Ex-drug executive Shkreli congratulates Australian students

Former pharmaceutical executive Martin Shkreli has congratulated a group of Australian students who reproduced the active ingredient for a life-saving, anti-parasitic drug at the center of a drug-price controversy involving his former company.

The students from Sydney Grammar School drew global media attention this week after they said they had produced the drug Daraprim for about $2 a dose, a fraction of the current list price of $750 per dose.

Shkreli is a former chief executive of Turing Pharmaceuticals, where he sparked outrage among patients and U.S. lawmakers for raising the price of Daraprim by more than 5,000 percent.

“These Australian students are proof that the 21st century economy will solve problems of human suffering through science and technology,” Shkreli said in a video message posted on YouTube.

“We should congratulate these students for their interest in chemistry and I’ll be excited about what is to come in this STEM-focused 21st century,” he said, referring to science, technology, engineering and mathematics.

Daraprim is used to fight parasitic infections in AIDS patients, pregnant women and others.

The six students and their science teacher worked under the guidance of the University of Sydney and members of the Open Source Malaria consortium, which allows scientists to share research on anti-malaria drugs.

“We had to repeat a lot of the reactions and try different reaction conditions in order to see which materials in which things would react to make the Daraprim,” student Brandon Lee told Reuters Television on Friday.

Turing made front-page headlines after it bought the rights to Daraprim in August 2015. With no rival manufacturers making the drug, Turing quickly raised the price for a tablet of Daraprim to $750 from $13.50.

Overnight, the tiny company was vilified as an example of pharmaceutical industry greed, drawing fire from politicians and medical groups. Turing later said it would cut the cost of the drug to hospitals.

Lee said the students wanted to show that “these compounds which you think are only accessible to these large, large-scale companies are actually able to be accessed and produced by ordinary citizens”.

Shkreli stepped down as Turing’s chief executive in December 2015 after being indicted on charges that he engaged in a Ponzi-like scheme at a hedge fund and Retrophin Inc, a company he once headed. He has pleaded not guilty.

(Reporting by Reuters Television; Writing by Darren Schuettler; Editing by Robert Birsel)

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Jardiance Wins CV Prevention Indication

WASHINGTON — The diabetes drug empagliflozin (Jardiance) may be marketed for prevention of cardiovascular death in patients with type 2 diabetes and co-existing cardiovascular disease, the FDA said Friday.

It’s the first such claim ever allowed for a diabetes drug.

Empagliflozin, first approved in 2014, is an inhibitor of the sodium-glucose co-transporter 2 (SGLT2) pathway, reducing blood glucose by causing it to be excreted in urine.

Its benefit for cardiovascular risk reduction was demonstrated in the so-called EMPA-REG trial, results of which were reported in 2015.

But whether the FDA would approve the indication was unclear, after an advisory committee delivered a split vote in June. Members opposing the claim pointed to problems with the trial that included interim unblinding, protocol changes, and irregularities in some of the data.

In announcing the approval, the agency said the “availability of anti-diabetes therapies that can help people live longer by reducing the risk of cardiovascular death is an important advance for adults with type 2 diabetes.”

The announcement also noted that the drug “is not intended for patients with type 1 diabetes mellitus or for the treatment of diabetic ketoacidosis.” It also recited the list of contraindications and adverse effects already noted on the drug’s label.

Since the EMPA-REG results were released, trials of two other diabetes drugs — liraglutide (Victoza) and the investigational drug semaglutide — also have demonstrated a cardiovascular benefit.


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Stop the flu before it starts

Flu season is back, which means it’s time to protect yourself and loved ones by getting a free flu shot.

Flu viruses change from year to year, so it’s important to get a flu shot each flu season. It’s free for people with Medicare, once per flu season when you get it by doctors or other health care providers (like senior centers and pharmacies) that take Medicare.

National Influenza Vaccination Week is December 4–10. You can stop the flu before it stops you.

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Study reveals new way to improve stability of common protein drugs

Gaining access to important biopharmaceuticals needed to treat illnesses and autoimmune diseases is one of the biggest obstacles developing countries face. Costs can be astronomical where these medications are needed most, and when doctors are able to acquire those medications they face another challenge – time. Drugs are perishable and some require refrigeration, which can be difficult to provide in the world’s poorest regions.

