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Family And Friends Petition Drug Giant For Compassionate Use Of Experi


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#1 Steve Heard

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Posted 24 March 2014 - 09:24 AM

Last November, Mikaela Knapp was diagnosed with an aggressive strain of kidney cancer, which metastasized to her brain. Knapp is 25.

 

Prior to her diagnosis, Knapp had worked as an account coordinator at SparkPR, a technology-focused public relations outfit in San Francisco. At Spark, Knapp developed an affinity and appreciation for technology. Meredith Obendorfer, who worked closely with Knapp, describes her as “a typical AC — bright, bubbly and eager to develop her media relationships.”

 

In the wake of her diagnosis, Knapp’s family and former colleagues have banded together to rally the media. The goal is to put pressure on Merck, one of the largest pharmaceutical companies in the world, to include Knapp in a potentially life-saving clinical trial.

 

As Obendorfer puts it: “We’re fortunate to possess skills that help spread impactful stories fast and far. Mikaela’s struggle with cancer and her fight to get the drugs she needs to survive popped up over social media and quickly gained momentum, particularly given the social nature of our community.”

 

After six months of treatment, Knapp is running out of FDA-approved therapies. After perusing the Internet, her husband (and high school sweetheart) Keith stumbled upon research into new therapies that can attack cancer by inhibiting a cloaking mechanism that tumors use to disguise themselves from the immune system. These drugs, called anti-PD-1 or anti-PD-L1, are under development by pharma giants, like Bristol-Myers Squibb, Merck, and Genentech.

 

More: http://venturebeat.c...-kidney-cancer/


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#2 The Average Joe

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Posted 24 March 2014 - 07:06 PM

I think that people with life threatening illnesses should be able to use experimental treatments at their risk, HOWEVER, they forfeit all rights to sue the company making the experimental drug if there are side effects. I see it as a win win. No need for the government to ALLOW you to take the risk. If I was dying, I wouldn't want some faceless bureaucrat deciding that an experimental treatment was too risky for me. Understanding the world, some asshat would then sue the company if there were side effects. Make the law protect the researching company, and everybody wins.


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#3 Steve Heard

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Posted 24 March 2014 - 08:50 PM

I agree. It reminds me of the Dallas Buyer's Club, in which the FDA banned substances which were legal in other countries, and some even legal here, except they weren't legal for treating AIDS. 


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#4 Carl G

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Posted 25 March 2014 - 08:07 AM

I agree too, but if the drugs save a life but cause some other long term damage, we all end up having to pay for it.  Even so, if you are going to die anyway, why not give it a shot.



#5 Deb aka Resume Lady

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Posted 25 March 2014 - 08:42 AM

If I understand correctly, the issue is that drug companies can only offer drugs that are being tested to people who are participating in their trials. As such, they have specific criteria in order to participate and all of the results are included in their studies. Because Mikaela's cancer is in a late stage (in contrast to their criteria), drug companies are concerned the results of giving the drugs to Mikaela will skew the overall results of the study. If the drugs are not effective in the late stages of cancer,  the overall results of the study will be negatively impacted and the drug companies risk not getting their drugs approved for anyone. This sucks for people who are willing to take the risks of a trial medication, but do not meet study criteria. I do understand the dilemma of both sides.

 

I wish there was some sort of compromise position the FDA would allow for such people -- allowing them to try the meds without officially be included in studies. However, millions of people might want to try trial drugs as a last resort and I don't think this is feasible for a plethora of reasons.

 

My heart goes out to Mikaela and I hope something can be done for her.


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