What the HHT?

A Voice for the HHT Community

Cancer, HHT and Avastin: What’s the Connection?


By Cassi Friday

avastinOne of the things I love about Cure HHT is the focus on research and clinical trials for patients. I’d like to use this post to talk about the Avastin clinical trial, which I recently saw posted on Cure HHT’s media pages.

Before delving into the background on this drug and its potential benefits to HHT patients, let me put out a disclaimer. Please talk with your physician if you think you may benefit from participating in a clinical trial. I am not a physician, nor am I a practicing medical professional. I am a scientific researcher and this post serves the purpose to summarize information about Avastin and its potential uses for the HHT community.

If you search for bevacizumab, or its brand name, Avastin, you’ll see it’s an FDA approved drug given to cancer patients to help minimize tumor growth. Your first thoughts may be ‘I don’t have cancer. Why should I try to take a cancer drug to stop my nose bleeds? Cancer drugs are harsh and scary!’

Related imageBevacizumab is something called a monoclonal antibody, meaning it will bind to a specific target. Its target is vascular endothelial growth factor, or VEGF. In cancer, tumors secrete a lot of VEGF and it helps grow blood vessels, and therefore helps grow and spread the tumor. Bevacizumab binds to VEGF and tries to stop blood vessel formation.

About 10 years ago, a couple case studies were published after HHT patients treated for cancer with bevacizumab saw dramatic improvements in the frequency and severity of nosebleeds and hepatic bleeding (Bose, Holter, & Selby, 2009; Flieger, Hainke, & Fischbach, 2006). These patients received the drug through an IV at a high dose to treat their cancer, but the unexpected benefits to HHT symptoms sparked interest in the drug as a therapy for epistaxis.

There are a lot of drugs that have had intended uses, but researchers saw positive effects on an unrelated symptom and changed the drug’s intention. Viagra, for example, was developed as a drug to treat high blood pressure and chest pain, as it dilates blood vessels. However, blood vessel dilation in other areas were a happy side effect and now the drug has a different use!

This is one of the many reasons why clinical trials are so important. Not only can a well-developed research study pinpoint good side effects, it can also pinpoint negative ones as well.

clinical trial

Click for enlarged image

With science, nothing is ever simple and there are always a lot of caveats. It would be great if this drug could be given in an IV and nosebleeds would get better, right? The tricky part is VEGF is something we need to be healthy, so if we try to disable it throughout our entire body, there could be other serious complications.

Researchers have tried administering the drug in a lower dose IV as well as with nasal spray, topical ointment and a submucosal injection. All have been shown to improve nosebleeds in patients with HHT, and since the dose of Avastin is so low there were no negative side effects.

Case closed, right? Well, these research studies did not directly compare its effects to a control, they did not have very many patients and some were merely case studies of a single patient (Simonds, Miller, Mandel, & Davidson, 2009; Suppressa, Liso, & Sabba, 2011; Thompson et al., 2014).

This does not mean these research studies should be discredited. They are all important pieces of information along the journey to see bevacizumab used as a treatment for HHT.

These studies and their findings led to the initiation of a clinical trial across six HHT centers of excellence comparing bevacizumab with a placebo and two other drugs in the North American Study on Epistaxis (NOSE) (Whitehead et al., 2016). In this study, patients either received a saline spray (the control for the study) or one of three drugs to spray in their nose twice a day.

NOSE Study LogoThe study concluded and found there were no differences between the four groups of patients receiving the different medicines or placebo. The study lists a couple of important limitations: 1) they did not have patients keep a diary of nosebleeds before the study started, so there was no way to quantitatively compare patients before and after treatment and 2) they were not sure how effective the drug delivery was because the nose can be pretty crusty in HHT patients due to dried blood that may block the medicine’s entry.

After all this (and much more past research), a phase IV clinical trial is now ongoing to study bevacizumab injection in the nose right after electrocautery treatment. All patients will receive electrocautery treatment, half will get an injection of bevacizumab and the other half will receive a placebo. This should ideally enhance the drug’s effects, as the nose will be cleaned of debris, cauterized and injected with the medicine.

Researchers at Stanford need several more volunteers in order to help complete this clinical trial. If you have talked with your physician, met the eligibility criteria for the study found here and are interested in helping advance the science behind this drug, I encourage you to contact the study team! If you were on the fence about participating in this trial, I hope the interesting background of Avastin has helped you make a decision!



Bose, P., Holter, J. L., & Selby, G. B. (2009). Bevacizumab in hereditary hemorrhagic telangiectasia. N Engl J Med, 360(20), 2143-2144. doi: 10.1056/NEJMc0901421

Flieger, D., Hainke, S., & Fischbach, W. (2006). Dramatic improvement in hereditary hemorrhagic telangiectasia after treatment with the vascular endothelial growth factor (VEGF) antagonist bevacizumab. Ann Hematol, 85(9), 631-632. doi: 10.1007/s00277-006-0147-8

Simonds, J., Miller, F., Mandel, J., & Davidson, T. M. (2009). The effect of bevacizumab (Avastin) treatment on epistaxis in hereditary hemorrhagic telangiectasia. Laryngoscope, 119(5), 988-992. doi: 10.1002/lary.20159

Suppressa, P., Liso, A., & Sabba, C. (2011). Low dose intravenous bevacizumab for the treatment of anaemia in hereditary haemorrhagic telangiectasia. Br J Haematol, 152(4), 365. doi: 10.1111/j.1365-2141.2010.08481.x

Thompson, A. B., Ross, D. A., Berard, P., Figueroa-Bodine, J., Livada, N., & Richer, S. L. (2014). Very low dose bevacizumab for the treatment of epistaxis in patients with hereditary hemorrhagic telangiectasia. Allergy Rhinol (Providence), 5(2), 91-95. doi: 10.2500/ar.2014.5.0091

Whitehead, K. J., Sautter, N. B., McWilliams, J. P., Chakinala, M. M., Merlo, C. A., Johnson, M. H., . . . Gossage, J. R. (2016). Effect of Topical Intranasal Therapy on Epistaxis Frequency in Patients

With Hereditary Hemorrhagic Telangiectasia: A Randomized Clinical Trial. JAMA, 316(9), 943-951. doi: 10.1001/jama.2016.11724



3 thoughts on “Cancer, HHT and Avastin: What’s the Connection?

  1. Thanks Cassi


  2. c
    Could it help people with HHT in the stomach?


    • Hi Raymond – The clinical trial is injecting Avastin in the nose, so it is only designed to help with nosebleeds. If a person receives this antibody (bevacizumab) to their entire body in a higher dose through an IV, it may improve gastrointestinal bleeding.


      Here is a link to a study done on a man who had HHT and also cancer that was getting worse. As part of his cancer treatment, they gave him bevacizumab and watched his HHT symptoms. As a result of the therapy, he did not require as many transfusions and his hemoglobin levels went up drastically.

      Click to access WJGS-8-792.pdf

      Here is another study of a woman with HHT who did not have cancer. They treated her with bevacizumab every two weeks and noticed a great improvement in her gastrointestinal bleeding and need for transfusions.

      Hope this helps. I would talk to the physician who treats you for HHT and discuss the best options for you. At the low doses used in the clinical trial for nose bleeding, this drug does not have side effects. However, at high doses throughout the whole body, the drug can have some serious side effects.


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