Federal Funding Needed for Clinical Trials

Therapies are Needed for Severely Ill Patients with COVID-19

More than 11 million Americans have tested positive for COVID-19 and nearly 250,000 Americans have died. In its most severe form, COVID-19 leads to acute respiratory distress syndrome (ARDS)—a life-threatening lung injury that allows fluid to leak into the lungs and makes it difficult for patients to breathe.

According to multiple recent studies cited by the Centers for Disease Control and Prevention (CDC):

  • 14% of patients in the U.S. were hospitalized, and among all hospitalized patients, 26 to 32% were admitted to the intensive care unit (ICU). 
  • Anywhere from 20 to 42% of hospitalized patients and 67 to 85% of patients admitted to the ICU develop ARDS.
  • 39 to 72% of patients admitted to the ICU for COVID-19, die.
  • Elderly patients and minorities are at greatest risk of serious complications and death.

While significant progress is being made on vaccines and therapies are beginning to emerge, to date no approved therapy has shown significant benefit for severe cases.  There is an urgent need for new modalities–that are broad-acting and safe—to help severely ill patients with COVID-19. 

Regenerative Medicine and Cell Therapies Show Promising Results

Early evidence shows that regenerative medicine and cell therapies (RMCTs)—and in particular, mesenchymal stromal cells (MSCs)—can play a key role in helping severely ill patients with COVID-19, given their immunomodulatory and anti-inflammatory characteristics.

  • A recent systematic review and meta-analysis led by a team at the Mayo Clinic showed a positive trend toward outcomes (pulmonary function and reduced lung inflammation) and reduced mortality for patients with ARDS.
  • More than 90 published review articles highlight the potential of MSCs in treating patients with COVID-19.
  • More than 90 clinical trials are now underway exploring the potential of RMCTs for patients with COVID-19, 70 of which are focused specifically on the use of MSCs. 
  • Of the 70 clinical trials exploring use of MSCs for COVID-19, 23 are being conducted in the United States.
  • Early published results from 12 clinical studies exploring use of MSCs for COVID-19 show safety and efficacy.

Federal Investment is Lacking

Early clinical studies are promising, but larger-scale, multi-center, randomized, controlled trials are needed to confirm early results and help bring safe and effective treatments to patients in need. The key barrier to conducting these large-scale, randomized controlled trials is lack of federal funding.

  • Of the 23 clinical trials exploring use of MSCs for COVID-19 patients in the United States, only one Phase 1 trial—directed towards cancer patients—is being funded by the National Institutes of Health (NIH) through the National Cancer Institute.
  • Of the more than $10 billion in supplemental funding Congress appropriated for vaccines and therapies, no funds have been directed towards promising regenerative medicine and cell therapy treatments for patients with COVID-19.
  • Less than one-half of one percent of total NIH funding has been used to support clinical trials for regenerative medicine and cell therapies.
  • And while the 21st Century Cures Act provided $30 million in funding for regenerative medicine research, those funds expired on September 30, 2020. And, none of the funds were directed toward clinical trials using MSCs.

Patients Can’t Wait – Congress Should Act

  • As COVID-19 is surging across the country, there are no approved therapies for seriously ill patients in the ICU. While a vaccine is coming, it will take months for Americans to get vaccinated, and in the meantime, millions of Americans are getting sick and thousands are dying.
  • Regenerative medicine and cell therapies—and in particular, MSCs—show promise, but large-scale, randomized, placebo-controlled trials cannot move forward without federal partnership and support.
  • Congress should assure that a portion of federal funds directed through a COVID Supplemental or FY 2021 Appropriations toward the Department of Health and Human Services—including BARDA and the NIH—should support clinical trials for regenerative medicine and cell therapies, including MSCs—for COVID-19 and other serious, life-threatening conditions.