Learn the Terms and Acronyms of Myeloma


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Learn the Terms and Acronyms of Myeloma
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Guide to Terms and Acronyms

Everytime you come across a term you do not understand, check the glossary.

Guide to Drug Names
What do carfilzomib and PR-171 have in common? They are the same drug! Between the original name, the generic name and the brand name, it is hard to keep myeloma drugs straight. Here's a handy guide put together by IMF Medical Editor Debbie Birns.

VAD, VAMP, DT-PACE...Is your head swimming from all the acronyms being thrown around? We are here to help.

AskDrDurie: What is remission?

Good info - I am out 6 years, with a few strange after effects.

Excellent news on the leukemia front...


Since the condition seems rather individualised, therapy should be too...

Modified T-cells in the freezer at the Clinical Cell and Vaccine Production Facility at the University of Pennsylvania. Credit Peggy Peterson Photography/Penn Medicine
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An experimental therapy has brought prolonged remissions to a high proportion of patients who were facing death from advanced leukemia after standard treatments had failed, researchers are reporting.
The therapy involves genetically programming cells from the patient’s own immune system to fight the disease.
The research included 30 patients: five adults ages 26 to 60, and 25 children and young adults ages 5 to 22. All were severely ill, with acute lymphoblastic leukemia, and had relapsed several times or had never responded to typical therapies. In more than half, the disease had come back even after a stem-cell transplant, which usually gives patients the best hope of surviving. Their life expectancy was a few months, or in some cases just weeks.
Six months after being treated, 23 of the 30 patients were still alive, and 19 of them have remained in complete remission.
The study, by researchers at the Children’s Hospital of Philadelphia and the Hospital of the University of Pennsylvania, is being published in The New England Journal of Medicine.
Tiny magnetic beads force the larger T-cells, an immune cell, to multiply to produce more cancer-killing cells. Credit Penn Medicine
“We have a number of patients who are a year or more out and are in remission and not requiring other therapies,” said Dr. Stephan A. Grupp, who led the part of the study done at Children’s Hospital of Philadelphia. He said those long remissions gave the researchers hope that the treatment would have lasting effects.
Earlier reports by the same researchers involved only a handful of patients, some with chronic rather than acute leukemia. The scientists say the growing number of patients treated helps demonstrate that the findings are real.
“With the initial patients, we didn’t know if it was just lucky,” said Dr. Carl H. June, the director of translational research at the university’s cancer center. “It turns out it’s reproducible.”
He and Dr. Grupp said that other hospitals around the country would soon test the experimental treatment in children with advanced acute lymphoblastic leukemia.
Similar research, also with encouraging results, is being done at other centers, including the National Cancer Institute and Memorial Sloan Kettering Cancer Center in New York.
Each year in the United States, acute lymphoblastic leukemia affects about 2,400 people older than 20, and 3,600 younger. It has a cure rate in adults of only about 40 percent, compared with 80 percent to 90 percent in children. About 1,170 adults die from the disease each year, compared with 270 people under age 20.
The experimental treatment uses patients’ own T-cells, a type of immune cell. Researchers extract the T-cells and then genetically engineer them, using a disabled virus to slip new genetic material into the cells. The new genetic material reprograms the T-cells to recognize and kill any cell that carries a particular protein on its surface. Then the cells are dripped back into the patient, like a transfusion.
The cells are also programmed to multiply, so that each one can yield as many as 10,000 more cancer-killing cells.
The protein they search for, called CD19, is found on B-cells, which are also part of the immune system. It was chosen as the target because these patients have a type of leukemia that affects B-cells, so the goal is to train the patients’ T-cells to destroy B-cells. Healthy B-cells — which make antibodies to fight infection — are killed along with cancerous ones, but that side effect is treatable.
The treatment clearly does not work for everyone. Seven of the 30 patients died, including a few who had complete remissions at first and then relapsed. In three, the leukemia came roaring back in B-cells that lacked the target protein and therefore were not vulnerable to the treatment.
Even so, Dr. June described the effectiveness of the treatment as “beyond my expectations.”
Of the 19 patients who stayed in remission, 15 did so without any additional treatment. One of them is Emma Whitehead, now 9, who was treated more than two years ago.
Some researchers have thought that, to be on the safe side, any patient who went into remission after T-cell treatment should have a stem cell transplant, because stem cells are considered the standard of care for this type of leukemia. But Dr. June and Dr. Grupp said the long remissions suggested that transplants might not be needed. They hope that eventually the T-cell treatment will be used instead of stem cell transplants, which are risky and arduous.
But the T-cell treatment has its own side effects, in particular a phenomenon known as cytokine release syndrome. It occurs when the T-cells churn out hormones called cytokines that can cause fever, aches, drops in blood pressureand breathing trouble. The more cancer there is to destroy, the worse the syndrome, Dr. June said.
Dawn Carie, from Chesterfield, Mich., said her daughter, Lexie, then 16, had the T-cell treatment a year ago, after multiple types of chemotherapy and a stem-cell transplant had failed. Within hours of receiving the T-cells, Lexie’s temperature shot up to 105 degrees, and she became disoriented and had frightening hallucinations. But when it was all over, she was in remission. For the first time since Lexie was 2, her mother said, tests found no signs whatsoever of leukemic cells.
Because her disease had been so severe, Lexie had two more infusions of T-cells a few months after the first one. She remains in remission.
Lexie is now a senior in high school, busy applying to colleges, Ms. Carie said, adding, “You know, it’s odd to feel normal, because we really never have.”
In July, the Food and Drug Administration designated the T-cell treatment a “breakthrough therapy” for relapsed and treatment-resistant acute lymphoblastic leukemia in adults and children. The designation recognizes experimental drugs “that may demonstrate substantial improvement over existing therapies” for life-threatening conditions, and is meant to speed their development and review.
Currently, the patients’ T-cells are processed at the University of Pennsylvania. But the drug company Novartis, which helped pay for the study, has invested heavily in the research, holds licenses to the technology and is expected to take over the cell processing. Dr. June, Dr. Grupp and some of the other study authors developed the technologies and may profit from them.

