Tag Archives: clinical trials

The Limits of Science

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A few weeks ago I was cruising the Internet and found an article about how an American man with AIDS was “cured” after he got leukemia, and got a bone marrow transplant from someone who had a rare mutation that made him HIV resistant. After this transplant, not only did the patient (Timothy Ray Brown) no longer have leukemia, but he was also HIV-negative! He underwent countless blood tests and tissue tests over the next year, and none of them could find any trace of the virus, even when he stopped any antiviral treatment. This discovery caused mass excitement in the scientific community. Could a cure finally be close at hand?

HIV: the pesky virus that has been causing so many problems for humanity

HIV: the pesky virus that has just won’t leave

Well, that turned out to be a false alarm because just this Saturday, the virus was detected again in both Timothy Brown and another man who went through the same procedure. It had just not been found earlier because the levels were too low to be detected with current methods of screening.

Dr. Timothy Hendrich, the doctor who had treated both patients, explained that all this hadn’t excitement and disappointment been for nothing; “through this research we have discovered the HIV reservoir is deeper and more persistent than previously known and that our current standards of probing for HIV may not be sufficient to inform us if long-term HIV remission is possible if antiretroviral therapy is stopped.”

This reminds us why science can be so complicated– just a few blogs ago I was talking about how long and tedious clinical trials are and how many steps a drug has to go through before it can be approved. Now I guess it makes sense– it took more than a year to discover that this treatment actually wasn’t a treatment at all. What would have happened if wealthy HIV-positive people had decided to get bone marrow transplants within this time? It would have been unpleasant and somewhat pointless.

What are your thoughts on this event? Are you disappointed, or do you see it as a chance for us to learn more about the nature of HIV and work harder on other treatments?

 

Source: http://www.cnn.com/2013/12/07/health/hiv-patients/index.html?hpt=hp_t2

(Special Health Issue) Clinical Trials and Tribulations

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In 2008, New Zealand lecturer Sarah Broom developed a cough while she was pregnant with her third child. She had to deliver her baby three months early in order to get a biopsy, but by then the lung cancer had become advanced-stage and not much could be done. Chemotherapy didn’t help– doctors soon found a new plum-sized tumor in her ovary. Broom contacted friends all over the world to ask for help, including someone in Boston who managed to enroll her in a clinical trial in Australia for a brand-new compound called crizotinib that had only been tested in two patients before. This drug worked briefly. Two years later, the tumors came back and Broom developed new ones in her brain after receiving treatment in Boston. Her last hope was a drug called LDK378– however, it had not been released yet because it was still going through clinical trials.

The idea behind clinical trials is that a new drug is tested on patients who have not been responding to other treatments; the company that developed the drug would track the patients’ condition to see if the drug is effective, and if it causes side effects. Each drug has to go through three stages of trials before it can be released to the public. In the United States, this process could take about 10 years, and the process of applying for a clinical trial is a huge pain for patients. Just look at this flow chart about the application process (don’t read it– I have no idea what these words mean):

clinicaltrial

In the end, Broom and her family wrote letters to Novartis, the drug company that developed LDK378, to request compassionate access (when everything is skipped and patients get immediate access to the medicine). The drug worked, and everything turned out fine.

But clinical trials aren’t just another example of paperwork and bureaucracy– there have been examples of drugs that were thought to be effective, only for people to learn that the side effects are worse than the diseases they were supposed to treat. Interferon gamma, which was developed for a lung disease, actually caused respiratory infections that killed patients faster. The sedative thalidomide, which was used for women in labor, was responsible for thousands of birth defects. Clinical trials could have discovered this long before these drugs were released.

 

Source:

http://www.nytimes.com/2013/11/03/magazine/how-dying-patients-get-access-to-experimental-drugs.html?pagewanted=4&_r=0&ref=science

[Th]e [S]hutdown: Why It Matters

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Everyone knows about the really obvious effects of the government shutdown on science— the Smithsonian museums are closed, national parks are closed, NASA is pretty much closed, and this thing pops up whenever you try to do research for a science class:

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However, there are two other slightly more serious consequences that haven’t been publicized as much: many patients with serious illness are being kept from possible treatments, and our food is no longer being inspected.

Even though people are still being enrolled in clinical trials at the National Institutes of Health, the process has become much slower. Only 12 patients have been enrolled since October 1st, when the shutdown began. Normally, about 200 patients, many with cancer, are enrolled each week and more than 1,400 trials run at any given time. But because of the lapse in funding, only people who are in immediate danger of dying are being added to the list.

It’s important to note that clinical trials are often a last hope for patients—they’ve gone through other known treatments but the disease hasn’t improved, so they decide to try new, experimental treatments that are still being studied. So while lawmakers and politicians are arguing about how health care should work, other people are trying to decide which 12 patients (out of 200) with long-term, life-threatening illnesses need treatment the most, since 75% of the NIH staff has been furloughed and the agency just doesn’t have enough funding to treat everyone.

I guess this won’t be happening anytime soon:

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The CDC and FDA have also suffered cuts, losing 68% and 45% of their staff, respectively. Among this staff are epidemiologists who had been tracing a salmonella outbreak that started in California and spread through 18 states, before they were furloughed. There have been no inspections of domestically produced food this week because inspectors, lab technicians, and many other staff members have also been sent home.

Food inspections have led to a lot of important information being made public in the past, most recently the fact that arsenic was used in chicken, turkey, and pig feed. If the government shutdown lasts for many more weeks, it would be almost impossible for anyone to know what new things are added to the food that our meal plans pay for.

These issues are frustrating because even though they are so severe, they are so easily fixed; if funding is resumed, the NIH could continue to perform clinical trials, and the CDC and FDA could go back to working on public health.

Hopefully our government will pause their health care debate long enough for that to happen.

 

Sources:

http://www.nytimes.com/2013/10/10/us/politics/risk-to-food-safety-seen-in-furloughs.html?src=recg

http://www.nytimes.com/2013/10/10/health/in-shutdown-clinical-trial-enrollment-slows-but-doesnt-halt.html?ref=science