Pediatric Blood Cancer Initiatives

Dr. Gwen Nichols

As LLS’s chief medical officer (CMO), Gwen Nichols, M.D., plays a critical role in advancing cures through a unique combination of clinical, academic and pharmaceutical experience. She oversees LLS’s scientific research portfolio, patient services and policy and advocacy initiatives. A physician and scientific researcher, she has dedicated her career to advancing cures for cancers.

Most recently, Dr. Nichols was oncology site head of the Roche Translational Clinical Research Center, where she worked to develop new cancer therapies, translating them from the laboratory to clinical trials. Prior to joining Roche in 2007, Dr. Nichols was at Columbia University for more than ten years, where she served as the director of the Hematologic Malignancies Program.

Dr. Nichols trained in internal medicine at the University of Chicago and completed post-doctoral research and a hematology-oncology fellowship at Memorial Sloan-Kettering Cancer Center where she was an attending physician. She received her medical degree with honors from the State University of New York at Buffalo.

Dr. Nichols:

• LLS started 70 years ago by a family who lost their child to leukemia. In the 1940s, patients were limited to very few treatment options. We have kept true to that mission of finding new treatments to  blood cancers. LLS has a rigorous research program, and we have realized our advances have been few for the Pediatric population. Thankfully, childhood cancers are rare, but it doesn’t mean they shouldn’t have access to the newest therapies. So, we decided to create the LLS Children’s Initiative to try to increase research within the pediatric sphere. We wanted to advocate and create resources for the children and families affected by blood cancer. 
• Pediatrics is not a big market, and so it is rarely the choice for a drug developer to bring a new molecule forward. We must allow the patient and the doctor the choice to select multiple methods of treatment. We are now selecting multiple developers for a Master Trial. Our goal is to allow patients and doctors more choices when relapse occurs. And we believe the less toxic nature of these new therapies will allow new options to even eradicate the need for Bone Marrow Transplants. 

Dr. Kevin Curran

Dr. Curran received his medical degree from Georgetown University.  His residency was at Tufts-New England Medical Center. His fellowship was in Pediatric Hematology/Oncology at Memorial Sloan Kettering Cancer Center.

Dr. Curran is a pediatric oncologist who specializes in bone marrow transplantation. He treats children and young adults who have hematologic malignancies such as leukemia, disorders of the bone marrow that impair the ability to make normal blood cells, and disorders of the immune system that impair the body’s ability to fight infection.

His research focus is on the development of novel treatment approaches for leukemia and lymphoma that do not respond to current therapies. Specifically, He and his colleagues use genetic manipulation of immune cells to recognize and kill cancer cells. This is a promising form of gene therapy and is currently being tested in clinical trials at Memorial Sloan Kettering Cancer Center for children and adults with high-risk or relapsed hematologic cancers. Through these are highly innovative studies, we hope to provide a novel and successful treatment option for all patients with cancer.

Dr. Curran:

• Thank you so much for the opportunity to talk about Pediatric Cancer. Really, I’m going to echo a lot of things Dr. Nichols has been saying. Like Dr. Nichols mentioned, it is a rare disease, but it is not a rare disease to the patients and their families. The beautiful thing about pediatric cancer is we have come a long way. However, many of our available treatments can be toxic, but our goal is to create less toxic treatments with better long-term outcomes. Yet, we still have a ways to go, especially in reducing the effects of GVHD. 
• My real focus is to eliminate bone marrow transplants. That is the goal of my career. Now, we have a couple trials going for less-toxic treatments. Car-T cells pull out the T-cells and use genetic modification to infuse back into the patient to eradicate the cancer. We are hoping the cell therapy field will expand beyond blood cancers and help eliminate other cancers as well. But now, we need to focus on, “how do we get it to the patient”?  

Michelle Marks

Michelle W. Marks, lives in Denver, Colorado with her husband Rich, 14-year old daughter Lainey and 13-year old son Quade.  Michelle earned a Bachelor of Science degree from University of Colorado at Boulder and a Master of Business Administration from University of Denver.  She has spent close to 25-years within the medical device industry developing and commercializing innovative medical treatments.  Currently she is Director of Global Marketing for Terumo BCT, a global leader in blood management, blood safety, therapeutic apheresis, cell therapy and cell collections.