Here is the Ebola Czar!



As the Ebola situation in West Africa continues to deteriorate, some U.S. officials are claiming that they would have been able to better deal with the public health threat if only they had more money.
Dr. Francis Collins, who heads the National Institutes of Health (NIH), told The Huffington Post, “Frankly, if we had not gone through our 10-year slide in research support, we probably would have had a vaccine in time for this that would’ve gone through clinical trials and would have been ready.” Hillary Clinton also claimed that funding restrictions were to blame for inability to combat Ebola.
Conservative critics have pointed out that the federal government has spent billions upon billions of dollars on unnecessary programs promoting a political agenda rather than targeting those funds to the fight against health threats.
Other limited government types point to the Progressive utopian foolishness seen in opposing political factions, both sides of which seem to agree humanity could somehow escape calamity if only we had a properly functioning government. People who don’t want an all-powerful government shouldn’t blame it for not having competence when crisis strikes.
What’s particularly interesting about this discussion, then, is that nobody has even discussed the fact that the federal government not ten years ago created and funded a brand new office in the Health and Human Services Department specifically to coordinate preparation for and response to public health threats like Ebola. The woman who heads that office, and reports directly to the HHS secretary, has been mysteriously invisible from the public handling of this threat. And she’s still on the job even though three years ago she was embroiled in a huge scandal of funneling a major stream of funding to a company with ties to a Democratic donor—and away from a company that was developing a treatment now being used on Ebola patients.
Before the media swallow implausible claims of funding problems, perhaps they could be more skeptical of the idea that government is responsible for solving all of humanity’s problems. Barring that, perhaps the media could at least look at the roles that waste, fraud, mismanagement, and general incompetence play in the repeated failures to solve the problems the feds unrealistically claim they will address. In a world where a $12.5 billion slush fund at the Centers for Disease Control and Prevention is used to fight the privatization of liquor stores, perhaps we should complain more about mission creep and Progressive faith in the habitually unrealized magic of increased government funding.

