107th Congress

Senate Appropriations Subcommittee on Labor, HHS, and Education -- Blood Cancers: Leukemia, Lymphoma, and Multiple Myeloma -- June 21, 2001

Members Present

Senators Tom Harkin (D-IA), Chairman; Arlen Specter (R-PA), Ranking Minority Member; Patty Murray (D-WA); Barbara Mikulski (D-MD); Kay Bailey Hutchsion (D-TX); Ted Stevens (R-AK). (Note: Senator Mikulski is not a member of the Subcommittee but came to lend support to one of the witnesses, former House Member Geraldine Ferraro (D-NY), who has multiple myeloma.)

Witnesses

Panel I: Richard Klausner, M.D., Director, National Cancer Institute (NCI), NIH

Panel II: Sandra J. Horning, M.D., Professor, Div. of Oncology, StanfORD Medical School; Mr. Larry Lucchino, President and CEO, San Diego Padres; Hagop Kantarjian, M.D., Professor of Medicine & Internist, Anderson Cancer Center; Mr. Miles Pendleton, Chronic Lymphocytic Leukemia.

Panel III: Geraldine Ferraro; Kenneth Anderson, M.D., Professor, Harvard Medical School; Ms. Kathy Giusti, President, Multiple Myeloma Research Foundation; John W. Holaday, Ph.D., EntreMed, Inc., Rockville, MD.

Summary

Purpose of Hearing: The hearing was held at the request of Senator Kay Bailey Hutchison (R-TX) and the blood cancer advocate community. Senator Hutchison, whose brother suffers from multiple myeloma, expressed enthusiasm and support for the hearing and called attention to the large number of sufferers of these diseases and their families who were in attendance from across the Nation. Although there were individuals who spoke for each of these diseases, this marked the first time these groups had formed a coalition to raise awareness and support for funding. There were several hundred spectators waiting in the hall who were unable to come into the hearing because of space constraints. Throughout the hearing, Members asked to have them invited in, to sit on the dais, on the floor, in the aisles, and stand, as many had traveled over 3000 miles to attend this hearing.

The witnesses were, despite the poor prognosis for these diseases, upbeat and positive about steps taken by NCI to address the problem, and particularly the recommendations of the NCI Report of the Leukemia, Lymphoma, and Multiple Myeloma Review Group (LLM-PRG), released in May 2001. They seemed heartened by recent research advances and pointed to new drugs which have been found to be successful in treatment of multiple myeloma, such as Gleevec and a new use for an old drug Thalidomide. Several witnesses referenced the LLM-PRG report, particularly recommendations on improved coordination among NIH, the Food and Drug Administration (FDA), and industry to bring new drugs to market sooner; and enactment of a patient's bill of rights bill that includes comprehensive clinical trials coverage. Questions raised to the first two panels included the anticipated value of human embryonic stem cell research for cures for these specific diseases and whether the cost for clinical trial coverage was in excess of current treatment costs. Senator Hutchison announced the introduction of a bill the day of the hearing with her colleague Senator Mikulski that will direct the NCI to establish a program for research of lymphoma, multiple myeloma and leukemia. It would authorize $250 million for that purpose and add $25 million for education efforts to be carried out by the Center for Disease Control (CDC).

Senator Specter noted an issue raised at a House hearing the previous day, which discussed the cloning of embryos. AccORDing to news reports, there was testimony that studies on stem cells from five-day-old cloned human embryos offer the best chance of developing promising new therapies for a variety of debilitating diseases. Dr. Klausner was pressed by Senator Specter whether cloned embryonic stem cells work better than human embryonic stem cells. Dr. Klausner responded that, "if we cannot do experiments to compare embryonic stem cells to nonembryonic or adult stem cells, we can't answer the question about what advantages they might have and what we may be missing. As I said, our best experience is from the mouse where the differences are quite clear and the advantages of embryonic stem cells for scientific research are clear." Senator Murray asked about the relative costs of clinical trials versus standard treatment. Dr. Klausner noted that all progress against diseases is the result of clinical trials and pointed to the analysis from four studies which have shown that there are no significant additional costs associated with clinical trials over standard treatment.

Questions:

Senator Specter

There has been testimony from biomedical researchers who believe that studies on stem cells from five-day-old cloned human embryos offer the best chance of developing promising new therapies for a variety of debilitating diseases. Are the cloned embryos, which produce stem cells superior to human embryonic stem cells, derived from embryos? I think it would be good for you to put on the record the superiority of embryonic stem cells in scientific research to which you have been referring.

I would like to have your verification and comment on other statements made by you that in cancer treatment you destroy cells; it is a destructive process to try to eliminate cancer. And then the stem cells are critically important as they come into the human body to replace the cells which have been destroyed. Would you amplify on that please?

From your response to the letter I wrote, you make the comment, probably the most dramatic advancement is the drug Gleevec in the treatment of CML. Two-part question: 1) What are the prospects for stem cells to be equally as effective on other forms of cancer? 2) How badly would you be disadvantaged if you couldn't use embryonic stem cells in the work that you're pursuing?

(To Mr. Pendleton and Mr. Lucchino) How do you feel about a situation where embryos are available, which are going to be discarded? And these embryos can produce stem cells which have enormous promise for answering and providing a cure for precisely the kind of ailment which you have. How do you feel about that?

(To Drs. Kantarjian and Hornig) How important do you think the potential for stem cells are in curing cancer?

What you think you can do within five years, what progress against disease, Dr. Anderson?

Senator Hutchison

What do you see going forward with the recommendations of the progress review group, and do you foresee the NCI going in a certain direction on the blood diseases now?

In the last 3 years, it seems that there has been more success at stemming the fatalities, the mortality of the blood diseases, and I just wondered if that means that you can do more in research because you have more to go on. And where do you think the best shots are in the near-term future?

Are you willing to say that you will be able to put more focus on these blood diseases now that you do have a little more to go on?

(To Dr. Kantarjian) Please define the term clinical trial. Does NCI do enough to help in the clinical trials?

Dr. Holaday, could you expand on the trial or study that is going on at Mayo and what you hope to gain from the results.

Senator Murray

Some of the opponents of the patient's bill of rights legislation have been arguing that coverage for patients in clinical trials is too costly. It seems to me that if we save lives and move forward that those costs are offset. Could you talk about how important clinical trials are in treating blood related cancers?

(To Drs. Kantarjian or Dr. Hornig) We're debating the Patient Bill of Rights and one of the contentious issues is whether or not patients should have access to clinical trials. Could either of you comment on that?

Senator Harkin

(To Dr. Klausner) A report from the Washington Post yesterday said that we are focused so much on finding a cure that we are neglecting research on how to care for people who are dying. Please comment.

Extramural research laboratories are now authorized for $250 million. Does it make sense to only spend $97 million of that in the next fiscal year?

The great breakthrough with the cancer drug Gleevec- how much of NIH research support went into its development? This drug may be applicable for other cancers? But the cost-I am wondering about the pricing of this drug. What do we do about the pricing, and how much of it comes back to NIH? We have to figure out what we are going to do about this.

Senator Mikulski

(To Mr. Lucchino) I am concerned about men's health. What advice or insight would you give to encourage men to see their physicians on a regular basis so that they can take advantage of early detection of disease as you were able to do? Do you think men would really be influenced by sports, and sports figures and through public service announcements?

Prepared by Anne Houser/OLPA