Go-ahead for the optim­iz­a­tion of new immunotherapies

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Go-ahead for the optim­iz­a­tion of new immunotherapies

Fight­ing autoim­mune dis­eases and can­cer with human immune cells more effi­ciently: medi­cine is mov­ing a step closer to this vis­ion with new pro­cesses developed by ISAR Bioscience GmbH. The Bav­arian research company’s con­cepts for the rapid, reli­able and at the same time eco­nom­ical pro­duc­tion of thera­peutic immune cells have now been pos­it­ively eval­u­ated by the Ger­man reg­u­lat­ory author­ity. The Paul Ehr­lich Insti­tute (PEI) con­siders the pro­cesses to be prom­ising – and thus gives the go-ahead for the fur­ther devel­op­ment at ISAR Bioscience.

So-called C(A)ART cells are the key ele­ments of the new ther­apies. The abbre­vi­ation stands for Chi­meric (Auto-) Anti­gen Receptor Cells and thus for a prom­ising thera­peutic approach that is inten­ded to strengthen the fight­ing abil­ity of the body’s own immune cells (T cells) against ser­i­ous dis­eases such as can­cer, but also in autoim­mune diseases.

The pre­par­a­tion of the highly per­son­al­ized ther­apy is com­plex and takes sev­eral weeks. First, T cells are obtained from a patient’s white blood cells. In the case of can­cer, these so-called auto­log­ous cells are then genet­ic­ally mod­i­fied in the labor­at­ory so that they can recog­nize and des­troy tumor cells in the body. Finally, the patients receive “their” spe­cific­ally optim­ized cells back by infu­sion. In Ger­many, this very expens­ive ther­apy has already been approved for cer­tain leuk­emias and lymphomas.

A new tech­no­logy plat­form at ISAR Bioscience prom­ises a much faster and more cost-effect­ive pro­ced­ure. In this pro­cess, stem cells are mod­i­fied by gen­ome edit­ing in order to reduce a pos­sible patho­lo­gical autoim­mune reac­tion against the body’s own tis­sue. Sub­sequently, the mod­i­fied stem cells are used to per­man­ently gen­er­ate thera­peutic “battle-ready” chi­meric T cells. The new plat­form allows for the mass pro­duc­tion of stand­ard­ized batches of cells that can be used over and over again in the long term. This, it is expec­ted, will sig­ni­fic­antly reduce treat­ment costs com­pared to cur­rent clin­ic­ally approved auto­log­ous CART cell therapies.

A second innov­a­tion is expec­ted to allow T cells to be used in autoim­mune dis­eases. CAART cells are expec­ted to make this pos­sible. The second A in this abbre­vi­ation stands for auto-anti­gen: These are the body’s pro­teins that are attacked by its own immune sys­tem. CAART cells are inten­ded to sup­press this attack. The first dis­ease the research­ers at ISAR Bioscience want to tackle is hyper­thyroid­ism triggered by anti­bod­ies. How­ever, other autoim­mune dis­eases, such as rheum­atic dis­eases, may also be amen­able to such therapy.

The Bav­arian research com­pany spe­cial­izes in the pro­duc­tion of cus­tom­ized stem cells and aims to bring med­ical research rap­idly to applic­a­tion. The encour­aging sig­nal for the new tech­no­logy plat­form for the use of human pluri­po­tent stem cells (hiPSC) for the gen­er­a­tion of thera­peutic immune cells is the res­ult of a “Sci­entific Advice” meet­ing recently held with the Paul Ehr­lich Institute.

The pos­it­ive eval­u­ation is asso­ci­ated with advice from the PEI on the fur­ther devel­op­ment of the plat­form. This involves, for example, the gen­er­a­tion of a Mas­ter Cell Bank (MCB) in the labor­at­or­ies of ISAR Bioscience at the company’s site in Planegg near Munich. The planned MCB will enable con­tinu­ous cell pro­duc­tion in accord­ance with “Good Man­u­fac­tur­ing Prac­tice” (GMP). Spe­cific­a­tions for the release of the MCB have already been jointly defined by ISAR Bioscience and PEI. Fur­ther pre­clin­ical stud­ies are in pre­par­a­tion. There­after, tox­ic­o­lo­gical tests are planned and, after their com­ple­tion, clin­ical tests in humans.

Back­ground inform­a­tion: https://www.pei.de/DE/home/home-node.html