Science & Medicine

Posts tagged ‘MPN Clinical Trials’

Summertime is here

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Hi, there… It’s just minutes before midnight and the Summer Issue of MPNforum is ready to ship to Subscribers.

That is: links should work, most everything is in place, it’s been proofread and edited.  Still, we could use your help in reviewing  stories and passing along  any errors or changes as well as comments before we launch to the Internet.

There are serious stories in this issue and some good news about friends and heavy lifting done by fellow patient Ellen Jacquardt to get the new expanded list together. And, as ever, we’re indebted to the doctors who generously provide their advice and counsel and support.

So enjoy…and please, let’s hear from you… via comments or e-mail (ourMPNforum@gmail.com)..

The Summer Issue is here”  https://mpnforum.com/summer-2014/

Cheers,

Zhen

 

* Remember, the “Back to Contents” link on each page will take you to the old MPNforum home page. We switch at noon tomorrow.

TSR – Summertime, 2014

No drugs. No surprise.

Zhen

Zhen

For a very few of us, it happens in the beginning. For most, it’s a final option, an endpoint with hopes for a fresh start.

That’s what clinical trial and stem cell transplant have in common.

Headed into the Summer, now that reports are available from the big US and European hematology conferences we can see the naked truth.

We have no good drug options beyond, possibly for some, the interferons.

No FDA approved drugs at all except Jakafi for symptomatic relief of high risk myelofibrosis. Our basic meds are all off label. Their MPN use is not indicated on the professional label. Hydroxyurea, interferon…off label.

Pain or distress prevents many of us from considering why we don’t have good drugs.

Simple cause and effect. All those reports from hematology conferences on clinical trials, complete responses, promising results, etc. and the lack of meds to relieve and cure our myeloproliferative neoplasms are directly related.

No drugs. That’s the inevitable result of a clinical trial system that demands nearly $1 billion and several years of trial and error, testing a single blood cancer drug with uncertain outcomes. No brilliant graduate student, no small biotech need apply. This game is for the big dogs only.

For the past five years, while we suffer and die, drug companies have been chasing the same damned JAK2 inhibitor with lookalike drugs, clogging drug development channels and squandering resources – billions of dollars that might otherwise have been spent on real scientific exploration.

There is an answer. Until this ancient, flawed clinical trial system gets reformed, until genetics, information technology and biotechnology are wedded in a new medical research paradigm, we can take evasive action.\\
We can embrace a healthy lifestyle and off label drugs to the extent possible. We can avoid all dosage and toxicity trials if possible. We can’t afford the time or impact on our bodies. And we can determine to enter late stage trials only with some definite and specific anticipation of benefit. We are patients, not lab mice. Choking off the supply of willing subjects — if possible — is one way to bring about change.

Guiding us in this effort are our personal hematologists and trusted MPN specialists. Doctors Ruben Mesa, Claire Harrison, Serge Verstovsek all featured in this Summer issue of the Forum — are workers in the tangled obscure vineyard of MPN. They are our allies and worthy of trust and respect.

As principal investigators into new drugs, however, they are also beneficiaries of funds from Novartis/Incyte and other drug companies.

We can trust them to report objectively and honestly about drugs produced by Novartis/Incyte and others. We can’t expect them to publicly criticize the companies or the clinical trial process in which they are embedded.

But we can. We can look at this stuff with an open mind, open heart and clear head, share our findings with each other, criticize when we must, and act together to change things.

Aren’t you sick and tired of being sick and tired?

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finger pointing smallComing up TOMORROW in the Summertime MPNforum… Reviews of the PRM-151 and Ruxo/PV trials…Preview of the MQJ SCT discussion by panel of transplant experts with intro by David Steensma and Claire Harrison, perspective by Chris Harper. Stories from patients emerging from the clinical trial and SCT experiences, Harvey Gould, five days post transplant tells all…Voncille, Patient F in the PRM-151 trial tells how it is….and a major expansion of the List of Hematologists (thank you Ellen Jacquardt). See you all tomorrow.

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finger pointing small Dr. Ruben Mesa needs us to step up and take his survey. We now have assurances of privacy and an arm’s length relationship with sponsor Incyte. Our reservations about Incyte’s use of marketing data from the survey are trumped by Mesa’s need for these data to complete his Fatigue research. Our fatigue research.

Think about it. No one has done more for us than Ruben Mesa, investigating MPNs, teaching, treating patients, counseling, We owe him a debt of gratitude and we owe him this one. Please. Click here to go to the survey. Thank you.

Advance copy Jan.-Feb. MPNforum here

 

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Because some stories can’t wait a month to tell, MPNforum for the first time is publishing multiple issues over the next two weeks. 

Later today we publish  “Lamentations and Celebrations” – tributes to Ian and Patsy…  announcement of the re-opening of the MPNclinic …and headlines and abstracts of the month’s articles.

Your advance copy is here: https://mpnforum.com/january-february-2014/

The dark cloud hanging over MPN Clinical Trials is the subject of “What Sanofi doesn’t want us to know,” an investigative report. Beyond the abrupt termination of a late stage clinical trial, a more pressing question remains:   Should have known months earlier that an unanticipated and deadly side effect was working it way through that trial?

This first MPNforum issue of 2014 will be broadly published this afternoon so if you find anything that needs attention, please let me know. (zhenyasenyak@gmail.com)

Thanks,

Zhen

 

 

 

 

 

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