StemCell Therapy

LONDON (Reuters) - Gene therapy, which aims to patch faulty genes with working DNA, has been a long time in development. The following are major milestones:

1972 - Researchers first suggest gene therapy as a treatment for genetic diseases but oppose its use in humans "for the foreseeable future", pending greater understanding of the technology.

1990 - A four-year-old girl with severe immunodeficiency became the first patient to undergo gene therapy in the United States.

1999 - American patient Jesse Gelsinger dies following a gene therapy experiment, setting the field back several years as U.S. regulators put some experiments on hold.

2002-03 - Cases of leukaemia are diagnosed in French children undergoing gene therapy in a further blow to the field.

2003 - The world's first gene therapy is approved in China for the treatment of head and neck cancer.

2007 - Doctors carry out the world's first operation using gene therapy to treat a serious sight disorder caused by a genetic defect.

2012 - Europe approves Glybera, the first gene therapy in a Western market, for an ultra-rare blood disorder.

2016 - Europe approves Strimvelis for a very rare type of immunodeficiency.

2017 or 2018 - The first gene therapy could be approved in United States.

Reporting by Ben Hirschler; editing by David Stamp

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Timeline: Gene therapy's long road to market - Reuters

LONDON - The science of gene therapy is finally delivering on its potential, and drugmakers are now hoping to produce commercially viable medicines after tiny sales for the first two such treatments in Europe.

Thanks to advances in delivering genes to targeted cells, more treatments based on fixing faulty DNA in patients are coming soon, including the first ones in the United States.

Yet the lack of sales for the two drugs already launched to treat ultra-rare diseases in Europe highlights the hurdles ahead for drugmakers in marketing new, extremely expensive products for genetic diseases.

After decades of frustrations, firms believe there are now major opportunities for gene therapy in treating inherited conditions such as haemophilia. They argue that therapies offering one-off cures for intractable diseases will save health providers large sums in the long term over conventional treatments which each patient may need for years.

In the past five years, European regulators have approved two gene therapies - the first of their kind in the world, outside China - but only three patients have so far been treated commercially.

UniQure's Glybera, for a very rare blood disorder, is now being taken off the market given the lack of demand.

The future of GlaxoSmithKline's Strimvelis for ADA-SCID - or "bubble boy" disease, where sufferers are highly vulnerable to infections - is uncertain after the company decided to review and possibly sell its rare diseases unit.

READ:Researchers use gene editing on human embryo for first time in US

Glybera, costing around $1-million (R13-million) per patient, has been used just once since approval in 2012. Strimvelis, at about $700,000, has seen two sales since its approval in May 2016, with two more patients due to be treated later this year.

"It's disappointing that so few patients have received gene therapy in Europe," said KPMG chief medical adviser Hilary Thomas. "It shows the business challenges and the problems faced by publicly-funded healthcare systems in dealing with a very expensive one-off treatment."

These first two therapies are for exceptionally rare conditions - GSK estimates there are only 15 new cases of ADA-SCID in Europe each year - but both drugs are expected to pave the way for bigger products.

The idea of using engineered viruses to deliver healthy genes has fuelled experiments since the 1990s. Progress was derailed by a patient death and cancer cases, but now scientists have learnt how to make viral delivery safer and more efficient.

Spark Therapeutics hopes to win US approval in January 2018 for a gene therapy to cure a rare inherited form of blindness, while Novartis could get the USgo-ahead as early as next month for its gene-modified cell therapy against leukaemia - a variation on standard gene therapy.

At the same time, academic research is advancing by leaps and bounds, with last week's successful use of CRISPR-Cas9 gene editing to correct a defect in a human embryo pointing to more innovative therapies down the line.

Pay-for-performance

Spark Chief Executive Jeffrey Marrazzo thinks there are specific reasons why Europe's first gene therapies have sold poorly, reflecting complex reimbursement systems, Glybera's patchy clinical trials record and the fact Strimvelis is given at only one clinic in Italy.

He expects Spark will do better. It plans to have treatment centres in each country to address a type of blindness affecting about 6,000 people around the world.

Marrazzo admits, however, there are many questions about how his firm should be rewarded for the $400-million it has spent developing the drug, given that healthcare systems are geared to paying for drugs monthly rather than facing a huge upfront bill.

A one-time cure, even at $1-million, could still save money over the long term by reducing the need for expensive care, in much the same way that a kidney transplant can save hundreds of thousands of dollars in dialysis costs.

But gene therapy companies - which also include Bluebird Bio, BioMarin, Sangamo and GenSight - may need new business models.

One option would be a pay-for-performance system, where governments or insurers would make payments to companies that could be halted if the drug stopped working.

READ:20 years after cloning Dolly: Everything you always wanted to know

"In an area like haemophilia I think that approach is going to make a tonne of sense since the budget impact there starts to get more significant," Marrazzo said.

Haemophilia, a hereditary condition affecting more than 100,000 people in markets where speciality drug makers typically operate, promises to be the first really big commercial opportunity. It offers to free patients from regular infusions of blood-clotting factors that can cost up to $400,000 a year.

Significantly, despite its move away from ultra-rare diseases, GSK is still looking to use its gene therapy platform to develop treatments for more common diseases, including cancer and beta-thalassaemia, another inherited blood disorder.

Rivals such as Pfizer and Sanofi are also investing, and overall financing for gene and gene-modified cell therapies reached $1-billion in the first quarter of 2017, according to the Alliance of Regenerative Medicine.

Shire CEO Flemming Ornskov - who has a large conventional haemophilia business and is also chasing Biomarin and Spark in hunting a cure for the bleeding disorder - sees both the opportunities and the difficulties of gene therapy.

"Is it something that I think will take market share mid- to long-term if the data continues to be encouraging? Yes. But I think everybody will have to figure out a business model."

Reuters

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Drugmakers' hopes for gene therapy rise despite tiny sales in Europe - eNCA

Updated Aug. 8, 2017 at 7:02 a.m.

Published: 2017-08-07 16:07:00 Updated: 2017-08-08 07:02:05

Sanford, N.C. Pharmaceutical giant Pfizer Inc. plans to invest $100 million in its Sanford operations as part of a push into gene therapy, officials said Monday.

The effort builds on a technology developed at the University of North Carolina at Chapel Hill and will create 40 jobs in Sanford.

"Pfizer is proud to further expand our presence in North Carolina, particularly as we build our leadership in gene therapy," Lynn Bottone, site leader at Pfizer Sanford, said in a statement. "We look forward to the next phase of this expansion as we build a clinical and commercial manufacturing facility."

Preliminary work on the expansion and initial hiring have already begun. The 230-acre campus employs about 450 people, reports the N.C. Biotechnology Center.

