See also our related blog for the Pediatric Preclinical Testing Initiative.

Wednesday, December 16, 2009

Sachiko & Tohru present at AACR Brain Tumor meeting

Sachiko and Tohru recently attended the AACR special conference on the Genetics and Biology of Brain Tumors in San Diego. This conference had a high content of adult cancer research, especially glioblastoma multiforme, but also had notable pediatric brain tumor representation including that of our collaborator, Dr. Richard Gilbertson of St Jude. At the meeting Sachiko presented her work on the ubiquitin-proteasome system in medulloblastoma, and Tohru presented his work on the cell of origin of pituitary tumors.

[ above right: Tohru and Sachiko ]

Tuesday, December 15, 2009

Lab Holiday Lunch in Helotes

Today we had our seasonal lab holiday party at the John T. Floore Country Store in Helotes, Texas. It has been a great year, with seven peer-reviewed publications in print or in press... so needless to say - everyone has worked very hard and has a much deserved few weeks of rest ahead. We were pleased that our alumist, Dr. Aoife Kilcoyne, could come back from her Radiology residency in Ireland to join us for the lab lunch (front left in the picture). We have a lot of faith in Aoife bringing a research-oriented approach to pediatric and adult cancer one day. We also celebrated the successful Masters thesis defense of Melinda Garcia (on the right, one person back), as well as Corrine Chua who successfully defended her thesis but couldn't be with us today. And as per our tradition, we'll be making a group contribution to the local women and children's shelter.

[ not shown are lab members Sachiko Ohshima and Tohru Hosoyama, who are at an AACR Brain Tumor conference. ]

Friday, December 11, 2009

NCI Rare Cancer Workshop

Pediatric sarcoma research had good representation at the NCI Rare Cancers with High Mortality: Challenges for Cancer Prevention and Treatment workshop in Bethesda yesterday and today. This workshop of more than 200 investigators, consumer advocates, patient advocates and policy makers examined approaches that might be taken to promote the development of resources and infrastructure necessary for new therapy development (ie, biorepositories, cancer genome and epi-genome studies, mouse models, clinical trials infrastructure and patient advocacy networks. At the workshop, Charles presented a main session talk on Mouse Models, and colleagues Drs. Frederic Barr (rhabdomyosarcoma, U Penn) and Jeff Toretsky (Ewing's sarcoma, Georgetown University) were moderators for a breakout session.

[above right: Jeff, Charles and Fred]

Monday, December 7, 2009

Steve Lessnick visits GCCRI

We are grateful to our colleagues, Dr. Stephen Lessnick from the Huntsman Cancer Institute, to have given a seminar on Ewing's Sarcoma at GCCRI. Steve is a fellow pediatric hematologist-oncologist and physician-scientist who studies the genetics and biochemistry of the Ews:Fli translocation product in Ewing's Sarcoma. Steve also plays an important role in the Children's Oncology Group, demonstrating his commitment to move new findings from the bench to the bedside.

[above right: Steve and Charles at Mi Tierra, a favorite San Antonio breakfast spot.]

Friday, December 4, 2009

Dr. Catchpoole visits from Australia

It was an absolute pleasure to have had Dr. Daniel Catchpoole visit GCCRI from the Childhood Cancer Research Unit at the Children's Hospital at Westmead, Sydney, Australia. Dan presented results of his studies of novel topoisomerase inhibitors that have specific efficacy in rhabdomyosarcoma. Part of this work was done in collaboration with our laboratory as well, with more studies together to come. If you have time, please visit Dan's childhood cancer tumor bank website. Dan is one of the key people across the world working to increase the access that researchers have to tumor material -- so that scientific discovery and better treatments are found more quickly.

[ above right, top:  Dan is a cave guide.  Here he is braving a very narrow passage at Jenolan Cave, Australia ]

[above right, bottom:  Dan at the Far Country Cave, Australia ]

Saturday, November 7, 2009

Welcoming Dustin !

We are happy to welcome graduate student Dustin Green for a rotation in our laboratory. Dustin graduated with a MS in Biology from Louisiana Tech University where his research in the lab of Dr. Mark DeCoster utilized a novel molecular printer to spatially control glial cell line growth. Dustin’s career goal is to perform research at the forefront of translational, bench-to-bedside approaches.

