But to turn the bright prospects into real incomes is not yet very successful - officially approved methods of cell therapy in the world barely get a couple dozen, and estimates of current and future revenue for different market analysts differ by an order of magnitude. There is nothing definite on the boiling frontier of science - even the terminology has not yet settled, let alone the profits.
The systematic use of stem cells in medicine began in the mid-1950s in the United States, where Dr. Edward Donnall Thomas developed bone marrow transplantation technology for the treatment of leukemia. It turned out that the cells of the donor's bone marrow introduced into the bloodstream of the donor (and in fact this is the stem hemopoietic cells) are able to "find their own way", "settle" in the patient's bones and start producing normal blood cells. To avoid rejection of foreign tissues by the immune system, the donor and recipient were tested for compatibility. For these developments Edward Thomas in 1990 was awarded the Nobel Prize.
So, is cellular technology the use of stem cells? Not only. To the same group of methods include, for example, cancer treatment with a chimeric antigenic receptor (CAR-T) - the human own lymphocytes are extracted, multiplied, engineered to "recognize" tumor cells and injected back into the patient's bloodstream so that genetically modified lymphocytes began to kill cancer cells. The method in different publications is called "cellular" therapy, then "gene", and even "immunotherapy." Well, and the treatment of genetic diseases through the introduction of genetically modified virus into the body, which is introduced into the cells and "corrects" their DNA, can be attributed to cell therapy?
In general, in order to avoid confusion, the term "regenerative medicine" has recently been applied in the United States. Established in 2009 to coordinate the efforts of the industry, the Alliance of Regenerative Medicine (ARM) defines the scope of its activities as: "Regenerative medicine includes gene, cell therapy and tissue engineering aimed at enhancing, treating, replacing or regenerating organs, tissues, cells, genes and metabolic processes in the body. "
The European Medical Agency (EMA) arrived even simpler - introducing for all new products the term "Advanced methods of medical therapy" (ATMP), which are defined as treatment methods based on the engineering of genes, cells or tissues.The volatility of definitions prevents analysts from trying to measure the market of regenerative medicine in monetary terms. Vademecum studied several expert reports and found a discrepancy in their final indicators by an order of magnitude. Estimating the current volume of the market ranges from $ 1.7 billion to $ 17 billion, and the invariably optimistic forecast of its growth in the next five to eight years gives the spread even more - from $ 5.6 billion to $ 83 billion. For comparison, the global volume of the pharmaceutical market in 2017 is analytical the company IQVIA estimated at $ 1.1 trillion.
Perhaps this is why ARM in its reports on the state of the industry evades such assessments, focusing on figures that can be verified. According to the most recent data, the development of regenerative medicine in the world is conducted by 860 companies, more than half (464) of them are located in the US, 233 in Europe and Israel, and only 126 in Asia. True, the last figure can also be doubted - very few know exactly what is happening in China, except that cell technologies are developing rapidly in the absence of strict regulatory and ethical barriers.
"Any major hospital in China" does "cells. They very quickly adapt new technologies, many centers have laboratories for the production of cell lines, "Michel Sadelain, director of the Center for Cell Therapy and Engineering of the Cancer Center of the American Clinic Sloan Kettering and inventor of the CAR-T method, told during his visit to St. Petersburg.
In the world, according to ARM estimates, 959 clinical trials (CIs) of regenerative techniques are now being carried out, of which only 90 are in the final phase of III. Among the total number of studies, this drop is in the sea - on the portal clinicaltrials.gov now registered almost 280 thousand CI of which 35%, the rest - international). Global funding for companies in the field of regenerative medicine in ARM was estimated at $ 20 billion in 2017, with $ 9.1 billion of them accounted for M & A transactions, and this is no accident.
With modest financial indices of the industry, research costs are enormous, and the results are unpredictable. "Initially, our development was funded by the National Cancer Institute of the United States, but when it came to clinical research, we had to turn to philanthropy. Then no one believed in the technology of CAR-T, "- recalls Sadelain.
Large pharma companies are in no hurry to finance advanced development. On the contrary, they often get rid of high-risk research in the field of regenerative medicine, so as not to spoil the reports. For example, Swiss Novartis disbanded its research unit in the field of cellular and gene therapy in 2016, and Pfizer transferred the CAR-T development portfolio to Allogene Therapeutics in 2018 in exchange for 25% of the latter.
