Roni Gamzu looks up at Health Ministry meeting.
(photo credit: Judy Siegel-Itzkovich)
Patients suffering from bone-marrow failure could have better quality of life and longer survival rates following approval by the Health Ministry to extend a clinical trial of a new placenta-based cell therapy in Israeli hospitals.
The ministry approved the launch of a phase I trial of Pluristem Therapeutics Inc.’s PLX-R18 cell therapy as treatment for insufficient hematopoietic recovery following hematopoietic cell transplantation (HCT, or bone-marrow transplant). The trials will take place at Hadassah Medical Center in Jerusalem and Rambam Hospital in Haifa.
A similar trial was approved by the United States Food and Drug Administration, and recruitment is ongoing in the US. Up to 30 patients will be recruited in both countries in the two studies.
Previous studies with PLXR18 placenta cells have yielded promising results, according to Pluristem’s co-CEO and chairman Zami Aberman. Five years ago, Prof. Reuven Or, from the Bone Marrow Transplantation and Cancer Immunotherapy Research Center at Hadassah University Medical Center in Jerusalem, tested the new methodology for assisting three patients who underwent failed bone-marrow transplants, injecting a simple shot of placenta-derived cells into the thighs of each patient.
The results showed their bodies starting to create new blood cells, repopulating their blood system and improving their health.
“It worked in all three cases,” Or said. The cell therapy currently falls under the umbrella of compassionate use, the treatment of a seriously ill patient using an unapproved drug when no other treatments are available, he said.
On average, more than 13,000 Americans are diagnosed annually with myelodysplastic syndromes, a group of blood disorders associated with abnormal blood-cell production and bone-marrow failure.
Further, bone-marrow failure can also be a side effect of radiation or chemotherapy treatment for cancer. The average length of survival depends on each case, but the prognosis is often poor, and usually the patients require HCT.
Additionally, according to the Institute for Justice, at least 3,000 people in need of a bone-marrow transplant die each year because they cannot find a matching donor. With PLX-R18, because the cells are grown using Pluristem’s proprietary 3-D micro-environmental technology and are an “off-theshelf” product, the process does not require the matching of tissues between donor and recipient and may support grafting from an unmatched donor.
Rather than using stem cells as a replacement part, PLXR18 cells secrete a range of specific proteins that trigger the regeneration of bone-marrow hematopoietic cells, thereby supporting the recovery of blood-cell production This is not the company’s only placenta-based therapy.
Pluristem’s PLX-R18 cells are in late-stage development as a treatment for acute radiation syndrome in conjunction with the US National Institute of Health, the Defense Department and the FDA. Aberman said this approval could fast-track the cell-product use for other indications related to damaged hematological systems, resulting in improved treatment for millions of people.
“We are very pleased with the Israeli Ministry of Health’s vote of confidence in our innovative therapies,” Aberman said.