Friday, October 30, 2009

Holiday Fun

It's hard to say how exactly this Halloween-related movie got composited, but it represents our lab's complete postdoctoral team. and yes, that is actually Koichi's real hair.

Wednesday, October 28, 2009

Hunter (lab alumnist) travels to Copenhagen

As a workstudy student, Hunter was a valuable member of our laboratory this past year, especially where we were interfacing biology, engineering and bioinformatics. Having recently graduated from UTSA, he naturally headed oversees to get a broader world view. Of course, we're sure his next step should be graduate school - although that would be a loss to the music field, Hunter's other passion.

Sunday, October 18, 2009

Scientific Exchange with Mayo Clinic Jacksonville

We are grateful to Dr. Al Copland and colleagues for the opportunity to share our work on Overcoming Resistance to RTK Inhibitors in Rhabdomyosarcoma with the outstanding group of 8 faculty in the Cancer Basic Science Research program at Mayo Clinic - Jacksonville. This group of highly accomplished scientists has, in a short number of years, created a highly synergistic and productive research unit with some of the most interesting work in cell & molecular signaling and tumor microenvironment work in cancer research. Charles is especially appreciative of his colleague, Al, for this invitation to present our work and share ideas on addressing key issues in rare (and sometimes not so rare) cancers.

[ above right: Charles & Al ]

Saturday, October 17, 2009

Student Alumnist Grant Chen at UCSF

It is exciting to see that our past 2007 Summer Student intern, Grant Chen, is doing well now as a medical student on a sabbatical research year at UCSF conducting modeling of stroke and arterio-venous malformations (in mouse models). We are proud that Grant is following in the physician-scientist tradition!

[above right: Grant and his fiance ]

Friday, October 2, 2009

Medulloblastoma studies presented at PBTF Conference

Sachiko and Charles were excited to have presented our work, "Bortezomib inhibits Progression in Medulloblastoma" at the Pediatric Neuro-Oncology Basic & Translational Research Conference in Ashville, NC. This conference was jointly sponsored by the Pediatric Brain Tumor Foundation and the Society of NeuroOncology. We are grateful for this opportunity to have presented our work (our medulloblastoma model development was originally developed with a grant from the PBTF), and to exchange ideas with the other thought leaders in the field of childhood brain tumor research.

[above right: Sachiko with Dianne Traynor, Director of Research Funding and Advocacy of the PBTF ]

Thursday, October 1, 2009

Welcoming Xiaolan

We are excited to have Dr. Xiaolan Yi join our team as a research scientist. Xiaolan graduated from Hunan Medical University and worked as a physician for many years before coming to the U.S. to do research in 2000. Most recently Xiaolan was a valued member of the neuroscience laboratory of Dr. Lori Redmond in the Department of Pharmacology at the Medical College of Georgia. Xiaolan is interested in research that bridges clinical medicine and basic biomedical science... a great fit for our laboratory. Welcome, Xiaolan!

Wednesday, September 30, 2009

Childhood Cancer Research presented at Millennium Pharmaceuticals

Sachiko and Charles were privileged to be able to present our basic and therapeutic research at the R&D Grand Rounds at Millennium Pharmaceuticals this week. In our presentation, "Pediatric Tumors and the Millennium Pipeline" we were able to have a bidirectional dialogue on how drugs developed for adult cancer could be used to improve the treatment of cancer in children. We are grateful for the warm reception and genuinely insightful comments made by Millennium scientists.

[above right: Sachiko and Charles with Dr. Owen Lockerbie, Medical Science Liason - Oncology at Millennium Pharmaceuticals ]

Sunday, September 27, 2009

Congratulations Again, Suresh !

Our biomedical engineer team member, Suresh, seems to be on a roll this year. Today we received notice that his paper on the crimson carrier contrast agent for studying muscle injury & regeneration, as well as muscle cancer, has been accepted to Muscle and Nerve. Many thanks to co-author collaborators Carlo Martinez, Joel Michalek, Linda McManus, Brian Rubin and Paula Shireman, as well as Keller lab members Jinu Abraham and Mandy McCleish (emeritus).

It is worth noting that this is Suresh's 6th paper accepted or published in the last year, and his 3rd first-author paper in the same period. In addition, Suresh also has one other co-authored paper in review as well as a mature draft of another first-author paper in preparation. Congratulations, Suresh, your hard work is paying off.