Gene therapy is a potentially transformational technology for patients that involves highly specialized, one-time treatments to address the root cause of diseases caused by genetic mutation. The technology involves introducing genetic material into the body to deliver a correct copy of a gene to a patients cells to compensate for a defective or missing gene.

Last year, Pfizer acquired Bamboo Therapeutics Inc., a privately held biotechnology company in Chapel Hill focused on developing gene therapies for the potential treatment of patients with certain rare diseases related to neuromuscular conditions and those affecting the central nervous system. Pfizer also committed $4 million to support postdoctoral fellowships in North Carolina universities for training in gene therapy research.

"We are excited that Carolinas research will improve lives and create jobs for North Carolinians," UNC-Chapel Hill Chancellor Carol Folt said in a statement. "This is a perfect example of how placing innovation at the center of our university creates new opportunities. We are proud to be a part of the technologies, expertise and infrastructure that went into Bamboo Therapeutics and helped make this manufacturing expansion in Sanford possible. Gene therapy is a strength at Carolina, and we look forward to continue to help advance this industry."

Pfizer is also expanding a drug-manufacturing facility in Rocky Mount that it acquired from Hospira in 2015. The $190 million project will add 65,000 square feet of sterile injectable facilities but will not create any new jobs. The plant employs about 300 people.

Gov. Roy Cooper visited Pfizers Sanford facility last week to take a tour and meet with the companys senior leaders.

"North Carolina is one of the few places in the country with the biotech resources to take an idea all the way from the lab to the manufacturing line," Cooper said in a statement. "Pfizers investment in Lee County is a prime example of how North Carolinas world-class universities and cutting-edge industries work together to move our state forward."

Pfizer qualified for a performance-based grant of $250,000 from the One North Carolina Fund, which provides state assistance matched by local governments to help attract economic investment and create jobs. Companies receive no money upfront and must meet job and investment targets to obtain payment.

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Pfizer to invest $100M in Sanford gene therapy operation, add jobs ... - WRAL Tech Wire

Bluebird Bio plans to bring its gene therapies to market in Europe before the U.S., thanks to a favorable regulatory pathway.

Bluebird's head of Europe, Andrew Obenshain, told the Daily Telegraph that the company is already in negotiations with the EMA and the U.K.'s Medicine and Healthcare products Regulatory Agency (MHRA) on possible regulatory filings.

The EMA's adaptive pathways processwhich allows new therapies to be approved in stages based on stepwise collection of datais a key part of that decision, as is the fact that the agency "works very closely with companies coming forward with new methodologies," said Morgan. And with Brexit looming, it makes sense to discuss these plans with the MHRA separately.

Two years ago, Bluebirdwhich targets severe genetic diseases and cancerwas hit hard when the NorthStar trial of lead therapy LentiGlobin failed to hit the mark in sickle cell disease and beta thalassemia, mainly because of variable patient responses to the treatment.

In a recent SEC filing, the company said that combined data from Northstar and other trials, including a follow-up Northstar-2 study, "could support the filing of a marketing authorization application in the EU" for transfusion-dependent thalassemiaprovided they all meet the primary objective of freeing patients from the need for regular blood transfusions.

So far, no approved gene therapies have been in the U.S., while Europe has seen two approvals, namely for UniGene's Glybera (alipogene tiparvovec) for lipoprotein lipase deficiency and GlaxoSmithKline's Strimvelis for the ultrarare "bubble boy syndrome," or ADA-SCID.

Even getting approval is no guarantee of success, however. Glybera was taken off the market in April due to a lack of demand for the 1 million (around $1.2 million)-per-year therapy, with only one patient receiving it commercially since its launch in 2012.

GSK, meanwhile, has priced Strimvelis at a lower rate (around $650,000 a year) to try to encourage takeup, but hasn't given any updates and said last week it may put its rare disease unit up for sale. Rare disease head Carlo Russo moved to Italian biotech Genenta in January.

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Bluebird Bio sees Europe as first market for its gene therapies - FierceBiotech

A University of Chicago-based research team has overcome challenges that have limited gene therapy and demonstrated how their novel approach with skin transplantation could enable a wide range of gene-based therapies to treat many human diseases.

In a study inthe journal Cell Stem Cell, the researchers provide proof-of-concept. They describe gene-therapy administered through skin transplants to treat two related and extremely common human ailments: Type 2 diabetes and obesity.

We resolved some technical hurdles and designed a mouse-to-mouse skin transplantation model in animals with intact immune systems, said study author Xiaoyang Wu, assistant professor in the Ben May Department for Cancer Research at the University of Chicago. We think this platform has the potential to lead to safe and durable gene therapy in mice and, we hope, in humans, using selected and modified cells from skin.

Beginning in the 1970s, physicians learned how to harvest skin stem cells from a patient with extensive burn wounds, grow them in the laboratory, then apply the lab-grown tissue to close and protect a patients wounds. This approach is now standard. However, the application of skin transplants is better developed in humans than in mice.

The mouse system is less mature, Wu said. It took us a few years to optimize our 3-D skin organoid culture system.

This study is the first to show that an engineered skin graft can survive long term in wild-type mice with intact immune systems. We have a better than 80 percent success rate with skin transplantation, Wu said. This is exciting for us.

The researchers focused on diabetes because it is a common non-skin disease that can be treated by the strategic delivery of specific proteins.

They inserted the gene for glucagon-like peptide 1 (GLP1), a hormone that stimulates the pancreas to secrete insulin. This extra insulin removes excessive glucose from the bloodstream, preventing the complications of diabetes. GLP1 can also delay gastric emptying and reduce appetite.

Using CRISPR, a tool for precise genetic engineering, they modified the GLP1 gene. They inserted one mutation, designed to extend the hormones half-life in the blood stream, and fused the modified gene to an antibody fragment so that it would circulate in the blood stream longer. They also attached an inducible promoter, which enabled them to turn on the gene to make more GLP1, as needed, by exposing it to the antibiotic doxycycline. Then they inserted the gene into skin cells and grew those cells in culture.

When these cultured cells were exposed to an air/liquid interface in the laboratory, they stratified, generating what the authors referred to as a multi-layered, skin-like organoid. Next, they grafted this lab-grown gene-altered skin onto mice with intact immune systems. There was no significant rejection of the transplanted skin grafts.

When the mice ate food containing minute amounts of doxycycline, they released dose-dependent levels of GLP1 into the blood. This promptly increased blood-insulin levels and reduced blood-glucose levels.

When the researchers fed normal or gene-altered mice a high-fat diet, both groups rapidly gained weight. They became obese. When normal and gene-altered mice got the high-fat diet along with varying levels of doxycycline, to induce GLP1 release, the normal mice grew fat and mice expressing GLP1 showed less weight gain.

