Support of HB 371: Stem cell funding for sickle cell disease

By Nancy Paltell, Ph.D, Associate Director of Respect for Life

House Health and Government Operations Committee, February 23, 2010

This statement, in support of HB 371, is conveyed on behalf of the Catholic bishops serving Maryland and their dioceses. Read the attachments here.

We urge a favorable report for HB 371 because the treatment of sickle cell disease using adult stem cells is a golden opportunity for the Maryland Stem Cell Research Fund (MSCRF) to shine by funding research that will likely rapidly result in a cure for a devastating disease. Maryland would be well on its way to establishing itself as a center of excellence for the treatment of sickle cell disease.

Just two months ago, the New England Journal of Medicine published the results of a clinical trial in which sickle cell disease was successfully reversed in nine out of 10 adult patients in the trial. This is considered a true breakthrough by experts in the field. The work was done in Maryland, and involved the use of adult stem cells obtained from bone marrow. Since the research used stem cells and was done in Maryland, it would qualify for funding from the MSCRF.

The value of using adult stem cells to reverse sickle cell disease has been known for many years. However, it’s important to note that in both 2008 and 2009, Maryland researchers applied for a grant from the MSCRF to study the use of adult stem cells to treat sickle cell disease and their grant applications were rejected.

Sickle cell disease (SCD) is a debilitating disease that results in a shortened life span. Other sources of funding for research into SCD are scarce. Yet medical researchers are so close to producing a routine cure for SCD – isn’t nine out of 10 patients successfully treated assurance enough that this type of research is worth funding?

To be clear, the recent breakthrough is fantastic news, and there is more work to be done. The clinical trial was small, just 10 people, so a larger trial must be conducted. The clinical trial used adult stem cells from matched bone marrow; now the work must be expanded to use adult stem cells from either non-matching bone marrow or umbilical cord blood. This type of applied research is just the reason the MSCRF was established, yet Maryland researchers have been unsuccessful in securing a grant for research using adult stem cells to treat sickle cell disease.

When the MSCRF was started in 2006, citizens of Maryland were promised cures for devastating diseases and injuries. The fund was supposed to be used for both basic and applied research.

Basic: adjective, constituting or serving as the basis or starting point.

Applied: adjective, put to practical use, e.g., applied sciences.

As the definition from Webster’s Dictionary implies, basic research is considered very close to the beginning, far removed from use in patients. Applied research takes those basic concepts and applies them so they have practical use. In the case of medical research, it takes the basic research to the next step and uses it to produce treatments that can actually be used to relieve patients’ suffering.

Read all the attachments here. A robust research program strikes a balance between basic and applied research. Both are needed. However, an analysis of how the money from the MSCRF is being spent shows that the majority of the money is being spent on basic research. For 2009, looking at just the large-scale investigator-initiated grants, 100% went to basic research (see Attachment 1). Not a single grant went to applied research, such as adult stem cell research. Fully 100% of the $9.3 million went to basic research using stem cells that cannot be injected into patients (pluripotent stem cells). It’s encouraging to see that half the money was invested into iPS research, which doesn’t have the ethical issues associated with embryonic stem cell research, but both iPS and embryonic stem cell research use pluripotent stem cells, which are known to cause tumors. See Attachment 2, the abstract of a project funded by the MSCRF at $1.73 million, to see confirmation that embryonic stem cells form tumors. Worldwide, many millions of dollars have been invested to try to figure out how to stop embryonic stem cells from forming a certain type of tumor called a teratoma, but to no avail. Adult stem cells, however, do NOT form tumors; hence they may be safely injected into human patients.

An analysis of all three types of grants awarded in 2009, the investigator-initiated grants, exploratory grants, and fellowships, reveals that overall only 20% of the MSCRF is being spent on applied research, research that is either already helping patients or is very close to doing so. Applied research includes adult stem cell research and research into ways to destroy cancer stem cells. See Attachment 3.

While applied research that had already demonstrated proof-of-concept using adult stem cells to treat sickle cell disease was rejected by the Maryland Stem Cell Commission, it’s ironic that basic research using embryonic stem cells and induced pluripotent stem cells was given $1.1 million from the MSCRF. A year later, despite the fact that the research team was no closer to a clinical trial to treat blood disorders, insult was added to injury when the same research proposal received an additional $1.73 million. Thus nearly $3 million has been devoted to exploring the use of speculative pluripotent stem cells to treat blood disorders, while no money has been awarded to expanding the use of adult stem cells to treat sickle cell disease. This despite the fact that worldwide, the only cures for sickle cell disease have involved adult and umbilical cord blood stem cells. What will it take to convince the Stem Cell Commission that the research deserves funding?!

Some legislators may have concerns that once a small percentage of the MSCRF is set aside for one particular disease, other disease-advocacy groups may come forward and request that money be designated for research into cures for other diseases. To this the response should be: if the disease is debilitating and results in a shortened life span, if other funding sources for the research are scarce, and if researchers have already demonstrated the potential for success through a clinical trial in which the majority of patients were cured, then the request for funding should be seriously considered. After all, the reason the MSCRF was started was to provide cures to the people of Maryland. Opportunities to facilitate the rapid delivery of such cures should be considered by the General Assembly.

Three years after its inception, the General Assembly should look at whether or not the MSCRF is delivering on the promises made to the people of Maryland. We maintain that it is not. And because the MSCRF is not delivering the cures the people of Maryland expect, using a small amount of the money to fund research that is highly likely to produce a cure is a win-win situation. Globally, people suffering from the devastation of sickle cell disease win because a cure is truly on the horizon, and the Stem Cell Commission wins because they will be able to tout the sickle cell disease progress as an accomplishment made possible because of the MSCRF. We urge a favorable report for HB 371.