Thursday, September 17, 2009

Barron presents at Imaging 2020

Congratulations to Barron who presented our lab's work on preclinical imaging of medulloblastoma at the Imaging 2020 meeting in Grand Teton, Wyoming. The highlight of the meeting might have been the keynote address of 2008 Nobel laureate, Roger Tsien, but overall the meeting was a very provocative and productive meeting of thought leaders in research and clinical imaging (for cancer, mainly). Barron did a terrific job presenting his work, and the questions and suggestions of the conference attendees were greatly appreciated.

Thursday, September 10, 2009

Congratulations, Suresh !

Many congratulations to Suresh on the publication of his paper in Circulation, entitled "Microscopic computed tomography-based virtual histology for visualization and morphometry of atherosclerosis in diabetic apolipoprotein e mutant mice". This was a joint project between Suresh and co-first author Hernan Martinez from the laboratory of Seema Ahuja at our university. As is our mantra, multi-disciplinary collaborations often lead to interesting studies and advances... this one in the field of heart disease.

[ above right: rendering of atherosclerotic plaque, made possible through freeware available from the Scientific Computing and Imaging Institute. ]

Friday, August 28, 2009

COG CNS Biology Committee Meeting in Bethesda

Yesterday and today the Children's Oncology Group Biology Committee met to discuss important new ways to help risk stratify patients with childhood brain tumors, as well as exciting potential new therapeutic approaches. We presented some of our work on targeting the ubiquitin-proteasome system in medulloblastoma. The highlight of the meeting, though, was the collaborative nature and the great scientific exchanges.

Tuesday, August 25, 2009

Welcoming Judy !

We are excited to welcome UTHSCSA graduate student, Chi-Yun 'Judy' Chang to our laboratory for her Fall rotation. Judy graduated with a BS in Biochemistry from The University of Texas at Austin. During college, she worked in the laboratory of Dr. John T. McDevitt, developing a particle based flow cytometric assay for CA-125 for comparison with the UT Nano-Biochip system. Judy hopes to be involved in research in with cancer in the future.

Friday, August 21, 2009

Aislynn receives Guarino Award

At Convocation today, Aislynn was acknowledged as the recipient of the 2009 Armand J. Guarino Award for Academic Excellence in Master of Science Studies. As Aislynn described in her acceptance speech, her project on childhood brain tumors (medulloblastoma) was a truly multi-disciplinary endeavor. Aislynn's strong communication skills and ability to bring in collaborators across diverse disciplines made her project possible. The second of two publications from her work will be submitted this coming week. We are so proud of what Aislynn has achieved. We wish her good luck and great success in Chicago!

[ above-right: Aislynn and her parents at the awards ceremony ]

Monday, August 10, 2009

Scott Carter Foundation BIG SHOW & Golf Classic

Koichi and Charles were privileged to participate in this year's annual Scott Carter Foundation fundraiser weekend, that included the "Big Show" and the Golf Tournament at the Indian Springs Country Club in Tulsa. Scott's Family and the Foundation's many selfless volunteers make ongoing important contributions of profound impact to childhood cancer research and advocacy. We appreciate all that they do!

Friday, July 31, 2009

Welcoming Courtney !

We are excited to have a new student research assistant in our lab, Courtney Kubicek. Courtney received her first Bachelor's degree in Biomedical Science from Texas A&M in May of 2009. While at Texas A&M she was an executive officer of the safe ride program CARPOOL, which pioneered and set the standard for safe ride programs nationwide. Currently Courtney is a nursing student at the UTHSCSA, and she will work part-time assisting our PPTI program (drug testing for childhood cancers using genetically-engineered mouse models). Courtney has a long term goal of obtaining my Master's in Anesthesiology.

Thursday, July 30, 2009

Congratulations to Corrine, a TST Scholar !

Many congratulations to Corrine on being selected by the Institute for Integration of Medicine and Science as Translational Science Training (TST) Program Scholar for 2009/2010! This competitive CTSA program, and Corrine was selected as one of 6 recipients by a 16-member interdisciplinary committee. The award encompasses a stipend, travel to a national meeting and funds for research. We are all so proud of Corrine's accomplishment!

Friday, July 24, 2009

Sarcoma Awareness Fun Run/ Walk & Social a Success !

