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Key Findings

Completed Studies

Skin delivery of Endoxifen for Breast Cancer Prevention:

Oral forms of the drugs used for the prevention of breast cancer, like oral Tamoxifen, a selective estrogen receptor modulator (SERM), can cause side effects throughout the body which makes it less appealing. Our study aimed to find an alternative to oral Tamoxifen in preventing breast cancer for high-risk women, exploring the use of topical Endoxifen, the key active metabolite of tamoxifen that plays a crucial role in its preventive effects. Our study found that topical Endoxifen is safe and penetrated through the breast skin. This underscores the importance of further investigation into the relationship between drug dose and skin absorption to enhance outcomes.

Skin deliv

Topical Tamoxifen's Potential in Preventing Breast Cancer

Oral tamoxifen (oral-TAM) benefits women with DCIS but concerns about side effects limit its use. Earlier studies suggested a topical gel form, 4-hydroxytamoxifen (4OHT-gel), might work as well with fewer side effects. In this study, we compared the 4OHT-gel to oral tamoxifen, but the gel didn't prove to be equally effective. We are now working on a new gel formula that will deliver drug into the breast at higher amounts.

Topical Tam

Iloprost as a Preventive Measure for Lung Cancer in Former Smokers

Iloprost is a synthetic analog of prostacyclin, a naturally occurring anti-inflammatory substance in the body. In a previous study, it helped improve abnormal cell changes known as dysplasia in ex-smokers and showed promise in preventing lung cancer in early tests. We studied using Iloprost as an inhalant in ex-smokers as another way of delivering the drug to the lung and found it was well tolerated. Molecular analysis suggests that Iloprost promotes immunity within the lungs and slows cell growth. We also discovered that a patient’s lung cells when grown in the laboratory can tell us whether iloprost will be effective for cancer prevention in a particular person. This test predicts response to Iloprost and can be used for future trials for lung cancer prevention.


Telapristone as a Topical Approach to Breast Cancer Prevention

Telapristone Acetate is a drug thought to block the effects of progesterone, a hormone linked to the development and growth of certain types of breast cancer. Like other oral medicines they can cause side effects in other organs. Therefore, healthy women at high risk for breast cancer can be reluctant to take them. We investigated the topical application of telapristone acetate to the breast skin and compared its ability to penetrate breast skin and distribute throughout the breast, to oral administration. Our study showed that despite limited skin absorption, the drug distribution in the breast was similar to the distribution when taken orally. This research advanced our understanding of delivering drugs to breast tissue through the skin and has broader implications for developing new topical treatments.


Simvastatin's Impact on Recurrent Pancreatitis

Acute pancreatitis can progress to chronic pancreatitis and hence increases the risk of pancreatic cancer. A cholesterol lowering drug called simvastatin has been shown to potentially reduce pancreatic cancer risk. We carried out a multicenter study to see how simvastatin affects people with recurrent acute pancreatitis, using the endoscopy to collect pancreatic fluid samples that tell us whether pancreatic function improved after treatment with simvastatin. We enrolled a small number of participants and learned that this study highlights the need to consider recruitment feasibility in future trials.


Exploring Berberine's Potential in Colon Cancer prevention for people with Ulcerative Colitis

Berberine, a natural product from China, is commonly used there for digestive and rheumatologic Diseases. While it's considered generally safe, its safety for people with ulcerative colitis isn't confirmed. We conducted a study in China to test its usefulness for colon cancer prevention and found that berberine, when taken orally, is well-tolerated by patients with ulcerative colitis. It also reduced inflammation in the colon. These results suggest that berberine has good potential for the prevention of colorectal cancer and deserves to be studied further.


Colon Cancer Prevention in People with Inflammatory Bowel Disease

Myo-inositol is a compound that has shown promise as an anti-inflammatory agent. People with inflammatory bowel disease and abnormal colonic cells are at increased risk for colon cancer.  We found that the use of myo-inositol decreased the molecular changes that are associated with colon cancer risk, and further development of myo-inositol in this setting may therefore be useful.


Tamoxifen as a Pill vs. a Gel for Safer Breast Cancer Prevention

Tamoxifen is a well proven medication for breast cancer prevention and the treatment of duct carcinoma in situ (DCIS, or Stage zero ductal cancer). However, many women decline tamoxifen pills for fear of side effects. In this study we tested a new way of delivering an active form of tamoxifen (4-hydroxytamoxifen or 4-OHT) as a gel applied to the breast skin in women who needed surgery for DCIS. We found that the 4-OHT gel was as effective as tamoxifen pills in decreasing the growth rate of DCIS cells, suggesting that 4-OHT gel may be substituted for oral tamoxifen in future studies. Particularly important was the fact that very little 4-OHT appeared in the circulation of women in the gel group, raising expectations that 4-OHT gel would cause few side effects and would be more acceptable to women. Additional studies of this approach are now ongoing.


Colon Cancer Prevention with a Colon-Cleansing Laxative

Polyethylene glycol (PEG) is a laxative that is used to cleanse the colon in preparation for colonoscopy.  Findings from the laboratories at Northwestern and other universities suggested that PEG may also prevent colon cancer. We conducted the first human trial to evaluate early signals of colon protection, using two different doses of PEG versus a placebo.  We found that the use of PEG in this setting is safe, and changes suggestive of colonic protection were seen.  Larger trials are now needed to establish PEG as a strategy for colon cancer prevention.

Colon 2

Photodynamic Therapy for Prevention of Mouth Cancer

Over the past three decades, photodynamic therapy (PDT) has developed as a light-based tool which aids in the destruction of abnormalities that occur on the surface of organs, such as the skin, the lining of the mouth, the lung, and the gut. It involves the use of a drug that makes cancer and pre-cancer cells sensitive to laser light; when laser light is then applied to the abnormal areas, these areas are destroyed. People at risk for cancer may develop whitish patches in the mouth called leukoplakia which can then progress to cancer.  This trial tested the safety and effectiveness of a newer form of PDT, and found that this was safe and had some effectiveness. These findings help  form the basis for additional, larger trials in the future.

Photo mouth


Selenium is a food component that has shown promise for cancer prevention, but clinical trials have shown mixed results.  In this study, we looked at a particular form of selenium given to healthy men, in three doses, to better understand the absorption of this compound when given by mouth, and the effects of different doses.


In a second selenium study, we conducted additional investigations to see what is the best form of selenium to use in cancer prevention studies. Healthy male volunteers took two different forms of selenium, to study relative absorption and excretion.  These two studies will guide the design of future prevention trials using selenium in people who are selenium-deficient.


Aspirin and Cancer Prevention

Aspirin use has been shown in many studies to have protective effects against colon cancer.  In this study, we tested a new way of looking at colon cells, to see if the beneficial effects of aspirin could be assessed after a short time of aspirin use,  by shining a light on the colon cells.  The study was successful in this regard; after three months of treatment, participants in the aspirin group showed normalization of these markers, but participants who received placebo did not.  Therefore, this new and accurate way of evaluating colon cells is being tested further in our ongoing studies and will be an important aid to studies of colon cancer prevention.



The goal of this study was to find out if anti-estrogen compounds extracted from soy beans could have a cancer prevention effect in healthy women at high risk for breast cancer.  The study found that in postmenopausal women, no significant good or bad effects were seen on the breast. But in pre-menopausal women, there was a possibility that these soy compounds could increase the growth rate of breast cells, which is undesirable.  The outcome of the study was therefore to caution women - particularly younger women - against the use of soy extracts, although the consumption of whole soy foods (like edemame or tofu) has no known harmful effects.

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