Expression of GLP1 also lowered glucose levels and reduced insulin resistance.

Together, our data strongly suggest that cutaneous gene therapy with inducible expression of GLP1 can be used for the treatment and prevention of diet-induced obesity and pathologies, the authors wrote.

When they transplanted gene-altered human cells to mice with a limited immune system, they saw the same effect. These results, the authors wrote, suggest that cutaneous gene therapy for GLP1 secretion could be practical and clinically relevant.

This approach, combining precise genome editing in vitro with effective application of engineered cells in vivo, could provide significant benefits for the treatment of many human diseases, the authors note.

We think this can provide a long-term safe option for the treatment of many diseases, Wu said. It could be used to deliver therapeutic proteins, replacing missing proteins for people with a genetic defect, such as hemophilia. Or it could function as a metabolic sink, removing various toxins.

Skin progenitor cells have several unique advantages that are a perfect fit for gene therapy. Human skin is the largest and most accessible organ in the body. It is easy to monitor. Transplanted skin can be quickly removed if necessary. Skins cells rapidly proliferate in culture and can be easily transplanted. The procedure is safe, minimally invasive and inexpensive.

There is also a need. More than 100 million U.S. adults have either diabetes (30.3 million) or prediabetes (84.1 million), according the Centers for Disease Control and Prevention. More than two out of three adults are overweight. More than one out of three are considered obese.

Additional authors of the study were Japing Yue, Queen Gou, and Cynthia Li from the University of Chicago and Barton Wicksteed from the University of Illinois at Chicago. The National Institutes of Health, the American Cancer Society and the V Foundation funded the study.

Article originally appeared on Science Life.

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Gene therapy via skin could treat diseases such as obesity - UChicago News

BioMarin Pharmaceutical on Monday dedicated its new Novato manufacturing facility which is expected to be key in its continuing clinical trials on a drug the company believes will potentially genetically repair the cause of hemophilia A.

Before a crowd of 300 to 400 people, the company, which manufactures drugs mostly for rare diseases, called its new production location the largest gene-therapy-manufacturing facility in the world. The project was completed 11 months ahead of schedule, employing 300 people in 200,000 construction hours, according to Robert Baffi, the firms executive vice president, Technical Operations.

Jean-Jacques Bienaim, chairman and CEO of BioMarin, said the drug to be produced at the location, BMN 270 gene therapy for hemophilia A, has the potential to change what future doctors learn about hemophilia.

Because of a genetic flaw, the blood of those who have hemophilia does not clot. The mutation takes places in a single gene that provides instructions to make a protein called Factor VIII, which is essential for blood to clot normally.

According to the company, the drug thus far in investigational clinical trials has shown the ability to genetically correct the problem and allow patients to manufacture and maintain a constant level of Factor VIII. Production of the drug to be used in those continuing trials will begin as soon as possible in Novato.

Among those affected by the hemophilia is the son of Christine Orr a speaker at todays event. Genetic roulette resulted in an older son being born without the problem.

But soon after her younger son was born, it became apparent he had little or no clotting factor. Every other day, home infusions of clotting factor have helped curb the problem, but she said her son experienced the stigma of parents being afraid to invite him to birthday parties or play dates over what might happen if he were to be hurt.

She said a one-shot treatment to potentially genetically treat and cure the disease gives her hope that yes, a cure is on my horizon, and he can choose his path in life and not have hemophilia choose it for him.

On Aug. 2, BioMarin Pharmaceutical reported it reaped $317 million in second-quarter revenue, up 6 percent from the same quarter in 2016.

It operated a loss of $37 million for the second quarter, but far less than the $419 million loss in the same quarter last year. The last quarters losses amounted to 21 cents per diluted share.

BioMarin, which has six main drugs on the market, had two huge contributors to second-quarter revenue: Kuvan, with $102 million, and Vimizim, with $103 million.

Kuvan, sapropterin dihydrochloride, treats a genetic disorder called phenylketonuria. BioMarin bought global rights to Kuvan in 2015 from Merck for 340 million euros, about $405 million. PKU is rare, and causes amino acid phenylalanine to build up in the body. The buildup of the amino acid can cause grave health problems.

Vimizim treats patients with mucopolysaccharidosis type IV-A, also called Morquio A syndrome, which is a metabolic disorder that inhibits the bodys ability to process certain mucopolysaccharides. It is usually inherited.

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BioMarin Pharmaceutical launches gene therapy drug plant in Novato - North Bay Business Journal

SAN DIEGO, Aug. 8, 2017 /PRNewswire/ --AVACEN Medical (AVACEN) announced partnering with Morulaa HealthTech of Chennai, India to introduce its FDA-cleared AVACEN 100, Class II medical device, in India for treating the joint pain associated with Arthritis which is currently being used in the USA.

According to the World Health Organization, Arthritis affects approximately 200 million people in India. This prevalence is higher than many well-known diseases such as diabetes and cancer. This has a devastating impact on those who suffer from the arthritis and their personal support systems. In addition, arthritis imposes a large economic burden on public and private healthcare providers.

AVACEN recently received CE (Conformit Europenne) Mark approval to market the AVACEN 100 to the European Union's 28 member countries for treating the joint pain associated with Arthritis. AVACEN believes this is the first time a medical device has been approved for treating whole-body arthritis pain.

Currently used in the USA the AVACEN 100 uses an entirely new concept to treat Arthritis pain. It noninvasively and safely infuses heat into the circulatory system forcing the body to dilate peripheral capillaries to radiate off this excess heat.

AVACEN Medical CEO Thomas Muehlbauer described the AVACEN 100 as "the only medical device on the market today able to provide non-invasive, whole-body treatment, using a single point of contact. It is the ideal drug-free and safe alternative for rapid relief of arthritis pain from the inside". Muehlbauer added, "With approximately 2 million safe treatments, we frequently hear reports dramatic results such as being able to return to playing piano, gardening, and chopping vegetables. Even gnarled hand joints have been reported to disappear."

About AVACEN 100 Patents

The US Patent Office has issued 4 Patents to AVACEN which include apparatus claims directed to features of the Heat Therapy apparatus manufactured by AVACEN and method claims directed to specific methods of use, referred to by the company as the AVACEN Treatment Method. The patents also cover innovations embodied in the AVACEN 100 system, which is expected to allow multiple therapeutic uses to alleviate symptoms associated with a circulatory, neurological, lymphatic, or endocrinal dysfunction, or any combination thereof.

Patents have also been issued for China, Australia, Japan, UK, France, Germany, Spain, Sweden and Canada. Patents are pending in India and Hong Kong.