Now, a new study appearing in the Proceedings of the National Academy of Sciences reveals a new way to improve the stability of common protein drugs and extend shelf life. Complex structures and poor chemical stability of biopharmaceuticals such as proteins, peptides and antibodies used to treat various illnesses and autoimmune diseases including rheumatoid arthritis, diabetes, multiple sclerosis and lymphoma can, over time, render drugs ineffective – the medicine simply becomes inactive.

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The study, led by Matthew Webber, assistant professor in the Department of Chemical and Biomolecular Engineering in the College of Engineering at the University of Notre Dame, tested a novel route for non-covalent protein modification of insulin and glucagon, both used in treating diabetes, as well as an antibody drug used in treating leukemia, lymphoma and autoimmune disease. The results demonstrated significant stability for this new additive.

“Our molecules interact with protein drugs reversibly, but when they are bound, they provide a protective shell,” said Webber. “This keeps the proteins from aggregating. This shell also protects the proteins from sticking to the wall of their storage vial, a major reason protein drugs become denatured and inactive.”

Stability of insulin when formulated with the new additive increased from approximately 14 hours to over 100 days while maintaining complete activity even under stressed conditions. “We are simulating stressed environmental conditions for storage,” Webber said. “So by keeping these drugs at elevated temperatures with agitation, we provide maximal stress to the formulation to understand the stabilizing effects of our additive on protein drugs.”

The glucagon samples, which typically lose stability in solution in under an hour, remained soluble for at least 24 hours. The therapeutic antibody also maintained its activity when stressed.

“We have reason to believe this additive would be effective in many biopharmaceuticals beyond those evaluated in our research,” Webber said. However, Webber notes that “regulatory approval and financial support would be needed to for mass distribution and use – a process that could take several years or more.”


University of Notre Dame

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New data on risk vs benefit for potent CAR-T cancer drugs

By Deena Beasley

A promising but risky new group of customized cancer drugs will be in focus this weekend at the annual meeting of the American Society of Hematology (ASH), where clinical trial results will help clarify their potential for doctors and investors.

Experimental chimeric antigen receptor T-cells, or CAR-Ts, are made by genetically altering a patients’ own T-cells in the lab to help the immune system find and kill cancer cells. The altered cells are then infused back into the patient.

Early excitement over the drugs has propelled investor interest in biotech Kite Pharma Inc, whose shares have tripled since a 2014 initial public offering, as well as rival Juno Therapeutics Inc, whose therapy JCAR015 has generated concerns after five leukemia patients died due to severe brain swelling. Juno shares now trade about 14 percent below their IPO price.

“Juno has dug themselves into such a deep hole,” said Brad Loncar, manager of the Loncar Cancer Immunotherapy ETF. “My guess is that they may drop the JCAR015 program.” He will be watching closely to see whether data at ASH on another Juno candidate, JCAR017, shows similar issues.

Data on CAR-T drugs from Kite and Novartis AG will also be presented at the meeting.

In early trials, CAR-Ts eliminated all trace of leukemia and lymphoma in 40 percent to 90 percent of patients who had run out of other options. But it is not yet clear how long those remissions will last. Also, the drugs can overestimate the immune system, which can cause dangerous side effects.

If they work safely, Wall Street expects annual sales for CAR-T therapies in the billions of dollars.

Shares of Bluebird Bio Inc closed 14 percent higher on Thursday after a small study showed that its CAR-T, bb2121, induced remission in several patients with advanced multiple myeloma with no worrisome side effects. The Bluebird drug is being developed in partnership with Celgene Inc.

“The data look good, but these are very small trials,” said Les Funtleyder, healthcare portfolio manager at E Squared Asset Management in New York, which does not currently hold positions in CAR-T companies. “We would definitely like to see larger sample sizes and longer duration.” He is also looking for clarity on pricing. Estimates have run into hundreds of thousands of dollars per treatment. It is not yet clear if patients will need more than one treatment or whether that can even be done safely.

The CAR-T technique is being tried initially against blood cancers, which contain specific proteins that can be differentiated from normal tissue, limiting a possible immune system attack on healthy organs. Drugs like Juno’s JCAR015 target a protein called CD19, which is found on the surface of a type of white blood cell.

Juno is slated to present early-stage data at the ASH meeting in San Diego on JCAR017, which also targets CD19.