Farmgigants prefer to leave risks to researchers, buying the rights to those drugs and techniques that have already shown encouraging results. A license for CAR-T-therapy of acute lymphoblastic leukemia was purchased by Novartis from scientists at the University of Pennsylvania. The use of a similar technology for the treatment of certain types of non-Hodgkin's lymphoma was developed by the American-Israeli Kite Pharma - it was bought by Gilead Sciences in 2017 for $ 12 billion. In 2017, Novartis technology was approved for use in the US under the name Kymriah - and was the world's first officially registered CAR-T-therapy. Soon approved and Yescarta from Gilead. Innovation is expensive - Kymriah will cost the patient $ 475,000, Yescarta - at $ 373,000.According to Sadelain, about 2,000 patients received CAR-T treatment in the US, but mostly free of charge - during clinical trials. How many people can afford to buy such drugs can be estimated according to Gilead reports for the II quarter of 2018. According to Bloomberg, Yescarta sales amounted to $ 68 million - it turns out that the procedure was 180 patients. This is clearly not yet a blockbuster, if you look at the Gilead drug developed by hepatitis C Sovaldi, which for the first full quarter of sales brought $ 2.27 billion.
The first approved gene therapy using a genetically modified virus will cost even more. The procedure Luxturna for the treatment of a rare hereditary disease of the retina - amaurosis Leber - at the time of registration in early 2018 was announced by the developers of Spark Therapeutics at a price of $ 850,000.
And what about stem cells? As a typical example, you can take the California Institute of Regenerative Medicine (CIRM) - one of the foremost research institutions in the US in this field. The history of CIRM began in 2004 with a vivid advertising campaign featuring famous actors Michael J. Fox and Christopher Reeve. Fox suffers from parkinsonism, and Reeve was paralyzed after falling from the horse. Both came out with a warm support for the development of stem cells, promising the emergence of drugs for their diseases, as well as many others. Under pressure from research enthusiasts stem cells from the public organization Americans for Cures in California adopted a special law, according to which was created and received funding of $ 3 billion CIRM.
"And now, many billions of dollars, and what do we have 14 years later? No approved drug from stem cells, but CIRM in 2020 is going to ask for another $ 5 billion from California, "- resents the Californian TV and radio company KQED. "Well, what is $ 5 billion? - retorts the head of Americans for Cures Robert Klein. "The state spent $ 6.5 billion on a new bridge last year, it's a transport infrastructure, and we are creating a scientific one!"
CIRM has something to show in its defense. For example, the six-year-old Evangeline Padillo-Vaccarro, who was to die four years ago. The girl was born in 2012 with severe combined immunodeficiency (SCID). Children with such a congenital disease are forced to live in complete isolation, but still usually do not live up to two years - their immune system is "off", and a common cold can become lethal. CIRM, along with scientists from the University of California, Los Angeles, took the hematopoietic stem cells of the girl, corrected the genetic defect and brought it back. Since then she lives a normal life. Later 40 children with SCID were treated in a similar way. But the technique is officially still only in the II phase of CI. A total of 43 clinical trials are conducted with the participation of CIRM, of which only five are in Phase III, according to the Institute's website. In general, the CIRM recognizes that their promises in 2004 were somewhat exaggerated.
Opponents of the new round of CIRM funding do not like this. Barbara Koenig, the head of the bioethics program at the University of California, San Francisco, is not against stem cell research, but believes that public $ 5 billion can be used more effectively. "There is too much noise around the stem cells and statements that a miracle cure is about to be invented. We need to be honest - while we invest in research. And this money can be spent on ensuring that every child in California receives clean water, quality food and excellent basic medical care, "König KQED said.
But the "noise" learned to use unscrupulous operators and just scammers who promise "miraculous healings" from almost everything with the help of injections of stem cells. Cells Stem Cells magazine in 2016 published a study according to which only in the US were found 550 clinics that advertise illegal options for stem cell treatment. A year later, the journal "Regenerative Medicine" received an even more alarming message - several "cellular" dealers managed to penetrate the clinicaltrials.gov portal and sell their services under the guise of "paid clinical studies", none of which, of course, received official approval FDA. The regulator had to intervene and publish a warning: "Some unscrupulous clinics offer treatment methods that are both not approved at the same time and do not have proven effectiveness. Beware of these potentially dangerous procedures, be sure to carefully study all the information before starting therapy. Any stem cell preparations or clinical studies of stem cells should be approved in accordance with FDA procedures. "
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