Hospital Israelita Albert Einstein, in São Paulo, is set to open a new center dedicated to cell and gene therapies and products derived from biological tissue engineering. The aim of the EMBRAPII Competence Center for Advanced Therapies is to function as a platform for basic and applied research, to produce knowledge about diseases that still lack satisfactory treatment options, such as certain types of cancer, to work in partnership with scientists from other institutions and businesses, and to help provide therapies and diagnostic methods that are not currently available.
“We want to create an ecosystem, based on the concept of open innovation, in which users can both extract and add value, where researchers working in networks can generate knowledge relevant to complex problems that would not be achieved without cooperation,” stated Chico Saboya, president of the Brazilian Agency for Industrial Research and Innovation (EMBRAPII), the organization leading the initiative, in an interview with Pesquisa FAPESP’s podcast, Pesquisa Brasil. “It will also be an environment capable of training scientists, hosting research groups, and creating startups.” The center plans to establish partnerships with similar institutions abroad, which will include researcher exchanges.
According to the Brazilian Health Regulatory Agency (ANVISA), advanced therapy is defined as the creation of therapeutic biological products from human cells and tissues that are artificially manufactured or reformulated or based on recombinant DNA with the purpose of repairing, replacing, adding, or removing a genetic sequence, or modifying the expression of a gene. Einstein Hospital has been researching advanced therapies for eight years—including some studies in partnership with Brazil’s Ministry of Health, under the scope of the Support Program for Institutional Development of the Unified Health System (Proadi-SUS).
Olga Maslova / Wikimedia Commons | NIAID / NIH
Aggregate of mesenchymal stem cells (above) and natural killer (NK) cells under an electron microscope: research subjectsOlga Maslova / Wikimedia Commons | NIAID / NIHThe center will seek innovation with an emphasis on the treatment of cancer and rare monogenic diseases—lines of research in which Einstein Hospital has already invested. Last year, the institution received ANVISA approval to begin using CAR T-cells (T lymphocytes with chimeric antigen receptors) produced in its own laboratory to treat humans with lymphoma, leukemia, and multiple myeloma. “I always tell my students that the first advanced cellular therapy was blood transfusion and the second was bone marrow transplantation. Much of the current focus in advanced therapies is on malignant diseases in general, as well as blood diseases and chronic diseases,” says hematologist José Mauro Kutner, medical manager of Einstein Hospital’s Department of Advanced Therapies and general manager of the new center.
In CAR T-cell therapy, the patient’s own immune system is activated to fight against cancer. Immune cells known as T lymphocytes, which typically fight against infections, are extracted from the patient’s own blood and genetically modified in a lab so that when injected back into the patient, they recognize the cancer as foreign. They then reproduce to create an army to combat the tumor (see Pesquisa FAPESP issue nº 286). Einstein Hospital is also researching natural killer (NK) cells—white blood cells that destroy infected and diseased cells, such as cancerous cells—to treat acute myeloid leukemia (AML); gene therapy for sickle cell anemia, a hereditary disease that alters red blood cells, making them shaped like a sickle; and the use of mesenchymal stem cells to treat congenital malformations and diabetes.
Another study being carried out by the organization’s researchers is tissue engineering for chronic knee injuries. The objective is to give the patient tissue with an increased number of cells, which improves the chances of recovery. This type of research is carried out in the cleanroom—a laboratory with ultrafiltered air that the hospital opened five years ago. Cells can thus be produced without the risk of contamination. Saboya, from EMBRAPII, says the long-term objective is also to develop new therapies based on gene editing in the country. “Brazil needs to establish competences in cutting-edge techniques like this and then make them available to the population.”
Einstein Hospital’s choice was made via a call for proposal issued last year by EMBRAPII, a public organization created 10 years ago, funded by the federal government—especially by the ministries of Science, Technology, and Innovation; of Education; of Health; and of Development, Industry, Commerce, and Services—to invest in private innovation projects, in addition to connecting companies with scientists from its 96 accredited research units. The institution has already invested R$3.2 billion in more than 2,500 studies carried out by its units, located in universities and public and private research institutions around the country.
Einstein Hospital
Scientists manipulate reagents and cells in the cleanroom at the Einstein Cell Therapy LaboratoryEinstein HospitalUnder the contractual rules, EMBRAPII did not reveal how many advanced therapy research proposals it received and stated that a series of factors were considered in choosing Einstein Hospital, such as the work that was already being done by the organization and the infrastructure available. The origin of the funds, in this case, was the Ministry of Health’s Department of Science, Technology, Innovation, and Strategic Inputs, which saw the chance to link the competence center with an initiative launched in 2020 that is still in its incipient stages: Genomas Brazil, or the National Program for Genomics and Precision Health, is aiming to create a national database containing the complete genomes of 100,000 Brazilians. Genes from patients with rare, infectious, cardiac, and cancerous diseases will be sequenced. The diseases to be included were selected based on their prevalence and cost to the national health system (SUS).
One element of Einstein’s proposal that stood out was the idea that the R$15 million invested in the center by EMBRAPII could be boosted by additional contributions from private partners and the use of the organization’s existing infrastructure. The advanced therapies center is expected to attract investments from companies in the pharmaceutical and biotechnology sector interested in its scientific expertise and the provision of specialized services that could increase revenue. The funding from the Ministry of Health must be invested for specific purposes, such as expanding physical infrastructure, training personnel, seeking partners, and founding startups.
“Investment in advanced technologies is always welcome, but R$15 million is not enough to obtain robust results in this type of research, in which costs are very high,” said Elize Massard da Fonseca, a professor of public administration at Fundação Getulio Vargas (FGV) who researches innovation and health. She mentions a study published in the scientific journal Pharmaceutical Medicine last June, according to which the development of a new cell and gene therapy requires an investment of US$1.9 billion—the figure was calculated based on 11 therapies approved or under approval by the US Food and Drug Administration (FDA).
The amount being invested in the advanced therapies center is lower than the funding provided for other competence centers recently created by EMBRAPII in technology fields, such as 5G and 6G connectivity, immersive technologies applied to virtual worlds, and intelligent hardware platforms. A total of R$480 million was allocated to eight initiatives, at an average of R$60 million for each.
Although the primary objective is to create innovative treatments, the Einstein center has the potential to stimulate the creation of high-skilled jobs and technology-based startups. “We developed an approach that facilitates the transfer of knowledge obtained in research to applications in industry, reducing costs and risks,” says Edelvicio Souza Júnior, director of innovation and digital transformation at EMBRAPII.
Einstein Hospital launched a new science communication initiative in July. The Science Arena website (sciencearena.org) was created to share information about science conducted in Brazil and worldwide. More than 60 texts have already been published, including reports written by science journalists, essays, columns, and interviews, with the aim of giving context to the latest news and creating a space for open dialogue. “The essays and columns are written by researchers who collaborate by sharing original works with Science Arena. Nothing we publish has been published before in other media,” says journalist Bruno de Pierro, a member of Einstein’s institutional communication team and editor in chief of Science Arena. He emphasizes that the platform is not just for Einstein Hospital’s research: it also covers studies carried out by other research institutions in Brazil and abroad, with all content published in Portuguese and English.
