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Fellowships

A Great Beginning

Greetings partners. We are sorry this update is so much later than we anticipated. We think we will make up for that by sharing what wonderful things are going on thanks to your donation.

We officially made the donation mid summer. The fellows were already hired and had begun working on our leukodystrophy (lysosomal disorder) issues. While we were down in North Carolina for Maddie and Sam’s nine month check up, we met one of the fellows. The supervising doctors, Dr. Maria Escolar and Dr. Joanne Kurtzberg, are involved both in the fellowship program and in Maddie and Sam’s care. They were very appreciative and excited about the work to be done.

We are, of course, very hopeful for all leukodystrophy kids. We cannot express enough, that this could not have been accomplished without you.

The first fellow is working at Duke under Dr. Kurtzberg’s direction. We have established at Duke the ‘Ross Joiner Fellowship for the Study of Lysosomal Storage Diseases in Children’. Here is the description that Dr. Kurtzberg sent to us.

“The fellow's name is Donald Beam. He is a trained pediatrician and has completed his first year as a fellow in clinical hematology oncology. He will be in the lab from 7/1/04 through 6/30/06 but may stay longer if his project is going well and we get additional funding.

Don's project is entitled "Isolation and propagation of oligodendrocytes from human umbilical cord blood". Don is taking fresh umbilical cord blood donations, isolating mononuclear cells, placing these cells in tissue culture with hormones that promote the growth of neural cells and growing them for 10-14 days. At this time, cells with the characteristics of oligodendrocytes are seen. Don then takes these cells and transfects them with a gene that contains the chemical luciferase. This is the same chemical that fireflies have in their cells causing them to glow. When cells are transfected with this gene, they can be induced to glow too. This allows scientists to track and follow the cells after they are injected into mice and rats under study. Don will take the transfected cells and inject them into the blood, ventricular fluid (fluid around the brain) and abdominal cavity of immunodeficient mice as well as mice with Krabbe disease (Twitcher mice) or MLD (Shiverer Mice). He will see where the cells go (homing) and see which route of injection delivers the most cells to the brain. He will also study subsets of cord blood cells to find out which cells give rise to neural precursors. When he's answered this question, he will work out ways to grow large quantitites of these cells in tissue culture.

The long term goals of this work is to create a cell therapy product that can be used to treat children with lysosomal storage diseases. This therapy might augment or ultimately replace a traditional transplant.

We are very excited about this fellowship opportunity and are confident that it will facilitate and accelerate progress in the understanding and treatment of lysosomal storage diseases (like metachromatic leukodystrophy) in children. “

The second fellow is working at the University of North Carolina under the direction of Dr. Escolar. Dr. Escolar is the doctor who monitors Max, Maddie and Sam’s development. She is on staff at both UNC and Duke and works closely with Dr. Kurtzberg. Dr. Holly Martin is the fellow at UNC and here is what she sent about herself and the work she will be doing.

“My origins began in Ohio, but quickly expanded to Lexington, Kentucky where I completed both my Bachelor of Science and Medical degrees. While in medical school, I met my husband, Geoff A. Martin and we had a beautiful daughter named Sylena. I graduated medical school in May of 2001 from the University of Kentucky. I have spent the last three years at the University of Louisville, also in Kentucky, completing a Pediatric residency. One short year ago, my husband also completed his residency in Emergency Medicine. In one month, we will be having an addition to our family, as I am due to have a baby girl. (Sorry, we are unable to provide a name at this time.)
Throughout my medical career, I have been interested in rare and genetic diseases, making an extra effort to educate myself on less common genetic syndromes seen in children. I am delighted at the opportunity to work with Dr. Maria Escolar following the behavior and neurodevelopment of children with lysosomal storage disorders and rare neurological diseases. For me, this incorporates two areas of special interest, as well as allowing me to establish long-term patient care relationships. As a bonus, I am excited to join a field of Pediatrics with such rich opportunities for growth in patient management and research.”

Dr. Escolar also sent a letter of thanks.

“I wanted to express my deep gratitude for your generosity in establishing the Trimpers’ Clinical Fellowship for Research in Lysosomal Storage Disorders.

Thanks to your support, Dr. Holly Martin will spend the first two years of her Child Development and Behavioural Pediatrics clinical fellowship focusing on lysosomal storage disorders. She will receive additional training in research methodology and will complete a research project in this area. In addition, your generous gift will allow our program to continue to describe the natural history of these disorders and longitudinally track outcomes of children treated with hematopoietic stem cell transplantation.

Your donation will also allow us to work in collaboration with the UNC Neurodevelopmental Research Center, and NIH funded center that promotes scientific interaction among biomedical researchers from different disciplines in the field of neurodevelopment. This partnership will increase awareness among participating community of investigators, impact training in related fields and advance future research in the area of lysosomal storage disorders.

Most importantly, your support will allow clinicians in the field of developmental and behavioral pediatrics to work toward improving identification, clinical care, and the quality of life of patients suffering from lysosomal storage disorders.”

The doctors are very appreciative. The fellows are very appreciative. We are so very, very appreciative.

If you haven’t had the chance to send your first donation, it isn’t too late. We will keep you updated on the progress being made in North Carolina and will remind you when the second set of donations is needed.

Team leaders, please share this with your partners. Post them, send them, just make sure they keep informed.

Thank you so much!




Dr. Donald Beam





Dr. Holly Martin






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