Access to Success: Fairness in Post Clinical Trials
What is the purpose of a clinical trial? Is it to feed the “machine” of trials? Is it to repeatedly “try” and never really look at the outcome? Is it to ask participants to be willing to be human guinea pigs with no regard for their lives?
Don participated in a TauBasket trial. The University of California, San Francisco, sponsored the trial, and Biogen provided the drug BIIB092, Gosuranemab. This basket trial was composed of four rare diseases; Corticobasal Syndrome (CBS) being one of them. There were eight patients assigned to the CBS group. Don began to improve immediately. His physical and cognitive changes were remarkable. Those changes were continuous and cumulative while taking the drug. We have videotaped neurology exams and a mountain of anecdotal evidence. Even with all the success Don achieved, Biogen refuses to grant him access to this life-changing drug. We have appealed under Compassionate Use and the Right-To- Try Act.
In Don’s case, we have a definitive documented change as a result of a trial. Don was willing to risk his life in the hopes of advancing science, treatment, and cures, but there is no mechanism in place for him to have access to the medication. We have no recourse but are at the mercy of a pharmaceutical company. The “science” of clinical trials must be addressed immediately. If a drug company is unwilling to supply the medication that a participant has shown to be efficacious in an individual case, the NIH and FDA should not allow them the ability to sell their drugs on the open market.
While a pharmaceutical company is under no obligation to manufacture a unique drug for one patient, they are currently producing 654 doses of Gosuranemab in their Alzheimer’s trial. Don’s last dose was December 11, 2019, and as of June 2020, he has declined 20-25% verified by his neurologist and speech pathologist.
Ironically, Biogen is willing to donate $10 million to help fight the COVID-19 Pandemic in which over 90 of their employees are infected, but is unwilling to help save Don Sladek’s life by providing their medication BIIB092, Gosuranemab to him.
Number of People with Neurodegenerative Disorders and How that Influences Biogen
The following statistics are for people in the United States and are important in understanding what has happened in Don’s case:
Alzheimer’s disease has about 5.8 million
Parkinson’s has about 1 million
Progressive Supranuclear Palsy has about 5 or 6 per 100,000
Corticobasal Syndrome (CBS) is very rare with about 5 people per million. There has not been a proper study, but it is believed to be about 10% the rate of (PSP).
These are current and past trials where Biogen’s drug, BIIB092, Gosuranemab has been used.
654 Alzheimer's Disease (AD) Patients
ClinicalTrials.gov Identifier: NCT03352557
Phase 2 Study of BIIB092 in Participants With Early Alzheimer's Disease (TANGO)
Estimated Completion Date May 25, 2021
490 Patients for Progressive Supranuclear Palsy (PSP)
ClinicalTrials.gov Identifier: NCT03068468
Study of BIIB092 in Participants With Progressive Supranuclear Palsy (PASSPORT)
Ended because it failed to meet their endpoint goals
ClinicalTrials.gov Identifier: NCT03658135
BIIB092 in Primary Tauopathies: CBS, nfvPPA, sMAPT, and TES (TauBasket)
12/19/19-Terminated - Biogen refuses access to the medication.
A couple of notes will set the stage for the problem that has led us to seek the public’s help in appealing to Biogen on Don’s behalf. First, while Biogen provided the drug for the trial, Biogen did not sponsor the trial. Dr. Boxer and UCSF were the sponsors of the trial. Second, concurrently, Biogen was sponsoring a clinical trial for Progressive Supranuclear Paly (PSP) patients: NCT03068468 Study of BIIB092 in Participants With Progressive Supranuclear Palsy (PASSPORT), with a total of 490 patients. It is my understanding that the trial did not meet its endpoint goals. As a result, this Biogen-sponsored trial was halted December 13, 2019, and because the medication was halted for this study, it was also halted for Don’s TauBasket Trial. Finally, Biogen is currently investigating the same drug in the following clinical trial for Alzheimer’s Disease: NCT03352557 Phase 2 Study of BIIB092 in Participants With Early Alzheimer's Disease (TANGO), with 654 patients.
Here is our problem:
1. Biogen’s drug, BIIB092, changed Don’s life and he is quite possibly alive today only because of it. Without it, he will degenerate and we will lose him.
2. Biogen refuses to allow access to BIIB092 due to the failure of the PSP trial.
3. Biogen has refused to acknowledge TauBasket, the UCSF-sponsored trial. The company has not reviewed any of the data and refuses to communicate with us. We just keep getting referred back to UCSF, and UCSF no longer has access to the drug—see previous point.
