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Alex's Lemonade Stand Foundation Blog

ALSF Innovation grantee Darrell Yamashiro works in the lab at Columbia University Medical Center. Dr. Yamashiro works with Dr. Julia Bender-Glade (mentioned below) developing therapies for relapsed childhood cancer. 

by Trish Adkins

Gone are the days of just chemotherapy and radiation. Today’s researchers and oncologists are combining the traditional tools with cutting-edge biological medicine, genetic analysis and novel therapeutics in the labs and clinics. 

Childhood cancer researchers are working hard towards better treatments and more cures every single day. Here are five trends in research today:

1. Treating the patient, not just the cancer

For over 40 years, scientists have known about oncogenes, the abnormal genes that can drive the growth of abnormal cells that become cancer. Now, childhood cancer researchers are discovering more about oncogenes and finding new ways to stop the development of cancer by targeting these “bad” genes. Researchers are also studying the biology of children with cancer to determine the best way to treat cancer within their body. 

“Every cancer is different, because every child is different,” said Dr. Julia Glade-Bender, a member of the ALSF Scientific Advisory Board and an oncologist at New York-Presbyterian Hospital/Morgan Stanley Children’s Hospital. Dr. Glade-Bender directs the Developmental Therapeutics Program at NYP, which works to identify the molecular drivers of each child’s cancer. Researchers will then use that information to personalize treatment using novel, biologically targeted agents during clinical trials.  

2. CAR T cell immunotherapy 

In August 2017, the FDA approved CAR (Chimeric Antigen Receptor) T cell immunotherapy as a treatment for certain types of acute lymphoblastic leukemia (ALL). This is the first gene therapy to achieve FDA approval in the United States and one of the few major breakthroughs and approvals for pediatric oncology in recent years. 

Like other types of immunotherapy, CAR T cell therapy harnesses a patient’s immune system to fight and destroy cancer cells. The FDA approval does not just bring hope for children battling relapsed ALL; it opens the door for more innovative research for all types of childhood cancer.

“The anticipation is that this is the first step towards changing the landscape of how we treat pediatric leukemia. The vision is that ultimately, we can remove a lot of the standard chemotherapy that we use and replace it with CAR T cell therapy. It’s not something that is going to happen overnight, but I truly believe it is where we will end up,” said Dr. Rebecca Gardner, a physician at Fred Hutchinson Cancer Research Center and ALSF Young Investigator Grantee. 

Dr. Gardner recently led a clinical trial using CAR T cell therapy as a treatment for relapsed leukemia. In her trial, 93% of patients reached remission, after struggling to reach remission using traditional leukemia treatments. 

3. Big data for big cures

Collaboration in the pediatric oncology research community has driven science closer to cures; however, with millions of disconnected data points, critical information is often filed and forgotten.

The Childhood Cancer Data Lab, powered by ALSF, is building a data refinery. This is a central location to collect, harmonize, analyze and share childhood cancer data. The data refinery will provide access to the entire field of pediatric oncology.

Researchers will quickly connect their findings to the findings of other colleagues from around the world. This allows them to identify common patterns and apply these hidden connections toward the development of new therapies to accelerate cures for children.

The data refinery is set to launch in beta in March 2018.

4. Engineering cures 

ALSF Young Investigator Grantee, Dr. Steven Jonas from UCLA is developing tools to make the delivery of treatments like CAR T cell immunotherapy, quicker and more cost-effective.  

His research project focuses on developing and applying microfluidics—the technology that makes things like ink-jet printers work—to speed up the process of altering individual children’s T cells into the immunotherapy drugs that could ultimately cure their cancer. 

Researchers are also using nanotechnology to support immunotherapy treatments. Dr. Christopher Jewel, ALSF ‘A’ Award Grantee from the University of Maryland, studied the use of nanoparticles—small biodegradable particles—in conjunction with an experimental cancer vaccine for neuroblastoma. The nanoparticles are loaded with signals that stimulate the body’s immune system to fight cancer. 

5. Harnessing viruses to kill cancer

Researchers have long known that cancer cells are hiding from the body’s immune system. In addition to uncovering these cells through immunotherapy, researchers are also using viruses to infect cancer cells and trick the immune system into killing cancer cells. Called oncolytic virotherapy, this cutting-edge research uses altered versions of viruses like HIV, polio and herpes to fight leukemia, brain tumors and other solid cell tumors.  

