Blavatnik Family Foundation Grants

Transforming Medical Care with Foundational Research

The Blavatnik Family Foundation is well-regarded for supporting innovative, breakthrough research to expedite the translation of scientific discovery into treatments and cures. Through its partnership with Harvard Medical School, the foundation has embarked on a series of initiatives to address some of the world’s most widespread health challenges.

Three leading BIDMC physician–scientists have recently been awarded prestigious Blavatnik Foundation grants, totaling more than $2.2 million. This critically important funding will help move their key laboratory discoveries into the clinic.

“Our guiding principle is the belief that science and education hold the key to unlocking the vast potential for improving the human condition,” said Len Blavatnik, founder and chairman of Access Industries, and head of the Blavatnik Family Foundation. “We support leading academic and research institutions across the globe that are committed to treating and curing disease. The Blavatnik Family Foundation is proud to contribute to the advancement of the innovative, transformational work taking place at BIDMC.”

Discovery Points the Way to a New Diabetes Treatment

Christiane Ferran, MD, PhD: Blavatnik Therapeutics Challenge Award Recipient

For more than 25 years, Christiane Ferran, MD, PhD, has been tapping into the human body's built-in genetic defenses to fight disease, developing highly innovative gene-based therapies for a number of critical conditions.

Recently, she and her team made a stunning discovery: when mice with type 1 diabetes were treated with one of these novel therapies, the animals' blood sugar levels normalized. In other words, the mice were no longer diabetic.

"We were extremely pleased, surprised, and encouraged," says Ferran, a physician–scientist in the Division of Vascular Surgery and Nephrology at BIDMC and the Lewis Thomas Professor of Surgery at Harvard Medical School. "A gene-based therapy could be administered intravenously in a single dose that could last for years. This would give patients much greater control over their blood sugar and help protect them from dangerous complications."

Type 1 diabetes leaves patients unable to produce insulin, the hormone that enables blood sugar to be used as energy. Therapeutic insulin, administered through multiple daily injections or a pump attached to the body, was discovered nearly a century ago and remains the only treatment available to these patients today.

"Maintaining glycemic control through insulin therapy can be arduous and exhausting for patients," says Ferran. "Patients with type 1 diabetes have a lot of ups and a lot of downs." When glucose levels rise too high or sink too low, life-threatening complications can develop, which is why patients' life expectancy is diminished by 11 to 13 years compared to the average adult.

Unlike therapeutic insulin, Ferran's insulin-independent gene therapy approach would allow patients with type 1 diabetes to keep blood sugar on an even keel without injections and constant monitoring. This discovery has the potential to fundamentally change—and advance—the way this debilitating disease is treated.

"If our research is borne out, having this type of built-in blood sugar control could not only improve patients' lifespans, but also their quality of life," says Ferran. “My team and I are so grateful for the Blavatnik Family Foundation’s philanthropy and support, which is helping us continue to propel this work forward.”

Preventing a Dangerous Side Effect of Cancer

Elliot Chaikof, MD, PhD: Blavatnik Therapeutics Challenge Award Recipient

Among the myriad challenges faced by patients with cancer is venous thromboembolism (VTE), the development of potentially life-threatening blood clots in the deep veins of the legs. Elliot Chaikof, MD, PhD, has been working to shed light on this dangerous condition in hopes of improving this population’s quality of life.

"For reasons that are not yet understood, patients with all types of cancer—in particular, cancers of the gastrointestinal system such as pancreatic and colorectal cancer—are at high risk of VTE," says Chaikof, chair of the Roberta and Stephen R. Weiner Department of Surgery at BIDMC and the Johnson and Johnson Professor of Surgery at Harvard Medical School.

When these clots break free from the leg and travel to the lungs, the result can be fatal, making VTE the leading cause of death among patients with cancer—after cancer itself.

Compounding the problem are the blood thinning anticoagulants used to treat the condition. "When it comes to VTE, there are two sides to the same coin," says Chaikof. "On the one hand, we want to stop clotting to reduce the risk of life-threatening complications. On the other hand, we want to maintain the normal hemostatic mechanisms that keep us safe.”

These mechanisms are critical in the event of an everyday minor bleeding injury—for example, accidentally cutting oneself shaving or cooking or a minor contusion that could lead to bruising. While all patients taking anticoagulant medication are at risk of bleeding, Chaikof says that patients with cancer face a much higher risk.

As the principal investigator of a Blavatnik Therapeutics Challenge Award, Chaikof is developing new and safer therapies to prevent VTE. His work focuses on a class of drugs called selectin inhibitors, which work by acting on white blood cells to halt abnormal clotting.

To date, his results have been promising.

"We have completed the drug discovery phase of our research and will now be able to move into the drug development phase, including safety testing, toxicology studies, and other pre-clinical efforts," says Chaikof.

"Our team is thankful for the support we’ve received from the Blavatnik Family Foundation, which is tremendously helping our work."

A Test of Nerves

Seward Rutkove, MD: Blavatnik Sensory Disorders Research Award Recipient

Damage to the peripheral nerves of the hands and feet can develop for any number of reasons. Diabetes complications are a common source of these painful neuropathies, but by no means the only cause.

According to Seward Rutkove, MD, infections, genetics, and side effects of medications and cancer treatments can also lead to sensory nerve disorders, which are more likely to affect elderly populations.

As chair of the Department of Neurology at BIDMC and Nancy Lurie Marks Professor of Neurology at Harvard Medical School, Rutkove conducts research focused on assessing and improving muscle and nerve health.

"Most people who receive chemotherapy develop some degree of numbness in their feet that doesn't go away," says Rutkove. "Although their cancer may be cured, these troublesome sensory issues endure."

Sensory neuropathies can cause both hypersensitivity and chronic pain, as well as tingling, numbness, and loss of sensation. As nerve damage worsens, patients often have difficulty walking and carrying out day-to-day activities, and are at greater risk of falls and other complications.

"There are no medications to specifically improve sensory function," says Rutkove, adding that a major issue preventing the development of new therapies is the wide variability in patients' self-reporting of either pain or numbness. "We don't have any individualized measures of nerve activity to guide us."

Together with colleagues at BIDMC and Massachusetts General Hospital, Rutkove is developing a new instrument to assess peripheral nerve function by measuring the nerve's level of electrical sensitivity.

"If a nerve is healthy, it responds appropriately to an electrical stimulus," says Rutkove. "But if the nerve is sick, it may be sluggish, it may be jumpy, or it may not respond at all." Knowing exactly how excitable a nerve is can provide clinicians with a much more accurate and quantifiable assessment of nerve damage.

"Through this generous award from the Blavatnik Family Foundation, we are on our way to a more thorough understanding of these sensory nerve disorders that impact so many lives," says Rutkove. "This will enable us to conduct more efficient clinical trials of therapies designed to reduce pain or boost sensation and eventually to help effectively tailor therapies to individuals impacted by these common conditions."

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