Two important articles that smashed the "precise medical" bubble

Two important articles that smashed the "precise medical" bubble

October 28, 2016 Source: Science Network

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In September, two articles on “Precision Medicine” were put on the trial platform, which attracted widespread attention. One published in the journal Nature; the other in the New England Journal of Medicine:

Two articles summarize the clinical results of some of the recently published individualized treatments for cancer, but all give very pessimistic comments. The first article argues that "the precise strategy does not bring benefits to most cancer patients." "Precise treatment of tumors is only a hypothesis to be proved."

"...most people with cancer do not benefit from the precision strategy, nor has this approach been show to imporve outcomes in controlled studies. Precision oncology remains a hypothesis in need of verification"

“Objectively, the prospects for precision cancer treatment are not optimistic. The best result is a brief relief in a small number of patients. What happens is the side effects and expensive treatment costs.”

"When measured objectively, the prospects and potential of precision oncology are sobering. At best, we may expect short-lived responses in a tiny fraction of patients, with the inevitable toxicity of targeted therapies and inflated cost that this approach guarantees."

The second article warns us that "this (precise medical) concept is attractive to patients and institutions that support cancer research, and despite the lack of useful evidence, molecular analysis of tumors has been sold directly to patients."

"This concept is appealing to patients and to foundations that support cancer research, and the molecular characterization of tumor is being marketed directly to patients, despite a lack of evidence of benefit."

Several large clinical trials to date have shown that after molecular diagnostic analysis such as sequencing, approximately 30-50% of patients can find related mutations that can explain tumor malignancy. Because of the limited availability of drugs, only 3-13% of patients can find "precise" drugs. Even with the paired drugs, only a small percentage (30%) of patients have efficacy. Several discounts were scored, and the last patient benefited was only 1.5% of all patients .

The article points out that there are several reasons why the current precision medical care is not ideal: (1) Most of the existing targeted therapeutic drugs can only partially block the cell proliferative pathway; (2) Try to completely block the malignant proliferative pathway by merging drugs or Increasing the dose, both will increase side effects; (3) tumor heterogeneity, that is, the same patient's tumor cells have multiple mutant strains, cured a part of the cells, and another part grows. (4) It is also the cause of tumor heterogeneity. Drugs that are effective at the beginning (the other cell) quickly develop resistance.

Some people think that with the strengthening of diagnosis and the continuous listing of new drugs, precision medicine will become better. However, the authors of the article believe that there are two difficult conditions for the precise treatment of tumors : (1) tumor-related driver mutations are present in all cancerous cells, and the drugs against the mutations are absolutely effective; (2) Tumor heterogeneity itself can be controlled.

To put it bluntly, the target of precision treatment cannot be kept moving, otherwise it is difficult to talk accurately.

The author also mentions the limitations of liquid biopsy: most of the tumor DNA leaking into the blood comes from the killed tumor cells, and the tumor cells that have not been killed are the targets that really need to be attacked.

The author concludes by stating: “We are not suggesting a complete abandonment of personalized medicine, but rather suggesting some small-scale, well-designed collaborative research to try to deal with the limitations mentioned above. At the same time, we should also give patients a Clear information: Individualized treatment of tumors has not shown substantial efficacy and is still in clinical validation. "

" " " " " " " " " " " " " Not led to gains in survival or its quality and is an appropriate strategy only within well-designed clinical trials."

According to statistics, at least 200 companies involved in "precise medical care" in China now have received hundreds of millions of dollars in financing. Some have already brought a variety of accurate medical tests to the market, and bubbles have formed.

When I just put forward the slogan of "precise medical care", I got a piece of cold water. The purpose of this article is also to hope that everyone can think more calmly about some of the problems of precision medicine.

Investors, whether it is government money or private investment, should weigh the risks and returns. Unlike investments in the Internet, investment risks in health care are primarily scientific and not operational. Buying more than one sequencer and hiring several people will not speed up the bottleneck of the concept of precision medicine itself. At least at this stage, before the scientific barrier (the heterogeneity of the tumor) has not been overcome, large-scale commercial operations can only accelerate the bursting of the bubble. I hope that more rational investors can seriously study the project and invest in truly innovative things instead of tracking the concept of being sizzled.

Companies that have already received the wind and money need to be cautious, put good steel on the cutting edge, do not force profit pressure to push not very mature products and services, so burning money is a trivial matter, bad market reputation is a big deal. The sequelae caused by the rapid marketization of PCR, cell therapy, etc. are all lessons. I believe that a really good company can always find new development opportunities.

As for patients, they should know how to protect themselves. If only 1.5% of patients in the United States benefit from “precise medical care,” how many people in China can benefit? If I am a patient, instead of spending thousands of dollars to do accurate medical testing, it is better to find a high-end restaurant and family to eat more accurately.

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