Juno Chief Executive Hans Bishop said JCAR017, and the similar JCAR014, are safer than JCAR015 because of a manufacturing process that allows for active control of the composition of the T-cells that make up the final product, said

“We think when you get to the extremes … that is likely a source of variability,” he said.

Novartis plans to file next year for U.S. Food and Drug Administration approval for its drug, CTL019, in children with acute lymphoblastic leukemia who have exhausted other options, based on Phase 2 trial results to be released at ASH on Saturday.

The study results show “what it looks like when you roll this out to a bunch of different centers across the world,” said Dr Stephan Grupp, research director at Children’s Hospital of Philadelphia’s childhood cancer center and the study’s lead investigator.

He said most patients in the Novartis trial experienced a serious side effect known as cytokine release syndrome, or CRS, as well as neurological toxicity, including confusion and seizures, but all responded to treatment for those side effects and none had severe brain swelling.

Kite is expected to report further details from a Phase 2 trial of patients with an aggressive type of lymphoma whose cancer returned despite treatment with chemotherapy or a bone marrow transplant. Interim results showed that a month after treatment 47 percent of patients achieved complete remission, but that rate dropped to 33 percent after three months. The company, which said it will begin a rolling FDA application, expects to have data with six months of follow-up in the first quarter of next year.

“These are the first results from a large multi-center trial,” Jeff Wiezorek, head of clinical development at Kite, told Reuters. “The safety and efficacy look similar to what we saw in smaller trials.”

(Corrects to Juno candidate JCAR017 instead of Kite, paragraph 4)

(Reporting By Deena Beasley; Editing by David Gregorio)

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Cornell researchers develop chemical probe activated by UV light to control inflammation

Black light does more than make posters glow. Cornell researchers have developed a chemical tool to control inflammation that is activated by ultraviolet (UV) light.

The method will allow scientists to study inflammation and the immune system, and may one day prove effective as a targeted therapy for inflammatory diseases, while minimizing side effects to healthy tissues.

The researchers, who reported their results in a study published in October in the journal Chemical Science, designed a small molecule that is capable of controlling an immune response when exposed to UV light radiation.

“Currently, there aren’t a lot of tools that are able to manipulate the immune system in a spatio-temporal fashion,” said Pamela Chang, assistant professor of microbiology and immunology, and the paper’s senior author. Bibudha Parasar, a graduate student in Chang’s lab, is the paper’s first author.

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“We are pushing the forefront of developing new technologies to control inflammation and the immune system, with the ultimate goal of being able to study these biological pathways and perhaps develop therapies for inflammatory diseases,” Chang said.

Inflammation is caused by the immune system as one of the body’s responses to eliminate infectious pathogens and other foreign or dangerous agents. When inflammation becomes chronic, it can lead to a host of diseases, including asthma, multiple sclerosis, rheumatoid arthritis, colitis and Crohn’s disease.

The researchers created a chemical probe that inhibits a reaction mediated by enzymes called histone deacetylases (HDACs). These enzymes regulate genes that turn on when the immune system is challenged and promote inflammation. HDACs also have inhibitors that suppress this inflammatory response, and the probe specifically activates these inhibitors but only in the presence of UV light. This is especially useful since HDACs are ubiquitous and have other biological effects, and most drugs affect the entire system, leading to unintended consequences.

“If you turned off all the HDACs in the body, you would probably be hitting a lot of pathways that you didn’t want to turn off,” said Chang. “We can control when and where we turn off the HDACs using light. The idea is that you can actually target the tissue that has chronic inflammation and regulate it by selectively inhibiting HDACs in the tissue that’s affected.”

Photodynamic therapies are being developed and have existing infrastructure in the clinic for the potential use of the new tool to inhibit inflammation in patients with inflammatory diseases, Chang said.


Cornell University

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'Patients at risk' from length of GP consultations

‘Patients at risk’ from length of GP consultations

  • 29 August 2016
  • From the section Health
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GP handing out prescriptionImage copyright
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Patients are being put at risk by GPs being forced to carry out complex consultations in 10 minutes or under, the British Medical Association says.

The doctors’ union said patient care was undermined when GPs were forced to see as many as 60 patients a day.

It called for more funding to allow GPs to spend at least 15 minutes with each patient.

NHS England said consultation lengths were up to doctors and there were no national limits suggesting 10 minutes.

