(Blood Cancer) ASCO — Zalmoxis and Haploidentical Stem Cell Transplantation
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- June 27, 2016
- Reading time: 1 minute
Zalmoxis, developed by MolMed in Milan, is an ex vivo gene therapy. For patients with high-risk hematological malignancies undergoing haploidentical hematopoietic stem cell transplantation, HSV-TK and nerve growth factor genes are introduced into T cells extracted from donor blood. This approach overcomes limitations of conventional cell and gene therapies that previously lacked reliable mechanisms of action.
Haploidentical transplant donors are primarily close relatives, which confers the advantage of potent anti-tumor activity against malignant cells in the patient. However, there is a risk of graft-versus-host disease (GvHD) attacking healthy cells. With Zalmoxis, concurrent use of ganciclovir (an antiviral drug) allows selective elimination of the engineered T cells when needed.
In a clinical trial, 30 volunteers were enrolled and 23 achieved successful immune reconstitution. Of these, 10 developed GvHD; however, following ganciclovir treatment, 9 patients were spared from death and severe complications. Compared to another clinical trial, the one-year survival rate was 49% — an increase of 37 percentage points over the benchmark figure of 140 cases of haploidentical hematopoietic stem cell transplantation.
MolMed was established 20 years ago through the merger of cell therapy divisions from Boehringer Ingelheim and Science Park Raf. In May of the same year, Strimvelis — a home-based gene therapy for ADA-SCID patients developed jointly with MolMed — received EU approval.
References:
- Zalmoxis
- New cell-based therapy to support stem cell transplantation in patients with high-risk blood cancer
- MolMed receives positive CHMP opinion recommending conditional marketing authorisation for Zalmoxis®, the first immunogene therapy to treat high-risk haematological malignancies in patients receiving haplo-identical haematopoietic stem cell transplantation
#Leukemia #HematologicalMalignancies
- Clinical trial drugs
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