AboutAVACEN Medical

AVACEN Medical is dedicated to the innovation and design of safe, easy to use, noninvasive drug-free alternatives for wound-healing and the management of pain associated with numerous chronic and acute conditions including the temporary relief of minor muscle and joint pain and stiffness associated with arthritis and potentially other conditions that can cause joint pain, such as CRPS, Reynaud's, and Lyme Disease. Founded in 2009, AVACEN Medical is headquartered in San Diego. Contact: Ryan Jeffcoat at (888) 428-2236 x 711 or info@AVACEN.com.

About Morulaa HealthTech

Established by a family with over 100 years of experience in business, Morulaa HealthTech provides turnkey solutions for manufacturers and distributors in the Medical Device sectors. The unique Morulaa model for Product Registration and Distribution, uses in-house regulatory and marketing teams working in-sync with clients to commercialize healthcare products across India and South East Asia. Morulaa HealthTech currently has clients from America, Europe, Asia and Oceania. For more information: http://www.morulaa.com

IMPORTANT NOTES: The AVACEN 100 is not for sale in the U.S. or E.U. for any non-cleared or non-approved indication mentioned in this document.

E.U. CE-Approval: A heat therapy system indicated for the temporary relief of minormuscle and joint pain and stiffness; the temporary relief of joint pain associated witharthritis, muscle spasms, minor strains and sprains;the temporary relief of widespreadpain associated with fibromyalgia; muscular relaxation; andthe temporary increase ofmicrocirculation.

U.S. FDA-Clearance: A heat therapy system indicated for the temporary relief of minor muscle and joint pain and stiffness; the temporary relief of joint pain associated with arthritis; muscle spasms; minor strains and sprains; muscular relaxation; and the temporary increase of local circulation where applied.

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AVACEN Medical Introduces New Concept for Treating Arthritis Pain to India - PR Newswire (press release)

Joint pain and stiffness are common complaints doctors hear from patients. Personal health advocate Carrie Bloemers says, arthritis is a common problem that can lead to joint swelling and pain. Its really uncomfortable and its something that people experience and live with daily.

There are natural things patients can do to minimize the pain without taking medication. The foods we eat can increase the inflammation levels in our body so therefore, if we are able to follow an anti-inflammatory diet with antioxidants we can help control some of the symptoms of arthritis and inflammation, said Bloemers.

Antioxidants lower levels of inflammation throughout the body. The goal is to minimize the overall symptoms so the aches and the pains from arthritis so they may have a lower dependency on some of the medications they are taking, said Bloemers.

Foods high in antioxidants like blueberries, grapes, and greens, can help with joint inflammation. But processed foods and foods in high fat and sugar can keep your joints inflamed. Not only do we need our blues and our purples, we need red, yellow, orange, and green nutrients so plant based materials every day too, said Bloemers.

A regular healthy diet can help with day to day pain and swelling. Eating blueberries one time isnt going to make your knee feel better. Its your habits of including a healthy diet high in antioxidants, low in inflammatory foods, like high fatty processed meats or high sugar foods that over time are going to help lower the inflammation in your body, said Bloemers.

In addition to fruits and vegetables, health experts also recommend patients maintain an active lifestyle to prevent joint stiffness.

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Health Matters: Nutrition for Inflammation and Arthritis - NBC2 News

Scientists from IIT Guwahati have synthesised mats made of silk-proteins and bioactive glass fibres that they believe can assist the growth of bone cells and repair worn-out joints in arthritis patients.

The disease most commonly affects joints in the knees, hips, hands, feet, and spine and is marked by the breakdown of joint cartilage and underlying bones. Left untreated, it can cause severe pain, swelling, and eventually limited range of movement.

Current clinical treatment methods are limited by lack of viable tissue substitutes to aid the repair process, Biman B. Mandal from institute said.

Joint disease

To develop a suitable tissue substitute, scientists, including those from the University College London in the U.K., looked into the natural bone-cartilage interface and tried to mimic it synthetically in lab conditions.

Knee osteoarthritis is the most common bone and joint disease in India. However, Mr. Mandal pointed out that the available clinical grafts were expensive.

Enhances healing

We used silk, a natural protein to fabricate electrospun mats to mimic the cartilage portion and bioactive glass to develop a composite material, similar to the natural tissue, said Mr. Mandal.

For the mat, scientists used a kind of silk easily available in northeast India.

Muga [Assam] silk is endowed with properties that enhance the healing process, Mr. Mandal said.

The researchers adopted a green fabrication approach for the developing the silk composite mats electrospinning. It is similar to knitting, except that it utilises electric high-voltage force to draw ultrafine fibres, Mr. Mandal said.

A layer by layer approach was followed, where the bone layer was first formed, on top of which the cartilage layer was developed. The resulting composite mat resembled the architecture of the bone-cartilage interface.

To assist the regenerative process, the mats would be grafted in the defected joint with cells harvested from the patient.

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Silk mats that could treat arthritis - The Hindu

A middle-aged tiger has been put down by vets after the endangered predator suffered for years with ARTHRITIS.

Indah, the 12-year-old Sumatran tiger, was a favourite among visitors to Howletts Wild Animal Park with her tiger boyfriend Amir.

But sadly, she was being treated by vets and a leading orthopaedic surgeon but in recent weeks was suffering increasing pain which could not be controlled and had to be put down.

The average Sumatran tiger lives to the age of 24, meaning poor Indah died in the middle of her life, leaving her mate Amir all alone.

A park spokesman said: Following numerous evaluations and discussions, our animal director, vet team and keepers made the difficult decision to euthanise Indah.

This was the most necessary and humane course of action.

Indah was a very special animal, born in 2005 at Dudley Zoo.

Tigress Indah who has had to be put down at Howletts Wild Animal Park.

She arrived at Howletts Wild Animal Park as part of a breeding programme in 2006.

She had a lovely character and was a favourite with visitors and staff alike.

She had a very special bond with Amir our male Sumatran tiger.

The pair would always enjoy a good head rub in the morning and they enjoyed nothing more than sitting out on the high platform in their enclosure, soaking up the sunshine.

We are sure that our visitors and supporters will be as saddened by this news as we are.

Visitors to the park were quick to pay tribute to Indah.

Ellie Hadlington said: My heart goes out to her keepers, who are no doubt distraught.

I commend the keepers and the vet team for the amazing work they have done with her and the other animals.

Tracy Butler added: I met this beautiful tiger when i had time feeding the tigers and gave her meds in piece of meat, such a beautiful creature and a great loss, well done to Howletts for everything they done for her.

Jackie Smith said: Such sad news. So glad I got to see her a few weeks ago. Such a beautiful tiger. Thoughts are with her keepers.