ALSF Reach Grantees, Drs. Michael Burke and Jeffrey Medin, from the Children’s Hospital of Wisconsin, are using a virus to treat acute myeloid leukemia (AML).  Drs. Burke and Medin take a small number of leukemia cells from a patient and infect those cells with a virus outside of the body. The virus then tricks leukemia cells into making a protein called interleukin 12 (IL-12). When IL-12 cells are returned to the patient’s body, their immune system sees the infected cells and begins attacking the leukemia cells. 

The ALSF Grants Program is designed to fill critical voids in pediatric cancer research. ALSF works to fund all phases of research through 13 different grant programs. Each potential project is given careful consideration and reviewed by a team of leading scientists and clinicians. The result: ALSF is able to power breakthroughs and move closer to cures for childhood cancer.  Learn more about the ALSF grants program here

 

Categories: 
Research
Instead of hibernating on these cold winter days, warm yourself up and help Alex’s Lemonade Stand Foundation (ALSF) fight childhood cancer. From swapping lemonade for hot chocolate to using your winter sport to fundraise, there are so many great ways you can fund childhood cancer research!

Instead of hibernating on these cold winter days, warm yourself up and help Alex’s Lemonade Stand Foundation (ALSF) fight childhood cancer.  From swapping lemonade for hot chocolate to using your winter sport to fundraise, there are so many great ways you can fund childhood cancer research! 

1. Swap Lemonade for Hot Chocolate

ALSF supporter and childhood cancer hero Matthew hosted “Matthew’s Miraculous Hot Coco Stand for Pediatric Cancer Research.” Matthew, who was diagnosed with a rare kidney cancer in 2014, took his stand to a local park. The result was over $8,000 raised!  You can be like Matthew and host a hot chocolate stand at your local park, in your front yard or even in conjunction with a local business. The ALSF stand kit has everything you need to get started, whether the temperatures are low or high. Sign up here

2. Host a Photo Challenge

Started in memory of Sadie Hulsey, who died from juvenile myelomonocytic leukemia (JMML), the Childhood Cancer Christmas Tree Challenge invites everyone to mimic one of Sadie’s mom’s favorite holiday photo. In the picture, Sadie is wearing her holiday best and her younger sister, Lanier is napping under the tree. Supporters can recreate the picture themselves (with some hilarious results!), share it and donate via the event’s fundraising page. 

Anyone can host a similar challenge. Simply pick one of your favorite, slightly silly photos, start a fundraising page on the ALSF website and ask supporters to share their copycat photos on social media along with your fundraising page link. 

3. Become a Champion

Whether you are dunking basketballs, a goalie making saves, a wrestler tacking pins or gearing up for spring sports, you can become a Champion for Kids with Cancer! Throughout the season, your athletic goals can also be fundraising goals. Through a personalized fundraising web page, your supporters can make a one-time donation or pledge a dollar amount per goal, save or accomplishment. 

It is never too late in the season to sign-up to be a Champion.

4. Host a Cook-Off

Gather all your friends and neighbors for a cook-off or bake-off. Ask for a donation entry-fee for all cooks and have non-cooking guests make a donation to vote for their favorite dishes. No idea what to cook? Grab a copy of Alex’s Table, the ALSF cookbook, packed with great recipes from chefs all over the world. 

5. Get Social with Facebook Fundraising

Facebook makes it easy to fundraise! Head to the ALSF Facebook page (www.facebook.com/alexslemonade) and select “create” to start a Facebook fundraiser!   Then, invite your network to donate. You can host your Facebook fundraiser in honor of your birthday, anniversary or life event. You can also host one just because you feel inspired to help kids with cancer.  

6. Snowman Building Competition

Gather all your neighborhood snow friends and builders for a snowman building competition! Have participants make a donation to compete. The spectators can vote on who has the best snow creation by putting a small donation in the bucket of their favorite snowman. In the end, whichever snowman has the most donations, wins!  (Insider tip: order some ALSF gear to decorate your snow creation from Alex’s Shop!) 

No snow where you are? Turn your snowman competition into a sand castle competition (and of course serve lemonade!) 

7. Dinner Party

Give all your friends and neighbors a reason to skip making dinner one night! Call a local restaurant and ask to host a fundraising night benefiting ALSF. Whether a chain restaurant or a locally owned dinner spot, most restaurants have options for guests to dine and donate! 

