The subject of this month's ILD Support Group was research. Archer Eller, the Clinical Trial Manager, gave a very detailed update to the group on studies and research for Idiopathic Pulmonary Fibrosis (IPF) patients. Of all ILDs, the clear majority of patients have IPF, therefore that group of people are used in studies. When a medication to treat IPF is approved, it can be prescribed off-label to patients with other ILDs.
The research goal is to insure that the research is safe, to create good data and to involve a specific population. The results are only trusted when the study is randomized, includes a placebo control and is a double-blind study.
Archer began with a short tutorial on the process of research. It begins with an idea then tried with chemicals in a petri dish. Humans are brought in during Phase 1 who are usually poor, healthy college students paid to try, for example, a pill. This phase usually involves 5-10 people.
Phase 2 involves a target population from 50 to maybe even 200 people. The question is whether the new medication is safe, see if it works and to fine-tune the dosage.
Phase 3 includes hundreds of people, usually 500-600. Specifically to our group, they usually include IPF and transplant patients. This is the final phase before FDA review and approval.
Phase 4 is after the drug is marketed by following any side effects.
We began the discussion of the saga of the research on the drug Pirfenidone, which was the first anti-fibrotic drug ever. It has been approved in India, Japan, Europe, Australia and parts of South America. At the last moment, before it was expected to get FDA approval in the US, they requested more research.
The first Pirfenidone trials began in Japan then my university hospital enrolled seventeen patients in the 004 study then there was also 006. The name of the study was CAPACITY. They took the results to the FDA who found it to be helpful in treating IPF and five people continued with the study. Six years later, it was expected to be approved to open label, which could then be used on patients with other ILD diseases. The long term effects would be studied. But, it didn't happen. The FDA wanted another one-year study.
The FDA found that the people who had a good result were in a specific sub group: people who had IPF but had specific other numbers. This Phase 3 study is called ASCEND. The specifics to be included into this study included:
Diagnosed longer than six months but not more than four years
Forced Vital Capacity of 50-90% predicted (mild or moderate)
FEV/FVC (Blow out 1 second) greater than .8 or 80%.
At my hospital, they tested fifteen people for this study but nine failed to meet the requirements.
The process began with the initial screening visits, which sometime lasted two weeks or months, enrollment was next in which the patients were randomized to insure a double-blind study then, when the study finally begins it will last 52-weeks. They anticipate that it will begin in 2013 and completed in 2014 when the data will be analyzed for 3-6 months. The FDA will also study the results so the earliest the drug will be approved in the US market will be 2015.
But, here is the problem. Once it is approved for use in the US as the first anti-fibrotic drug, there will be no attention paid to only treat this subgroup of people that showed a positive result. So, the drug itself will be VERY expensive yet probably not do anything for a huge number of IPF and ILD patients.
IPF is an orphan disease. It does not have a lot of public awareness. Apparently, there is not a lot of money to be made by the drug companies because there are not a lot of people with the disease to take their new drug. Why spend all the money on research?
We were told an interesting story of another drug that had been discarded by the developing drug company and picked up by a non-profit. It was a drug for cystic fibrosis, an autoimmune disease. The Cystic Fibrosis Foundation bought the rights to the drug, developed it and now owns it. They have made enough money from it that they are now doing other research on other drugs. A success story.
The hope is that the Coalition for Pulmonary Fibrosis or the Pulmonary Fibrosis Foundation will step up and begin to develop these drugs as a non-profit, just like the story above.
In the following blogs, I will report about the latest in the hopeful BIBF trials, PANTHER NAC trials, the National institute of Health's trial of connective tissue studies, Scleroderma Lung Study regarding cytoyan, and observational study regarding GERD, biomarkers and the suspected reason why ILD patients develop GERD.
