Table 3. Summary of efficacy, safety, and clinical response outcome data collected at Month 3.
Each row represents cell product data collected from a unique PACT approved project or clinical trial having the unique attributes of cell product type, administration of fresh or cryopreserved cells, and route of administration unless as otherwise footnoted below. A form data entry field without a response was included in the ‘unknown’ category for this analysis. The product source is indicated in parentheses.
| Cell Product Type | Indication | Product Administrations (2010-2014) | Efficacy | Safety | Clinical Response | |||
|---|---|---|---|---|---|---|---|---|
| BM-MNCs (auto, BM-derived)a | Cardiovascular | 26 | Unknown | 26 | Unknown | 26 | Unknown | 26 |
| CTLs (allo, PB-derived)b | Other disease indication | 8 | Achieved | 6 | No toxicity | 6 | Complete | 2 |
| Unknown | 1 | Mild toxicity | 1 | Unknown | 1 | |||
| Not Applicable | 1 | Unknown | 1 | Partial | 3 | |||
| -- | -- | -- | -- | None | 2 | |||
| CTLs (allo, BM-derived) | Other disease indication | 1 | Achieved | 1 | No toxicity | 1 | Complete | 1 |
| HSCs (allo, UCB-derived) | Hematology | 29 | Unknown | 29 | Unknown | 29 | Unknown | 29 |
| MSCs (allo, BM-derived) | Hematology | 10 | Unknown | 10 | Unknown | 10 | Unknown | 10 |
| NK cells (auto and allo, leukapheresis) | Other disease indication | 7 | Achieved | 1 | No toxicity | 5 | None | 5 |
| Not Achieved | 5 | Unknown | 2 | Complete | 1 | |||
| Not Applicable | 1 | -- | -- | Unknown | 1 | |||
| NK cells (allo, apheresis) | BMT | 6 | Unknown | 6 | Unknown | 6 | Unknown | 6 |
| Tregs (allo, UCB-derived) | BMT | 3 | Unknown | 3 | Unknown | 3 | Unknown | 3 |
| Total product administrations with outcome data | 90 | |||||||
Source: auto = autologous; allo = allogeneic; BM-derived = bone marrow-derived; PB-derived = peripheral blood-derived; UCB-derived = umbilical cord blood-derived. BM-MNCs = bone marrow mononuclear cells; CTLs = cytotoxic T lymphocytes; DCs = dendritic cells; MSCs = mesenchymal stem/stromal cells; NK = natural killer cells; Tregs = T regulatory cells; UCB-derived HSCs = umbilical cord blood-derived hematopoietic stem cells.
Data collected from 2 single-site trials.
Data collected from 2 single-site trials, 1 multi-site trial, and from third party use under compassionate use INDs.