Need more ideas? Well, we have 25 ways you can support ALSF and fight childhood cancer all winter long. Just head here

Categories: 
Fundraising Ideas
Over 700 new children are affected by cancer every day. For these kids, years are too long to wait. To help researchers get to the clinical trial phase quicker, ALSF established the Reach grants. The program, which awards three multi-year grants annually, accelerates researchers closer to clinical trial.

The process of bringing an idea from the lab to clinical trial can take years. Researchers are not only required to prove the effectiveness of their science, they also need to get FDA approval to provide an experimental therapy to actual patients. On top of all this, researchers also must ensure that their home hospital has the correct infrastructure in place to administer a clinical trial.

Over 700 new children are affected by cancer every day. For these kids, years are too long to wait. To help researchers get to the clinical trial phase quicker, ALSF established the Reach grants. The program, which awards multi-year grants annually, accelerates researchers closer to clinical trial. 

This year’s grantees are embracing cutting-edge trends in cancer research and working towards cures for kids battling some of the deadliest cancers. Meet our 2017 Reach grantees:

1. Dr. Gianpietro Dotti and Dr. Barbara Savoldo, University of North Carolina 

In August, the FDA approved CAR T cell immunotherapy as a standard of care for some types of relapsed leukemia. T cells, an essential part of the immune system, are collected from patients and then modified, so when they are returned to the body, they find leukemia cells and kill them. 

However, the potential of CAR T cell immunotherapy as a cure for different types of cancer does not stop there. CAR T cells have also been shown to kill glioblastoma (GBM) cells in the lab.

Dr. Dotti and Dr. Savoldo are developing a similar strategy as a potential treatment for GBM, a type of brain tumor that is notoriously hard to eliminate surgically or with chemotherapy and radiation. 

Dr. Dotti’s team has found that CAR T cells kill the majority of GBM tumor cells, but some GBM cells survive by blocking the altered T cell. Now, the team is working to make the CAR T cells unable to be blocked by GBM cells. The hope is this research will lead to a major breakthrough for children battling GBM, providing a treatment option with minimal side effects. 

Dr. Dotti’s work is co-funded with Bear Necessities Pediatric Cancer Foundation.

2. Dr. Michael Burke and  Dr. Jeffrey Medin, Children’s Hospital of Wisconsin

Acute myeloid leukemia (AML) is the second most common type of pediatric leukemia. AML progresses quickly and about half of all children relapse. Relapsed AML has a dismal survival rate—the need for treatments is urgent. 

Dr. Burke and Dr. Medin are studying the use of oncolytic virotherapy as a treatment for AML. Oncolytic virotherapy uses altered virus cells that are injected into cancer cells. The infected cancer cells can be seen by the immune system, which then goes to work to kill cancer and rid the body of infection. 

Dr. Burke and Dr. Medin take a small number of leukemia cells from a patient and infect those cells with a virus outside of the body. The virus then tricks leukemia cells into making a protein called interleukin 12 (IL-12). When IL-12 cells are returned to the patient’s body, their immune system sees the infected cells and begins attacking the leukemia cells. 

A similar approach has been successfully used with adults battling relapsed AML. If successful, this approach would not only provide a treatment option to children with relapsed disease, but also provide an alternative to intensive chemotherapy and the side effects associated with current AML treatment. 

3. Dr. Alex Kentsis, Memorial Sloan-Kettering Cancer Center

The causes of genetic mutations that lead to the development of cancer are still poorly understood. This is particularly true for solid tumors of children and young adults that express the PGBD5 mutation. This mutation is expressed in several solid tumors, including rhabdoid tumors, lethal tumors that can develop in any part of the body including the lungs, kidneys, brain, liver and soft tissues. 

Dr. Kentsis recently identified that the PGBD5 mutation causes the genes to rearrange and leads to the development of cancer. This phenomenon is called genomic rearrangement and is common in a majority of childhood solid tumors. Dr. Kentsis hopes that his study will lead to the development of a new therapeutic strategy to inhibit the rearrangement and move directly to the clinical trial phase, providing improved therapeutic options for children battling solid tumors. 

ALSF began the Reach grant program in 2013. Read more about our Reach grant projects here

 

Categories: 
Research

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