Could new stem cell technology eliminate need for donors’ cells?

“Look at that! Life!” said Deidra Flowers-Williams, a sickle cell disease patient, as she held up a tube with stem cells donated by her sister, Tanisha Flowers. It was just minutes before doctors at the NIH Clinical Center would use the cells – extracted from her sister’s bone marrow – for a transplant they hoped would cure the devastating blood disease the 40-year-old had endured most of her life.

So, Flowers-Williams’ elation was understandable — not simply because of the promise, she said, of “a new chapter in my life, free of the chronic pain, the narcotics, the constant ER visits,” but also because she knew how lucky she was to get a donor whose cells were a 100% match for her own.

More often than not, she knew, patients with life-threatening blood and immune diseases never get a donor at all, much less a perfect one. This scarcity turns out to be a major hurdle not only for the patients, but for researchers who need stem cells to test potential cures or treatments.

Now Garuda Therapeutics, a company creating off-the-shelf, durable blood stem cell therapies, wants to make the anxiety of finding a donor a thing of the past. Aided by research funded by NHLBI, the company says it is creating “the world’s first off-the-shelf blood stem cell platform,” which will produce self-renewing blood stem cells on demand. Their goal: to give patients rapid access to durable, properly matched cells for use in life-saving therapies they otherwise might not get.

Just launched in September with an initial investment of $72 million, the platform could lead to the treatment or cure of more than 70 diseases and increase access to a host of existing stem cell therapies, the company said.

“Eliminating the need for donor or patient cells while gaining the ability to exploit stem cell engineering, would democratize blood stem cell transplants,” said Dhvanit Shah, Ph.D., co-founder, president, and CEO of Garuda Therapeutics. And, he added, it would ensure that “this vital, and often curative, therapy can be made accessible to any bone marrow transplant eligible patient in need.”

Blood stem cells, or hematopoietic stem cells, can develop into all types of blood cells, including white, red, and platelets. Currently, healthcare providers try to find immune-compatible donors or use the patients’ own cells to have a chance at successful stem cell procedures. Yet the quality of the donor’s materials often turns out to be poor – the stem cells might have been damaged during storage, or they be weak or too old. Sometimes, there’s just not enough of them.

The company is banking on none of these being an issue if the platform works as hoped. And that means people with scores of diseases, including sickle cell disease, beta thalassemia, bone marrow failure, and blood cancers, could have a shot at a new life. 

“It is hard to overstate the importance of making stem cell therapy more accessible to millions of people around the world suffering from treatable and even curable diseases,” said W. Keith Hoots, M.D., director of the NHLBI’s Division of Blood Diseases and Resources (DBDR). “That’s why it is so exciting to see NHLBI-funded science leading to technologies that could really address a host of diseases in our research portfolio.”

Some of these are also rare diseases that affect minority populations and patients with mixed ancestry or ethnicity, for whom finding a matching donor has proven even more difficult. “NHLBI has a vested interest in expanding access to equitable treatments for these groups which are so often underserved,” Hoots said.

Although other companies are trying to produce off-the-shelf therapies, what distinguishes Garuda’s platform, according to Shah, is that its developers have taken the need for donor cells, and the unpredictability associated with it, out of the equation.

Shah, who received an NHLBI’s early-stage investigator grant at Brigham and Women’s Hospital, is “very grateful” for the NHLBI’s past support and looks forward to future opportunities for collaboration “to improve the lives of patients with life-threatening blood diseases.” He also hopes Garuda’s ability to translate and industrialize this technology “will attract more funding in hematopoietic stem cell research at NHLBI in the future.”

For Asif Rizwan, Ph.D., DBDR program officer, Garuda’s successful launch shows the importance of NHLBI’s grants supporting early-stage investigators. “As a young researcher, the funds are obviously essential,” Rizwan said. “But just as critical are the vetting that your idea receives, the mentoring, the validation.”

This is particularly true when these investigators, still new in their fields, come up with innovative concepts or novel hypothesis, “the type that might turn into a solution to the scarcity of stem cell donors, for instance,” Rizwan concluded.

For Flowers-Williams, not having to agonize over whether she could find a perfect donor would have been great. On September 9, 2015, Flowers-Williams tweeted a picture of herself undergoing a blood transfusion with the caption: “One day this will no longer be my life! #SickleCellAwareness.”

As it happened, she lucked out. Two months later, on November 19, that day came: She received a stem cell transplant and is now sickle cell-free.