Many heartfelt thanks to Melinda Garcia and Corrine Chua for the wonderful planning that made for today's very successful "Team Sarcoma San Antonio" event. We met at Pavilion 3 in McAllister Park where we had a fun run/walk followed by a cook-out and social. “Sarcoma Knows No Borders” bracelets benefiting the Liddy Shriver Sarcoma Initiative went quickly. We were especially pleased that this year's event was well shaded and nearby a great playground for the children of participants.

For other recent Sarcoma Awareness events across Texas, click here.

To send your comments about this year's event and suggestions for next year's, feel free to email Melinda at

pictured: Team Sarcoma San Antonio (TeSSA) 2009 event!

Thursday, July 23, 2009

Drug Development Conference

The Cancer Molecular Therapeutics Research Association (CMTRA) annual meeting was held at Stanford University from July 19 -22. This is an interesting intersection of academics and the pharmaceutical industry whereby confidential data presentations allow free and open intellectual exchanges about the science and implementation of drug development and clinical trials. For those interested in molecular therapeutics, this meeting is a true gem. From our lab, Jinu's work on IGF receptor inhibition was very well received.

[ pictured top right: an artwork on the Stanford campus by Auguste Rodin, entitled The Gates of Hell. After all, it was a cancer meeting. ]

Wednesday, July 15, 2009

Our lab featured as UTHSCSA 'Research Stars'

We are grateful for the acknowledgement of our lab's mission in the Summer 2009 UTHSCSA publication entitled, "The reSearch is On: Research-Discover-Cure" emphasizing the importance of the bench to bedside research paradigm. Vice President for Research, Dr. Brian Herman, was a critical reason we endeavored to start our research enterprise at the newly formed Greehey Children's Cancer Research Institute, and with his support we are able to move closer to fulfilling our mission every day.

Tuesday, July 7, 2009

Congratulations to Navaline!

Many congratulations to our collaborators Navaline Quach and her mentor, Tom Rando, at Stanford on their publication, "Focal Adhesion Kinase Signaling Regulates the Expression of Caveolin 3 and beta1 Integrin, Genes Essential for Normal Myoblast Fusion" in Molecular Biology of the Cell. Our lab appreciates the opportunity to have contributed to this study, which dissects the role of focal adhesion kinase in myoblast fusion and differentiation.

Wednesday, July 1, 2009

Pediatric Drug Development website Officially Launched!

The Pediatric Preclinical Testing Initiative at the Greehey Children's Cancer Research Institute has officially launched its new website, . We're excited about this program, which uses genetically-engineered mice to validate novel molecularly-targeted therapies for use in children with cancer.

We sincerely thank our early and current sponsors, including a kind anonymous donor, the Joanna MacAfee Foundation and the Rally Foundation.

Monday, June 22, 2009

Welcoming Steven

Summer intern Steven McCarthy is a welcome new addition the our pediatric cancer research team. Steven is an undergraduate at Florida State University (Tallahassee) where he's majoring in Biomedical Engineering with minors in physics and biology. Steven is also captain for the FSU Men's Water Polo team and recently joined the Biomedical Engineering Society, a prestigious distinction in his field of study.

Cross-disciplinary contributors like Steven make new approaches possible. Welcome, Steven!

Thursday, June 11, 2009

upcoming Sarcoma Awareness Fun Run/ Walk & Social !

When: Friday, July 24, at 6:00 pm
Where: McAllister Park (13102 Jones Maltsberger Rd), San Antonio, Texas

This is a free event open to all. The fun run/walk will begin at 6:00pm at Pavilion 3 in McAllister Park. At 6:30pm everyone will meet up at the pavilion where we’ll have a cook-out and social until 8pm. “Sarcoma Knows No Borders” bracelets benefiting the Liddy Shriver Sarcoma Initiative will be available at $5 each for our participants on a first come first serve basis.

Many thanks to this year's organizer, Melinda Garcia! For more details* or to RSVP, email .

shown above-right: Team Sarcoma San Antonio (TeSSA) at the 2008 event!
[ for other Sarcoma Awareness events across Texas, click here. ]

* This community event is intended for sarcoma awareness and not specifically a fundraiser or a function of the UTHSCSA.

