-cap biotech names begin to rally again, but over the long-term this ETF should hold up well to IBB given its more diversified portfolio.

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Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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BBP: A Diversified Biotech ETF - Seeking Alpha

The Technical Chart For Puma Biotechnology, Inc. (PBYI) Is Very Revealing Today NY Stock News The technicals for Puma Biotechnology, Inc. (PBYI) has spoken via its technical chart and the message is loud and clear. Based on that message, this is the relevant information necessary to make sense of that current setup. Often the difference between ... and more »

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The Technical Chart For Puma Biotechnology, Inc. (PBYI) Is Very Revealing Today - NY Stock News