If not YOU, then who?
If not NOW, then when?
How exactly does clinical research work?
In general, basic scientists work with cells and animal models to learn about the mechanisms that cause a particular disease to behave the way it does – such as how AVMs form in HHT. Based on these mechanisms, we propose drugs that might block these mechanisms to stop or reverse the disease. With the approval of the Food and Drug Administration (FDA, in the USA), a research study is designed in which people with that disease will receive a drug under close supervision to monitor for benefits and side effects.
In some research studies a placebo may be included. A placebo is a phony drug (e.g. a sugar pill that looks just like the real pill) that you might take during the research study and neither you nor your doctor would know if you were getting the real pill or the phony pill. By comparing the results from patients taking placebo to the results from patients taking real drug, we can figure out which benefits and side effects are due to the drug and which are due to the natural history of the disease.
I can see that research is important, but why should participate in it?
You might be thinking “I would rather get help now from a non-experimental drug and let someone else do the research.” Of course, if everyone said that, there would be no “someone else” left, and no one to help advance the treatment in HHT. The case for participating in research is particularly strong in HHT.
Hereditary - Even if the research treatment doesn’t help you during the study, your participation may help to prove the drug helpful so that it is available when someone else in your family is in need – your son, your daughter, a parent, an aunt, a brother, a niece, a cousin. You have a chance to help prove a treatment that may one day save the life of a close relative – how cool is that!
Cost can be another reason to participate in research. In most research studies the drugs are free of charge to participants. If your insurance does not cover a particular treatment – and that is often the case for experimental treatments – you may be able to get that treatment for free through a research study. For some treatments, your only access to that drug may be through a research study if it is not approved for use in your country.
Civic Pride - A research patient at the 2012 HHT Patient Conference in Orlando came up with another reason; he said that he initially joined the HHT NOSE study to get help for his nosebleeds. Although the benefits to his nosebleeds were minor, he was ultimately really glad that he joined because he felt a considerable sense of civic pride to be participating in a cause to help other HHT patients – it was his small contribution to a big cause!
So even if you don’t have other family with this disease, you are still helping doctors and scientists to figure out how to treat a disease and therefore help fellow citizens and society as a whole.
What if I don’t get better or have side effects while in a research study?
If you don’t get better in a research study it could be because the research drug is not effective for you or because you are on placebo. If you get worse during the study, your research doctor may take you out of the study and prescribe a different treatment for you. All research studies are set up so that you can leave the study at any time for any reason, and still have access to all of the approved treatments that you would have had prior to enrolling in the study. Concerning side effects, almost all drugs have them. Most drugs have undergone extensive testing in animals and healthy persons to understand the profile of side effects. A research study is designed to minimize the risk of side effects and monitor for their occurrence. If side effects do occur, they can usually be managed with standard medical care.
But what if my HHT is not bad enough to make me eligible to join a research study?
You can still help! Research is expensive to do the correct way and much of the HHT research so far has been funded by the HHT Foundation. Click on DONATE and either make a general contribution to research or to a specific research area that may be close to your heart because of an affected friend or family member – say someone with severe nosebleeds or brain AVM.