Timeframe: 2014 – 2018
Goals: Develop and implement a clinical trial for combined liver and bone marrow transplantation as a treatment for liver-confined FLC
Principal Investigator: Ephraim Fuchs, MD
Study overview: This grant supported the development of a phase II, single center study to assess the feasibility and effectiveness of combining bone marrow transplantation and partial liver transplant from a matched living donor in patients with FLC.
The primary objective of this trial was to characterize recurrence-free survival at 1 year following bone marrow transplantation among recipients of prior partial liver transplantation from the same donor.
Under the proposed protocol, patients would have received a combination of:
- A living related donor partial liver transplantation
- Total body irradiation, and
- Bone marrow transplant from same donor.
The treatment was meant to apply to patients whose cancer remained confined to the liver but was too widespread to be removed by surgery or treated by a liver transplant from a deceased donor. These patients have a poor prognosis, with a median survival of 20 months. This protocol was designed to prolong the survival of these patients or perhaps even cure their disease. The purpose of the combined treatment was to reduce the risk of the cancer coming back after the liver transplant. The study team believed that bone marrow transplantation could reduce the risk of cancer relapse in two ways:
- Patients who have combined bone marrow and solid organ transplants may minimize the use of anti-rejection drugs, which inhibit the immune system from destroying cancer cells.
- The donor’s bone marrow contains cells of their immune system, which can attack any cancer cells that remain after the liver transplant.
Details of the proposed trial approach can be found at: https://clinicaltrials.gov/study/NCT02702960
Results: The clinical study was withdrawn in 2018 before any patient enrollment. Most surgeons were concerned about management of the risks to the living donor as a consequence of such a high risk transplant approach.