The Sumatran tiger has been listed as critically endangered since 2008, as the population was estimated at 441 to 679.

More worrying is the staggering decline in independent, investigative journalism. It costs a lot to produce, so many publications facing an uncertain future can no longer fund it.

With nobody to hold the rich and powerful to account, or report on the issues that don't fit with the mainstream 'narrative', your help is needed.

You can help support free, independent journalism for as little as 50p. Every penny we collect from donations supports vital investigative journalism.

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HeartbreakingSumatran tiger had to be put down because she had arthritis - The London Economic

Rheumatoid arthritis (RA) isn't the same as grandma's stiff joints (aka osteoarthritis). It's an autoimmune disease that causes a persons immune system to attack their joints by mistake, causing pain and swellingand it most often shows up between the ages of 30 and 50, per the American College of Rheumatology.

RA isnt super common among all cases of arthritisabout 1.3 million people are affected by it, according to the American College of Rheumatology. But for those who do have it, it can be debilitating. Heres what you need to know.

We dont really know the cause, even to date, Orrin Troum, M.D., a rheumatologist at Providence Saint John's Health Center in Santa Monica, California, tells SELF. What we do know is that it's an autoimmune disease and that there is evidence that it runs in families. According to Dr. Troum, there are some factors that have been linked to RA, including smoking cigarettes, poor dental hygiene, and genetics.

Your genes dont actually cause you to develop rheumatoid arthritis, but they can make you more susceptible to environmental factors, like bacterial or viral infections, that could potentially trigger the disease, according to the Mayo Clinic. In other words, having a genetic predisposition doesn't mean that you'll get RA; rather, it means you inherited genetic variations from your parents that could make it harder for your body to fend off infection (which can then contribute to the development of a disease).

People tend to associate the word arthritis with osteoarthritis, which causes joint inflammation that typically comes from the wear and tear of age. But rheumatoid arthritis is an immune condition that impacts the lining of a person's joints. In some patients, RA can impact the skin (via noduleslumps that form beneath the skinand rashes), cause inflammation in the lungs and whites of the eyes, lead to plaque buildup in the heart and an increased risk of heart attack and stroke, and damage the blood vessels, according to the Arthritis Foundation.

Rheumatoid arthritis starts in a persons joints, but it can be felt all over. Symptoms typically include joint pain, tenderness, swelling, or stiffness (especially in the morning), according to the Arthritis Foundation. People can also experience fatigue, loss of appetite, and a low-grade fever, the organization says.

While you have pain in your joints, your whole body [feels] sick, Marcy OKoon, senior director of consumer health at the Arthritis Foundation, tells SELF. People just feel ill. They have fatigue because they dont sleep well due to the pain. The first symptoms tend to happen in the joints of the hands and feet and then spread to other areas of the body, Reddog Sina, D.O., assistant professor in the department of family and community medicine at Michigan State University, tells SELF.

Depending on how severe their condition is, people with RA can feel fine one day and terrible the next. These flare-ups can be unpredictable and debilitating, the Arthritis Foundation says. Some flare-ups have known triggers, like overexerting yourself and having swollen joints the next day, but others have no known cause.

About 75 percent of patients in the U.S. with RA are women, according to the American College of Rheumatology, and 1 to 3 percent of women may get RA in their lifetime. This is true for several other autoimmune conditions like lupus and thyroid disease, Dr. Troum says, and experts arent sure why it happens. Women with RA tend to feel better during pregnancy, only to have a flare-up afterward, O'Koon says. It may have something to do with hormonal differences, but we just dont know, Dr. Troum says.

Since rheumatoid arthritis can get worse with time, early diagnosis is key, Dr. Troum says. But diagnosis can be difficult. Often, patients arent recognized as having rheumatoid arthritis at first and are treated with over-the-counter medications, he says. It's not uncommon for RA patients to originally be misdiagnosed with osteoarthritis, Dr. Troum says.

Once a person receives a diagnosis, verified through a blood test and sometimes an X-ray, theyre often put on disease-modifying antirheumatic drugs (DMARDS) which can stop the progression of the disease and prevent disability, Dr. Troum says. Typically patients stay on the drugs for life, but they dont have a positive effect on everyone. Its very individually dependent. Some people function incredibly well with medication their entire lives, and others dont, Dr. Sina says. Getting good sleep, eating a healthy diet, and trying to decrease stress may also help people manage their RA, Dr. Troum says.

If youre diagnosed with RA, know this: You can still have a good, active life with proper care. Most of my patients lead normal, healthy, productive lives, Dr. Troum says.

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6 Things You Need to Know About Rheumatoid Arthritis - SELF

The Gorilla NC Fusion Plating System is the latest addition to Paragon 28's robust foot and ankle specific portfolio. The system is made up offour plates, left and right specific, ranging from 28 35.5 mm in length to span the Naviculocuneiform joint. The plates providefive different screw options for compression across multiple joints. The patent-pending NC Plate with PRECISION Guide technology allows for a crossing screw that passes from the medial cuneiform to the lateral aspect of the navicular. A locking screw in the plate allows for fixation between the proximal medial navicular to the intermediate cuneiform. A second locking screw in the navicular helps guard against plantar gapping, while two distal screws in the medial cuneiform have the ability to be placed across one, two or three cuneiforms. All plates are anatomically contoured for optimal fixation and low profile to minimize prominence and avoid soft tissue irritation.

The system also includes templates to facilitate proper plate placement and screw alignment across each joint. The patent-pending NC Fusion PRECISION Guide provides three options for the crossing screw trajectory to account for variations among patient anatomy and can accommodate a 3.5 mm, 4.0 mm, 4.5 mm or 5.5 mm cannulated cross screw. Compression can be achieved by use of a partially threaded cross screw or with external compression using a fully threaded cross screw.

Key Features and Benefits of the System:

Guided Screw Targeting simplifies screw placement and helps to ensure fixation is achieved in highest quality boneAnatomic Plate Contouring cylindrical curvature minimizes the need for interoperative manipulation of plate to conform to anatomyTemplating and Trialing System ensures plates and screws are prepositioned to achieve best fit and intended compressionCompression through and Outside the Plate maximizes distribution of compression across fusion sites

Paragon 28 is grateful for the significant contributions Dr. Byron Hutchinson, DPM, made as the surgeon designer of this system.