Wednesday, June 10, 2009

Interesting Rhabdomyosarcoma Study

In the June issue of The Journal of Clinical Investigation, Durbin and colleagues from the laboratory of David Malkin presented an interesting distinction between the biologies of embryonal and alveolar rhabdomyosarcoma. This distinction centers on the Integrin-linked Kinase signaling pathway. For the original article, click here. For the commentary by Charles and co-authors Paul McDonald and Shoukat Dedhar, click here.

Sunday, June 7, 2009

Welcoming Lena

Elena Wisely will be joining our research team at GCCRI for her 2009 Summer rotation. Lena is an incoming MD/PhD student at UTHSCSA and plans to pursue translational biomedical research. She is a graduate of Texas State University in San Marcos, where she spent more than 2 years in Dr. Joseph Koke's lab investigating the role of the FOXP2 transcription factor responsible for regulating human language development and acquisition. Lena, welcome to our team!

Friday, May 22, 2009

Aislynn Graduates!

We couldn't be more proud of Aislynn Samano who graduated today with a Masters Degree from UTHSCSA. Aislynn's thesis encompassed a multi-disciplinary approach to evaluating drug efficacy in genetically-engineered mouse models of medulloblastoma. The results of her study are especially important to academic and pharmaceutical approaches to preclinical therapeutics. Important contributors included lab members Eri Taniguchi (alumnist now), Sachiko Ohshima and Aoife Kilcoyne as well as imaging collaborators Osamu Togao, Ai-Ling Lin, Duff Davis, and Tim Duong and engineering collaborators Brent Nowak and Thomas Whitney. We plan to submit a manuscript on Aislynn's exciting work later this month. Congratulations, Aislynn!

[ Update (7/15/09): Exciting News! Aislynn has been selected as the recipient of the 2009 Armand J. Guarino Award for Academic Excellence in Master of Science Studies !! ]

Tuesday, May 12, 2009

Koichi featured by ALSF News

Koichi has been featured in the "newsroom" of the Alex's Lemonade Stand Foundation website as a past recipient of an ALSF career development award. To see the ALSF news spotlight, click here.

Monday, May 11, 2009

Sachiko Wins Poster Presentation

Congratulations to Sachiko, who won 1st Place in the poster competition for the 7th Annual Center for Biomedical Neuroscience Retreat today. Sachiko's poster explores the role of receptor proteins as therapeutic targets in metastatic medulloblastoma. way to go, Sachiko!

Friday, May 1, 2009

Welcoming Ashwathi!

Ashwathi Mohan will be joining our research team as a GCCRI 2009 Summer intern. Ashwathi is currently an undergraduate student at Texas A&M University and is a graduate of the Northside Health Careers High School in San Antonio. After completing her Molecular and Cell Biology Major and a Classics (Latin) minor, she plans on pursuing a combined M.D. and Ph.D. Her goal is to both practice medicine and continue biomedical research.

[ 06/09/2009: Congratulations to Ashwathi, who was recently selected as a University Scholar scholarship from Texas A&M University. This prestigious award is based on academic achievement and the intent to pursue postgraduate studies. ]

Sunday, April 19, 2009

Strategic Planning Feature in NCURA

Thanks to our colleague Cory Hallam, our laboratory retreat last month has been featured in a essay and case study on Bringing Strategic Planning to the Research Lab for the National Council of University Research Administrator’s magazine (as a “Feature”). The article is written by Cory (Assistant Vice President of Commercialization at UTSA) and co-authored by Charles. The strategy, however, is a true lab team effort. To see the NCURA Magazine feature, click here and go to page 42.

Tuesday, April 7, 2009

KENS TV Coverage (muscle stem cells)

We sincerely thank Wendy Rigby of KENS Channel 5 for her coverage of our recent muscle stem cell publication. It was thoughtful of Wendy and her camerman, Richard, to have included so much of our team in the story. To see the local video, click here. For the CBS national link, click here.

[ to right: Mandy with mus musculus (a house mouse) ]

Monday, April 6, 2009

Lor Randall visits GCCRI

It was our pleasure to host our colleague and collaborator, Dr. Lor Randall, who visited UTHSCSA to give an Orthopaedic Surgery Grand Rounds presentation entitled, "Sarcomatology: A Transdisciplinary Approach". An orthopedic oncologist and researcher himself, Lor is the Director of the Huntsman Cancer Institute Sarcoma Service - and our frequent collaborator and good friend. We appreciate his time to visit with us to share his recent work.

