CAR-T does not lack basic research, it lacks a high-quality clinical trial translation platform.

Recently, the pricing of two domestically-listed CAR-T products have been announced.
Fosun Kate’s Akilunza injection is expected to be listed at a price of 1.
2 million yuan per injection, and WuXi Juno’s Ruiji Orenza injection is expected to be listed.
The net price is 1.
29 million yuan per piece
.

Payment has become a major factor preventing CAR-T products from entering the market quickly
.

This year, Akirensai injection passed the preliminary review list of medical insurance negotiations, but in the end, it was overpriced and stopped on the final list of negotiations

A scientist who has been deeply involved in the CAR-T industry pointed out that in the next few years, CAR-T therapy will still be a treatment for some patients in China.
This is a painful stage that scientific development must pass through, because each field has a , The process of continuous development and finally maturity
.

Universal CAR-T is an important direction to solve the payment problem
.

The aforementioned scientists believe that the foreign body will be resolved in the next 5 years.

01.
How to treat the homogeneity of CAR-T product targets?

01.

As of the end of November this year, in the global CAR-T therapy development pipeline, CD19, as a target for a variety of therapies already on the market, ranked first in the number of research and development
.

Among the TOP20 targets in the global CAR-T field, CD19 accounts for 40%

The world does not need so many CAR-Ts with the same target, nor does China
.

For enterprises, even if the CD19 CAR-T is developed, the market prospects are most likely not optimistic

However, there are still quite a number of companies carrying out clinical development on mature targets such as CD19 and BCMA
.

Regarding the issue of homogeneity, an expert from the Institute of Hematology pointed out that some overlaps will inevitably be caused when new technologies are first developed.

In fact, many researchers, experts, and PIs in hospitals are doing basic research.
Pharmaceutical companies can seek some joints.
The joints between basic and clinical transformation can also provide more new targets
.

An entrepreneur who has been doing CAR-T products for a long time also said that the crowding phenomenon is temporary
.

He also gave an example, “Foreign technical research is relatively large, especially solid tumor research, so do not get together

02.
Difficulties and pain points of CAR-T

02.

"The future of CAR-T is allogeneic (i.
e.
universal), not autologous
.

" Almost all CAR-T scientists have reached such a consensus

Experts from the Institute of Hematology believe that CAR-T faces two problems, one is the relatively long preparation cycle, and the other is the relatively high preparation cost in terms of the preparation process and preparation process, which makes a universal CAR-T very necessary
.

In clinical studies initiated by researchers, some preliminary results have been seen

The aforementioned entrepreneurs who do CAR-T also believe that universal CAR-T must be an inevitable direction for the development of cell therapy
.
Looking at it now, using gene editing to knock out a few genes, reduce the graft-versus-host response, and reduce the ability to resist heterogeneous treatment, these can all be achieved, but in the end, it will achieve the same effect as autologous CAR-T.
In fact, there are still many A long way to go
.

On the other hand, the current domestic CAR-T is only doing better on hematological tumors, but there is almost a blank on solid tumors.
It is not that CAR-T is ineffective on solid tumors.
It may be due to inappropriate target selection and tumor Insufficient knowledge of the immune microenvironment, or T cells do not play a role in solid tumors
.
It can be combined with other treatments, such as the introduction of antibody sequences in CAR-T cells or some sequences that allow CAR-T to be released in blood vessels, or even T cell modification, so that one end recognizes tumor cells and the other end recognizes NK cells.
Activate
.

The reason why the development of solid tumors is more embarrassing is that there are more classifications of solid tumors, and the treatment needs of each tumor type are different, and each one needs to be explored and confirmed to prove its efficacy
.
In the future, we have to focus on certain molecular types of certain tumors and tumors of a certain period
.
Second, in the treatment of solid tumors, the problem of side effects is his most critical point.
Compared with the risk of hematoma itself, the risk of the entire treatment has always existed.
However, the treatment process of solid tumors is relatively gentle, so the side effects The response is more prominent
.
In this regard, CAR-T still has a long way to go
.

There is also a hematoma that has been successfully treated now, all of which are for B cells.
CAR-T cells can help proliferate after entering, so the amount of input cells is not large
.
However, for solid tumors, the amount of cells required for treatment should be much larger than the total amount of hematoma, because it does not help proliferate, all rely on input, and there is also the problem of tumor microenvironment suppression
.
Therefore, if the body's immune system can be mobilized through modification or transformation, it will finally be able to make a breakthrough in solid tumors.
This is the direction of future research and development
.

03.
The bottleneck of clinical transformation

03.

What is the current status of CAR-T transformation?

Experts from the Institute of Hematology and Oncology said that thanks to the benign interaction between clinical needs and clinical research institutions, as well as the development of high-end corporate talents, pipeline research in the laboratory is relatively easier than landing in the clinic, because the clinical export is narrowed.
Each R&D product has the opportunity to be quickly verified clinically, to explore its effectiveness or simply look at its safety
.

"Too many products and clinical trial institutions and subjects are still a little imbalanced.
This requires us to develop and explore new clinical research resources in order to move from a technological power to a technological power
.
When drug research is becoming a powerful country, clinical Institutions play a considerable role, including our clinical research capabilities and numbers, which cannot cover development needs at this stage.
Capability cultivation in this area is an important challenge facing the country and an important link in helping the development of innovative drugs and innovative technologies
.
"

The status of hematoma transformation is like this, and the situation of solid tumors is even more embarrassing
.

Basic research in China is not lacking in imagination, and clinical patient needs are also great.
The biggest bottleneck is how to successfully transfer to patients, which is very difficult in practice
.
Experts from Tianjin Cancer Hospital said that there are several reasons for the problem.
One is government supervision and the other is the lack of a high-quality clinical trial platform
.
The clinical trial platform is growing very fast now, including the construction of secondary hospitals, but there is an obvious contradiction between quality and quantity.
How to overcome this contradiction is a very important topic before us
.
Take Tianjin Cancer Hospital as an example.
It is one of the five largest cancer hospitals in China.
The entire clinical research evaluation is also gradually growing.
However, we still feel that there are big defects in the transformation of cell therapy, especially CAR-T.
Other platforms also have more or less shortcomings, and this is the real bottleneck
.

In terms of supervision, cell therapy implements a dual-track system, which has many advantages but also has shortcomings
.
For example, if you want to apply for an IND in a U.
S.
hospital, you can apply for an IND on your own, and companies can assist the hospital, without the need for a research institute
.
In China, only pharmaceutical companies can apply for IND, and hospital researchers need to set up companies if they want to go to the clinic
.
The aforementioned entrepreneur said, “The clinical research in hospitals cannot be done, and it is quite difficult to catch up with foreign countries
.
”