Where The Money Goes

Here are a few articles describing where the money we raise goes.

December 1999 Dr. Melvin Freedman Reports Research Progress

Since the inception of the Neutropenia Support Association ten years ago, we made a strong commitment to raise funds to support research of neutropenia and related disorders. We recognized that new information which can benefit patients can only come through research. We are also aware that funding from traditional granting sources was difficult to obtain, even by qualified investigators. To correct this deficiency, we pledged our support to raise funds through the Association to assist researchers in their efforts.

Recently, it was described in the "Journal of Experimental Medicine" the identification of a new abnormality of the G-CSF receptor in a child with congenital neutropenia who failed to respond to G-CSF treatment. The abnormality, called a point mutation, resulted in a faulty interaction between G-CSF and the abnormal receptor on marrow neutrophil precursors. The biochemical signaling that is supposed to result from the interaction was defective and interfered with the proliferation of the precursors. Remarkably, when the cells were exposed in the test tube to corticoidsteroid medicine such as prednisone the receptor abnormality was reversed and allowed G-CSF to perform its normal function. When the patient was given prednisone and G-CSF treatment in combination, neutrophils were produced normally for the first time.

Thus, these new data add to our understanding of why some patients are refractory to G-CSF treatment. Currently, we are looking at the 10% of patients who don’t respond well to treatment in order to define the magnitude of this receptor problem as an explanation for the poor response.

A second project that was completed and published in "Blood" addressed the issue of bone marrow failure in Shwachman-Diamond syndrome. This is an inherited, multisystem disorder characterized by abnormal pancreatic function, various degrees of bone marrow failure, and a striking tendency to develop leukemic transformation. The data has been reported on 13 patients with the syndrome and 11 healthy marrow donors. Patients with the syndrome had significantly lower numbers of marrow blood forming stem cells and an impaired ability for them to proliferate. In addition to this stem cell defect, we found that patients with the syndrome also had abnormal function of the marrow structural cells in terms of their ability to support and maintain neutrophil production. This dual defect has not been described previously and opens the door to research on the Shwachman-Diamond Syndrome at a higher level.

In 1999 Lorna Stevens, on behalf of the Neutropenia Support Association Inc., magnamimously presented The Toronto Hospital for Sick Children with a cheque for $10,000.00(raised through a charity golf tournament) to invest in a new project that is currently a "hot" area of research activity. It was proposed to measure the telomere length in inherited bone marrow failure disorders including congenital neutropenia and Shwachman-Diamond syndrome. Telomeres are sequences of DNA protein which cap each end of human chromosomes. They protect the genetic part of DNA from breakdown and they interact with factors that control the growth of cells. Each time bone marrow cells divide, telomeres shorten. Studies have shown that telomere length correlates with the age of the cells and the remaining capacity for them to divide. Measuring telomere length in inherited marrow failure disorders will facilitate our understanding of how and why the bone marrow function is impaired. We may also be able to understand the relationship of telomere shortening with respect to transformation into leukemia that occurs in some of these conditions.

Thus, the Association’s charitable donations have been a precious resource for bone marrow research in Toronto. It has made work much easier and has pushed the field ahead with new knowledge that hopefully will benefit patients directly.

As of January 2000 we remain the only support group charity helping families with this rare life-threatening blood disorder. Since January 1990 when the Neutropenia Support Association was registered as a Canadian charity, over $250,000.00 has been raised and contributed to neutrophil education and research. As volunteers we currently serve 25 countries with information. Many research teams in various provinces have received our support. The cyclic neutropenia has been identified. This will lead, we hope, to further lifesaving treatments.


Letter of Thanks from The Hospital for Sick Children to The Neutropenia Support Association Inc.

November 1999

Thank you so much for your continued support of The Hospital for Sick Children. A receipt for your generous gift of $10,000.00 is enclosed.

Your donation to support Dr. Freedman’s work in bone marrow research is working to ensure that Sick Kids can continue on its mission to better the health of children. By endeavoring to increase understanding of diseases affecting children, this type of research can translate into improved treatments and, ultimately, cures.

I hope you will take pride in knowing you have helped Sick Kids make kids better. Once again, thank you for your support - it is very much appreciated.


Thanks For Helping Us Help Sick Children

The Children's Hospital Research Foundation

Dear Members of the Neutropenia Support Association:

THANKS FOR HELPING US HELP SICK CHILDREN!

Through the generous and active support of groups such as yours, the Children’s Hospital Research Foundation is able to fund researchers like Dr. Bonnie Cham. As you know, she has conducted extensive research on neutrophils and has established a Canadian registry for people with Neutropenia.

Thanks to your contribution, children in Manitoba benefit from the most up-to-date knowledge and treatment in this area. Only through this type of generosity and commitment can we hope to maintain a standard of excellence in meeting the special health care needs of children.

On behalf of the Children’s Hospital Research Foundation, Winnipeg’s Children’s Hospital and, above all, the children of Manitoba, thank you for your donation. Your help means a brighter future for many young Manitobans and their families.


Why Not?

Neutropenia Support Association Newsletter, Volume 6, 1994

Dr. Fred Saunders and I want to thank you for the generous donation for support of neutropenia research. We will be using the funds immediately for the study of bone marrow cells from a child with SCN that has not responded to G-CSF therapy. The research question in this case is..."why not"?

The Hospital for Sick Children

Toronto, Ontario

Editors note: The Ontario Chapter and Head Office (Winnipeg) of the Neutropenia Support Association Inc. combined donation: $2500.00


Fundraising News

Pediatric Hematology/Oncology - Newfoundland

I was glad to hear that Mrs. Diane Cooper was able to raise such a significant amount of money in her community and that she is willing to help out here at the Janeway. You asked about a project for this fund and one area of concern that I have is the microscopes that we are using presently. Our senior hematology technician, Mr. Bill Hayward, tells me two of them came with the hospital twenty-seven years ago. I would hope to eventually purchase one with these funds to better enable us to look at blood films as well as do research.

Note: The goal to raise is $5000.00 A challenge, teamwork and enthusiasm.