Blavatnik Therapeutics Challenge Awards (BTCA)

Harvard Medical School has just announced the winners of their inaugural BTCA. All project winners will receive one million dollars over a period of two years. These awards are funded by the Flavatnik Family Foundation. Each winner will be provided support from Harvard’s Translator program which supports drug discovery, development, and licensing. This program will provide both logistic support and scientific advice.

The ultimate aim of these grants is to speed up the development of novel therapies which can support patients, and the speed with which these therapies get to their hands.

This year, the winning projects are focused on the following conditions-

• Asthma
• Type 1 Diabetes
• Venous Thromboembolism (VTE)
• Frontotemporal Dementia (FTD)
• Severe Congenital Neutropenia (SCN)

Each of these projects is ready to move to human clinical trials, the costliest stage of development. Without the  support of this grant, both financially and scientifically, many of these projects would be unable to move forward. A bit of information about each project is provided below.

Asthma

Asthma patients often suffer from control of their condition even with high doses of corticosteroids. 1.8 million individuals go to the ER each year for asthma.

Researchers are developing therapeutics that could treat the underlying cellular mechanisms of this condition as well as other inflammatory diseases.

Type 1 Diabetes

Type 1 Diabetes is caused by the inability of the pancreas to make insulin, which is critical for the regulating of blood glucose.

Researchers are working to develop a gene therapy that would eliminate the need for patients to supplement with insulin, something that can impact their daily quality of life. This approach would provide glycemic control without risking hypoglycemia.

VTE

Cancer patients have a higher risk than the general public for developing VTE. In fact, VTEs have become the 2nd leading reason of death for cancer patients, as the blood clots in the veins can become lethal.

Researchers are working on a therapeutic that could prevent VTEs in cancer patients that provides lower risk of bleeding than the current anticoagulant therapies.  

FTD

FTD is similar in presentation to Alzheimer’s disease, and no treatment currently exists for the condition. Onset of symptoms for most patients is between age 40 and 60.

Researchers are working to treat the underlying cause of the condition by restoring the function of the defective gene in a specific subtype of the disease.

SCN

SCN is a rare genetic disease characterized by low levels of white blood cells. This means patients are extremely prone to infection. Unfortunately, the only curative treatment currently available is a bone marrow transplant, which is a risky venture.

Researchers plan to use gene editing to correct the genetic mutation which causes this condition.

You can read more about these awards here.