Aleta
Member
- Joined
- Dec 10, 2009
- Messages
- 26
- Reason
- CALS
- Diagnosis
- 03/2008
- Country
- US
- State
- TN
- City
- Thompsons Station
I wrote to Mr. Petzinger about getting compassionate use for this drug nd this was his response to me. Time is running out for my husband and other ALS patients.
What else can be done? Any ideas?
Dear Aleta,
Please call me Tom.
I want to thank you for your note. We receive many communications from patients and families, as you clearly recognize, but some, such as yours, stand out as real statements from the heart. It’s a reminder—as if we needed one—about why we set up our company in the first place. Our sole focus is advancing a meaningful treatment for ALS.
Indeed, that’s the very reason why I have to disappoint your request, much as I might wish it were otherwise.
I don’t know the particulars of Charlie’s case and, as a layman, I’m certainly unqualified to evaluate it. But as a general matter, getting a drug approved in this disease, especially after so many failures, requires rigorous adherence to protocol requirements, including enrollment criteria. Meeting the goal also means limiting exposure of the drug to people enrolled in well-controlled studies reviewed and approved by regulatory bodies in the U.S. and Europe. To put it simply, we have to conduct our studies by the book if we’re to have the best possible chance of success against this disease.
Finally, we haven’t established in a large population that the drug is safe for ALS patients. We’re especially mindful that in some recent large clinical trials, drug candidate actually worsened the conditions of ALS patients, despite showing earlier promise.
I admit this reasoning may ring hollow to someone living with the disease now, but it’s motivated by a commitment to advancing a drug that ultimately may serve the entire community of people with ALS, long into the future.
Thank you again for writing. Best wishes to you and Charlie.
Sincerely,
Tom Petzinger
--------------------------------------------------------
Tom Petzinger
EVP, Business Development and Public Affairs
Knopp Neurosciences Inc.
2100 Wharton Street - Suite 615
Pittsburgh PA USA 15203
+ 412.488.1776
Knopp Neurosciences
What else can be done? Any ideas?
Dear Aleta,
Please call me Tom.
I want to thank you for your note. We receive many communications from patients and families, as you clearly recognize, but some, such as yours, stand out as real statements from the heart. It’s a reminder—as if we needed one—about why we set up our company in the first place. Our sole focus is advancing a meaningful treatment for ALS.
Indeed, that’s the very reason why I have to disappoint your request, much as I might wish it were otherwise.
I don’t know the particulars of Charlie’s case and, as a layman, I’m certainly unqualified to evaluate it. But as a general matter, getting a drug approved in this disease, especially after so many failures, requires rigorous adherence to protocol requirements, including enrollment criteria. Meeting the goal also means limiting exposure of the drug to people enrolled in well-controlled studies reviewed and approved by regulatory bodies in the U.S. and Europe. To put it simply, we have to conduct our studies by the book if we’re to have the best possible chance of success against this disease.
Finally, we haven’t established in a large population that the drug is safe for ALS patients. We’re especially mindful that in some recent large clinical trials, drug candidate actually worsened the conditions of ALS patients, despite showing earlier promise.
I admit this reasoning may ring hollow to someone living with the disease now, but it’s motivated by a commitment to advancing a drug that ultimately may serve the entire community of people with ALS, long into the future.
Thank you again for writing. Best wishes to you and Charlie.
Sincerely,
Tom Petzinger
--------------------------------------------------------
Tom Petzinger
EVP, Business Development and Public Affairs
Knopp Neurosciences Inc.
2100 Wharton Street - Suite 615
Pittsburgh PA USA 15203
+ 412.488.1776
Knopp Neurosciences