Product Page:http://www.paragon28.com/products/ncfusionplatingsystem

CONTACT:Jim Edson, Director of Product Management and Marketing, jedson@paragon28.com

View original content with multimedia:http://www.prnewswire.com/news-releases/paragon-28-launches-plating-and-guided-screw-system-to-address-naviculocuneiform-arthritis-and-flatfoot-gorilla-nc-fusion-plating-system-300501564.html

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Paragon 28 Launches Plating and Guided Screw System to Address Naviculocuneiform Arthritis and Flatfoot- Gorilla ... - PR Newswire (press release)

(Reuters) - The benefits of Johnson & Johnson's experimental rheumatoid arthritis drug sirukumab do not outweigh the risks, an advisory panel to the U.S. Food and Drug Administration concluded on Wednesday.

The panel voted 12-1 that the drug should not be approved, citing safety concerns, including an imbalance in the number of deaths in patients taking sirukumab compared with those taking a placebo. The most common causes of death were major heart problems, infection and malignancies.

"The safety is not there," said Dr. Beth Jonas, interim head of the division of rheumatology at the University of North Carolina School of Medicine.

The FDA is not obliged to act on the recommendation of its advisory panels but typically does so.

J&J originally developed the drug with GlaxoSmithKline Plc. GSK recently said it would return all rights to J&J. GSK held rights to the drug in North, Central and South America.

Sirukumab blocks a cytokine in the body known as interleukin 6 (IL-6) that can contribute to the inflammation associated with rheumatoid arthritis, an autoimmune disorder that affects more than 1.3 million.

Panelists said they were especially reluctant to recommend approval of sirukumab because there are two other drugs on the market in the same class. These are Roche Holding AG's Actemra and Sanofi SA and Regeneron Pharmaceuticals Inc.'s

"If this was a new agent I would probably be a little more enthusiastic," said Dr. Maria Suarez-Almazor, rheumatology section chief at the University of Texas MD Anderson Cancer Center. "There is no reason to think that this new drug will act in a tremendously different way."

The FDA, in briefing documents released on Monday, noted that the trend toward increased overall mortality seemed unique to the sirukumab program.

Reporting by Toni Clarke in Washington; Editing by Jonathan Oatis and Steve Orlofsky

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FDA panel votes against approval of J&J arthritis drug - Reuters

Scientists from IIT Guwahati have synthesised mats made of silk-proteins and bioactive glass fibres that they believe can assist the growth of bone cells and repair worn-out joints in arthritis patients.

The disease most commonly affects joints in the knees, hips, hands, feet, and spine and is marked by the breakdown of joint cartilage and underlying bones. Left untreated, it can cause severe pain, swelling, and eventually limited range of movement.

Current clinical treatment methods are limited by lack of viable tissue substitutes to aid the repair process, Biman B. Mandal from institute said.

Joint disease

To develop a suitable tissue substitute, scientists, including those from the University College London in the U.K., looked into the natural bone-cartilage interface and tried to mimic it synthetically in lab conditions.

Knee osteoarthritis is the most common bone and joint disease in India. However, Mr. Mandal pointed out that the available clinical grafts were expensive.

Enhances healing

We used silk, a natural protein to fabricate electrospun mats to mimic the cartilage portion and bioactive glass to develop a composite material, similar to the natural tissue, said Mr. Mandal.

For the mat, scientists used a kind of silk easily available in northeast India.

Muga [Assam] silk is endowed with properties that enhance the healing process, Mr. Mandal said.

The researchers adopted a green fabrication approach for the developing the silk composite mats electrospinning. It is similar to knitting, except that it utilises electric high-voltage force to draw ultrafine fibres, Mr. Mandal said.

A layer by layer approach was followed, where the bone layer was first formed, on top of which the cartilage layer was developed. The resulting composite mat resembled the architecture of the bone-cartilage interface.

To assist the regenerative process, the mats would be grafted in the defected joint with cells harvested from the patient.

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Silk mats can help treat arthritis - The Hindu

Klotho, in ancient Greek mythology, is one of the Fates controlling the span of human destiny tasked as she was with cutting the string that determined the length of a persons life. Klotho is also a naturally occurring hormone in the body. More than two decades ago, Japanese researchers discovered that this hormone plays a role in aging. People with more klotho in their body, tend to live longer and to retain more of their facultiesthat is to stay sharpwell into old age.

There are many elderly people who walk around and have completely normal, sharp vibrant brain function despite the fact that they are much older and the fact that they have a lot of disease toxins in the brain, Dr. Dena Dubal a neurobiologist at the University of California, San Franciscos Memory and Aging Center told Popular Science.

Dubal is the author on a study released today in the journal Cell Reports that looks at what happens to brain function when you inject klotho protein into mice.

Aging researchers like Dubal arent interested in extending life for its own sakethey dont have a Methuselah complexbut rather because aging is the biggest factor for disease. If they can find a way to slow aging, or at least its effects, they might find a way to allow us to avoid the cognitive declines, and diseases such as stroke, heart disease, and diabetes that tend to come as we flip through more pages on the calendar.

A few years ago, we discovered in collaboration with several people that in human populations those people that had higher levels of klotho had better cognitive functions in normal aging, says Dual.

Which is great if you happen to be genetically blessed with higher levels of klotho, less great if youre not.

Earlier studies have looked at the impacts of klotho on longevity and health, including brain health, but they tended to rely on genetically modified mice. The genes of these mice had been modified to allow them to produce more klotho or less.

Those studies found that mice modified to produce more klotho lived longer, which is promising. But despite recent advances, genetically modifying humans is still an ethical minefield away in the future, especially if the modifications are being made to improve wellbeing as opposed to preventing disease. And, genetically altering embryos might help future populations, but it doesnt do much for those of us already living. So, Dubal wanted to see is if mice injected with klothoacting essentially a hormonal supplementwould show cognitive improvements.

The answer, seems to be yes.

To get the result, Dubal and her colleagues injected three types of mice with a portion of the protein. They injected young mice, aged mice, and mice genetically altered to have brains similar to that which we would see in Alzheimers or Parkinsons patients in humans.

Within hours they showed better cognitive function, says Dubal.

Since you cant exactly administer a mouse an IQ test, they assessed brain power based on the mices ability to navigate a series of water mazes, in an experiment that sounds on par with human a trip to Wisconsins famed waterslide park, The Dells.

They found that mice that had daily injections and were better able to navigate the maze (as measured by the distance traveled to find a hidden platform) than their control group peers. In a classic example of work smarter, not harder, the klotho mice were just much more efficient seekers.

We tested them two weeks later in a different cognitive test and they were still smarter, says Dubal, which suggested that getting the klotho protein into their bodies combined with brain training and stimulation had a long-lasting effect in their brain. Because the half-life of the protein is only seven and a half hours long, any of the protein should have been long out of their system.

There are a few caveats.

First, this was an experiment in micenot in humans. While its incredibly promising, the study results are short term and they werent looking for side effects. The pace at which a promising scientific study is turned into a supplement of dubious efficacy is stunning these days, so please dont subject yourself to some back-alley klotho injection. Between resveratrol and superfoods weve been there before.

