Regenerative medicine

Main article: Cancer treatment

History

2023: Reprogramming stem cells and organs "from a test tube." How regenerative medicine develops in Russia and in the world

In October 2023, Professor from Iran Massud Vosukh held a training event at the Institute of Regenerative Medicine of the Scientific and Technological Park of Biomedicine Sechenov University Ministry of Health of Russia. Among the topics covered are the principles of regenerative medicine; personalized; medicine cellular and; gene therapy the future of medicine, as well as the translational challenges of regenerative medicine.

In particular, the leading international articles of the world's leading scientists were discussed. One was dedicated to iPS cells (induced pluripotent stem cells). They are obtained from human or animal cells by epigenetic reprogramming, they are able to convert into cells of muscles, bones, heart, liver, vessels, nervous system. This allows new organs and tissues to be grown from them.

Professor Massoud Vosuch attends a training event at the Institute of Regenerative Medicine

Also, the participants of the workshop touched upon the development of drugs for cancer, as well as about human mesenchymal stromal cells - MSC. They are obtained from fat, blood, gum mucosa, tooth pulp, bone and other tissues. MSCs are considered one of the most accessible for cell therapy, since their sampling in humans does not violate ethical standards and does not harm the patient. One work proposes a set of standard criteria for determining human MSCs suitable for laboratory and preclinical studies.

In Russia, the main promising areas in the field of regenerative medicine were determined by the scientific community together with the Ministry of Health in accordance with the "Strategy for the Development of Medical Science in the Russian Federation for the Period up to 2025" and coincide with global trends in this area. Thus, Russian scientists created a prototype of a biomaterial based on highly purified matrices of proteins and a balanced complex of regenerative factors secreted by MSC of human adipose tissue to stimulate tissue regeneration. They also developed various forms of biomaterial in the form of injection gel, dressings and dense membranes.^[1]

2022: Kidney cells restored for the first time in the world

A team of researchers from Singapore and Germany has demonstrated for the first time in the world the possibility of regenerative therapy to restore kidney function. The results of the work were released in early December 2022.

The program was attended by specialists from the Duke-NUS School of Medicine, as well as the National Heart Center of Singapore (NHCS). The results of the preclinical study are presented in the journal Nature Communications. Scientists have experimented with interleukin-11 (IL-11), a protein that causes scarring in various organs, including the liver, lungs and heart. In addition, IL-11 is involved in acute and chronic kidney diseases.

First-Time Renal Cell Repair Therapy

The researchers found that the named protein triggers a sequence of molecular processes in response to kidney damage, leading to inflammation, fibrosis (scarring) and loss of organ function. In addition, scientists have found that inhibiting IL-11 with neutralizing antibodies can prevent and even reverse kidney damage.

Specifically, renal tubular cells have been shown to release IL-11 in response to injury, ultimately leading to cell growth arrest and promoting kidney dysfunction. However, turning off this process by administering an antibody contributes to the proliferation of renal tubular cells, stopping fibrosis and relieving inflammation. As a result, the damaged cells are regenerated and renal function is restored.

This discovery could be a real breakthrough in the treatment of chronic kidney disease, which is a serious public health problem around the world, and will bring us one step closer to gaining the benefits promised by regenerative medicine, "said Professor Stuart Cook, one of the program participants.[2]

2021: Investments in Regerative Medicine Reach $22.7 Billion

In 2021, companies and researchers working in the field of regenerative medicine attracted a record investment of $22.7 billion. This is evidenced by data from the Regenerative Medicine Alliance (ARM).

Six new cell, gene and tissue therapies were approved in 2021, according to the report. In addition, CAR-T therapy presented data justifying the transition to a higher step in cancer treatment, and venture capital began to actively invest in this area.

Investments in regerative medicine reached $22.7 billion

Some major advances in this area have yet to be made in 2022, according to ARM. About nine therapies are pending approval, and another seven FDA are under regulatory review. EU Among them, the company's omidubicel cell therapy Gamida Cell and ValRox, BioMarin for the treatment of A hemophilias. In 2023 USA , two drugs for the treatment of sickle cell disease will be considered - gene therapy bb1111 and CRISPR Tx manufactured by bluebird bio and CTX001 manufactured. Vertex Pharmaceuticals

There is much debate about the mechanisms for providing gene therapies that ARM says hinder research, which also affected results in 2021. However, ARM noted that the FDA will not administer a universal dose limit for AAV-based gene therapy.

AAVs were used in 145 (or 46%) clinical trials of gene therapy. Other mechanisms included lentivirus, which participated in 24 clinical trials, or 8%, and adenovirus, 22, or 7% of trials. In 2021, 41 gene editing studies were conducted, a third of which were in phase 1 and the rest in phase 2. About 80% of them use CRISPR technology. These include the drug Intellia for the treatment of transthyretin amyloidosis, which was talked about a lot in the summer of 2021. In February 2021, the company presented additional data from a study that showed a dose-dependent result and began answering a question about longevity related to gene editing.

More than half of all gene editing trials are in cancer. In 2021, CRISPR Tx and ViaCyte launched the first study in the field of gene therapy for a more common disease - type 1 diabetes. As for investments, companies engaged in gene and cell therapies received most of the funds - $10.2 billion and $10.1 billion, respectively. Cell therapy received $2 billion, and tissue engineering biotechnology - $341 million. Most of the money came from venture capital, which brought in $9.8 billion, which is 75% more than in 2020. IPOs helped companies raise $4.8 billion, up 30% from 2020.

Biotechnology has seen a boom in partnerships with large pharmaceutical companies, and these deals helped raise $2.3 billion in advance payments in 2021, up 23% from 2020. ARM noted an advance payment of $900 million that Vertex offered CRISPR Tx to work on the CTX001, and an AbbVie check of $370 million for REGENXBIO for gene therapy for eye diseases.

Cancer is the most active indicator for regenerative medicine, with 1,246 trials in oncology, representing 52% of all research in this field in the world. For a long time, the sector worked on hematological cancers, for which 685 studies were conducted, or 55%. However, in 2021, ARM noted a shift towards solid tumors, the number of which grew to 561, or 45% of studies. The leading indication here was cancers of the gastrointestinal tract - 130 studies, or 23% of all ongoing studies of solid tumors, followed by cancers of the brain and spinal cord - 71 studies, or 13%.^[3]

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