President's Letter (2010 Conference)

Dear Scientists, Physicians and Patients,

Preparing for our annual conference always forces me to reflect on how far we have come and how far we have to go. Formed in September 2005, the Jain Foundation had its inception in discussions between Ajit and me following his son Akshay’s diagnosis of dysferlinopathy. We had already funded a few isolated projects, but felt a more comprehensive approach was needed to find a therapy for dysferlinopathy as quickly and efficiently as possible. In order to do this, we needed to develop a novel paradigm for foundation-funded research.

In the typical foundation model, much of the organization’s time and resources must go toward fundraising — an ongoing and labor-intensive process. Only after funds are raised, a research program is formulated and grants awarded. Typically, this is done by an external advisory committee consisting of specialists in the field, chosen from among the limited number of scientists with the necessary expertise. This approach calls on researchers to do double duty and, as is typical in scientific grant giving, can lead to a focus on individual grants and research topics rather than a holistic and coordinated strategy towards finding a cure. Another challenge foundations face is systemic and institutional barriers to sharing results and tools between researchers across their field. The sharing of information, laboratory tools and results is essential to finding a therapy as quickly as possible.

Because of the inefficiency and hurdles faced by the typical foundation model, I have worked to build a new foundation model that will hasten discovery of a cure for dysferlinopathy, and to assemble a team uniquely capable of carrying out that work. Toward that end, the Jain Foundation is staffed with a full-time, in-house scientific team instead of an external advisory committee. This in-house team closely coordinates and communicates with funded researchers—including identifying projects, soliciting proposals, modifying proposals, and monitoring the progress of projects—to ensure that each research project we fund is aligned with the research strategy that we believe is optimal for moving the field forward as a whole (see inside back cover). Because the Jain Foundation is not involved in fundraising all of our time is dedicated to coordinating and facilitating research. Finally, we are strongly committed to removing barriers to sharing data, and actively mediate collaborations among researchers (see page 3).

Our new model is paying off. Just four years after the Jain Foundation’s first annual conference (see page 12), research on dysferlin has dramatically increased (see page 3). In addition, I want to highlight some important achievements:

  • As a direct result of a small group of dedicated researchers, several hundred patients have been identified and diagnosed with dysferlinopathy at the genetic level, no small feat given the rarity of the disease. Their work has influenced the worldwide diagnosis of this disease.
  • Other researchers have made important dysferlin-deficient mouse models available not only to all Jain Foundation projects but, at our request, to the larger global research community.
  • Several outstanding researchers have taken on the unglamorous task of creating and sharing critically needed resources in the dysferlin field.
  • Many researchers have taken our message of collaboration to heart and by working together, they have made their own research programs stronger and increased the pace of progress in the field as a whole. 

Several investigators have shown a remarkable commitment to our approach, working hand in hand with us and adopting unconventional methods to work towards the foundation’s goal. I applaud every one of these researchers for their selfless dedication to this cause and look forward to their continued partnership in the years to come.

Together, my team and these committed researchers bring to our cause more than I could have ever hoped. Even more importantly, I hope we can convince each and every one of you to treat dysferlin research with this same passion and motivation — and thereby help us reach our goal of making a real difference in patients’ lives.

Sincerely,

Plavi Mittal, PhD
President & CEO, Jain Foundation