The DM Disease Activity Collaborative Study Group, and later the International
Myositis Outcome Assessment Collaborative Study Group recommended development of outcome measures in all forms of IIM - Idiopathic Inflammatory Myopathies - in adults and children, and
coordination and facilitation of clinical trials in adult and juvenile myositis.
The main reason stopping this group from undertaking large multi-center
trials in myositis is the lack of validated disease activity and damage indices.
For this reason, the first in a series of two workshops were held to address the
following:
1. Use of the core set of outcome measures recently identified to
develop a myositis disease activity index, a preliminary definition of
improvement, and damage index, and
2. to develop consensus on issues related to
the general conduct of clinical trials, extend of improvement and damage,
development of inclusion and exclusion criteria and appropriate therapy, and
recommendations in regard to the trial duration, dropout criteria, safety
assessment, and post-trial evaluations. NINDS 2001
Since this was written things have progressed more on this soon. (Sorry, my research is slow)
Please continue to write more because it’s unusual that someone has something interesting to say about this. Will be waiting for more…
ReplyDeleteWe all learned in Kindergarten that life is not fair. But we also learned to recognize injustice when we see it and I feel it is necessary to reveal an injustice about which very few in this country will ever know.
ReplyDeleteThis injustice will affect thousands like me. You see, I am a victim of the disease called Inclusion Body Myositis, (IBM), a serious muscle wasting disease. IBM is similar to ALS (Lou Gehrig's Disease). While ALS attacks the central nervous system, IBM attacks the skeletal muscles. IBM usually runs its course in 15 to 20 years while ALS is terminal in 3 to 5 years. I, like thousands of ALS and IBM patients, am now wheelchair-bound for the rest of my life. Both conditions are terminal but we all must come to terms with that fact. But a recent development in corporate America has added an intolerable insult to our lot in life.
Several American biotech companies benefited from federal grants and donated money generated through donations, including the Ice Bucket Challenge. One of those biotech companies is CytRx Corporation based in Los Angeles, CA. With those donated funds and federal grants, CytRx developed a drug called Arimoclomol. That drug is currently the only known potential treatment for IBM and ALS. Early investigations of Arimoclomol showed it was safe and demonstrated clinical improvement in Phase I and Phase II trials for both IBM and ALS patients.
With the promise of success, Arimoclomol was sold for millions to a Danish biotech company called Orphazyme based in Copenhagen, Denmark. To those of us with IBM and ALS, the only known treatment for our condition being sold to a company in a foreign country after it was developed using US taxpayers' funds goes beyond unjust. It seems criminal. For a foreign company to complete Phase III trials and acquire FDA approval for use in the US could be another five years. Many of us with ALS and IBM wonder if we have that long. If this drug was still in the hands of a US company, victims have the opportunity to apply for compassionate use of the experimental drug. That option was lost when CytRx cashed out. Many of us have tried to reach out to Orphazyme Biotech about the possibility of compassionate applications or to simply find out about progress on FDA approval. I do not know of a single call or email that has been returned or answered.
American taxpayers and donors expect a modicum of accountability. We all hope people and corporations will do the right thing for the greater good. But this is one injustice that can have quality of life ramifications or even become a matter of life or death for thousands. But that hope for greater good and accountability in this case was sold overseas.
To whom can we turn for help?
On a brighter note Orphazyme plans now to release Arimoclomol to market late 2020.