However, the NHS Choices website does state that “GPs spend an average of 8-10 minutes with each patient” and advises patients to “plan ahead to make sure you cover everything you want to discuss”.

The BMA, which published a report on “safe working in general practice” earlier this month, called for a reorganisation and warned that GPs faced “unsustainable pressure” from increasing workload and staff shortages.

The report said it was intended to “stimulate discussion” and put forward a model that could be used across the UK.

‘Unsafe levels’

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Dr Nicola Hulme, a GP in Cheshire, said she found NHS England’s comment about there being no national limit on appointment times “insulting”.

“With the high levels of demand, we have to run 10-minute appointments,” she said.

“To offer longer with the same number of appointments would extend our day to beyond the 12 hours we currently routinely work.

“I often run late so I can deal thoroughly with my patients’ issues.

“Paperwork gets started routinely after 7pm. I rarely get home before 8pm, having started generally at 7.30am.

“Nobody goes into medicine for an easy ride, we are all hard workers, but the intensity and the demand are now at unsafe levels.”

Dr Ali Alibhai, who works in central London, said GPs now had to manage so many chronic diseases such as diabetes that a consultation as short as eight minutes was “not appropriate any more for safe patient care”.

Dr Brian Balmer, of the BMA’s GPs’ committee, said that, in an ageing population, many patients had complex multiple conditions that needed longer to treat.

He warned that many GPs were being forced to truncate care and deliver an “unsafe number of consultations”.

‘New approach’

He said consultations should be limited to 25 a day, about the same number recommended in many other EU countries.

Dr Balmer added: “We need a new approach that shakes up the way patients get their care from their local GP practice.

“The consultation time needs to increase to 15 minutes with the government providing on its promised funding to make this work.”

Health think tank the King’s Fund said its analysis showed GPs were under “huge pressure due to an increase in workload which has not been matched by a growth in funding or the workforce”.

It also found the average length of consultations had increased due to the rising number of older patients with more complex conditions.

King’s Fund chief executive Chris Ham said general practice needed “new investment to stop it falling apart”, but GPs must also embrace new methods such as giving more advice by phone and email.

NHS England said the length of appointments was “at the discretion of individual GP practices, based on patient need, and there are no national limits suggesting 10 minutes should be the norm”.

It accepted that GPs were “under pressure”, and said it was “substantially increasing investment and reforming care to free up GPs to spend more time with patients”.

In 2013, a rule saying GP appointment slots in England must be for a minimum of 10 minutes was scrapped.

‘I would be dead’

One patient, John White, from Somerset, said: “I think those who get a 10-minute consultation with their GPs are lucky.

“At my doctors’ surgery I cannot even book an appointment to see my GP. All we are offered is to book a ‘telephone triage consultation’ where your doctor will call you by telephone and decide whether a face-to-face appointment is warranted.

“I have had to wait in excess of two weeks for the telephone appointment.”

Another patient, Maggy Jackson, said: “I have a brilliant GP who sees me for as long as I need. He always runs on time too.

“All I can say is if it wasn’t for this practice I would be dead. They diagnosed a gangrenous hernia two years ago when I presented with horrendous pain and no appointment. I was given a full examination, morphine and was in hospital an hour later after their intervention.”

Nurse Liz Wright said appointment length was also an issue in nursing.

“In years gone by, I had either 15 or 20-minute appointments to see my patients,” she said.

“This has now been cut to 10 minutes, in which to carry out triage, cervical smears, dressings, injections, contraceptive discussions and issuing of prescriptions and a whole range of ailments.

“This means that in a typical 7.5-hour shift, I see up to 42 patients, well above the recommended daily amount.

“In my opinion this is unsafe practice and I have decided to take early retirement.”

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Do Your Part for the Environment—Go Paperless!

On June 5, more than 100 countries worldwide will be celebrating World Environment Day—a day for encouraging awareness and action for the environment. How can you do your part to help the environment?  One great way is to sign up to get your “Medicare & You” handbook electronically.

If you have an eReader (like an iPad, Surface, or Kindle Fire) you can download a free digital version of the “Medicare & You” handbook to your eReader and take it with you anywhere you go.

Don’t have an eReader? You can still sign up to get a paperless version in a few simple steps. We’ll send you an email in September when the new eHandbook is available. The email will explain that instead of getting a paper copy in your mailbox each October, you’ll get an email linking you to the online version of “Medicare & You.” This online version of the handbook contains all the same information as the printed version.

