Consider this please:
Testimony By Dr. Robert Goldstein, Chief Scientific Officer of the Juvenile Diabetes Research Foundation International (JDRF) Regarding Adult Stem Cell Research
Before the Senate Commerce Committee Subcommittee on Science, Technology and Space
July 14, 2004
Chairman Brownback and members of this Subcommittee, thank you for the opportunity to appear before you today to participate in this important hearing on adult stem cell research. I am Robert Goldstein, Chief Scientific Officer of the Juvenile Diabetes Research Foundation (JDRF). I am joined today by the Langbein family who represent the millions of families who struggle with the daily challenges and fears of caring for a loved one with juvenile diabetes. Jamie was diagnosed at the age of one, and she has been on an insulin pump since the age of four. Jamies diabetes affects her life every day, all day. Her parents must test her blood sugar eight times a day, and every time she eats, exercises, or goes to a birthday party, Jamie must account for what she eats or how much exercise she does and adjust her dose of insulin accordingly so she doesnt end up in the hospital or in a coma. Her mom gave up her career as an attorney so that she could always be nearby if Jamie had problems with her pump or blood sugar while at school, and her parents get up frequently during the night to check her blood sugar level. Jamie worries about being different from her friends in school, and her parents worry about the long-term complications of diabetes and their daughters future and whether their other children will be diagnosed with the disease. This is just one child of the nearly two million people who battle juvenile diabetes each and every day.
JDRF is the leading charitable funder of juvenile diabetes research worldwide. Established more than 30 years ago by parents of children with juvenile diabetes, our mission is to find a cure for juvenile diabetes and its complications. Over the years, JDRF has provided some $800 million in grants for diabetes research at most of the worlds leading universities, laboratories, and hospitals. To fund that science, JDRF volunteers do their part every day to raise money in our communities across the country through walks, galas, and other events -- and we are proud of the strong partnership for funding research that we have developed with the federal government.
JDRF, as the worlds leading charitable funder of diabetes research, aggressively pursues all avenues of promising research and makes its funding decisions based upon vigorous scientific review based, in many ways, upon the NIH model. In the area of stem cell science, JDRF funds scientists exploring the opportunities created by both adult and embryonic stem cell research. In Fiscal year 2004, JDRF commitments in the area of stem cell research total $8.2 million. Of this amount, $6.3 million is spent in the area of embryonic stem cell research and less than $2 million is spent on other areas of stem cell research, including adult stem cells. We focus on both areas as well as dozens of other avenues of scientific investigation because no one can predict what area of research will produce new therapies or a cure for juvenile diabetes.
Adult stem cell research has been pursued for more than 35 years, and as you know, embryonic stem cells were just discovered in 1998. JDRF will continue to support both adult and embryonic stem cell research so that we can pursue a cure as strongly as possible. However, the research community believes that embryonic stem cells offer more promise in the area of diabetes. Let me explain why, using pancreatic islet cell transplantation as an example. Islet transplantation has been a spectacular breakthrough in diabetes research. In islet transplantation, the beta or insulin-producing cells are isolated from a cadaver pancreas and then infused into a person with juvenile diabetes through a catheter inserted into the portal vein of their liver. Once transplanted, these new islets recognize blood sugar levels and begin to produce and release insulin into the patients body. Islet transplantation had been attempted since the 1970s with limited success. However, in the year 2000, researchers made a breakthrough in the procedure, and since that time nearly 300 people have received islet transplants and the majority of them lead significantly better and healthier lives. In most of these individuals, therapeutic control of their diabetes has improved remarkably, and in many instances they do not even have to take insulin injections. Furthermore, many of the patients have reported a reversal in some of their complications, especially hypoglycemia unawareness but also improvement in vision and less pain from neuropathy.
These results are very exciting, but there are significant hurdles in moving this from an experimental procedure to a standard therapy that could benefit the millions of people with diabetes many of them children. One such hurdle is the severe shortage of donated pancreases. In 2001, approximately 400 pancreata were available for islet transplantation and research, compared to the almost two million Americans with juvenile diabetes.