At the same time, we know that klotho levels can be affected not only by genetics - but also by stress. If you want to help your body keep its klotho levels at your own peak you might want to try proven stress reduction techniques like exercise and chilling out about your own impending mortality.

Finally, the researchers arent sure how klotho seems to be generating this effect because the protein is too big to pass from the body into the brain. For a long time, weve studied the brain in isolation from the body with the brain mostly telling the body what to do, and the body acting like a series of censors that give the brain useful data. But this study like our growing understanding of the connection between our microbiome and brain health, and exercise and brain health, this study touches on our growing understanding of how the body connects to the brain to help make the brain more resilient.

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An extra dose of this longevity hormone helped make mice smarter - Popular Science

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Monday, August 07, 2017

EDITORIAL

Tom Brady - is his brain immune from injury?

It has been nearly eight years since Malcolm Gladwell wrote his piece in The New Yorker about the long-term tragic impact of football on the brain of those that play the game. It outlines the post-football life, There were men with aching knees and backs and hands, from all those years of playing football. But their real problem was with their heads, the one part of their body that got hit over and over again.

Gladwell's article, Offensive Play How different are dogfighting and football? is one of a growing number of looks at the depressing post-football life of the players - viewed by many as American heroes. He wrote it 2009.

My Life Sucks

Now in 2017, Jim Plunkett, who was a Super Bowl hero back in the day and now 69, told the San Jose Mercury News, My life sucks. Plunkett's body is broken and he is in constant pain. Once a man like Tom Brady - overcoming obstacles and realizing NFL quarterback hero status -- he is now devastated physically and his wife says in the same interview that Plunketts mind is being ravaged by the years of head injuries.

Math 99% of NFL

In July, a paper in the JAMA found that Chronic Traumatic Encephalopathy, known as CTE, was found in 99% of deceased NFL players' brains those that were donated for scientific research. And, CTE was found in three of the 14 high school players and 48 of the 53 college players whose brains were tested. CTE is directly linked tomemory loss, confusion, impaired judgment, aggression, depression, anxiety, impulse control issues, and suicidal behavior. A slew of former NFL players have committed suicide in the past few years and their brains were found to have CTE.

Beautiful Brady

While so many love the amazing story of Bradys greatest of all-time title and marvel at his longevity built of his dedication to physical performance, luck, genetics, and kale salads, the reality is none of those things can protect his brain from the impact of years of missed blocks or blindside corner blitzes.

The collective failure of those missed blocks and defensive scheming of opponents may not have stopped the Patriots from being the great team in NFL history, but they have taken their toll on jarring the brain of Brady no less than any other player. The reality is that the longer he plays, the more his brain is impacted.

His longevity is now working against his future the statistics prove it. The list of NFL players who were found to have CTE is long and depressingly catastrophic. The Patriots alone can claim Junior Seau, Most Tatupu, and Kevin Turner -- all tested after death and all suffered from CTE. Seau killed himself at age 43, Tatupu was 54 at the time of his death, and Turner died of ALS believed to be linked to CTE at the age of 46.

Deviant Behavior

Gladwell ends the article with a passage from Dogmen and Dogfights, an academic analysis of deviant behavior by Rhonda Evans and Craig Forsyth about those involved with dogfighting.

The authors write:

When one views a staged dog fight between pit bulls for the first time, the most macabre aspect of the event is that the only sounds you hear from these dogs are those of crunching bones and cartilage. The dogs rip and tear at each other; their blood, urine and saliva splatter the sides of the pit and clothes of the handlers. . . . The emotions of the dogs are conspicuous, but not so striking, even to themselves, are the passions of the owners of the dogs. Whether they hug a winner or in the rare case, destroy a dying loser, whether they walk away from the carcass or lay crying over it, their fondness for these fighters is manifest."

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EDITORIAL: 99%, My Life Sucks, Dogfighting, and Brady's Brain - GoLocalProv

This press release was orginally distributed by SBWire

Guildford, UK -- (SBWIRE) -- 08/08/2017 -- When it comes to eye health and vision, the needs of every individual differ. As we are subjected to an increasing girth of information regarding sight correction procedures and vision health, researching choices and options can be overwhelming. With the advancements in technology, ground breaking treatments such as LASIK, LASEK, 3rd Generation ReLEx SMILE and Lens Replacement can transform eyesight overnight thereby reducing the need to rely on optical aids like glasses or contact lenses.

Attending Optegra's informative Open Evening on August 9, 2017 (6 PM) affords all Londoners an ideal opportunity to learn about powerful lens replacement techniques. Ideal for those in the Capital aged 50+ yearning for perfect sight, the event will help provide answers to any and all vision correction concerns and queries.

The North London event will be held at Optegra's state of the art hospital conveniently located at 6 The Technology Park Colindeep Lane, London, NW9 6BX.The evening will be led by Optegra's Consultant Mr. Mark Hulbert along with Optegra's Eye Health Care team. Mr. Hulbert is a specialist in general ophthalmology and has a particular interest in glaucoma and neuro-opthalmology.

The informal structure of the North London event encourages attendees to clarify doubts and discuss any unfounded fears they may have regarding vision correction. The open evening also provides participants an opportunity to learn about past patient experiences and get an understanding about Optegra's clinical outcomes and outstanding success rate standards. Due to the high popularity of the event, prior booking is encouraged to attend the North London Open Evening.

To find out why Optegra is UK's most trusted eye health care provider recommended by GPs & optometrists, its wide range of treatments and money saving offer visit Optegra.com or call 0808 273 6059 Mon-Fri 8am-8pm, Sat 9am-4pm.

For media enquiries contact Tukshad Engineer Head of Digital at Optegra - 0755 499 4155 | tukshad.engineer@optegra.com.

About Optegra Eye Health CareOptegra Eye Health Care is a specialist provider of ophthalmic services in the UK, China, Czech Republic, Poland and Germany. Optegra operates nationwide eye hospitals and clinics in the UK and brings together leading edge research and medical expertise, state-of-the-art surgical equipment and top ophthalmic surgeons renowned for their areas of expertise to offer excellent clinical outcomes in laser eye surgery, lens replacement (Clarivu), cataract removal, AMD, vitreoretinal and oculoplastic procedures all carried out in five-star patient facilities.

Optegra Eye Health Care operates seven dedicated eye hospitals based in Birmingham, Central London, Hampshire, Manchester, North London, Surrey and Yorkshire. For more information please visit Optegra.com.