4. Biogen has clearly stated to Dr. Borderlon that it will not offer the drug under Compassionate Use or the Right-To-Try Act, and no one at Biogen is willing to discuss or even entertain the fact that Don’s was the TauBasket trial and not the PSP trial.
5. We understand that we cannot force Biogen to make a drug; however, they are currently producing BIIB092. Biogen is manufacturing BIIB092 for the TANGO Phase 2 trial for Alzheimer’s Disease, which has an estimated enrollment of 654 patients. The trial began in May 2018 and is expected to last 3 years and 2 months, which would mean continued manufacturing of it at least until May 2021, with the potential for long-term extension depending on the success of the trial. Evidence of BIIB092’s use can be found at https://www.biogentriallink.com/en-us/clinical-indications/alzheimers-disease/tango.html.
Alzheimer’s disease has about 5.8 million
Parkinson’s has about 1 million
Progressive Supranuclear Palsy has about 5 or 6 per 100,000
Corticobasal Syndrome (CBS) is very rare with about 5 people per million. There has not been a proper study, but it is believed to be about 10% the rate of (PSP).
These are current and past trials where Biogen’s drug, BIIB092, Gosuranemab has been used.
654 Alzheimer's Disease (AD) Patients
ClinicalTrials.gov Identifier: NCT03352557
Phase 2 Study of BIIB092 in Participants With Early Alzheimer's Disease (TANGO)
Estimated Completion Date May 25, 2021
490 Patients for Progressive Supranuclear Palsy (PSP)
ClinicalTrials.gov Identifier: NCT03068468
Study of BIIB092 in Participants With Progressive Supranuclear Palsy (PASSPORT)
Ended because it failed to meet their endpoint goals
ClinicalTrials.gov Identifier: NCT03658135
BIIB092 in Primary Tauopathies: CBS, nfvPPA, sMAPT, and TES (TauBasket)
12/19/19-Terminated - Biogen refuses access to the medication.
A couple of notes will set the stage for the problem that has led us to seek the public’s help in appealing to Biogen on Don’s behalf. First, while Biogen provided the drug for the trial, Biogen did not sponsor the trial. Dr. Boxer and UCSF were the sponsors of the trial. Second, concurrently, Biogen was sponsoring a clinical trial for Progressive Supranuclear Paly (PSP) patients: NCT03068468 Study of BIIB092 in Participants With Progressive Supranuclear Palsy (PASSPORT), with a total of 490 patients. It is my understanding that the trial did not meet its endpoint goals. As a result, this Biogen-sponsored trial was halted December 13, 2019, and because the medication was halted for this study, it was also halted for Don’s TauBasket Trial. Finally, Biogen is currently investigating the same drug in the following clinical trial for Alzheimer’s Disease: NCT03352557 Phase 2 Study of BIIB092 in Participants With Early Alzheimer's Disease (TANGO), with 654 patients.
Here is our problem:
1. Biogen’s drug, BIIB092, changed Don’s life and he is quite possibly alive today only because of it. Without it, he will degenerate and we will lose him.
2. Biogen refuses to allow access to BIIB092 due to the failure of the PSP trial.
3. Biogen has refused to acknowledge TauBasket, the UCSF-sponsored trial. The company has not reviewed any of the data and refuses to communicate with us. We just keep getting referred back to UCSF, and UCSF no longer has access to the drug—see previous point.
4. Biogen has clearly stated to Dr. Borderlon that it will not offer the drug under Compassionate Use or the Right-To-Try Act, and no one at Biogen is willing to discuss or even entertain the fact that Don’s was the TauBasket trial and not the PSP trial.
5. We understand that we cannot force Biogen to make a drug; however, they are currently producing BIIB092. Biogen is manufacturing BIIB092 for the TANGO Phase 2 trial for Alzheimer’s Disease, which has an estimated enrollment of 654 patients. The trial began in May 2018 and is expected to last 3 years and 2 months, which would mean continued manufacturing of it at least until May 2021, with the potential for long-term extension depending on the success of the trial. Evidence of BIIB092’s use can be found at https://www.biogentriallink.com/en-us/clinical-indications/alzheimers-disease/tango.html.
© COPYRIGHT 2020. All rights reserved. Unless attribution otherwise noted.
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THANK YOU“I would like to thank all of you from the bottom of my heart that took the time to read this site and those that took action concerning my situation with CBS. The clinical trial has helped my quality of life. My goal has always been to help science to help others with this terrible disease. God bless and thank you all.”
- Don Sladek |
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