FDA Approves First Gene Therapy for Cancer Treatment in U.S.

Dennis Thompson wrote . . . . . . .

The U.S. Food and Drug Administration broke new ground in cancer care Wednesday by approving the first gene therapy for patients in the United States.

Kymriah (tisagenlecleucel) genetically tweaks a patient’s own immune system cells into what scientists call “a living drug” to battle a form of acute lymphoblastic leukemia (ALL).

The immunotherapy now can be used in children and young adults with B-cell ALL that will not respond to other therapies, the FDA announced.

Wednesday’s announcement will “change the face of modern medicine and drug development,” FDA Commissioner Dr. Scott Gottlieb said at a news briefing.

“Gene therapy products are now being studied in many diseases and conditions, including genetic disorders, autoimmune diseases, heart disease, cancer, diabetes and HIV/AIDS,” Gottlieb added.

ALL is a cancer of the bone marrow and blood in which the body makes abnormal lymphocytes, a type of white blood cell. According to the U.S. National Cancer Institute, about 3,100 patients aged 20 and younger are diagnosed with ALL each year.

About 15 percent to 20 percent of patients with B-cell ALL have cancer that either did not respond to treatment or has recurred, the FDA says. It’s these patients for whom Kymriah is intended.

In the treatment, doctors collect the patient’s own T-cells — one of the immune system’s main cell types — and genetically reprogram them to target and attack leukemia cells. They are then reintroduced back into the patient to do battle against the tumor.

Dr. Kenneth Anderson is president of the American Society of Hematology, which focuses on blood cancers. In a statement, he said that Wednesday’s approval “marks an important shift in the blood cancer treatment paradigm. We now have proof that it is possible to eradicate cancer by harnessing the power of a patient’s own immune system. This is a potentially curative therapy in patients whose leukemia is unresponsive to other treatments.”

Dr. Otis Brawley, chief medical officer at the American Cancer Society, agreed.

“This is big. Big,” he said. “I’m going to predict this technology will spread to other diseases over the next few years. It’s a new method of treating cancer that I think we will successfully further exploit over the next decade.”

However, Anderson stressed that “this approval only pertains to a small population of children [with ALL].”

“More research is needed to make this therapy more effective for a broader population, to reduce the severe side effects that patients experience during treatment, and ultimately to find a broader application beyond blood cancers,” he said.

The FDA based its decision on a clinical trial involving 63 patients with B-cell ALL. After three months of treatment, 83 percent of the patients remained cancer-free.

Gottlieb said the FDA approval was expedited, coming just seven months after the agency received the initial application.

However, Kymriah comes with the potential for severe side effects. The worst is cytokine release syndrome, a common immunotherapy complication that causes potentially life-threatening fever and flu-like symptoms. The therapy also can cause neurological events, serious infections, low blood pressure and acute kidney injury.

Because of these safety concerns, the FDA will require that hospitals receive special certification to use Kymriah, the agency said.

And then there’s the issue of cost. Kymriah’s maker, Novartis, hasn’t provided a price for the drug. But speaking earlier this year to The New York Times, drug industry analysts estimated that individualized therapies could cost more than $300,000.

Other genetic therapies for cancer are also in the research pipeline. In June, a clinical trial in China showed promise in treating another blood cancer, multiple myeloma, using reprogrammed T-cells in much the same way Kymriah does.

According to Gottlieb, the FDA has already granted more than 550 active investigational new drug applications related to gene therapy products, including 76 active investigational new drug applications related to CAR-T cell products like Kymriah.

And while the path to safe and effective gene therapies has been a long one, “Today, a pivotal leg in that journey is complete,” Gottlieb said.

“The science has reached a point of superiority, where enough of the components of these endeavors have worked out,” he explained. “We can deliver effective therapies to patients. We can deliver on the original promise.”

Source: HealthDay

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Apple Sherbet

Ingredients

2/3 cup dry white wine
1/4 cup soft light brown sugar
1 strip of thinly-pared lemon rind
2 tablespoons lemon juice
1 piece of fresh root ginger, peeled, cored and sliced
1 lb cooking apples, peeled, cored and sliced
small angelica or other herb leaves, to decorate

Method

  1. Put the wine, sugar, lemon rind, lemon juice and ginger into a pan and stir over a low heat until the sugar has dissolved. Increase the heat and bring to the boil.
  2. Add the apple slices and poach them for 8-10 minutes, or until they are tender. Remove from the heat and leave to cool.
  3. Discard the lemon rind and ginger and puree the fruit and juice in a blender.
  4. Pour the mixture into a container, cover with foil and freeze for 1 hour.
  5. Turn the mixture into a chilled bowl and beat it to break down the ice crystals.
  6. Return the mixture to the freezer for 3-4 hours, until it is firm.
  7. To serve, transfer the sherbet to the refrigerator for about 30 minutes. Serve it in scoops, decorated with the herb leaves.

Makes 4 servings.

Source: Cooking Naturally

Sweet Burgers

The burgers feature ice cream wrapped daifuku (大福) and 4 different sauces sandwiched between two bun halves.