Even better, the handbook information on Medicare.gov is updated regularly, so you can be confident that you have the most up-to-date Medicare information!

Make a difference this World Environment Day – Go paperless, and sign up to get your “Medicare & You” information electronically.

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'Concerning' variation in birth care

Birth care variation in hospitals in England ‘concerning’

  • 23 March 2016
  • From the section Health

A midwife talking to a pregnant womanImage copyright

The variation in care women get when giving birth in hospital in England is concerning, experts say.

The Royal College of Obstetricians and Gynaecologists’ warning follows a review of more than 550,000 births.

It found “substantial variation” in practice between maternity units, and said this may suggest not all women get the best possible care.

Ministers said the NHS was a safe place to give birth but the report would help it improve.

The review – carried out with the London School of Hygiene and Tropical Medicine – looked at official data on births in 2013-14, excluding non-standard deliveries such as twins, triplets and pre-term babies.

It highlighted different rates of emergency caesarian sections, instrumental deliveries and episiotomies, which is when the area between the vagina and anus is cut.

In some maternity units 8% of mothers needed emergency c-sections, but in others the figure was 15%.

Just under one in five first-time mothers needed help with instruments while giving birth naturally in some units – but in other units this figure rose to almost three in 10.

The number of women who needed an episiotomy ranged from 29% to 44%.

‘Postcode lottery’

RCOG president Dr David Richmond said: “We are concerned about the amount of variation identified in this report.

“Although the exact causes are difficult to establish, it is paramount that maternity units have information about their services, as well as the ability to compare themselves to the national average and to their peers.”

To help with that, the college has collated the results on an interactive website.

Elizabeth Duff of the National Childbirth Trust said: “Pregnant women should not have to endure a postcode lottery, and it is unacceptable these variations in maternity care are reported year after year.

“We urge NHS trusts to use this data to examine their practices and ensure the best possible outcomes for mothers and babies.”

Health minister Ben Gummer said: “Mothers and their babies deserve the very highest standards of care regardless of where they live.

“The NHS is already a safe place to give birth and by being open and honest about variations in care, RCOG’s report will help the NHS to improve.”

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Scientists solve atomic structure of ubiquitin ligase complex that plays key role in protein degradation

Scientists at Sanford Burnham Prebys Medical Discovery Institute (SBP) have solved the atomic structure of a unique ubiquitin ligase complex. Ubiquitin is best known for its role in protein degradation, but more recently seen as important for cell signaling, DNA repair, anti-inflammatory, and immune responses.

The study, published today in Nature, opens the door for developing a novel class of drug targets for cancer as well as inflammatory diseases such as rheumatoid arthritis, Crohn’s disease and psoriasis.

“Our new research revealing the fully active structure of an RBR E3 ligase holds significant therapeutic potential in oncology and immunology,” said Bernhard Lechtenberg, Ph.D., postdoctoral fellow at SBP and lead author of the study. “The three-dimensional structure we present provides detailed insights into the molecular architecture of the complex and allows us to draw conclusions about how it works.”

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Ubiquitin is a small protein that helps regulate the function of other proteins in the body. E3 ligases carry out a process known as ubiquitination, where ubiquitin is attached to a substrate protein, changing the way it functions.

There are three classes of E3 ligases, but members of the RBR type have most recently emerged as a novel and relatively untapped class of targets for drug discovery because of their role in modulating the immune system.

“We were surprised to find how the active form of the E3 ligase we analyzed, called HOIP, attaches ubiquitin in a markedly different way—an elongated fashion—compared to the other types of E3 ligases,” said Stefan Riedl, Ph.D., associate professor in SBP’s NCI-designated Cancer Center and senior author of the paper. “This may be key to its role in activating the NF-kB pathway, a signaling process that is well established as a regulator of cell survival and death, and helps coordinate the immune system.

“NF-kB is the master regulator of inflammation inside cells, and its activation is believed to promote cancer development by inhibiting cell death and promoting inflammation. This study removes a significant technical barrier that has prevented exploiting RBR E3 ligases as a drug target for cancer and inflammatory disorders.

“Our next step is to continue to work very closely with our biology and immunology collaborators to more fully understand the regulation of RBR E3 ligases,” added Riedl.


Sanford Burnham Prebys Medical Discovery Institute

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