Leukemia genotherapy: new stage

Intellectual partner of the project

Yesterday we wrote about bad news: the experimental technique for treating HIV turned out to be ineffective. But such is the nature of any non -philanthropic ways: good luck alternate with failures, you have to overcome many unforeseen difficulties. Today is the turn of good news.

In March of this year, Proscience spoke about the experimental methodology for treating cancer of the lymphatic system developed at the University of Pennsylvania. Leukemias are diseases in which blood cells (mature cells or predecessor cells) begin to share uncontrollably. This method is suitable for the treatment of diseases in which B lymphocytes begin to reproduce uncontrollably-those cells that are normally produce antibodies.

The treatment method consists in the fact that patients take their own T-lymphocytes and modify them in the test tube. T-lymphocytes are cells (not all, but a significant part of them) that the sore cells of the body (cancer, infected with the virus, etc.) recognize “cancer” and help them die. T-lymphocytes were modified so that they recognize and kill all the cells on the surface of which there are a CD19 molecule, and it is on the surface of all B-lymphocytes: both healthy and patients.

Modified cells were introduced back to patients. Patients developed a typical, sometimes quite strong, picture of inflammation (ordinary symptoms of “colds” like high temperature and general ailment are caused by the activity of T-lecocytes struggling with infected cells). After some time, health was normalized and, if everything went well, there were no sick cells in the patient’s body, and the modified remained - theoretically, for the rest of their lives.

On their own, scientists conducted the first clinical trials for about a dozen patients, among whom were two children. The happy story of Emma Whitehead has circumvented many media outlets (she has been doing well for 20 months). Some patients have a complete remission, some have a partial improvement. Two treatment did not help. If we take into account that classical medicine with its chemotherapy and bone marrow transplant could no longer help these people, then this is an amazing result.

But ten people are only ten people, and, for example , in 2000, a quarter of a million people fell over with leukemia around the world. It can not allow yourself to be full -scale clinical trials necessary for approval of the drug for mass use - it can not - it is very expensive, and the cost of the method is great. But recently, the transnational pharmaceutical giant novartis became interested in the method. On December 7, they published the results of more large -scale clinical trials. These results are extremely optimistic. Of the 22 children with acute lymphoblastic leukemia, 19 came a complete remission. In adult patients with chronic lympholecosis, improvements occurred in 15 patients out of 32, of these 15 in 7 - complete remission.

This story is quite typical for modern drug development. First, scientists involved in fundamental science clarify the situation. It was fundamental studies that showed how T- and B-lymphocytes differ, how they interact with other cells, how the molecules look on their surface, during fundamental studies, phenomena were discovered, which formed the basis of cell modification. Then, scientists involved in applied science, facing which a specific goal is to “come up with treatment for the disease”, armed with fundamental knowledge, develop a method and show its effectiveness on a small group of patients.

Further, if the development looks promising, as a rule, large pharmaceutical companies enter the game. Of course, they do this in the hope of future benefits, but it must be understood that only a few drugs during clinical trials show themselves better than existing analogues, and, often, we are talking about a little superiority. The same story is notable for the fact that the method under study seems to be extremely effective, it is a great success for both the developer and for future patients.