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Promising New Cell Therapies to Prevent Graft vs. Host Disease

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At present, the global Graft versus Host Disease (GvHD) treatment market is expanding due to a rise in the prevalence and incidence of cancer patients, and is driven by those patients who require bone marrow transplant surgeries (source).


Bone marrow transplants offer patients with leukemia and lymphoma a potentially curative treatment, as the donor bone marrow which contains healthy immune cells targets the recipient's cancer cells and prevents tumor relapse. However, those very same donor immune cells can also recognize the recipient's other healthy tissues as foreign and begin to attack them, a life-threatening phenomenon known as Graft vs Host Disease.

To date, the standard current treatment is a fine balance of immunosuppression in order to achieve tumor relapse prevention while at the same time maintain healthy recipient tissues as they are. However, this leaves patients at high risk for infection as well as tumor recurrence. Additionally, even with the standard immunosuppression, 30-50% of patients will still experience some form of GvHD (source), leading the field of oncology desperately requiring additional treatment options to aid in these patients recovery.


GvHD is leading the number of clinical trials within autoimmune conditions. Clinical trials investigating GvHD are still mainly in early phases as therapeutic treatments are developed and investigated.


Source: Signals Pharma Playbook, Sept 2014 – Aug 2019


As the use of cell and gene therapy as a treatment option is changing the clinical landscape, those aiming to develop novel GvHD therapies are taking advantage of the promising tool and investigating how it can make waves in the GvHD sector as well. In fact, using Pharma Playbook, we can see that the leading modality being investigated in clinical trials for GvHD are cell therapy options.


Most clinical trials are using cell therapy as an investigative modality to treat GvHD in oncology patients.


Source: Signals Pharma Playbook, Sept 2014 - Aug 2019


Digging deeper into the data, we can identify which exciting cell therapies are coming out of this ecosystem. Mesoblast, an Australian-based company who focuses on regenerative medicine, is developing remestemcel-L, cultured adult human mesenchymal stem cells, for the treatment of GvHD. Remestemcel-L regulates the T-cell mediated inflammatory response by inhibiting T-cell proliferation and the production of TNF-alpha and IFN-gamma.

  • They are currently in Phase 3 clinical trials showing positive results with overall Day 180 survival rates reaching 69% compared to the standard steroid therapy which shows overall Day 180 survival rates of 10-30%.

  • Mesoblast is now working towards a pre-BLA meeting with the FDA in the next few months.


Cynata Therapeutics uses their unique Cymerus™ technology, based on the versatile mesenchymoangioblasts, to overcome the challenges of manufacturing mesenchymal stem cells in order to commercialize their stem cell therapy options (source).

  • GvHD is Cynata’s first target indication and a Phase I clinical trial was recently completed using the lead Cymerus™ product candidate, CYP-001, developed from allogeneic induced pluripotent stem cells (iPSC’s). The clinical trial ended with favorable safety and efficacy results with overall survival by Day 100 at 87% (source).

  • A Phase 2 clinical trial is expected to be conducted in late 2019.


Kiadis Pharma is developing their lead product, ATIR101, specifically designed to deplete the T-cells which lead to GvHD and retain the T-cells which are protective against tumor cells and infections. The company uses their proprietary selective cytotoxic compound, TH9402, in order to do so, and enables to provision of a lymphocyte infusion from a healthy, haploidentical stem cell donor with minimal risk of causing GvHD. 

  • Phase II data demonstrated substantial and clinically relevant improvements compared to standard therapy and Kiadis Pharma is conducting a Phase III trial with ATIR101 across Europe and North America.

  • ATIR101 has been granted the Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA as well as Orphan Drug Designations both in the U.S. and Europe.

The use of novel cell therapy options to treat GvHD appears to hold much potential as survival rates soar beyond the current standard immunosuppressive therapy. As the cell therapy field develops more, we should expect to see even more companies utilizing this modality and creating promising therapeutics for the ever-expanding patient population who needs them.


Insights powered by Signals Analytics’ Pharma Playbook




Written by Shir Miodownik

Research Analyst at Signals Analytics

Shir completed her Masters in Genetics and Molecular Biology at Bar Ilan University, and is currently a medical student at Ben Gurion University.


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