A second drug trial to treat the pulmonary fibrosis of Hermansky-Pudlak Syndrome is planned to open this spring, according to the announcement made by Dr. Markello from the National Institutes of Health at the Hermansky-Pudlak Syndrome Family Conference last week.
This protocol is designed to treat patients that are found to be too ill for the ongoing Pirfenidone protocol. Patients must have a forced vital capacity (FVC) of less than 45 for this trial.
The trial will test a cocktail of medications, all of which are already on the market for other uses. It’s NIH’s hope that these medications, in combination with one another, will slow down the disease process.
The protocol will recruit six to 12 patients for this study. They will be brought to the NIH (at NIH’s expense). The first hospitalization is estimated to be three weeks as each medication is added gradually and any adverse reactions or side effects are monitored. Then patients will return to NIH for approximately three days every three months during the duration of the trial.
The drugs to be used in the cocktail include:
Pravastatin
Losartan
Zileuton
N-acetyl-cysteine
Erythromycin (this is an antibiotic that some people have bad reactions to and another antibiotic may be substituted that is easier to tolerate if that’s a problem.)
If you want to know about why each drug was chosen, and what it’s side effects are, or more information about the protocol its self, please feel free to get in touch with me (I’ve got more notes) or with the HPS Network.
I’m excited about this protocol for several reasons. First, it gives HPS patients with advanced lung disease another option. Getting on a lung transplant list can be difficult and all the while the patient isn’t getting any better. Thus, this gives patients another chance.
I’m also excited because I see it as a signal of NIH’s commitment to the HPS community and continued research. NIH is biting off quite a bit with this protocol. It means bringing in patients that could be quite sick and require a lot of care – a major investment for NIH.
I’d also like to give a big BRAVO to Dr. Markello on his presentation. Dr. Markello was surveying everyone in Champions about how he could make his slides easier to see for everyone. I was in the front row, and I actually could read most of them with my monocular. Way to go Dr. Markello! Thanks for caring so much!
This protocol is designed to treat patients that are found to be too ill for the ongoing Pirfenidone protocol. Patients must have a forced vital capacity (FVC) of less than 45 for this trial.
The trial will test a cocktail of medications, all of which are already on the market for other uses. It’s NIH’s hope that these medications, in combination with one another, will slow down the disease process.
The protocol will recruit six to 12 patients for this study. They will be brought to the NIH (at NIH’s expense). The first hospitalization is estimated to be three weeks as each medication is added gradually and any adverse reactions or side effects are monitored. Then patients will return to NIH for approximately three days every three months during the duration of the trial.
The drugs to be used in the cocktail include:
Pravastatin
Losartan
Zileuton
N-acetyl-cysteine
Erythromycin (this is an antibiotic that some people have bad reactions to and another antibiotic may be substituted that is easier to tolerate if that’s a problem.)
If you want to know about why each drug was chosen, and what it’s side effects are, or more information about the protocol its self, please feel free to get in touch with me (I’ve got more notes) or with the HPS Network.
I’m excited about this protocol for several reasons. First, it gives HPS patients with advanced lung disease another option. Getting on a lung transplant list can be difficult and all the while the patient isn’t getting any better. Thus, this gives patients another chance.
I’m also excited because I see it as a signal of NIH’s commitment to the HPS community and continued research. NIH is biting off quite a bit with this protocol. It means bringing in patients that could be quite sick and require a lot of care – a major investment for NIH.
I’d also like to give a big BRAVO to Dr. Markello on his presentation. Dr. Markello was surveying everyone in Champions about how he could make his slides easier to see for everyone. I was in the front row, and I actually could read most of them with my monocular. Way to go Dr. Markello! Thanks for caring so much!
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