Lay of the Land

Collins’ NIH is part of the Health and Human Services Department. Real spending at that agency has increased nine-fold since 1970 and now tops $900 billion. Oh, if we could all endure such “funding slides,” eh?
Whether or not Dr. Collins’ effort to get more funding for NIH will be successful—if the past is prologue, we’ll throw more money at him—the fact is that Congress passed legislation with billions of dollars in funding specifically to coordinate preparation for public health threats like Ebola not 10 years ago. And yet the results of such funding have been hard to evaluate.
See, in 2004, Congress passed The Project Bioshield Act. The text of that legislation authorized up to $5,593,000,000 in new spending by NIH for the purpose of purchasing vaccines that would be used in the event of a bioterrorist attack. A major part of the plan was to allow stockpiling and distribution of vaccines.
Just two years later, Congress passed the Pandemic and All-Hazards Preparedness Act, which created a new assistant secretary for preparedness and response to oversee medical efforts and called for a National Health Security Strategy. The Act establishedBiomedical Advanced Research and Development Authority as the focal point within HHS for medical efforts to protect the American civilian population against naturally occurring threats to public health. It specifically says this authority was established to give “an integrated, systematic approach to the development and purchase of the necessary vaccines, drugs, therapies, and diagnostic tools for public health medical emergencies.”
Last year, Congress passed the Pandemic and All-Hazards Preparedness Reauthorization Act of 2013 which keep the programs in effect for another five years.
If you look at any of the information about these pieces of legislation or the office and authorities that were created, this brand new expansion of the federal government was sold to us specifically as a means to fight public health threats like Ebola. That was the entire point of why the office and authorities were created.
In fact, when Sen. Bob Casey was asked if he agreed the U.S. needed an Ebola czar, which some legislators are demanding, he responded: “I don’t, because under the bill we have such a person in HHS already.”

The Invisible Dr. Lurie

So, we have an office for public health threat preparedness and response. And one of HHS’ eight assistant secretaries is the assistant secretary for preparedness and response, whose job it is to “lead the nation in preventing, responding to and recovering from the adverse health effects of public health emergencies and disasters, ranging from hurricanes to bioterrorism.”
In the video below, the woman who heads that office, Dr. Nicole Lurie, explains that the responsibilities of her office are “to help our country prepare for, respond to and recover from public health threats.” She says her major priority is to help the country prepare for emergencies and to “have the countermeasures—the medicines or vaccines that people might need to use in a public health emergency. So a large part of my office also is responsible for developing those countermeasures.”
Or, as National Journal rather glowingly puts it, “Lurie’s job is to plan for the unthinkable. A global flu pandemic? She has a plan. A bioterror attack? She’s on it. Massive earthquake? Yep. Her responsibilities as assistant secretary span public health, global health, and homeland security.” A profile of Lurie quoted her as saying, “I have responsibility for getting the nation prepared for public health emergencies—whether naturally occurring disasters or man-made, as well as for helping it respond and recover. It’s a pretty significant undertaking.” Still another refers to her as “the highest-ranking federal official in charge of preparing the nation to face such health crises as earthquakes, hurricanes, terrorist attacks, and pandemic influenza.”
Now, you might be wondering why the person in charge of all this is a name you’re not familiar with. Apart from a discussion of Casey’s comments on how we don’t need an Ebola czar because we already have one, a Google News search for Lurie’s name at the time of writing brings up nothing in the last hour, the last 24 hours, not even the last week! You have to get back to mid-September for a few brief mentions of her name in minor publications. Not a single one of those links is confidence building.
So why has the top official for public health threats been sidelined in the midst of the Ebola crisis? Only the not-known-for-transparency Obama administration knows for sure. But maybe taxpayers and voters should force Congress to do a better job with its oversight rather than get away with the far easier passing of legislation that grants additional funds before finding out what we got for all that money we allocated to this task over the last decade. And then maybe taxpayers should begin to puzzle out whether their really bad return on tax investment dollars is related to some sort of inherent problem with the administrative state.