In April 2016, Michelle and her husband, Rich, were presented with devastating news their then 9-year old son, Quade, was diagnosed with Acute Myeloid Leukemia (AML) with monosomy-7, a deadly type of blood cancer not common in pediatrics.  Quade was given 4-8 weeks to live without treatment.  After months of inpatient hospitalization, four rounds of intensive chemotherapy, countless blood transfusions (all of which were collected on equipment developed by Michelle’s employer Terumo BCT), and a stem cell transplant, Quade is now cancer free!

With strong passion for advancing medical research and proven business leadership, Michelle is a strong advocate for advancing cancer research, ensuring access to affordable care and innovative therapies, and improving the lives of cancer patients and their families.

Michelle:

• I am a mom to a pediatric cancer survivor. It all started on April 15, 2016. I woke up in Tokyo, but I ended it with a Pediatric Oncologist in Denver. I wasn’t thinking about all of the bumps and bruises on Quade’s legs because he was an active kid. He was playing sports and was always getting into the action of things. However, I was called in to Quade’s school after he had complained to the school nurse that he had a swollen hand and a strange rash. Shortly after taking him to the doctor, we were told we needed to go to the emergency room. After countless tests and a long overnight visit, Quade received his diagnosis. What followed was three rounds of chemo and a stem cell transplant. We learned how to live in the hospital. We experienced the inevitable ups and downs of childhood cancer. But yes, we beat it and we are now in the category of survivors. We are joyful that the leukemia hasn’t returned, but there is this nagging fear of long-term complications or even relapse. One of the biggest misconceptions of pediatric cancer is that once treatment is over, it’s over. But only 1 in every four treatment survivors lives past five years of the end of treatment. However, I am filled with hope after hearing about these new possibilities spoken about today. Today, Quade continues to play lacrosse, snowboard, and stay active however he can. 
• You know, the diagnosis leaves scars. We have now learned to let the petty stuff go. We know we can’t control tomorrow, but we appreciate what we have today. So here’s my advice: Use your resources. Educate yourself. Advocate for your child. Find the best medical team. Don’t be afraid to ask for help. Keep a positive attitude and try to laugh. Vent when you need to. And finally, have hope that there is incredible innovation on the horizon.

Questions

Question 1: What would you tell someone just starting the cancer diagnosis journey?

Michelle: Know your resources, don’t be afraid to ask questions. Connect with people who’ve been through it.

Question 2: What do you think about supplemental therapies?

Dr. Curran: If you are a parent, you know your child best. Do what you can to ease their journey. We welcome a lot of things to help decrease side effects. There are websites and journals you can find that will tell you if the supplement you are curious about has any interactions with chemotherapy.

Question 3: If families themselves want to look at clinical trials, do you have a resource for families to look at research?

Dr. Nichols: LLS has a clinical research source to help you learn more about trials, whether or not they are open, etc. Call LLS and ask to talk to a clinical trial navigation nurse about trials appropriate for the patient. They can match you with a list of available, open, and appropriate trials to take back to your own doctor to discuss.

Question 4: How did you choose a pediatric center?

Michelle: We met with the two hospitals available to see which medical team felt like a good fit for us. I always encourage patients and caregivers to seek the best fit for them.

Question 5: Michelle, You mentioned a 504 plan. Did you have to organize this, or did someone at the school initiate it?

Michelle:

We had to initiate it ourselves. This all felt like a second full-time job, learning and advocating for our son, but it’s imperative to educate yourself and seek options for your child.

Question 6: For an ALL patient, what do you do about swelling?

Dr. Curran: If his kidneys and heart are functioning well, then it’s okay. If it’s clearly not an allergic reaction, that’s okay too. But if you are unhappy with your answer, you can get a second opinion from another physician. You are your child’s best advocate.

Question 7: How do you ease anxiety in children?

Michelle: Our whole family was in therapy while Quaid was in treatment and afterwards. Talking about it really helped. Venting and finding ways to relax was crucial. For kids, art, or athletics could help decrease anxiety. There are many resources available, but don’t be afraid to experiment and find what works for your family.

Dr. Curran: You have to take care of your mental health and find an outlet. Always try to find support groups or counseling, even if things are good. It’s just helpful to have that resource. You can even find online support.