Friday, April 3, 2009

Rally Foundation funds PPTI Study

We are grateful to the Rally Foundation for a recent grant in the amount of $17,076 which will fund testing of one novel therapeutic compound for medulloblastoma and another for rhabdomyosarcoma in our genetically-engineered mouse models. The Rally Foundation for Childhood Cancer Research is a non-profit organization, which through volunteers across the country, aims to raise awareness and funds specifically for childhood cancer research to find better treatments and cures for childhood cancers.

These awards fromt he Rally Foundation are in honor of Matthew Butterfield and in memory of Coleman Larson.

[ This blog entry is also mirrored at the Rally Foundation blog site. ]

Tuesday, March 31, 2009

Koichi's paper to be Highlighted in Cancer Research

We've just been informed that Koichi's paper has been selected for the Highlights section of the upcoming issue of Cancer Research. Congratulations, Koichi!

Friday, March 27, 2009

Tuesday, March 24, 2009

Bench to Bedside

We're excited to share that Charles has been appointed as a full time member of the Children's Oncology Group (COG) Soft Tissue Sarcoma Committee. This committee designs clinical and translational trials for rhabdomyosarcoma and other soft tissue sarcomas. This appointment follows other COG activities in recent years including ad hoc participation in the Soft Tissue Sarcoma Committee since 2001. Also, Charles has also been an awardee of the COG Young Investigator Award (2001) and a recipient of the COG Translational Research Award (2001-2002). The results of the latter are published as a COG report entitled, "Rhabdomyosarcomas utilize developmental, myogenic growth factors for disease advantage". This study was the foundation upon which our more recent from-the-bench-towards-bedside efforts have been focused. Our lab looks forward to being able to contribute more to clinical and translational trial design as new, molecularly-targeted drugs become available to pediatric patients.

Collaborator Andrew Brack visits GCCRI

Friend and collaborator, Dr. Andrew S. Brack, gave a terrific seminar presentation at GCCRI today. Andrew is an Assistant Professor at the Center for Regenerative Medicine at MGH, as well as a member of the Harvard Stem Cell Institute. A recent trainee of our other valued colleague, Dr. Thomas A. Rando at Stanford, Andrew has established a productive independent laboratory of his own and is a young rising star in the muscle biology field. As ever, the cross-pollination between muscle cancer biologists and muscle biologists leads to interesting conversations, novel ideas, and new outlooks on ways to treat muscle cancer. We are grateful to Andrew for making the trip to San Antonio and giving such a great seminar on satellite cell (muscle stem cell) renewal.

[ PICTURED: Andrew (left) with Koichi (right)
at the Alamo ]

Thursday, March 5, 2009

Koichi and Tohru's paper accepted to The FASEB Journal

Congratulation to Koichi and Tohru as first and second author on a muscle stem cell physiology / rhabdomyosarcoma study that was accepted today to The FASEB Journal. Other lab members who co-authored this work include Bev, Suresh and Mandy - as well as lab alumnist Ali Bahadur. We are grateful for the contributions of our collaborators, especially Chris Bjornson who is a member of the laboratory of Thomas Rando and our pathologist collaborator, Brian Rubin.  We are grateful, too, to the Scott Carter Foundation for sponsoring Koichi's fellowship.  

[ pictured on above right: a green myotube and its blue nucleus with an adjacent pink-white muscle stem cell (satellite cell). Pictured below, Koichi (left) and Tohru (right). ]

[ Koichi & Tohru's paper is now online. ]

Wednesday, March 4, 2009

Imran awarded AMA Seed Grant

Congratulations to Imran, who has been awarded a 2009 Medical Student Seed Grant from the American Medical Association Foundation. Imran's project will investigate the role of interleukin receptors in rhabdomyosarcoma biology and therapeutic strategies. This is a great start to Imran's project as a candidate for the M.D. with Distinction in Research Program at our medical school. We're very impressed with the trajectory of this future physician-scientist.