The Ron Perelman Scandal

There are a few interesting things about the scandal Lurie was embroiled in years ago. You can—and should—read all about it in the Los Angeles Times‘ excellent front-page expose from November 2011, headlined: “Cost, need questioned in $433-million smallpox drug deal: A company controlled by a longtime political donor gets a no-bid contract to supply an experimental remedy for a threat that may not exist.” This Forbespiece is also interesting.
The donor is billionaire Ron Perelman, who was controlling shareholder of Siga. He’s a huge Democratic donor but he also gets Republicans to play for his team, of course. Siga was under scrutiny even back in October 2010 when The Huffington Post reported that it had named labor leader Andy Stern to its board and “compensated him with stock options that would become dramatically more valuable if the company managed to win the contract it sought with HHS—an agency where Stern has deep connections, having helped lead the year-plus fight for health care reform as then head of the Service Employees International Union.”
The award was controversial from almost every angle—including disputes about need, efficacy, and extremely high costs. There were also complaints about awarding a company of its size and structure a small business award as well as the negotiations involved in granting the award. It was so controversial that even Democrats in tight election races were calling for investigations.
Last month, Siga filed for bankruptcy after it was found liable for breaching a licensing contract. The drug it’s been trying to develop, which was projected to have limited utility, has not really panned out—yet the feds have continued to give valuable funds to the company even though the law would permit them to recoup some of their costs or to simply stop any further funding.
The Los Angeles Times revealed that, during the fight over the grant, Lurie wrote to Siga’s chief executive, Dr. Eric A. Rose, to tell him that someone new would be taking over the negotiations with the company. She wrote, “I trust this will be satisfactory to you.” Later she denied that she’d had any contact with Rose regarding the contract, saying such contact would have been inappropriate.
The company that most fought the peculiar sole-source contract award to Siga was Chimerix, which argued that its drug had far more promise than Siga’s. And, in fact, Chimerix’s Brincidofovir is an antiviral medication being developed for treatment of smallpox but also Ebola and adenovirus. In animal trials, it’s shown some success against adenoviruses, smallpox, and herpes—and preliminary tests show some promise against Ebola. On Oct. 6, the FDA authorized its use for some Ebola patients.
It was given to Ebola patient Thomas Eric Duncan, who died, and Ashoka Mukpo, who doctors said had improved. Mukpo even tweeted that he was on the road to recovery.

Back to that Budget

Consider again how The Huffington Post parroted Collins’ claims:
Money, or rather the lack of it, is a big part of the problem. NIH’s purchasing power is down 23 percent from what it was a decade ago, and its budget has remained almost static. In fiscal year 2004, the agency’s budget was $28.03 billion. In FY 2013, it was $29.31 billion—barely a change, even before adjusting for inflation.
Of course, between the fiscal years 2000 and 2004, NIH’s budget jumped a whopping 58 percent. HHS’s 70,000 workers will spend a total of $958 billion this year, or about $7,789 for every U.S. household. A 2012 report on federal spending including the following nuggets about how NIH spends its supposedly tight funds:
  • a $702,558 grant for the study of the impact of televisions and gas generators on villages in Vietnam.
  • $175,587 to the University of Kentucky to study the impact of cocaine on the sex drive of Japanese quail.
  • $55,382 to study hookah smoking in Jordan.
  • $592,527 to study why chimpanzees throw objects.
Last year there were news reports about a $509,840 grant from NIH to pay for a study that will send text messages in “gay lingo” to meth-heads. There are many other shake-your-head examples of misguided spending that are easy to find.
And we’re not even getting into the problems at the CDC or the confusing mixed messages on Ebola from the administration. CDC director Tom Frieden noted:
Indeed. The Progressive belief that a powerful government can stop all calamity is misguided. In the last 10 years we passed multiple pieces of legislation to create funding streams, offices, and management authorities precisely for this moment. That we have nothing to show for it is not good reason to put even more faith in government without learning anything from our repeated mistakes. Responding to the missing Ebola Czar and her office’s corruption by throwing still more money, more management changes, and more bureaucratic complexity in her general direction is madness.

Ad Blames GOP Budget Cuts For Ebola Outbreak

Ad Blames GOP Budget Cuts For Ebola Outbreak

Now, as a partisan I am not afraid of this.  Just a desperate attack ad at the last minute in hopes of suppressing the GOP vote.

As a student of public health, this is evil.  Diverting attention from the public health issue, they are turning it into a political issue.  Political and racial issue.  Centered on funding.

If the CDC has lost any real money in the last 10 years, it is only because Barack Obama has not passed a budget since he entered  office.

So in the future you can "Vote for Democrats, we hate Ebola!" and "GOP is nothing but EBOLA peddlers!"

Sad, sad state of politics.