Red Bean Sauce

Red Bean and Strawberry Sauce

Almond and Mitarashi Sauce

Cheese Sauce

The sweet burgers are available at Lotteria stores in Japan for a limited time at 230 yen each (tax included).

‘Gluten-free water’ Shows Absurdity of Trend in Labeling What’s Absent

Brandon McFadden wrote . . . . . .

The food labeling craze coupled with banner headlines about the dangers of gluten, genetically modified organisms (GMOs) and hormones are leading to increasingly absurd results.

For example, you can now buy “premium” water that’s not only free of GMOs and gluten but certified kosher and organic. Never mind that not a single drop of water anywhere contains either property or is altered in any way by those designations.

While some labels provide useful information that is not readily detectable by consumers, others contain misleading claims that exploit a knowledge gap with consumers and take advantage of their willingness to pay a premium for so-called process labels. For example, details on a product’s country of origin are helpful; labeling a bottle of water “gluten free” and “non-GMO” much less so.

In my experience as a food economist, such “fake transparency” does nothing to inform consumers about the nature of their foods. Moreover, it can actually decrease well-being when accompanied by a higher price tag. A new labeling law set to take effect next year will only make matters worse.

Brief history of food labels

Until the late 1960s, consumers knew very little about the nutritional content of the prepared foods they purchased.

The dramatic growth in processed foods changed this and led to a system of voluntary and mandatory nutrition labeling in the early ‘70s. As we learned more about the relationship between diet and health, Congress sought to provide consumers more information by passing the Nutrition Labeling and Education Act of 1990, which gave the Food and Drug Administration (FDA) the authority to require companies to list certain nutrients and other details on food packages.

Since then, food labeling has only gotten wilder. Some labels, such as “organic,” follow strict federal guidelines, while others aren’t regulated, such as “natural.” Eggs might come from chickens that are “cage-free” (which isn’t regulated) or “free range” (which is), while your milk could come from cows that are “grass-fed” (no standard) or “hormone-free” (requires verification).

These labels are largely the result of the consumer desire to know more about the way food is produced – and the willingness to pay more for the claims, spurious or not.

Characteristics of a product

To understand how all this labeling drives consumer behavior, let’s turn to economics.

The economist Kevin Lancaster hypothesized that consumers derive happiness not from a product they might buy but from its characteristics.

For example, when purchasing a car, it’s the characteristics – color, brand, size, price or fuel efficiency – that make you want to buy it. Browsing online even allows us to refine searches by these characteristics. Some of these characteristics, such as size and color, are visible and verifiable to they eye before purchase, while others, like a car’s fuel efficiency, can’t be confirmed until you sign on the dotted line and collect the keys.

In other words, the company knows more about the car than you do, something economists call asymmetric information. Economist George Akerlof won a Nobel Prize for his work on asymmetric information and how it leads to terrible market outcomes.

Similarly, food has characteristics that can be observed only after purchase. You can pick up an apple and see whether it has any blemishes, but you don’t really know how it will taste, and you cannot know how many calories it has even after consumption. That’s where food labels can help.

Exploiting the knowledge gap

Unfortunately, the problem of asymmetric information can never be eliminated entirely, and consumers may never have as much knowledge as they’d like when making purchases.

Mandated labeling has helped narrow this gap, particularly when the additional information increases consumer well-being, such as knowledge that a food contains 160 calories or 60 percent of the recommended daily does of vitamin C.

Some companies, however, use food labels to exploit this knowledge gap by preying on consumer concerns about a certain ingredient or process in order to collect a premium or increase market share. One of the ways they do this is by providing fake transparency through so-called absence labels (like “does not contain”), which are increasingly found on products that could not possibly have the ingredient in the first place.

While the water example I mentioned earlier is the most clear-cut illustration of this, others only require a bit more knowledge to see that they don’t serve a purpose. Since federal regulation requires that hormones not be used in pork or poultry, advertising a chicken breast as “hormone-free” doesn’t make sense – yet doing so allows a company to charge more or help its products stand out from the less-labeled competition.

The FDA allows a business to use the phrase as long as the label also notes that “federal regulations prohibit the use of hormones.”

Signaling safety

A new law that makes GMO labeling of some foods mandatory will likely compound these problems once it takes effect in the summer of 2018.

To understand why, let’s return to asymmetric information and a related economic theory called the signaling effect. A signaling effect occurs when a buyer receives an implicit message from an explicit cue. For example, a food labeled “low sodium” may implicitly communicate that salt should be avoided. When the government is involved in the signaling effect, such as when a label is mandatory, the impact tends to become stronger.

Thus the new GMO labeling law is bound to signal to consumers that bioengineered foods are somehow bad. While some countries have banned the use of GMOs, such as in Europe, the FDA has said that “credible evidence has demonstrated that foods from the GE plant varieties marketed to date are as safe as comparable, non-GE foods.”

As a result of the new law, companies selling products without GMOs will likely slap “GMO free” on the label even though the law doesn’t apply to those foods.

