HSCI's mission is to help patients, but many years and research dollars are required to move a potential therapeutic through the clinical pipeline. We accelerate the process by:
- fueling collaborations between academic researchers and clinician-scientists;
- providing the scientific community with shared resources, such as the Trans Lab at the Center for Human Cell Therapy; and
- partnering with companies and investors to commercialize the science, including startup formation.
The following therapies discovered by HSCI faculty members are currently being translated for the clinic or are in patient trials:
In Clinical Trials
PROHEMA® for cord blood transplants
Fate Therapeutics' lead product candidate, PROHEMA®, is inspired by work from the lab of HSCI Executive Committee chair Leonard Zon, MD. His team discovered that a compound, called prostaglandin E2, expands blood stem cell populations. Affiliated Faculty member Corey Cutler, MD, MPH, helped identify the compound's potential for cord blood transplants and led clinical research of prostaglandin E2 at Dana-Farber Cancer Institute and Massachusetts General Hospital. Now in Phase II clinical trials, PROHEMA® has been shown to improve the success of cord blood transplants by enhancing blood stem cell homing to the bone marrow niche as well as increasing blood stem cell proliferation and survival.
Parathyroid hormone/FORTEO® for blood stem cells
A discovery from the HSCI laboratory of David Scadden, MD, demonstrated that blood stem and progenitor cells increased with administration of parathyroid hormone (PTH) to modify the stem cell niche. This led to two multicenter, NIH sponsored clinical trials led by Karen Ballen, MD, at Massachusetts General Hospital studying the use of a human parathyroid hormone (PTH)/(Teriparatide or FORTEO®), an FDA-approved treatment for osteoporosis. In the first study, FORTEO® was used to improve mobilization efficacy for patients with lymphoma who did not mobilize stem cells well. PTH was well tolerated in this setting and led to successful stem cell harvesting in half the patients. In the second study, FORTEO® was used after umbilical cord blood transplant. Engraftment was not improved with this approach although survival was comparable to other published reports.
Gene therapy for blood diseases
There are currently three open clinical trials (and two other trials in late stages of development) where genetic diseases and relapsed B cell leukemia are being treated by blood stem cell transplantation using the patients own stem cells that have been genetically modified to express the normal or malfunctioning gene. The open trials are investigating the safety and efficacy of gene therapy for the following diseases: X-linked SCID, Wiskott-Aldrich syndrome, and X-linked Adrenoleukodystrophy. Several HSCI faculty are Principal Investigators and IND Sponsors of these trials; including Principal Faculty members David Williams, MD, and Luigi Notarangelo, MD, and Affiliated Faculty member Sung-Yun Pai, MD. To learn more about the trials and the eligibility criteria, please visit the Dana-Farber Cancer Center/Boston Children's Hospital Cancer and Blood Disorders Center website.
POTIGA® (retigabine) for ALS
A drug approved in 2010 by the Food and Drug Administration for epilepsy was found to reverse ALS abnormalities of patient motor neurons grown in a laboratory dish.The work, done by HSCI Principal Faculty members Kevin Eggan, PhD, and Clifford Woolf, MD, PhD, is being translated to a Phase I clinical trial to test the safety of retigabine in ALS patients. The trial is being led by Merit Cudkowicz, MD, at Massachusetts General Hospital.
A therapy that has shown potential in disease models are tested for feasibility and safety in animals under FDA guidance:
- HSCI Principal Faculty member Ole Isacson, MD, has shown that stem cell transplants for Parkinson's disease are safe in non-human primates and is now researching whether these safer techniques can have positive behavioral impacts on primate subjects.
- HSCI Affiliated Faculty member Rona Carroll, PhD, is developing the use of mesenchymal stem cell-based therapies for human brain tumors.
- HSCI Affiliated Faculty member Dario Fauza, MD, PhD, is testing the clinical potential of tissue engineering for congenital diaphragmatic hernia and other birth defects.
- HSCI Affiliated Faculty member Ula Jurkanas, MD, is investigating how to use patient corneal stem cells for the treatment of corneal stem cell deficiency.
Click here for more useful links related to stem cell research and clinical trials.