Here, then, is one reason why we are so excited about recent advances in embryonic stem cell research. Recent studies have demonstrated the ability to coax embryonic stem cells into insulin-producing cells in the lab. We have good reason to believe that embryonic stem cells will one day be able to grow large amounts of insulin-producing beta cells for transplant, but more work needs to be done. Unfortunately, adult stem cells have not shown the same promise when it comes to diabetes. Last month, Harvard University researcher Douglas Melton published a paper in Nature pointing out that in mice, new beta cells in the pancreas are formed through the replication of existing beta cells rather than through the differentiation of adult stem cells. This finding indicates that adult stem cells in the pancreas do not contribute to beta cell formation, and that embryonic stem cells may prove to be the only stem cells that will be useful to generate beta cells for the treatment of Type 1 diabetes. Other studies indicate that mouse embryonic stem cells can be differentiated into insulin-producing cells, and several studies suggest that this can be done using human embryonic stem cells.
JDRF funds research to develop beta cells from adult stem cells, or to regenerate beta cells from existing precursor cells. Researchers have reported that human adult duct tissue might have the potential to develop into beta cells. Other groups have results that indicate that transplanted bone marrow cells may be able to show insulin production. Some have used these findings to argue that adult stem cells may be the answer for curing juvenile diabetes. JDRF takes the position that research using both embryonic and adult stem cells, perhaps even in side-by-side comparisons, will get us to our goal fastest.
Mr. Chairman, adult stem cells may one day prove to be the answer to alleviating the pain and suffering caused by certain diseases I certainly hope that is the case. We have heard some remarkable stories from some of the witnesses today. But we have no idea of knowing which diseases those may be, and unfortunately we are not certain of the widespread application of these treatments. We do know that to date, adult stem cells have not been shown to hold as much promise for juvenile diabetes as embryonic stem cells. Given this reality, how can we turn our backs on other exciting research opportunities, such as embryonic stem cell research, thereby potentially delaying life-saving therapies and cures for millions of people? And how can we adequately compare the effectiveness of adult and embryonic stem cell research unless both avenues are pursued simultaneously and with equal rigor?
We are in an extraordinary time of opportunity in the area of medical research, and this country is leading the way. Scientists around the world agree that stem cell research holds tremendous promise for hundreds of millions of people. I applaud you for continuing to monitor advances in the area of adult stem cell research, and I encourage you to do the same for embryonic stem cell research. For certain diseases such as juvenile diabetes, embryonic stem cells hold the most promise, and we cant afford to lose any more time.
While we have made great strides towards our goal of a cure, more needs to be done, and we dont have time to wait. Insulin is not a cure for juvenile diabetes, nor does it prevent the onset of complications such as kidney failure, blindness, heart disease and amputations. Diabetic retinopathy is the leading cause of adult blindness in the United States; ninety percent of patients have evidence of retinopathy after fifteen years of diabetes with approximately 25,000 new cases of blindness per year. Diabetes is also the leading cause of renal failure in the United States, accounting for forty percent of new cases per year. Greater than half of all patients with diabetes develop neuropathy, making diabetic neuropathy the most common cause of non-traumatic amputations and autonomic failure. In his or her lifetime, a diabetic patient with neuropathy has a fifteen percent chance to undergo one or more amputations. Mr. Chairman, in the battle against diabetes, we are in a race against time.
Not a day goes by that JDRF doesnt receive calls or letters or email messages from mothers or fathers of children with type 1 diabetes asking, When will my child be cured? On the one hand, it is extremely difficult to explain the pace of science, particularly to a mother whose five-year-old has to prick his finger six or seven times a day to test his blood sugar, who needs three or four injections of insulin every day, who is afraid to go to sleepovers or summer camp for fear of falling into a coma, and who is at constant risk of developing a host of complications that could cut short his life. But on the other hand, it is downright tragic to have to explain how the pace of science could be slowed even further by focusing on one area of research and excluding another.
To put the urgency of finding a cure into perspective, Id like to share some words from Mary Tyler Moore, JDRFs International Chairman, that she shared with Members of the House. Mary states that in the nearly six years since human embryonic stem cells were first successfully cultured in a lab,
diabetes has contributed to the deaths of as many as 3 million people and cost our nation over $750 billion. It has caused nearly 500,000 amputations, rendered over 100,000 people blind, and forced a quarter million people to require kidney transplants or dialysis. And 120,000 moms have been told that their child has Type 1 diabetes a disease which during that time period would require each of these children to have 8,700 injections of insulin and 17,500 pricks of their fingers to check blood sugar levels just for that child to survive.
Thank you again for the opportunity to appear before you today. I am happy to answer any questions you may have.