My worry is that consumers will become ever more mystified as more businesses make increasingly absurd claims on their labels so that their products stand out from the competition in the grocery store aisle. I expect that the only thing consumers will get in return for these “fake transparency” labels is a higher price tag.

Source: The Conversation

Low-fat Diet Does Not Reduce Risk of Premature Death from Cardiovascular Diseases

Sheryl Ubelacker wrote . . . . . . .

A large Canadian study is challenging conventional wisdom that says a low-fat diet is optimal for cardiovascular health and reduces the risk of premature death.

The McMaster University study of more than 135,000 people in 18 countries found that eating a moderate amount of all types of fat is linked to a reduced risk of early mortality compared to the much-touted low-fat diet — while consuming a high-carbohydrate diet is associated with an increased risk of dying early.

“Contrary to popular belief, increased consumption of dietary fats is associated with a lower risk of death,” said lead author Mahshid Dehghan, a nutrition epidemiologist at the Hamilton university’s Population Health and Research Institute.

“Those with a high-fat intake, about 30 per cent of energy intake, had a 23 per cent lower risk of mortality and an 18 per cent lower risk of stroke, compared to the low-intake group, which had 11 per cent energy from fat,” Dehghan said from Barcelona, where she presented the findings Tuesday to the European Society of Cardiology Congress.

“The association with lower mortality was also seen with all major types of fat, by which I mean saturated, monounsaturated and polyunsaturated fatty acids.”

Saturated fat is found in meat and dairy products, while monounsaturated fat is contained in nuts, avocados, and vegetable and olive oils. Polyunsaturated fat is found in walnuts, sunflower and flax seeds, fish, corn, soybean and safflower oils.

Current global guidelines recommend that 50 to 65 per cent of daily calories come from carbohydrates, and less than 10 per cent from saturated fats. But Dehghan said that advice is mostly based on evidence from studies in North America and Europe.

Cardiovascular disease is a global epidemic, with 80 per cent of the burden of disease in low- and middle-income countries. Diet is a key modifiable risk factor for cardiovascular disease, experts say.

Dehghan said the healthiest diet would be made up of 50 to 55 per cent carbohydrates and 35 per cent total fat, including both saturated and unsaturated types.

“We found no evidence that below 10 per cent of energy from saturated fat is beneficial — and going below seven per cent is even harmful,” she said, adding that a diet containing 10 to 13 per cent of energy from saturated fat was found to be beneficial.

A diet that provides more than 60 per cent of energy from carbohydrates — one common among populations in China and South Asia — was associated with a 28 per cent higher risk of premature death, researchers found.

“The message of our study is moderation as opposed to very low or very high intake in consumption of both fats and carbohydrates.”

“We’re not advocating an extreme diet,” agreed co-author Andrew Mente. “We’re not saying that people should go on a low-carb, very high-fat diet because we didn’t find any benefit with a very low-carb diet either.

“There’s a sweet spot for carbohydrates, which is about 55 per cent of energy intake.”

The PURE (Prospective Urban Rural Epidemiology) study was published Tuesday in The Lancet. In a linked commentary in the journal, Drs. Christopher Ramsden and Anthony Domenichiello of the U.S. National Institute on Aging called the research “an impressive undertaking that will contribute to public health for years to come.”

“The relationships between diet, cardiovascular disease and death are topics of major public health importance…. Initial PURE findings challenge conventional diet-disease tenets that are largely based on observational associations in European and North American populations, adding to the uncertainty about what constitutes a healthy diet. This uncertainty is likely to prevail until well-designed randomized controlled trials are done.”

Mente, also a nutrition epidemiologist at the Population Health and Research Institute, was lead author of a second analysis from the PURE study presented Tuesday at the cardiology meeting.

That paper — one of three from PURE published in The Lancet — found that eating three to four servings of fruit, vegetables and legumes per day reduces the risk of premature death.

“And consuming higher amounts, pretty much you have the same level of risk,” Mente said from Barcelona. “There’s no added benefit with consuming more than four servings.

“This is important because existing guidelines recommend that people consume at least five servings per day, which is less affordable in the poorer countries because fruits and vegetables — particularly fruits — are more expensive as a proportion of people’s incomes.”

Lower-income Canadians may also be unable to afford the five to 10 daily servings of fruits and vegetables recommended in the country’s Food Guide.

“So what our study shows is you can achieve maximum benefit through fruits and vegetables and legumes, and it’s also affordable at the same time.”

Mente said the study also showed raw vegetables appear to confer greater health benefits than those that are cooked because of a loss of nutrients from being exposed to heat.

With the federal government in the process of revamping Canada’s Food Guide, the research could be a timely addition to consultations on what Canadians should be eating, Mente suggested.

“We would hope that independent thinkers perhaps reconsider the guidelines and look at our data, and perhaps rather than putting limits on total fat and saturated fat, perhaps we should be putting limits on the amount of carbohydrates that people consume.”

Source: Winnipeg Free Press


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