HSCI Program Director Claudia Rizzini, PhD, receives hundreds of queries each year asking the same thing: ‘When will your research help me or my loved one?’ At times, she has been told that the work going on in one of HSCI’s laboratories is someone’s last hope.
Rizzini fields these questions with empathy and honesty. “I tell them that most of the work is very much in progress and will not be ready for translation to humans for another 5-10 years,” she said. “I also warn patients and their families to be careful, as stem cell ‘treatments’ advertised elsewhere are not proven to be safe or effective.”
HSCI’s mission is to help patients, but many years and research dollars are required to move potential drugs through the clinical pipeline. The Institute is set up to accelerate the process by fueling collaborations between academic researchers and clinician-scientists, providing the scientific community with shared resources, and partnering with companies and investors to commercialize the science.
Despite HSCI’s relatively short existence in drug discovery years—the average drug takes 12 years to go from lab to patient—these strategies have already helped bring a handful of therapeutics into patient trials and set up many more for rapid translation into the clinic.
California-based biopharmaceutical Fate Therapeutics’ lead drug candidate PROHEMA® was inspired by the discovery in an HSCI lab of a compound, called prostaglandin E2, that expands blood stem cell populations. Dana-Farber Cancer Institute and Massachusetts General Hospital clinicians helped identify the value of the compound for patients undergoing cord blood transplants. Now in Phase II clinical trials, the drug 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.
Another set of clinical trials was sparked by a find made by HSCI scientists that blood stem and progenitor cells increased in number in the presence of added parathyroid hormone (currently marketed as FORTEO®, an FDA-approved treatment for osteoporosis). In one trial, the hormone was used to help lymphoma patients move stem cells into the blood. It was well tolerated and led to successful stem cell harvesting in half of the participants. In a second trial, FORTEO® was used after umbilical cord blood transplant to improve engraftment, but in that case the drug did not have a marked effect.
One upcoming trial that’s drawn attention will test the potential of an FDA-approved drug for epilepsy (POTIGA®) to help patients with ALS, also known as Lou Gehrig’s disease, which results from the degeneration of motor neurons. Using patient neurons grown in a laboratory dish, HSCI scientists screened drugs that could correct the defect causing the neurons to die. POTIGA® was found to be the most effective in the dish, and its results in patients will be watched as proof-of-concept that patient stem-cell screens can be an effective tool for new drug discovery.
Other HSCI labs are working to ensure that future trials will be safe for humans. Therapies now in translation include the use of stem cell transplants to help Parkinson’s disease and corneal stem cell deficiency; stem cell-based therapies for human brain tumors; and tissue engineering strategies for congenital hernia and
other birth defects.