A word on Leukemia and progress from Dr. Emil J. Freireich


Just 50 years ago, the diagnosis of acute leukemia was a death warrant. Patients looked forward to an immediate onset of bleeding from mouth, nose, urine, rectum, major infection, and a lifespan of approximately two months on average, with virtually 100% mortality by one year after diagnosis.

Today, 80% of children with the diagnosis of acute lymphoblastic leukemia can be cured. Approximately 40% of adults with acute lymphoblastic leukemia can be cured. For patients with the myeloid leukemias, the overall cure rate is approximately 25%, but there is a subset of patients who receive individualized treatment where cure rates are much higher.

In acute promyelocytic leukemia, which is between 6 and 10% of patients, today’s therapy gives over 90%—5 year survival. For patients with other cytogenetic abnormalities, such as Inversion 16, and Translocation 8/21, cure rates are reported between 40 and 60%.

Similar progress has been made in the treatment of the more differentiated or chronic leukemias. The most dramatic advance in the last decade has been the discovery of the drug Gleevec. This is a small molecule which is administered in pill form by mouth and with this single treatment - one pill a day. A disease which had an average survival of 3½ years and over 90% mortality by end of 5 years is now (resulting from Gleevec therapy) over 90% of the patients alive at 10 years post-diagnosis – a phenomenally dramatic change in the natural history of this disease.

Likewise for chronic lymphocytic leukemia, current therapy benefits over 90% of patients with control of all the signs and symptoms of the disease for extended periods. One form of chronic lymphocytic leukemia, hairy cell leukemia, which makes up approximately 3% of these patients, have had their disease controlled indefinitely with current available therapy.

Importantly, the progress that has been made over the last 50 years has been accelerating over that period of time. So the promise for the next 50 years is for even more effective therapy to be developed.

Impact of regulation on development of new and more effective therapies

In an effort to provide maximum protection for patients and for the public at large, the United States government has established an elaborate regulatory process operated by the Food and Drug Administration to ensure that individual are not harmed as a result of the availability of therapies. Although these legislative activities have the best of intentions, as is frequently the case with well-intentioned legislation, the consequences have been very harmful to those patients who have serious and life-threatening diseases. These regulations came into force as a reaction to the thalidomide scandal where a drug was marketed to the general public as a sedative but turned out to have terrible effects on the babies that were being carried by women who used this medication. New regulations certainly would and will be effective in preventing this type of activity, but the unfortunate consequence is that the legislation did not distinguish between pregnant females with babies in their uterus and patients with life-threatening illnesses who are desperately in need in the development of new, effective treatments.

It has become progressively obvious that the regulatory process has greatly delayed the development of new treatment. Current estimates are that from time of conception to availability to patients can be as long as 15 years, and almost two-thirds of that 15 years, i.e. 9 years, is spent testing this treatment in experimental animals, in tissue culture, bacteria, and so on – the so-called IND or investigational new drug application process. It is clear to patients with life-threatening illness and to those who provide professional care to these individuals that this process has to be accelerated for the benefit of those patients without comprising the protection for healthy individuals or pregnant women. I am strongly supportive of legislation to improve access to investigational treatments for patients with life-threatening illnesses. —Emil J. Freireich, M.D., D.Sc.

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