[ pictured at right: Imran hard at work in the lab following a set of medical school exams. ]

[ update 05/22/09: Imran has been accepted to the MD with Distinction program at UTHSCSA! Congratulations, Imran! ]

Saturday, February 28, 2009

an informal word on the research in the Keller Laboratory

Our laboratory focuses on long term and near-term treatments for the childhood muscle cancer, rhabdomyosarcoma, and the childhood brain tumor, medulloblastoma. To say long term, we mean basic science investigation of how these tumors work – such things as what kind of normal cell gives rise to one particular kind of cancer. And in saying near-term, we mean finding molecules in these cancers to directly turn off or turn on by drugs so that the tumor stops growing. Behind both approaches are some rather exciting genetically-engineered mice… modified from before birth so that at a certain age, and in a certain tissue, the same mutations found in a child’s cancer are activated in the mouse. Then the tumor can be followed to see how it grows and spreads… even to test a treatment to see whether the tumor growth can be reversed. That these mice have normal immune systems is a real plus, too, because white blood cells play an important role in how tumors evolve and respond to therapy.

While this use of mouse models makes our lab slightly unique, our greatest asset is that we have a very multi-disciplinary team. Biomedical engineers for building and operating imaging and diagnostic instruments, biochemists for understanding the molecules, molecular biologists and electrical engineers for understanding how tumors express genes in certain ways, an orthopedic oncologist and me (the board-certified pediatric oncologist) helping bring it all together in a focused way. Sometimes we venture beyond the ordinary, such as the use of 25¢ fertilized chicken eggs (instead of mice or petri dish experiments) to study anti-cancer drugs. That last project is one graciously sponsored by the Alex’s Lemonade Stand Foundation. We’re even starting to work with the National Cancer Institute’s Pediatric Preclinical Testing Program to try exciting new drugs right out of the pharmaceutical development pipeline in our novel mice.

I’d like to think that tangibly better treatments for rhabdomyosarcoma and medulloblastoma can be found in a matter of years, instead of tens of years. I go to a fundraiser golf tournament for the Scott Carter Foundation every year. They sponsored my research training in Mario Capecchi’s lab years ago, and now sponsor my postdoctoral fellow, Koichi Nishijo. Standing at the 18th hole every year, I get the same question about every 5 minutes, “Doctor, do you have anything new for these kids yet?” These questions led my lab to put a heavy emphasis on therapeutics about 3 years ago. As a result, we’re finding drug targets, as well as drugs to hit those targets that are less inclined to result in relapse for our patients (ok – yes, it’s just mice so far, but we did present our results at a Children’s Oncology Group symposium a few weeks ago to help select a non-chemotherapy drug to add to the next COG trial for rhabdomyosarcoma).

We’re fortunate that despite the economic downturns that we’re doing pretty well so far… R01 funding from the National Cancer Institute, grants from the ALSF, the St Baldrick’s Foundation, the National Brain Tumor Society, the Joanna McAfee Foundation and more. Seven publications in print or accepted in 2008. This are truly exciting times. On top of all this is our mouse drug testing program mentioned above, which is open to public participation (for more details, see the left column of our blog). And just to put a plug in for a colleague’s new lab, try visiting Dr. David Langenau’s website . David has made a zebrafish model of embryonal rhabdomyosarcoma. He treats these little fish with drugs, right in the tank water. Also see Dr. Rene Galindo's website on using fruitflies to understand rhabdomyosarcoma. It will probably take a lot of new approaches and cooperation between groups to make the big impact we’re all hoping for, but it can be done.

Where there is a will, there is a way. Change can be tangible.

Charles Keller, MD

[ many thanks to collaborator Dr. Bryan W. Jones who took this picture on a hike in Utah. ]

Friday, February 27, 2009

GCCRI Symposium

In celebration of the 5th anniversary of the GCCRI we held a symposium on the Genetics and Biology of Childhood Cancer at the Hotel Contessa February 26 - 27th. Our founding director Dr. Sharon Murphy also attended (Sharon is now a Scholar-in-Residence at the Institute of Medicine). The Keller lab hosted the session on disease models and preclinical studies. Speakers in this session included Tom Look (DFCI) who gave a great talk on ALK that follows his Fall report in Nature, Rene Galindo spoke on drosophila models of alveolar rhabdomyosarcoma, Pat Reynolds talked about exciting new retinoid therapies for neuroblastoma and leukemia, and Peter Houghton reviewed the tremendous progress by the Pediatric Preclinical Testing Program. We can't thank the speakers enough for making this an exciting, interactive exchange.