Contact:Tukshad Engineer, Head of Digital at OptegraCompany: Optegra Eye Health CarePhone: 0755 499 4155Address: The Surrey Research Park, 10 Alan Turing Rd, Guildford GU2 7YF, United KingdomEmail: tukshad.engineer@optegra.comWebsite:http://optegraeyehealthcare.com/

For more information on this press release visit: http://www.sbwire.com/press-releases/have-questions-about-an-eye-condition-or-vision-problems-attend-optegras-informative-open-evening-in-north-london-on-august-9-2017-844882.htm

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Have Questions About an Eye Condition or Vision Problems? Attend Optegra's Informative Open Evening in North ... - Digital Journal

"Any attempt to treat veterans with traumatic brain injuries begins with an accurate diagnosis," says Randall Benson, M.D., CNS Vice President and Medical Director. "Combat injuries are frequently mis-diagnosed resulting in inappropriate and unsuccessful treatment. We can and must do better. Better diagnoses lead to improved outcomes."

Hope After Combat combines diagnostic testing including neurology, neuro-opthalmology and neuropsychology enhanced by CNS' advanced magnetic resonance imaging techniques (MRI), diffusion tensor imaging and susceptibility-weighted imaging. These advanced MRI techniques developed at CNS are significantly more sensitive than traditional MRI imaging and have worked successfully on hundreds of non-combat brain-injury victims.

In the Hope After Combat study, once diagnostic testing is complete a treatment plan is developed and the veteran takes part in an individualized 60-day rehabilitation program based on his or her specific diagnosis. All disciplines continue to provide input and care for the victim and his/her family. The final study is expected to include more than 50 combat victims.

"These veterans and their families are desperate for answers and we're trying to provide them," says John D. Russell, CEO and President of CNS. "The veterans have been real troopers in every sense of the word and we're learning from their individual efforts and progress. But our donors are the ones who've made the study possible. Without them we couldn't bring all these disciplines together for such an important study."

For further information or to participate in or make a contribution to the Hope After Combat study, contact John Russell at 313-228-0930 or visit the CNS website, http://neurologicstudies.com

CNS was founded in 2011 with the objective of advancing scientific research for neurovascular disease. CNS staff are available for interview on brain injuries and brain-related diseases.

View original content:http://www.prnewswire.com/news-releases/military-brain-injuries-are-the-subject-of-center-for-neurological-studies-hope-after-combat-study-300499425.html

SOURCE Center for Neurological Studies

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Military Brain Injuries are the Subject of Center for Neurological Studies "Hope After Combat" Study - PR Newswire (press release)

Mobilizing the forces of the immune system to the fight has changed the game of cancer treatment in recent years.

The key to success has been first identifying how cancer cells disarm the T-cell fighters of the immune system and then finding drugs to restore their firepower.

Using drugs called checkpoint inhibitors, patients with incurable cancers like advanced melanoma have shown long-term responses.

Forty percent of melanoma patients will still fail to respond to the treatment, however, which means that cancer cells must have other means ones that are not addressed by checkpoint inhibitors to disable the immune systems weaponry.

To discover what they are, a research team led by Nicholas Restifo at the US National Cancer Institute began with human melanoma cells growing in a dish, and systematically disabled every gene in the melanoma cells using the CRISPR gene-editing technique.

They then tested the ability of the T-cells fighters to recognize each one. It turned out about 100 different genes activated by the cancer were able to prevent the attack by the T-cells.

Of particular interest was a gene called APLNR. While it has been implicated as contributing to some cancers, this was the first evidence that it played a role in disarming T-cells.

If we can truly understand mechanisms of resistance to immunotherapy, we might be able to develop new therapeutics, comments Restifo.

The research is published in Nature.

Link:
The cancer genes that disarm the immune system | Cosmos - Cosmos

After decades with little progress, cancer researchers have made major breakthroughs in the last few years in treatingmelanoma(the most serious type of skin cancer). The key to these advances?Immunotherapy is helping patients live longer.

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Chemotherapy which is typically a first-line treatment targets cancers directly. But immunotherapy spurs your immune system to attack cancer cells. This approach can offer new hope if you have melanoma.

Researcher and plastic surgeon Brian Gastman, MD, works with the latest advances in using immunotherapy to treat melanoma. He explains how they work.

One of the newest options is injectinga modified form of the herpes virus directly into the tumor, Dr. Gastman says.

This modified virus is called talimogene laherparepvec, or T-VEC.Once doctors inject it, the virus replicates within the cancer cells and causes them to rupture and die.

Doctors use T-VEC to treat melanoma at the skin level, under the skin or in deep soft tissues, he says.

One of the main reasons we give T-VEC is for whats called in-transit metastases, Dr. Gastman says.

He explains, Lets say someone had a melanoma in their foot. Typically, it would spread from the foot to the lymph nodes and from the lymph nodes all over the body.

But sometimes cancer cells get caught between the original site and the lymph nodes and cause little satellite lesions, he says. Those are in-transit metastases.

If its on the arm or leg, you can inject the arm or leg with chemotherapy and it will kill the cancer, but it will also damage the arm or leg, Dr. Gastman says.

T-VEC offers several advantages over chemotherapy in this situation:

Doctors still use ipilimumab, the first immunotherapy drug used to disrupt the growth and spread of cancer, for high-risk patients, but mostly as a help to other treatments. Its sometimes used alongside or after the initial treatment, Dr. Gastman says.

In this case, patients receive a high dose of ipilimumab after surgery to help prevent the cancer from coming back. Its significant because it is helping patients live longer, he says.

This is the first time that an adjuvant (helper) therapy showed an improvement in survival rates over a placebo, he says.

T-VEC and the new use for ipilimumab are the two major advances the FDA approved in recent years. Theres also a lot of exciting research happening, too, Dr. Gastman says.

Now, another immunotherapy drug called Pembrolizumab is being used a lot, he says. In 2016, there was data showing that 40 percent of stage IV melanoma patients treated only with pembrolizumab were alive in three years. Thats very exciting news.

Theres also ongoing research that finds promise in a treatment that combines ipilimumab with another immunotherapy drug, nivolumab.

The study, which focuses on patients with advanced melanoma who received this combination, reported a two-year overall survival rate of 64 percent. Early resultsshow that using the two drugs together is more effective than using either drug alone.

Dr. Gastman also notes that outcomes are improving as doctors learn more about how to prevent or better manage side effects from these drugs. The side effects, which are sometimes significant, may include:

Were much better now than we were five or six years ago at handling the toxic side effects, he says.

Immunotherapies wont completely replace other treatments like chemotherapy and surgery. But, they offer new and effective options for patients with melanoma. And ongoing research continues to make advances, Dr. Gastman says.

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These New Skin Cancer Treatments Unleash Your Immune System ... - Health Essentials from Cleveland Clinic (blog)