The following was excerpted from the February 2011 edition of eNewsline, where we featured a Q & A with Susan R. Rheingold, an LLS funded researcher and Team In Training alumni:
What is the biomedical problem/issue that you are trying to resolve?
My clinical and research interest is childhood and adolescent ALL, especially relapsed ALL. Currently, a research grant from the Leukemia and Lymphoma Society (LLS) is helping me translate our laboratory findings into new therapies, especially for the patients who have relapsed and desperately need more effective treatments.
At The Children's Hospital of Philadelphia (CHOP), we found that new anti-cancer agents that target/inhibit a molecule called mTOR (mammalian target of rapamycin) can be effective in patients with certain types of leukemia and lymphoma. The mTOR enzyme is part of a network of interacting molecules (pathway) that normally control cell growth, proliferation and survival and are frequently abnormally active in cancer cells. We are presently studying which standard chemotherapy drugs and which other molecularly targeted drugs can be combined with mTOR inhibitor drugs to increase the likelihood of cure.
What's novel or innovative about your approach?
We are studying mTOR inhibition in childhood ALL with a 360 degree approach. Using ALL cell lines and mice who develop ALL, my colleague Stephen Grupp, MD, PhD, and his laboratory have shown that mTOR inhibiting drugs increase leukemia cell killing and “patient” survival. Now, using leukemia cells collected from the children enrolled on our trials, Dr. Grupp is able to study drug effects on human leukemia cells in real time. He can also grow these cells in mice so that we can study in detail the various pathways being affected by the drug. Importantly, we can also use these mice to learn which drugs work best together.
How will your work one day help patients?
By targeting mTOR with molecularly designed drugs, we can inhibit mTOR and thereby inhibit a key cancer cell growth pathway. Our goal is to increase cure rates for young patients with leukemia and lymphoma. Importantly, the toxicity of targeted agents is much less than with classic chemotherapy and can be combined without adding more side effects to patients.
Are you close to clinical trials?
We translated our mouse model findings into a Phase I trial of the oral mTOR inhibitor drug, sirolimus (rapamycin,) in children and adolescents with relapsed acute leukemia or lymphoma. Expanding upon this trial, we are currently enrolling patients at CHOP in a Phase 2 efficacy trial of sirolimus combined with oral methotrexate for children and adolescents with relapsed and refractory ALL and NHL. Two other trials, using a second generation mTOR inhibitor that can be given intravenously, are going through administrative review and will hopefully be opened at many sites through the COG and TACL cooperative groups in 2011.
What other projects are you excited about and believe will benefit patients?
In the past several years our ability to predict which ALL patients are at high risk of relapse has improved based upon cutting-edge molecular tests. Using those same tests, we can look for better anti-leukemia drugs that will target the abnormalities that makes a child’s ALL particularly aggressive. Translational laboratory research and clinical trials are paramount to introducing new drugs, not available otherwise, to keep the more aggressive cases of ALL from relapsing, and curing those that do relapse.
 < | What are some of your hobbies and non-research interests? My first interaction with LLS was over a decade ago when I was part of the inaugural Team-In-Training (TNT) Triathlon Fundraising Team. I did my first half Ironman that year and have subsequently continued competing in triathlons, including a full Ironman. I am the captain of Team CHOP which has raised hundreds of thousands of dollars at the Philadelphia Triathlon for pediatric cancer research. I also raised money for LLS by running the Dublin Marathon with TNT. It is great to be able to combine my athletic interests with my work! If you'd like to recieve eNewsline, our monthly summary of the latest in blood cancer news, subscriber at www.lls.org/news
To become a TNT alum like Dr. Rheingold.....sign up for Team In Training at www.teamintraining.org!ader 2 |
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